白消安联合氟达拉滨低毒预处理异基因造血干细胞移植治疗恶性血液病13例

 【摘要】　目的　探讨经白消安+氟达拉滨（Bu+Flu）方案低毒清髓预处理异基因造血干细胞移植（allo-HSCT）治疗恶性血液病的临床疗效。方法　对13例接受Bu+Flu方案预处理造血干细胞移植白血病患者的临床资料进行回顾性分析。预处理方案为Bu+Flu，同胞不全相合和非血缘移植患者加用兔抗人胸腺细胞免疫球蛋白（ATG）。用环孢素A+短疗程甲氨蝶呤或环孢素A+吗替麦考酚酯预防移植物抗宿主病（GVHD）。采用DNA短串联重复序列（STR）多态性分析方法鉴定供者干细胞植入情况。结果　13例患者均能耐受Bu+Flu预处理方案，未发生严重的预处理相关并发症。中性粒细胞植活中位时间11 d（9~15 d），血小板植活中位时间13 d（8~25 d）。10例患者造血重建，检测其外周血白细胞STR-DNA证实均为100 %完全供者植入。出现急性GVHD 5例（38.5 %），可评估的10例患者中，出现慢性GVHD 4例（40.0 %）；无Ⅱ度以上重型GVHD。中位随访11个月（1~39个月），总生存率为76.9 %（10／13），无病生存率为61.5%（8／13）。死亡病例原因均为疾病复发。结论　Bu+Flu方案低毒清髓预处理可减轻移植相关并发症，患者有很好耐受性和较好疗效。

Allogeneic peripheral blood stem cell transplantation using a low toxicity conditioning regimen with fludarabine and busulfan for 13 patients with hematological malignancies

Objective To discuss the clinical effect of fludarabine and busulfan （Bu＋Flu） as a low toxicity myeloablative conditioning regimen for allogeneic peripheral blood stem cell transplantation （allo- HSCT） in leukemia patients. Methods Clinical data of 13 patients with hematological malignancies receiving conditioning regimen with Bu＋Flu for allo-HSCT were analyzed retrospectively. Conditioning regimen was Bu＋Flu, compatriot mismatched and unrelated transplantation combined with rabbit anti-human thymocytes immune globulin （ATG）. CsA＋short course of methotrexate or CsA ＋ myeophenolate mofetil were used to prevent graft-versus-host disease （GVHD）. DNA sequencing of short tandem repeat （STR） polymorphism analysis method was performed for identification of donor stem cells implantation. Results 13 patients all tolerated with this conditioning regimen well, no serious complications occurred. