单倍体相合造血干细胞移植治疗高危白血病

背景：异基因造血干细胞移植是治疗高危白血病的主要方法，单倍体相合的造血干细胞移植扩展了移植的应用范围。 目的：观察“改良Bu/Cy+ATG”为预处理方案的单倍体相合造血干细胞移植治疗高危白血病的疗效。 方法：对19例高危白血病患者，均采用“改良Bu/Cy+ATG”预处理方案，采用外周血造血干细胞移植5例，外周血+骨髓造血干细胞移植14例。应用甲氨蝶呤，环孢素A，吗替麦考酚酯预防移植物抗宿主病。 结果与结论：①短期疗效：中性粒细胞恢复的中位时间为12(8~20) d；血小板恢复的中位时间为13(10~31) d；移植后100 d内，移植相关死亡率为(15.8±8.4)%。②移植物抗宿主病发生情况：Ⅰ~Ⅳ度急性移植物抗宿主病总发生率(63.1±11.1)%，慢性移植物抗宿主病发生率(54.54±15.0)%。③远期疗效：2年无病生存率为(28.2±15.5)%，2年总体生存率为(46.9±16.5)%。结果提示，高危白血病无人类白细胞抗原相合血缘供者及无人类白细胞抗原相合非血缘供者，而又急需进行挽救性移植时，“改良Bu/Cy+ATG”为预处理方案的单倍体相合造血干细胞移植是一种可行的选择。

Clinical effects of haploidentical hematopoietic stem cell transplantation on high-risk leukemia

BACKGROUND:Allogeneic hematopoietic stem cell transplantation is the main therapy of leukemia. The haploidentical hematopoietic stem cell transplantation can expand the application of the transplantation. OBJECTIVE:Observe the effect of “modi?ed Bu/Cy+ATG” conditioning regimen of haploidentical hematopoietic stem cell transplantation for patients with high-risk leukemia. METHODS:Nineteen patients with high-risk leukemia received a “modi?ed Bu/Cy+ATG” conditioning regimen: peripheral blood stem cells transplantation in 5 cases, and peripheral blood and bone marrow stem cells transplantation in 14 cases. Methotrexate, cyclosporine A, mycophenolate mofetil were used to prevent graft-versus-host disease (GVHD). Prostaglandin E1 was applied to prevent liver veno-occlusive disease (VOD). RESULTS AND CONCLUSION: ①Short term effect: the median neutrophil recovery time was 12 (8-20) d; the median platelet recovery time was 13 (10-31) d, and all the patients were successfully implanted. Transplant-related mortality rate was (15.8±8.4)% within 100 days after transplantation. ②GVHD incidence: the overall incidence of Ⅰ- Ⅳdegree of acute GVHD was (63.1±11.1)%, the incidence of chronic GVHD was (54.54±15.0)%. ③Long-term effect: 2-year disease-free survival rate was (28.2±15.5)%. 2-year overall survival rate was (46.9±16.5)%. For the patient with high-risk leukemia, who has not a HLA-matched related or unrelated donor but needs a salvage transplant, the haploidentical hematopoietic stem cell transplantation with “modi?ed Bu/Cy+ATG” conditioning regimen is a feasible selection.