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or its risk factors with the goal of improving visual acuity, publishing its final recommendation statement and evidence summary online Sept. 5 in JAMA.
A review of the latest evidence supports a B recommendation for vision screening at least once in children aged 3-5 years, but the evidence is insufficient to determine the balance of risks and benefits for vision screening in children younger than 3 years (meriting an I statement from the USPSTF). The recommendation updates the 2011 USPSTF recommendation, which also recommended vision screening for children aged 3-5 years with a B recommendation.
“The prevalence of amblyopia, strabismus, and anisometropia ranges from 1% to 6% among children younger than 6 years in the United States,” which can lead to permanent vision loss if left untreated, chair and corresponding author David C. Grossman, MD, of Kaiser Permanente Washington Health Research Institute, Seattle, and colleagues noted in the recommendation statement (JAMA. 2017;318:836-44).
The USPSTF found “inadequate evidence that treatment reduced the incidence of long-term amblyopia or improved school performance, functioning, or quality of life.” However, the USPSTF concluded that the harms of screening and treating preschool children for amblyopia and its risk factors were small, and that treatment improved visual acuity, “which is likely to result in permanent improvements throughout life.”
The benefits of early treatment were characterized as moderate because of the risk of permanent, uncorrectable vision loss associated with untreated amblyopia, “and the benefits of screening and treatment can be experienced over a child’s lifetime,” the researchers said.
The evidence report accompanying the recommendations contained data from 40 studies with 34,709 participants, and addressed issues including the benefits of screening, accuracy of vision screening tests, and the potential harms and benefits of treatments including eye patches and glasses (JAMA. 2017;318:845-58).
“Studies directly evaluating the effectiveness of screening were limited and do not establish whether vision screening in preschool children is better than no screening,” Daniel E. Jonas, MD, of RTI International–University of North Carolina at Chapel Hill Evidence-based Practice Center and his colleagues wrote in the evidence report.
Therefore, the Task Force called for additional research while recommending at least one screening.
The study was funded by the Agency for Healthcare Research and Quality. The researchers had no financial conflicts to disclose.
Read the complete recommendations online at http://www.uspreventiveservicestaskforce.org.
or its risk factors with the goal of improving visual acuity, publishing its final recommendation statement and evidence summary online Sept. 5 in JAMA.
A review of the latest evidence supports a B recommendation for vision screening at least once in children aged 3-5 years, but the evidence is insufficient to determine the balance of risks and benefits for vision screening in children younger than 3 years (meriting an I statement from the USPSTF). The recommendation updates the 2011 USPSTF recommendation, which also recommended vision screening for children aged 3-5 years with a B recommendation.
“The prevalence of amblyopia, strabismus, and anisometropia ranges from 1% to 6% among children younger than 6 years in the United States,” which can lead to permanent vision loss if left untreated, chair and corresponding author David C. Grossman, MD, of Kaiser Permanente Washington Health Research Institute, Seattle, and colleagues noted in the recommendation statement (JAMA. 2017;318:836-44).
The USPSTF found “inadequate evidence that treatment reduced the incidence of long-term amblyopia or improved school performance, functioning, or quality of life.” However, the USPSTF concluded that the harms of screening and treating preschool children for amblyopia and its risk factors were small, and that treatment improved visual acuity, “which is likely to result in permanent improvements throughout life.”
The benefits of early treatment were characterized as moderate because of the risk of permanent, uncorrectable vision loss associated with untreated amblyopia, “and the benefits of screening and treatment can be experienced over a child’s lifetime,” the researchers said.
The evidence report accompanying the recommendations contained data from 40 studies with 34,709 participants, and addressed issues including the benefits of screening, accuracy of vision screening tests, and the potential harms and benefits of treatments including eye patches and glasses (JAMA. 2017;318:845-58).
“Studies directly evaluating the effectiveness of screening were limited and do not establish whether vision screening in preschool children is better than no screening,” Daniel E. Jonas, MD, of RTI International–University of North Carolina at Chapel Hill Evidence-based Practice Center and his colleagues wrote in the evidence report.
Therefore, the Task Force called for additional research while recommending at least one screening.
The study was funded by the Agency for Healthcare Research and Quality. The researchers had no financial conflicts to disclose.
Read the complete recommendations online at http://www.uspreventiveservicestaskforce.org.
or its risk factors with the goal of improving visual acuity, publishing its final recommendation statement and evidence summary online Sept. 5 in JAMA.
A review of the latest evidence supports a B recommendation for vision screening at least once in children aged 3-5 years, but the evidence is insufficient to determine the balance of risks and benefits for vision screening in children younger than 3 years (meriting an I statement from the USPSTF). The recommendation updates the 2011 USPSTF recommendation, which also recommended vision screening for children aged 3-5 years with a B recommendation.
“The prevalence of amblyopia, strabismus, and anisometropia ranges from 1% to 6% among children younger than 6 years in the United States,” which can lead to permanent vision loss if left untreated, chair and corresponding author David C. Grossman, MD, of Kaiser Permanente Washington Health Research Institute, Seattle, and colleagues noted in the recommendation statement (JAMA. 2017;318:836-44).
The USPSTF found “inadequate evidence that treatment reduced the incidence of long-term amblyopia or improved school performance, functioning, or quality of life.” However, the USPSTF concluded that the harms of screening and treating preschool children for amblyopia and its risk factors were small, and that treatment improved visual acuity, “which is likely to result in permanent improvements throughout life.”
The benefits of early treatment were characterized as moderate because of the risk of permanent, uncorrectable vision loss associated with untreated amblyopia, “and the benefits of screening and treatment can be experienced over a child’s lifetime,” the researchers said.
The evidence report accompanying the recommendations contained data from 40 studies with 34,709 participants, and addressed issues including the benefits of screening, accuracy of vision screening tests, and the potential harms and benefits of treatments including eye patches and glasses (JAMA. 2017;318:845-58).
“Studies directly evaluating the effectiveness of screening were limited and do not establish whether vision screening in preschool children is better than no screening,” Daniel E. Jonas, MD, of RTI International–University of North Carolina at Chapel Hill Evidence-based Practice Center and his colleagues wrote in the evidence report.
Therefore, the Task Force called for additional research while recommending at least one screening.
The study was funded by the Agency for Healthcare Research and Quality. The researchers had no financial conflicts to disclose.
Read the complete recommendations online at http://www.uspreventiveservicestaskforce.org.
FROM JAMA
In California, medical vaccine exemptions tripled after personal belief exemption ban
The proportion of California kindergartners with medical exemptions from vaccination tripled after the state eliminated personal belief exemptions, a study has shown.
Furthermore, California counties that previously had the highest rates of personal belief exemptions now have the highest rates of medical exemptions, Paul L. Delamater, PhD, of the University of North Carolina at Chapel Hill, and his associates reported in a research letter in JAMA. Such trends undermine California Senate Bill 277, will “limit [the law’s] long-term benefits, if sustained,” and could lead to outbreaks of vaccine-preventable diseases in the near future, the researchers warned.
Beginning in fall 2016, SB 277 prohibited unvaccinated kindergartners from matriculating at a public or private school in California without a medical exemption from vaccination, having eliminated the personal belief exemption from the state’s school-entry vaccine requirements. But the new law also gave physicians broader discretion to grant the medical exemptions, prompting concerns that “vaccine-hesitant” parents might successfully obtain medical exemptions in lieu of personal belief exemptions. To explore that possibility, the researchers analyzed data from the California state health department on kindergarten enrollment, vaccination, and vaccination exemptions. These data covered about 95% of California kindergartners, Dr. Delamater and his associates noted (JAMA. 2017;318[9]:863-4).
