Opinion

The introduction of eculizumab, a monoclonal antibody targeting C5 of the complement cascade, revolutionized the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare hematologic disorder characterized by complement-mediated intravascular hemolysis, bone marrow failure, and thrombophilia. Treatment options for PNH were limited before eculizumab was approved by the Food and Drug Administration in 2007.

Its use resulted in the inhibition of intravascular hemolysis, hemoglobin stabilization, and substantial reductions in transfusion requirements. Moreover, eculizumab had the unexpected effect of reducing the risk of thromboembolic complications, the most severe complication of PNH. Patients treated with eculizumab experienced fewer thrombotic events (4%), compared with historical cohorts (27%). Importantly, 5-year overall survival rates for patients with PNH taking eculizumab improved more than 90%, compared wity the 80% reported historically.

More than 10 years later, we are tasked with assessing the impact of this drug. Unquestionably, eculizumab has done more for PNH than we could have hoped for. However, 10 years of additional data reveal the limitations of this groundbreaking therapy. Despite the overall sustained response and survival benefit, hematologic response remains variable. Complete normalization of hemoglobin occurs in less than one-third of patients. Transfusion requirements persist in many patients. Residual anemia during eculizumab therapy is at least partly attributed to bone marrow failure, a feature the complement inhibition does not address. Still, pharmacokinetic limitations of the drug also contribute to the lack of complete responses. There is residual intravascular hemolysis because of insufficient inhibition of C5 and the emergence of C3-mediated extravascular hemolysis constitutes an unanticipated mechanistic complication of all C5-mediated therapies.

The last few years have seen a surge in novel anticomplement agents, which improve upon the already well-established inhibition of C5 but also explore the efficacy of targeting earlier aspects of the complement pathway. During the American Society of Hematology (ASH) annual meeting, we had exciting updates on some of the promising new kids on the block.

Ravulizumab, the newest C5 monoclonal antibody approved by the FDA for PNH, displays more robust C5 inhibition, thereby reducing the breakthrough hemolysis still seen with eculizumab use. Crovalimab, also an anti-C5 humanized antibody, is engineered with Sequential Monoclonal Antibody Recycling Technology that improves the half-life of the drug and facilitates subcutaneous dosing while still achieving complete C5 inhibition. Some of the most exciting data is on danicopan, a small-molecule factor D inhibitor that targets the alternative pathway thereby inhibiting C3 convertase and blocking extravascular hemolysis. It has shown promise as a stand-alone agent, as well as with combined C5 inhibition, while promising safety, a reasonable concern as we explore the long-term risks of targeting the proximal complement pathway.

I was recently asked to comment on how the new complement inhibitors are addressing unmet needs in PNH. While the recent presentations at ASH demonstrate an improvement on the efficacy of C5 inhibition, pharmacokinetics, and drug delivery – all which translate to improved hemoglobin and reduced breakthrough hemolysis for PNH patients – I am most excited at the promise this new generation of drugs holds for other diseases. Since its approval for PNH, eculizumab has also been approved for use in atypical hemolytic uremic syndrome (aHUS), myasthenia gravis, and neuromyelitis optica spectrum disorder.

Perhaps the greatest potential I envision for the new generation of drugs is in aHUS, a chronic disease characterized by hemolytic anemia, thrombocytopenia, and end-stage renal disease that cannot be cured with renal transplantation. The pathophysiology involves dysregulation of complement activation because of genetic mutations or autoantibodies to key proteins in the complement cascade. Though we have experienced some success with eculizumab, responses can be incomplete, particularly in patients with C5 mutations. The newer drugs offer the opportunity to inhibit complement activation at both proximal and distal aspects of the cascade, which may prove ideal in a disease in which the affected protein is not consistent. Moreover, preclinical and clinical trials have shown promise for these novel complement inhibitors in other autoimmune diseases: antibody-mediated vasculitis, C3 glomerulopathy, catastrophic antibody syndrome, membranous nephropathy, and lupus nephritis.

The surge of new complement inhibitors could revolutionize our strategy for treatment of autoimmune-mediated diseases, in which downstream complement activation can manifest with life-threatening tissue injury. Inhibition of complement offers a promising strategy for blocking downstream immune-mediated effector mechanisms of injury common in several autoimmune diseases.

As the results from various clinical trials come to fruition, it will be exciting to determine how to best use this new generation of drugs to target new diseases and whether the next decade is poised to eclipse the progress in complement therapy already established by eculizumab.
 

Dr. Sosa is a benign hematologist at Fox Chase Cancer Center in Philadelphia. Her research interests are in thromboembolic disease, with a focus in racial and gender disparities.

The introduction of eculizumab, a monoclonal antibody targeting C5 of the complement cascade, revolutionized the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare hematologic disorder characterized by complement-mediated intravascular hemolysis, bone marrow failure, and thrombophilia. Treatment options for PNH were limited before eculizumab was approved by the Food and Drug Administration in 2007.

Its use resulted in the inhibition of intravascular hemolysis, hemoglobin stabilization, and substantial reductions in transfusion requirements. Moreover, eculizumab had the unexpected effect of reducing the risk of thromboembolic complications, the most severe complication of PNH. Patients treated with eculizumab experienced fewer thrombotic events (4%), compared with historical cohorts (27%). Importantly, 5-year overall survival rates for patients with PNH taking eculizumab improved more than 90%, compared wity the 80% reported historically.

More than 10 years later, we are tasked with assessing the impact of this drug. Unquestionably, eculizumab has done more for PNH than we could have hoped for. However, 10 years of additional data reveal the limitations of this groundbreaking therapy. Despite the overall sustained response and survival benefit, hematologic response remains variable. Complete normalization of hemoglobin occurs in less than one-third of patients. Transfusion requirements persist in many patients. Residual anemia during eculizumab therapy is at least partly attributed to bone marrow failure, a feature the complement inhibition does not address. Still, pharmacokinetic limitations of the drug also contribute to the lack of complete responses. There is residual intravascular hemolysis because of insufficient inhibition of C5 and the emergence of C3-mediated extravascular hemolysis constitutes an unanticipated mechanistic complication of all C5-mediated therapies.

The last few years have seen a surge in novel anticomplement agents, which improve upon the already well-established inhibition of C5 but also explore the efficacy of targeting earlier aspects of the complement pathway. During the American Society of Hematology (ASH) annual meeting, we had exciting updates on some of the promising new kids on the block.

Ravulizumab, the newest C5 monoclonal antibody approved by the FDA for PNH, displays more robust C5 inhibition, thereby reducing the breakthrough hemolysis still seen with eculizumab use. Crovalimab, also an anti-C5 humanized antibody, is engineered with Sequential Monoclonal Antibody Recycling Technology that improves the half-life of the drug and facilitates subcutaneous dosing while still achieving complete C5 inhibition. Some of the most exciting data is on danicopan, a small-molecule factor D inhibitor that targets the alternative pathway thereby inhibiting C3 convertase and blocking extravascular hemolysis. It has shown promise as a stand-alone agent, as well as with combined C5 inhibition, while promising safety, a reasonable concern as we explore the long-term risks of targeting the proximal complement pathway.

I was recently asked to comment on how the new complement inhibitors are addressing unmet needs in PNH. While the recent presentations at ASH demonstrate an improvement on the efficacy of C5 inhibition, pharmacokinetics, and drug delivery – all which translate to improved hemoglobin and reduced breakthrough hemolysis for PNH patients – I am most excited at the promise this new generation of drugs holds for other diseases. Since its approval for PNH, eculizumab has also been approved for use in atypical hemolytic uremic syndrome (aHUS), myasthenia gravis, and neuromyelitis optica spectrum disorder.

Perhaps the greatest potential I envision for the new generation of drugs is in aHUS, a chronic disease characterized by hemolytic anemia, thrombocytopenia, and end-stage renal disease that cannot be cured with renal transplantation. The pathophysiology involves dysregulation of complement activation because of genetic mutations or autoantibodies to key proteins in the complement cascade. Though we have experienced some success with eculizumab, responses can be incomplete, particularly in patients with C5 mutations. The newer drugs offer the opportunity to inhibit complement activation at both proximal and distal aspects of the cascade, which may prove ideal in a disease in which the affected protein is not consistent. Moreover, preclinical and clinical trials have shown promise for these novel complement inhibitors in other autoimmune diseases: antibody-mediated vasculitis, C3 glomerulopathy, catastrophic antibody syndrome, membranous nephropathy, and lupus nephritis.

The surge of new complement inhibitors could revolutionize our strategy for treatment of autoimmune-mediated diseases, in which downstream complement activation can manifest with life-threatening tissue injury. Inhibition of complement offers a promising strategy for blocking downstream immune-mediated effector mechanisms of injury common in several autoimmune diseases.

As the results from various clinical trials come to fruition, it will be exciting to determine how to best use this new generation of drugs to target new diseases and whether the next decade is poised to eclipse the progress in complement therapy already established by eculizumab.
 

Dr. Sosa is a benign hematologist at Fox Chase Cancer Center in Philadelphia. Her research interests are in thromboembolic disease, with a focus in racial and gender disparities.

The introduction of eculizumab, a monoclonal antibody targeting C5 of the complement cascade, revolutionized the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare hematologic disorder characterized by complement-mediated intravascular hemolysis, bone marrow failure, and thrombophilia. Treatment options for PNH were limited before eculizumab was approved by the Food and Drug Administration in 2007.

Its use resulted in the inhibition of intravascular hemolysis, hemoglobin stabilization, and substantial reductions in transfusion requirements. Moreover, eculizumab had the unexpected effect of reducing the risk of thromboembolic complications, the most severe complication of PNH. Patients treated with eculizumab experienced fewer thrombotic events (4%), compared with historical cohorts (27%). Importantly, 5-year overall survival rates for patients with PNH taking eculizumab improved more than 90%, compared wity the 80% reported historically.

More than 10 years later, we are tasked with assessing the impact of this drug. Unquestionably, eculizumab has done more for PNH than we could have hoped for. However, 10 years of additional data reveal the limitations of this groundbreaking therapy. Despite the overall sustained response and survival benefit, hematologic response remains variable. Complete normalization of hemoglobin occurs in less than one-third of patients. Transfusion requirements persist in many patients. Residual anemia during eculizumab therapy is at least partly attributed to bone marrow failure, a feature the complement inhibition does not address. Still, pharmacokinetic limitations of the drug also contribute to the lack of complete responses. There is residual intravascular hemolysis because of insufficient inhibition of C5 and the emergence of C3-mediated extravascular hemolysis constitutes an unanticipated mechanistic complication of all C5-mediated therapies.

The last few years have seen a surge in novel anticomplement agents, which improve upon the already well-established inhibition of C5 but also explore the efficacy of targeting earlier aspects of the complement pathway. During the American Society of Hematology (ASH) annual meeting, we had exciting updates on some of the promising new kids on the block.

Ravulizumab, the newest C5 monoclonal antibody approved by the FDA for PNH, displays more robust C5 inhibition, thereby reducing the breakthrough hemolysis still seen with eculizumab use. Crovalimab, also an anti-C5 humanized antibody, is engineered with Sequential Monoclonal Antibody Recycling Technology that improves the half-life of the drug and facilitates subcutaneous dosing while still achieving complete C5 inhibition. Some of the most exciting data is on danicopan, a small-molecule factor D inhibitor that targets the alternative pathway thereby inhibiting C3 convertase and blocking extravascular hemolysis. It has shown promise as a stand-alone agent, as well as with combined C5 inhibition, while promising safety, a reasonable concern as we explore the long-term risks of targeting the proximal complement pathway.

I was recently asked to comment on how the new complement inhibitors are addressing unmet needs in PNH. While the recent presentations at ASH demonstrate an improvement on the efficacy of C5 inhibition, pharmacokinetics, and drug delivery – all which translate to improved hemoglobin and reduced breakthrough hemolysis for PNH patients – I am most excited at the promise this new generation of drugs holds for other diseases. Since its approval for PNH, eculizumab has also been approved for use in atypical hemolytic uremic syndrome (aHUS), myasthenia gravis, and neuromyelitis optica spectrum disorder.

Perhaps the greatest potential I envision for the new generation of drugs is in aHUS, a chronic disease characterized by hemolytic anemia, thrombocytopenia, and end-stage renal disease that cannot be cured with renal transplantation. The pathophysiology involves dysregulation of complement activation because of genetic mutations or autoantibodies to key proteins in the complement cascade. Though we have experienced some success with eculizumab, responses can be incomplete, particularly in patients with C5 mutations. The newer drugs offer the opportunity to inhibit complement activation at both proximal and distal aspects of the cascade, which may prove ideal in a disease in which the affected protein is not consistent. Moreover, preclinical and clinical trials have shown promise for these novel complement inhibitors in other autoimmune diseases: antibody-mediated vasculitis, C3 glomerulopathy, catastrophic antibody syndrome, membranous nephropathy, and lupus nephritis.

The surge of new complement inhibitors could revolutionize our strategy for treatment of autoimmune-mediated diseases, in which downstream complement activation can manifest with life-threatening tissue injury. Inhibition of complement offers a promising strategy for blocking downstream immune-mediated effector mechanisms of injury common in several autoimmune diseases.

As the results from various clinical trials come to fruition, it will be exciting to determine how to best use this new generation of drugs to target new diseases and whether the next decade is poised to eclipse the progress in complement therapy already established by eculizumab.
 

Dr. Sosa is a benign hematologist at Fox Chase Cancer Center in Philadelphia. Her research interests are in thromboembolic disease, with a focus in racial and gender disparities.

When Elizabeth Wurtzel wrote “Prozac Nation,” an autobiographical account published in 1994 of her experience with depression and psychiatric medications, she helped shift the public dialogue about mental illness, and in doing so chipped away at the stigma that continues to haunt many of our patients.

Ms. Wurtzel, who died recently at age 52, wrote about depression passionately and matter-of-factly.

As she stated in “Prozac Nation”: “Depression was the loneliest &*%$ thing on earth. There were no halfway houses for depressives, no Depression Anonymous meetings that I knew of. Yes, of course, there were mental hospitals like McLean and Bellevue and Payne Whitney and the Menninger Clinic, but I couldn’t hope to end up in one of those places unless I made a suicide attempt serious enough to warrant oxygen or stitches or a stomach pump.

“I used to wish – to pray to God for the courage and strength – that I’d have the guts not to get better, but to slit my wrists and get a whole lot worse so I could land in some mental ward, where real help might have been possible.”

Think of where the public consciousness was in 1994. Peter D. Kramer, MD, had started the conversation on Prozac a year earlier with his book, “Listening to Prozac,” Kurt Cobain died by suicide in 1994, and 2 years later President Bill Clinton signed the Personal Responsibility and Work Opportunity Reconciliation Act, a law that “reformed” welfare and some say made it more difficult for low-income Americans to secure psychiatric and addiction services (Milbank Q. 2005 Mar;83[1]:65-99).

Prozac, as we know, was the first SSRI on the market in the late 1980s and was hailed as a major medication breakthrough in the treatment of depression. It lacked the side effects of the tricyclic antidepressants of previous years and did not have the potentially dangerous food restrictions associated with monoamine oxidase inhibitors.

Interestingly, the major reviews of Ms. Wurtzel’s book, mainly written by men, were negative. Those reviews focused more on the lifestyle of Ms. Wurtzel, her introspection, and how difficult life was for her, rather than the importance of the book. To me, her writing skills were exceptional, as was her willingness to put her lifestyle and suffering on the line.

The literary critics failed to recognize the book’s importance in unmasking the massive denial of mental illnesses and what Ms. Wurtzel was trying to get across. There have been many successful male writers over the years whose lives were difficult and replete with emotional pain and suffering, and their work was lauded. Regardless of the reviews’ negativity, readers found her book open and enlightening, making it a bestseller – thus paving the way for better and more-open discussion of mental disorders. It also became a touchstone in discussions of antidepressants in the psychiatric literature (Lancet. 1998 Sep 26. doi: 10.1016/S0140-6736(98)08418-9; Lancet. 2015 Oct 1. doi: 10.1016/S2215-0366[15]00430-7; and Biol Psychiatry. 2018 Dec 1;84[11]:e73-5).

However, unfortunately, the stigma still exists on many levels, often starting with the medical profession itself. In my experience over the years in teaching and supervising medical students, many of those not interested in becoming a psychiatrist all too often could not wait for their psych rotation to be over. Generally, they did not take the rotation seriously. I’ve even heard students making light of the delusions and paranoia seen in the suffering of acutely ill patients.

We can take this even further within the profession. I have had many referrals from far too many extremely competent physicians, across many medical specialties, who would refer to their patient as “sort of crazy.” Those physicians want the best for their patients, clearly, in making the referral, but they need to change their thinking and, therefore, their vocabulary about mental disorders. I’d like to see these physicians be more respectful of our patients – just as I would be if I were referring a patient complaining of fatigue and joint pain to a general internist or rheumatologist.

I once knew a brilliant orthopedic surgeon who, when he made a referral, would sit down with the patient and clearly explain why they were not crazy but had an anxiety or a mood problem that he didn’t treat but had a person to refer to who could help. Likewise, I know an ophthalmologist who tells his patients with some emotional symptoms that they are experiencing a difficult situation and would benefit from help that he is not able to provide but could be resolved with another type of specialist who works with their “difficulties.” We clearly need more docs like this who go out of their specialty to explain what patients might need – despite the administrative burden exacted by EMRs on doctors’ time and energy.

