TY - JOUR
T1 - Growth hormone research society workshop summary
T2 - Consensus guidelines for recombinant human growth hormone therapy in Prader-Willi syndrome
AU - Deal, Cheri L.
AU - Tony, Michèle
AU - Hoÿbye, Charlotte
AU - Allen, David B.
AU - Tauber, Maïth́e
AU - Christiansen, Jens Sandahl
AU - Ambler, Geoffrey R.
AU - Battista, Renaldo
AU - Beauloye, Véronique
AU - Berall, Glenn
AU - Biller, Beverly M K
AU - Butler, Merlin G.
AU - Cassidy, Suzanne B.
AU - Chihara, Kazuo
AU - Cohen, Pinchas
AU - Craig, Maria
AU - Farholt, Stense
AU - Goetghebeur, Mireille
AU - Goldstone, Anthony P.
AU - Greggi, Tiziana
AU - Grugni, Graziano
AU - Hokken-Koelega, Anita C.
AU - Johannsson, Gudmundur
AU - Johnson, Keegan
AU - Kemper, Alex
AU - Kopchick, John J.
AU - Malozowski, Saul
AU - Miller, Jennifer
AU - Mogul, Harriette R.
AU - Muscatelli, Françoise
AU - Nergårdh, Ricard
AU - Nicholls, Robert D.
AU - Radovick, Sally
AU - Rosenthal, M. Sara
AU - Sipilä, Ilkka
AU - Tarride, Jean Eric
AU - Vogels, Annick
AU - Waters, Michael J.
PY - 2013/6
Y1 - 2013/6
N2 - 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.
AB - 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.
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U2 - 10.1210/jc.2012-3888
DO - 10.1210/jc.2012-3888
M3 - Article
C2 - 23543664
AN - SCOPUS:84878475228
SN - 0021-972X
VL - 98
JO - Journal of Clinical Endocrinology and Metabolism
JF - Journal of Clinical Endocrinology and Metabolism
IS - 6
ER -