title: GrowthHormone Research Society workshop summary: consensus guidelines for recombinant human growth hormone therapy in Prader-Willi syndrome.
abstract: BACKGROUND
  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.
  
  
  METHODS
  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.
  
  
  METHODS
  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.
authors.affiliation: Research Center and Department of Pediatrics, Centre Hospitalier Universitaire Sainte-Justine, Montréal, Québec, Canada H3T 1C5. Cheri.L.Deal@umontreal.ca
authors.forename: Cheri L
authors.initials: CL
authors.lastname: Deal
authors.forename: Michèle
authors.initials: M
authors.lastname: Tony
authors.forename: Charlotte
authors.initials: C
authors.lastname: Höybye
authors.forename: David B
authors.initials: DB
authors.lastname: Allen
authors.forename: Maïthé
authors.initials: M
authors.lastname: Tauber
authors.forename: Jens Sandahl
authors.initials: JS
authors.lastname: Christiansen
mesh_terms: D000328:Adult; D002648:Child; D019382:Human Growth Hormone / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D017410:Practice Guidelines as Topic; D011218:Prader-Willi Syndrome / Q000188:drug therapy* / Q000503:physiopathology; D011994:Recombinant Proteins / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D019382:Human Growth Hormone
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