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

Deal, Cheri L., Tony, Michele, Hoybye, Charlotte, Allen, David B., Tauber, Maithe, Christiansen, Jens S., 2011 Growth Hormone in Prader-Willi Syndrome Clinical Care Guidelines Workshop Participants, Ambler, Geoffrey R., Battista, Renaldo, Beauloye, Veronique, Berall, Glenn, Biller, Beverly M. K., Butler, Merlin G., Cassidy, Suzanne B., Chihara, Kazuo, Cohen, Pinchas, Craig, Maria, Farholt, Stense, Goetghebeur, Mireille, Goldstone, Anthony P., Greggi, Tiziana, Grugni, Graziano, Hokken-Koelega, Anita C., Johannsson, Gudmundur, Johnson, Keegan, Kemper, Alex, Kopchick, John J., Malozowski, Saul, Miller, Jennifer, Mogul, Harriette R., Muscatelli, Francoise, Nergardh, Richard, Nicholls, Robert D., Radovick, Sally, Rosenthal, M. Sara, Sipila, Ilkka, Tarride, Jean-Eric, Vogels, Annick and Waters, Michael 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: E1072-E1087. doi:10.1210/jc.2012-3888


Author Deal, Cheri L.
Tony, Michele
Hoybye, Charlotte
Allen, David B.
Tauber, Maithe
Christiansen, Jens S.
2011 Growth Hormone in Prader-Willi Syndrome Clinical Care Guidelines Workshop Participants
Ambler, Geoffrey R.
Battista, Renaldo
Beauloye, Veronique
Berall, Glenn
Biller, Beverly M. K.
Butler, Merlin G.
Cassidy, Suzanne B.
Chihara, Kazuo
Cohen, Pinchas
Craig, Maria
Farholt, Stense
Goetghebeur, Mireille
Goldstone, Anthony P.
Greggi, Tiziana
Grugni, Graziano
Hokken-Koelega, Anita C.
Johannsson, Gudmundur
Johnson, Keegan
Kemper, Alex
Kopchick, John J.
Malozowski, Saul
Miller, Jennifer
Mogul, Harriette R.
Muscatelli, Francoise
Nergardh, Richard
Nicholls, Robert D.
Radovick, Sally
Rosenthal, M. Sara
Sipila, Ilkka
Tarride, Jean-Eric
Vogels, Annick
Waters, Michael J.
Total Author Count Override 7
Title Growth hormone research society workshop summary: Consensus guidelines for recombinant human growth hormone therapy in Prader-Willi syndrome
Journal name Journal of Clinical Endocrinology and Metabolism   Check publisher's open access policy
ISSN 0021-972X
1945-7197
Publication date 2013
Year available 2013
Sub-type Article (original research)
DOI 10.1210/jc.2012-3888
Open Access Status DOI
Volume 98
Issue 6
Start page E1072
End page E1087
Total pages 16
Place of publication Chevy Chase, MD United States
Publisher The Endocrine Society
Collection year 2014
Language eng
Subject 1303 Specialist Studies in Education
1308 Clinical Biochemistry
1310 Endocrinology
2704 Biochemistry, medical
2712 Endocrinology, Diabetes and Metabolism
2700 Medicine
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. Copyright
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ

Document type: Journal Article
Sub-type: Article (original research)
Collections: Official 2014 Collection
Institute for Molecular Bioscience - Publications
 
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