As we grow more tolerant in our culture and eliminate distasteful words about people and groups, maybe we should try and avoid the word crazy – even in our general vocabulary. Furthermore, in social situations, while out to dinner with friends, at the gym, or even while in the workplace, just as we may refer to our primary care doc as the best or report we have the best cardiologist or dermatologist, we rarely hear someone being open about the best psychiatrist, psychologist, or therapist in the same manner.

“If ‘Prozac Nation’ has any particular purpose,” she wrote in the afterword, “it would be to come out and say that clinical depression is a real problem, that it ruins lives, that it ends lives, that it very nearly ended my life, that it afflicts many, many people, many very bright and worthy and thoughtful and caring people, people who could probably save the world or at the very least do it some real good.” Those people are our patients, and medicine should take the lead in working further to destigmatize mental illness.

Dr. London is a practicing psychiatrist and has been a newspaper columnist for 35 years, specializing in and writing about short-term therapy, including cognitive-behavioral therapy and guided imagery. He is author of “Find Freedom Fast” (New York: Kettlehole Publishing, 2019). He has no conflicts of interest.

When Elizabeth Wurtzel wrote “Prozac Nation,” an autobiographical account published in 1994 of her experience with depression and psychiatric medications, she helped shift the public dialogue about mental illness, and in doing so chipped away at the stigma that continues to haunt many of our patients.

Ms. Wurtzel, who died recently at age 52, wrote about depression passionately and matter-of-factly.

As she stated in “Prozac Nation”: “Depression was the loneliest &*%$ thing on earth. There were no halfway houses for depressives, no Depression Anonymous meetings that I knew of. Yes, of course, there were mental hospitals like McLean and Bellevue and Payne Whitney and the Menninger Clinic, but I couldn’t hope to end up in one of those places unless I made a suicide attempt serious enough to warrant oxygen or stitches or a stomach pump.

“I used to wish – to pray to God for the courage and strength – that I’d have the guts not to get better, but to slit my wrists and get a whole lot worse so I could land in some mental ward, where real help might have been possible.”

Think of where the public consciousness was in 1994. Peter D. Kramer, MD, had started the conversation on Prozac a year earlier with his book, “Listening to Prozac,” Kurt Cobain died by suicide in 1994, and 2 years later President Bill Clinton signed the Personal Responsibility and Work Opportunity Reconciliation Act, a law that “reformed” welfare and some say made it more difficult for low-income Americans to secure psychiatric and addiction services (Milbank Q. 2005 Mar;83[1]:65-99).

Prozac, as we know, was the first SSRI on the market in the late 1980s and was hailed as a major medication breakthrough in the treatment of depression. It lacked the side effects of the tricyclic antidepressants of previous years and did not have the potentially dangerous food restrictions associated with monoamine oxidase inhibitors.

Interestingly, the major reviews of Ms. Wurtzel’s book, mainly written by men, were negative. Those reviews focused more on the lifestyle of Ms. Wurtzel, her introspection, and how difficult life was for her, rather than the importance of the book. To me, her writing skills were exceptional, as was her willingness to put her lifestyle and suffering on the line.

The literary critics failed to recognize the book’s importance in unmasking the massive denial of mental illnesses and what Ms. Wurtzel was trying to get across. There have been many successful male writers over the years whose lives were difficult and replete with emotional pain and suffering, and their work was lauded. Regardless of the reviews’ negativity, readers found her book open and enlightening, making it a bestseller – thus paving the way for better and more-open discussion of mental disorders. It also became a touchstone in discussions of antidepressants in the psychiatric literature (Lancet. 1998 Sep 26. doi: 10.1016/S0140-6736(98)08418-9; Lancet. 2015 Oct 1. doi: 10.1016/S2215-0366[15]00430-7; and Biol Psychiatry. 2018 Dec 1;84[11]:e73-5).

However, unfortunately, the stigma still exists on many levels, often starting with the medical profession itself. In my experience over the years in teaching and supervising medical students, many of those not interested in becoming a psychiatrist all too often could not wait for their psych rotation to be over. Generally, they did not take the rotation seriously. I’ve even heard students making light of the delusions and paranoia seen in the suffering of acutely ill patients.

We can take this even further within the profession. I have had many referrals from far too many extremely competent physicians, across many medical specialties, who would refer to their patient as “sort of crazy.” Those physicians want the best for their patients, clearly, in making the referral, but they need to change their thinking and, therefore, their vocabulary about mental disorders. I’d like to see these physicians be more respectful of our patients – just as I would be if I were referring a patient complaining of fatigue and joint pain to a general internist or rheumatologist.

I once knew a brilliant orthopedic surgeon who, when he made a referral, would sit down with the patient and clearly explain why they were not crazy but had an anxiety or a mood problem that he didn’t treat but had a person to refer to who could help. Likewise, I know an ophthalmologist who tells his patients with some emotional symptoms that they are experiencing a difficult situation and would benefit from help that he is not able to provide but could be resolved with another type of specialist who works with their “difficulties.” We clearly need more docs like this who go out of their specialty to explain what patients might need – despite the administrative burden exacted by EMRs on doctors’ time and energy.

As we grow more tolerant in our culture and eliminate distasteful words about people and groups, maybe we should try and avoid the word crazy – even in our general vocabulary. Furthermore, in social situations, while out to dinner with friends, at the gym, or even while in the workplace, just as we may refer to our primary care doc as the best or report we have the best cardiologist or dermatologist, we rarely hear someone being open about the best psychiatrist, psychologist, or therapist in the same manner.

“If ‘Prozac Nation’ has any particular purpose,” she wrote in the afterword, “it would be to come out and say that clinical depression is a real problem, that it ruins lives, that it ends lives, that it very nearly ended my life, that it afflicts many, many people, many very bright and worthy and thoughtful and caring people, people who could probably save the world or at the very least do it some real good.” Those people are our patients, and medicine should take the lead in working further to destigmatize mental illness.

Dr. London is a practicing psychiatrist and has been a newspaper columnist for 35 years, specializing in and writing about short-term therapy, including cognitive-behavioral therapy and guided imagery. He is author of “Find Freedom Fast” (New York: Kettlehole Publishing, 2019). He has no conflicts of interest.

When Elizabeth Wurtzel wrote “Prozac Nation,” an autobiographical account published in 1994 of her experience with depression and psychiatric medications, she helped shift the public dialogue about mental illness, and in doing so chipped away at the stigma that continues to haunt many of our patients.

Ms. Wurtzel, who died recently at age 52, wrote about depression passionately and matter-of-factly.

As she stated in “Prozac Nation”: “Depression was the loneliest &*%$ thing on earth. There were no halfway houses for depressives, no Depression Anonymous meetings that I knew of. Yes, of course, there were mental hospitals like McLean and Bellevue and Payne Whitney and the Menninger Clinic, but I couldn’t hope to end up in one of those places unless I made a suicide attempt serious enough to warrant oxygen or stitches or a stomach pump.

“I used to wish – to pray to God for the courage and strength – that I’d have the guts not to get better, but to slit my wrists and get a whole lot worse so I could land in some mental ward, where real help might have been possible.”

Think of where the public consciousness was in 1994. Peter D. Kramer, MD, had started the conversation on Prozac a year earlier with his book, “Listening to Prozac,” Kurt Cobain died by suicide in 1994, and 2 years later President Bill Clinton signed the Personal Responsibility and Work Opportunity Reconciliation Act, a law that “reformed” welfare and some say made it more difficult for low-income Americans to secure psychiatric and addiction services (Milbank Q. 2005 Mar;83[1]:65-99).

Prozac, as we know, was the first SSRI on the market in the late 1980s and was hailed as a major medication breakthrough in the treatment of depression. It lacked the side effects of the tricyclic antidepressants of previous years and did not have the potentially dangerous food restrictions associated with monoamine oxidase inhibitors.

Interestingly, the major reviews of Ms. Wurtzel’s book, mainly written by men, were negative. Those reviews focused more on the lifestyle of Ms. Wurtzel, her introspection, and how difficult life was for her, rather than the importance of the book. To me, her writing skills were exceptional, as was her willingness to put her lifestyle and suffering on the line.

The literary critics failed to recognize the book’s importance in unmasking the massive denial of mental illnesses and what Ms. Wurtzel was trying to get across. There have been many successful male writers over the years whose lives were difficult and replete with emotional pain and suffering, and their work was lauded. Regardless of the reviews’ negativity, readers found her book open and enlightening, making it a bestseller – thus paving the way for better and more-open discussion of mental disorders. It also became a touchstone in discussions of antidepressants in the psychiatric literature (Lancet. 1998 Sep 26. doi: 10.1016/S0140-6736(98)08418-9; Lancet. 2015 Oct 1. doi: 10.1016/S2215-0366[15]00430-7; and Biol Psychiatry. 2018 Dec 1;84[11]:e73-5).

However, unfortunately, the stigma still exists on many levels, often starting with the medical profession itself. In my experience over the years in teaching and supervising medical students, many of those not interested in becoming a psychiatrist all too often could not wait for their psych rotation to be over. Generally, they did not take the rotation seriously. I’ve even heard students making light of the delusions and paranoia seen in the suffering of acutely ill patients.

We can take this even further within the profession. I have had many referrals from far too many extremely competent physicians, across many medical specialties, who would refer to their patient as “sort of crazy.” Those physicians want the best for their patients, clearly, in making the referral, but they need to change their thinking and, therefore, their vocabulary about mental disorders. I’d like to see these physicians be more respectful of our patients – just as I would be if I were referring a patient complaining of fatigue and joint pain to a general internist or rheumatologist.

I once knew a brilliant orthopedic surgeon who, when he made a referral, would sit down with the patient and clearly explain why they were not crazy but had an anxiety or a mood problem that he didn’t treat but had a person to refer to who could help. Likewise, I know an ophthalmologist who tells his patients with some emotional symptoms that they are experiencing a difficult situation and would benefit from help that he is not able to provide but could be resolved with another type of specialist who works with their “difficulties.” We clearly need more docs like this who go out of their specialty to explain what patients might need – despite the administrative burden exacted by EMRs on doctors’ time and energy.

As we grow more tolerant in our culture and eliminate distasteful words about people and groups, maybe we should try and avoid the word crazy – even in our general vocabulary. Furthermore, in social situations, while out to dinner with friends, at the gym, or even while in the workplace, just as we may refer to our primary care doc as the best or report we have the best cardiologist or dermatologist, we rarely hear someone being open about the best psychiatrist, psychologist, or therapist in the same manner.

“If ‘Prozac Nation’ has any particular purpose,” she wrote in the afterword, “it would be to come out and say that clinical depression is a real problem, that it ruins lives, that it ends lives, that it very nearly ended my life, that it afflicts many, many people, many very bright and worthy and thoughtful and caring people, people who could probably save the world or at the very least do it some real good.” Those people are our patients, and medicine should take the lead in working further to destigmatize mental illness.

Dr. London is a practicing psychiatrist and has been a newspaper columnist for 35 years, specializing in and writing about short-term therapy, including cognitive-behavioral therapy and guided imagery. He is author of “Find Freedom Fast” (New York: Kettlehole Publishing, 2019). He has no conflicts of interest.

In this edition of “How I Will Treat My Next Patient,” I highlight the potential role of circulating tumor DNA (ctDNA) monitoring in patients with a history of nonmetastatic colorectal cancer (CRC) and the approval of a new targeted agent for a subset of patients with gastrointestinal stromal tumors (GISTs). Taken together, this information may guide the management of selected patients with gastrointestinal malignancies, now and in the future.

ctDNA in colorectal cancer

Unfortunately, among patients with CRC with standard blood monitoring, multiple, incurable metastases are the predominant finding when relapse occurs. To improve upon the detection of potentially curable recurrent disease, researchers performed a cross-sectional, observational study of routine monitoring of ctDNA in patients with stage I-III CRC. The patients were also monitored with carcinoembryonic antigen (CEA) levels and imaging in accordance with guidelines from the National Comprehensive Cancer Network (Cancer. 2020 Jan 7. doi: 10.1002/cncr.32695). The investigators used the COLVERA assay of methylated CBAT1/IKZF1 for ctDNA monitoring and the LIAISON CEA plasma test.

Among the 50 patients with recurrence and 177 without recurrence of CRC who met all blood and imaging collection criteria, ctDNA testing offered a sensitivity of 68.1%, compared with 31.9% for CEA (P = .0002), with comparable specificity (97.9% vs. 96.4%; P = 1.000). Over about a 4-year time period, ctDNA detected an additional 18 patients (38%) with recurrence who did not have an elevated plasma CEA, whereas there was only 1 case (2.1%) with an elevated plasma CEA and negative ctDNA test.

Among recurrences considered amenable to surgery with curative intent, (n = 20), ctDNA was positive in 60% of patients, compared with only 20% for CEA (P = .010). Multivariate analysis indicated that ctDNA was an independent predictor of recurrence, whereas CEA was not.

The authors concluded that the methylated BCAT1/IKZF1 ctDNA test was superior to CEA monitoring after initial treatment for potentially curable CRC.

How these results influence practice

The current study adds to the body of work showing superior sensitivity of ctDNA monitoring in the detection of recurrence in patients with solid tumors. In May 2019, this column highlighted the work of Yuxuan Wang, MD, PhD, and colleagues in 58 patients with stages I-III CRC; over a similar duration of follow-up as in the current study (JAMA Oncol. 2019;5[8]:1118-23), Dr. Wang found that 10 of 13 recurrences (77%) were detected by monitoring ctDNA levels. CEA levels were detected 63% of recurrences.

The central, critical hypothesis of these efforts is that the earlier detection of metastatic disease will lead to improved survival for patients with CRC. As the authors state in their discussion, that hypothesis remains tantalizing but unproven.

Prospective, randomized trials like the recently opened COBRA trial (NRG-GI005), which tests ctDNA assay–directed therapy in patients with stage IIA CRC, deserve our enthusiastic support. Research with similar designs will establish the value of ctDNA monitoring as a biomarker for early intervention in patients with stage IV disease and for selecting patients after primary therapy who are unlikely to have been cured and who could potentially benefit from adjuvant therapy.

Monitoring ctDNA is a research tool that could be practice changing in the near future. It has shown enough promise to justify prospective, randomized trials to determine whether its superior sensitivity translates into improved survival.
 

Avapritinib for mutated GIST

The platelet-derived growth factor receptor–alpha (PDGFRA) gene encodes PDGFRA, a member of the type III tyrosine kinase receptor family, which includes the stem cell factor receptor, KIT (present in 95% of GISTs). PDGFRA and KIT have domains with specific roles in tyrosine kinase activation. Several PDGF isoforms bind and activate PDGFRA.

Among patients with GISTs, clinical responses to imatinib and other tyrosine kinase inhibitors (TKIs) correlate with tumor genotype. For example, many GISTs that progress within 6 months of TKI initiation lack mutations in KIT or PDGFRA or have a PDGFRA D842 mutation.

The Food and Drug Administration recently approved avapritinib (Ayvakit) for adults with unresectable or metastatic GISTs with a PDGFRA exon 18 mutation. The approval was based on the results of the NAVIGATOR trial, a single-arm, multicenter, open-label study of 43 patients with PDGFRA exon 18 mutations, including 38 patients with a PDGFRA D842V mutation.

The patients received 300 mg avapritinib once daily and 84% responded to avapritinib, though most were partial responses. Among patients with a D842V mutation, the response rate was also high – at 89% – but most responses were partial. At a median follow-up of 10.6 months, median response duration was not reached, but 61% of responses exceeded 6 months. Toxicity included asthenia, gastrointestinal and central nervous system side effects (including intracranial hemorrhage), hair color changes, lacrimation, and dizziness.

Avapritinib is the first drug approved specifically for patients with advanced or unresectable GISTs with a PDGFRA exon 18 mutation.

How these results influence practice

PDGFRA exon 18 mutations occur in 5%-7% of GISTs and are believed to stabilize the kinase activation loop, perhaps accounting for slow, steady growth of these tumors over a long time period and resistance to TKIs. The most frequent mutation results in an exon 18 D842V substitution (75% of all PDGFRA-mutated tumors). Although dramatic progress has been made in the treatment of patients with GIST since 2000, in patients with GISTs harboring PDGFRA exon 18 mutations, responses to treatment are rare and, when they occur, are more abbreviated.

With a high response rate and impressive response duration, avapritinib will be a valuable resource for oncologists treating this uncommon subset of patients with GISTs. The opportunity to convert unresectable tumors to potentially curable ones seems within reach.

The difficult dilemma will be to decide when treatment is needed and drug-related toxicity is justified in patients with slowly progressive tumors and few symptoms. As with so many decisions in oncology, until newer agents with fewer toxicities and higher complete response rates are developed, the proper time for any individual patient to embark on treatment with avapritinib will be found at the intersection of “precision medicine” and “clinical judgment.”

Dr. Lyss has been a community-based medical oncologist and clinical researcher for more than 35 years, practicing in St. Louis. His clinical and research interests are in the prevention, diagnosis, and treatment of breast and lung cancers and in expanding access to clinical trials to medically underserved populations.

In this edition of “How I Will Treat My Next Patient,” I highlight the potential role of circulating tumor DNA (ctDNA) monitoring in patients with a history of nonmetastatic colorectal cancer (CRC) and the approval of a new targeted agent for a subset of patients with gastrointestinal stromal tumors (GISTs). Taken together, this information may guide the management of selected patients with gastrointestinal malignancies, now and in the future.

ctDNA in colorectal cancer

Unfortunately, among patients with CRC with standard blood monitoring, multiple, incurable metastases are the predominant finding when relapse occurs. To improve upon the detection of potentially curable recurrent disease, researchers performed a cross-sectional, observational study of routine monitoring of ctDNA in patients with stage I-III CRC. The patients were also monitored with carcinoembryonic antigen (CEA) levels and imaging in accordance with guidelines from the National Comprehensive Cancer Network (Cancer. 2020 Jan 7. doi: 10.1002/cncr.32695). The investigators used the COLVERA assay of methylated CBAT1/IKZF1 for ctDNA monitoring and the LIAISON CEA plasma test.

