Pediatrics

The field of transgender health is growing. What began as a lone German physician in 1918 defying the norms of treating gender identity as a disease now has burgeoned into a field that includes 1,079 PubMed articles,two medical guidelines^1,2, and a multitude of books. As we learn more about the complexity of gender and gender identity, we also are discovering potential problems that occur when providing care to our transgender patients. One is eating disorders.

A systematic review by Jones et al. showed only a handful of studies on eating disorders in transgender individuals, most of them restricted to case studies.³ In some situations, the issue of gender identity arises during treatment for an eating disorder, as the individual realizes that body dissatisfaction is due to the gender identity instead of a fear of gaining weight. In other cases, a transgender person in the process of transitioning to the affirmed gender develops an eating disorder.

There are two larger quantitative studies on eating disorders among transgender individuals. One study of 289,024 college students reveals that transgender students, compared to cisgender students, are almost five times as likely to report an eating disorder and two times as likely to use unhealthy compensatory methods (e.g., vomiting) for weight control.⁴ Another study of almost 2,500 teenagers shows that transgender individuals are almost three times as likely to restrict their eating, almost nine times as likely to take diet pills, and seven times as likely to take laxatives.⁵

The most commonly suggested reason for the possible elevated risk for eating disorders among transgender individuals is that many of them are trying to achieve the unrealistic standards of the ideal masculine or feminine body type. Another explanation is that eating disorders among transgender individuals are maladaptive coping mechanisms to stress from antitrans stigma and discrimination.⁴ However, these explanations are not mutually exclusive and could simultaneously drive disordered eating among transgender individuals.

To further appreciate the relationship between these two conditions, one must understand their similarities and differences. The Diagnostic Statistical Manual of Mental Disorders V characterizes eating disorders as “a persistent disturbance of eating or eating-related behavior that results in the altered consumption or absorption of food and ... significantly impairs physical health or psychosocial functioning.”⁶ Anorexia nervosa and bulimia nervosa are driven by fear of gaining weight or by a self-esteem unduly influenced by weight or appearance.⁶

Gender dysphoria, in comparison, is the distress caused by the incongruence between one’s gender identity and one’s anatomy, along with the desire to have the characteristics of one’s affirmed gender identity. This condition also could severely alter physical and psychosocial functioning,⁷ partly because of the distress from the incongruence, and partly because of the stress from antitrans stigma and discrimination, as an individual attempts to match the body with the gender identity⁸ (e.g., wearing clothing to match the gender identity).

The higher risk of developing an eating disorder among transgender individuals makes sense. Dissatisfaction with one’s body characterizes both conditions. The high standards on what is masculine or feminine affects everyone, especially transgender individuals who may feel that they’re “far behind” when they begin to transition to their affirmed gender. In addition, both involve identity. Those who have anorexia nervosa also incorporate this into their own identity.⁹ This is why treating an eating disorder can be very difficult.

Finally, individuals afflicted by an eating disorder or gender dysphoria engage in certain behaviors to achieve their desired appearance. However, this is where the similarities end. One major distinction between an eating disorder and gender dysphoria is the treatment approach. The goal in treating an eating disorder is to discourage the disordered behavior and encourage healthier eating habits and a more positive body image. Affirming the identity of someone with an eating disorder can be deadly, as it will encourage more disordered eating.¹⁰ In contrast, affirming the identity of someone with gender dysphoria through social transition, cross-sex hormones, and/or surgical reassignment is life-saving and therapeutic.¹¹

There is little guidance on how to treat the these disorders simultaneously. What complicates treating both conditions at the same time is that when an eating disorder is accompanied by another mental health disorder (e.g., substance use), one condition over the other is prioritized.¹² There is no guidance on whether the eating disorder or gender dysphoria should take priority over the other, or if it is possible to treat both conditions at the same time.

Strandjord et al. suggest a hierarchal approach, in which life-threatening issues (such as suicide or electrolyte disturbances) take priority.¹³ In addition, if the patient is malnourished, weight restoration should be the initial focus. A patient who is severely malnourished may not have the cognitive capacity nor the physiological ability to manage comorbidities such as anxiety or depression,¹² much less have the capacity to process something as complex as gender and gender identity, nor understand the steps necessary for a successful transition to the affirmed gender. However, this does not mean providers should wait to successfully manage an eating disorder before addressing gender dysphoria. Studies have suggested that gender-affirming medical therapies (e.g., cross sex hormones) can be therapeutic for both gender dysphoria and eating disorder symptoms.¹⁴ Finally, because of the two ways a transgender patient with an eating disorder can present, I recommend screening for eating disorders in transgender individuals and inquiring about gender identity among those with an eating disorder. Doing so may save a life.

 

References

1. J Clin Endocrinol Metab. 2009 Sep;94(9):3132-54.

2. Adv Urol. 2012;2012:581712.

3. Int Rev Psychiatry. 2016;28(1):81-94.

4. J Adolesc Health. 2015 Aug;57(2):144-9.

5. J Adolesc Health. 2016. doi: 10.1016/j.jadohealth.2016.08.027.

6. Feeding and Eating Disorders. Diagnostic and Statistical Manual of Mental Disorders. 5th ed. (Washington: American Psychiatric Association, 2013).

7. Gender Dysphoria. Diagnostic and Statistical Manual of Mental Disorders. 5th ed. (Washington: American Psychiatric Association, 2013).

8. Psychol Bull. 2003 Sep;129(5):674-97.

9. Int J Law Psychiatry. 2003 Sep-Oct;26(5):533-48.

10. Arch Gen Psychiatry. 2011 Jul;68(7):724-31.

11. Clin Endocrinol (Oxf). 2010 Feb;72(2):214-31.

12. CNS drugs. 2006;20(8):655-63.

13. Int J Eat Disord. 2015 Nov;48(7):942-5.

14. Eat Disord. 2012;20(4):300-11.

Dr. Montano is clinical instructor of pediatrics at the University of Pittsburgh and an adolescent medicine physician at Children’s Hospital of Pittsburgh of UPMC. Email him at pdnews@frontlinemedcom.com.

The field of transgender health is growing. What began as a lone German physician in 1918 defying the norms of treating gender identity as a disease now has burgeoned into a field that includes 1,079 PubMed articles,two medical guidelines^1,2, and a multitude of books. As we learn more about the complexity of gender and gender identity, we also are discovering potential problems that occur when providing care to our transgender patients. One is eating disorders.

A systematic review by Jones et al. showed only a handful of studies on eating disorders in transgender individuals, most of them restricted to case studies.³ In some situations, the issue of gender identity arises during treatment for an eating disorder, as the individual realizes that body dissatisfaction is due to the gender identity instead of a fear of gaining weight. In other cases, a transgender person in the process of transitioning to the affirmed gender develops an eating disorder.

There are two larger quantitative studies on eating disorders among transgender individuals. One study of 289,024 college students reveals that transgender students, compared to cisgender students, are almost five times as likely to report an eating disorder and two times as likely to use unhealthy compensatory methods (e.g., vomiting) for weight control.⁴ Another study of almost 2,500 teenagers shows that transgender individuals are almost three times as likely to restrict their eating, almost nine times as likely to take diet pills, and seven times as likely to take laxatives.⁵

The most commonly suggested reason for the possible elevated risk for eating disorders among transgender individuals is that many of them are trying to achieve the unrealistic standards of the ideal masculine or feminine body type. Another explanation is that eating disorders among transgender individuals are maladaptive coping mechanisms to stress from antitrans stigma and discrimination.⁴ However, these explanations are not mutually exclusive and could simultaneously drive disordered eating among transgender individuals.

To further appreciate the relationship between these two conditions, one must understand their similarities and differences. The Diagnostic Statistical Manual of Mental Disorders V characterizes eating disorders as “a persistent disturbance of eating or eating-related behavior that results in the altered consumption or absorption of food and ... significantly impairs physical health or psychosocial functioning.”⁶ Anorexia nervosa and bulimia nervosa are driven by fear of gaining weight or by a self-esteem unduly influenced by weight or appearance.⁶

Gender dysphoria, in comparison, is the distress caused by the incongruence between one’s gender identity and one’s anatomy, along with the desire to have the characteristics of one’s affirmed gender identity. This condition also could severely alter physical and psychosocial functioning,⁷ partly because of the distress from the incongruence, and partly because of the stress from antitrans stigma and discrimination, as an individual attempts to match the body with the gender identity⁸ (e.g., wearing clothing to match the gender identity).

The higher risk of developing an eating disorder among transgender individuals makes sense. Dissatisfaction with one’s body characterizes both conditions. The high standards on what is masculine or feminine affects everyone, especially transgender individuals who may feel that they’re “far behind” when they begin to transition to their affirmed gender. In addition, both involve identity. Those who have anorexia nervosa also incorporate this into their own identity.⁹ This is why treating an eating disorder can be very difficult.

Finally, individuals afflicted by an eating disorder or gender dysphoria engage in certain behaviors to achieve their desired appearance. However, this is where the similarities end. One major distinction between an eating disorder and gender dysphoria is the treatment approach. The goal in treating an eating disorder is to discourage the disordered behavior and encourage healthier eating habits and a more positive body image. Affirming the identity of someone with an eating disorder can be deadly, as it will encourage more disordered eating.¹⁰ In contrast, affirming the identity of someone with gender dysphoria through social transition, cross-sex hormones, and/or surgical reassignment is life-saving and therapeutic.¹¹

There is little guidance on how to treat the these disorders simultaneously. What complicates treating both conditions at the same time is that when an eating disorder is accompanied by another mental health disorder (e.g., substance use), one condition over the other is prioritized.¹² There is no guidance on whether the eating disorder or gender dysphoria should take priority over the other, or if it is possible to treat both conditions at the same time.

Strandjord et al. suggest a hierarchal approach, in which life-threatening issues (such as suicide or electrolyte disturbances) take priority.¹³ In addition, if the patient is malnourished, weight restoration should be the initial focus. A patient who is severely malnourished may not have the cognitive capacity nor the physiological ability to manage comorbidities such as anxiety or depression,¹² much less have the capacity to process something as complex as gender and gender identity, nor understand the steps necessary for a successful transition to the affirmed gender. However, this does not mean providers should wait to successfully manage an eating disorder before addressing gender dysphoria. Studies have suggested that gender-affirming medical therapies (e.g., cross sex hormones) can be therapeutic for both gender dysphoria and eating disorder symptoms.¹⁴ Finally, because of the two ways a transgender patient with an eating disorder can present, I recommend screening for eating disorders in transgender individuals and inquiring about gender identity among those with an eating disorder. Doing so may save a life.

 

References

1. J Clin Endocrinol Metab. 2009 Sep;94(9):3132-54.

2. Adv Urol. 2012;2012:581712.

3. Int Rev Psychiatry. 2016;28(1):81-94.

4. J Adolesc Health. 2015 Aug;57(2):144-9.

5. J Adolesc Health. 2016. doi: 10.1016/j.jadohealth.2016.08.027.

6. Feeding and Eating Disorders. Diagnostic and Statistical Manual of Mental Disorders. 5th ed. (Washington: American Psychiatric Association, 2013).

7. Gender Dysphoria. Diagnostic and Statistical Manual of Mental Disorders. 5th ed. (Washington: American Psychiatric Association, 2013).

