Cy-VP16-fTBI预处理的干细胞移植治疗恶性血液病的长期疗效

Long-term efficacy of Cy-VP16-fTBI conditioning regimen in hematopoietic stem cell transplantation for hematologic malignancies

目的Cy-VP16-fTBI预处理方案的造血干细胞移植治疗血液系统恶性疾病的长期疗效观察。方法造血干细胞移植治疗16例恶性血液病患者，其中异基因外周血干细胞移植8例，异基因骨髓移植5例，自体外周血干细胞移植3例。所有患者均予环磷酰胺（Cy）＋依托泊苷（VP16）＋标准分次全身放疗（fTBI）预处理方案。结果所有患者均成功植活，白细胞、血小板植入中位时间分别为第15（10-22）天和第20（10-65）天。Ⅱ-Ⅳ度急性移植物抗宿主病（aGVHD）3例（19%），慢性GVHD（cGVHD）7例（43%）。11例（68.75%）患者长期生存，中位生存期75（34-82）个月。6年总体生存率（OS）68.75%，其中第一次完全缓解或慢性粒细胞白血病慢性期患者6年OS为78.75%。6年复发率14.29%，移植满2 a后无患者复发。结论Cy-VP16-fTBI预处理方案的造血干细胞移植安全、有效，其长期疗效明显优于常规化疗。aGVHD发生率较低，但cGVHD在异基因外周血干细胞移植中发生率较高；其他毒副作用较少。

Objective To investigate the long-term efficacy of Cy-VP16-fractionated TBI（frBI） conditioning regimen in hematopoietic stem cell transplantation （PBSCT or BMT） for hematologic malignancies. Methods Sixteen patients with hematologic malignancies received hematopoietic stem cell transplantation. Allo-PBSCT was performed for 8 patients, allo-BMT for 5 and auto-PBSCT for the other 3. Cy-VP16-frBI conditioning regimen was administered to all the patients. Results All the patients were engrafted. The median time （range） to ANC 〉0.5 × 10^9/L and platelet 〉20 × 10^9/L was 15 （10 -22） d and 20 （10 -65） d post transplantation, respectively. Grade Ⅱ-Ⅳ acute GVHD （aGVHD） occurred in 3 （19%） patients and chronic GVHD （cGVHD） in 7 （43%）. Eleven patients （68.75%） were alive with a median of 75 months （34 -82 months） , all of whom were in continuous complete remission（CR）. The 6- year probability of overall survival （OS） for all the 16 patients was 68.75% , and the 6-year OS in the patients transplanted at first CR or chronic phase was 78. 75%. The 6-year relapse rate was 14.29% with no more relapse 2 years post transplantation. Conclusion The application of Cy-VP16-tTBI conditioning regimen in hematopoietic stem cell transplantation is safe and effective with the long-term efficacy significantly better than the regular chemotherapy. The incidence is lower for aGVHD, while higher for cGVHD in allo-transplant. The other side effects are not frequently observed.