Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations.
Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations.
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DOI：
10.1530/eje-16-0111
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发表时间：
2016-06
影响因子：
5.8
通讯作者：
Yuen K
中科院分区：
文献类型：
--
作者：
Christiansen JS;Backeljauw PF;Bidlingmaier M;Biller BM;Boguszewski MC;Casanueva FF;Chanson P;Chatelain P;Choong CS;Clemmons DR;Cohen LE;Cohen P;Frystyk J;Grimberg A;Hasegawa Y;Haymond MW;Ho K;Hoffman AR;Holly JM;Horikawa R;Höybye C;Jorgensen JO;Johannsson G;Juul A;Katznelson L;Kopchick JJ;Lee KO;Lee KW;Luo X;Melmed S;Miller BS;Misra M;Popovic V;Rosenfeld RG;Ross J;Ross RJ;Saenger P;Strasburger CJ;Thorner MO;Werner H;Yuen K
生长激素（GH）研究协会（GRS）召开了一次研讨会，以解决有关试验设计，疗效和长效生长激素制剂（LAGH）的安全性的重要问题。55名具有GH专业知识的国际科学家的闭门会议，包括儿科和成人内分泌学家，基础科学家，监管科学家和制药行业的参与者。对现有文献进行了审查，以确定知识差距。专家意见用于建议需要进行研究以解决潜在的安全性和有效性问题。在总结文献的全体发言之后，分组讨论了规划委员会提出的问题。与会者在每次分组会议后重新召集，分享小组报告。一个编写小组将分组会议的报告汇编成一份文件草案，在闭幕日的一个公开论坛上进行了讨论和修订。这份报告经过进一步编辑，然后分发给学术机构的与会者，供会后审查。来自制药公司的与会者没有参与研讨会最后一天的计划、写作或讨论和文本修订。来自行业和监管机构的科学家审查了手稿，以确定任何事实错误。LAGH化合物可能代表了每日GH注射的进步，因为增加了便利性和不同的药效学特性，提供了改善依从性和结局的潜力。必须制定和验证评估遵守情况的更好方法。长期监测登记，包括疗效，成本效益，疾病负担，生活质量和安全性的评估是必不可少的了解持续暴露于LAGH制剂的影响。
The Growth Hormone (GH) Research Society (GRS) convened a workshop to address important issues regarding trial design, efficacy, and safety of long-acting growth hormone preparations (LAGH). A closed meeting of 55 international scientists with expertise in GH, including pediatric and adult endocrinologists, basic scientists, regulatory scientists, and participants from the pharmaceutical industry. Current literature was reviewed for gaps in knowledge. Expert opinion was used to suggest studies required to address potential safety and efficacy issues. Following plenary presentations summarizing the literature, breakout groups discussed questions framed by the planning committee. Attendees reconvened after each breakout session to share group reports. A writing team compiled the breakout session reports into a draft document that was discussed and revised in an open forum on the concluding day. This was edited further and then circulated to attendees from academic institutions for review after the meeting. Participants from pharmaceutical companies did not participate in the planning, writing, or in the discussions and text revision on the final day of the workshop. Scientists from industry and regulatory agencies reviewed the manuscript to identify any factual errors. LAGH compounds may represent an advance over daily GH injections because of increased convenience and differing phamacodynamic properties, providing the potential for improved adherence and outcomes. Better methods to assess adherence must be developed and validated. Long-term surveillance registries that include assessment of efficacy, cost-benefit, disease burden, quality of life, and safety are essential for understanding the impact of sustained exposure to LAGH preparations.