North West London National Health Services Trust, Harrow, Middlessex, United Kingdom.
J Clin Oncol. 2010 Nov 20;28(33):4926-34. doi: 10.1200/JCO.2009.27.3607. Epub 2010 Oct 18.
Allogeneic stem-cell transplantation (alloSCT) is a curative therapeutic option for patients with low-grade lymphoid malignancies. Information regarding alloSCT in Waldenström macroglobulinemia (WM) is limited. This study presents the long-term outcome of a large series of patients with WM treated with alloSCT.
A total of 86 patients received allograft by using either myeloablative (MAC; n = 37) or reduced-intensity conditioning (RIC; n = 49) regimens and were retrospectively studied. The median age was 49 years (range, 23 to 64 years); 47 patients had received three or more previous lines of therapy, and eight patients had experienced failure on a prior autologous stem-cell transplantation. A total of 59 patients (68.6%) had chemotherapy-sensitive disease at the time of alloSCT. Median follow-up of the surviving patients was 50 months (7 to 142 months).
Nonrelapse mortality (NRM) at 3 years was 33% for MAC and 23% for RIC. The overall response rate was 75.6%. The relapse rates (RRs) at 3 years were 11% for MAC and 25% for RIC. Fourteen patients received donor lymphocyte infusions (DLIs) for disease relapse. PFS and OS at 5 years were 56% and 62% for MAC and 49% and 64% for RIC, respectively. The occurrence of chronic graft-versus-host disease (cGVHD) was associated with a higher NRM and a lower RR, leading to an improvement in PFS.
alloSCT can induce durable remissions in a selected population of young and heavily pretreated patients with WM. The low RR, the achievement of additional disease responses after DLIs, and the lower RR in patients developing cGVHD suggest the existence of a clinically relevant graft-versus-WM effect.
异基因造血干细胞移植(alloSCT)是治疗低级别淋巴恶性肿瘤患者的一种有治愈希望的治疗选择。关于 Waldenström 巨球蛋白血症(WM)患者接受 alloSCT 的信息有限。本研究介绍了一组接受 alloSCT 治疗的 WM 患者的长期结果。
共有 86 例患者接受了同种异体移植,其中 37 例采用了清髓性(MAC)预处理方案,49 例采用了强度降低的预处理方案(RIC)。中位年龄为 49 岁(范围为 23 岁至 64 岁);47 例患者接受了三线或以上的治疗,8 例患者在接受自体造血干细胞移植后复发。86 例患者中,59 例(68.6%)在接受 alloSCT 时患有化疗敏感疾病。存活患者的中位随访时间为 50 个月(7 至 142 个月)。
MAC 和 RIC 组 3 年非复发死亡率(NRM)分别为 33%和 23%。总缓解率为 75.6%。MAC 和 RIC 组 3 年的复发率(RR)分别为 11%和 25%。14 例患者因疾病复发接受了供者淋巴细胞输注(DLI)。MAC 和 RIC 组 5 年的无进展生存率(PFS)和总生存率(OS)分别为 56%和 62%、49%和 64%。慢性移植物抗宿主病(cGVHD)的发生与较高的 NRM 和较低的 RR 相关,从而改善了 PFS。
alloSCT 可以在年轻且接受过多线治疗的 WM 患者亚群中诱导持久缓解。低 RR、DLI 后获得额外的疾病缓解以及发生 cGVHD 的患者 RR 较低,提示存在有临床意义的移植物抗 WM 效应。