At the county level, rates of medical exemptions strongly correlated with historic rates of personal belief exemptions (P less than .001). Thus, counties with the highest historic rates of personal belief exemptions had the largest increases in rates of medical exemptions in 2016, that is, a change of between –1.00% and 3.38% of kindergartners. Furthermore, vaccination rates among all elementary school-aged children in California are even lower because SB 277 permitted children who previously entered kindergarten with personal belief exemptions to continue attending school without receiving vaccines until seventh grade. “Because the largest increases in [medical exemption] percentage occurred in regions with high past–[personal belief exemption] use, portions of California may remain susceptible to vaccine-preventable disease outbreaks in the near future,” Dr. Delamater and his associates concluded.
The investigators reported having no conflicts of interest.
The proportion of California kindergartners with medical exemptions from vaccination tripled after the state eliminated personal belief exemptions, a study has shown.
Furthermore, California counties that previously had the highest rates of personal belief exemptions now have the highest rates of medical exemptions, Paul L. Delamater, PhD, of the University of North Carolina at Chapel Hill, and his associates reported in a research letter in JAMA. Such trends undermine California Senate Bill 277, will “limit [the law’s] long-term benefits, if sustained,” and could lead to outbreaks of vaccine-preventable diseases in the near future, the researchers warned.
Beginning in fall 2016, SB 277 prohibited unvaccinated kindergartners from matriculating at a public or private school in California without a medical exemption from vaccination, having eliminated the personal belief exemption from the state’s school-entry vaccine requirements. But the new law also gave physicians broader discretion to grant the medical exemptions, prompting concerns that “vaccine-hesitant” parents might successfully obtain medical exemptions in lieu of personal belief exemptions. To explore that possibility, the researchers analyzed data from the California state health department on kindergarten enrollment, vaccination, and vaccination exemptions. These data covered about 95% of California kindergartners, Dr. Delamater and his associates noted (JAMA. 2017;318[9]:863-4).
At the county level, rates of medical exemptions strongly correlated with historic rates of personal belief exemptions (P less than .001). Thus, counties with the highest historic rates of personal belief exemptions had the largest increases in rates of medical exemptions in 2016, that is, a change of between –1.00% and 3.38% of kindergartners. Furthermore, vaccination rates among all elementary school-aged children in California are even lower because SB 277 permitted children who previously entered kindergarten with personal belief exemptions to continue attending school without receiving vaccines until seventh grade. “Because the largest increases in [medical exemption] percentage occurred in regions with high past–[personal belief exemption] use, portions of California may remain susceptible to vaccine-preventable disease outbreaks in the near future,” Dr. Delamater and his associates concluded.
The investigators reported having no conflicts of interest.
The proportion of California kindergartners with medical exemptions from vaccination tripled after the state eliminated personal belief exemptions, a study has shown.
Furthermore, California counties that previously had the highest rates of personal belief exemptions now have the highest rates of medical exemptions, Paul L. Delamater, PhD, of the University of North Carolina at Chapel Hill, and his associates reported in a research letter in JAMA. Such trends undermine California Senate Bill 277, will “limit [the law’s] long-term benefits, if sustained,” and could lead to outbreaks of vaccine-preventable diseases in the near future, the researchers warned.
Beginning in fall 2016, SB 277 prohibited unvaccinated kindergartners from matriculating at a public or private school in California without a medical exemption from vaccination, having eliminated the personal belief exemption from the state’s school-entry vaccine requirements. But the new law also gave physicians broader discretion to grant the medical exemptions, prompting concerns that “vaccine-hesitant” parents might successfully obtain medical exemptions in lieu of personal belief exemptions. To explore that possibility, the researchers analyzed data from the California state health department on kindergarten enrollment, vaccination, and vaccination exemptions. These data covered about 95% of California kindergartners, Dr. Delamater and his associates noted (JAMA. 2017;318[9]:863-4).
At the county level, rates of medical exemptions strongly correlated with historic rates of personal belief exemptions (P less than .001). Thus, counties with the highest historic rates of personal belief exemptions had the largest increases in rates of medical exemptions in 2016, that is, a change of between –1.00% and 3.38% of kindergartners. Furthermore, vaccination rates among all elementary school-aged children in California are even lower because SB 277 permitted children who previously entered kindergarten with personal belief exemptions to continue attending school without receiving vaccines until seventh grade. “Because the largest increases in [medical exemption] percentage occurred in regions with high past–[personal belief exemption] use, portions of California may remain susceptible to vaccine-preventable disease outbreaks in the near future,” Dr. Delamater and his associates concluded.
The investigators reported having no conflicts of interest.
FROM JAMA
Key clinical point:
Major finding: In 2016, 0.51% of California kindergartners had medical exemptions, a threefold rise from 2015.
Data source: An analysis of reportable state health department data from 2001 to 2016.
Disclosures: The investigators reported having no conflicts of interest.
Midyear formulary changes wreak havoc on diabetes care
Instability of formulary coverage for drug and insulin can make it tough to treat patients with diabetes, and the problem is getting worse.
Just one example: CVS Caremark removed 22 insulin products and drugs to treat diabetes in its July 2017 formulary update.
“This is a real problem,” Philip Levy, MD, an endocrinologist at Banner University Medical Group in Phoenix, said in an interview. “We have people that are doing well on one medication and then all of a sudden, their insurance carrier says you have to use a different medication and they don’t tolerate it. It’s a real problem.”
Claresa Levetan, MD, with Chestnut Hill Endocrinology, Diabetes & Metabolic Associates of Philadelphia, agreed. “It’s a very big problem. ... In my practice, it’s a nightmare because I cannot choose to put them on the insulin or the medications that would be best for them.”
The American Diabetes Association also is concerned. The association is “deeply concerned with recent trends in prescription drug formulary designs that result in frequent changes in drug coverage for individuals with diabetes,” according to a statement.
The situation is made more challenging by the sheer volume of money spent on insulins and diabetes drugs.
Diabetes prescription ranked first among therapeutic classes in terms of total spend (13%), ahead of autoimmune (11.9%) and multiple sclerosis and HIV treatments (both tied at 4.7% each) for its commercial clients, according to pharmacy benefit manager (PBM) Prime Therapeutics. Three of the top 10 drugs by drug spend are diabetes medication (No. 4: Lantus; No. 7: NovoLog; and No. 8: Victoza). For its Medicare Part D clients, diabetes drugs also topped the list in terms of total spend per therapeutic class (14.2%), ahead of oral cancer therapies (11.3%) and respiratory (6.5%). Three of the top 10 drugs by drug spend among Medicare Part D clients are diabetes drugs (No. 4: Lantus; No. 6: Januvia; and No. 10: Humalog KwikPen).
Express Scripts reports similar trends. Diabetes prescriptions were the second highest therapeutic class in terms of per member per year spending in 2016 at $108.80, behind inflammatory conditions ($118.21) and ahead of oncology ($60.70).
Utilization of diabetes drugs increased 5.3%, according to the PBM, while unit costs for these treatments rose 14.1%. Express Scripts identified the top five most costly medications as Lantus, Humalog KwikPen, metformin, Januvia, and Invokana.
“Formulary changes made during the year are particularly troubling because most individuals do not have the option of changing to a different insurance plan that would cover his/her prescription medication,” the ADA said in its statement. “Therefore, the association is strongly opposed to formulary changes such as removing medications from formularies or moving medications to a higher tier during the plan year.”
