• 小剂量骨髓联合外周血造血干细胞移植治疗重型再生障碍性贫血18例临床研究
  • 彭文.小剂量骨髓联合外周血造血干细胞移植治疗重型再生障碍性贫血18例临床研究[J].内科急危重症杂志,2024,30(3):229-234
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    DOI:10.11768/nkjwzzzz20240307
    中文关键词:  小剂量骨髓  外周造血干细胞  异基因造血干细胞移植  重型再生障碍性贫血
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    彭文 重庆医科大学附属第一医院 
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    中文摘要:
          摘要 目的:探讨小剂量骨髓联合外周血造血干细胞(PBSC)移植治疗重型再生障碍性贫血(SAA)的疗效及安全性。方法:纳入以小剂量骨髓(中位体积200 mL)联合PBSC移植治疗的18例SAA患者的临床资料,回顾分析其造血重建情况、移植物抗宿主病(GVHD)发生率、感染发生率及生存率等指标。结果:18例患者的中位年龄27(13~52)岁,男11例,女7例;SAA -Ⅰ型15例,SAA -Ⅱ型3例;单倍体相合供者15例,同胞全相合供者3例;预处理:氟达拉滨+环磷酰胺+抗人胸腺球蛋白方案2例,氟达拉滨+环磷酰胺+移植后环磷酰胺方案8例,氟达拉滨+环磷酰胺+抗人胸腺球蛋白+移植后环磷酰胺方案8例;采用环孢素或他克莫司+短疗程甲氨蝶呤联合吗替麦考酚酯预防GVHD。所有患者均获得造血重建,仅1例患者出现继发性移植物功能不良;10例(55.6%)出现总计19例次移植后感染,其中10例次为巨细胞病毒(CMV)或EB病毒(EBV)血症,6例次细菌感染;急性GVHD发生率为16.7%,其中Ⅰ度1例,Ⅱ度2例;慢性GVHD发生率为16.7%,均为轻度;中位随访26(2~64)个月,移植后2年总生存率为72.2%(95%CI: 51.4~93.0)。5例患者死亡,4例死于肺部感染,1例死于颅内感染。结论:采用小剂量骨髓联合PBSC是SAA患者进行造血干细胞移植的有效且可行的移植物选择策略。
    英文摘要:
          Abstract Objective: We analyzed the outcome of patients with severe aplastic anemia (SAA) undergoing allogeneic hematopoietic stem cell transplantation who received both low-dose bone marrow and peripheral blood stem cells (PBSC), and assessed the efficacy and safety of this stem cell source. Methods: In this retrospective cohort study, we included patients with newly diagnosed SAA who received hematopoietic stem cell transplantation with low-dose bone marrow (median 200mL, range 80-280mL) and PBSC. The analysis focused on their hematopoietic reconstruction, incidence of graft-versus-host disease (GVHD), occurrence of infections, overall survival rate, and other relevant indicators. Results: The median age of the 18 patients was 27 years (range, 13-52). There were 11 males and 11 females. There were 15 cases of severe aplastic anemia type I (SAA-I) and 3 cases of type Ⅱ (SAA-Ⅱ). There were 15 cases of haploidentical donors and 3 cases of matched sibling donors. The conditioning regimen consisted of the following: Fludarabine/cyclophosphamide/anti thymocyte globulin regimen (n= 2), Fludarabine/cyclophosphamide and post-transplant cyclophosphamide regimen (n= 8), Fludarabine/cyclophosphamide/anti thymocyte globulin and post-transplant cyclophosphamide regimen (n= 8). Cyclosporine or tacrolimus in combination with methotrexate and mycophenolate mofetil was used to GVHD prophylaxis. All patients achieved durable engraftment, but one patient experienced secondary poor graft function. A total of 10 (55.6%) individual patients experienced infections after transplant. Of a total of 19 documented infection events, 10 were attributed to CMV or EBV reactivation, and 6 to bacterial infections. The incidence of acute GVHD was 16.7%, including one case of grade I and two cases of grade II, and that of mild chronic GVHD was 16.7%. The two-year overall survival rate was 72.2% (95% CI: 51.4-93.0). The mortality of 5 patients was attributed to infectious complications, including 4 cases of pulmonary infections and one case of intracranial infection. Conclusion: The combination of low-dose bone marrow and peripheral blood appears to be a potentially effective and feasible graft source for hematopoietic stem cell transplantation in SAA patients.