Among the 50 patients with recurrence and 177 without recurrence of CRC who met all blood and imaging collection criteria, ctDNA testing offered a sensitivity of 68.1%, compared with 31.9% for CEA (P = .0002), with comparable specificity (97.9% vs. 96.4%; P = 1.000). Over about a 4-year time period, ctDNA detected an additional 18 patients (38%) with recurrence who did not have an elevated plasma CEA, whereas there was only 1 case (2.1%) with an elevated plasma CEA and negative ctDNA test.

Among recurrences considered amenable to surgery with curative intent, (n = 20), ctDNA was positive in 60% of patients, compared with only 20% for CEA (P = .010). Multivariate analysis indicated that ctDNA was an independent predictor of recurrence, whereas CEA was not.

The authors concluded that the methylated BCAT1/IKZF1 ctDNA test was superior to CEA monitoring after initial treatment for potentially curable CRC.

How these results influence practice

The current study adds to the body of work showing superior sensitivity of ctDNA monitoring in the detection of recurrence in patients with solid tumors. In May 2019, this column highlighted the work of Yuxuan Wang, MD, PhD, and colleagues in 58 patients with stages I-III CRC; over a similar duration of follow-up as in the current study (JAMA Oncol. 2019;5[8]:1118-23), Dr. Wang found that 10 of 13 recurrences (77%) were detected by monitoring ctDNA levels. CEA levels were detected 63% of recurrences.

The central, critical hypothesis of these efforts is that the earlier detection of metastatic disease will lead to improved survival for patients with CRC. As the authors state in their discussion, that hypothesis remains tantalizing but unproven.

Prospective, randomized trials like the recently opened COBRA trial (NRG-GI005), which tests ctDNA assay–directed therapy in patients with stage IIA CRC, deserve our enthusiastic support. Research with similar designs will establish the value of ctDNA monitoring as a biomarker for early intervention in patients with stage IV disease and for selecting patients after primary therapy who are unlikely to have been cured and who could potentially benefit from adjuvant therapy.

Monitoring ctDNA is a research tool that could be practice changing in the near future. It has shown enough promise to justify prospective, randomized trials to determine whether its superior sensitivity translates into improved survival.
 

Avapritinib for mutated GIST

The platelet-derived growth factor receptor–alpha (PDGFRA) gene encodes PDGFRA, a member of the type III tyrosine kinase receptor family, which includes the stem cell factor receptor, KIT (present in 95% of GISTs). PDGFRA and KIT have domains with specific roles in tyrosine kinase activation. Several PDGF isoforms bind and activate PDGFRA.

Among patients with GISTs, clinical responses to imatinib and other tyrosine kinase inhibitors (TKIs) correlate with tumor genotype. For example, many GISTs that progress within 6 months of TKI initiation lack mutations in KIT or PDGFRA or have a PDGFRA D842 mutation.

The Food and Drug Administration recently approved avapritinib (Ayvakit) for adults with unresectable or metastatic GISTs with a PDGFRA exon 18 mutation. The approval was based on the results of the NAVIGATOR trial, a single-arm, multicenter, open-label study of 43 patients with PDGFRA exon 18 mutations, including 38 patients with a PDGFRA D842V mutation.

The patients received 300 mg avapritinib once daily and 84% responded to avapritinib, though most were partial responses. Among patients with a D842V mutation, the response rate was also high – at 89% – but most responses were partial. At a median follow-up of 10.6 months, median response duration was not reached, but 61% of responses exceeded 6 months. Toxicity included asthenia, gastrointestinal and central nervous system side effects (including intracranial hemorrhage), hair color changes, lacrimation, and dizziness.

Avapritinib is the first drug approved specifically for patients with advanced or unresectable GISTs with a PDGFRA exon 18 mutation.

How these results influence practice

PDGFRA exon 18 mutations occur in 5%-7% of GISTs and are believed to stabilize the kinase activation loop, perhaps accounting for slow, steady growth of these tumors over a long time period and resistance to TKIs. The most frequent mutation results in an exon 18 D842V substitution (75% of all PDGFRA-mutated tumors). Although dramatic progress has been made in the treatment of patients with GIST since 2000, in patients with GISTs harboring PDGFRA exon 18 mutations, responses to treatment are rare and, when they occur, are more abbreviated.

With a high response rate and impressive response duration, avapritinib will be a valuable resource for oncologists treating this uncommon subset of patients with GISTs. The opportunity to convert unresectable tumors to potentially curable ones seems within reach.

The difficult dilemma will be to decide when treatment is needed and drug-related toxicity is justified in patients with slowly progressive tumors and few symptoms. As with so many decisions in oncology, until newer agents with fewer toxicities and higher complete response rates are developed, the proper time for any individual patient to embark on treatment with avapritinib will be found at the intersection of “precision medicine” and “clinical judgment.”

Dr. Lyss has been a community-based medical oncologist and clinical researcher for more than 35 years, practicing in St. Louis. His clinical and research interests are in the prevention, diagnosis, and treatment of breast and lung cancers and in expanding access to clinical trials to medically underserved populations.

In this edition of “How I Will Treat My Next Patient,” I highlight the potential role of circulating tumor DNA (ctDNA) monitoring in patients with a history of nonmetastatic colorectal cancer (CRC) and the approval of a new targeted agent for a subset of patients with gastrointestinal stromal tumors (GISTs). Taken together, this information may guide the management of selected patients with gastrointestinal malignancies, now and in the future.

ctDNA in colorectal cancer

Unfortunately, among patients with CRC with standard blood monitoring, multiple, incurable metastases are the predominant finding when relapse occurs. To improve upon the detection of potentially curable recurrent disease, researchers performed a cross-sectional, observational study of routine monitoring of ctDNA in patients with stage I-III CRC. The patients were also monitored with carcinoembryonic antigen (CEA) levels and imaging in accordance with guidelines from the National Comprehensive Cancer Network (Cancer. 2020 Jan 7. doi: 10.1002/cncr.32695). The investigators used the COLVERA assay of methylated CBAT1/IKZF1 for ctDNA monitoring and the LIAISON CEA plasma test.

Among the 50 patients with recurrence and 177 without recurrence of CRC who met all blood and imaging collection criteria, ctDNA testing offered a sensitivity of 68.1%, compared with 31.9% for CEA (P = .0002), with comparable specificity (97.9% vs. 96.4%; P = 1.000). Over about a 4-year time period, ctDNA detected an additional 18 patients (38%) with recurrence who did not have an elevated plasma CEA, whereas there was only 1 case (2.1%) with an elevated plasma CEA and negative ctDNA test.

Among recurrences considered amenable to surgery with curative intent, (n = 20), ctDNA was positive in 60% of patients, compared with only 20% for CEA (P = .010). Multivariate analysis indicated that ctDNA was an independent predictor of recurrence, whereas CEA was not.

The authors concluded that the methylated BCAT1/IKZF1 ctDNA test was superior to CEA monitoring after initial treatment for potentially curable CRC.

How these results influence practice

The current study adds to the body of work showing superior sensitivity of ctDNA monitoring in the detection of recurrence in patients with solid tumors. In May 2019, this column highlighted the work of Yuxuan Wang, MD, PhD, and colleagues in 58 patients with stages I-III CRC; over a similar duration of follow-up as in the current study (JAMA Oncol. 2019;5[8]:1118-23), Dr. Wang found that 10 of 13 recurrences (77%) were detected by monitoring ctDNA levels. CEA levels were detected 63% of recurrences.

The central, critical hypothesis of these efforts is that the earlier detection of metastatic disease will lead to improved survival for patients with CRC. As the authors state in their discussion, that hypothesis remains tantalizing but unproven.

Prospective, randomized trials like the recently opened COBRA trial (NRG-GI005), which tests ctDNA assay–directed therapy in patients with stage IIA CRC, deserve our enthusiastic support. Research with similar designs will establish the value of ctDNA monitoring as a biomarker for early intervention in patients with stage IV disease and for selecting patients after primary therapy who are unlikely to have been cured and who could potentially benefit from adjuvant therapy.

Monitoring ctDNA is a research tool that could be practice changing in the near future. It has shown enough promise to justify prospective, randomized trials to determine whether its superior sensitivity translates into improved survival.
 

Avapritinib for mutated GIST

The platelet-derived growth factor receptor–alpha (PDGFRA) gene encodes PDGFRA, a member of the type III tyrosine kinase receptor family, which includes the stem cell factor receptor, KIT (present in 95% of GISTs). PDGFRA and KIT have domains with specific roles in tyrosine kinase activation. Several PDGF isoforms bind and activate PDGFRA.

Among patients with GISTs, clinical responses to imatinib and other tyrosine kinase inhibitors (TKIs) correlate with tumor genotype. For example, many GISTs that progress within 6 months of TKI initiation lack mutations in KIT or PDGFRA or have a PDGFRA D842 mutation.

The Food and Drug Administration recently approved avapritinib (Ayvakit) for adults with unresectable or metastatic GISTs with a PDGFRA exon 18 mutation. The approval was based on the results of the NAVIGATOR trial, a single-arm, multicenter, open-label study of 43 patients with PDGFRA exon 18 mutations, including 38 patients with a PDGFRA D842V mutation.

The patients received 300 mg avapritinib once daily and 84% responded to avapritinib, though most were partial responses. Among patients with a D842V mutation, the response rate was also high – at 89% – but most responses were partial. At a median follow-up of 10.6 months, median response duration was not reached, but 61% of responses exceeded 6 months. Toxicity included asthenia, gastrointestinal and central nervous system side effects (including intracranial hemorrhage), hair color changes, lacrimation, and dizziness.

Avapritinib is the first drug approved specifically for patients with advanced or unresectable GISTs with a PDGFRA exon 18 mutation.

How these results influence practice

PDGFRA exon 18 mutations occur in 5%-7% of GISTs and are believed to stabilize the kinase activation loop, perhaps accounting for slow, steady growth of these tumors over a long time period and resistance to TKIs. The most frequent mutation results in an exon 18 D842V substitution (75% of all PDGFRA-mutated tumors). Although dramatic progress has been made in the treatment of patients with GIST since 2000, in patients with GISTs harboring PDGFRA exon 18 mutations, responses to treatment are rare and, when they occur, are more abbreviated.

With a high response rate and impressive response duration, avapritinib will be a valuable resource for oncologists treating this uncommon subset of patients with GISTs. The opportunity to convert unresectable tumors to potentially curable ones seems within reach.

The difficult dilemma will be to decide when treatment is needed and drug-related toxicity is justified in patients with slowly progressive tumors and few symptoms. As with so many decisions in oncology, until newer agents with fewer toxicities and higher complete response rates are developed, the proper time for any individual patient to embark on treatment with avapritinib will be found at the intersection of “precision medicine” and “clinical judgment.”

Dr. Lyss has been a community-based medical oncologist and clinical researcher for more than 35 years, practicing in St. Louis. His clinical and research interests are in the prevention, diagnosis, and treatment of breast and lung cancers and in expanding access to clinical trials to medically underserved populations.

Pyrrolidone carboxylic acid (PCA), the primary constituent of the natural moisturizing factor (NMF),¹ including its derivatives – such as simple² and novel³ esters as well as sugar complexes⁴ – is the subject of great interest and research regarding its capacity to moisturize the stratum corneum via topical application.

Creams and lotions containing the sodium salt of PCA are widely reported to aid in hydrating the skin and ameliorating dry flaky skin conditions.^5,6 In addition, the zinc salt of L-pyrrolidone carboxylate is a longtime cosmetic ingredient due to antimicrobial and astringent qualities. This column briefly addresses the role of PCA in skin health.⁷

Dry skin

In a comprehensive literature review from 1981, Clar and Fourtanier reported conclusive evidence that PCA acts as a hydrating agent and that all the cosmetic formulations with a minimum of 2% PCA and PCA salt that they tested in their own 8-year study enhanced dry skin in short- and long-term conditions given suitable vehicles (no aqueous solutions).⁶

In a 2014 clinical study of 64 healthy white women with either normal or cosmetic dry skin, Feng et al. noted that tape stripped samples of stratum corneum revealed significantly lower ratios of free amino acids to protein and PCA to protein. This was associated with decreased hydration levels compared with normal skin. The investigators concluded that lower NMF levels across the depth of the stratum corneum and reduced cohesivity characterize cosmetic dry skin and that these clinical endpoints merit attention in evaluating the usefulness of treatments for dry skin.⁸

In 2016, Wei et al. reported on their assessment of the barrier function, hydration, and dryness of the lower leg skin of 25 female patients during the winter and then in the subsequent summer. They found that PCA levels were significantly greater during the summer, as were keratins. Hydration was also higher during the summer, while transepidermal water loss and visual dryness grades were substantially lower.⁹
 

Atopic dermatitis

A 2014 clinical study by Brandt et al. in patients with skin prone to developing atopic dermatitis (AD) revealed that a body wash composed of the filaggrin metabolites arginine and PCA was well tolerated and diminished pruritus. Patients reported liking the product and suggested that it improved their quality of life.¹⁰

Later that year, Jung et al. characterized the relationship of PCA levels, and other factors, with the clinical severity of AD. Specifically, in a study of 73 subjects (21 with mild AD, 21 with moderate to severe AD, 13 with X-linked ichthyosis as a negative control for filaggrin gene mutation, and 18 healthy controls), the investigators assessed transepidermal water loss, stratum corneum hydration, and skin surface pH. They found that PCA levels and caspase-14 were lower in inflammatory lesions compared with nonlesional skin in subjects with AD. These levels also were associated with clinical AD severity as measured by eczema area and severity index scores as well as skin barrier function.¹¹
 

PCA as a biomarker

In 2009, Kezic et al. determined that the use of tape stripping to cull PCA in the stratum corneum was effective in revealing that PCA concentration in the outermost skin layer is a viable biomarker of filaggrin genotype.¹²

Raj et al. conducted an interesting study in 2016 in which they set out to describe the various markers for total NMF levels and link them to the activities of plasmin and corneocyte maturation in the photoexposed cheek and photoprotected postauricular regions of healthy white, black African, and albino African women in South Africa. PCA levels were highest among the albino African group, followed by black African and then white participants. The investigators also found that bleomycin hydrolase was linked to PCA synthesis, as suggested by higher bleomycin levels in albino African participants. In this group, corneocyte maturation was also observed to be impeded.¹³

The next year, the same team studied stratum corneum physiology and biochemistry of the cheeks in 48 white women with sensitive skin. The goal was to ascertain the connections between bleomycin hydrolase and calpain-1, PCA levels, corneocyte maturation, and transglutaminase and plasmin activities. Capsaicin sensitivity was observed in 52% of subjects, with PCA levels and bleomycin hydrolase activity found to be lower in the capsaicin-sensitive panel and correlated in subjects not sensitive to capsaicin. The researchers concluded that reduced levels of PCA, bleomycin hydrolase, and transglutaminase combined with a larger volume of immature corneocytes suggest comparatively poor stratum corneum maturation in individuals with sensitive skin.¹⁴

Other uses

In 2012, Takino et al. used cultured normal human dermal fibroblasts to show that zinc l-pyrrolidone carboxylate blocked UVA induction of activator protein-1, diminished matrix metalloproteinase-1 synthesis, and spurred type I collagen production. The researchers suggested that such results suggest the potential of zinc PCA for further investigation as an agent to combat photoaging.⁷

Conclusion

Pyrrolidone carboxylic acid is clearly established as the main component of the NMF. Recent research suggests that it may serve as an important biomarker of filaggrin, NMF levels, and skin hydration. In addition, new data point to its usefulness as a gauge for ADs. More investigations are necessary to ascertain the feasibility of adjusting PCA levels through topical administration and what effects topically applied PCA may have on various skin parameters.

Dr. Baumann is a private practice dermatologist, researcher, author, and entrepreneur in Miami. She founded the Cosmetic Dermatology Center at the University of Miami in 1997. Dr. Baumann wrote two textbooks, “Cosmetic Dermatology: Principles and Practice” (New York: McGraw-Hill, 2002) and “Cosmeceuticals and Cosmetic Ingredients” (New York: McGraw-Hill, 2014), as well as a New York Times Best Sellers book for consumers, “The Skin Type Solution” (New York: Bantam Dell, 2006). Dr. Baumann has received funding for advisory boards and/or clinical research trials from Allergan, Evolus, Galderma, and Revance. She is the founder and CEO of Skin Type Solutions Franchise Systems LLC. Write to her at dermnews@mdedge.com.

References

1. Björklund S et al. Soft Matter. 2014 Jul 7;10(25):4535-46.

2. Hall KJ, Hill JC. J Soc Cosmet Chem. 1986;37(6):397-407.

3. Tezuka T et al. Dermatology. 1994;188(1):21-4.

4. Kwoya Hakko Kogyo Co. Pyrrolidone carboxylic acid esters containing composition used to prevent loss of moisture from the skin. Patent JA 48 82 046 (1982).

5. Org Santerre. l-pyrrolidone carboxylic acid-sugar compounds as rehydrating ingredients in cosmetics. Patent Fr 2 277 823 (1977).