Beyond cost, Dr. Levetan suggested that another issue is the lack of clinician input at the PBM level.
“I think the problem is not only just companies switching to a cheaper bid mid-year between [manufacturers], but there is really no input from physicians, especially the endocrinologists and the specialists,” she said. “This is potentially life threatening.”
Finding alternatives can also have adverse effects on outcomes and can also be time consuming.
“If somebody is doing well, there is no real reason to change that or to stop it,” Dr. Levy said. “There are some medications that are pretty interchangeable, but others, they all have slightly different side effects and we try to use what we think is the best medication for that patient based on the patient’s characteristics.”
ADA is recommending that PBMs, insurance plans, and employers “provide an expedited and standard process for gaining access to medications not included in the plan’s formulary. Responses to exception requests must be timely to ensure no delay in obtaining a needed medication, thereby preventing a gap in treatment. In addition, the association recommends PBMs, plans, and employers temporarily cover nonformulary drugs as if they are on formulary during the first 30 days after a formulary list is changed.”
Instability of formulary coverage for drug and insulin can make it tough to treat patients with diabetes, and the problem is getting worse.
Just one example: CVS Caremark removed 22 insulin products and drugs to treat diabetes in its July 2017 formulary update.
“This is a real problem,” Philip Levy, MD, an endocrinologist at Banner University Medical Group in Phoenix, said in an interview. “We have people that are doing well on one medication and then all of a sudden, their insurance carrier says you have to use a different medication and they don’t tolerate it. It’s a real problem.”
Claresa Levetan, MD, with Chestnut Hill Endocrinology, Diabetes & Metabolic Associates of Philadelphia, agreed. “It’s a very big problem. ... In my practice, it’s a nightmare because I cannot choose to put them on the insulin or the medications that would be best for them.”
The American Diabetes Association also is concerned. The association is “deeply concerned with recent trends in prescription drug formulary designs that result in frequent changes in drug coverage for individuals with diabetes,” according to a statement.
The situation is made more challenging by the sheer volume of money spent on insulins and diabetes drugs.
Diabetes prescription ranked first among therapeutic classes in terms of total spend (13%), ahead of autoimmune (11.9%) and multiple sclerosis and HIV treatments (both tied at 4.7% each) for its commercial clients, according to pharmacy benefit manager (PBM) Prime Therapeutics. Three of the top 10 drugs by drug spend are diabetes medication (No. 4: Lantus; No. 7: NovoLog; and No. 8: Victoza). For its Medicare Part D clients, diabetes drugs also topped the list in terms of total spend per therapeutic class (14.2%), ahead of oral cancer therapies (11.3%) and respiratory (6.5%). Three of the top 10 drugs by drug spend among Medicare Part D clients are diabetes drugs (No. 4: Lantus; No. 6: Januvia; and No. 10: Humalog KwikPen).
Express Scripts reports similar trends. Diabetes prescriptions were the second highest therapeutic class in terms of per member per year spending in 2016 at $108.80, behind inflammatory conditions ($118.21) and ahead of oncology ($60.70).
Utilization of diabetes drugs increased 5.3%, according to the PBM, while unit costs for these treatments rose 14.1%. Express Scripts identified the top five most costly medications as Lantus, Humalog KwikPen, metformin, Januvia, and Invokana.
“Formulary changes made during the year are particularly troubling because most individuals do not have the option of changing to a different insurance plan that would cover his/her prescription medication,” the ADA said in its statement. “Therefore, the association is strongly opposed to formulary changes such as removing medications from formularies or moving medications to a higher tier during the plan year.”
Beyond cost, Dr. Levetan suggested that another issue is the lack of clinician input at the PBM level.
“I think the problem is not only just companies switching to a cheaper bid mid-year between [manufacturers], but there is really no input from physicians, especially the endocrinologists and the specialists,” she said. “This is potentially life threatening.”
Finding alternatives can also have adverse effects on outcomes and can also be time consuming.
“If somebody is doing well, there is no real reason to change that or to stop it,” Dr. Levy said. “There are some medications that are pretty interchangeable, but others, they all have slightly different side effects and we try to use what we think is the best medication for that patient based on the patient’s characteristics.”
ADA is recommending that PBMs, insurance plans, and employers “provide an expedited and standard process for gaining access to medications not included in the plan’s formulary. Responses to exception requests must be timely to ensure no delay in obtaining a needed medication, thereby preventing a gap in treatment. In addition, the association recommends PBMs, plans, and employers temporarily cover nonformulary drugs as if they are on formulary during the first 30 days after a formulary list is changed.”
Instability of formulary coverage for drug and insulin can make it tough to treat patients with diabetes, and the problem is getting worse.
Just one example: CVS Caremark removed 22 insulin products and drugs to treat diabetes in its July 2017 formulary update.
“This is a real problem,” Philip Levy, MD, an endocrinologist at Banner University Medical Group in Phoenix, said in an interview. “We have people that are doing well on one medication and then all of a sudden, their insurance carrier says you have to use a different medication and they don’t tolerate it. It’s a real problem.”
Claresa Levetan, MD, with Chestnut Hill Endocrinology, Diabetes & Metabolic Associates of Philadelphia, agreed. “It’s a very big problem. ... In my practice, it’s a nightmare because I cannot choose to put them on the insulin or the medications that would be best for them.”
The American Diabetes Association also is concerned. The association is “deeply concerned with recent trends in prescription drug formulary designs that result in frequent changes in drug coverage for individuals with diabetes,” according to a statement.
The situation is made more challenging by the sheer volume of money spent on insulins and diabetes drugs.
Diabetes prescription ranked first among therapeutic classes in terms of total spend (13%), ahead of autoimmune (11.9%) and multiple sclerosis and HIV treatments (both tied at 4.7% each) for its commercial clients, according to pharmacy benefit manager (PBM) Prime Therapeutics. Three of the top 10 drugs by drug spend are diabetes medication (No. 4: Lantus; No. 7: NovoLog; and No. 8: Victoza). For its Medicare Part D clients, diabetes drugs also topped the list in terms of total spend per therapeutic class (14.2%), ahead of oral cancer therapies (11.3%) and respiratory (6.5%). Three of the top 10 drugs by drug spend among Medicare Part D clients are diabetes drugs (No. 4: Lantus; No. 6: Januvia; and No. 10: Humalog KwikPen).
Express Scripts reports similar trends. Diabetes prescriptions were the second highest therapeutic class in terms of per member per year spending in 2016 at $108.80, behind inflammatory conditions ($118.21) and ahead of oncology ($60.70).
Utilization of diabetes drugs increased 5.3%, according to the PBM, while unit costs for these treatments rose 14.1%. Express Scripts identified the top five most costly medications as Lantus, Humalog KwikPen, metformin, Januvia, and Invokana.
“Formulary changes made during the year are particularly troubling because most individuals do not have the option of changing to a different insurance plan that would cover his/her prescription medication,” the ADA said in its statement. “Therefore, the association is strongly opposed to formulary changes such as removing medications from formularies or moving medications to a higher tier during the plan year.”
Beyond cost, Dr. Levetan suggested that another issue is the lack of clinician input at the PBM level.
“I think the problem is not only just companies switching to a cheaper bid mid-year between [manufacturers], but there is really no input from physicians, especially the endocrinologists and the specialists,” she said. “This is potentially life threatening.”
Finding alternatives can also have adverse effects on outcomes and can also be time consuming.
“If somebody is doing well, there is no real reason to change that or to stop it,” Dr. Levy said. “There are some medications that are pretty interchangeable, but others, they all have slightly different side effects and we try to use what we think is the best medication for that patient based on the patient’s characteristics.”
ADA is recommending that PBMs, insurance plans, and employers “provide an expedited and standard process for gaining access to medications not included in the plan’s formulary. Responses to exception requests must be timely to ensure no delay in obtaining a needed medication, thereby preventing a gap in treatment. In addition, the association recommends PBMs, plans, and employers temporarily cover nonformulary drugs as if they are on formulary during the first 30 days after a formulary list is changed.”
Withdrawn AML drug back on market in US
The US Food and Drug Administration (FDA) has approved use of gemtuzumab ozogamicin (GO, Mylotarg), a treatment that was initially approved by the agency in 2000 but later pulled from the US market.
GO is an antibody-drug conjugate that consists of the cytotoxic agent calicheamicin attached to a monoclonal antibody targeting CD33.
GO is now approved to treat adults with newly diagnosed, CD33-positive acute myeloid leukemia (AML) and patients age 2 and older with CD33-positive, relapsed or refractory AML.
GO can be given alone or in combination with daunorubicin and cytarabine.
The prescribing information for GO includes a boxed warning detailing the risk of hepatotoxicity, including veno-occlusive disease or sinusoidal obstruction syndrome, associated with GO.
GO originates from a collaboration between Pfizer and Celltech, now UCB. Pfizer has sole responsibility for all manufacturing, clinical development, and commercialization activities for this molecule.
Market withdrawal and subsequent trials
GO was originally approved under the FDA’s accelerated approval program in 2000 for use as a single agent in patients with CD33-positive AML who had experienced their first relapse and were 60 years of age or older.
In 2010, Pfizer voluntarily withdrew GO from the US market due to the results of a confirmatory phase 3 trial, SWOG S0106.
This trial showed there was no clinical benefit for patients who received GO plus daunorubicin and cytarabine over patients who received only daunorubicin and cytarabine.
In addition, the rate of fatal, treatment-related toxicity was significantly higher in the GO arm of the study.
Because of the unmet need for effective treatments in AML, investigators expressed an interest in evaluating different doses and schedules of GO.
These independent investigators, with Pfizer’s support, conducted clinical trials that yielded more information on the efficacy and safety of GO.
The trials—ALFA-0701, AML-19, and MyloFrance-1—supported the new approval of GO. Updated data from these trials are included in the prescribing information, which is available for download at www.mylotarg.com. 
The US Food and Drug Administration (FDA) has approved use of gemtuzumab ozogamicin (GO, Mylotarg), a treatment that was initially approved by the agency in 2000 but later pulled from the US market.
GO is an antibody-drug conjugate that consists of the cytotoxic agent calicheamicin attached to a monoclonal antibody targeting CD33.
GO is now approved to treat adults with newly diagnosed, CD33-positive acute myeloid leukemia (AML) and patients age 2 and older with CD33-positive, relapsed or refractory AML.
GO can be given alone or in combination with daunorubicin and cytarabine.
The prescribing information for GO includes a boxed warning detailing the risk of hepatotoxicity, including veno-occlusive disease or sinusoidal obstruction syndrome, associated with GO.
GO originates from a collaboration between Pfizer and Celltech, now UCB. Pfizer has sole responsibility for all manufacturing, clinical development, and commercialization activities for this molecule.
Market withdrawal and subsequent trials
GO was originally approved under the FDA’s accelerated approval program in 2000 for use as a single agent in patients with CD33-positive AML who had experienced their first relapse and were 60 years of age or older.
In 2010, Pfizer voluntarily withdrew GO from the US market due to the results of a confirmatory phase 3 trial, SWOG S0106.
This trial showed there was no clinical benefit for patients who received GO plus daunorubicin and cytarabine over patients who received only daunorubicin and cytarabine.
In addition, the rate of fatal, treatment-related toxicity was significantly higher in the GO arm of the study.
Because of the unmet need for effective treatments in AML, investigators expressed an interest in evaluating different doses and schedules of GO.
These independent investigators, with Pfizer’s support, conducted clinical trials that yielded more information on the efficacy and safety of GO.
The trials—ALFA-0701, AML-19, and MyloFrance-1—supported the new approval of GO. Updated data from these trials are included in the prescribing information, which is available for download at www.mylotarg.com.
The US Food and Drug Administration (FDA) has approved use of gemtuzumab ozogamicin (GO, Mylotarg), a treatment that was initially approved by the agency in 2000 but later pulled from the US market.
GO is an antibody-drug conjugate that consists of the cytotoxic agent calicheamicin attached to a monoclonal antibody targeting CD33.
GO is now approved to treat adults with newly diagnosed, CD33-positive acute myeloid leukemia (AML) and patients age 2 and older with CD33-positive, relapsed or refractory AML.
GO can be given alone or in combination with daunorubicin and cytarabine.
The prescribing information for GO includes a boxed warning detailing the risk of hepatotoxicity, including veno-occlusive disease or sinusoidal obstruction syndrome, associated with GO.
GO originates from a collaboration between Pfizer and Celltech, now UCB. Pfizer has sole responsibility for all manufacturing, clinical development, and commercialization activities for this molecule.
Market withdrawal and subsequent trials
GO was originally approved under the FDA’s accelerated approval program in 2000 for use as a single agent in patients with CD33-positive AML who had experienced their first relapse and were 60 years of age or older.
In 2010, Pfizer voluntarily withdrew GO from the US market due to the results of a confirmatory phase 3 trial, SWOG S0106.
This trial showed there was no clinical benefit for patients who received GO plus daunorubicin and cytarabine over patients who received only daunorubicin and cytarabine.
In addition, the rate of fatal, treatment-related toxicity was significantly higher in the GO arm of the study.
Because of the unmet need for effective treatments in AML, investigators expressed an interest in evaluating different doses and schedules of GO.
These independent investigators, with Pfizer’s support, conducted clinical trials that yielded more information on the efficacy and safety of GO.
The trials—ALFA-0701, AML-19, and MyloFrance-1—supported the new approval of GO. Updated data from these trials are included in the prescribing information, which is available for download at www.mylotarg.com.
FDA reapproves gemtuzumab ozogamicin for CD33-positive AML treatment
The Food and Drug Administration has approved gemtuzumab ozogamicin (Mylotarg) for the treatment of newly diagnosed CD33-positive acute myeloid leukemia in adults, according to a press release.
Approval was based on results from three clinical trials. In the first, newly diagnosed AML patients who received gemtuzumab ozogamicin plus chemotherapy had significantly longer event-free survival than did patients who received chemotherapy alone. In a second trial, patients who received gemtuzumab ozogamicin alone had better overall survival compared to those who received only best supportive care. In the third clinical trial, 26% of patients who had experienced a relapse and received gemtuzumab ozogamicin experienced a remission.
Common side effects of gemtuzumab ozogamicin include fever, nausea, infection, vomiting, bleeding, thrombocytopenia, stomatitis, constipation, rash, headache, elevated liver function tests, and neutropenia; it is not recommended for women who are pregnant or breastfeeding.
Gemtuzumab ozogamicin was also approved to treat patients older than 2 years old who have experienced a relapse or have not responded to initial treatment.
“Mylotarg’s history underscores the importance of examining alternative dosing, scheduling, and administration of therapies for patients with cancer, especially in those who may be most vulnerable to the side effects of treatment,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in the press release.
The Food and Drug Administration has approved gemtuzumab ozogamicin (Mylotarg) for the treatment of newly diagnosed CD33-positive acute myeloid leukemia in adults, according to a press release.