8. Psychol Bull. 2003 Sep;129(5):674-97.

9. Int J Law Psychiatry. 2003 Sep-Oct;26(5):533-48.

10. Arch Gen Psychiatry. 2011 Jul;68(7):724-31.

11. Clin Endocrinol (Oxf). 2010 Feb;72(2):214-31.

12. CNS drugs. 2006;20(8):655-63.

13. Int J Eat Disord. 2015 Nov;48(7):942-5.

14. Eat Disord. 2012;20(4):300-11.

Dr. Montano is clinical instructor of pediatrics at the University of Pittsburgh and an adolescent medicine physician at Children’s Hospital of Pittsburgh of UPMC. Email him at pdnews@frontlinemedcom.com.

The field of transgender health is growing. What began as a lone German physician in 1918 defying the norms of treating gender identity as a disease now has burgeoned into a field that includes 1,079 PubMed articles,two medical guidelines^1,2, and a multitude of books. As we learn more about the complexity of gender and gender identity, we also are discovering potential problems that occur when providing care to our transgender patients. One is eating disorders.

A systematic review by Jones et al. showed only a handful of studies on eating disorders in transgender individuals, most of them restricted to case studies.³ In some situations, the issue of gender identity arises during treatment for an eating disorder, as the individual realizes that body dissatisfaction is due to the gender identity instead of a fear of gaining weight. In other cases, a transgender person in the process of transitioning to the affirmed gender develops an eating disorder.

There are two larger quantitative studies on eating disorders among transgender individuals. One study of 289,024 college students reveals that transgender students, compared to cisgender students, are almost five times as likely to report an eating disorder and two times as likely to use unhealthy compensatory methods (e.g., vomiting) for weight control.⁴ Another study of almost 2,500 teenagers shows that transgender individuals are almost three times as likely to restrict their eating, almost nine times as likely to take diet pills, and seven times as likely to take laxatives.⁵

The most commonly suggested reason for the possible elevated risk for eating disorders among transgender individuals is that many of them are trying to achieve the unrealistic standards of the ideal masculine or feminine body type. Another explanation is that eating disorders among transgender individuals are maladaptive coping mechanisms to stress from antitrans stigma and discrimination.⁴ However, these explanations are not mutually exclusive and could simultaneously drive disordered eating among transgender individuals.

To further appreciate the relationship between these two conditions, one must understand their similarities and differences. The Diagnostic Statistical Manual of Mental Disorders V characterizes eating disorders as “a persistent disturbance of eating or eating-related behavior that results in the altered consumption or absorption of food and ... significantly impairs physical health or psychosocial functioning.”⁶ Anorexia nervosa and bulimia nervosa are driven by fear of gaining weight or by a self-esteem unduly influenced by weight or appearance.⁶

Gender dysphoria, in comparison, is the distress caused by the incongruence between one’s gender identity and one’s anatomy, along with the desire to have the characteristics of one’s affirmed gender identity. This condition also could severely alter physical and psychosocial functioning,⁷ partly because of the distress from the incongruence, and partly because of the stress from antitrans stigma and discrimination, as an individual attempts to match the body with the gender identity⁸ (e.g., wearing clothing to match the gender identity).

The higher risk of developing an eating disorder among transgender individuals makes sense. Dissatisfaction with one’s body characterizes both conditions. The high standards on what is masculine or feminine affects everyone, especially transgender individuals who may feel that they’re “far behind” when they begin to transition to their affirmed gender. In addition, both involve identity. Those who have anorexia nervosa also incorporate this into their own identity.⁹ This is why treating an eating disorder can be very difficult.

Finally, individuals afflicted by an eating disorder or gender dysphoria engage in certain behaviors to achieve their desired appearance. However, this is where the similarities end. One major distinction between an eating disorder and gender dysphoria is the treatment approach. The goal in treating an eating disorder is to discourage the disordered behavior and encourage healthier eating habits and a more positive body image. Affirming the identity of someone with an eating disorder can be deadly, as it will encourage more disordered eating.¹⁰ In contrast, affirming the identity of someone with gender dysphoria through social transition, cross-sex hormones, and/or surgical reassignment is life-saving and therapeutic.¹¹

There is little guidance on how to treat the these disorders simultaneously. What complicates treating both conditions at the same time is that when an eating disorder is accompanied by another mental health disorder (e.g., substance use), one condition over the other is prioritized.¹² There is no guidance on whether the eating disorder or gender dysphoria should take priority over the other, or if it is possible to treat both conditions at the same time.

Strandjord et al. suggest a hierarchal approach, in which life-threatening issues (such as suicide or electrolyte disturbances) take priority.¹³ In addition, if the patient is malnourished, weight restoration should be the initial focus. A patient who is severely malnourished may not have the cognitive capacity nor the physiological ability to manage comorbidities such as anxiety or depression,¹² much less have the capacity to process something as complex as gender and gender identity, nor understand the steps necessary for a successful transition to the affirmed gender. However, this does not mean providers should wait to successfully manage an eating disorder before addressing gender dysphoria. Studies have suggested that gender-affirming medical therapies (e.g., cross sex hormones) can be therapeutic for both gender dysphoria and eating disorder symptoms.¹⁴ Finally, because of the two ways a transgender patient with an eating disorder can present, I recommend screening for eating disorders in transgender individuals and inquiring about gender identity among those with an eating disorder. Doing so may save a life.

 

References

1. J Clin Endocrinol Metab. 2009 Sep;94(9):3132-54.

2. Adv Urol. 2012;2012:581712.

3. Int Rev Psychiatry. 2016;28(1):81-94.

4. J Adolesc Health. 2015 Aug;57(2):144-9.

5. J Adolesc Health. 2016. doi: 10.1016/j.jadohealth.2016.08.027.

6. Feeding and Eating Disorders. Diagnostic and Statistical Manual of Mental Disorders. 5th ed. (Washington: American Psychiatric Association, 2013).

7. Gender Dysphoria. Diagnostic and Statistical Manual of Mental Disorders. 5th ed. (Washington: American Psychiatric Association, 2013).

8. Psychol Bull. 2003 Sep;129(5):674-97.

9. Int J Law Psychiatry. 2003 Sep-Oct;26(5):533-48.

10. Arch Gen Psychiatry. 2011 Jul;68(7):724-31.

11. Clin Endocrinol (Oxf). 2010 Feb;72(2):214-31.

12. CNS drugs. 2006;20(8):655-63.

13. Int J Eat Disord. 2015 Nov;48(7):942-5.

14. Eat Disord. 2012;20(4):300-11.

Dr. Montano is clinical instructor of pediatrics at the University of Pittsburgh and an adolescent medicine physician at Children’s Hospital of Pittsburgh of UPMC. Email him at pdnews@frontlinemedcom.com.

 

Among periviable infants born at 11 tertiary care centers in 2000 through 2011, the rate of survival without neurodevelopmental impairment increased a small but significant 4%, according to a report published online Feb. 16 in the New England Journal of Medicine.

The rate of survival with neurodevelopmental impairment also increased, although to a lesser extent (1%).

herjua/Thinkstock

The rise in the survival rate of these extremely premature infants born at 22-24 weeks’ gestation “was not associated with a disproportionate increase in the rate of survival with neurodevelopmental impairment; rather, the rate of survival without neurodevelopmental impairment and the rate of survival with neurodevelopmental impairment increased similarly (adjusted relative risk, 1.27),” said Noelle Younge, MD, and her associates in the Eunice Kennedy Shriver National Institute of Child Health and Human Development Neonatal Research Network.

“These findings are important for guiding counseling and decision making with respect to periviable birth. Prognosis continues to be guarded; in the most recent epoch [time period in our study], mortality was 64%, and 43% of surviving infants had neurodevelopmental impairment,” they noted.

The investigators defined such impairment as moderate or severe cerebral palsy, Gross Motor Function Classification System level of at least 2 on a scale of 1-5, profound hearing loss requiring amplification in both ears, profound visual impairment in both eyes, or cognitive impairment such as a Mental Developmental Index score of less than 70 or a Cognitive Composite score of less than 85.

To examine time trends in the outcomes of periviable infants, Dr. Younge of Duke University, Durham, N.C., and her associates analyzed data from the network’s registry of births at 11 academic tertiary care centers nationwide. They focused on 4,274 infants who were born during 3 epochs – 2000-2003, 2004-2007, and 2008-2011 – and were evaluated for motor function, sensory impairment, and cognitive delay at a corrected age of 18-22 months.

The percentage of infants who survived without neurodevelopmental impairment increased over time, from 16% during the first epoch to 20% during the third epoch. However, the percentage who survived with neurodevelopmental impairment also increased, from 15% during the first epoch to 16% during the third epoch (New Engl. J. Med. 2017 Feb 16. doi: 10.1056/NEJMoa1605566).

The rates of active treatment of these periviable infants didn’t change significantly over time. Overall, 22% of infants born at 22 weeks, 71% of those born at 23 weeks, and 95% of those born at 24 weeks received active treatment at birth. Therefore, the overall decrease in mortality and the 4% improvement in neurodevelopmental outcomes wasn’t attributable to greater use of active treatment for periviable infants over time, said Dr. Younge and her associates.

Despite these small but significant improvements in outcomes, “the incidence of death, neurodevelopmental impairment, and other adverse outcomes remains high in this population,” they noted.

This study was supported by the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institutes of Health, the National Center for Research Resources, and the National Center for Advancing Translational Sciences for the Neonatal Research Network’s Generic Database and Follow-up Studies. Dr. Younge reported having no relevant financial disclosures; two of her associates reported ties to Pediatrix Medical Group and rEVO Biologics.

 

Among periviable infants born at 11 tertiary care centers in 2000 through 2011, the rate of survival without neurodevelopmental impairment increased a small but significant 4%, according to a report published online Feb. 16 in the New England Journal of Medicine.

The rate of survival with neurodevelopmental impairment also increased, although to a lesser extent (1%).

herjua/Thinkstock

The rise in the survival rate of these extremely premature infants born at 22-24 weeks’ gestation “was not associated with a disproportionate increase in the rate of survival with neurodevelopmental impairment; rather, the rate of survival without neurodevelopmental impairment and the rate of survival with neurodevelopmental impairment increased similarly (adjusted relative risk, 1.27),” said Noelle Younge, MD, and her associates in the Eunice Kennedy Shriver National Institute of Child Health and Human Development Neonatal Research Network.

“These findings are important for guiding counseling and decision making with respect to periviable birth. Prognosis continues to be guarded; in the most recent epoch [time period in our study], mortality was 64%, and 43% of surviving infants had neurodevelopmental impairment,” they noted.

The investigators defined such impairment as moderate or severe cerebral palsy, Gross Motor Function Classification System level of at least 2 on a scale of 1-5, profound hearing loss requiring amplification in both ears, profound visual impairment in both eyes, or cognitive impairment such as a Mental Developmental Index score of less than 70 or a Cognitive Composite score of less than 85.

To examine time trends in the outcomes of periviable infants, Dr. Younge of Duke University, Durham, N.C., and her associates analyzed data from the network’s registry of births at 11 academic tertiary care centers nationwide. They focused on 4,274 infants who were born during 3 epochs – 2000-2003, 2004-2007, and 2008-2011 – and were evaluated for motor function, sensory impairment, and cognitive delay at a corrected age of 18-22 months.

The percentage of infants who survived without neurodevelopmental impairment increased over time, from 16% during the first epoch to 20% during the third epoch. However, the percentage who survived with neurodevelopmental impairment also increased, from 15% during the first epoch to 16% during the third epoch (New Engl. J. Med. 2017 Feb 16. doi: 10.1056/NEJMoa1605566).

The rates of active treatment of these periviable infants didn’t change significantly over time. Overall, 22% of infants born at 22 weeks, 71% of those born at 23 weeks, and 95% of those born at 24 weeks received active treatment at birth. Therefore, the overall decrease in mortality and the 4% improvement in neurodevelopmental outcomes wasn’t attributable to greater use of active treatment for periviable infants over time, said Dr. Younge and her associates.

