27 Jan Guidelines Address Treating Short Children With Hormone Therapy
MedicalResearch.com Interview with:
Adda Grimberg, MD
Associate Professor of Pediatrics,
Perelman School of Medicine, University of Pennsylvania
Scientific Director, Diagnostic and Research Growth Center
The Children’s Hospital of Philadelphia
Philadelphia, PA 19104
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: This study sought to update the last guidelines for the use of growth hormone (GH) by the Drug and Therapeutics Committee of the Pediatric Endocrine Society (PES), published in 2003, and was the first to be endorsed also by the Ethics Committee of the PES. To facilitate evidence-based decision making, it was the first such GH guidelines to follow the approach recommended by the Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) group. Because idiopathic short stature (ISS) remains a controversial indication, and diagnostic challenges often blur the distinction between ISS, growth hormone deficiency (GHD), and primary insulin-like growth factor-I deficiency (PIGFD), this guidelines statement focused on these three diagnoses and added recombinant IGF-I therapy to the GH guidelines for the first time.
MedicalResearch.com: What should readers take away from your report?
Response: Short stature itself is not a disease, and part of the complexity of the issue is about whether children without actual hormone deficiency should receive a costly, nightly treatment, in which long-term risks are unknown. There are strong supporting evidence and general agreement regarding the restoration of hormonal normalcy in children with severe deficiency of growth hormone or IGF-I. More complex are issues related to the use of growth hormone treatment to increase growth rates and heights of otherwise healthy short children with either ISS or “partial” isolated idiopathic GHD, who are distinguished from each other only by their GH testing results.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: This extensive evidence review highlighted areas that need further research, including fundamental questions that heretofore have been based mostly on underlying assumptions about height, quality of life, and the value of treating short stature per se with growth hormone. The two most pressing research needs are the need for better outcome metrics and for better diagnostics. Evidence-based practice innovation will be hampered without clearly defining what we are trying to achieve and whom we are studying.
MedicalResearch.com: Is there anything else you would like to add?
Response: The rigorous evidence-based methodologies of the GRADE approach resulted in a more conservative guidelines than previous documents. The lack of studies of sufficient quality in support of a practice is not the same as evidence against the practice; until such studies can be performed, individualization of clinical care remains the central tenet of therapy.
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Adda Grimberg, Sara A. DiVall, Constantin Polychronakos, David B. Allen, Laurie E. Cohen, Jose Bernardo Quintos, Wilma C. Rossi, Chris Feudtner, Mohammad Hassan Murad. Hormone Research in Paediatrics, 2016; 86 (6): 361 DOI: 10.1159/000452150
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