Growth hormone research society workshop summary: Consensus guidelines for recombinant human growth hormone therapy in Prader-Willi syndrome

Cheri L. Deal, Michèle Tony, Charlotte Hoÿbye, David B. Allen, Maïth́e Tauber, Jens Sandahl Christiansen, Geoffrey R. Ambler, Renaldo Battista, Véronique Beauloye, Glenn Berall, Beverly M K Biller, Merlin G. Butler, Suzanne B. Cassidy, Kazuo Chihara, Pinchas Cohen, Maria Craig, Stense Farholt, Mireille Goetghebeur, Anthony P. Goldstone, Tiziana GreggiGraziano Grugni, Anita C. Hokken-Koelega, Gudmundur Johannsson, Keegan Johnson, Alex Kemper, John J. Kopchick, Saul Malozowski, Jennifer Miller, Harriette R. Mogul, Françoise Muscatelli, Ricard Nergårdh, Robert D. Nicholls, Sally Radovick, M. Sara Rosenthal, Ilkka Sipilä, Jean Eric Tarride, Annick Vogels, Michael J. Waters

Research output: Contribution to journalArticle

Abstract

Context: Recombinant human GH (rhGH) therapy in Prader-Willi syndrome (PWS) has been used by the medical community and advocated by parental support groups since its approval in the United States in 2000 and in Europe in 2001. Its use in PWS represents a unique therapeutic challenge that includes treating individuals with cognitive disability, varied therapeutic goals that are not focused exclusively on increased height, and concerns about potential life-threatening adverse events. Objective: The aim of the study was to formulate recommendations for the use of rhGH in children and adult patients with PWS. Evidence: We performed a systematic review of the clinical evidence in the pediatric population, including randomized controlled trials, comparative observational studies, and long-term studies (>3.5 y). Adult studies included randomized controlled trials of rhGH treatment for ≥6 months and uncontrolled trials. Safety data were obtained from case reports, clinical trials, and pharmaceutical registries. Methodology: Forty-three international experts and stakeholders followed clinical practice guideline development recommendations outlined by the AGREE Collaboration (www.agreetrust.org). Evidence was synthesized and graded using a comprehensive multicriteria methodology (EVIDEM) (http://bit.ly.PWGHIN). Conclusions: Following a multidisciplinary evaluation, preferably by experts, rhGH treatment should be considered for patients with genetically confirmed PWS in conjunction with dietary, environmental, and lifestyle interventions. Cognitive impairment should not be a barrier to treatment, and informed consent/assent should include benefit/risk information. Exclusion criteria should include severe obesity, uncontrolled diabetes mellitus, untreated severe obstructive sleep apnea, active cancer, or psychosis. Clinical outcome priorities should vary depending upon age and the presence of physical, mental, and social disability, and treatment should be continued for as long as demonstrated benefits outweigh the risks.

Original languageEnglish (US)
JournalJournal of Clinical Endocrinology and Metabolism
Volume98
Issue number6
DOIs
StatePublished - Jun 2013

ASJC Scopus subject areas

  • Biochemistry
  • Clinical Biochemistry
  • Endocrinology
  • Biochemistry, medical
  • Endocrinology, Diabetes and Metabolism
  • Medicine(all)

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    Deal, C. L., Tony, M., Hoÿbye, C., Allen, D. B., Tauber, M., Christiansen, J. S., Ambler, G. R., Battista, R., Beauloye, V., Berall, G., Biller, B. M. K., Butler, M. G., Cassidy, S. B., Chihara, K., Cohen, P., Craig, M., Farholt, S., Goetghebeur, M., Goldstone, A. P., ... Waters, M. J. (2013). Growth hormone research society workshop summary: Consensus guidelines for recombinant human growth hormone therapy in Prader-Willi syndrome. Journal of Clinical Endocrinology and Metabolism, 98(6). https://doi.org/10.1210/jc.2012-3888