Despite these small but significant improvements in outcomes, “the incidence of death, neurodevelopmental impairment, and other adverse outcomes remains high in this population,” they noted.

This study was supported by the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institutes of Health, the National Center for Research Resources, and the National Center for Advancing Translational Sciences for the Neonatal Research Network’s Generic Database and Follow-up Studies. Dr. Younge reported having no relevant financial disclosures; two of her associates reported ties to Pediatrix Medical Group and rEVO Biologics.

 

Among periviable infants born at 11 tertiary care centers in 2000 through 2011, the rate of survival without neurodevelopmental impairment increased a small but significant 4%, according to a report published online Feb. 16 in the New England Journal of Medicine.

The rate of survival with neurodevelopmental impairment also increased, although to a lesser extent (1%).

herjua/Thinkstock

The rise in the survival rate of these extremely premature infants born at 22-24 weeks’ gestation “was not associated with a disproportionate increase in the rate of survival with neurodevelopmental impairment; rather, the rate of survival without neurodevelopmental impairment and the rate of survival with neurodevelopmental impairment increased similarly (adjusted relative risk, 1.27),” said Noelle Younge, MD, and her associates in the Eunice Kennedy Shriver National Institute of Child Health and Human Development Neonatal Research Network.

“These findings are important for guiding counseling and decision making with respect to periviable birth. Prognosis continues to be guarded; in the most recent epoch [time period in our study], mortality was 64%, and 43% of surviving infants had neurodevelopmental impairment,” they noted.

The investigators defined such impairment as moderate or severe cerebral palsy, Gross Motor Function Classification System level of at least 2 on a scale of 1-5, profound hearing loss requiring amplification in both ears, profound visual impairment in both eyes, or cognitive impairment such as a Mental Developmental Index score of less than 70 or a Cognitive Composite score of less than 85.

To examine time trends in the outcomes of periviable infants, Dr. Younge of Duke University, Durham, N.C., and her associates analyzed data from the network’s registry of births at 11 academic tertiary care centers nationwide. They focused on 4,274 infants who were born during 3 epochs – 2000-2003, 2004-2007, and 2008-2011 – and were evaluated for motor function, sensory impairment, and cognitive delay at a corrected age of 18-22 months.

The percentage of infants who survived without neurodevelopmental impairment increased over time, from 16% during the first epoch to 20% during the third epoch. However, the percentage who survived with neurodevelopmental impairment also increased, from 15% during the first epoch to 16% during the third epoch (New Engl. J. Med. 2017 Feb 16. doi: 10.1056/NEJMoa1605566).

The rates of active treatment of these periviable infants didn’t change significantly over time. Overall, 22% of infants born at 22 weeks, 71% of those born at 23 weeks, and 95% of those born at 24 weeks received active treatment at birth. Therefore, the overall decrease in mortality and the 4% improvement in neurodevelopmental outcomes wasn’t attributable to greater use of active treatment for periviable infants over time, said Dr. Younge and her associates.

Despite these small but significant improvements in outcomes, “the incidence of death, neurodevelopmental impairment, and other adverse outcomes remains high in this population,” they noted.

This study was supported by the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institutes of Health, the National Center for Research Resources, and the National Center for Advancing Translational Sciences for the Neonatal Research Network’s Generic Database and Follow-up Studies. Dr. Younge reported having no relevant financial disclosures; two of her associates reported ties to Pediatrix Medical Group and rEVO Biologics.

 

A wide range of attitudes and practices for the process of withdrawing medications in pediatric patients with clinically inactive juvenile idiopathic arthritis (JIA) exist among clinician members of the Childhood Arthritis and Rheumatology Research Alliance (CARRA), according to findings from an anonymous survey.

The cross-sectional, electronic survey found that respondents varied in the amount of time they thought was necessary to spend in clinically inactive disease before beginning withdrawal of medications and in the amount of time to spend during tapering or stopping medications, for both methotrexate and biologics.

Many studies of discontinuing therapy after achieving clinically inactive disease have been small and retrospective and have been unable to identify predictors of successful withdrawal of medications, according to the authors of the survey study, led by Daniel B. Horton, MD, of the division of pediatric rheumatology at Rutgers Biomedical and Health Sciences, New Brunswick, N.J. Some of these studies found that discontinuation of biologics with or without methotrexate led to disease flare in one-half to two-thirds of patients within 1 year. Results from one of these smaller retrospective studies found that better outcomes were associated with longer durations of medicated remission and medication taper instead of immediate discontinuation, but the results were not supported by the only published randomized trial on medication discontinuation in JIA, which involved methotrexate. However, two single-center observational studies have shown improved outcomes with the use of medication reduction, rather than discontinuation, using low-dose etanercept, or by continuing biologics after withdrawing methotrexate.

To better understand how clinicians care for patients with clinically inactive disease, the investigators emailed the survey to 388 clinician members of the CARRA in the United States and Canada over a 4-week period during November-December 2015 (J Rheumatol. 2017 Feb 1. doi: 10.3899/jrheum.161078).

The survey, which the investigators thought to be “the first comprehensive evaluation of influential factors and approaches for the clinical management of children with clinically inactive JIA,” did not include questions about systemic JIA, inflammatory bowel disease, psoriasis, and uveitis in order “to simplify responses and encourage participation, because in practice, the manifestations and outcomes of these diseases could substantially influence treatment decisions for children with JIA.”

They received complete responses from 124 of the 132 clinicians who responded to the survey email. Of the 121 respondents who reported taking clinical care of patients with JIA, 87% were physicians, and the same number reported taking care of pediatric patients only. About three-quarters spent half of their professional time in clinical care, and about half had more than 10 years of post-training clinical experience.

When deciding about withdrawing JIA medications, more than one-half of respondents said that the time that a patient spent in clinically inactive disease and a history of drug toxicity are very important factors. Most participants ranked those two factors most highly and most often among their top five factors for decision making.

Respondents also commonly ranked these factors as important:

• JIA duration before attaining clinically inactive disease.
• Patient/family preferences.
• Presence of JIA-related damage.
• JIA category.

The factors that consistently appeared in responses fit into three clusters that included JIA features and time spent in clinically inactive disease (JIA category and total disease duration), JIA severity and resistance to treatment (disease duration before clinically inactive disease, number of drugs needed to attain inactivity, joint damage, and a history of sacroiliac or temporomandibular disease), and the patient’s experience (drug toxicity and family preference).

The respondents indicated that they would be least likely to stop medications for children with rheumatoid factor (RF)–positive polyarthritis (85%), which is “consistent with prior studies showing that RF-positive polyarthritis is associated with higher rates of flares than other JIA categories,” the investigators wrote. However, respondents said they would be most likely to stop medications for children with persistent oligoarthritis (87%) “even though rates of flares in this category appear similar to other JIA types. This method may reflect a belief that flares in children with persistent oligoarticular JIA will be less severe and easier to control.”

When patients met all criteria for clinically inactive disease for a “sufficient amount of time” and families were interested in stopping medications, some factors continued to make respondents reluctant to withdraw medications. These factors were most often a history of erosions (81%), asymptomatic joint abnormalities on ultrasound or MRI (72%), and failure of multiple prior disease-modifying antirheumatic drugs or biologics to control disease (64%). The definition of clinically inactive disease is a composite of no active arthritis, uveitis, or systemic JIA symptoms; the best possible clinical global assessment; inflammatory markers normal or elevated for reasons other than JIA; and no more than 15 minutes of joint stiffness.

A little over half of respondents said they would wait until clinically inactive disease had lasted 12 months before considering stopping or tapering methotrexate or biologic monotherapy, but a substantial minority said they would wait for only 6 months for methotrexate (31%) or biologic monotherapy (23%). A smaller number would wait for 18 months for methotrexate (13%) or biologics (18%), and another 3%-5% said they could not give a time frame.

The strategies varied for how actual withdrawing of medications occurred. Most methotrexate monotherapy withdrawals involved tapering over 2-6 months, one-third over longer periods, and the fewest reported tapering for less than 2 months (7%) or immediate withdrawal (17%).

Withdrawal of biologics was generally said to occur more gradually than with methotrexate, with one-third of respondents citing over 2-6 months, a quarter more slowly, and another 29% in less than 2 months or immediately. Some wrote that they preferred spacing out the interval between doses, but none decreased the dose. When children took combination therapy with methotrexate plus a biologic, 63% said that they began tapering or stopping methotrexate first, but a quarter said that the order was strongly context dependent, and the most commonly cited reason for deciding was history of toxicity or intolerance.

Imaging played a role in less than half of the decisions to withdraw medications, with it being used often by 9% and sometimes by 36%. And while it’s assumed that patients and family consideration played an important role in decision making, only 25% of respondents reported using specific patient-reported outcomes in deciding to withdraw medications.

The study was funded by grants from Rutgers Biomedical and Health Sciences and the National Institute of Arthritis and Musculoskeletal and Skin Diseases.

 

jevans@frontlinemedcom.com

 

A wide range of attitudes and practices for the process of withdrawing medications in pediatric patients with clinically inactive juvenile idiopathic arthritis (JIA) exist among clinician members of the Childhood Arthritis and Rheumatology Research Alliance (CARRA), according to findings from an anonymous survey.

The cross-sectional, electronic survey found that respondents varied in the amount of time they thought was necessary to spend in clinically inactive disease before beginning withdrawal of medications and in the amount of time to spend during tapering or stopping medications, for both methotrexate and biologics.

Many studies of discontinuing therapy after achieving clinically inactive disease have been small and retrospective and have been unable to identify predictors of successful withdrawal of medications, according to the authors of the survey study, led by Daniel B. Horton, MD, of the division of pediatric rheumatology at Rutgers Biomedical and Health Sciences, New Brunswick, N.J. Some of these studies found that discontinuation of biologics with or without methotrexate led to disease flare in one-half to two-thirds of patients within 1 year. Results from one of these smaller retrospective studies found that better outcomes were associated with longer durations of medicated remission and medication taper instead of immediate discontinuation, but the results were not supported by the only published randomized trial on medication discontinuation in JIA, which involved methotrexate. However, two single-center observational studies have shown improved outcomes with the use of medication reduction, rather than discontinuation, using low-dose etanercept, or by continuing biologics after withdrawing methotrexate.

To better understand how clinicians care for patients with clinically inactive disease, the investigators emailed the survey to 388 clinician members of the CARRA in the United States and Canada over a 4-week period during November-December 2015 (J Rheumatol. 2017 Feb 1. doi: 10.3899/jrheum.161078).

The survey, which the investigators thought to be “the first comprehensive evaluation of influential factors and approaches for the clinical management of children with clinically inactive JIA,” did not include questions about systemic JIA, inflammatory bowel disease, psoriasis, and uveitis in order “to simplify responses and encourage participation, because in practice, the manifestations and outcomes of these diseases could substantially influence treatment decisions for children with JIA.”

They received complete responses from 124 of the 132 clinicians who responded to the survey email. Of the 121 respondents who reported taking clinical care of patients with JIA, 87% were physicians, and the same number reported taking care of pediatric patients only. About three-quarters spent half of their professional time in clinical care, and about half had more than 10 years of post-training clinical experience.

When deciding about withdrawing JIA medications, more than one-half of respondents said that the time that a patient spent in clinically inactive disease and a history of drug toxicity are very important factors. Most participants ranked those two factors most highly and most often among their top five factors for decision making.

