Division of Hematology, Mayo Clinic, 200 First Street SW, Rochester, MN, 55905, USA.
Department of Internal Medicine, Mayo Clinic, Rochester, MN, USA.
Ann Hematol. 2018 Aug;97(8):1417-1425. doi: 10.1007/s00277-018-3311-z. Epub 2018 Apr 3.
The treatment approaches for Waldenstrom macroglobulinemia (WM) are largely based upon information from single-arm phase II trials, without comparative data. We compared the efficacy of two commonly used regimens in routine practice (bendamustine-rituximab (BR) and dexamethasone, rituximab plus cyclophosphamide (DRC)) and evaluated their activity with respect to the patients' MYD88 mutation status. Of 160 consecutive patients, 60 received BR (43 with relapsed/refractory WM) and 100 received DRC (50 had relapsed/refractory WM). In the treatment-naïve setting, overall response rate (ORR) was 93% with BR versus 96% with DRC (p = 0.55). Two-year progression-free survival (PFS) with BR and DRC was 88 and 61%, respectively (p = 0.07). In salvage setting, ORR was 95% with BR versus 87% with DRC, p = 0.45; median PFS with BR was 58 versus 32 months with DRC (2-year PFS was 66 versus 53%; p = 0.08). Median disease-specific survival was not reached with BR versus 166 months with DRC (p = 0.51). The time-to-event endpoints and depth of response were independent of the MYD88 mutation status. Grade ≥ 3 adverse events of both regimens were comparable. A trend for longer PFS was observed with BR although the regimens have comparable toxicities. The activity of BR and DRC appears to be unaffected by patients' MYD88 mutation status.
华氏巨球蛋白血症（WM）的治疗方法主要基于单臂 II 期临床试验的信息，没有对照数据。我们比较了两种在常规实践中常用的方案（苯达莫司汀-利妥昔单抗（BR）和地塞米松、利妥昔单抗加环磷酰胺（DRC））的疗效，并评估了它们在患者 MYD88 突变状态方面的活性。在 160 例连续患者中，60 例接受 BR（43 例为复发/难治性 WM），100 例接受 DRC（50 例为复发/难治性 WM）。在初治患者中，BR 的总缓解率（ORR）为 93%，而 DRC 的 ORR 为 96%（p=0.55）。BR 和 DRC 的 2 年无进展生存率（PFS）分别为 88%和 61%（p=0.07）。在挽救治疗中，BR 的 ORR 为 95%，而 DRC 的 ORR 为 87%（p=0.45）；BR 的中位 PFS 为 58 个月，而 DRC 的中位 PFS 为 32 个月（2 年 PFS 为 66%比 53%；p=0.08）。BR 的中位疾病特异性生存率为未达到，而 DRC 的中位疾病特异性生存率为 166 个月（p=0.51）。无事件生存时间和反应深度与 MYD88 突变状态无关。两种方案的 3 级及以上不良事件发生率相当。BR 的 PFS 有延长趋势，尽管两种方案的毒性相当。BR 和 DRC 的活性似乎不受患者 MYD88 突变状态的影响。
