Abstract：
BACKGROUND: Bone and soft tissue sarcomas are rare malignancies, and their heterogeneity has limited the development of novel drugs. This study aimed to apply two validated tools to evaluate the clinical benefits of novel drug therapies for sarcoma developed over the last decade.
METHODS: The PubMed and Embase databases were searched for randomized controlled trials (RCTs) of systemic therapies for sarcomas published between 2013 and 2023. Each trial was scored according to the European Society of Medical Oncology-Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS) and the American Society of Clinical Oncology-Value Framework version 2 (ASCO-VF).
RESULTS: We included 52 RCTs in this study, of which 17 (32.7%) reported positive results that favored the experimental arm. The ESMO-MCBS grades were determined in 14/17 positive trials, and three of them (21.4%) met the threshold for meaningful clinical benefit. Likewise, ASCO-VF scores were calculated for 11/17 positive trials, and three of them (27.3%) met the threshold for meaningful clinical benefit. Weak correlation (r = 0.38, P = 0.277) and agreement (κ = 0.211, P = 0.490) were observed between the two frameworks.
CONCLUSIONS: Only a few RCTs with positive results have demonstrated substantial patient benefits for bone and soft tissue sarcomas over the past decade.
METHODS: The PubMed and Embase databases were searched for randomized controlled trials (RCTs) of systemic therapies for sarcomas published between 2013 and 2023. Each trial was scored according to the European Society of Medical Oncology-Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS) and the American Society of Clinical Oncology-Value Framework version 2 (ASCO-VF).
RESULTS: We included 52 RCTs in this study, of which 17 (32.7%) reported positive results that favored the experimental arm. The ESMO-MCBS grades were determined in 14/17 positive trials, and three of them (21.4%) met the threshold for meaningful clinical benefit. Likewise, ASCO-VF scores were calculated for 11/17 positive trials, and three of them (27.3%) met the threshold for meaningful clinical benefit. Weak correlation (r = 0.38, P = 0.277) and agreement (κ = 0.211, P = 0.490) were observed between the two frameworks.
CONCLUSIONS: Only a few RCTs with positive results have demonstrated substantial patient benefits for bone and soft tissue sarcomas over the past decade.
摘要：
背景：骨和软组织肉瘤是罕见的恶性肿瘤，它们的异质性限制了新药的开发。这项研究旨在应用两种经过验证的工具来评估在过去十年中开发的新型药物治疗肉瘤的临床益处。
方法：在PubMed和Embase数据库中搜索了2013年至2023年发表的肉瘤全身治疗的随机对照试验（RCT）。根据欧洲医学肿瘤学会临床获益量表1.1版（ESMO-MCBS）和美国临床肿瘤学会价值框架2版（ASCO-VF）对每项试验进行评分。
结果：我们在这项研究中纳入了52项RCT，其中17人(32.7%)报告了有利于实验臂的阳性结果。ESMO-MCBS等级在14/17个阳性试验中确定，其中3例(21.4%)达到了有意义的临床获益阈值.同样，ASCO-VF评分计算了11/17个阳性试验，其中3人(27.3%)达到了有意义的临床获益阈值。两种框架之间存在弱相关性（r=0.38，P=0.277）和一致性（κ=0.211，P=0.490）。
结论：在过去的十年中，只有少数阳性结果的随机对照试验证明了骨和软组织肉瘤对患者的实质性益处。
方法：在PubMed和Embase数据库中搜索了2013年至2023年发表的肉瘤全身治疗的随机对照试验（RCT）。根据欧洲医学肿瘤学会临床获益量表1.1版（ESMO-MCBS）和美国临床肿瘤学会价值框架2版（ASCO-VF）对每项试验进行评分。
结果：我们在这项研究中纳入了52项RCT，其中17人(32.7%)报告了有利于实验臂的阳性结果。ESMO-MCBS等级在14/17个阳性试验中确定，其中3例(21.4%)达到了有意义的临床获益阈值.同样，ASCO-VF评分计算了11/17个阳性试验，其中3人(27.3%)达到了有意义的临床获益阈值。两种框架之间存在弱相关性（r=0.38，P=0.277）和一致性（κ=0.211，P=0.490）。
结论：在过去的十年中，只有少数阳性结果的随机对照试验证明了骨和软组织肉瘤对患者的实质性益处。