Respondents also commonly ranked these factors as important:

• JIA duration before attaining clinically inactive disease.
• Patient/family preferences.
• Presence of JIA-related damage.
• JIA category.

The factors that consistently appeared in responses fit into three clusters that included JIA features and time spent in clinically inactive disease (JIA category and total disease duration), JIA severity and resistance to treatment (disease duration before clinically inactive disease, number of drugs needed to attain inactivity, joint damage, and a history of sacroiliac or temporomandibular disease), and the patient’s experience (drug toxicity and family preference).

The respondents indicated that they would be least likely to stop medications for children with rheumatoid factor (RF)–positive polyarthritis (85%), which is “consistent with prior studies showing that RF-positive polyarthritis is associated with higher rates of flares than other JIA categories,” the investigators wrote. However, respondents said they would be most likely to stop medications for children with persistent oligoarthritis (87%) “even though rates of flares in this category appear similar to other JIA types. This method may reflect a belief that flares in children with persistent oligoarticular JIA will be less severe and easier to control.”

When patients met all criteria for clinically inactive disease for a “sufficient amount of time” and families were interested in stopping medications, some factors continued to make respondents reluctant to withdraw medications. These factors were most often a history of erosions (81%), asymptomatic joint abnormalities on ultrasound or MRI (72%), and failure of multiple prior disease-modifying antirheumatic drugs or biologics to control disease (64%). The definition of clinically inactive disease is a composite of no active arthritis, uveitis, or systemic JIA symptoms; the best possible clinical global assessment; inflammatory markers normal or elevated for reasons other than JIA; and no more than 15 minutes of joint stiffness.

A little over half of respondents said they would wait until clinically inactive disease had lasted 12 months before considering stopping or tapering methotrexate or biologic monotherapy, but a substantial minority said they would wait for only 6 months for methotrexate (31%) or biologic monotherapy (23%). A smaller number would wait for 18 months for methotrexate (13%) or biologics (18%), and another 3%-5% said they could not give a time frame.

The strategies varied for how actual withdrawing of medications occurred. Most methotrexate monotherapy withdrawals involved tapering over 2-6 months, one-third over longer periods, and the fewest reported tapering for less than 2 months (7%) or immediate withdrawal (17%).

Withdrawal of biologics was generally said to occur more gradually than with methotrexate, with one-third of respondents citing over 2-6 months, a quarter more slowly, and another 29% in less than 2 months or immediately. Some wrote that they preferred spacing out the interval between doses, but none decreased the dose. When children took combination therapy with methotrexate plus a biologic, 63% said that they began tapering or stopping methotrexate first, but a quarter said that the order was strongly context dependent, and the most commonly cited reason for deciding was history of toxicity or intolerance.

Imaging played a role in less than half of the decisions to withdraw medications, with it being used often by 9% and sometimes by 36%. And while it’s assumed that patients and family consideration played an important role in decision making, only 25% of respondents reported using specific patient-reported outcomes in deciding to withdraw medications.

The study was funded by grants from Rutgers Biomedical and Health Sciences and the National Institute of Arthritis and Musculoskeletal and Skin Diseases.

 

jevans@frontlinemedcom.com

 

A wide range of attitudes and practices for the process of withdrawing medications in pediatric patients with clinically inactive juvenile idiopathic arthritis (JIA) exist among clinician members of the Childhood Arthritis and Rheumatology Research Alliance (CARRA), according to findings from an anonymous survey.

The cross-sectional, electronic survey found that respondents varied in the amount of time they thought was necessary to spend in clinically inactive disease before beginning withdrawal of medications and in the amount of time to spend during tapering or stopping medications, for both methotrexate and biologics.

Many studies of discontinuing therapy after achieving clinically inactive disease have been small and retrospective and have been unable to identify predictors of successful withdrawal of medications, according to the authors of the survey study, led by Daniel B. Horton, MD, of the division of pediatric rheumatology at Rutgers Biomedical and Health Sciences, New Brunswick, N.J. Some of these studies found that discontinuation of biologics with or without methotrexate led to disease flare in one-half to two-thirds of patients within 1 year. Results from one of these smaller retrospective studies found that better outcomes were associated with longer durations of medicated remission and medication taper instead of immediate discontinuation, but the results were not supported by the only published randomized trial on medication discontinuation in JIA, which involved methotrexate. However, two single-center observational studies have shown improved outcomes with the use of medication reduction, rather than discontinuation, using low-dose etanercept, or by continuing biologics after withdrawing methotrexate.

To better understand how clinicians care for patients with clinically inactive disease, the investigators emailed the survey to 388 clinician members of the CARRA in the United States and Canada over a 4-week period during November-December 2015 (J Rheumatol. 2017 Feb 1. doi: 10.3899/jrheum.161078).

The survey, which the investigators thought to be “the first comprehensive evaluation of influential factors and approaches for the clinical management of children with clinically inactive JIA,” did not include questions about systemic JIA, inflammatory bowel disease, psoriasis, and uveitis in order “to simplify responses and encourage participation, because in practice, the manifestations and outcomes of these diseases could substantially influence treatment decisions for children with JIA.”

They received complete responses from 124 of the 132 clinicians who responded to the survey email. Of the 121 respondents who reported taking clinical care of patients with JIA, 87% were physicians, and the same number reported taking care of pediatric patients only. About three-quarters spent half of their professional time in clinical care, and about half had more than 10 years of post-training clinical experience.

When deciding about withdrawing JIA medications, more than one-half of respondents said that the time that a patient spent in clinically inactive disease and a history of drug toxicity are very important factors. Most participants ranked those two factors most highly and most often among their top five factors for decision making.

Respondents also commonly ranked these factors as important:

• JIA duration before attaining clinically inactive disease.
• Patient/family preferences.
• Presence of JIA-related damage.
• JIA category.

The factors that consistently appeared in responses fit into three clusters that included JIA features and time spent in clinically inactive disease (JIA category and total disease duration), JIA severity and resistance to treatment (disease duration before clinically inactive disease, number of drugs needed to attain inactivity, joint damage, and a history of sacroiliac or temporomandibular disease), and the patient’s experience (drug toxicity and family preference).

The respondents indicated that they would be least likely to stop medications for children with rheumatoid factor (RF)–positive polyarthritis (85%), which is “consistent with prior studies showing that RF-positive polyarthritis is associated with higher rates of flares than other JIA categories,” the investigators wrote. However, respondents said they would be most likely to stop medications for children with persistent oligoarthritis (87%) “even though rates of flares in this category appear similar to other JIA types. This method may reflect a belief that flares in children with persistent oligoarticular JIA will be less severe and easier to control.”

When patients met all criteria for clinically inactive disease for a “sufficient amount of time” and families were interested in stopping medications, some factors continued to make respondents reluctant to withdraw medications. These factors were most often a history of erosions (81%), asymptomatic joint abnormalities on ultrasound or MRI (72%), and failure of multiple prior disease-modifying antirheumatic drugs or biologics to control disease (64%). The definition of clinically inactive disease is a composite of no active arthritis, uveitis, or systemic JIA symptoms; the best possible clinical global assessment; inflammatory markers normal or elevated for reasons other than JIA; and no more than 15 minutes of joint stiffness.

A little over half of respondents said they would wait until clinically inactive disease had lasted 12 months before considering stopping or tapering methotrexate or biologic monotherapy, but a substantial minority said they would wait for only 6 months for methotrexate (31%) or biologic monotherapy (23%). A smaller number would wait for 18 months for methotrexate (13%) or biologics (18%), and another 3%-5% said they could not give a time frame.

The strategies varied for how actual withdrawing of medications occurred. Most methotrexate monotherapy withdrawals involved tapering over 2-6 months, one-third over longer periods, and the fewest reported tapering for less than 2 months (7%) or immediate withdrawal (17%).

Withdrawal of biologics was generally said to occur more gradually than with methotrexate, with one-third of respondents citing over 2-6 months, a quarter more slowly, and another 29% in less than 2 months or immediately. Some wrote that they preferred spacing out the interval between doses, but none decreased the dose. When children took combination therapy with methotrexate plus a biologic, 63% said that they began tapering or stopping methotrexate first, but a quarter said that the order was strongly context dependent, and the most commonly cited reason for deciding was history of toxicity or intolerance.

Imaging played a role in less than half of the decisions to withdraw medications, with it being used often by 9% and sometimes by 36%. And while it’s assumed that patients and family consideration played an important role in decision making, only 25% of respondents reported using specific patient-reported outcomes in deciding to withdraw medications.

The study was funded by grants from Rutgers Biomedical and Health Sciences and the National Institute of Arthritis and Musculoskeletal and Skin Diseases.

 

jevans@frontlinemedcom.com

Our dermatology department recently sponsored a pediatric dermatology lecture series for the pediatric residency program. Within this series, Antonella Tosti, MD, a professor at the University of Miami Health System, Florida, and a renowned expert in nail disorders and allergic contact dermatitis, presented her clinical expertise on the presentation and management of common pediatric nail diseases. This article highlights pearls from her unique and enlightening lecture.

Pearl: Hand-foot-and-mouth disease is a recognized trigger for onychomadesis 

An arrest in nail matrix activity is responsible for onychomadesis, or shedding of the nail. Its presentation in children can be further divided based upon the degree of involvement. If a few nails are affected, trauma should be implicated. In contrast, if all nails are involved, a systemic etiology should be suspected. Hand-foot-and-mouth disease (HFMD) has been recognized as a trigger for onychomadesis in school-aged children. Onychomadesis presents with characteristic proximal nail detachment (Figure 1). The association of HFMD with onychomadesis and Beau lines was first reported in 2000. Five patients who resided within close proximity and shared a physician-diagnosed case of HFMD presented with representative nail findings 4 weeks after illness.¹ Hypotheses for these changes include viral-induced nail pathology, inflammation from cutaneous lesions of HFMD, and systemic effects from the disease.² Given the prevalence of HFMD and benign outcome, clinicians should be cognizant of this unique cutaneous manifestation. 

Pearl: Management of pediatric melanonychia can take a wait-and-see approach

Melanonychia is the presence of a longitudinal brown-black band extending from the proximal nail fold. The cause of melanonychia can be due to either activation or hyperplasia. Activation is the less common etiology in children; however, if present, activation can be due to Laugier-Hunziker syndrome or trauma such as onychotillomania. Melanonychia in children usually is the result of hyperplasia of melanocytes and can manifest as a lentigo, nevus, or more rarely melanoma. Nail matrix nevi are typically exhibited on the fingernails, particularly the thumb, and frequently are junctional nevi (Figure 2). Spontaneous fading of nevi is expected with time due to decreased melanin production. Therapeutic options for melanonychia include regular clinical monitoring, biopsy, or excision. Dr. Tosti explained that one must be wary when pursuing a biopsy, as it can result in a false-negative finding due to missed pathology. If clinically indicated, a shave biopsy of the nail matrix can be performed to best analyze the lesion. She noted that if more than 3 mm of the matrix is removed, a resultant scar will ensue. Conservative management is recommended given the indolent clinical behavior of the majority of cases of melanonychia in children.³

 

Pearl: Congenital hypertrophy of the lateral nail folds can be treated with tape

Congenital hypertrophy of the lateral nail folds is relatively common in children and normally improves with age. Koilonychia may also occur simultaneously and can be viewed as a physiologic process in this age group. The etiology of the underlying disorder is due to anomalous periungual soft-tissue changes of the bilateral halluces; the resulting overgrowth can partially cover the nail plate. Although usually a self-limiting condition, the changes can cause inflammation and discomfort due to an ingrown nail.⁴ Dr. Tosti advised that by simply taping and retracting the bilateral overgrowth, the condition can be more readily resolved. This simple treatment can be demonstrated in the office and subsequently performed at home.