Approval was based on results from three clinical trials. In the first, newly diagnosed AML patients who received gemtuzumab ozogamicin plus chemotherapy had significantly longer event-free survival than did patients who received chemotherapy alone. In a second trial, patients who received gemtuzumab ozogamicin alone had better overall survival compared to those who received only best supportive care. In the third clinical trial, 26% of patients who had experienced a relapse and received gemtuzumab ozogamicin experienced a remission.
Common side effects of gemtuzumab ozogamicin include fever, nausea, infection, vomiting, bleeding, thrombocytopenia, stomatitis, constipation, rash, headache, elevated liver function tests, and neutropenia; it is not recommended for women who are pregnant or breastfeeding.
Gemtuzumab ozogamicin was also approved to treat patients older than 2 years old who have experienced a relapse or have not responded to initial treatment.
“Mylotarg’s history underscores the importance of examining alternative dosing, scheduling, and administration of therapies for patients with cancer, especially in those who may be most vulnerable to the side effects of treatment,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in the press release.
The Food and Drug Administration has approved gemtuzumab ozogamicin (Mylotarg) for the treatment of newly diagnosed CD33-positive acute myeloid leukemia in adults, according to a press release.
Approval was based on results from three clinical trials. In the first, newly diagnosed AML patients who received gemtuzumab ozogamicin plus chemotherapy had significantly longer event-free survival than did patients who received chemotherapy alone. In a second trial, patients who received gemtuzumab ozogamicin alone had better overall survival compared to those who received only best supportive care. In the third clinical trial, 26% of patients who had experienced a relapse and received gemtuzumab ozogamicin experienced a remission.
Common side effects of gemtuzumab ozogamicin include fever, nausea, infection, vomiting, bleeding, thrombocytopenia, stomatitis, constipation, rash, headache, elevated liver function tests, and neutropenia; it is not recommended for women who are pregnant or breastfeeding.
Gemtuzumab ozogamicin was also approved to treat patients older than 2 years old who have experienced a relapse or have not responded to initial treatment.
“Mylotarg’s history underscores the importance of examining alternative dosing, scheduling, and administration of therapies for patients with cancer, especially in those who may be most vulnerable to the side effects of treatment,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in the press release.
Young cancer patients want more digital support, survey says
A recent survey of young cancer patients in the UK revealed their desire for additional digital oncology resources.
The survey showed that many of the patients already used digital resources to access information about their cancer.
However, many also expressed a need for additional resources such as online counseling or psychological support.
Esha Abrol, of Camden and Islington NHS Foundation Trust in London, UK, and her colleagues reported these findings in the Journal of Cancer Survivorship.
The researchers surveyed 102 cancer patients, ages 13 to 24, about digital media use.
The patients reported having active digital lives, with 41.6% of them rating digital resources as “essential” to their lives.
The patients reported using a variety of healthcare-related digital resources to access information about their disease, including independent sources and ones recommended to them by the professional team treating them.
Half (51%) of respondents said they kept in contact through digital means with other patients they had met during treatment. Twelve percent contacted others or started new digital relationships with people they had never met in person.
However, most of the patients were still most likely to get information about their treatment from professionals in a face-to-face environment, such as when visiting their doctor.
“This is a reassuring and appropriate finding, as this is the conventional means by which teenage and young adult oncology care is delivered by the multidisciplinary team, whether the young people choose to engage or not,” Abrol said.
At the same time, many survey respondents expressed a desire for virtual online groups (54.3%), online counseling or psychological support (43.5%), and the ability to receive (66.3%) and share (48.9%) clinical information online.
Young adults (ages 19 to 24) were more interested than adolescents (ages 13 to 18) in online counseling options and preferred receiving clinical information online.
The researchers said this may reflect young adults’ greater independence, resilience, breadth of experience in the digital world, and confidence in discussing clinical matters online.
Abrol believes these preliminary results can help inform the development of local, national, and global services to teenagers and young adults with cancer to address their unmet needs.
“These digital support resources have the potential to improve patient experience and engagement for an important subsection of teenagers and young people treated for cancer,” Abrol said.
A recent survey of young cancer patients in the UK revealed their desire for additional digital oncology resources.
The survey showed that many of the patients already used digital resources to access information about their cancer.
However, many also expressed a need for additional resources such as online counseling or psychological support.
Esha Abrol, of Camden and Islington NHS Foundation Trust in London, UK, and her colleagues reported these findings in the Journal of Cancer Survivorship.
The researchers surveyed 102 cancer patients, ages 13 to 24, about digital media use.
The patients reported having active digital lives, with 41.6% of them rating digital resources as “essential” to their lives.
The patients reported using a variety of healthcare-related digital resources to access information about their disease, including independent sources and ones recommended to them by the professional team treating them.
Half (51%) of respondents said they kept in contact through digital means with other patients they had met during treatment. Twelve percent contacted others or started new digital relationships with people they had never met in person.
However, most of the patients were still most likely to get information about their treatment from professionals in a face-to-face environment, such as when visiting their doctor.
“This is a reassuring and appropriate finding, as this is the conventional means by which teenage and young adult oncology care is delivered by the multidisciplinary team, whether the young people choose to engage or not,” Abrol said.
At the same time, many survey respondents expressed a desire for virtual online groups (54.3%), online counseling or psychological support (43.5%), and the ability to receive (66.3%) and share (48.9%) clinical information online.
Young adults (ages 19 to 24) were more interested than adolescents (ages 13 to 18) in online counseling options and preferred receiving clinical information online.
The researchers said this may reflect young adults’ greater independence, resilience, breadth of experience in the digital world, and confidence in discussing clinical matters online.
Abrol believes these preliminary results can help inform the development of local, national, and global services to teenagers and young adults with cancer to address their unmet needs.
“These digital support resources have the potential to improve patient experience and engagement for an important subsection of teenagers and young people treated for cancer,” Abrol said.
A recent survey of young cancer patients in the UK revealed their desire for additional digital oncology resources.
The survey showed that many of the patients already used digital resources to access information about their cancer.
However, many also expressed a need for additional resources such as online counseling or psychological support.
Esha Abrol, of Camden and Islington NHS Foundation Trust in London, UK, and her colleagues reported these findings in the Journal of Cancer Survivorship.
The researchers surveyed 102 cancer patients, ages 13 to 24, about digital media use.
The patients reported having active digital lives, with 41.6% of them rating digital resources as “essential” to their lives.
The patients reported using a variety of healthcare-related digital resources to access information about their disease, including independent sources and ones recommended to them by the professional team treating them.
Half (51%) of respondents said they kept in contact through digital means with other patients they had met during treatment. Twelve percent contacted others or started new digital relationships with people they had never met in person.
However, most of the patients were still most likely to get information about their treatment from professionals in a face-to-face environment, such as when visiting their doctor.
“This is a reassuring and appropriate finding, as this is the conventional means by which teenage and young adult oncology care is delivered by the multidisciplinary team, whether the young people choose to engage or not,” Abrol said.
At the same time, many survey respondents expressed a desire for virtual online groups (54.3%), online counseling or psychological support (43.5%), and the ability to receive (66.3%) and share (48.9%) clinical information online.
Young adults (ages 19 to 24) were more interested than adolescents (ages 13 to 18) in online counseling options and preferred receiving clinical information online.
The researchers said this may reflect young adults’ greater independence, resilience, breadth of experience in the digital world, and confidence in discussing clinical matters online.
