Children’s Doctor News and Updates: Winter 2018

When to test thyroid function in children with alopecia areata

Experts from CHOP’s Division of Endocrinology and Diabetes and the Dermatology Section analyzed data from 298 children with alopecia areata (AA) and found a history of Down syndrome, atopy, and family history of thyroid disease had significant association with the incidence of thyroid abnormalities, but the patient’s age at diagnosis, duration of the disease, and pattern of AA did not.

“Because hair thinning and hair loss are commonly found in patients with hypothyroidism, a significant percent of patients with alopecia areata have thyroid function testing performed in an effort to find an etiology and potential treatment,” says endocrinologist Andrew Bauer, MD, Medical Director of the Pediatric Thyroid Center and co-author of the study in JAMA Pediatrics.

“This study helps frame when to test children with new alopecia areata for thyroid abnormalities so that unnecessary blood testing is avoided,” says dermatologist and co-author Leslie Castelo-Soccio, MD, PhD. Other benefits include reducing healthcare costs and standardizing clinical practices to characterize and establish guidelines for screening thyroid function in children with AA. Read more about the study.

Study: bone density lags behind height

CHOP researchers Shana McCormack, MD, MTR, an endocrinologist, and Babette Zemel, PhD, Director of the Nutrition and Growth Laboratory, were leaders of a multi-institution study team that found that bone mineral density develops at different rates in different parts of the skeleton and that teenagers continue to accumulate approximately 10% of their bone mass after they have reached their adult height.

The researchers analyzed data from 2014 healthy children who participated in the Bone Mineral Density in Childhood Study, which included sophisticated bone and growth measurements via dual-energy X-ray absorptiometry at annual visits for up to 7 years from 2002 through 2010. The participants were ages 5 to 19 when they joined the study, which was published in JAMA Pediatrics.

“This study reinforces that understanding and suggests that late adolescence may be an under-recognized period to intervene in this important area of public health,” Zemel says. The “lag” in bone mineral accrual may help explain the high fracture rates among children and adolescents. Thirty percent to 50% of children experience at least 1 bone fracture prior to adulthood.

CHOP co-leads development of guidelines for managing mitochondrial disease

Physicians who see patients with mitochondrial disease now have a set of guidelines for managing and caring for those patients. Occurring in at least 1 in 4,500 individuals, mitochondrial disease is caused by defects in genes affecting the function of mitochondria, crucial energy-producing structures found in every cell. This complex disease can affect nearly every organ or system in the body.

“Standards of care are not uniform across centers or clinicians,” says study co-leader Amy Goldstein, MD, Clinical Director of the Mitochondrial Medicine at CHOP and current President of the Mitochondrial Medicine Society (MMS), which issued the consensus statement. “To take just one example, clinicians need to know how often to order laboratory tests to monitor the health of patients with mitochondrial disease. Our guidelines reflect expert consensus based on our current knowledge of mitochondrial medicine.”

The MMS patient care standards for primary mitochondrial disease were published in Genetics in Medicine. It is a follow-up to MMS’s 2015 guide to diagnosis of mitochondrial disease.

Families report favorable quality of life for cardiac patients treated with ECMO

CHOP cardiologists Matthew D. Elias, MD, and Matthew J. O'Connor, MD, led a study that revealed that patients and parents of cardiac patients treated in childhood with extracorporeal membrane oxygenation (ECMO) overwhelmingly reported positive outcomes regarding health and physical limitations. More than 90% of families reported good or excellent health, and approximately 86% reported no or mild physical limitations.

However, the authors noted a discrepancy between family-reported favorable outcomes and a relatively high rate of medical and behavioral issues revealed by more detailed questioning. Almost 25% of patients had below-average school performance and required special education, and almost 50% had parental-reported learning disabilities.

Elias recommends a multisite study, and he says, “In the meantime, our findings should allow for improved family counseling in discussing long-term quality of life for children with heart disease.” Read more about the study.

Proton lowers treatment side effects in pediatric head and neck cancer patients

Pediatric patients with head and neck cancer can be treated with proton beam therapy (PBT) instead of traditional photon radiation, and it will result in similar outcomes with less impact on quality of life. Researchers from the Perelman School of Medicine at the University of Pennsylvania and Children’s Hospital analyzed cases of 69 pediatric head and neck cancer treated with PBT between 2010 and 2016 and found similar rates of tumor control and lower rates of toxicity than what is historically expected from photon radiation.

The head/neck area’s sensitivity means the effects of treatment can lower patient quality of life due to symptoms including loss of appetite, difficulty swallowing, or mucositis. For example, 4% of patients treated with PBT reported mucositis at grade 3 (on a 1 to 5 scale) while 46% of patients treated with photon radiation historically report grade 3 or 4 mucositis. Study findings were published in Pediatric Blood and Cancer. Read more about the study.