Growth Hormone Research Society Workshop Summary: Consensus Guidelines for Recombinant Human Growth Hormone Therapy in Prader-Willi Syndrome

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 aimof the studywas 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, includingrandomizedcontrolledtrials,comparativeobservationalstudies,andlong-termstudies(3.5 y). Adult studies included randomized controlled trials of rhGH treatment for 6months and uncon-trolled trials. Safetydatawereobtained fromcase reports, clinical trials, andpharmaceutical registries. Methodology: Forty-three international experts and stakeholders followed clinical practice guide-line development recommendations outlined by the AGREE Collaboration (www.agreetrust.org). Evidencewas synthesized and graded using a comprehensivemulticriteriamethodology (EVIDEM