Cherny N I, Oosting S F, Dafni U, Latino N J, Galotti M, Zygoura P, Dimopoulou G, Amaral T, Barriuso J, Calles A, Kiesewetter B, Gomez-Roca C, Gyawali B, Piccart M, Passaro A, Roitberg F, Tarazona N, Trapani D, Curigliano G, Wester R, Zarkavelis G, Zielinski C, de Vries E G E
Cancer Pain and Palliative Medicine Service, Department of Medical Oncology, Shaare Zedek Medical Center, Jerusalem, Israel.
Department of Medical Oncology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
Ann Oncol. 2025 May 21. doi: 10.1016/j.annonc.2025.04.006.
The ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) is a validated tool to assess the magnitude of clinical benefit from new cancer therapies, with planned updates based upon recognition of new needs and shortcomings. This paper describes the development of ESMO-MCBS v2.0.
The revision process incorporates nine steps: (i) review of critiques and suggestions and identification of problems in the application of ESMO-MCBS v1.1; (ii) identification of shortcomings for revision in the upcoming version; (iii) drafting solutions addressing identified shortcomings; (iv) field-testing of solutions; (v) preparation of a near-final revised version for peer review for reasonableness by members of the ESMO Faculty and ESMO Guidelines Committee; (vi) amendments based on peer review for reasonableness; (vii) near-final review by members of the ESMO-MCBS Working Group; (viii) final amendments; (ix) final review and approval by members of the ESMO-MCBS Working Group and the ESMO Executive Board.
Seventeen issues for revision or amendment were considered, and 13 amendments were formulated to address identified shortcomings. In the curative setting, studies evaluated based on disease-free survival now credit improved time without treatment or disease even when overall survival is not significantly improved, and studies with small absolute gain in disease-free survival are credited more conservatively. Additionally, acute and persistent toxicity annotations are added. In the non-curative setting, the approach to crediting a difference in the tail of overall survival and progression-free survival curves is more statistically valid, and the toxicity evaluation has been revised. In peer review all amendments were found to be either reasonable or mostly reasonable. The amendments changed the scoring of 85/353 of evaluated studies.
The amendments incorporated into ESMO-MCBS v2.0 change the scores of 13.6% of evaluated studies (10.5% downgraded, 3.1% upgraded) and add toxicity annotations to 45.5% of the studies in the curative setting, and improve its discriminatory capacity and utility.
欧洲肿瘤内科学会临床获益量表（ESMO-MCBS）是一种经过验证的工具，用于评估新型癌症疗法的临床获益程度，并根据对新需求和缺陷的认识进行计划更新。本文描述了ESMO-MCBS v2.0的开发过程。
修订过程包括九个步骤：（i）审查批评意见和建议，识别ESMO-MCBS v1.1应用中的问题；（ii）确定即将发布版本中需要修订的缺陷；（iii）起草解决已识别缺陷的方案；（iv）对方案进行实地测试；（v）准备接近最终的修订版本，供ESMO教员和ESMO指南委员会成员进行合理性同行评审；（vi）根据同行评审的合理性进行修订；（vii）由ESMO-MCBS工作组成员进行接近最终评审；（viii）最终修订；（ix）由ESMO-MCBS工作组和ESMO执行委员会成员进行最终评审和批准。
共考虑了17个修订或修正问题，并制定了13项修正措施以解决已识别出的缺陷。在治愈性治疗背景下，基于无病生存期评估的研究，即使总生存期未显著改善，现在也会认可无治疗或无疾病时间的改善，而无病生存期绝对获益较小的研究评分更为保守。此外，增加了急性和持续性毒性注释。在非治愈性治疗背景下，对总生存期和无进展生存期曲线尾部差异的评分方法在统计学上更合理，并且毒性评估也进行了修订。在同行评审中，所有修订均被认为合理或基本合理。这些修订改变了85/353项评估研究的评分。
纳入ESMO-MCBS v2.0的修订改变了13.6%的评估研究的评分（10.5%下调，3.1%上调），并在治愈性治疗背景下为45.5%的研究增加了毒性注释，提高了其区分能力和实用性。