Pearl: Onychomycosis is uncommon in children

Onychomycosis occurs in less than 1% of children.⁵ Several factors are responsible for this decreased prevalence. More rapid nail growth and smaller nail surface area decreases the ability of the fungi to penetrate the nail plate.⁶ Furthermore, children have a diminished rate of tinea pedis, leading to less neighboring infection. When onychomycosis does affect this patient population, it commonly presents as distal subungual onychomycosis and favors the fingernails over the toenails. Treatment options usually parallel those of the adult population; however, all medications for children are considered off-label use by the US Food and Drug Administration. Dr. Tosti explained that oral granules of terbinafine can be sprinkled on food to help with pediatric ingestion. Topical therapies should also be considered; children usually respond better than their adult counterparts due to their thinner nails, which grant enhanced drug delivery and penetration.⁶

Pearl: Acute paronychia can be due to nail-biting and sucking 

Acute paronychia is inflammation of the proximal nail fold. In children, it frequently is a result of mixed flora induced by nail-biting and sucking. Management involves culturing the affected lesions and is effectively treated with warm soaks alone. Dr. Tosti highlighted that Candida in the subungual space is a common colonizer and is typically self-limiting in nature if isolated. Candida can be cultured more readily in premature infants, immunosuppressed patients, and those with chronic mucocutaneous candidiasis. Patients with chronic mucocutaneous candidiasis can exhibit periungual inflammation involving several digits. The differential can include nail psoriasis, as both can demonstrate dystrophic changes. The differential for localized paronychia includes herpetic whitlow and can manifest as vesicles under the proximal nail fold.  

Final Thoughts

These clinical pearls are shared to help deliver utmost care to our pediatric patients presenting with nail pathology. For example, a child exhibiting melanonychia can cause alarm due to the possibility of underlying melanoma; given the rarity of neoplasia in these patients, a conservative approach is favored to help avoid unnecessary biopsies and subsequent scarring. Similarly, it is important to be aware of the common colonizers of the subungual area, particularly Candida, to avoid unessential medications with potential side effects. The examples demonstrated help shed light on the management of pediatric nail diseases.

Acknowledgment

This article is possible thanks to the help of Antonella Tosti, MD (Miami, Florida), who contributed her time and expertise at the University of Miami Pediatric Grand Rounds to expand the foundation and knowledge of pediatric nail diseases.

Our dermatology department recently sponsored a pediatric dermatology lecture series for the pediatric residency program. Within this series, Antonella Tosti, MD, a professor at the University of Miami Health System, Florida, and a renowned expert in nail disorders and allergic contact dermatitis, presented her clinical expertise on the presentation and management of common pediatric nail diseases. This article highlights pearls from her unique and enlightening lecture.

Pearl: Hand-foot-and-mouth disease is a recognized trigger for onychomadesis 

An arrest in nail matrix activity is responsible for onychomadesis, or shedding of the nail. Its presentation in children can be further divided based upon the degree of involvement. If a few nails are affected, trauma should be implicated. In contrast, if all nails are involved, a systemic etiology should be suspected. Hand-foot-and-mouth disease (HFMD) has been recognized as a trigger for onychomadesis in school-aged children. Onychomadesis presents with characteristic proximal nail detachment (Figure 1). The association of HFMD with onychomadesis and Beau lines was first reported in 2000. Five patients who resided within close proximity and shared a physician-diagnosed case of HFMD presented with representative nail findings 4 weeks after illness.¹ Hypotheses for these changes include viral-induced nail pathology, inflammation from cutaneous lesions of HFMD, and systemic effects from the disease.² Given the prevalence of HFMD and benign outcome, clinicians should be cognizant of this unique cutaneous manifestation. 

Pearl: Management of pediatric melanonychia can take a wait-and-see approach

Melanonychia is the presence of a longitudinal brown-black band extending from the proximal nail fold. The cause of melanonychia can be due to either activation or hyperplasia. Activation is the less common etiology in children; however, if present, activation can be due to Laugier-Hunziker syndrome or trauma such as onychotillomania. Melanonychia in children usually is the result of hyperplasia of melanocytes and can manifest as a lentigo, nevus, or more rarely melanoma. Nail matrix nevi are typically exhibited on the fingernails, particularly the thumb, and frequently are junctional nevi (Figure 2). Spontaneous fading of nevi is expected with time due to decreased melanin production. Therapeutic options for melanonychia include regular clinical monitoring, biopsy, or excision. Dr. Tosti explained that one must be wary when pursuing a biopsy, as it can result in a false-negative finding due to missed pathology. If clinically indicated, a shave biopsy of the nail matrix can be performed to best analyze the lesion. She noted that if more than 3 mm of the matrix is removed, a resultant scar will ensue. Conservative management is recommended given the indolent clinical behavior of the majority of cases of melanonychia in children.³

 

Pearl: Congenital hypertrophy of the lateral nail folds can be treated with tape

Congenital hypertrophy of the lateral nail folds is relatively common in children and normally improves with age. Koilonychia may also occur simultaneously and can be viewed as a physiologic process in this age group. The etiology of the underlying disorder is due to anomalous periungual soft-tissue changes of the bilateral halluces; the resulting overgrowth can partially cover the nail plate. Although usually a self-limiting condition, the changes can cause inflammation and discomfort due to an ingrown nail.⁴ Dr. Tosti advised that by simply taping and retracting the bilateral overgrowth, the condition can be more readily resolved. This simple treatment can be demonstrated in the office and subsequently performed at home.

Pearl: Onychomycosis is uncommon in children

Onychomycosis occurs in less than 1% of children.⁵ Several factors are responsible for this decreased prevalence. More rapid nail growth and smaller nail surface area decreases the ability of the fungi to penetrate the nail plate.⁶ Furthermore, children have a diminished rate of tinea pedis, leading to less neighboring infection. When onychomycosis does affect this patient population, it commonly presents as distal subungual onychomycosis and favors the fingernails over the toenails. Treatment options usually parallel those of the adult population; however, all medications for children are considered off-label use by the US Food and Drug Administration. Dr. Tosti explained that oral granules of terbinafine can be sprinkled on food to help with pediatric ingestion. Topical therapies should also be considered; children usually respond better than their adult counterparts due to their thinner nails, which grant enhanced drug delivery and penetration.⁶

Pearl: Acute paronychia can be due to nail-biting and sucking 

Acute paronychia is inflammation of the proximal nail fold. In children, it frequently is a result of mixed flora induced by nail-biting and sucking. Management involves culturing the affected lesions and is effectively treated with warm soaks alone. Dr. Tosti highlighted that Candida in the subungual space is a common colonizer and is typically self-limiting in nature if isolated. Candida can be cultured more readily in premature infants, immunosuppressed patients, and those with chronic mucocutaneous candidiasis. Patients with chronic mucocutaneous candidiasis can exhibit periungual inflammation involving several digits. The differential can include nail psoriasis, as both can demonstrate dystrophic changes. The differential for localized paronychia includes herpetic whitlow and can manifest as vesicles under the proximal nail fold.  

Final Thoughts

These clinical pearls are shared to help deliver utmost care to our pediatric patients presenting with nail pathology. For example, a child exhibiting melanonychia can cause alarm due to the possibility of underlying melanoma; given the rarity of neoplasia in these patients, a conservative approach is favored to help avoid unnecessary biopsies and subsequent scarring. Similarly, it is important to be aware of the common colonizers of the subungual area, particularly Candida, to avoid unessential medications with potential side effects. The examples demonstrated help shed light on the management of pediatric nail diseases.

Acknowledgment

This article is possible thanks to the help of Antonella Tosti, MD (Miami, Florida), who contributed her time and expertise at the University of Miami Pediatric Grand Rounds to expand the foundation and knowledge of pediatric nail diseases.

Our dermatology department recently sponsored a pediatric dermatology lecture series for the pediatric residency program. Within this series, Antonella Tosti, MD, a professor at the University of Miami Health System, Florida, and a renowned expert in nail disorders and allergic contact dermatitis, presented her clinical expertise on the presentation and management of common pediatric nail diseases. This article highlights pearls from her unique and enlightening lecture.

Pearl: Hand-foot-and-mouth disease is a recognized trigger for onychomadesis 

An arrest in nail matrix activity is responsible for onychomadesis, or shedding of the nail. Its presentation in children can be further divided based upon the degree of involvement. If a few nails are affected, trauma should be implicated. In contrast, if all nails are involved, a systemic etiology should be suspected. Hand-foot-and-mouth disease (HFMD) has been recognized as a trigger for onychomadesis in school-aged children. Onychomadesis presents with characteristic proximal nail detachment (Figure 1). The association of HFMD with onychomadesis and Beau lines was first reported in 2000. Five patients who resided within close proximity and shared a physician-diagnosed case of HFMD presented with representative nail findings 4 weeks after illness.¹ Hypotheses for these changes include viral-induced nail pathology, inflammation from cutaneous lesions of HFMD, and systemic effects from the disease.² Given the prevalence of HFMD and benign outcome, clinicians should be cognizant of this unique cutaneous manifestation. 

Pearl: Management of pediatric melanonychia can take a wait-and-see approach

Melanonychia is the presence of a longitudinal brown-black band extending from the proximal nail fold. The cause of melanonychia can be due to either activation or hyperplasia. Activation is the less common etiology in children; however, if present, activation can be due to Laugier-Hunziker syndrome or trauma such as onychotillomania. Melanonychia in children usually is the result of hyperplasia of melanocytes and can manifest as a lentigo, nevus, or more rarely melanoma. Nail matrix nevi are typically exhibited on the fingernails, particularly the thumb, and frequently are junctional nevi (Figure 2). Spontaneous fading of nevi is expected with time due to decreased melanin production. Therapeutic options for melanonychia include regular clinical monitoring, biopsy, or excision. Dr. Tosti explained that one must be wary when pursuing a biopsy, as it can result in a false-negative finding due to missed pathology. If clinically indicated, a shave biopsy of the nail matrix can be performed to best analyze the lesion. She noted that if more than 3 mm of the matrix is removed, a resultant scar will ensue. Conservative management is recommended given the indolent clinical behavior of the majority of cases of melanonychia in children.³

 

Pearl: Congenital hypertrophy of the lateral nail folds can be treated with tape

Congenital hypertrophy of the lateral nail folds is relatively common in children and normally improves with age. Koilonychia may also occur simultaneously and can be viewed as a physiologic process in this age group. The etiology of the underlying disorder is due to anomalous periungual soft-tissue changes of the bilateral halluces; the resulting overgrowth can partially cover the nail plate. Although usually a self-limiting condition, the changes can cause inflammation and discomfort due to an ingrown nail.⁴ Dr. Tosti advised that by simply taping and retracting the bilateral overgrowth, the condition can be more readily resolved. This simple treatment can be demonstrated in the office and subsequently performed at home.