Abrol believes these preliminary results can help inform the development of local, national, and global services to teenagers and young adults with cancer to address their unmet needs.
“These digital support resources have the potential to improve patient experience and engagement for an important subsection of teenagers and young people treated for cancer,” Abrol said.
Developing vaccines against enterovirus-A71 called a priority
MADRID – Is there a need for an enterovirus-A71 vaccine?
This is a new question for North American and European physicians, but not so new in Asia.
“China says yes, with more than 15 million cases of hand, foot, and mouth disease resulting in 3,500 deaths since surveillance started in 2009,” Heli Harvala, MD, said at the annual meeting of the European Society for Paediatric Infectious Diseases.
Seroconversion rates 28 days after the second dose of these vaccines, both directed specifically against viral subgenotype C-4, are 92%-100%. Vaccine efficacy is 91%-97%, according to Dr. Harvala, a consultant medical virologist at University College London.
It remains a mystery why major outbreaks of severe EV-A71 disease have mostly occurred in Asia, with the notable exception of a Spanish outbreak of EV-A71 encephalitis in 2016. The possibility of much wider spread is concerning.
The Chinese monovalent EV-A71 vaccines, however, are seen as a stopgap. For one thing, recent evidence suggests that it’s probably not the specific EV-A71 C-4 viral subgenotype that accounts for all severe disease.
“I think we have to aim for a multivalent vaccine,” Dr. Harvala said.
Now in clinical trials, investigational bivalent vaccines are directed against other EV-A71 subgenotypes in addition to C-4, and also against another enterovirus, coxsackievirus serotype A16, the most common cause of classic hand, foot, and mouth disease in the United States. But that’s probably not enough, according to Dr. Harvala. She noted that coxsackievirus A6, which was first identified more than 50 years ago, abruptly became the main cause of mild hand, foot, and mouth disease in China in 2013 and again in 2015. Moreover, its role in severe cases is growing, and there have been important outbreaks in the United States in recent years. These severe cases come in three main presentations, resembling either erythema multiforme, chicken pox, or eczema herpeticum.
Dr. Harvala added that a next-generation vaccine probably also should offer protection against enterovirus-D68. In 2014, there were 1,153 laboratory-confirmed EV-D68 infections and 14 deaths in the United States and Canada. This infection poses a diagnostic challenge: while the virus is readily detectable on throat swabs, it’s only rarely present in stool or cerebrospinal fluid samples.
“It’s important to keep in mind that this infection is still underdiagnosed. We are not really looking for it,” she said.
No specific treatment for enterovirus infections is available. Three capsid-binding antiviral agents now are in clinical trials: pleconaril, vapendavir, and pocapavir. In addition, translational studies have demonstrated that the SSRI fluoxetine inhibits enterovirus replication, but there have been no clinical trials as yet.
Although development of antivirals effective against enterovirus is an active area of research, Dr. Harvala thinks drug resistance will be an issue, underscoring the importance of vaccine development.
She reported having no financial conflicts of interest regarding her presentation.
MADRID – Is there a need for an enterovirus-A71 vaccine?
This is a new question for North American and European physicians, but not so new in Asia.
“China says yes, with more than 15 million cases of hand, foot, and mouth disease resulting in 3,500 deaths since surveillance started in 2009,” Heli Harvala, MD, said at the annual meeting of the European Society for Paediatric Infectious Diseases.
Seroconversion rates 28 days after the second dose of these vaccines, both directed specifically against viral subgenotype C-4, are 92%-100%. Vaccine efficacy is 91%-97%, according to Dr. Harvala, a consultant medical virologist at University College London.
It remains a mystery why major outbreaks of severe EV-A71 disease have mostly occurred in Asia, with the notable exception of a Spanish outbreak of EV-A71 encephalitis in 2016. The possibility of much wider spread is concerning.
The Chinese monovalent EV-A71 vaccines, however, are seen as a stopgap. For one thing, recent evidence suggests that it’s probably not the specific EV-A71 C-4 viral subgenotype that accounts for all severe disease.
“I think we have to aim for a multivalent vaccine,” Dr. Harvala said.
Now in clinical trials, investigational bivalent vaccines are directed against other EV-A71 subgenotypes in addition to C-4, and also against another enterovirus, coxsackievirus serotype A16, the most common cause of classic hand, foot, and mouth disease in the United States. But that’s probably not enough, according to Dr. Harvala. She noted that coxsackievirus A6, which was first identified more than 50 years ago, abruptly became the main cause of mild hand, foot, and mouth disease in China in 2013 and again in 2015. Moreover, its role in severe cases is growing, and there have been important outbreaks in the United States in recent years. These severe cases come in three main presentations, resembling either erythema multiforme, chicken pox, or eczema herpeticum.
Dr. Harvala added that a next-generation vaccine probably also should offer protection against enterovirus-D68. In 2014, there were 1,153 laboratory-confirmed EV-D68 infections and 14 deaths in the United States and Canada. This infection poses a diagnostic challenge: while the virus is readily detectable on throat swabs, it’s only rarely present in stool or cerebrospinal fluid samples.
“It’s important to keep in mind that this infection is still underdiagnosed. We are not really looking for it,” she said.
No specific treatment for enterovirus infections is available. Three capsid-binding antiviral agents now are in clinical trials: pleconaril, vapendavir, and pocapavir. In addition, translational studies have demonstrated that the SSRI fluoxetine inhibits enterovirus replication, but there have been no clinical trials as yet.
Although development of antivirals effective against enterovirus is an active area of research, Dr. Harvala thinks drug resistance will be an issue, underscoring the importance of vaccine development.
She reported having no financial conflicts of interest regarding her presentation.
MADRID – Is there a need for an enterovirus-A71 vaccine?
This is a new question for North American and European physicians, but not so new in Asia.
“China says yes, with more than 15 million cases of hand, foot, and mouth disease resulting in 3,500 deaths since surveillance started in 2009,” Heli Harvala, MD, said at the annual meeting of the European Society for Paediatric Infectious Diseases.
Seroconversion rates 28 days after the second dose of these vaccines, both directed specifically against viral subgenotype C-4, are 92%-100%. Vaccine efficacy is 91%-97%, according to Dr. Harvala, a consultant medical virologist at University College London.
It remains a mystery why major outbreaks of severe EV-A71 disease have mostly occurred in Asia, with the notable exception of a Spanish outbreak of EV-A71 encephalitis in 2016. The possibility of much wider spread is concerning.
The Chinese monovalent EV-A71 vaccines, however, are seen as a stopgap. For one thing, recent evidence suggests that it’s probably not the specific EV-A71 C-4 viral subgenotype that accounts for all severe disease.
“I think we have to aim for a multivalent vaccine,” Dr. Harvala said.
Now in clinical trials, investigational bivalent vaccines are directed against other EV-A71 subgenotypes in addition to C-4, and also against another enterovirus, coxsackievirus serotype A16, the most common cause of classic hand, foot, and mouth disease in the United States. But that’s probably not enough, according to Dr. Harvala. She noted that coxsackievirus A6, which was first identified more than 50 years ago, abruptly became the main cause of mild hand, foot, and mouth disease in China in 2013 and again in 2015. Moreover, its role in severe cases is growing, and there have been important outbreaks in the United States in recent years. These severe cases come in three main presentations, resembling either erythema multiforme, chicken pox, or eczema herpeticum.
Dr. Harvala added that a next-generation vaccine probably also should offer protection against enterovirus-D68. In 2014, there were 1,153 laboratory-confirmed EV-D68 infections and 14 deaths in the United States and Canada. This infection poses a diagnostic challenge: while the virus is readily detectable on throat swabs, it’s only rarely present in stool or cerebrospinal fluid samples.
