Bortezomib, lenalidomide, and dexamethasone induction chemotherapy (VRd), followed by autologous stem cell transplantation (ASCT), are the standard of care for patients with newly diagnosed multiple myeloma (NDMM). Pomalidomide is currently approved for relapsed-refractory multiple myeloma. This single-arm, open-label, phase 2 study was the prospective evaluation of the efficacy and safety of bortezomib, pomalidomide, and dexamethasone (VPd) induction for NDMM. We used Fleming’s two-stage design for sample size calculation. We included transplant-eligible and ineligible patients aged 18–75 years in the study. The patients received four cycles of VPd induction followed by response assessment. Thirty-four patients were included in the study, of which 31 completed all four cycles of induction. The median age was 52 years (32–72). Thirty (91%) patients had multiple myeloma, and three had multiple plasmacytomas with less than 10% bone marrow involvement. Nine (27%) had ISS-I, 9 (27%) had ISS-II, and 15 (46%) had ISS-III myeloma. Three patients had high-risk cytogenetic abnormalities. After four cycles of VPd induction, ten patients (32%) achieved stringent CR, nine had CR (29%), eight (26%) had VGPR, and 4 (13%) had PR. Fifteen (48%) had a complete metabolic response (CMR) on PET-CT. Two patients developed SAEs. Anemia was the most common hematological toxicity. Peripheral neuropathy and constipation were the most common non-hematological toxicities. Patients with ≥VGPR had significantly better 12-month PFS than those with PR. Patients with ≥VGPR and CMR on PET-CT had significantly better 12-month OS. Our study showed VPd induction is safe and efficacious in NDMM. Further Phase 3 studies are necessary to establish the superiority and survival benefits.
硼替佐米、来那度胺联合地塞米松诱导化疗（VRd）序贯自体干细胞移植（ASCT）是新诊断多发性骨髓瘤（NDMM）的标准治疗方案。泊马度胺目前获批用于复发难治性多发性骨髓瘤。本项单臂、开放标签的2期研究前瞻性评估了硼替佐米、泊马度胺联合地塞米松（VPd）诱导方案在NDMM中的疗效与安全性。我们采用弗莱明两阶段设计进行样本量计算。研究纳入年龄18-75岁、适合或不适合移植的患者。患者接受4周期VPd诱导治疗后进行疗效评估。共纳入34例患者，其中31例完成全部4周期诱导治疗。中位年龄52岁（范围32-72岁）。30例（91%）为多发性骨髓瘤，3例为骨髓浸润<10%的多发性浆细胞瘤。9例（27%）为ISS分期Ⅰ期，9例（27%）为Ⅱ期，15例（46%）为Ⅲ期。3例患者存在高危细胞遗传学异常。完成4周期VPd诱导后，10例患者（32%）达到严格完全缓解，9例（29%）达完全缓解，8例（26%）达非常好的部分缓解，4例（13%）达部分缓解。15例（48%）患者PET-CT显示完全代谢缓解。2例患者发生严重不良事件。贫血是最常见的血液学毒性，周围神经病变和便秘是最常见的非血液学毒性。达到≥非常好的部分缓解的患者其12个月无进展生存期显著优于部分缓解者。PET-CT显示≥非常好的部分缓解且完全代谢缓解的患者12个月总生存期显著更优。本研究证实VPd诱导方案在NDMM中安全有效，尚需开展3期研究以明确其优势及生存获益。
肿瘤(癌症)患者之家