[匹配同胞供体异基因造血干细胞移植治疗骨髓纤维化的临床结果]。

R Ma, B R Tang, T T Han, X Y Luo, W Han, Y Chen, X D Mo, L P Xu, X H Zhang, Y Wang, X J Huang, Y Q Sun
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引用次数: 0

摘要

目的评估配对同胞供者异基因造血干细胞移植(allo-HSCT)治疗骨髓纤维化(MF)的有效性和安全性。方法:本病例系列回顾性研究了2008年12月至2023年12月在北京大学人民医院血液科接受异基因造血干细胞移植的18例骨髓纤维化患者的临床资料。研究采用 Kaplan-Meier 生存分析和竞争风险模型评估了 3 年总生存期(OS)、无病生存期(DFS)、累积复发率(CIR)和移植相关死亡率(TRM)的概率。此外,还对移植相关并发症进行了分析。结果在纳入的 18 名患者中,男性 12 人,女性 6 人,中位年龄为 50 岁(范围:28-64 岁)。所有 18 名患者均实现了中性粒细胞移植,中性粒细胞移植时间[M(Q1,Q3)]为 16.0(11.8,18.0)天。12名患者实现了血小板移植,血小板移植时间为21.0(16.2,43.2)天。6名患者出现Ⅱ级至Ⅳ级急性移植物抗宿主病(GVHD),6名患者出现慢性GVHD。移植后3年的OS率和DFS率分别为62.2%和52.2%。3年CIR和TRM分别为29.7%和24.6%。四名患者在随访期间死亡,主要死因是感染。结论配型同胞异体造血干细胞移植是治疗骨髓纤维化的可行方案。
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[Clinical outcomes of allogeneic hematopoietic stem cell transplantation from matched sibling donor for myelofibrosis].

Objective: To evaluate the efficacy and safety of matched sibling donor allogeneic hematopoietic stem cell transplantation (allo-HSCT) for the treatment of myelofibrosis (MF). Methods: In this case series, the clinical data of 18 patients with MF who received allo-HSCT in the Department of Hematology, Peking University People's Hospital from December 2008 to December 2023 were retrospectively studied. Kaplan-Meier survival analysis and competitive risk model were used to evaluate the probabilities of 3-year overall survival (OS), disease-free survival (DFS), cumulative incidence of relapse (CIR), and transplant related mortality (TRM). The transplant related complications were also analyzed. Results: Among the 18 patients included, there were 12 males and 6 females, with a median age of 50 (range: 28-64) years. All 18 patients achieved neutrophil engraftment, and the time of neutrophil engraftment [M (Q1, Q3)] was 16.0 (11.8, 18.0) days. Twelve patients achieved platelet engraftment, and the platelet engraftment time was 21.0 (16.2, 43.2) days. Six patients had grade Ⅱ to Ⅳ acute graft-versus-host disease (GVHD), and six patients had chronic GVHD. The 3-year OS rate and DFS rate after transplantation were 62.2% and 52.2%, respectively. The 3-year CIR and TRM were 29.7% and 24.6%, respectively. Four patients died during follow-up, with the main cause of death being infections. Conclusion: Matched sibling allo-HSCT is a feasible option for the treatment of MF.

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