Pearl: Onychomycosis is uncommon in children

Onychomycosis occurs in less than 1% of children.⁵ Several factors are responsible for this decreased prevalence. More rapid nail growth and smaller nail surface area decreases the ability of the fungi to penetrate the nail plate.⁶ Furthermore, children have a diminished rate of tinea pedis, leading to less neighboring infection. When onychomycosis does affect this patient population, it commonly presents as distal subungual onychomycosis and favors the fingernails over the toenails. Treatment options usually parallel those of the adult population; however, all medications for children are considered off-label use by the US Food and Drug Administration. Dr. Tosti explained that oral granules of terbinafine can be sprinkled on food to help with pediatric ingestion. Topical therapies should also be considered; children usually respond better than their adult counterparts due to their thinner nails, which grant enhanced drug delivery and penetration.⁶

Pearl: Acute paronychia can be due to nail-biting and sucking 

Acute paronychia is inflammation of the proximal nail fold. In children, it frequently is a result of mixed flora induced by nail-biting and sucking. Management involves culturing the affected lesions and is effectively treated with warm soaks alone. Dr. Tosti highlighted that Candida in the subungual space is a common colonizer and is typically self-limiting in nature if isolated. Candida can be cultured more readily in premature infants, immunosuppressed patients, and those with chronic mucocutaneous candidiasis. Patients with chronic mucocutaneous candidiasis can exhibit periungual inflammation involving several digits. The differential can include nail psoriasis, as both can demonstrate dystrophic changes. The differential for localized paronychia includes herpetic whitlow and can manifest as vesicles under the proximal nail fold.  

Final Thoughts

These clinical pearls are shared to help deliver utmost care to our pediatric patients presenting with nail pathology. For example, a child exhibiting melanonychia can cause alarm due to the possibility of underlying melanoma; given the rarity of neoplasia in these patients, a conservative approach is favored to help avoid unnecessary biopsies and subsequent scarring. Similarly, it is important to be aware of the common colonizers of the subungual area, particularly Candida, to avoid unessential medications with potential side effects. The examples demonstrated help shed light on the management of pediatric nail diseases.

Acknowledgment

This article is possible thanks to the help of Antonella Tosti, MD (Miami, Florida), who contributed her time and expertise at the University of Miami Pediatric Grand Rounds to expand the foundation and knowledge of pediatric nail diseases.

A pair of transcription factors protect B cells from malignant transformation by keeping the cells’ glucose and energy levels low, according to research published in Nature.

“While transformation to cancer and childhood leukemia takes large amounts of energy, we discovered that low energy levels in B cells protects from malignant transformation toward leukemia and cancer,” said study author Markus Müschen, MD, PhD, of City of Hope Comprehensive Cancer Center in Duarte, California.

“The low energy levels in normal B cells are simply too low to allow transformation to leukemia.”

Dr Müschen and his colleagues found that PAX5 and IKZF1, transcription factors that are critical for early B-cell development, “enforce a state of chronic energy deprivation” that results in constitutive activation of the energy-stress sensor AMPK.

However, dominant-negative mutants of PAX5 and IKZF1 reverse this effect.

Past research has suggested that mutations and deletions in the PAX5 and IKZF1 genes occur in more than 80% of cases of pre-B-cell acute lymphoblastic leukemia (ALL).

In the current study, Dr Müschen and his colleagues found that heterozygous deletion of Pax5 in a mouse model of pre-B ALL greatly increased glucose uptake and ATP levels.

Similarly, when they reconstituted PAX5 and IKZF1 in samples from patients with pre-B ALL, the investigators observed “an energy crisis” that prompted leukemic cell death.

Dr Müschen and his colleagues also performed a CRISPR/Cas9-based screen of PAX5 and IKZF1 transcriptional targets. They said this revealed that NR3C1, TXNIP, and CNR2 are central effectors of B-lymphoid restriction of glucose and energy.

To build upon this finding, the investigators tested TXNIP and CNR2 agonists as well as a small-molecule AMPK inhibitor. They found these compounds synergized with glucocorticoids in patient-derived pre-B ALL cells.

The team therefore concluded that TXNIP, CNR2, and AMPK are potential therapeutic targets for pre-B ALL.

The investigators also said the results of this study support a previous finding that obese children with high blood sugar levels are much more likely to develop drug-resistant leukemia than children who are not overweight. So dieting could be an important consideration for children who have survived leukemia.

“Avoiding obesity and excessive energy supply may help to decrease the risk of leukemia relapse,” said study author Lai Chan, PhD, also of City of Hope.

To test that theory, Drs Chan and Müschen and their colleagues plan to perform experiments in animal models to evaluate the efficacy of dietary restriction on patient-derived childhood leukemia cells, and to assess the activity of drugs that reduce leukemia cells’ glucose and energy supply.

“Based on the outcome of these studies, we plan to introduce dietary restriction and/or glucose-restricting drugs into a clinical trial for children who are at risk to develop leukemia relapse,” Dr Müschen said.

A pair of transcription factors protect B cells from malignant transformation by keeping the cells’ glucose and energy levels low, according to research published in Nature.

“While transformation to cancer and childhood leukemia takes large amounts of energy, we discovered that low energy levels in B cells protects from malignant transformation toward leukemia and cancer,” said study author Markus Müschen, MD, PhD, of City of Hope Comprehensive Cancer Center in Duarte, California.

“The low energy levels in normal B cells are simply too low to allow transformation to leukemia.”

Dr Müschen and his colleagues found that PAX5 and IKZF1, transcription factors that are critical for early B-cell development, “enforce a state of chronic energy deprivation” that results in constitutive activation of the energy-stress sensor AMPK.

However, dominant-negative mutants of PAX5 and IKZF1 reverse this effect.

Past research has suggested that mutations and deletions in the PAX5 and IKZF1 genes occur in more than 80% of cases of pre-B-cell acute lymphoblastic leukemia (ALL).

In the current study, Dr Müschen and his colleagues found that heterozygous deletion of Pax5 in a mouse model of pre-B ALL greatly increased glucose uptake and ATP levels.

Similarly, when they reconstituted PAX5 and IKZF1 in samples from patients with pre-B ALL, the investigators observed “an energy crisis” that prompted leukemic cell death.

Dr Müschen and his colleagues also performed a CRISPR/Cas9-based screen of PAX5 and IKZF1 transcriptional targets. They said this revealed that NR3C1, TXNIP, and CNR2 are central effectors of B-lymphoid restriction of glucose and energy.

To build upon this finding, the investigators tested TXNIP and CNR2 agonists as well as a small-molecule AMPK inhibitor. They found these compounds synergized with glucocorticoids in patient-derived pre-B ALL cells.

The team therefore concluded that TXNIP, CNR2, and AMPK are potential therapeutic targets for pre-B ALL.

The investigators also said the results of this study support a previous finding that obese children with high blood sugar levels are much more likely to develop drug-resistant leukemia than children who are not overweight. So dieting could be an important consideration for children who have survived leukemia.

“Avoiding obesity and excessive energy supply may help to decrease the risk of leukemia relapse,” said study author Lai Chan, PhD, also of City of Hope.

To test that theory, Drs Chan and Müschen and their colleagues plan to perform experiments in animal models to evaluate the efficacy of dietary restriction on patient-derived childhood leukemia cells, and to assess the activity of drugs that reduce leukemia cells’ glucose and energy supply.

“Based on the outcome of these studies, we plan to introduce dietary restriction and/or glucose-restricting drugs into a clinical trial for children who are at risk to develop leukemia relapse,” Dr Müschen said.

A pair of transcription factors protect B cells from malignant transformation by keeping the cells’ glucose and energy levels low, according to research published in Nature.

“While transformation to cancer and childhood leukemia takes large amounts of energy, we discovered that low energy levels in B cells protects from malignant transformation toward leukemia and cancer,” said study author Markus Müschen, MD, PhD, of City of Hope Comprehensive Cancer Center in Duarte, California.

“The low energy levels in normal B cells are simply too low to allow transformation to leukemia.”

Dr Müschen and his colleagues found that PAX5 and IKZF1, transcription factors that are critical for early B-cell development, “enforce a state of chronic energy deprivation” that results in constitutive activation of the energy-stress sensor AMPK.

However, dominant-negative mutants of PAX5 and IKZF1 reverse this effect.

Past research has suggested that mutations and deletions in the PAX5 and IKZF1 genes occur in more than 80% of cases of pre-B-cell acute lymphoblastic leukemia (ALL).

In the current study, Dr Müschen and his colleagues found that heterozygous deletion of Pax5 in a mouse model of pre-B ALL greatly increased glucose uptake and ATP levels.

Similarly, when they reconstituted PAX5 and IKZF1 in samples from patients with pre-B ALL, the investigators observed “an energy crisis” that prompted leukemic cell death.

Dr Müschen and his colleagues also performed a CRISPR/Cas9-based screen of PAX5 and IKZF1 transcriptional targets. They said this revealed that NR3C1, TXNIP, and CNR2 are central effectors of B-lymphoid restriction of glucose and energy.

To build upon this finding, the investigators tested TXNIP and CNR2 agonists as well as a small-molecule AMPK inhibitor. They found these compounds synergized with glucocorticoids in patient-derived pre-B ALL cells.

The team therefore concluded that TXNIP, CNR2, and AMPK are potential therapeutic targets for pre-B ALL.

The investigators also said the results of this study support a previous finding that obese children with high blood sugar levels are much more likely to develop drug-resistant leukemia than children who are not overweight. So dieting could be an important consideration for children who have survived leukemia.

“Avoiding obesity and excessive energy supply may help to decrease the risk of leukemia relapse,” said study author Lai Chan, PhD, also of City of Hope.

To test that theory, Drs Chan and Müschen and their colleagues plan to perform experiments in animal models to evaluate the efficacy of dietary restriction on patient-derived childhood leukemia cells, and to assess the activity of drugs that reduce leukemia cells’ glucose and energy supply.

“Based on the outcome of these studies, we plan to introduce dietary restriction and/or glucose-restricting drugs into a clinical trial for children who are at risk to develop leukemia relapse,” Dr Müschen said.

Population-based surveillance data has revealed patterns of emergency department (ED) visits among Californians with sickle cell disease (SCD).

Previous research suggested that between one-half and two-thirds of SCD patients’ ED visits end in a discharge from the ED, called a treat-and-release visit.

The remainder result in admission to a hospital or other treatment facility.

The purpose of the current study was to use data from the Sickle Cell Data Collection program to describe patterns of ED use for treat-and-release visits by California’s SCD population and compare these new findings with results of previous studies.

The current study was published in Pediatric Blood and Cancer.

Researchers looked at ED and hospital discharge data in California from 2005 to 2014. This included 4636 patients with SCD.

The data showed that 88% of patients had 1 or more treat-and-release ED visits during the 10-year study period.

This group of 4100 patients had 90,904 treat-and-release ED visits. The average number of visits each year was 2.1 (rage, 0-185).

In a single year (2005):

• 53% of patients had no treat-and-release ED visits (no ED use)
• 35% had between 1 and 3 visits (low ED use)
• 9% had between 4 and 10 visits (medium ED use)
• 3% had 11 or more visits (high ED use).

The youngest patients (age 0 to 9.9) and the oldest patients (80 and older) were the least likely to have at least 1 treat-and-release ED visit.

The proportion of patients with at least 1 ED visit over the study period was:

• 68% among patients age 0 to 9.9 at the close of the study
• 80% among patients age 10 to 19.9
• 92% among patients age 20 to 29.9
• 94% among patients age 30 to 39.9
• 93% among patients age 40 to 49.9
• 92% among patients age 50 to 59.9
• 92% among patients age 60 to 69.9
• 85% among patients age 70 to 79.9
• 73% among patients age 80 and older.

The researchers said this study highlights the utility of a multisource, longitudinal data collection effort for SCD. And further study of patients with the highest ED utilization may highlight areas where changes could improve and extend the lives of patients with SCD.

Population-based surveillance data has revealed patterns of emergency department (ED) visits among Californians with sickle cell disease (SCD).

Previous research suggested that between one-half and two-thirds of SCD patients’ ED visits end in a discharge from the ED, called a treat-and-release visit.