6. Clar EJ, Fourtanier A. Int J Cosmet Sci. 1981 Jun;3(3):101-13.

7. Takino Y et al. Int J Cosmet Sci. 2012 Feb;34(1):23-8.

8. Feng L et al. Int J Cosmet Sci. 2014 Jun;36(3):231-8.

9. Wei KS et al. J Cosmet Sci. 2016 May-Jun;67(3):185-203.

10. Brandt S et al. J Drugs Dermatol. 2014 Sep;13(9):1108-11.

11. Jung M et al. J Dermatol Sci. 2014 Dec;76(3):231-9.

12. Kezic S et al. Br J Dermatol. 2009 Nov;161(5):1098-104.

13. Raj N et al. Int J Cosmet Sci. 2016 Dec;38(6):567-75.

14. Raj N et al. Int J Cosmet Sci. 2017 Feb;39(1):2-10.

Pyrrolidone carboxylic acid (PCA), the primary constituent of the natural moisturizing factor (NMF),¹ including its derivatives – such as simple² and novel³ esters as well as sugar complexes⁴ – is the subject of great interest and research regarding its capacity to moisturize the stratum corneum via topical application.

Creams and lotions containing the sodium salt of PCA are widely reported to aid in hydrating the skin and ameliorating dry flaky skin conditions.^5,6 In addition, the zinc salt of L-pyrrolidone carboxylate is a longtime cosmetic ingredient due to antimicrobial and astringent qualities. This column briefly addresses the role of PCA in skin health.⁷

Dry skin

In a comprehensive literature review from 1981, Clar and Fourtanier reported conclusive evidence that PCA acts as a hydrating agent and that all the cosmetic formulations with a minimum of 2% PCA and PCA salt that they tested in their own 8-year study enhanced dry skin in short- and long-term conditions given suitable vehicles (no aqueous solutions).⁶

In a 2014 clinical study of 64 healthy white women with either normal or cosmetic dry skin, Feng et al. noted that tape stripped samples of stratum corneum revealed significantly lower ratios of free amino acids to protein and PCA to protein. This was associated with decreased hydration levels compared with normal skin. The investigators concluded that lower NMF levels across the depth of the stratum corneum and reduced cohesivity characterize cosmetic dry skin and that these clinical endpoints merit attention in evaluating the usefulness of treatments for dry skin.⁸

In 2016, Wei et al. reported on their assessment of the barrier function, hydration, and dryness of the lower leg skin of 25 female patients during the winter and then in the subsequent summer. They found that PCA levels were significantly greater during the summer, as were keratins. Hydration was also higher during the summer, while transepidermal water loss and visual dryness grades were substantially lower.⁹
 

Atopic dermatitis

A 2014 clinical study by Brandt et al. in patients with skin prone to developing atopic dermatitis (AD) revealed that a body wash composed of the filaggrin metabolites arginine and PCA was well tolerated and diminished pruritus. Patients reported liking the product and suggested that it improved their quality of life.¹⁰

Later that year, Jung et al. characterized the relationship of PCA levels, and other factors, with the clinical severity of AD. Specifically, in a study of 73 subjects (21 with mild AD, 21 with moderate to severe AD, 13 with X-linked ichthyosis as a negative control for filaggrin gene mutation, and 18 healthy controls), the investigators assessed transepidermal water loss, stratum corneum hydration, and skin surface pH. They found that PCA levels and caspase-14 were lower in inflammatory lesions compared with nonlesional skin in subjects with AD. These levels also were associated with clinical AD severity as measured by eczema area and severity index scores as well as skin barrier function.¹¹
 

PCA as a biomarker

In 2009, Kezic et al. determined that the use of tape stripping to cull PCA in the stratum corneum was effective in revealing that PCA concentration in the outermost skin layer is a viable biomarker of filaggrin genotype.¹²

Raj et al. conducted an interesting study in 2016 in which they set out to describe the various markers for total NMF levels and link them to the activities of plasmin and corneocyte maturation in the photoexposed cheek and photoprotected postauricular regions of healthy white, black African, and albino African women in South Africa. PCA levels were highest among the albino African group, followed by black African and then white participants. The investigators also found that bleomycin hydrolase was linked to PCA synthesis, as suggested by higher bleomycin levels in albino African participants. In this group, corneocyte maturation was also observed to be impeded.¹³

The next year, the same team studied stratum corneum physiology and biochemistry of the cheeks in 48 white women with sensitive skin. The goal was to ascertain the connections between bleomycin hydrolase and calpain-1, PCA levels, corneocyte maturation, and transglutaminase and plasmin activities. Capsaicin sensitivity was observed in 52% of subjects, with PCA levels and bleomycin hydrolase activity found to be lower in the capsaicin-sensitive panel and correlated in subjects not sensitive to capsaicin. The researchers concluded that reduced levels of PCA, bleomycin hydrolase, and transglutaminase combined with a larger volume of immature corneocytes suggest comparatively poor stratum corneum maturation in individuals with sensitive skin.¹⁴

Other uses

In 2012, Takino et al. used cultured normal human dermal fibroblasts to show that zinc l-pyrrolidone carboxylate blocked UVA induction of activator protein-1, diminished matrix metalloproteinase-1 synthesis, and spurred type I collagen production. The researchers suggested that such results suggest the potential of zinc PCA for further investigation as an agent to combat photoaging.⁷

Conclusion

Pyrrolidone carboxylic acid is clearly established as the main component of the NMF. Recent research suggests that it may serve as an important biomarker of filaggrin, NMF levels, and skin hydration. In addition, new data point to its usefulness as a gauge for ADs. More investigations are necessary to ascertain the feasibility of adjusting PCA levels through topical administration and what effects topically applied PCA may have on various skin parameters.

Dr. Baumann is a private practice dermatologist, researcher, author, and entrepreneur in Miami. She founded the Cosmetic Dermatology Center at the University of Miami in 1997. Dr. Baumann wrote two textbooks, “Cosmetic Dermatology: Principles and Practice” (New York: McGraw-Hill, 2002) and “Cosmeceuticals and Cosmetic Ingredients” (New York: McGraw-Hill, 2014), as well as a New York Times Best Sellers book for consumers, “The Skin Type Solution” (New York: Bantam Dell, 2006). Dr. Baumann has received funding for advisory boards and/or clinical research trials from Allergan, Evolus, Galderma, and Revance. She is the founder and CEO of Skin Type Solutions Franchise Systems LLC. Write to her at dermnews@mdedge.com.

References

1. Björklund S et al. Soft Matter. 2014 Jul 7;10(25):4535-46.

2. Hall KJ, Hill JC. J Soc Cosmet Chem. 1986;37(6):397-407.

3. Tezuka T et al. Dermatology. 1994;188(1):21-4.

4. Kwoya Hakko Kogyo Co. Pyrrolidone carboxylic acid esters containing composition used to prevent loss of moisture from the skin. Patent JA 48 82 046 (1982).

5. Org Santerre. l-pyrrolidone carboxylic acid-sugar compounds as rehydrating ingredients in cosmetics. Patent Fr 2 277 823 (1977).

6. Clar EJ, Fourtanier A. Int J Cosmet Sci. 1981 Jun;3(3):101-13.

7. Takino Y et al. Int J Cosmet Sci. 2012 Feb;34(1):23-8.

8. Feng L et al. Int J Cosmet Sci. 2014 Jun;36(3):231-8.

9. Wei KS et al. J Cosmet Sci. 2016 May-Jun;67(3):185-203.

10. Brandt S et al. J Drugs Dermatol. 2014 Sep;13(9):1108-11.

11. Jung M et al. J Dermatol Sci. 2014 Dec;76(3):231-9.

12. Kezic S et al. Br J Dermatol. 2009 Nov;161(5):1098-104.

13. Raj N et al. Int J Cosmet Sci. 2016 Dec;38(6):567-75.

14. Raj N et al. Int J Cosmet Sci. 2017 Feb;39(1):2-10.

Pyrrolidone carboxylic acid (PCA), the primary constituent of the natural moisturizing factor (NMF),¹ including its derivatives – such as simple² and novel³ esters as well as sugar complexes⁴ – is the subject of great interest and research regarding its capacity to moisturize the stratum corneum via topical application.

Creams and lotions containing the sodium salt of PCA are widely reported to aid in hydrating the skin and ameliorating dry flaky skin conditions.^5,6 In addition, the zinc salt of L-pyrrolidone carboxylate is a longtime cosmetic ingredient due to antimicrobial and astringent qualities. This column briefly addresses the role of PCA in skin health.⁷

Dry skin

In a comprehensive literature review from 1981, Clar and Fourtanier reported conclusive evidence that PCA acts as a hydrating agent and that all the cosmetic formulations with a minimum of 2% PCA and PCA salt that they tested in their own 8-year study enhanced dry skin in short- and long-term conditions given suitable vehicles (no aqueous solutions).⁶

In a 2014 clinical study of 64 healthy white women with either normal or cosmetic dry skin, Feng et al. noted that tape stripped samples of stratum corneum revealed significantly lower ratios of free amino acids to protein and PCA to protein. This was associated with decreased hydration levels compared with normal skin. The investigators concluded that lower NMF levels across the depth of the stratum corneum and reduced cohesivity characterize cosmetic dry skin and that these clinical endpoints merit attention in evaluating the usefulness of treatments for dry skin.⁸

In 2016, Wei et al. reported on their assessment of the barrier function, hydration, and dryness of the lower leg skin of 25 female patients during the winter and then in the subsequent summer. They found that PCA levels were significantly greater during the summer, as were keratins. Hydration was also higher during the summer, while transepidermal water loss and visual dryness grades were substantially lower.⁹
 

Atopic dermatitis

A 2014 clinical study by Brandt et al. in patients with skin prone to developing atopic dermatitis (AD) revealed that a body wash composed of the filaggrin metabolites arginine and PCA was well tolerated and diminished pruritus. Patients reported liking the product and suggested that it improved their quality of life.¹⁰

Later that year, Jung et al. characterized the relationship of PCA levels, and other factors, with the clinical severity of AD. Specifically, in a study of 73 subjects (21 with mild AD, 21 with moderate to severe AD, 13 with X-linked ichthyosis as a negative control for filaggrin gene mutation, and 18 healthy controls), the investigators assessed transepidermal water loss, stratum corneum hydration, and skin surface pH. They found that PCA levels and caspase-14 were lower in inflammatory lesions compared with nonlesional skin in subjects with AD. These levels also were associated with clinical AD severity as measured by eczema area and severity index scores as well as skin barrier function.¹¹
 

PCA as a biomarker

In 2009, Kezic et al. determined that the use of tape stripping to cull PCA in the stratum corneum was effective in revealing that PCA concentration in the outermost skin layer is a viable biomarker of filaggrin genotype.¹²

Raj et al. conducted an interesting study in 2016 in which they set out to describe the various markers for total NMF levels and link them to the activities of plasmin and corneocyte maturation in the photoexposed cheek and photoprotected postauricular regions of healthy white, black African, and albino African women in South Africa. PCA levels were highest among the albino African group, followed by black African and then white participants. The investigators also found that bleomycin hydrolase was linked to PCA synthesis, as suggested by higher bleomycin levels in albino African participants. In this group, corneocyte maturation was also observed to be impeded.¹³

The next year, the same team studied stratum corneum physiology and biochemistry of the cheeks in 48 white women with sensitive skin. The goal was to ascertain the connections between bleomycin hydrolase and calpain-1, PCA levels, corneocyte maturation, and transglutaminase and plasmin activities. Capsaicin sensitivity was observed in 52% of subjects, with PCA levels and bleomycin hydrolase activity found to be lower in the capsaicin-sensitive panel and correlated in subjects not sensitive to capsaicin. The researchers concluded that reduced levels of PCA, bleomycin hydrolase, and transglutaminase combined with a larger volume of immature corneocytes suggest comparatively poor stratum corneum maturation in individuals with sensitive skin.¹⁴

Other uses

In 2012, Takino et al. used cultured normal human dermal fibroblasts to show that zinc l-pyrrolidone carboxylate blocked UVA induction of activator protein-1, diminished matrix metalloproteinase-1 synthesis, and spurred type I collagen production. The researchers suggested that such results suggest the potential of zinc PCA for further investigation as an agent to combat photoaging.⁷

Conclusion

Pyrrolidone carboxylic acid is clearly established as the main component of the NMF. Recent research suggests that it may serve as an important biomarker of filaggrin, NMF levels, and skin hydration. In addition, new data point to its usefulness as a gauge for ADs. More investigations are necessary to ascertain the feasibility of adjusting PCA levels through topical administration and what effects topically applied PCA may have on various skin parameters.

Dr. Baumann is a private practice dermatologist, researcher, author, and entrepreneur in Miami. She founded the Cosmetic Dermatology Center at the University of Miami in 1997. Dr. Baumann wrote two textbooks, “Cosmetic Dermatology: Principles and Practice” (New York: McGraw-Hill, 2002) and “Cosmeceuticals and Cosmetic Ingredients” (New York: McGraw-Hill, 2014), as well as a New York Times Best Sellers book for consumers, “The Skin Type Solution” (New York: Bantam Dell, 2006). Dr. Baumann has received funding for advisory boards and/or clinical research trials from Allergan, Evolus, Galderma, and Revance. She is the founder and CEO of Skin Type Solutions Franchise Systems LLC. Write to her at dermnews@mdedge.com.

References

1. Björklund S et al. Soft Matter. 2014 Jul 7;10(25):4535-46.

2. Hall KJ, Hill JC. J Soc Cosmet Chem. 1986;37(6):397-407.

3. Tezuka T et al. Dermatology. 1994;188(1):21-4.

4. Kwoya Hakko Kogyo Co. Pyrrolidone carboxylic acid esters containing composition used to prevent loss of moisture from the skin. Patent JA 48 82 046 (1982).

5. Org Santerre. l-pyrrolidone carboxylic acid-sugar compounds as rehydrating ingredients in cosmetics. Patent Fr 2 277 823 (1977).

6. Clar EJ, Fourtanier A. Int J Cosmet Sci. 1981 Jun;3(3):101-13.

7. Takino Y et al. Int J Cosmet Sci. 2012 Feb;34(1):23-8.

8. Feng L et al. Int J Cosmet Sci. 2014 Jun;36(3):231-8.

9. Wei KS et al. J Cosmet Sci. 2016 May-Jun;67(3):185-203.

10. Brandt S et al. J Drugs Dermatol. 2014 Sep;13(9):1108-11.

11. Jung M et al. J Dermatol Sci. 2014 Dec;76(3):231-9.

12. Kezic S et al. Br J Dermatol. 2009 Nov;161(5):1098-104.

13. Raj N et al. Int J Cosmet Sci. 2016 Dec;38(6):567-75.

14. Raj N et al. Int J Cosmet Sci. 2017 Feb;39(1):2-10.

Did you notice that your practice slows down in February? In fact, if you plot your patient census over a few years, you may find that it dips every February. And you will discover other slow periods, perhaps in December, and busy months during other parts of the year.

Seasonality is yet another of those basic business concepts that most physicians have never heard of, because of the conspicuous lack of business training in medical schools. Seasonal fluctuations exist in one form or another in every business, including private medical practices. Why are people more or less willing to spend money at certain times of the year? Analysts usually blame slow business during January or February on reluctance to buy products or services after the holiday season. They attribute summer peaks to everything from warm weather to an increased propensity to buy when students are out of school, and summer slumps to vacationing customers. It is not always easy – or necessary – to explain seasonality. The point is that such behavior patterns do exist.

It would seem that this behavior would be easy to change, by running some ads, or doing an e-mail blast; but unfortunately, altering a seasonal pattern is not an option for a small private practice. It can be done, but it is a deep pockets game requiring long, expensive campaigns that are only practical for large corporations.

For example, soup was traditionally consumed during the winter months since time immemorial. After years of pervasive advertising extolling its nutritional virtues (remember “Soup is Good Food”?), the soup industry succeeded in convincing the public to use their product year-round. Obviously, that kind of large-scale behavior modification is not practical for a local medical practice.

Does that mean there is nothing we can do about our practices’ seasonal variations? Not at all; but we must work within the realities of our patients’ seasonal behavior, rather than attempting to change that behavior outright.

First, you need to know what that behavior is, because it varies from practice to practice, even within the same state or city. Plotting your seasonality is easy; you can make a graph on Excel in a few minutes. Ask your office manager or accountant for month-by-month billing figures for the last 2 or 3 years. (Make sure it’s the amount billed, not collected, since the latter lags the former by several weeks at least.) Plot those figures on the vertical arm and time (in months) on the horizontal. Alternatively you can plot patient visits per month, if you wish; I do both.

Once you know your seasonality, review your options. Modify your own habits when necessary. If you typically take a vacation in August, for example, that’s not a great idea if August is one of your busiest months; consider vacationing during predictable slow periods instead.

Though I have said that you can’t change most seasonal behavior, it is possible to “retrain” some of your long-time, loyal patients to come in during your slower periods for at least some of their care. Use insurance company rules as a financial incentive, where possible. Many of my patients are on Medicare, so I send a notice to all of them in early November each year, urging them to come in during December (one of my light months) before their deductible has to be paid again.

If you advertise your services, do the bulk of it during your busiest months. That might seem counterintuitive; why not advertise during slow periods to fill those empty slots? But once again, you cannot change seasonal behavior with a low-budget, local advertising campaign; physicians who attempt it invariably get a poor response to their ads. So don’t try to move the mountain to Mohammed. Advertise during your busy periods, when seasonal patterns predict that potential patients are more willing to spend money and are more likely to respond to your message.

In short, then, try to “flatten” your seasonal dips by persuading as many existing patients as possible to return during slower seasons. You can then encourage new patients to make appointments when they are receptive to purchasing new services, your seasonal peaks. Once in your practice, some of them can then be shifted into your slower periods, especially for predictable, periodic care.

