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

Context: rhGH therapy in Prader-Willi syndrome (PWS) has been used by the medical community andadvocatedbyparental supportgroups since itsapproval in theUS in2000and inEurope in2001. Its use in PWS represents a unique therapeutic challenge which 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: To formulate recommendations for the use of rhGH in children and adult patients with PWS. Evidence: A systematic review of the clinical evidence in the pediatric population, including ran-domized controlled trials (RCTs), comparative observational studies and long term studies (3.5 years). Adult studies included RCTs of rhGH treatment for [mteq] 6months 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 guide-line development recommendations outlined by the AGREE Collaboration (www.agreetrust.org). Evidencewas synthesized and graded using a comprehensivemulticriteriamethodology (EVIDEM) (www.evidem.org/praderwilli)