Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Department of Hematology, Japan Baptist Hospital, Kyoto, Japan.
Hematology. 2022 Dec;27(1):239-248. doi: 10.1080/16078454.2022.2032915.
We conducted a phase II trial to prospectively evaluate the efficacy and safety of bortezomib-cyclophosphamide-dexamethasone (VCD) induction, autologous stem cell transplantation (ASCT), VCD consolidation, and bortezomib maintenance in transplant-eligible newly diagnosed multiple myeloma (NDMM) patients in Japan (UMIN000010542).
From 2013 to 2016, 42 patients with a median age of 58 (range 42-65) years with NDMM were enrolled in 15 centers. The primary endpoint was the complete response (CR) /stringent CR (sCR) rate after transplantation, and overall/progression-free survival rates were also evaluated.
Following induction therapy, the overall response rate was obtained in 71% of patients, including a CR/sCR of 10% and a very good partial response (VGPR) of 26%. Twenty-six of the 42 patients completed ASCT following the protocol and CR/sCR and VGPR rate 100 days after ASCT was 26% and 17%, respectively. During consolidation therapy, 3 of the 24 patients achieved deeper responses. Eight of the 18 patients completed 2-year bortezomib maintenance without disease progression and grade 3/4 toxicities. Five patients were VGPR or partial response after ASCT but maintained response with 2-year bortezomib maintenance. Two-year overall and progression-free survival rates were 92.5% (95% confidence interval [CI]: 78.5%-97.5%) and 62.6% (95% CI: 45.8%-75.5%), respectively. Grade 3/4 toxicities (≥ 10%) included neutropenia (19%) and anemia (17%) in induction, and thrombocytopenia (29%) in consolidation.
VCD induction/consolidation and bortezomib maintenance with ASCT for NDMM resulted in a high CR/sCR rate and provided good overall/progression-free survival in Japan.
我们进行了一项 II 期临床试验，前瞻性评估硼替佐米-环磷酰胺-地塞米松（VCD）诱导、自体干细胞移植（ASCT）、VCD 巩固和硼替佐米维持治疗在日本适合移植的新诊断多发性骨髓瘤（NDMM）患者中的疗效和安全性（UMIN000010542）。
2013 年至 2016 年，在 15 个中心共纳入 42 例中位年龄为 58 岁（范围 42-65 岁）的 NDMM 患者。主要终点是移植后完全缓解（CR）/严格 CR（sCR）率，同时还评估了总生存（OS）/无进展生存（PFS）率。
诱导治疗后，71%的患者获得了总体缓解，包括 10%的 CR/sCR 和 26%的非常好的部分缓解（VGPR）。42 例患者中有 26 例按照方案完成了 ASCT，ASCT 后 100 天的 CR/sCR 和 VGPR 率分别为 26%和 17%。在巩固治疗期间，24 例患者中有 3 例获得了更深的缓解。18 例患者中有 8 例完成了 2 年硼替佐米维持治疗，没有疾病进展和 3/4 级毒性。5 例患者在 ASCT 后获得了 VGPR 或部分缓解，但通过 2 年硼替佐米维持治疗保持缓解。2 年 OS 和 PFS 率分别为 92.5%（95%置信区间[CI]：78.5%-97.5%）和 62.6%（95% CI：45.8%-75.5%）。≥10%的 3/4 级毒性包括诱导治疗中的中性粒细胞减少症（19%）和贫血（17%），以及巩固治疗中的血小板减少症（29%）。
硼替佐米诱导/巩固和 ASCT 联合硼替佐米维持治疗 NDMM 可获得较高的 CR/sCR 率，并提供了较好的 OS/PFS。