Dr. Eastern practices dermatology and dermatologic surgery in Belleville, N.J. He is the author of numerous articles and textbook chapters, and is a longtime monthly columnist for Dermatology News. Write to him at dermnews@mdedge.com.

Did you notice that your practice slows down in February? In fact, if you plot your patient census over a few years, you may find that it dips every February. And you will discover other slow periods, perhaps in December, and busy months during other parts of the year.

Seasonality is yet another of those basic business concepts that most physicians have never heard of, because of the conspicuous lack of business training in medical schools. Seasonal fluctuations exist in one form or another in every business, including private medical practices. Why are people more or less willing to spend money at certain times of the year? Analysts usually blame slow business during January or February on reluctance to buy products or services after the holiday season. They attribute summer peaks to everything from warm weather to an increased propensity to buy when students are out of school, and summer slumps to vacationing customers. It is not always easy – or necessary – to explain seasonality. The point is that such behavior patterns do exist.

It would seem that this behavior would be easy to change, by running some ads, or doing an e-mail blast; but unfortunately, altering a seasonal pattern is not an option for a small private practice. It can be done, but it is a deep pockets game requiring long, expensive campaigns that are only practical for large corporations.

For example, soup was traditionally consumed during the winter months since time immemorial. After years of pervasive advertising extolling its nutritional virtues (remember “Soup is Good Food”?), the soup industry succeeded in convincing the public to use their product year-round. Obviously, that kind of large-scale behavior modification is not practical for a local medical practice.

Does that mean there is nothing we can do about our practices’ seasonal variations? Not at all; but we must work within the realities of our patients’ seasonal behavior, rather than attempting to change that behavior outright.

First, you need to know what that behavior is, because it varies from practice to practice, even within the same state or city. Plotting your seasonality is easy; you can make a graph on Excel in a few minutes. Ask your office manager or accountant for month-by-month billing figures for the last 2 or 3 years. (Make sure it’s the amount billed, not collected, since the latter lags the former by several weeks at least.) Plot those figures on the vertical arm and time (in months) on the horizontal. Alternatively you can plot patient visits per month, if you wish; I do both.

Once you know your seasonality, review your options. Modify your own habits when necessary. If you typically take a vacation in August, for example, that’s not a great idea if August is one of your busiest months; consider vacationing during predictable slow periods instead.

Though I have said that you can’t change most seasonal behavior, it is possible to “retrain” some of your long-time, loyal patients to come in during your slower periods for at least some of their care. Use insurance company rules as a financial incentive, where possible. Many of my patients are on Medicare, so I send a notice to all of them in early November each year, urging them to come in during December (one of my light months) before their deductible has to be paid again.

If you advertise your services, do the bulk of it during your busiest months. That might seem counterintuitive; why not advertise during slow periods to fill those empty slots? But once again, you cannot change seasonal behavior with a low-budget, local advertising campaign; physicians who attempt it invariably get a poor response to their ads. So don’t try to move the mountain to Mohammed. Advertise during your busy periods, when seasonal patterns predict that potential patients are more willing to spend money and are more likely to respond to your message.

In short, then, try to “flatten” your seasonal dips by persuading as many existing patients as possible to return during slower seasons. You can then encourage new patients to make appointments when they are receptive to purchasing new services, your seasonal peaks. Once in your practice, some of them can then be shifted into your slower periods, especially for predictable, periodic care.

Dr. Eastern practices dermatology and dermatologic surgery in Belleville, N.J. He is the author of numerous articles and textbook chapters, and is a longtime monthly columnist for Dermatology News. Write to him at dermnews@mdedge.com.

Did you notice that your practice slows down in February? In fact, if you plot your patient census over a few years, you may find that it dips every February. And you will discover other slow periods, perhaps in December, and busy months during other parts of the year.

Seasonality is yet another of those basic business concepts that most physicians have never heard of, because of the conspicuous lack of business training in medical schools. Seasonal fluctuations exist in one form or another in every business, including private medical practices. Why are people more or less willing to spend money at certain times of the year? Analysts usually blame slow business during January or February on reluctance to buy products or services after the holiday season. They attribute summer peaks to everything from warm weather to an increased propensity to buy when students are out of school, and summer slumps to vacationing customers. It is not always easy – or necessary – to explain seasonality. The point is that such behavior patterns do exist.

It would seem that this behavior would be easy to change, by running some ads, or doing an e-mail blast; but unfortunately, altering a seasonal pattern is not an option for a small private practice. It can be done, but it is a deep pockets game requiring long, expensive campaigns that are only practical for large corporations.

For example, soup was traditionally consumed during the winter months since time immemorial. After years of pervasive advertising extolling its nutritional virtues (remember “Soup is Good Food”?), the soup industry succeeded in convincing the public to use their product year-round. Obviously, that kind of large-scale behavior modification is not practical for a local medical practice.

Does that mean there is nothing we can do about our practices’ seasonal variations? Not at all; but we must work within the realities of our patients’ seasonal behavior, rather than attempting to change that behavior outright.

First, you need to know what that behavior is, because it varies from practice to practice, even within the same state or city. Plotting your seasonality is easy; you can make a graph on Excel in a few minutes. Ask your office manager or accountant for month-by-month billing figures for the last 2 or 3 years. (Make sure it’s the amount billed, not collected, since the latter lags the former by several weeks at least.) Plot those figures on the vertical arm and time (in months) on the horizontal. Alternatively you can plot patient visits per month, if you wish; I do both.

Once you know your seasonality, review your options. Modify your own habits when necessary. If you typically take a vacation in August, for example, that’s not a great idea if August is one of your busiest months; consider vacationing during predictable slow periods instead.

Though I have said that you can’t change most seasonal behavior, it is possible to “retrain” some of your long-time, loyal patients to come in during your slower periods for at least some of their care. Use insurance company rules as a financial incentive, where possible. Many of my patients are on Medicare, so I send a notice to all of them in early November each year, urging them to come in during December (one of my light months) before their deductible has to be paid again.

If you advertise your services, do the bulk of it during your busiest months. That might seem counterintuitive; why not advertise during slow periods to fill those empty slots? But once again, you cannot change seasonal behavior with a low-budget, local advertising campaign; physicians who attempt it invariably get a poor response to their ads. So don’t try to move the mountain to Mohammed. Advertise during your busy periods, when seasonal patterns predict that potential patients are more willing to spend money and are more likely to respond to your message.

In short, then, try to “flatten” your seasonal dips by persuading as many existing patients as possible to return during slower seasons. You can then encourage new patients to make appointments when they are receptive to purchasing new services, your seasonal peaks. Once in your practice, some of them can then be shifted into your slower periods, especially for predictable, periodic care.

Dr. Eastern practices dermatology and dermatologic surgery in Belleville, N.J. He is the author of numerous articles and textbook chapters, and is a longtime monthly columnist for Dermatology News. Write to him at dermnews@mdedge.com.

The time shared during clinical encounters provides small peeks into patients’ lives that get documented as episodic snapshots in electronic health records. But there is little information about how patients are doing outside of the office. With increasing emphasis on filling out mandatory parts of the EHR, there is less time available for in-depth, in-office conversations and phone follow-ups.

At the same time, it has become clear that it is not just the medicines we prescribe that affect our patients’ lives. Their behaviors outside of the office – being physically active, eating well, getting a good night’s rest, and adhering to medications – also impact their health outcomes.

The explosion of technology and personal data in our increasingly connected world provides powerful new sources of health and behavior information that generate new understanding of patients’ lives in their everyday settings.

The ubiquity and remarkable technological progress of personal computing devices – including wearables, smartphones, and tablets – along with the multitude of sensor modalities embedded within these devices, has enabled us to establish a continuous connection with people who want to share information about their behavior and daily life.

Such rich, longitudinal information, known as person-generated health data (PGHD), can be searched for physiological and behavioral signatures that can be used in combination with traditional clinical information to predict, diagnose, and treat disease. It can also be used to understand the safety and effectiveness of medical interventions.

PGHD is defined as wellness and/or health-related data created, recorded, or gathered by individuals. It reflects events and interactions that occur during an person’s everyday life. Systematically gathering this information and organizing it to better understand patients’ approach to their health or their unique experience living with disease provides meaningful insights that complement the data traditionally collected as part of clinical trials or periodic office visits.

PGHD can produce a rich picture of a person’s health or symptom burden with disease. It allows the opportunity to measure the real human burden of a patient’s disease and how it changes over time, with an opportunity to detect changes in symptoms in real time.

PGHD can also enable participation in health research.

An example would be the work of Evidation Health in San Mateo, Calif. Evidation provides a platform to run research studies utilizing technology and systems to measure health in everyday life. Its app, Achievement, collects continuous behavior-related data from smartphones, wearables, connected devices, and apps. That provides opportunities for participants to join research studies that develop novel measures designed to quantify health outcomes in a way that more accurately reflects an individual’s day-to-day activities and experience. All data collected are at the direction of and with the permission of the individual.

“Achievers” are given points for taking health-related actions such as tracking steps or their sleep, which convert to cash that can be kept or donated to their favorite charities. Achievement’s 3.5 million diverse participants also receive offers to join research studies. This paradigm shift dramatically expands access to research to increase diversity, shortens the time to first data through rapid recruitment, and enhances retention rates by making it easier to engage. To date, more than 1 million users have chosen to participate in research studies. The technology is bringing new data and insights to health research; it supports important questions about quality of life, medical products’ real-world effectiveness, and the development of hyperpersonalized health care services.

This new type of data is transforming medical research by creating real-world studies of unprecedented size, such as the Apple Heart Study – a virtual study with more than 400,000 enrolled participants – which was designed to test the accuracy of Apple Watches in safely identifying atrial fibrillation. The FDA has cleared two features on the Apple Watch: the device’s ability to detect and notify the user of an irregular heart rhythm, and the ability to take a single-lead EKG feature that can provide a rhythm strip for a clinician to review.

The FDA clearance letters specify that the apps are “not intended to replace traditional methods of diagnosis or treatment.” They provide extra information, and that information might be helpful – but the apps won’t replace a doctor’s visit. It remains to be seen how these data will be used, but they have the potential to identify atrial fibrillation early, leading to treatment that may prevent devastating strokes.

Another example of home-generated health data is a tool that has obtained FDA clearance as a diagnostic device with insurance reimbursement: WatchPAT, a portable sleep apnea diagnostic device. WatchPAT is worn like a simple wristwatch, with no need for belts, wires, or nasal cannulas.

Over time, in-home tests like these that are of minimal inconvenience to the patient and reflect a real-world experience may eclipse traditional sleep studies that require patients to spend the night in a clinic while attached to wires and monitors.

Health data generated by connected populations will yield novel insights that may help us better predict, diagnose, and treat disease. These are examples of innovations that can extend clinicians’ abilities to remotely monitor or diagnose health conditions, and we can expect that more will continue to be integrated into the clinical and research settings in the near future.

In part 2 of this series, we will discuss novel digital measures and studies utilizing PGHD to impact population health.
 

Dr. Skolnik is professor of family and community medicine at Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, and associate director, family medicine residency program, Abington (Pa.) Jefferson Health. Dr. Foschini is cofounder and chief data scientist at Evidation Health in San Mateo, Calif. Bray Patrick-Lake is a patient thought leader and director, strategic partnerships, at Evidation Health.

References

Determining real-world data’s fitness for use and the role of reliability, September 2019. Duke-Margolis Center for Health Policy.

N Engl J Med. 2019 Nov 14;381(20):1909-17.

The time shared during clinical encounters provides small peeks into patients’ lives that get documented as episodic snapshots in electronic health records. But there is little information about how patients are doing outside of the office. With increasing emphasis on filling out mandatory parts of the EHR, there is less time available for in-depth, in-office conversations and phone follow-ups.

At the same time, it has become clear that it is not just the medicines we prescribe that affect our patients’ lives. Their behaviors outside of the office – being physically active, eating well, getting a good night’s rest, and adhering to medications – also impact their health outcomes.

The explosion of technology and personal data in our increasingly connected world provides powerful new sources of health and behavior information that generate new understanding of patients’ lives in their everyday settings.

The ubiquity and remarkable technological progress of personal computing devices – including wearables, smartphones, and tablets – along with the multitude of sensor modalities embedded within these devices, has enabled us to establish a continuous connection with people who want to share information about their behavior and daily life.

Such rich, longitudinal information, known as person-generated health data (PGHD), can be searched for physiological and behavioral signatures that can be used in combination with traditional clinical information to predict, diagnose, and treat disease. It can also be used to understand the safety and effectiveness of medical interventions.

PGHD is defined as wellness and/or health-related data created, recorded, or gathered by individuals. It reflects events and interactions that occur during an person’s everyday life. Systematically gathering this information and organizing it to better understand patients’ approach to their health or their unique experience living with disease provides meaningful insights that complement the data traditionally collected as part of clinical trials or periodic office visits.

PGHD can produce a rich picture of a person’s health or symptom burden with disease. It allows the opportunity to measure the real human burden of a patient’s disease and how it changes over time, with an opportunity to detect changes in symptoms in real time.

PGHD can also enable participation in health research.

An example would be the work of Evidation Health in San Mateo, Calif. Evidation provides a platform to run research studies utilizing technology and systems to measure health in everyday life. Its app, Achievement, collects continuous behavior-related data from smartphones, wearables, connected devices, and apps. That provides opportunities for participants to join research studies that develop novel measures designed to quantify health outcomes in a way that more accurately reflects an individual’s day-to-day activities and experience. All data collected are at the direction of and with the permission of the individual.

“Achievers” are given points for taking health-related actions such as tracking steps or their sleep, which convert to cash that can be kept or donated to their favorite charities. Achievement’s 3.5 million diverse participants also receive offers to join research studies. This paradigm shift dramatically expands access to research to increase diversity, shortens the time to first data through rapid recruitment, and enhances retention rates by making it easier to engage. To date, more than 1 million users have chosen to participate in research studies. The technology is bringing new data and insights to health research; it supports important questions about quality of life, medical products’ real-world effectiveness, and the development of hyperpersonalized health care services.

This new type of data is transforming medical research by creating real-world studies of unprecedented size, such as the Apple Heart Study – a virtual study with more than 400,000 enrolled participants – which was designed to test the accuracy of Apple Watches in safely identifying atrial fibrillation. The FDA has cleared two features on the Apple Watch: the device’s ability to detect and notify the user of an irregular heart rhythm, and the ability to take a single-lead EKG feature that can provide a rhythm strip for a clinician to review.

The FDA clearance letters specify that the apps are “not intended to replace traditional methods of diagnosis or treatment.” They provide extra information, and that information might be helpful – but the apps won’t replace a doctor’s visit. It remains to be seen how these data will be used, but they have the potential to identify atrial fibrillation early, leading to treatment that may prevent devastating strokes.

Another example of home-generated health data is a tool that has obtained FDA clearance as a diagnostic device with insurance reimbursement: WatchPAT, a portable sleep apnea diagnostic device. WatchPAT is worn like a simple wristwatch, with no need for belts, wires, or nasal cannulas.

Over time, in-home tests like these that are of minimal inconvenience to the patient and reflect a real-world experience may eclipse traditional sleep studies that require patients to spend the night in a clinic while attached to wires and monitors.

Health data generated by connected populations will yield novel insights that may help us better predict, diagnose, and treat disease. These are examples of innovations that can extend clinicians’ abilities to remotely monitor or diagnose health conditions, and we can expect that more will continue to be integrated into the clinical and research settings in the near future.

In part 2 of this series, we will discuss novel digital measures and studies utilizing PGHD to impact population health.
 

Dr. Skolnik is professor of family and community medicine at Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, and associate director, family medicine residency program, Abington (Pa.) Jefferson Health. Dr. Foschini is cofounder and chief data scientist at Evidation Health in San Mateo, Calif. Bray Patrick-Lake is a patient thought leader and director, strategic partnerships, at Evidation Health.

References

Determining real-world data’s fitness for use and the role of reliability, September 2019. Duke-Margolis Center for Health Policy.

N Engl J Med. 2019 Nov 14;381(20):1909-17.

The time shared during clinical encounters provides small peeks into patients’ lives that get documented as episodic snapshots in electronic health records. But there is little information about how patients are doing outside of the office. With increasing emphasis on filling out mandatory parts of the EHR, there is less time available for in-depth, in-office conversations and phone follow-ups.

At the same time, it has become clear that it is not just the medicines we prescribe that affect our patients’ lives. Their behaviors outside of the office – being physically active, eating well, getting a good night’s rest, and adhering to medications – also impact their health outcomes.

The explosion of technology and personal data in our increasingly connected world provides powerful new sources of health and behavior information that generate new understanding of patients’ lives in their everyday settings.

The ubiquity and remarkable technological progress of personal computing devices – including wearables, smartphones, and tablets – along with the multitude of sensor modalities embedded within these devices, has enabled us to establish a continuous connection with people who want to share information about their behavior and daily life.

Such rich, longitudinal information, known as person-generated health data (PGHD), can be searched for physiological and behavioral signatures that can be used in combination with traditional clinical information to predict, diagnose, and treat disease. It can also be used to understand the safety and effectiveness of medical interventions.

PGHD is defined as wellness and/or health-related data created, recorded, or gathered by individuals. It reflects events and interactions that occur during an person’s everyday life. Systematically gathering this information and organizing it to better understand patients’ approach to their health or their unique experience living with disease provides meaningful insights that complement the data traditionally collected as part of clinical trials or periodic office visits.

