GrowthHormone Research Society workshop summary: consensus guidelines for recombinant human growth hormone therapy in Prader-Willi syndrome.
GrowthHormone Research Society workshop summary: consensus guidelines for recombinant human growth hormone therapy in Prader-Willi syndrome.
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DOI：
10.1210/jc.2012-3888
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发表时间：
2013-06
期刊：
影响因子：
--
通讯作者：
2011 Growth Hormone in Prader-Willi Syndrome Clinical Care Guidelines Workshop Participants
中科院分区：
文献类型：
--
作者：
Deal CL;Tony M;Höybye C;Allen DB;Tauber M;Christiansen JS;2011 Growth Hormone in Prader-Willi Syndrome Clinical Care Guidelines Workshop Participants
自2000年在美国和2001年在欧洲获得批准以来，重组人GH（rhGH）治疗普拉德-威利综合征（PWS）已被医学界使用，并得到家长支持团体的倡导。它在PWS中的使用代表了一种独特的治疗挑战，包括治疗患有认知障碍的个体，各种治疗目标并不完全集中在身高增加上，以及对潜在危及生命的不良事件的担忧。该研究的目的是制定在PWS儿童和成人患者中使用rhGH的建议。我们对儿科人群的临床证据进行了系统评价，包括随机对照试验、比较观察性研究和长期研究（>3.5年）。成人研究包括rhGH治疗≥ 6个月的随机对照试验和非对照试验。安全性数据来自病例报告、临床试验和药物注册。43名国际专家和利益相关者遵循AGREE协作组织（www.agreetrust.org）概述的临床实践指南制定建议。使用综合多标准方法（EVIDEM）（http：//bit.ly.PWGHIN）对证据进行综合和分级。经过多学科评估，最好是由专家，rhGH治疗应考虑与饮食，环境和生活方式干预与遗传证实PWS的患者。认知障碍不应成为治疗的障碍，知情同意书应包括获益/风险信息。排除标准应包括严重肥胖、未控制的糖尿病、未经治疗的严重阻塞性睡眠呼吸暂停、活动性癌症或精神病。临床结局优先顺序应根据年龄和存在的身体，精神和社会残疾而有所不同，只要证明获益大于风险，就应继续治疗。
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. The aim of the study was to formulate recommendations for the use of rhGH in children and adult patients with PWS. 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. 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). 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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DOI：
10.1111/j.1365-2788.2006.00878.x
发表时间：
2007-04-01
影响因子：
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作者：
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