We’ve had a flurry of activity this week at Children’s Hospital of Philadelphia Research Institute, including our first snowstorm of the year. In between shoveling, we took a few minutes to dig into the latest research news.
New Guidelines for Children With Growth Failure Highlight Need for More Research
A group of medical experts has weighed existing evidence and issued a new set of clinical guidelines for managing children and adolescents with growth failure. Written on behalf of the Pediatric Endocrine Society, the guidelines are the society’s first update since 2003.
“The nuances of this issue leave much room for open questions and differences of interpretation,” said study leader Adda Grimberg, MD, a pediatric endocrinologist at CHOP. “In developing these guidelines, we analyzed not only the results, but the strengths, limitations, and potential biases of studies in a large evidence base that continues to evolve.”
For children with certain clearly diagnosed medical conditions — growth hormone deficiency (GHD) and primary IGF-I deficiency (PIGFD) — the experts recommend hormonal treatments. When the cause of growth failure is unknown — idiopathic short stature (ISS) — they advise against routine growth hormone use and recommend a shared decision-making approach, weighing physical and psychological burdens for the child, along with a discussion of risks and benefits. Given the current state of knowledge, there are ethical questions in addition to medical questions surrounding growth hormone treatment for short children who don't have a disease.
“Administering growth hormone treatment may help very short children gain a few inches in height, but it also exposes them to a powerful hormone when we do not fully know the long-term implications,” said co-author Chris Feudtner, MD, PhD, a CHOP pediatrician and director of the hospital’s department of Medical Ethics.
While endorsing further research to address the many unanswered questions in the field, the authors recommend that only pediatric endocrinologists manage evaluation and treatment for GHD, PIGFD, and ISS in children.
National Trial Compares Lower, Higher Glucose Target Ranges for Critically Ill Children
Critically ill children don’t benefit from tight glycemic control medical practice, according to a landmark clinical trial called HALF-PINT (for Heart and Lung Failure Pediatric INsulin Titration Trial). Vinay Nadkarni, MD, a critical care specialist at CHOP, is co-principal investigator of the study.
The HALF-PINT trial focused on critically ill children without cardiac surgical conditions, an understudied population. The study team enrolled 713 critically ill children ranging from 2 weeks old to age 17 years, from 35 pediatric centers. All the children had hyperglycemia (high blood glucose levels). The children were randomized into two groups. In one group, clinicians aimed for lower glucose control targets — 80 to 110 milligrams per deciliter, while the other group had a higher target — 150 to 180 mg per deciliter.
Between the two treatment groups, there were no significant differences in ICU-free days, mortality, organ dysfunction, or ventilator-free days. While both levels of blood sugar control were relatively safe, control to lower levels increased the risk of a very low blood sugar (hypoglycemia) without offering a significant advantage.
“These results complement recent findings from two important studies in related pediatric critical care populations,” Dr. Nadkarni said. “It’s clear that high blood sugars are associated with worse outcomes in children, but controlling blood sugar to lower targets within the usual care range does not appear to provide additional benefit.”
Dr. Nadkarni added that follow-up research will continue with study patients, to evaluate survival and neurological outcomes a year after treatment.
The HALF-PINT study was sponsored by the National Institutes of Health, and the results appeared in the New England Journal of Medicine (NEJM), concurrent with a presentation in the late-breaking science session at the annual meeting of the Society for Critical Care Medicine (SCCM) in Honolulu.
Dr. Nadkarni and co-workers at CHOP participated in another multicenter pediatric clinical trial presented at the SCCM late-breaking research session and published in the same issue of NEJM. The THAPCA (Therapeutic Hypothermia After Pediatric Cardiac Arrest) in-hospital study compared two levels of targeted temperature management in 329 children who survived in-hospital cardiac arrests. The group found that targeting lower body temperature to 33 degrees Celsius (91 degrees Fahrenheit) had no benefit in survival with favorable functional outcome at one year, compared to targeted temperature management to 36.8 degrees Celsius (98 degrees Fahrenheit) for five days.
Read more in the CHOP press release.
What’s At Stake for Children if CHIP Funding Is Not Renewed
Children must not be left on the sidelines of the healthcare debate. CHOP President and Chief Executive Officer Madeline Bell explains why in an opinion piece featured in The Hill and discusses new PolicyLab at CHOP research that “shines a light on the urgent need to ensure Medicaid and CHIP continue to provide vital, affordable coverage to children in working families.”
Children in the U.S. have nearly universal coverage rates — 95 percent in 2015 — and this success relies on Medicaid and the Children’s Health Insurance Program (CHIP). A common misperception is that most children covered by Medicaid and CHIP are from families who are unemployed. In fact, a new study from PolicyLab at CHOP found that the fastest growing users of Medicaid and CHIP are families in which the parents are insured by their employer, but cannot afford, or were not offered, family coverage.
“But, without congressional action early this year, CHIP will cease to exist,” Bell writes. “And threatened changes to Medicaid would decimate the nearly universal children’s coverage rates we’ve achieved. It is imperative that neither the action nor the inaction of Congress force a decline in children’s health coverage.”
New Penn/CHOP Research Informs Interventions for Global Road Safety
“We know what works to improve traffic safety, such as seat belt use and driving attentively; however, how these play out and whether these behaviors are adopted has to be adapted 'glocally',” said Flaura Winston, MD, PhD, founder and scientific director of the Center for Injury Research and Prevention at CHOP. “This ‘glocal’ approach takes global issues and adapts them to a local context.”
That is the aim of a research team led by the University of Pennsylvania School of Nursing (Penn Nursing) and CIRP who worked with a major U.S. multinational corporation to investigate employee perceptions of road risks and strategies to reduce road traffic injuries. This research was conducted in two Indian cities with some of the highest road traffic injury rates worldwide that are also centers for multinational corporations in the software and technology sectors.
Through interviews, focus groups, and surveys of local representatives of a global workforce as part of health risk assessment tools or as a series of stand-alone inquiries, the study offers a replicable and straightforward methodology to gather employee perspective for local road traffic intervention strategies.
The study, “Road Safety Perspectives Among Employees of a Multinational Corporation in Urban India: Local Context for Global Injury Prevention,” appeared in the International Journal of Injury Control and Safety Promotion.
Read more in the Penn Nursing press release.
This week on Cornerstone we welcomed the Philadelphia Coalition for a Cure. The collaboration is the nation’s first city-wide brain tumor precision-medicine research partnership to benefit both pediatric and adult patients with brain tumors.
And here are our top headlines from our Jan. 27 segment of In the News:
- Consumer-Use Vital Signs Monitors May Cause Parents Undue Alarm
- Beckwith-Wiedemann Syndrome Registry Helping to Find Cancer Mechanisms
- Exploring CAR T-cell Therapy
- Three Developers of Medical Devices for Children Receive Grants
- CHOP Expert Signs Mitochondrial Medicine Research Agreement
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