PGHD can produce a rich picture of a person’s health or symptom burden with disease. It allows the opportunity to measure the real human burden of a patient’s disease and how it changes over time, with an opportunity to detect changes in symptoms in real time.

PGHD can also enable participation in health research.

An example would be the work of Evidation Health in San Mateo, Calif. Evidation provides a platform to run research studies utilizing technology and systems to measure health in everyday life. Its app, Achievement, collects continuous behavior-related data from smartphones, wearables, connected devices, and apps. That provides opportunities for participants to join research studies that develop novel measures designed to quantify health outcomes in a way that more accurately reflects an individual’s day-to-day activities and experience. All data collected are at the direction of and with the permission of the individual.

“Achievers” are given points for taking health-related actions such as tracking steps or their sleep, which convert to cash that can be kept or donated to their favorite charities. Achievement’s 3.5 million diverse participants also receive offers to join research studies. This paradigm shift dramatically expands access to research to increase diversity, shortens the time to first data through rapid recruitment, and enhances retention rates by making it easier to engage. To date, more than 1 million users have chosen to participate in research studies. The technology is bringing new data and insights to health research; it supports important questions about quality of life, medical products’ real-world effectiveness, and the development of hyperpersonalized health care services.

This new type of data is transforming medical research by creating real-world studies of unprecedented size, such as the Apple Heart Study – a virtual study with more than 400,000 enrolled participants – which was designed to test the accuracy of Apple Watches in safely identifying atrial fibrillation. The FDA has cleared two features on the Apple Watch: the device’s ability to detect and notify the user of an irregular heart rhythm, and the ability to take a single-lead EKG feature that can provide a rhythm strip for a clinician to review.

The FDA clearance letters specify that the apps are “not intended to replace traditional methods of diagnosis or treatment.” They provide extra information, and that information might be helpful – but the apps won’t replace a doctor’s visit. It remains to be seen how these data will be used, but they have the potential to identify atrial fibrillation early, leading to treatment that may prevent devastating strokes.

Another example of home-generated health data is a tool that has obtained FDA clearance as a diagnostic device with insurance reimbursement: WatchPAT, a portable sleep apnea diagnostic device. WatchPAT is worn like a simple wristwatch, with no need for belts, wires, or nasal cannulas.

Over time, in-home tests like these that are of minimal inconvenience to the patient and reflect a real-world experience may eclipse traditional sleep studies that require patients to spend the night in a clinic while attached to wires and monitors.

Health data generated by connected populations will yield novel insights that may help us better predict, diagnose, and treat disease. These are examples of innovations that can extend clinicians’ abilities to remotely monitor or diagnose health conditions, and we can expect that more will continue to be integrated into the clinical and research settings in the near future.

In part 2 of this series, we will discuss novel digital measures and studies utilizing PGHD to impact population health.
 

Dr. Skolnik is professor of family and community medicine at Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, and associate director, family medicine residency program, Abington (Pa.) Jefferson Health. Dr. Foschini is cofounder and chief data scientist at Evidation Health in San Mateo, Calif. Bray Patrick-Lake is a patient thought leader and director, strategic partnerships, at Evidation Health.

References

Determining real-world data’s fitness for use and the role of reliability, September 2019. Duke-Margolis Center for Health Policy.

N Engl J Med. 2019 Nov 14;381(20):1909-17.

Lichenoid dermatoses are a heterogeneous group of diseases with varying clinical presentations. The term “lichenoid” refers to the popular lesions of certain skin disorders of which lichen planus (LP) is the prototype. The papules are shiny, flat topped, polygonal, of different sizes, and occur in clusters creating a pattern that resembles lichen growing on a rock. Lichenoid eruptions are quite common in children and can result from many different origins. In most instances the precise mechanism of disease is not known, although it is usually believed to be immunologic in nature. Certain disorders are common in children, whereas others more often affect the adult population.

Lichen striatus, lichen nitidus (LN), and lichen spinulosus are lichenoid lesions that are more common in children than adults.

LN – as seen in the patient described here – is an uncommon benign inflammatory skin disease, primarily of children. Individual lesions are sharply demarcated, pinpoint to pinhead sized, round or polygonal, and strikingly monomorphous in nature. The papules are usually flesh colored, however, the color varies from yellow and brown to violet hues depending on the background color of the patient’s skin. This variation in color is in contrast with LP which is characteristically violaceous. The surfaces of the papules are flat, shiny, and slightly elevated. They may have a fine scale or a hyperkeratotic plug. The lesions tend to occur in groups, primarily on the abdomen, chest, glans penis, and upper extremities. The Koebner phenomenon is observed and is a hallmark for the disorder. LN is generally asymptomatic, unlike LP, which is exceedingly pruritic.

The cause of LN is unknown; however, it has been proposed that LN, in particular generalized LN, may be associated with immune alterations in the patient. The course of LN is slowly progressive with a tendency toward remission. The lesions can remain stationary for years; however, they sometimes disappear spontaneously and completely.

The differential diagnosis of LN beyond the entities discussed above includes frictional lichenoid eruption, lichenoid drug eruption, LP, and keratosis pilaris.

LP is the classic lichenoid eruption. It is rare in children and occurs most frequently in individuals aged 30-60 years. LP usually manifests as an extremely pruritic eruption of flat-topped polygonal and violaceous papules that often have fine linear white scales known as Wickham striae. The distribution is usually bilateral and symmetric with most of the papules and plaques located on the legs, flexor wrists, neck, and genitalia. The lesions may exhibit the Koebner phenomenon, appearing in a linear pattern along the site of a scratch. Generally, in childhood cases there is reported itching, and oral and nail lesions are less common.

Frictional lichenoid eruption occurs in childhood. The lesions consist of lichenoid papules with regular borders 1-2 mm in diameter that generally are asymptomatic, although they may be mildly pruritic. The papules are found in a very characteristic distribution with almost exclusive involvement of the backs of the hands, fingers, elbows, and knees with occasional involvement of the extensor forearms and cheeks. This disorder occurs in predisposed children who have been exposed to significant frictional force during play, and typically resolves spontaneously after removal of the stimulus.

Keratosis pilaris is a rash that usually is found on the outer areas of the upper arms, upper thighs, buttocks, and cheeks. It consists of small bumps that are flesh colored to red. The bumps generally don’t hurt or itch.

The lack of symptoms and spontaneous healing have rendered treatment unnecessary in most cases. LN generally is self-limiting, thus treatment may not be necessary. However, topical treatment with mid- to high-potency corticosteroids has hastened resolution of lesions in some children, as have topical dinitrochlorobenzene and systemic treatment with psoralens, astemizole, etretinate, and psoralen-UVA.

Dr. Eichenfield is chief of pediatric and adolescent dermatology at Rady Children’s Hospital–San Diego. He is vice chair of the department of dermatology and professor of dermatology and pediatrics at the University of California, San Diego. Dr. Bhatti is a research fellow in pediatric dermatology at Rady Children’s Hospital and the University of California, San Diego. Neither Dr. Eichenfield nor Dr. Bhatti has any relevant financial disclosures. Email them at pdnews@mdedge.com.

References

Pickert A. Cutis. 2012 Sep;90(3):E1-3. https://mdedge-files-live.s3.us-east-2.amazonaws.com/files/s3fs-public/Document/September-2017/0900300E1.pdf Tziotzios C et al. J Am Acad Dermatol. 2018 Nov;79(5):789-804. Tilly JJ et al. J Am Acad Dermatol. 2004 Oct;51(4):606-24.

Lichenoid dermatoses are a heterogeneous group of diseases with varying clinical presentations. The term “lichenoid” refers to the popular lesions of certain skin disorders of which lichen planus (LP) is the prototype. The papules are shiny, flat topped, polygonal, of different sizes, and occur in clusters creating a pattern that resembles lichen growing on a rock. Lichenoid eruptions are quite common in children and can result from many different origins. In most instances the precise mechanism of disease is not known, although it is usually believed to be immunologic in nature. Certain disorders are common in children, whereas others more often affect the adult population.

Lichen striatus, lichen nitidus (LN), and lichen spinulosus are lichenoid lesions that are more common in children than adults.

LN – as seen in the patient described here – is an uncommon benign inflammatory skin disease, primarily of children. Individual lesions are sharply demarcated, pinpoint to pinhead sized, round or polygonal, and strikingly monomorphous in nature. The papules are usually flesh colored, however, the color varies from yellow and brown to violet hues depending on the background color of the patient’s skin. This variation in color is in contrast with LP which is characteristically violaceous. The surfaces of the papules are flat, shiny, and slightly elevated. They may have a fine scale or a hyperkeratotic plug. The lesions tend to occur in groups, primarily on the abdomen, chest, glans penis, and upper extremities. The Koebner phenomenon is observed and is a hallmark for the disorder. LN is generally asymptomatic, unlike LP, which is exceedingly pruritic.

The cause of LN is unknown; however, it has been proposed that LN, in particular generalized LN, may be associated with immune alterations in the patient. The course of LN is slowly progressive with a tendency toward remission. The lesions can remain stationary for years; however, they sometimes disappear spontaneously and completely.

The differential diagnosis of LN beyond the entities discussed above includes frictional lichenoid eruption, lichenoid drug eruption, LP, and keratosis pilaris.

LP is the classic lichenoid eruption. It is rare in children and occurs most frequently in individuals aged 30-60 years. LP usually manifests as an extremely pruritic eruption of flat-topped polygonal and violaceous papules that often have fine linear white scales known as Wickham striae. The distribution is usually bilateral and symmetric with most of the papules and plaques located on the legs, flexor wrists, neck, and genitalia. The lesions may exhibit the Koebner phenomenon, appearing in a linear pattern along the site of a scratch. Generally, in childhood cases there is reported itching, and oral and nail lesions are less common.

Frictional lichenoid eruption occurs in childhood. The lesions consist of lichenoid papules with regular borders 1-2 mm in diameter that generally are asymptomatic, although they may be mildly pruritic. The papules are found in a very characteristic distribution with almost exclusive involvement of the backs of the hands, fingers, elbows, and knees with occasional involvement of the extensor forearms and cheeks. This disorder occurs in predisposed children who have been exposed to significant frictional force during play, and typically resolves spontaneously after removal of the stimulus.

Keratosis pilaris is a rash that usually is found on the outer areas of the upper arms, upper thighs, buttocks, and cheeks. It consists of small bumps that are flesh colored to red. The bumps generally don’t hurt or itch.

The lack of symptoms and spontaneous healing have rendered treatment unnecessary in most cases. LN generally is self-limiting, thus treatment may not be necessary. However, topical treatment with mid- to high-potency corticosteroids has hastened resolution of lesions in some children, as have topical dinitrochlorobenzene and systemic treatment with psoralens, astemizole, etretinate, and psoralen-UVA.

Dr. Eichenfield is chief of pediatric and adolescent dermatology at Rady Children’s Hospital–San Diego. He is vice chair of the department of dermatology and professor of dermatology and pediatrics at the University of California, San Diego. Dr. Bhatti is a research fellow in pediatric dermatology at Rady Children’s Hospital and the University of California, San Diego. Neither Dr. Eichenfield nor Dr. Bhatti has any relevant financial disclosures. Email them at pdnews@mdedge.com.

References

Pickert A. Cutis. 2012 Sep;90(3):E1-3. https://mdedge-files-live.s3.us-east-2.amazonaws.com/files/s3fs-public/Document/September-2017/0900300E1.pdf Tziotzios C et al. J Am Acad Dermatol. 2018 Nov;79(5):789-804. Tilly JJ et al. J Am Acad Dermatol. 2004 Oct;51(4):606-24.

Lichenoid dermatoses are a heterogeneous group of diseases with varying clinical presentations. The term “lichenoid” refers to the popular lesions of certain skin disorders of which lichen planus (LP) is the prototype. The papules are shiny, flat topped, polygonal, of different sizes, and occur in clusters creating a pattern that resembles lichen growing on a rock. Lichenoid eruptions are quite common in children and can result from many different origins. In most instances the precise mechanism of disease is not known, although it is usually believed to be immunologic in nature. Certain disorders are common in children, whereas others more often affect the adult population.

Lichen striatus, lichen nitidus (LN), and lichen spinulosus are lichenoid lesions that are more common in children than adults.

LN – as seen in the patient described here – is an uncommon benign inflammatory skin disease, primarily of children. Individual lesions are sharply demarcated, pinpoint to pinhead sized, round or polygonal, and strikingly monomorphous in nature. The papules are usually flesh colored, however, the color varies from yellow and brown to violet hues depending on the background color of the patient’s skin. This variation in color is in contrast with LP which is characteristically violaceous. The surfaces of the papules are flat, shiny, and slightly elevated. They may have a fine scale or a hyperkeratotic plug. The lesions tend to occur in groups, primarily on the abdomen, chest, glans penis, and upper extremities. The Koebner phenomenon is observed and is a hallmark for the disorder. LN is generally asymptomatic, unlike LP, which is exceedingly pruritic.

The cause of LN is unknown; however, it has been proposed that LN, in particular generalized LN, may be associated with immune alterations in the patient. The course of LN is slowly progressive with a tendency toward remission. The lesions can remain stationary for years; however, they sometimes disappear spontaneously and completely.

The differential diagnosis of LN beyond the entities discussed above includes frictional lichenoid eruption, lichenoid drug eruption, LP, and keratosis pilaris.

LP is the classic lichenoid eruption. It is rare in children and occurs most frequently in individuals aged 30-60 years. LP usually manifests as an extremely pruritic eruption of flat-topped polygonal and violaceous papules that often have fine linear white scales known as Wickham striae. The distribution is usually bilateral and symmetric with most of the papules and plaques located on the legs, flexor wrists, neck, and genitalia. The lesions may exhibit the Koebner phenomenon, appearing in a linear pattern along the site of a scratch. Generally, in childhood cases there is reported itching, and oral and nail lesions are less common.

Frictional lichenoid eruption occurs in childhood. The lesions consist of lichenoid papules with regular borders 1-2 mm in diameter that generally are asymptomatic, although they may be mildly pruritic. The papules are found in a very characteristic distribution with almost exclusive involvement of the backs of the hands, fingers, elbows, and knees with occasional involvement of the extensor forearms and cheeks. This disorder occurs in predisposed children who have been exposed to significant frictional force during play, and typically resolves spontaneously after removal of the stimulus.

Keratosis pilaris is a rash that usually is found on the outer areas of the upper arms, upper thighs, buttocks, and cheeks. It consists of small bumps that are flesh colored to red. The bumps generally don’t hurt or itch.

The lack of symptoms and spontaneous healing have rendered treatment unnecessary in most cases. LN generally is self-limiting, thus treatment may not be necessary. However, topical treatment with mid- to high-potency corticosteroids has hastened resolution of lesions in some children, as have topical dinitrochlorobenzene and systemic treatment with psoralens, astemizole, etretinate, and psoralen-UVA.

Dr. Eichenfield is chief of pediatric and adolescent dermatology at Rady Children’s Hospital–San Diego. He is vice chair of the department of dermatology and professor of dermatology and pediatrics at the University of California, San Diego. Dr. Bhatti is a research fellow in pediatric dermatology at Rady Children’s Hospital and the University of California, San Diego. Neither Dr. Eichenfield nor Dr. Bhatti has any relevant financial disclosures. Email them at pdnews@mdedge.com.

References

Pickert A. Cutis. 2012 Sep;90(3):E1-3. https://mdedge-files-live.s3.us-east-2.amazonaws.com/files/s3fs-public/Document/September-2017/0900300E1.pdf Tziotzios C et al. J Am Acad Dermatol. 2018 Nov;79(5):789-804. Tilly JJ et al. J Am Acad Dermatol. 2004 Oct;51(4):606-24.

In May 2019, SHM sent a letter to U.S. Senators Tina Smith and Bill Cassidy in support of the Reducing Administrative Costs and Burdens in Health Care Act of 2019. In excerpts from the letter below, the society details the link between administrative burdens and physician burnout. 

Providers and hospital systems expend countless resources, both time and dollars, adhering to unnecessary and excessive administrative burdens instead of investing those resources in providing quality patient care. National data suggests that more than 50 percent of the physician workforce is burned out. Excessive administrative burden is a major contributor to physician burnout, which negatively affects quality and safety within the hospital and further increases health care costs. Notably, the Reducing Administrative Costs and Burdens in Health Care Act calls for a 50% reduction of unnecessary administrative costs from the Department of Health and Human Services within the next ten years.  

Hospitalists are front-line clinicians in America's acute care hospitals whose professional focus is the general medical care of hospitalized patients. Their unique position in the healthcare system affords hospitalists a distinct perspective and systems-based approach to confronting and solving challenges at the individual provider and overall institutional level of the hospital. In this capacity, hospitalists experience multiple examples of administrative requirements directly detracting from patient care and redirecting finite resources away from care to meet compliance demands. 
By way of example, navigating the administrative rules around inpatient admissions and outpatient observation care, for example, requires a significant shift of healthcare resources away from patient care. While patients admitted under observation receive nearly identical care to those admitted as an inpatient, hospitalists report that, in addition to themselves as the direct healthcare provider, status determinations between inpatient admissions and outpatient observation care require the input of a myriad of staff including nursing, coding/compliance teams, utilization review, case managers and external review organizations. A recent study in the Journal of Hospital Medicine indicated that an average of 5.1 full time employees, not including case managers, are required to navigate the audit and appeals process associated with hospital stay status determinations. These are resources that should be directly used for patient care, but are redirected towards regulation compliance, increasing cost of care without increasing quality. 

