He Qiang, Wang Zhen, Mei Jie, Xie Chengxin, Sun Xin
Department of Orthopedics, Nanjing University of Chinese Medicine Affiliated Nanjing Hospital of Traditional Chinese Medicine, Nanjing, China.
Department of Orthopedics, Shandong University of Traditional Chinese Medicine Affiliated Hospital, Jinan, China.
Jpn J Clin Oncol. 2024 Dec 7;54(12):1272-1280. doi: 10.1093/jjco/hyae102.
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.
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).
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.
Only a few RCTs with positive results have demonstrated substantial patient benefits for bone and soft tissue sarcomas over the past decade.
骨肉瘤和软组织肉瘤是罕见的恶性肿瘤，其异质性限制了新型药物的研发。本研究旨在应用两种经过验证的工具来评估过去十年中开发的肉瘤新型药物疗法的临床益处。
检索PubMed和Embase数据库，查找2013年至2023年间发表的肉瘤全身治疗随机对照试验（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。
本研究纳入了52项RCT，其中17项（32.7%）报告了有利于试验组的阳性结果。在14/17项阳性试验中确定了ESMO-MCBS等级，其中三项（21.4%）达到了有意义临床获益的阈值。同样，在11/17项阳性试验中计算了ASCO-VF评分，其中三项（27.3%）达到了有意义临床获益的阈值。两个框架之间观察到弱相关性（r = 0.38，P = 0.277）和一致性（κ = 0.211，P = 0.490）。
在过去十年中，只有少数阳性结果的RCT证明了骨肉瘤和软组织肉瘤患者有显著获益。