The remainder result in admission to a hospital or other treatment facility.

The purpose of the current study was to use data from the Sickle Cell Data Collection program to describe patterns of ED use for treat-and-release visits by California’s SCD population and compare these new findings with results of previous studies.

The current study was published in Pediatric Blood and Cancer.

Researchers looked at ED and hospital discharge data in California from 2005 to 2014. This included 4636 patients with SCD.

The data showed that 88% of patients had 1 or more treat-and-release ED visits during the 10-year study period.

This group of 4100 patients had 90,904 treat-and-release ED visits. The average number of visits each year was 2.1 (rage, 0-185).

In a single year (2005):

• 53% of patients had no treat-and-release ED visits (no ED use)
• 35% had between 1 and 3 visits (low ED use)
• 9% had between 4 and 10 visits (medium ED use)
• 3% had 11 or more visits (high ED use).

The youngest patients (age 0 to 9.9) and the oldest patients (80 and older) were the least likely to have at least 1 treat-and-release ED visit.

The proportion of patients with at least 1 ED visit over the study period was:

• 68% among patients age 0 to 9.9 at the close of the study
• 80% among patients age 10 to 19.9
• 92% among patients age 20 to 29.9
• 94% among patients age 30 to 39.9
• 93% among patients age 40 to 49.9
• 92% among patients age 50 to 59.9
• 92% among patients age 60 to 69.9
• 85% among patients age 70 to 79.9
• 73% among patients age 80 and older.

The researchers said this study highlights the utility of a multisource, longitudinal data collection effort for SCD. And further study of patients with the highest ED utilization may highlight areas where changes could improve and extend the lives of patients with SCD.

Population-based surveillance data has revealed patterns of emergency department (ED) visits among Californians with sickle cell disease (SCD).

Previous research suggested that between one-half and two-thirds of SCD patients’ ED visits end in a discharge from the ED, called a treat-and-release visit.

The remainder result in admission to a hospital or other treatment facility.

The purpose of the current study was to use data from the Sickle Cell Data Collection program to describe patterns of ED use for treat-and-release visits by California’s SCD population and compare these new findings with results of previous studies.

The current study was published in Pediatric Blood and Cancer.

Researchers looked at ED and hospital discharge data in California from 2005 to 2014. This included 4636 patients with SCD.

The data showed that 88% of patients had 1 or more treat-and-release ED visits during the 10-year study period.

This group of 4100 patients had 90,904 treat-and-release ED visits. The average number of visits each year was 2.1 (rage, 0-185).

In a single year (2005):

• 53% of patients had no treat-and-release ED visits (no ED use)
• 35% had between 1 and 3 visits (low ED use)
• 9% had between 4 and 10 visits (medium ED use)
• 3% had 11 or more visits (high ED use).

The youngest patients (age 0 to 9.9) and the oldest patients (80 and older) were the least likely to have at least 1 treat-and-release ED visit.

The proportion of patients with at least 1 ED visit over the study period was:

• 68% among patients age 0 to 9.9 at the close of the study
• 80% among patients age 10 to 19.9
• 92% among patients age 20 to 29.9
• 94% among patients age 30 to 39.9
• 93% among patients age 40 to 49.9
• 92% among patients age 50 to 59.9
• 92% among patients age 60 to 69.9
• 85% among patients age 70 to 79.9
• 73% among patients age 80 and older.

The researchers said this study highlights the utility of a multisource, longitudinal data collection effort for SCD. And further study of patients with the highest ED utilization may highlight areas where changes could improve and extend the lives of patients with SCD.

 

Daptomycin was safe and effective at treating complicated skin and skin structure infections (cSSSI) in children aged 1-17 years, according to Dr. John Bradley of the University of California, San Diego, and his associates.

A total of 389 cSSSI patients were included in the study, with 257 receiving daptomycin and 132 receiving standard-of-care (SOC) treatment (mostly vancomycin or clindamycin). Clinical success rates were similar for both groups, at 91% in the daptomycin group and 87% in the SOC group. Therapeutic success rates also were similar, at 97% in the daptomycin group and 99% in the SOC group.

Courtesy U.S. National Institute of Allergy and Infectious Diseases

Adverse event incidence was similar in both groups, occurring in 14% of daptomycin patients and in 17% of SOC patients. The most common adverse events were diarrhea, occurring in 7% of daptomycin patients and 5% of SOC patients, and increased creatine phosphokinase, occurring in 6% of daptomycin patients and 5% of SOC patients.

“Daptomycin is a suitable once-daily alternative to vancomycin or clindamycin in the pediatric setting, particularly for suspected or confirmed MRSA [methicillin-resistant Staphylococcus aureus] infections,” the investigators concluded.

Find the full study in Pediatrics (doi: 10.1542/peds.2016-2477).
 

lfranki@frontlinemedcom.com

 

Daptomycin was safe and effective at treating complicated skin and skin structure infections (cSSSI) in children aged 1-17 years, according to Dr. John Bradley of the University of California, San Diego, and his associates.

A total of 389 cSSSI patients were included in the study, with 257 receiving daptomycin and 132 receiving standard-of-care (SOC) treatment (mostly vancomycin or clindamycin). Clinical success rates were similar for both groups, at 91% in the daptomycin group and 87% in the SOC group. Therapeutic success rates also were similar, at 97% in the daptomycin group and 99% in the SOC group.

Courtesy U.S. National Institute of Allergy and Infectious Diseases

Adverse event incidence was similar in both groups, occurring in 14% of daptomycin patients and in 17% of SOC patients. The most common adverse events were diarrhea, occurring in 7% of daptomycin patients and 5% of SOC patients, and increased creatine phosphokinase, occurring in 6% of daptomycin patients and 5% of SOC patients.

“Daptomycin is a suitable once-daily alternative to vancomycin or clindamycin in the pediatric setting, particularly for suspected or confirmed MRSA [methicillin-resistant Staphylococcus aureus] infections,” the investigators concluded.

Find the full study in Pediatrics (doi: 10.1542/peds.2016-2477).
 

lfranki@frontlinemedcom.com

 

Daptomycin was safe and effective at treating complicated skin and skin structure infections (cSSSI) in children aged 1-17 years, according to Dr. John Bradley of the University of California, San Diego, and his associates.

A total of 389 cSSSI patients were included in the study, with 257 receiving daptomycin and 132 receiving standard-of-care (SOC) treatment (mostly vancomycin or clindamycin). Clinical success rates were similar for both groups, at 91% in the daptomycin group and 87% in the SOC group. Therapeutic success rates also were similar, at 97% in the daptomycin group and 99% in the SOC group.

Courtesy U.S. National Institute of Allergy and Infectious Diseases

Adverse event incidence was similar in both groups, occurring in 14% of daptomycin patients and in 17% of SOC patients. The most common adverse events were diarrhea, occurring in 7% of daptomycin patients and 5% of SOC patients, and increased creatine phosphokinase, occurring in 6% of daptomycin patients and 5% of SOC patients.

“Daptomycin is a suitable once-daily alternative to vancomycin or clindamycin in the pediatric setting, particularly for suspected or confirmed MRSA [methicillin-resistant Staphylococcus aureus] infections,” the investigators concluded.

Find the full study in Pediatrics (doi: 10.1542/peds.2016-2477).
 

lfranki@frontlinemedcom.com

Granulocyte colony-stimulating factor (G-CSF) could prevent infertility in male cancer patients, according to preclinical research published in Reproductive Biology and Endocrinology.

Researchers said they found that G-CSF protects spermatogenesis after alkylating chemotherapy by stimulating the proliferation of surviving spermatogonia.

The team also found evidence to suggest that G-CSF may be useful as a fertility-restoring treatment.

The researchers have been pursuing initiatives to restore fertility in men who have lost their ability to have children as a result of cancer treatments they received as children.

While working on methods to restart sperm production, the team discovered a link between G-CSF and the absence of normal damage to reproductive ability.

“We were using G-CSF to prevent infections in our research experiments,” said study author Brian Hermann, PhD, of The University of Texas at San Antonio.

“It turned out that the drug also had the unexpected impact of guarding against male infertility.”

To test the fertility-related impact of G-CSF, the researchers treated male mice with G-CSF before and/or after treatment with busulfan.

The team then evaluated effects on spermatogenesis in these mice and control mice that only received busulfan.

G-CSF had a protective effect on spermatogenesis that was stable for at least 19 weeks after chemotherapy.

And mice treated with G-CSF for 4 days after busulfan showed modestly enhanced spermatogenic recovery compared to controls.

The researchers said these results suggest G-CSF promotes spermatogonial proliferation, leading to enhanced spermatogenic regeneration from surviving spermatogonial stem cells.

Granulocyte colony-stimulating factor (G-CSF) could prevent infertility in male cancer patients, according to preclinical research published in Reproductive Biology and Endocrinology.

Researchers said they found that G-CSF protects spermatogenesis after alkylating chemotherapy by stimulating the proliferation of surviving spermatogonia.

The team also found evidence to suggest that G-CSF may be useful as a fertility-restoring treatment.

The researchers have been pursuing initiatives to restore fertility in men who have lost their ability to have children as a result of cancer treatments they received as children.

While working on methods to restart sperm production, the team discovered a link between G-CSF and the absence of normal damage to reproductive ability.

“We were using G-CSF to prevent infections in our research experiments,” said study author Brian Hermann, PhD, of The University of Texas at San Antonio.

“It turned out that the drug also had the unexpected impact of guarding against male infertility.”

To test the fertility-related impact of G-CSF, the researchers treated male mice with G-CSF before and/or after treatment with busulfan.

The team then evaluated effects on spermatogenesis in these mice and control mice that only received busulfan.

G-CSF had a protective effect on spermatogenesis that was stable for at least 19 weeks after chemotherapy.

And mice treated with G-CSF for 4 days after busulfan showed modestly enhanced spermatogenic recovery compared to controls.

The researchers said these results suggest G-CSF promotes spermatogonial proliferation, leading to enhanced spermatogenic regeneration from surviving spermatogonial stem cells.

Granulocyte colony-stimulating factor (G-CSF) could prevent infertility in male cancer patients, according to preclinical research published in Reproductive Biology and Endocrinology.

Researchers said they found that G-CSF protects spermatogenesis after alkylating chemotherapy by stimulating the proliferation of surviving spermatogonia.

The team also found evidence to suggest that G-CSF may be useful as a fertility-restoring treatment.

The researchers have been pursuing initiatives to restore fertility in men who have lost their ability to have children as a result of cancer treatments they received as children.

While working on methods to restart sperm production, the team discovered a link between G-CSF and the absence of normal damage to reproductive ability.

“We were using G-CSF to prevent infections in our research experiments,” said study author Brian Hermann, PhD, of The University of Texas at San Antonio.

“It turned out that the drug also had the unexpected impact of guarding against male infertility.”

To test the fertility-related impact of G-CSF, the researchers treated male mice with G-CSF before and/or after treatment with busulfan.

The team then evaluated effects on spermatogenesis in these mice and control mice that only received busulfan.

G-CSF had a protective effect on spermatogenesis that was stable for at least 19 weeks after chemotherapy.

And mice treated with G-CSF for 4 days after busulfan showed modestly enhanced spermatogenic recovery compared to controls.

The researchers said these results suggest G-CSF promotes spermatogonial proliferation, leading to enhanced spermatogenic regeneration from surviving spermatogonial stem cells.

 

Clinical question: What are the epidemiology, risk factors, and associated morbidity and mortality of acute kidney injury (AKI) in critically ill children and young adults?