To read the entire letter, visit https://www.hospitalmedicine.org/policy--advocacy/letters/shm-supports-the-reducing-administrative-costs-and-burdens-in-health-care-act-of-2019/.

In May 2019, SHM sent a letter to U.S. Senators Tina Smith and Bill Cassidy in support of the Reducing Administrative Costs and Burdens in Health Care Act of 2019. In excerpts from the letter below, the society details the link between administrative burdens and physician burnout. 

Providers and hospital systems expend countless resources, both time and dollars, adhering to unnecessary and excessive administrative burdens instead of investing those resources in providing quality patient care. National data suggests that more than 50 percent of the physician workforce is burned out. Excessive administrative burden is a major contributor to physician burnout, which negatively affects quality and safety within the hospital and further increases health care costs. Notably, the Reducing Administrative Costs and Burdens in Health Care Act calls for a 50% reduction of unnecessary administrative costs from the Department of Health and Human Services within the next ten years.  

Hospitalists are front-line clinicians in America's acute care hospitals whose professional focus is the general medical care of hospitalized patients. Their unique position in the healthcare system affords hospitalists a distinct perspective and systems-based approach to confronting and solving challenges at the individual provider and overall institutional level of the hospital. In this capacity, hospitalists experience multiple examples of administrative requirements directly detracting from patient care and redirecting finite resources away from care to meet compliance demands. 
By way of example, navigating the administrative rules around inpatient admissions and outpatient observation care, for example, requires a significant shift of healthcare resources away from patient care. While patients admitted under observation receive nearly identical care to those admitted as an inpatient, hospitalists report that, in addition to themselves as the direct healthcare provider, status determinations between inpatient admissions and outpatient observation care require the input of a myriad of staff including nursing, coding/compliance teams, utilization review, case managers and external review organizations. A recent study in the Journal of Hospital Medicine indicated that an average of 5.1 full time employees, not including case managers, are required to navigate the audit and appeals process associated with hospital stay status determinations. These are resources that should be directly used for patient care, but are redirected towards regulation compliance, increasing cost of care without increasing quality. 

To read the entire letter, visit https://www.hospitalmedicine.org/policy--advocacy/letters/shm-supports-the-reducing-administrative-costs-and-burdens-in-health-care-act-of-2019/.

In May 2019, SHM sent a letter to U.S. Senators Tina Smith and Bill Cassidy in support of the Reducing Administrative Costs and Burdens in Health Care Act of 2019. In excerpts from the letter below, the society details the link between administrative burdens and physician burnout. 

Providers and hospital systems expend countless resources, both time and dollars, adhering to unnecessary and excessive administrative burdens instead of investing those resources in providing quality patient care. National data suggests that more than 50 percent of the physician workforce is burned out. Excessive administrative burden is a major contributor to physician burnout, which negatively affects quality and safety within the hospital and further increases health care costs. Notably, the Reducing Administrative Costs and Burdens in Health Care Act calls for a 50% reduction of unnecessary administrative costs from the Department of Health and Human Services within the next ten years.  

Hospitalists are front-line clinicians in America's acute care hospitals whose professional focus is the general medical care of hospitalized patients. Their unique position in the healthcare system affords hospitalists a distinct perspective and systems-based approach to confronting and solving challenges at the individual provider and overall institutional level of the hospital. In this capacity, hospitalists experience multiple examples of administrative requirements directly detracting from patient care and redirecting finite resources away from care to meet compliance demands. 
By way of example, navigating the administrative rules around inpatient admissions and outpatient observation care, for example, requires a significant shift of healthcare resources away from patient care. While patients admitted under observation receive nearly identical care to those admitted as an inpatient, hospitalists report that, in addition to themselves as the direct healthcare provider, status determinations between inpatient admissions and outpatient observation care require the input of a myriad of staff including nursing, coding/compliance teams, utilization review, case managers and external review organizations. A recent study in the Journal of Hospital Medicine indicated that an average of 5.1 full time employees, not including case managers, are required to navigate the audit and appeals process associated with hospital stay status determinations. These are resources that should be directly used for patient care, but are redirected towards regulation compliance, increasing cost of care without increasing quality. 

To read the entire letter, visit https://www.hospitalmedicine.org/policy--advocacy/letters/shm-supports-the-reducing-administrative-costs-and-burdens-in-health-care-act-of-2019/.

The mantra of community hospital administrators is that pediatric care does not pay. Neonatal intensive care pays. For pediatrics, it is similar to how football programs (Medicare patients) support minor sports (pediatrics and obstetrics) at colleges. However, fewer even mildly sick newborns are cared for at community hospitals, which has led to a centralization of neonatal and pediatric care and a loss of pediatric expertise at the affected hospitals.

JohnnyGreig/E+/Getty Images

Pediatric hospitalists are hired to cover the pediatric floor, the emergency department, and labor and delivery, then fired over empty pediatric beds. The rationale expressed is that pediatricians have done such a good job in preventive care that children rarely need hospitalization, so why have a pediatric inpatient unit? It is true that preventive care has been an integral part of primary care for children. Significantly less that 1% of child office visits result in hospitalization.

Advocate Health Care has closed inpatient pediatric units at Illinois Masonic, on Chicago’s North Side, Good Samaritan in Downers Grove, and Good Shepherd in Barrington. Units also have been closed at Mount Sinai in North Lawndale, Norwegian American on Chicago’s West Side, Little Company of Mary in Evergreen Park, and Alexian Brothers in Elk Grove.

As a Chicago-area pediatrician for more than 30 years, I have learned several things about community-based pediatric care:

1. Pediatrics is a geographic specialty. Parents will travel to shop, but would rather walk or have a short ride to their children’s medical providers. Secondary care should be community based, and hospitalization, if necessary, should be close by as well.

2. Hospitals that ceased delivering pediatric inpatient care lost their child-friendliness and pediatric competence, becoming uncomfortable delivering almost any care for children (e.g., sedated MRIs and EEGs, x-rays and ultrasounds, ECGs and echocardiograms, and emergency care).

3. In almost all hospitals, after pediatrics was gone eventually so passed obstetrics (another less remunerative specialty). Sick newborns need immediate, competent care. Most pediatric hospitalizations are short term, often overnight. Delaying newborn care is a medicolegal nightmare. Transferring a sick child to a distant hospital, to stay a day or 2, is counterintuitive, and exposes the child and his or her family to a potentially dangerous drive or helicopter ride.

4. As pediatric subspecialty care becomes more centralized, parents are asked to travel for hours to see a pediatric specialist. There are times when that is necessary (e.g., cardiovascular surgery). Pediatric subspecialists, such as pediatric otolaryngologists, then leave community hospitals, forcing even minor surgeries (e.g., ear ventilation tubes) to be done at a center. In rural areas, this could mean hours of travel, lost work days, and family disruption.

5. Children’s hospitals get uninsured and publicly insured children sent hundreds of miles, because there were no subspecialists in the community who would care for these children.

What is the solution, in our profit-focused health care system?

1. Hospitals’ Certificates of Need could include a mandate for pediatric care.

2. Children’s hospitals could be made responsible for community-based care within their geographic catchment areas.

3. The state or the federal government could mandate and financially support community-based hospital care.

4. Deciding what level of care might be appropriate for each community could depend upon closeness to a pediatric hospital, health problems in the community, and the availability of pediatric specialists.

5. A condition for medical licensure might be that a community-based pediatric subspecialist is required to care for a proportion of the uninsured or publicly insured children in his or her area.

6. Reimbursements for pediatric care need to rise enough to make caring for children worth it.

The major decision point regarding care for children cannot be financial, but must instead embrace the needs of each affected community. If quality health care is a right, and not a privilege, then it is time to stop closing pediatric inpatient units, and, instead, look for creative ways to better care for our children.

This process has led to pediatric care being available only in designated centers. The centralization of pediatric care has progressed from 30 years ago, when most community hospitals had inpatient pediatric units, to the search for innovative ways to fill pediatric beds in the mid-90s (sick day care, flex- or shared pediatric units), to the wholesale closure of community pediatric inpatient beds, from 2000 to the present. I have, unfortunately, seen this firsthand, watching the rise of pediatric mega-hospitals and the demise of community pediatrics. It is a simple financial argument. Care for children simply does not pay nearly as well as does care for adults, especially Medicaid patients. Pediatricians are the poorest paid practicing doctors (public health doctors are paid less).

It is true that pediatricians always have been at the forefront of preventive medicine, and that pediatric patients almost always get better, in spite of our best-intentioned interventions. So community-based pediatricians admit very few patients.

With the loss of pediatric units, community hospitals lose their comfort caring for children. This includes phlebotomy, x-ray, trauma, surgery, and behavioral health. And eroding community hospital pediatric expertise has catastrophic implications for rural hospitals, where parents may have to drive for hours to find a child-friendly emergency department.

Is there an answer?

1. Hospitals are responsible for the patients they serve, including children. Why should a hospital be able to close pediatric services so easily?

2. Every hospital that sees children, through the emergency department, needs to have a pediatrician available to evaluate a child, 24/7.

3. There needs to be an observation unit for children, with pediatric staffing, for overnight stays.

4. Pediatric hospitalists should be staffing community hospitals.

5. Pediatric behavioral health resources need to be available, e.g., inpatient psychiatry, partial hospitalization programs, intensive outpatient programs.

6. Telehealth communication is not adequate to address acute care problems, because the hospital caring for the child has to have the proper equipment and adequate expertise to carry out the recommendations of the teleconsultant.

If we accept that our children will shape the future, we must allow them to survive and thrive. Is health care a right or a privilege, and is it just for adults or for children, too?
 

Dr. Ochs is in private practice at Ravenswood Pediatrics in Chicago. He said he had no relevant financial disclosures. Email him at pdnews@mdedge.com.

The mantra of community hospital administrators is that pediatric care does not pay. Neonatal intensive care pays. For pediatrics, it is similar to how football programs (Medicare patients) support minor sports (pediatrics and obstetrics) at colleges. However, fewer even mildly sick newborns are cared for at community hospitals, which has led to a centralization of neonatal and pediatric care and a loss of pediatric expertise at the affected hospitals.

JohnnyGreig/E+/Getty Images

Pediatric hospitalists are hired to cover the pediatric floor, the emergency department, and labor and delivery, then fired over empty pediatric beds. The rationale expressed is that pediatricians have done such a good job in preventive care that children rarely need hospitalization, so why have a pediatric inpatient unit? It is true that preventive care has been an integral part of primary care for children. Significantly less that 1% of child office visits result in hospitalization.

Advocate Health Care has closed inpatient pediatric units at Illinois Masonic, on Chicago’s North Side, Good Samaritan in Downers Grove, and Good Shepherd in Barrington. Units also have been closed at Mount Sinai in North Lawndale, Norwegian American on Chicago’s West Side, Little Company of Mary in Evergreen Park, and Alexian Brothers in Elk Grove.

As a Chicago-area pediatrician for more than 30 years, I have learned several things about community-based pediatric care:

1. Pediatrics is a geographic specialty. Parents will travel to shop, but would rather walk or have a short ride to their children’s medical providers. Secondary care should be community based, and hospitalization, if necessary, should be close by as well.

2. Hospitals that ceased delivering pediatric inpatient care lost their child-friendliness and pediatric competence, becoming uncomfortable delivering almost any care for children (e.g., sedated MRIs and EEGs, x-rays and ultrasounds, ECGs and echocardiograms, and emergency care).

3. In almost all hospitals, after pediatrics was gone eventually so passed obstetrics (another less remunerative specialty). Sick newborns need immediate, competent care. Most pediatric hospitalizations are short term, often overnight. Delaying newborn care is a medicolegal nightmare. Transferring a sick child to a distant hospital, to stay a day or 2, is counterintuitive, and exposes the child and his or her family to a potentially dangerous drive or helicopter ride.

4. As pediatric subspecialty care becomes more centralized, parents are asked to travel for hours to see a pediatric specialist. There are times when that is necessary (e.g., cardiovascular surgery). Pediatric subspecialists, such as pediatric otolaryngologists, then leave community hospitals, forcing even minor surgeries (e.g., ear ventilation tubes) to be done at a center. In rural areas, this could mean hours of travel, lost work days, and family disruption.

5. Children’s hospitals get uninsured and publicly insured children sent hundreds of miles, because there were no subspecialists in the community who would care for these children.

What is the solution, in our profit-focused health care system?

1. Hospitals’ Certificates of Need could include a mandate for pediatric care.

2. Children’s hospitals could be made responsible for community-based care within their geographic catchment areas.

3. The state or the federal government could mandate and financially support community-based hospital care.

4. Deciding what level of care might be appropriate for each community could depend upon closeness to a pediatric hospital, health problems in the community, and the availability of pediatric specialists.

5. A condition for medical licensure might be that a community-based pediatric subspecialist is required to care for a proportion of the uninsured or publicly insured children in his or her area.

6. Reimbursements for pediatric care need to rise enough to make caring for children worth it.

The major decision point regarding care for children cannot be financial, but must instead embrace the needs of each affected community. If quality health care is a right, and not a privilege, then it is time to stop closing pediatric inpatient units, and, instead, look for creative ways to better care for our children.

This process has led to pediatric care being available only in designated centers. The centralization of pediatric care has progressed from 30 years ago, when most community hospitals had inpatient pediatric units, to the search for innovative ways to fill pediatric beds in the mid-90s (sick day care, flex- or shared pediatric units), to the wholesale closure of community pediatric inpatient beds, from 2000 to the present. I have, unfortunately, seen this firsthand, watching the rise of pediatric mega-hospitals and the demise of community pediatrics. It is a simple financial argument. Care for children simply does not pay nearly as well as does care for adults, especially Medicaid patients. Pediatricians are the poorest paid practicing doctors (public health doctors are paid less).

It is true that pediatricians always have been at the forefront of preventive medicine, and that pediatric patients almost always get better, in spite of our best-intentioned interventions. So community-based pediatricians admit very few patients.

With the loss of pediatric units, community hospitals lose their comfort caring for children. This includes phlebotomy, x-ray, trauma, surgery, and behavioral health. And eroding community hospital pediatric expertise has catastrophic implications for rural hospitals, where parents may have to drive for hours to find a child-friendly emergency department.

Is there an answer?

1. Hospitals are responsible for the patients they serve, including children. Why should a hospital be able to close pediatric services so easily?

2. Every hospital that sees children, through the emergency department, needs to have a pediatrician available to evaluate a child, 24/7.

3. There needs to be an observation unit for children, with pediatric staffing, for overnight stays.

4. Pediatric hospitalists should be staffing community hospitals.

5. Pediatric behavioral health resources need to be available, e.g., inpatient psychiatry, partial hospitalization programs, intensive outpatient programs.

6. Telehealth communication is not adequate to address acute care problems, because the hospital caring for the child has to have the proper equipment and adequate expertise to carry out the recommendations of the teleconsultant.

If we accept that our children will shape the future, we must allow them to survive and thrive. Is health care a right or a privilege, and is it just for adults or for children, too?
 

Dr. Ochs is in private practice at Ravenswood Pediatrics in Chicago. He said he had no relevant financial disclosures. Email him at pdnews@mdedge.com.

The mantra of community hospital administrators is that pediatric care does not pay. Neonatal intensive care pays. For pediatrics, it is similar to how football programs (Medicare patients) support minor sports (pediatrics and obstetrics) at colleges. However, fewer even mildly sick newborns are cared for at community hospitals, which has led to a centralization of neonatal and pediatric care and a loss of pediatric expertise at the affected hospitals.

JohnnyGreig/E+/Getty Images

Pediatric hospitalists are hired to cover the pediatric floor, the emergency department, and labor and delivery, then fired over empty pediatric beds. The rationale expressed is that pediatricians have done such a good job in preventive care that children rarely need hospitalization, so why have a pediatric inpatient unit? It is true that preventive care has been an integral part of primary care for children. Significantly less that 1% of child office visits result in hospitalization.

Advocate Health Care has closed inpatient pediatric units at Illinois Masonic, on Chicago’s North Side, Good Samaritan in Downers Grove, and Good Shepherd in Barrington. Units also have been closed at Mount Sinai in North Lawndale, Norwegian American on Chicago’s West Side, Little Company of Mary in Evergreen Park, and Alexian Brothers in Elk Grove.

As a Chicago-area pediatrician for more than 30 years, I have learned several things about community-based pediatric care:

1. Pediatrics is a geographic specialty. Parents will travel to shop, but would rather walk or have a short ride to their children’s medical providers. Secondary care should be community based, and hospitalization, if necessary, should be close by as well.

2. Hospitals that ceased delivering pediatric inpatient care lost their child-friendliness and pediatric competence, becoming uncomfortable delivering almost any care for children (e.g., sedated MRIs and EEGs, x-rays and ultrasounds, ECGs and echocardiograms, and emergency care).

3. In almost all hospitals, after pediatrics was gone eventually so passed obstetrics (another less remunerative specialty). Sick newborns need immediate, competent care. Most pediatric hospitalizations are short term, often overnight. Delaying newborn care is a medicolegal nightmare. Transferring a sick child to a distant hospital, to stay a day or 2, is counterintuitive, and exposes the child and his or her family to a potentially dangerous drive or helicopter ride.

4. As pediatric subspecialty care becomes more centralized, parents are asked to travel for hours to see a pediatric specialist. There are times when that is necessary (e.g., cardiovascular surgery). Pediatric subspecialists, such as pediatric otolaryngologists, then leave community hospitals, forcing even minor surgeries (e.g., ear ventilation tubes) to be done at a center. In rural areas, this could mean hours of travel, lost work days, and family disruption.

