Department of Hematology and Infectious Disease, Gifu University Hospital, Gifu, Japan.
Department of Hematology Oncology, Cancer Institute Hospital of Japanese Foundation for Cancer Research, Tokyo, Japan.
Jpn J Clin Oncol. 2021 Jul 1;51(7):1059-1066. doi: 10.1093/jjco/hyab066.
The International Myeloma Working Group response criteria require two consecutive assessments of paraprotein levels. We conducted an exploratory analysis to evaluate whether a single response assessment could be a substitute for the International Myeloma Working Group criteria using data from JCOG1105, a randomized phase II study on melphalan, prednisolone and bortezomib.
Of 91 patients with transplant-ineligible newly diagnosed multiple myeloma, 79 patients were included. We calculated the kappa coefficient to evaluate the degree of agreement between the International Myeloma Working Group criteria and the single response assessment.
Based on the International Myeloma Working Group criteria, 11 (13.9%), 20 (25.3%), 36 (45.6%) and 12 (15.2%) patients had stringent complete response/complete response, very good partial response, partial response and stable disease, respectively. Based on the single response assessment, 17 (21.5%), 19 (24.1%), 35 (44.3%) and 8 (10.1%) patients had stringent complete response/complete response, very good partial response, partial response and stable disease, respectively. The kappa coefficient was 0.76 (95% confidence interval, 0.65-0.88), demonstrating good agreement. The single response assessment was not inferior to the International Myeloma Working Group criteria in the median progression-free survival (3.8 and 2.9 years) in stringent complete response/complete response patients, suggesting that the single response assessment was not an overestimation.
The single response assessment could be a substitute for the current International Myeloma Working Group criteria for transplant-ineligible newly diagnosed multiple myeloma.
国际骨髓瘤工作组反应标准要求连续两次评估蛋白水平。我们进行了一项探索性分析，使用 JCOG1105（一项关于马法兰、泼尼松和硼替佐米的随机 II 期研究）的数据，评估单次反应评估是否可以替代国际骨髓瘤工作组标准。
在 91 例不适合移植的新诊断多发性骨髓瘤患者中，纳入了 79 例患者。我们计算了 κ 系数来评估国际骨髓瘤工作组标准与单次反应评估之间的一致性程度。
根据国际骨髓瘤工作组标准，11 例（13.9%）、20 例（25.3%）、36 例（45.6%）和 12 例（15.2%）患者分别达到严格完全缓解/完全缓解、非常好的部分缓解、部分缓解和疾病稳定。根据单次反应评估，17 例（21.5%）、19 例（24.1%）、35 例（44.3%）和 8 例（10.1%）患者分别达到严格完全缓解/完全缓解、非常好的部分缓解、部分缓解和疾病稳定。κ 系数为 0.76（95%置信区间，0.65-0.88），表明一致性较好。在严格完全缓解/完全缓解患者中，单次反应评估的中位无进展生存期并不劣于国际骨髓瘤工作组标准（3.8 年和 2.9 年），这表明单次反应评估并非高估。
单次反应评估可替代当前不适合移植的新诊断多发性骨髓瘤的国际骨髓瘤工作组标准。
