异基因造血细胞移植治疗骨髓增生性肿瘤急变期患者的结局：欧洲血液和骨髓移植学会慢性恶性肿瘤工作组的一项回顾性研究。

Outcomes after allogeneic hematopoietic cell transplant in patients diagnosed with blast phase of myeloproliferative neoplasms: A retrospective study from the Chronic Malignancies Working Party of the European Society for Blood and Marrow Transplantation.

机构信息

Department of Hematology, Vall d'Hebron University Hospital, Vall d'Hebron Institute of Oncology (VHIO), Barcelona, Spain.

EBMT Statistical Unit, Leiden, The Netherlands.

出版信息

Am J Hematol. 2023 Apr;98(4):628-638. doi: 10.1002/ajh.26833. Epub 2023 Jan 13.

DOI:10.1002/ajh.26833

PMID:36606718

Abstract

Allogeneic hematopoietic cell transplant (allo-HCT) provides the only potential route to long-term remission in patients diagnosed with blast phase transformation of myeloproliferative neoplasm (BP-MPN). We report on a large, retrospective European Society for Blood and Marrow Transplantation registry-based study of BP-MPN patients undergoing allo-HCT. BP-MPN patients undergoing first allo-HCT between 2005 and 2019 were included. A total of 663 patients were included. With a median follow-up of 62 months, the estimated 3-year overall survival (OS) was 36% (95% confidence interval [CI], 32-36). Factors associated with lower OS were Karnofsky Performance Score (KPS) <90 (hazard ratio [HR] 1.65, p < .001) and active disease at allo-HCT (HR 1.45, p < .001), whereas patients undergoing allo-HCT more recently associated with a higher OS (HR 0.96, p = .008). In a selected patient's population, the 3-year OS of patients undergoing allo-HCT in complete response (CR) and with a KPS ≥90 was 60%. KPS < 90 (HR 1.4, p = .001) and active disease (HR 1.44, p = .0004) were associated with a lower progression-free survival (PFS). Conversely, most recent allo-HCT associated with a higher PFS (HR 0.96, p = .008). Active disease at allo-HCT (HR 1.34, p = .03) was associated with a higher cumulative incidence of relapse (RI) and allo-HCT in earlier calendar years (HR 0.96, p = .02) associated with a lower RI. Last, KPS < 90 (HR 1.91, p < .001), active disease (HR 1.74, p = .003) and allo-HCT from mismatched related donors were associated with a higher non-relapse mortality (HR 2.66, p = .003). In this large series of BP-MPN patients, about one third were alive at 3 years after transplantation. Patients undergoing allo-HCT in the more recent era, with a KPS ≥90 and in CR at transplant had a better prognosis.

摘要

异基因造血细胞移植（allo-HCT）为诊断为骨髓增生性肿瘤（MPN）爆发期转化（BP-MPN）的患者提供了获得长期缓解的唯一潜在途径。我们报告了一项大型的、基于欧洲血液和骨髓移植协会注册的 BP-MPN 患者 allo-HCT 回顾性研究。纳入了 2005 年至 2019 年间首次 allo-HCT 的 BP-MPN 患者。共纳入 663 例患者。中位随访 62 个月后，估计 3 年总生存率（OS）为 36%（95%置信区间[CI]，32-36）。与较低 OS 相关的因素包括 Karnofsky 表现评分（KPS）<90（风险比[HR]1.65，p<.001）和 allo-HCT 时疾病活跃（HR 1.45，p<.001），而最近接受 allo-HCT 与较高的 OS 相关（HR 0.96，p=.008）。在一个选择的患者人群中，allo-HCT 在完全缓解（CR）且 KPS≥90 的患者的 3 年 OS 为 60%。KPS<90（HR 1.4，p=.001）和疾病活跃（HR 1.44，p=.0004）与无进展生存率（PFS）降低相关。相反，最近的 allo-HCT 与较高的 PFS 相关（HR 0.96，p=.008）。allo-HCT 时疾病活跃（HR 1.34，p=.03）与较高的累积复发率（RI）相关，而较早的日历年份进行 allo-HCT（HR 0.96，p=.02）与较低的 RI 相关。最后，KPS<90（HR 1.91，p<.001）、疾病活跃（HR 1.74，p=.003）和来自不相合亲缘供体的 allo-HCT 与较高的非复发死亡率（HR 2.66，p=.003）相关。在这项 BP-MPN 患者的大型系列研究中，约三分之一的患者在移植后 3 年时仍存活。在较新的时期接受 allo-HCT、KPS≥90 和移植时处于 CR 的患者具有更好的预后。