Division of Oncology, Department of Medicine I, Medical University of Vienna, Vienna, Austria.
Laboratory of Biostatistics, School of Health Sciences, National and Kapodistrian University of Athens, Athens; Frontier Science Foundation-Hellas, Athens, Greece.
Ann Oncol. 2023 Sep;34(9):734-771. doi: 10.1016/j.annonc.2023.06.002. Epub 2023 Jun 19.
The European Society for Medical Oncology (ESMO)-Magnitude of Clinical Benefit Scale (MCBS) has been accepted as a robust tool to evaluate the magnitude of clinical benefit reported in trials for oncological therapies. However, the ESMO-MCBS hitherto has only been validated for solid tumours. With the rapid development of novel therapies for haematological malignancies, we aimed to develop an ESMO-MCBS version that is specifically designed and validated for haematological malignancies.
ESMO and the European Hematology Association (EHA) initiated a collaboration to develop a version for haematological malignancies (ESMO-MCBS:H). The process incorporated five landmarks: field testing of the ESMO-MCBS version 1.1 (v1.1) to identify shortcomings specific to haematological diseases, drafting of the ESMO-MCBS:H forms, peer review and revision of the draft based on re-scoring (resulting in a second draft), assessment of reasonableness of the scores generated, final review and approval by ESMO and EHA including executive boards.
Based on the field testing results of 80 haematological trials and extensive review for feasibility and reasonableness, five amendments to ESMO-MCBS were incorporated in the ESMO-MCBS:H addressing the identified shortcomings. These concerned mainly clinical trial endpoints that differ in haematology versus solid oncology and the very indolent nature of nevertheless incurable diseases such as follicular lymphoma, which hampers presentation of mature data. In addition, general changes incorporated in the draft version of the ESMO-MCBS v2 were included, and specific forms for haematological malignancies generated. Here we present the final approved forms of the ESMO-MCBS:H, including instructions.
The haematology-specific version ESMO-MCBS:H allows now full applicability of the scale for evaluating the magnitude of clinical benefit derived from clinical studies in haematological malignancies.
欧洲肿瘤内科学会（ESMO）-临床获益幅度量表（MCBS）已被接受为评估肿瘤治疗临床试验报告的临床获益幅度的有力工具。然而，ESMO-MCBS 迄今为止仅在实体瘤中得到验证。随着新型血液恶性肿瘤治疗方法的快速发展，我们旨在开发一种专门针对血液恶性肿瘤的 ESMO-MCBS 版本。
ESMO 和欧洲血液学协会（EHA）合作开发血液恶性肿瘤版本（ESMO-MCBS:H）。该过程包括五个里程碑：现场测试 ESMO-MCBS 版本 1.1（v1.1），以确定特定于血液疾病的缺点，起草 ESMO-MCBS:H 表格，根据重新评分对草稿进行同行评审和修订（导致第二稿），评估生成的分数的合理性，ESMO 和 EHA 包括执行委员会的最终审查和批准。
根据 80 项血液学试验的现场测试结果和对可行性和合理性的广泛审查，对 ESMO-MCBS 进行了五项修订，纳入了 ESMO-MCBS:H，以解决已确定的缺点。这些主要涉及血液学与实体肿瘤临床试验终点的不同，以及滤泡性淋巴瘤等不可治愈疾病的惰性性质，这阻碍了成熟数据的呈现。此外，还纳入了 ESMO-MCBS 第二版草案中包含的一般变更，并生成了血液恶性肿瘤的特定表格。在这里，我们展示了最终批准的 ESMO-MCBS:H 表格，包括说明。
血液学专用版 ESMO-MCBS:H 现在允许充分应用该量表评估来自血液恶性肿瘤临床研究的临床获益幅度。