“It’s important to keep in mind that this infection is still underdiagnosed. We are not really looking for it,” she said.
No specific treatment for enterovirus infections is available. Three capsid-binding antiviral agents now are in clinical trials: pleconaril, vapendavir, and pocapavir. In addition, translational studies have demonstrated that the SSRI fluoxetine inhibits enterovirus replication, but there have been no clinical trials as yet.
Although development of antivirals effective against enterovirus is an active area of research, Dr. Harvala thinks drug resistance will be an issue, underscoring the importance of vaccine development.
She reported having no financial conflicts of interest regarding her presentation.
EXPERT ANALYSIS FROM ESPID 2017
The summer job
You probably aren’t surprised to learn that the jobless rate for young people aged 16-24 years has fallen to the lowest rate recorded since 1969. Those “Hiring” signs you see in every storefront tell the story. Although the jobless rate for young people is still twice that for adults, clearly there are jobs out there.
However, it appears that there are fewer young people looking for those jobs. In fact, the decline in what is referred to as the “labor force participation rate” is down significantly to 60.6% from a high point of 77.5% in 1989 (Summer Youth Unemployment Falls to Lowest Level Since 1969, by Eric Morath. Wall Street Journal. 2017 Aug 17).
But it may be that the concept of having a job, particularly a first job, lacks the appeal it did for my generation. While I’m sure my parents would have appreciated any financial contribution I could provide, I felt no direct pressure from them to get a summer job. My mother’s only concern was that without something to do, I would be getting into trouble or hanging around the house and getting in her way. She could easily find me work to do around the house that wasn’t going to be fun or pay me anything.
It was peer pressure that nudged me into working. I had watched my friends and their older siblings reaping the benefits of a summer job – disposable income. Money could buy an old car, pay for insurance and gas, fund dates, and buy 45 rpm records. The money provided some independence. Even the most menial job could allow you to feel a bit more like a grown-up.
While I don’t think it is our job as pediatricians to instill a work ethic in our patients, it doesn’t hurt to encourage those who seem to be at loose ends to consider getting a job. Unfortunately, many of the businesses hiring young people are offering hours that are certainly not schoolwork- and sleep-friendly. And we must caution our patients to avoid making bad compromises when facing the lure of a steady supply of spending money.
I would hate to see us return to the bad old days when children were enslaved in sweat shops, in dangerous and unhealthy working conditions. However, I fear that in some cases, in our zeal to protect young people from unsafe working conditions, we have made so many rules that we have seriously limited the opportunities for them to get a taste of the hands-on technical skills that our country desperately needs. Just try to get a plumber or electrician when you need one, and you will understand what I mean. A summer spent as an electrician’s gofer just might trigger a floundering 13-year-old to invest more energy in his studies when he sees them as a critical step to a well-paying job he would enjoy.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.”
You probably aren’t surprised to learn that the jobless rate for young people aged 16-24 years has fallen to the lowest rate recorded since 1969. Those “Hiring” signs you see in every storefront tell the story. Although the jobless rate for young people is still twice that for adults, clearly there are jobs out there.
However, it appears that there are fewer young people looking for those jobs. In fact, the decline in what is referred to as the “labor force participation rate” is down significantly to 60.6% from a high point of 77.5% in 1989 (Summer Youth Unemployment Falls to Lowest Level Since 1969, by Eric Morath. Wall Street Journal. 2017 Aug 17).
But it may be that the concept of having a job, particularly a first job, lacks the appeal it did for my generation. While I’m sure my parents would have appreciated any financial contribution I could provide, I felt no direct pressure from them to get a summer job. My mother’s only concern was that without something to do, I would be getting into trouble or hanging around the house and getting in her way. She could easily find me work to do around the house that wasn’t going to be fun or pay me anything.
It was peer pressure that nudged me into working. I had watched my friends and their older siblings reaping the benefits of a summer job – disposable income. Money could buy an old car, pay for insurance and gas, fund dates, and buy 45 rpm records. The money provided some independence. Even the most menial job could allow you to feel a bit more like a grown-up.
While I don’t think it is our job as pediatricians to instill a work ethic in our patients, it doesn’t hurt to encourage those who seem to be at loose ends to consider getting a job. Unfortunately, many of the businesses hiring young people are offering hours that are certainly not schoolwork- and sleep-friendly. And we must caution our patients to avoid making bad compromises when facing the lure of a steady supply of spending money.
I would hate to see us return to the bad old days when children were enslaved in sweat shops, in dangerous and unhealthy working conditions. However, I fear that in some cases, in our zeal to protect young people from unsafe working conditions, we have made so many rules that we have seriously limited the opportunities for them to get a taste of the hands-on technical skills that our country desperately needs. Just try to get a plumber or electrician when you need one, and you will understand what I mean. A summer spent as an electrician’s gofer just might trigger a floundering 13-year-old to invest more energy in his studies when he sees them as a critical step to a well-paying job he would enjoy.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.”
You probably aren’t surprised to learn that the jobless rate for young people aged 16-24 years has fallen to the lowest rate recorded since 1969. Those “Hiring” signs you see in every storefront tell the story. Although the jobless rate for young people is still twice that for adults, clearly there are jobs out there.
However, it appears that there are fewer young people looking for those jobs. In fact, the decline in what is referred to as the “labor force participation rate” is down significantly to 60.6% from a high point of 77.5% in 1989 (Summer Youth Unemployment Falls to Lowest Level Since 1969, by Eric Morath. Wall Street Journal. 2017 Aug 17).
But it may be that the concept of having a job, particularly a first job, lacks the appeal it did for my generation. While I’m sure my parents would have appreciated any financial contribution I could provide, I felt no direct pressure from them to get a summer job. My mother’s only concern was that without something to do, I would be getting into trouble or hanging around the house and getting in her way. She could easily find me work to do around the house that wasn’t going to be fun or pay me anything.
It was peer pressure that nudged me into working. I had watched my friends and their older siblings reaping the benefits of a summer job – disposable income. Money could buy an old car, pay for insurance and gas, fund dates, and buy 45 rpm records. The money provided some independence. Even the most menial job could allow you to feel a bit more like a grown-up.
While I don’t think it is our job as pediatricians to instill a work ethic in our patients, it doesn’t hurt to encourage those who seem to be at loose ends to consider getting a job. Unfortunately, many of the businesses hiring young people are offering hours that are certainly not schoolwork- and sleep-friendly. And we must caution our patients to avoid making bad compromises when facing the lure of a steady supply of spending money.
I would hate to see us return to the bad old days when children were enslaved in sweat shops, in dangerous and unhealthy working conditions. However, I fear that in some cases, in our zeal to protect young people from unsafe working conditions, we have made so many rules that we have seriously limited the opportunities for them to get a taste of the hands-on technical skills that our country desperately needs. Just try to get a plumber or electrician when you need one, and you will understand what I mean. A summer spent as an electrician’s gofer just might trigger a floundering 13-year-old to invest more energy in his studies when he sees them as a critical step to a well-paying job he would enjoy.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.”
FDA grants accelerated approval for treatment of Chagas disease in children
The Food and Drug Administration announced Aug. 29 the accelerated approval of benznidazole for use in children aged 2-12 years who have Chagas disease.
