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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 tr...
Autores principales: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Endocrine Society
2013
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3789886/ https://www.ncbi.nlm.nih.gov/pubmed/23543664 http://dx.doi.org/10.1210/jc.2012-3888 |
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author | Deal, Cheri L. Tony, Michèle Höybye, Charlotte Allen, David B. Tauber, Maïthé Christiansen, Jens Sandahl Ambler, Geoffrey R. Battista, Renaldo Beauloye, Véronique 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, Françoise Nergårdh, Ricard Nicholls, Robert D. Radovick, Sally Rosenthal, M. Sara Sipilä, Ilkka Tarride, Jean-Eric Vogels, Annick Waters, Michael J. |
author_facet | Deal, Cheri L. Tony, Michèle Höybye, Charlotte Allen, David B. Tauber, Maïthé Christiansen, Jens Sandahl Ambler, Geoffrey R. Battista, Renaldo Beauloye, Véronique 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, Françoise Nergårdh, Ricard Nicholls, Robert D. Radovick, Sally Rosenthal, M. Sara Sipilä, Ilkka Tarride, Jean-Eric Vogels, Annick Waters, Michael J. |
author_sort | Deal, Cheri L. |
collection | PubMed |
description | 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. |
format | Online Article Text |
id | pubmed-3789886 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2013 |
publisher | Endocrine Society |
record_format | MEDLINE/PubMed |
spelling | pubmed-37898862013-11-01 Growth Hormone Research Society Workshop Summary: Consensus Guidelines for Recombinant Human Growth Hormone Therapy in Prader-Willi Syndrome Deal, Cheri L. Tony, Michèle Höybye, Charlotte Allen, David B. Tauber, Maïthé Christiansen, Jens Sandahl Ambler, Geoffrey R. Battista, Renaldo Beauloye, Véronique 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, Françoise Nergårdh, Ricard Nicholls, Robert D. Radovick, Sally Rosenthal, M. Sara Sipilä, Ilkka Tarride, Jean-Eric Vogels, Annick Waters, Michael J. J Clin Endocrinol Metab JCEM Online: Advances in Genetics 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. Endocrine Society 2013-06 2013-03-29 /pmc/articles/PMC3789886/ /pubmed/23543664 http://dx.doi.org/10.1210/jc.2012-3888 Text en Copyright © 2013 by The Endocrine Society This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/3.0/us/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | JCEM Online: Advances in Genetics Deal, Cheri L. Tony, Michèle Höybye, Charlotte Allen, David B. Tauber, Maïthé Christiansen, Jens Sandahl Ambler, Geoffrey R. Battista, Renaldo Beauloye, Véronique 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, Françoise Nergårdh, Ricard Nicholls, Robert D. Radovick, Sally Rosenthal, M. Sara Sipilä, Ilkka Tarride, Jean-Eric Vogels, Annick Waters, Michael J. Growth Hormone Research Society Workshop Summary: Consensus Guidelines for Recombinant Human Growth Hormone Therapy in Prader-Willi Syndrome |
title | Growth Hormone Research Society Workshop Summary: Consensus Guidelines for Recombinant Human Growth Hormone Therapy in Prader-Willi Syndrome |
title_full | Growth Hormone Research Society Workshop Summary: Consensus Guidelines for Recombinant Human Growth Hormone Therapy in Prader-Willi Syndrome |
title_fullStr | Growth Hormone Research Society Workshop Summary: Consensus Guidelines for Recombinant Human Growth Hormone Therapy in Prader-Willi Syndrome |
title_full_unstemmed | Growth Hormone Research Society Workshop Summary: Consensus Guidelines for Recombinant Human Growth Hormone Therapy in Prader-Willi Syndrome |
title_short | Growth Hormone Research Society Workshop Summary: Consensus Guidelines for Recombinant Human Growth Hormone Therapy in Prader-Willi Syndrome |
title_sort | growth hormone research society workshop summary: consensus guidelines for recombinant human growth hormone therapy in prader-willi syndrome |
topic | JCEM Online: Advances in Genetics |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3789886/ https://www.ncbi.nlm.nih.gov/pubmed/23543664 http://dx.doi.org/10.1210/jc.2012-3888 |
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