以静脉注射和口服白消安为预处理行HLA半相合造血干细胞移植治疗恶性血液病的比较

Comparative study on clinical outcomes of HLA haploidentical hematopoietic stem cell transplantation for hematologic malignancies between intravenous and oral busulfan conditioning regimens

目的:比较以静脉注射和口服白消安(Bu)为预处理方案行亲属间HLA半相合外周血造血干细胞移植在造血重建、移植物抗宿主病(GVHD)、预处理相关毒性及并发症和疗效的差异,观察白消安注射液(ivBu)的疗效和安全性。方法:20例恶性血液病患者中,10例采用静脉注射Bu为预处理方案(ivBu组),10例采用口服Bu为预处理方案(口服Bu组),回顾性分析2组移植后造血重建、GVHD、预处理相关毒性及并发症和疗效情况。结果:ivBu组和口服Bu组移植后均获造血功能重建,2组中性粒细胞绝对值≥0.5×109/L的中位时间均为14d(P>0.05),血小板计数≥20×109/L的中位时间分别为12和13d(P>0.05),ivBu组急性GVHD发生率为10.0%,低于口服Bu组(50.0%)(P>0.05),慢性GVHD的发生率在2组中分别为20.0%和60.0%(P>0.05),2组在预处理相关毒性及并发症方面差异无统计学意义(P>0.05),ivBu组1例因间质性肺炎死亡,2组分别有1例和2例复发,余16例无病生存。结论:ivBu组和口服Bu组在造血重建、GVHD、预处理相关毒性及并发症等的差异均无统计学意义,以ivBu为主的预处理方案治疗血液病是安全有效的。

Objective： To compare the hematopoietic reconstitution, incidience of graft versus-host disease （GVHD）, transplantation-related complication and clinical outcomes of HLA haploidentical hematopoietic stem cell transplantation between intravenous and oral busulfan as conditioning regimens, and observe the efficacy and complications of intravenous busulfan （ivBu） used as conditioning regimen. Methods：Patients were randomly dis tributed into intravenous busulfan （ivBu） group （10 cases） and oral Bu group （10 cases）, the clinical results of two groups were compared retrospectively. Results： All the patients were engrafted successfully, compared ivBu group and oral Bu group, the median time of recovery of absolute neutrophil count （≥0.5 × 10^9/L） was 14 days for either （ P 〉0.05）, the median time of recovery of BPC （≥ 20 × 10^9/L） was 12 and 13 days, respectively （ P 〉0.05）. The incidence of acute GVHD （10.0% vs 50.0%）, chronic GVHD （20.0% vs 60.0%） and transplan tation-related complication between ivBu group and oral Bu group all have no statistical significance （ P 〉0.05）. Both transplant-related mortality and relapse rate were 10% in ivBu group, whereas those were 0% and 20% respectively in oral Bu group. Conditionging-related toxicity and complications were not significant different between two groups. 1 pateint died of interstitial pneumonia in ivBu group, 1 patient relapsed in ivBu group while 2 pa tients in oral Bu group. 16 patients were alive in a disease-free situation. Conclusion： Hematopoietic reeonstitution, incidienee of GVHD and transplantation-related complication between two groups all have no significant differences. The conditioning regimen of intravenous busulfan combined with cyclophosphamide is relatively easy to ad minister and well tolerated in patients.