Background: Adult studies on acute kidney injury have shown increasing mortality and morbidity when both plasma creatinine and urine output were used to diagnose AKI than when used alone. Studies of AKI in children have also been limited.

Study design: Prospective, observational study.

Setting: International (32 pediatric intensive care units across Asia, Australia, Europe, and North America).

Synopsis: 4,984 patients aged 3 months to 25 years with a predicted ICU stay of at least 48 hours were considered for enrollment, of which 4,683 were included in the final analysis. The primary outcome was 28-day mortality. Secondary outcomes were length of stay in the ICU, receipt and duration of mechanical ventilation, receipt of extracorporeal membrane oxygenation, and renal-replacement therapy. A total of 26.9% of patients developed AKI in the first 7 days of an ICU admission. Severe AKI increased mortality by day 28 (adjusted odds ratio, 1.77; 95% confidence interval, 1.17-2.68) and was associated with increased use of renal-replacement therapy and mechanical ventilation and longer stays in the ICU. Urine output predicted mortality more accurately than did plasma creatinine, and using plasma creatinine alone failed to identify two-thirds of patients with low urine output.

Bottom line: In critically ill young patients, AKI is a common occurrence and is associated with both an increased morbidity and mortality. In a majority of patients with low urine output, plasma creatinine was a poor discriminant of renal function.

Citations: Kaddourah A, Basu RK, Bagshaw SM, et al. Epidemiology of acute kidney injury in critically ill children and young adults. N Eng J Med. 2017; 376 (1):11-20.

Dr. Rachoin is an assistant professor of clinical medicine and associate division head, Hospital Medicine, at Cooper Medical School at Rowan University. He works as a hospitalist at Cooper University Hospital in Camden, N.J.

 

Clinical question: What are the epidemiology, risk factors, and associated morbidity and mortality of acute kidney injury (AKI) in critically ill children and young adults?

Background: Adult studies on acute kidney injury have shown increasing mortality and morbidity when both plasma creatinine and urine output were used to diagnose AKI than when used alone. Studies of AKI in children have also been limited.

Study design: Prospective, observational study.

Setting: International (32 pediatric intensive care units across Asia, Australia, Europe, and North America).

Synopsis: 4,984 patients aged 3 months to 25 years with a predicted ICU stay of at least 48 hours were considered for enrollment, of which 4,683 were included in the final analysis. The primary outcome was 28-day mortality. Secondary outcomes were length of stay in the ICU, receipt and duration of mechanical ventilation, receipt of extracorporeal membrane oxygenation, and renal-replacement therapy. A total of 26.9% of patients developed AKI in the first 7 days of an ICU admission. Severe AKI increased mortality by day 28 (adjusted odds ratio, 1.77; 95% confidence interval, 1.17-2.68) and was associated with increased use of renal-replacement therapy and mechanical ventilation and longer stays in the ICU. Urine output predicted mortality more accurately than did plasma creatinine, and using plasma creatinine alone failed to identify two-thirds of patients with low urine output.

Bottom line: In critically ill young patients, AKI is a common occurrence and is associated with both an increased morbidity and mortality. In a majority of patients with low urine output, plasma creatinine was a poor discriminant of renal function.

Citations: Kaddourah A, Basu RK, Bagshaw SM, et al. Epidemiology of acute kidney injury in critically ill children and young adults. N Eng J Med. 2017; 376 (1):11-20.

Dr. Rachoin is an assistant professor of clinical medicine and associate division head, Hospital Medicine, at Cooper Medical School at Rowan University. He works as a hospitalist at Cooper University Hospital in Camden, N.J.

 

Clinical question: What are the epidemiology, risk factors, and associated morbidity and mortality of acute kidney injury (AKI) in critically ill children and young adults?

Background: Adult studies on acute kidney injury have shown increasing mortality and morbidity when both plasma creatinine and urine output were used to diagnose AKI than when used alone. Studies of AKI in children have also been limited.

Study design: Prospective, observational study.

Setting: International (32 pediatric intensive care units across Asia, Australia, Europe, and North America).

Synopsis: 4,984 patients aged 3 months to 25 years with a predicted ICU stay of at least 48 hours were considered for enrollment, of which 4,683 were included in the final analysis. The primary outcome was 28-day mortality. Secondary outcomes were length of stay in the ICU, receipt and duration of mechanical ventilation, receipt of extracorporeal membrane oxygenation, and renal-replacement therapy. A total of 26.9% of patients developed AKI in the first 7 days of an ICU admission. Severe AKI increased mortality by day 28 (adjusted odds ratio, 1.77; 95% confidence interval, 1.17-2.68) and was associated with increased use of renal-replacement therapy and mechanical ventilation and longer stays in the ICU. Urine output predicted mortality more accurately than did plasma creatinine, and using plasma creatinine alone failed to identify two-thirds of patients with low urine output.

Bottom line: In critically ill young patients, AKI is a common occurrence and is associated with both an increased morbidity and mortality. In a majority of patients with low urine output, plasma creatinine was a poor discriminant of renal function.

Citations: Kaddourah A, Basu RK, Bagshaw SM, et al. Epidemiology of acute kidney injury in critically ill children and young adults. N Eng J Med. 2017; 376 (1):11-20.

Dr. Rachoin is an assistant professor of clinical medicine and associate division head, Hospital Medicine, at Cooper Medical School at Rowan University. He works as a hospitalist at Cooper University Hospital in Camden, N.J.

 

High adherence to bronchiolitis clinical pathway recommendations in health care settings is associated with shorter length of stay (LOS) and lower health care costs, according to Mersine A. Bryan, MD, of the University of Washington, Seattle and her associates.

In a retrospective cohort study, researchers looked at 267 patients less than 24 months old diagnosed with bronchiolitis from December 2009 to July 2012. Levels of adherence were then categorized into low, middle, and high tertiles. Results show that adherence was highest for the inpatient quality indicators (mean score, 95) and lowest for the emergency department (ED) quality indicators (mean score, 79). The mean ED LOS was significantly shorter for cases with ED adherence scores in the highest versus the lowest tertile (90 vs. 140 minutes; P less than .05). There were no significant differences in mean inpatient LOS by inpatient adherence score tertiles. “However, the mean inpatient LOS was approximately 17 hours shorter for cases with combined ED/inpatient adherence scores in the highest, compared with the lowest tertile,” they said.

peterspiro/Thinkstock

The mean ED costs for cases with ED adherence scores in the highest tertile were significantly lower than cases with scores in the lowest tertile (–$84; P less than .05). It is noted there were no significant differences in mean total costs by inpatient adherence score tertile, but “for cases where the combined ED/inpatient adherence scores were in the highest tertile, the mean total costs were significantly lower than for cases with combined adherence scores in the lowest tertile, the researchers noted. Also, cases with ED adherence scores in the highest tertile had lower odds of inpatient admission, compared with those with scores in the lowest tertile (odds ratio, 0.38). There were no significant differences in the odds of return ED visits or readmissions by adherence score tertile.

“Our study demonstrates that high adherence to evidence-based recommendations within a clinical pathway across the entire continuum of care, from the ED to the inpatient setting, is associated with lower costs and shorter LOS,” Dr. Bryan and associates concluded. “By improving adherence to evidence-based recommendations within a clinical pathway, we may be able to provide higher-value care by optimizing the quality of bronchiolitis care at lower costs and with shorter LOS.”

Read the full study in Pediatrics (doi: 10.1542/peds.2016-3432).

llaubach@frontlinemedcom.com

 

High adherence to bronchiolitis clinical pathway recommendations in health care settings is associated with shorter length of stay (LOS) and lower health care costs, according to Mersine A. Bryan, MD, of the University of Washington, Seattle and her associates.

In a retrospective cohort study, researchers looked at 267 patients less than 24 months old diagnosed with bronchiolitis from December 2009 to July 2012. Levels of adherence were then categorized into low, middle, and high tertiles. Results show that adherence was highest for the inpatient quality indicators (mean score, 95) and lowest for the emergency department (ED) quality indicators (mean score, 79). The mean ED LOS was significantly shorter for cases with ED adherence scores in the highest versus the lowest tertile (90 vs. 140 minutes; P less than .05). There were no significant differences in mean inpatient LOS by inpatient adherence score tertiles. “However, the mean inpatient LOS was approximately 17 hours shorter for cases with combined ED/inpatient adherence scores in the highest, compared with the lowest tertile,” they said.

peterspiro/Thinkstock

The mean ED costs for cases with ED adherence scores in the highest tertile were significantly lower than cases with scores in the lowest tertile (–$84; P less than .05). It is noted there were no significant differences in mean total costs by inpatient adherence score tertile, but “for cases where the combined ED/inpatient adherence scores were in the highest tertile, the mean total costs were significantly lower than for cases with combined adherence scores in the lowest tertile, the researchers noted. Also, cases with ED adherence scores in the highest tertile had lower odds of inpatient admission, compared with those with scores in the lowest tertile (odds ratio, 0.38). There were no significant differences in the odds of return ED visits or readmissions by adherence score tertile.

“Our study demonstrates that high adherence to evidence-based recommendations within a clinical pathway across the entire continuum of care, from the ED to the inpatient setting, is associated with lower costs and shorter LOS,” Dr. Bryan and associates concluded. “By improving adherence to evidence-based recommendations within a clinical pathway, we may be able to provide higher-value care by optimizing the quality of bronchiolitis care at lower costs and with shorter LOS.”

Read the full study in Pediatrics (doi: 10.1542/peds.2016-3432).

llaubach@frontlinemedcom.com

 

High adherence to bronchiolitis clinical pathway recommendations in health care settings is associated with shorter length of stay (LOS) and lower health care costs, according to Mersine A. Bryan, MD, of the University of Washington, Seattle and her associates.

In a retrospective cohort study, researchers looked at 267 patients less than 24 months old diagnosed with bronchiolitis from December 2009 to July 2012. Levels of adherence were then categorized into low, middle, and high tertiles. Results show that adherence was highest for the inpatient quality indicators (mean score, 95) and lowest for the emergency department (ED) quality indicators (mean score, 79). The mean ED LOS was significantly shorter for cases with ED adherence scores in the highest versus the lowest tertile (90 vs. 140 minutes; P less than .05). There were no significant differences in mean inpatient LOS by inpatient adherence score tertiles. “However, the mean inpatient LOS was approximately 17 hours shorter for cases with combined ED/inpatient adherence scores in the highest, compared with the lowest tertile,” they said.

peterspiro/Thinkstock

The mean ED costs for cases with ED adherence scores in the highest tertile were significantly lower than cases with scores in the lowest tertile (–$84; P less than .05). It is noted there were no significant differences in mean total costs by inpatient adherence score tertile, but “for cases where the combined ED/inpatient adherence scores were in the highest tertile, the mean total costs were significantly lower than for cases with combined adherence scores in the lowest tertile, the researchers noted. Also, cases with ED adherence scores in the highest tertile had lower odds of inpatient admission, compared with those with scores in the lowest tertile (odds ratio, 0.38). There were no significant differences in the odds of return ED visits or readmissions by adherence score tertile.

“Our study demonstrates that high adherence to evidence-based recommendations within a clinical pathway across the entire continuum of care, from the ED to the inpatient setting, is associated with lower costs and shorter LOS,” Dr. Bryan and associates concluded. “By improving adherence to evidence-based recommendations within a clinical pathway, we may be able to provide higher-value care by optimizing the quality of bronchiolitis care at lower costs and with shorter LOS.”

Read the full study in Pediatrics (doi: 10.1542/peds.2016-3432).

llaubach@frontlinemedcom.com