North West London, NHS Trust, Watford Rd, Harrow, Middlessex, HA1 3UJ, United Kingdom.
J Clin Oncol. 2010 May 1;28(13):2227-32. doi: 10.1200/JCO.2009.24.4905. Epub 2010 Apr 5.
The role of autologous stem-cell transplantation (ASCT) in Waldenström macroglobulinemia (WM) is not defined. The aim of this study was to analyze the results of ASCT in patients with WM and to determine the prognostic factors that have a significant impact on outcome.
We analyzed 158 adult patients with WM reported to the European Group for Blood and Marrow Transplantation (EBMT) between January 1991 and December 2005. Median time from diagnosis to ASCT was 1.7 years (range, 0.3 to 20.3 years), 32% of the patients experienced treatment failure with at least three lines of therapy, and 93% had sensitive disease at the time of ASCT. Conditioning regimen was total-body irradiation-based in 45 patients. Median follow-up for surviving patients was 4.2 years (range, 0.5 to 14.8 years).
Nonrelapse mortality was 3.8% at 1 year. Ten patients developed a secondary malignancy, with a cumulative incidence of 8.4% at 5 years. Relapse rate was 52.1% at 5 years. Progression-free survival (PFS) and overall survival were 39.7% and 68.5%, respectively, at 5 years and were significantly influenced by number of lines of therapy and chemorefractoriness at ASCT. The achievement of a negative immunofixation after ASCT had a positive impact on PFS after ASCT. When used as consolidation at first response, ASCT provided a PFS of 44% at 5 years.
ASCT is a feasible procedure in young patients with advanced WM. ASCT should not be offered to patients with chemoresistant disease and to those who received more than three lines of therapy.
自体干细胞移植(ASCT)在华氏巨球蛋白血症(WM)中的作用尚未确定。本研究旨在分析 WM 患者 ASCT 的结果,并确定对预后有显著影响的预后因素。
我们分析了 1991 年 1 月至 2005 年 12 月期间向欧洲血液和骨髓移植组(EBMT)报告的 158 例成人 WM 患者。从诊断到 ASCT 的中位时间为 1.7 年(范围,0.3 至 20.3 年),32%的患者经历了至少三种方案的治疗失败,93%的患者在 ASCT 时具有敏感疾病。45 例患者采用全身照射为基础的预处理方案。对存活患者的中位随访时间为 4.2 年(范围,0.5 至 14.8 年)。
1 年时非复发死亡率为 3.8%。10 例患者发生继发性恶性肿瘤,5 年累积发生率为 8.4%。复发率为 5 年时的 52.1%。无进展生存(PFS)和总生存(OS)分别为 5 年时的 39.7%和 68.5%,并且受治疗线数和 ASCT 时化疗耐药性的显著影响。ASCT 后免疫固定阴性的获得对 ASCT 后 PFS 有积极影响。ASCT 作为首次缓解的巩固治疗时,5 年时 PFS 为 44%。
ASCT 是年轻晚期 WM 患者可行的治疗方法。不应对化疗耐药的患者和接受过三线以上治疗的患者提供 ASCT。