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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| 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. |
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