University Medical Center Hamburg Eppendorf, Department of Stem Cell Transplantation, Hamburg, Germany.
Raisa Gorbacheva Memorial Institute for Children Hematology and Transplantology, Saint Petersburg, Russian Federation.
Bone Marrow Transplant. 2021 Nov;56(11):2834-2841. doi: 10.1038/s41409-021-01410-x. Epub 2021 Jul 30.
Allogeneic hematopoietic stem-cell transplantation (HSCT) remains the only curative option for patients with advanced chronic myeloid leukemia (CML). However, outcome is dismal and of short follow-up. The objective of the study was to determine long-term outcome and risk factors in patients with a history of CML Blast Crisis (BC; n = 96) or accelerated phase (n = 51) transplanted between 1990 and 2018. At transplant, patients had a median age of 39 (range 7-76) years and were in ≥CP2 (n = 70), in AP (n = 40) or in BC (n = 37) with a diagnosis-HSCT interval of median 1.9 (range 0.3-24.4) years. Overall survival (OS) amounted 34% (95% CI 22-46) and progression-free survival (PFS) 26% (95% CI 16-36) at 15 years. Adverse risk factors for OS and PFS were low CD34 count in the graft, donor age (>36 years) and BC. Cumulative incidence of Non-Relapse Mortality (NRM) was 28% (95% CI 18-38) and of relapse (RI) 43% (95% CI 33-53) at 15 years. PB-HSCT and HSCT after 2008 were favorable prognostic factors for NRM, while family donor and patient age >39 years were independently associated with higher RI. HSCT resulted in long-term OS in patients with advanced CML. OS was improved in non-BC patients, with donors ≤36 years and with higher CD34 dose in the graft.
异基因造血干细胞移植(HSCT)仍然是晚期慢性髓系白血病(CML)患者的唯一治愈选择。然而,结果令人沮丧,随访时间短。本研究的目的是确定 1990 年至 2018 年间接受 CML 急变期(BC;n=96)或加速期(AP;n=51)治疗的患者的长期结果和危险因素。移植时,患者的中位年龄为 39 岁(范围 7-76 岁),处于 CP2 期(n=70)、AP 期(n=40)或 BC 期(n=37),诊断-移植间隔中位数为 1.9 年(范围 0.3-24.4 年)。15 年时,总生存率(OS)为 34%(95%CI 22-46),无进展生存率(PFS)为 26%(95%CI 16-36)。OS 和 PFS 的不良危险因素是移植物中 CD34 计数低、供者年龄(>36 岁)和 BC。15 年时,非复发死亡率(NRM)累积发生率为 28%(95%CI 18-38),复发率(RI)为 43%(95%CI 33-53)。PB-HSCT 和 2008 年后的 HSCT 是 NRM 的有利预后因素,而亲缘供者和患者年龄>39 岁与更高的 RI 独立相关。HSCT 可使晚期 CML 患者获得长期 OS。非 BC 患者的 OS 得到改善,其供者年龄≤36 岁,移植物中 CD34 剂量较高。