Second allogeneic stem cell transplantation (alloSCT2) is among the most effective treatments for acute myeloid leukemia (AML) relapse after first alloSCT (alloSCT1). Long-term EBMT registry data were used to provide large scale, up-to-date outcome results and to identify factors for improved outcome. Among 1540 recipients of alloSCT2, increasing age, better disease control and performance status before alloSCT2, more use of alternative donors and higher conditioning intensity represented important trends over time. Between the first (2000–2004) and last (2015–2019) period, two-year overall and leukemia-free survival (OS/LFS) increased considerably (OS: 22.5–35%, LFS: 14.5–24.5%). Cumulative relapse incidence (RI) decreased from 64% to 50.7%, whereas graft-versus-host disease and non-relapse mortality (NRM) remained unchanged. In multivariable analysis, later period of alloSCT2 was associated with improved OS/LFS (HR = 0.47/0.53) and reduced RI (HR = 0.44). Beyond, remission duration, disease stage and patient performance score were factors for OS, LFS, RI, and NRM. Myeloablative conditioning for alloSCT2 decreased RI without increasing NRM, leading to improved OS/LFS. Haploidentical or unrelated donors and older age were associated with higher NRM and inferior OS. In summary, outcome after alloSCT2 has continuously improved over the last two decades despite increasing patient age. The identified factors provide clues for the optimized implementation of alloSCT2.
二次异基因干细胞移植(alloSCT2)是首次异基因干细胞移植(alloSCT1)后急性髓系白血病(AML)复发最有效的治疗方法之一。本研究利用欧洲血液与骨髓移植学会(EBMT)登记处的长期数据,旨在提供大规模、最新的疗效结果,并识别改善预后的影响因素。在1540例接受alloSCT2的患者中,随时间推移呈现以下重要趋势:患者年龄增长、移植前疾病控制及体能状态改善、更多使用替代供者以及预处理强度增强。从早期(2000–2004年)到近期(2015–2019年)阶段,两年总生存率与无白血病生存率(OS/LFS)显著提高(OS:22.5%–35%,LFS:14.5%–24.5%)。累积复发率从64%降至50.7%,而移植物抗宿主病和非复发死亡率保持不变。多变量分析显示,较晚时期的alloSCT2与OS/LFS改善(风险比 = 0.47/0.53)及复发率降低(风险比 = 0.44)相关。此外,缓解持续时间、疾病分期和患者体能评分是影响OS、LFS、复发率和非复发死亡率的重要因素。alloSCT2采用清髓性预处理可降低复发率且不增加非复发死亡率,从而改善OS/LFS。单倍体相合或无关供者以及高龄与较高的非复发死亡率和较差的OS相关。总之,尽管患者年龄不断增加,过去二十年中alloSCT2的疗效持续改善。本研究识别的影响因素为优化alloSCT2的临床应用提供了重要线索。