In two placebo-controlled clinical trials, researchers examined pediatric patients aged 6-12 with Chagas disease. In the first trial, about 60% of children treated with benznidazole had an antibody test change from positive to negative, compared with about 14% children who received a placebo. Similar findings were found in the second trial. In that one, about 55% of children treated with benznidazole had an antibody test change from positive to negative, compared with 5% who received a placebo. Also, a different study of the safety and pharmacokinetics of benznidazole in pediatric patients aged 2-12 provided information for dosing recommendations down to 2 years of age.
“The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
This is the first treatment approved in the United States for Chagas disease. According to the agency, additional study is needed to “verify and describe the anticipated clinical benefit of benznidazole.”
Read the full press release on the FDA’s website.
The Food and Drug Administration announced Aug. 29 the accelerated approval of benznidazole for use in children aged 2-12 years who have Chagas disease.
In two placebo-controlled clinical trials, researchers examined pediatric patients aged 6-12 with Chagas disease. In the first trial, about 60% of children treated with benznidazole had an antibody test change from positive to negative, compared with about 14% children who received a placebo. Similar findings were found in the second trial. In that one, about 55% of children treated with benznidazole had an antibody test change from positive to negative, compared with 5% who received a placebo. Also, a different study of the safety and pharmacokinetics of benznidazole in pediatric patients aged 2-12 provided information for dosing recommendations down to 2 years of age.
“The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
This is the first treatment approved in the United States for Chagas disease. According to the agency, additional study is needed to “verify and describe the anticipated clinical benefit of benznidazole.”
Read the full press release on the FDA’s website.
The Food and Drug Administration announced Aug. 29 the accelerated approval of benznidazole for use in children aged 2-12 years who have Chagas disease.
In two placebo-controlled clinical trials, researchers examined pediatric patients aged 6-12 with Chagas disease. In the first trial, about 60% of children treated with benznidazole had an antibody test change from positive to negative, compared with about 14% children who received a placebo. Similar findings were found in the second trial. In that one, about 55% of children treated with benznidazole had an antibody test change from positive to negative, compared with 5% who received a placebo. Also, a different study of the safety and pharmacokinetics of benznidazole in pediatric patients aged 2-12 provided information for dosing recommendations down to 2 years of age.
“The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
This is the first treatment approved in the United States for Chagas disease. According to the agency, additional study is needed to “verify and describe the anticipated clinical benefit of benznidazole.”
Read the full press release on the FDA’s website.
FDA approves first gene therapy – tisagenlecleucel for ALL
The U.S. Food and Drug Administration has approved tisagenlecleucel (Kymriah), a first-of-its-kind chimeric antigen receptor T-cell (CAR T) therapy, for the treatment of children and young adults up to age 25 years with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
Tisagenlecleucel will carry a boxed warning regarding the CRS risk. Additionally, due to the CRS risk and risk of neurological events, the approval requires a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use, according to an FDA press release.
Special certification will be required for hospitals and associated clinics that dispense tisagenlecleucel. As part of certification, staff will be trained in the prescribing, dispensing, or administering of the therapy, and to recognize and manage CRS and neurological events.
Novartis, the maker of tisagenlecleucel, will be required to conduct postmarketing observational study.
Indeed, FDA commissioner Scott Gottlieb, MD, said the approval marks the entry to a “new frontier in medical innovation.”
“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses,” he said in the press statement.
Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy involving customized treatment created using a patient’s own T cells. The T cells are genetically modified to include a chimeric antigen receptor that directs the T cells to target and kill leukemia cells with CD19 surface antigen, and are then infused back into the patient.
In a phase 2 clinical trial, the overall remission rate with tisagenlecleucel therapy was 83% in 63 children and young adults with relapsed/refractory B-cell precursor ALL for whom at least two prior lines of therapy had failed; the therapy was granted Fast Track, Priority Review, and Breakthrough Therapy designations.
“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research said in the press statement.
“Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that shows promising remission and survival rates in clinical trials.”
At its meeting in July, the FDA ODAC agreed nearly unanimously that the risk mitigation plan put forward by Novartis, including planned 15-year follow-up and other mitigation measures, would be adequate for detecting serious consequences of CAR T-cell therapy.
sworcester@frontlinemedcom.com
This article was updated August 30, 2017.
The U.S. Food and Drug Administration has approved tisagenlecleucel (Kymriah), a first-of-its-kind chimeric antigen receptor T-cell (CAR T) therapy, for the treatment of children and young adults up to age 25 years with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
Tisagenlecleucel will carry a boxed warning regarding the CRS risk. Additionally, due to the CRS risk and risk of neurological events, the approval requires a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use, according to an FDA press release.
Special certification will be required for hospitals and associated clinics that dispense tisagenlecleucel. As part of certification, staff will be trained in the prescribing, dispensing, or administering of the therapy, and to recognize and manage CRS and neurological events.
Novartis, the maker of tisagenlecleucel, will be required to conduct postmarketing observational study.
Indeed, FDA commissioner Scott Gottlieb, MD, said the approval marks the entry to a “new frontier in medical innovation.”
“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses,” he said in the press statement.
Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy involving customized treatment created using a patient’s own T cells. The T cells are genetically modified to include a chimeric antigen receptor that directs the T cells to target and kill leukemia cells with CD19 surface antigen, and are then infused back into the patient.
In a phase 2 clinical trial, the overall remission rate with tisagenlecleucel therapy was 83% in 63 children and young adults with relapsed/refractory B-cell precursor ALL for whom at least two prior lines of therapy had failed; the therapy was granted Fast Track, Priority Review, and Breakthrough Therapy designations.
“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research said in the press statement.
“Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that shows promising remission and survival rates in clinical trials.”
At its meeting in July, the FDA ODAC agreed nearly unanimously that the risk mitigation plan put forward by Novartis, including planned 15-year follow-up and other mitigation measures, would be adequate for detecting serious consequences of CAR T-cell therapy.
sworcester@frontlinemedcom.com
This article was updated August 30, 2017.
The U.S. Food and Drug Administration has approved tisagenlecleucel (Kymriah), a first-of-its-kind chimeric antigen receptor T-cell (CAR T) therapy, for the treatment of children and young adults up to age 25 years with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
Tisagenlecleucel will carry a boxed warning regarding the CRS risk. Additionally, due to the CRS risk and risk of neurological events, the approval requires a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use, according to an FDA press release.
Special certification will be required for hospitals and associated clinics that dispense tisagenlecleucel. As part of certification, staff will be trained in the prescribing, dispensing, or administering of the therapy, and to recognize and manage CRS and neurological events.
Novartis, the maker of tisagenlecleucel, will be required to conduct postmarketing observational study.
Indeed, FDA commissioner Scott Gottlieb, MD, said the approval marks the entry to a “new frontier in medical innovation.”
“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses,” he said in the press statement.
Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy involving customized treatment created using a patient’s own T cells. The T cells are genetically modified to include a chimeric antigen receptor that directs the T cells to target and kill leukemia cells with CD19 surface antigen, and are then infused back into the patient.
In a phase 2 clinical trial, the overall remission rate with tisagenlecleucel therapy was 83% in 63 children and young adults with relapsed/refractory B-cell precursor ALL for whom at least two prior lines of therapy had failed; the therapy was granted Fast Track, Priority Review, and Breakthrough Therapy designations.
“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research said in the press statement.
“Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that shows promising remission and survival rates in clinical trials.”
At its meeting in July, the FDA ODAC agreed nearly unanimously that the risk mitigation plan put forward by Novartis, including planned 15-year follow-up and other mitigation measures, would be adequate for detecting serious consequences of CAR T-cell therapy.
sworcester@frontlinemedcom.com
This article was updated August 30, 2017.