WHO Collaborating Center for Public Health Aspects of Musculo-Skeletal Health and Ageing, Division of Public Health, Epidemiology and Health Economics, University of Liège, Avenue Hippocrate 13, CHU B23, 4000, Liege, Belgium.
Chair for Biomarkers of Chronic Diseases, Biochemistry Department, College of Science, King Saud University, Riyadh, 11451, Saudi Arabia.
Aging Clin Exp Res. 2021 Jan;33(1):3-17. doi: 10.1007/s40520-020-01663-4. Epub 2020 Jul 31.
In 2016, an expert working group was convened under the auspices of the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis (ESCEO) and formulated consensus recommendations for the conduct of clinical trials for drugs to prevent or treat sarcopenia.
The objective of the current paper is to provide a 2020 update of the previous recommendations in accordance with the evidence that has become available since our original recommendations.
This paper is based on literature reviews performed by members of the ESCEO working group and followed up with face to face meetings organized for the whole group to make amendments and discuss further recommendations.
The randomized placebo-controlled double-blind parallel-arm drug clinical trials should be the design of choice for both phase II and III trials. Treatment and follow-up should run at least 6 months for phase II and 12 months for phase III trials. Overall physical activity, nutrition, co-prescriptions and comorbidity should be recorded. Participants in these trials should be at least 70-years-old and present with a combination of low muscle strength and low physical performance. Severely malnourished individuals, as well as bedridden patients, patients with extremely limited mobility or individuals with physical limitations clearly attributable to the direct effect of a specific disease, should be excluded. Multiple outcomes are proposed for phase II trials, including, as example, physical performance, muscle strength and mass, muscle metabolism and muscle-bone interaction. For phase III trials, we recommend a co-primary endpoint of a measure of functional performance and a Patient Reported Outcome Measure.
The working group has formulated consensus recommendations on specific aspects of trial design, and in doing so hopes to contribute to an improvement of the methodological robustness and comparability of clinical trials. Standardization of designs and outcomes would advance the field by allowing better comparison across studies, including performing individual patient-data meta-analyses, and different pro-myogenic therapies.
2016 年，在欧洲临床和经济骨质疏松和骨关节炎学会（ESCEO）的主持下，一个专家工作组召集会议，制定了药物临床试验的共识建议，以预防或治疗肌少症。
本文件的目的是根据自我们最初建议以来获得的证据，对先前的建议进行 2020 年更新。
本文基于 ESCEO 工作组成员进行的文献回顾，并通过组织全体会议进行跟进，以进行修订和讨论进一步的建议。
随机安慰剂对照双盲平行臂药物临床试验应该是 II 期和 III 期试验的首选设计。治疗和随访应至少进行 6 个月用于 II 期试验，12 个月用于 III 期试验。应记录总体身体活动、营养、共同处方和合并症。这些试验的参与者应至少 70 岁，并伴有低肌肉力量和低身体表现。应排除严重营养不良者、卧床不起的患者、活动能力极有限或身体限制明显归因于特定疾病直接影响的患者。对于 II 期试验，提出了多个结局，包括例如身体表现、肌肉力量和质量、肌肉代谢和肌肉-骨骼相互作用。对于 III 期试验，我们建议功能性表现和患者报告的结果测量作为联合主要终点。
工作组就试验设计的具体方面制定了共识建议，希望通过提高临床试验的方法学稳健性和可比性来为该领域做出贡献。设计和结局的标准化将通过允许更好地比较研究，包括进行个体患者数据荟萃分析和不同的促肌生成治疗，从而推动该领域的发展。