5. Children’s hospitals get uninsured and publicly insured children sent hundreds of miles, because there were no subspecialists in the community who would care for these children.

What is the solution, in our profit-focused health care system?

1. Hospitals’ Certificates of Need could include a mandate for pediatric care.

2. Children’s hospitals could be made responsible for community-based care within their geographic catchment areas.

3. The state or the federal government could mandate and financially support community-based hospital care.

4. Deciding what level of care might be appropriate for each community could depend upon closeness to a pediatric hospital, health problems in the community, and the availability of pediatric specialists.

5. A condition for medical licensure might be that a community-based pediatric subspecialist is required to care for a proportion of the uninsured or publicly insured children in his or her area.

6. Reimbursements for pediatric care need to rise enough to make caring for children worth it.

The major decision point regarding care for children cannot be financial, but must instead embrace the needs of each affected community. If quality health care is a right, and not a privilege, then it is time to stop closing pediatric inpatient units, and, instead, look for creative ways to better care for our children.

This process has led to pediatric care being available only in designated centers. The centralization of pediatric care has progressed from 30 years ago, when most community hospitals had inpatient pediatric units, to the search for innovative ways to fill pediatric beds in the mid-90s (sick day care, flex- or shared pediatric units), to the wholesale closure of community pediatric inpatient beds, from 2000 to the present. I have, unfortunately, seen this firsthand, watching the rise of pediatric mega-hospitals and the demise of community pediatrics. It is a simple financial argument. Care for children simply does not pay nearly as well as does care for adults, especially Medicaid patients. Pediatricians are the poorest paid practicing doctors (public health doctors are paid less).

It is true that pediatricians always have been at the forefront of preventive medicine, and that pediatric patients almost always get better, in spite of our best-intentioned interventions. So community-based pediatricians admit very few patients.

With the loss of pediatric units, community hospitals lose their comfort caring for children. This includes phlebotomy, x-ray, trauma, surgery, and behavioral health. And eroding community hospital pediatric expertise has catastrophic implications for rural hospitals, where parents may have to drive for hours to find a child-friendly emergency department.

Is there an answer?

1. Hospitals are responsible for the patients they serve, including children. Why should a hospital be able to close pediatric services so easily?

2. Every hospital that sees children, through the emergency department, needs to have a pediatrician available to evaluate a child, 24/7.

3. There needs to be an observation unit for children, with pediatric staffing, for overnight stays.

4. Pediatric hospitalists should be staffing community hospitals.

5. Pediatric behavioral health resources need to be available, e.g., inpatient psychiatry, partial hospitalization programs, intensive outpatient programs.

6. Telehealth communication is not adequate to address acute care problems, because the hospital caring for the child has to have the proper equipment and adequate expertise to carry out the recommendations of the teleconsultant.

If we accept that our children will shape the future, we must allow them to survive and thrive. Is health care a right or a privilege, and is it just for adults or for children, too?
 

Dr. Ochs is in private practice at Ravenswood Pediatrics in Chicago. He said he had no relevant financial disclosures. Email him at pdnews@mdedge.com.

Assessment and treatment of many of the more common behavioral disorders in childhood, such as ADHD and anxiety, should be considered within a pediatrician’s scope of practice, a stance made very clear by a recent policy statement published by the American Academy of Pediatrics entitled “Mental health competencies for pediatric practice.”¹ These competencies include medication treatment. As stated in the article, “certain disorders (ADHD, common anxiety disorders, depression), if associated with no more than moderate impairment, are amenable to primary care medication management because there are indicated medications with a well-established safety profile.”

This shift to shared ownership when it comes to mental health care is likely coming from multiple sources, not the least of them being necessity and an acknowledgment that there simply aren’t enough psychiatrists to take over the mental health care of every youth with a diagnosable psychiatric disorder. While the number of child and adolescent psychiatrists remains relatively flat, the youth suicide rate is rising, as are the numbers presenting to emergency departments in crisis – all for reasons still to be fully understood. And these trends all are occurring as the medical community overall is appreciating more and more that good mental health is a cornerstone of all health.

The response from the pediatric community, whether it be because of personal conviction or simply a lack of options, largely has been to step up to the plate and take on these new responsibilities and challenges while trying to get up to speed with the latest information about mental health best practices. Many pediatricians now fully endorse the idea that mental health problems should be considered as their “lane,” within certain boundaries. From my own experience doing evaluations and consultations from area primary care clinicians for over 15 years, the shift is noticeable. The typical patient now coming in has already seen a mental health counselor and tried at least one medication, while evaluations for diagnosis and treatment recommendations for things like uncomplicated and treatment-naive ADHD symptoms, for example, are becoming much more infrequent – although still far from extinct.

Nevertheless, there remain concerns about the extent of these new charges. Joe Nasca, MD, an experienced pediatrician who has been practicing in rural Vermont for decades, is worried that there is simply too much already for pediatricians to know and do to be able to add extensive mental health care. “There is so much to know in general peds [pediatrics] that I would guess a year or more of additional residency and experience would adequately prepare me to take this on,” he said in an interview. In comparing psychiatric care to other specialties, Dr. Nasca went on to say that, “I would not presume to treat chronic renal failure without the help of a nephrologist or a dilated aortic arch without a cardiologist.”

In a similar vein, however, it also is true that a significant percentage of children presenting to pediatricians for orthopedic problems, infections, asthma, and rashes are managed without referrals to specialists. The right balance, of course, will vary from clinician to clinician based on that pediatrician’s level of interest, experience, and available resources in the community. The AAP position papers don’t mandate or even encourage the notion that all pediatricians need to be at the same place when it comes to competency in assessment and treatment of mental health problems, although it is probably fair to say that there is a push for the pediatric community as a whole to raise the collective bar at least a notch or two.

In response, the mental health community has moved to support the primary care community in their expanded role. These efforts have taken many forms, most notably the model of integrated care, in which mental health clinicians of various types see patients in primary care offices rather than making patients come to them. There also are new consultation programs that provide easy access to a child psychiatrist or other mental health professional for case-related questions delivered by phone, email, or for single in-person consultations. Additional training and educational offerings also are now available for pediatricians either in training and for those already in practice. These initiatives are bolstered by research showing that, not only can good mental health care be delivered in pediatric settings, but there are cost savings that can be realized, particularly for nonpsychiatric medical care.² Despite these promising leads, however, there will remain some for whom anything less than the increased availability of a psychiatrist to “take over” a patient’s mental health care will be seen as a falling short of the clinical need.

To illustrate how things have and continue to change, consider the following three common clinical scenarios that generally present to a pediatrician:

• New presentation of ADHD symptoms.
• Anxiety or obsessive-compulsive problems.
• Return of a patient who has been psychiatrically hospitalized and now is taking multiple medications.

In the past, all three cases often would have resulted in a referral to a psychiatrist. Today, however, it is quite likely that only one of these cases would be referred because ADHD could be well diagnosed and managed within the primary care setting, and problems like anxiety and obsessive-compulsive disorder are sent first not to a psychiatrist but to a non-MD psychotherapist.

Moving forward, today’s pediatricians are expected to do more for the mental health care of patients themselves instead of referring to a psychiatrist. Most already do, despite having had little in the way of formal training. As the evidence grows that the promotion of mental well-being can be a key to future overall health, as well as to the cost of future health care, there are many reasons to be optimistic that support for pediatricians and collaborative care models for clinicians trying to fulfill these new responsibilities will get only stronger.
 

References

1. Pediatrics. 2019 Nov;144(5). pii: e20192757.

2. Pediatrics. 2019 Jul;144(1). pii: e20183243.
 

Dr. Rettew is a child and adolescent psychiatrist and associate professor of psychiatry and pediatrics at the University of Vermont, Burlington. Follow him on Twitter @PediPsych. Email him at pdnews@mdedge.com. Looking for more mental health training? Attend the 14th annual Child Psychiatry in Primary Care conference in Burlington on May 8, 2020 (http://www.med.uvm.edu/cme/conferences).

Assessment and treatment of many of the more common behavioral disorders in childhood, such as ADHD and anxiety, should be considered within a pediatrician’s scope of practice, a stance made very clear by a recent policy statement published by the American Academy of Pediatrics entitled “Mental health competencies for pediatric practice.”¹ These competencies include medication treatment. As stated in the article, “certain disorders (ADHD, common anxiety disorders, depression), if associated with no more than moderate impairment, are amenable to primary care medication management because there are indicated medications with a well-established safety profile.”

This shift to shared ownership when it comes to mental health care is likely coming from multiple sources, not the least of them being necessity and an acknowledgment that there simply aren’t enough psychiatrists to take over the mental health care of every youth with a diagnosable psychiatric disorder. While the number of child and adolescent psychiatrists remains relatively flat, the youth suicide rate is rising, as are the numbers presenting to emergency departments in crisis – all for reasons still to be fully understood. And these trends all are occurring as the medical community overall is appreciating more and more that good mental health is a cornerstone of all health.

The response from the pediatric community, whether it be because of personal conviction or simply a lack of options, largely has been to step up to the plate and take on these new responsibilities and challenges while trying to get up to speed with the latest information about mental health best practices. Many pediatricians now fully endorse the idea that mental health problems should be considered as their “lane,” within certain boundaries. From my own experience doing evaluations and consultations from area primary care clinicians for over 15 years, the shift is noticeable. The typical patient now coming in has already seen a mental health counselor and tried at least one medication, while evaluations for diagnosis and treatment recommendations for things like uncomplicated and treatment-naive ADHD symptoms, for example, are becoming much more infrequent – although still far from extinct.

Nevertheless, there remain concerns about the extent of these new charges. Joe Nasca, MD, an experienced pediatrician who has been practicing in rural Vermont for decades, is worried that there is simply too much already for pediatricians to know and do to be able to add extensive mental health care. “There is so much to know in general peds [pediatrics] that I would guess a year or more of additional residency and experience would adequately prepare me to take this on,” he said in an interview. In comparing psychiatric care to other specialties, Dr. Nasca went on to say that, “I would not presume to treat chronic renal failure without the help of a nephrologist or a dilated aortic arch without a cardiologist.”

In a similar vein, however, it also is true that a significant percentage of children presenting to pediatricians for orthopedic problems, infections, asthma, and rashes are managed without referrals to specialists. The right balance, of course, will vary from clinician to clinician based on that pediatrician’s level of interest, experience, and available resources in the community. The AAP position papers don’t mandate or even encourage the notion that all pediatricians need to be at the same place when it comes to competency in assessment and treatment of mental health problems, although it is probably fair to say that there is a push for the pediatric community as a whole to raise the collective bar at least a notch or two.

In response, the mental health community has moved to support the primary care community in their expanded role. These efforts have taken many forms, most notably the model of integrated care, in which mental health clinicians of various types see patients in primary care offices rather than making patients come to them. There also are new consultation programs that provide easy access to a child psychiatrist or other mental health professional for case-related questions delivered by phone, email, or for single in-person consultations. Additional training and educational offerings also are now available for pediatricians either in training and for those already in practice. These initiatives are bolstered by research showing that, not only can good mental health care be delivered in pediatric settings, but there are cost savings that can be realized, particularly for nonpsychiatric medical care.² Despite these promising leads, however, there will remain some for whom anything less than the increased availability of a psychiatrist to “take over” a patient’s mental health care will be seen as a falling short of the clinical need.

To illustrate how things have and continue to change, consider the following three common clinical scenarios that generally present to a pediatrician:

• New presentation of ADHD symptoms.
• Anxiety or obsessive-compulsive problems.
• Return of a patient who has been psychiatrically hospitalized and now is taking multiple medications.

In the past, all three cases often would have resulted in a referral to a psychiatrist. Today, however, it is quite likely that only one of these cases would be referred because ADHD could be well diagnosed and managed within the primary care setting, and problems like anxiety and obsessive-compulsive disorder are sent first not to a psychiatrist but to a non-MD psychotherapist.

Moving forward, today’s pediatricians are expected to do more for the mental health care of patients themselves instead of referring to a psychiatrist. Most already do, despite having had little in the way of formal training. As the evidence grows that the promotion of mental well-being can be a key to future overall health, as well as to the cost of future health care, there are many reasons to be optimistic that support for pediatricians and collaborative care models for clinicians trying to fulfill these new responsibilities will get only stronger.
 

References

1. Pediatrics. 2019 Nov;144(5). pii: e20192757.

2. Pediatrics. 2019 Jul;144(1). pii: e20183243.
 

Dr. Rettew is a child and adolescent psychiatrist and associate professor of psychiatry and pediatrics at the University of Vermont, Burlington. Follow him on Twitter @PediPsych. Email him at pdnews@mdedge.com. Looking for more mental health training? Attend the 14th annual Child Psychiatry in Primary Care conference in Burlington on May 8, 2020 (http://www.med.uvm.edu/cme/conferences).

Assessment and treatment of many of the more common behavioral disorders in childhood, such as ADHD and anxiety, should be considered within a pediatrician’s scope of practice, a stance made very clear by a recent policy statement published by the American Academy of Pediatrics entitled “Mental health competencies for pediatric practice.”¹ These competencies include medication treatment. As stated in the article, “certain disorders (ADHD, common anxiety disorders, depression), if associated with no more than moderate impairment, are amenable to primary care medication management because there are indicated medications with a well-established safety profile.”

This shift to shared ownership when it comes to mental health care is likely coming from multiple sources, not the least of them being necessity and an acknowledgment that there simply aren’t enough psychiatrists to take over the mental health care of every youth with a diagnosable psychiatric disorder. While the number of child and adolescent psychiatrists remains relatively flat, the youth suicide rate is rising, as are the numbers presenting to emergency departments in crisis – all for reasons still to be fully understood. And these trends all are occurring as the medical community overall is appreciating more and more that good mental health is a cornerstone of all health.

The response from the pediatric community, whether it be because of personal conviction or simply a lack of options, largely has been to step up to the plate and take on these new responsibilities and challenges while trying to get up to speed with the latest information about mental health best practices. Many pediatricians now fully endorse the idea that mental health problems should be considered as their “lane,” within certain boundaries. From my own experience doing evaluations and consultations from area primary care clinicians for over 15 years, the shift is noticeable. The typical patient now coming in has already seen a mental health counselor and tried at least one medication, while evaluations for diagnosis and treatment recommendations for things like uncomplicated and treatment-naive ADHD symptoms, for example, are becoming much more infrequent – although still far from extinct.

Nevertheless, there remain concerns about the extent of these new charges. Joe Nasca, MD, an experienced pediatrician who has been practicing in rural Vermont for decades, is worried that there is simply too much already for pediatricians to know and do to be able to add extensive mental health care. “There is so much to know in general peds [pediatrics] that I would guess a year or more of additional residency and experience would adequately prepare me to take this on,” he said in an interview. In comparing psychiatric care to other specialties, Dr. Nasca went on to say that, “I would not presume to treat chronic renal failure without the help of a nephrologist or a dilated aortic arch without a cardiologist.”

In a similar vein, however, it also is true that a significant percentage of children presenting to pediatricians for orthopedic problems, infections, asthma, and rashes are managed without referrals to specialists. The right balance, of course, will vary from clinician to clinician based on that pediatrician’s level of interest, experience, and available resources in the community. The AAP position papers don’t mandate or even encourage the notion that all pediatricians need to be at the same place when it comes to competency in assessment and treatment of mental health problems, although it is probably fair to say that there is a push for the pediatric community as a whole to raise the collective bar at least a notch or two.

In response, the mental health community has moved to support the primary care community in their expanded role. These efforts have taken many forms, most notably the model of integrated care, in which mental health clinicians of various types see patients in primary care offices rather than making patients come to them. There also are new consultation programs that provide easy access to a child psychiatrist or other mental health professional for case-related questions delivered by phone, email, or for single in-person consultations. Additional training and educational offerings also are now available for pediatricians either in training and for those already in practice. These initiatives are bolstered by research showing that, not only can good mental health care be delivered in pediatric settings, but there are cost savings that can be realized, particularly for nonpsychiatric medical care.² Despite these promising leads, however, there will remain some for whom anything less than the increased availability of a psychiatrist to “take over” a patient’s mental health care will be seen as a falling short of the clinical need.

To illustrate how things have and continue to change, consider the following three common clinical scenarios that generally present to a pediatrician:

• New presentation of ADHD symptoms.
• Anxiety or obsessive-compulsive problems.
• Return of a patient who has been psychiatrically hospitalized and now is taking multiple medications.

In the past, all three cases often would have resulted in a referral to a psychiatrist. Today, however, it is quite likely that only one of these cases would be referred because ADHD could be well diagnosed and managed within the primary care setting, and problems like anxiety and obsessive-compulsive disorder are sent first not to a psychiatrist but to a non-MD psychotherapist.

Moving forward, today’s pediatricians are expected to do more for the mental health care of patients themselves instead of referring to a psychiatrist. Most already do, despite having had little in the way of formal training. As the evidence grows that the promotion of mental well-being can be a key to future overall health, as well as to the cost of future health care, there are many reasons to be optimistic that support for pediatricians and collaborative care models for clinicians trying to fulfill these new responsibilities will get only stronger.
 

References

1. Pediatrics. 2019 Nov;144(5). pii: e20192757.

2. Pediatrics. 2019 Jul;144(1). pii: e20183243.
 

Dr. Rettew is a child and adolescent psychiatrist and associate professor of psychiatry and pediatrics at the University of Vermont, Burlington. Follow him on Twitter @PediPsych. Email him at pdnews@mdedge.com. Looking for more mental health training? Attend the 14th annual Child Psychiatry in Primary Care conference in Burlington on May 8, 2020 (http://www.med.uvm.edu/cme/conferences).