Department of Medical Oncology, Inselspital, University Hospital and University of Bern, Bern, Switzerland.
Department of Hematology, Inselspital, University Hospital and University of Bern, Bern, Switzerland.
Bone Marrow Transplant. 2022 Jun;57(6):990-997. doi: 10.1038/s41409-022-01681-y. Epub 2022 Apr 20.
Definite cure remains exceptional in myeloma patients even after high-dose chemotherapy (HDCT) with melphalan (Mel) and autologous stem cell transplantation (ASCT). Thus, improving efficacy of HDCT in MM remains an unresolved issue. This randomized phase II trial compared standard 200 mg/m Mel HDCT to experimental HDCT with 200 mg/m bendamustine, given both at days -4 and -3, combined with 100 mg/m melphalan at days -2 and -1 (BenMel) before ASCT as first-line consolidation in myeloma patients. The primary endpoint aimed to identify at least a 15% improvement in the complete remission rate (stringent CR + CR) after HDCT with BenMel compared with Mel alone. A total of 120 MM patients were 1:1 randomized. The rate of sCR/CR after ASCT was higher in BenMel than in Mel treated patients (70.0% vs. 51.7%; p = 0.039). Three patients in the BenMel group (5.0%) had reversible acute renal insufficiency compared with none in Mel patients. Minimal residual disease negativity (<10-5) by flow cytometry was observed in 26 (45.6%) BenMel patients and 22 (37.9%) in the Mel group (p = 0.375). Our data suggest that BenMel HDCT is safe and improves the sCR/CR rate compared with standard Mel alone.
即使在接受大剂量化疗（HDCT）联合马法兰（Mel）和自体干细胞移植（ASCT）后，骨髓瘤患者也很难获得根治。因此，提高 HDCT 在 MM 中的疗效仍然是一个悬而未决的问题。这项随机 II 期试验比较了标准的 200mg/m Mel HDCT 与实验性的 200mg/m 苯达莫司汀（Ben）在 -4 日和 -3 日给予，联合 ASCT 前 -2 日和 -1 日给予 100mg/m Mel（BenMel）作为骨髓瘤患者的一线巩固治疗。主要终点旨在确定与 Mel 单药治疗相比，BenMel 治疗后的 HDCT 完全缓解率（严格 CR+CR）至少提高 15%。共有 120 名 MM 患者 1:1 随机分组。ASCT 后 sCR/CR 率在 BenMel 组高于 Mel 组（70.0%比 51.7%；p=0.039）。BenMel 组有 3 例（5.0%）患者出现可逆性急性肾功能不全，而 Mel 组无患者出现。流式细胞术检测到 26 例（45.6%）BenMel 患者和 22 例（37.9%）Mel 组患者存在微小残留病阴性（<10-5）（p=0.375）。我们的数据表明，与标准的 Mel 单药治疗相比，BenMel HDCT 安全且可提高 sCR/CR 率。
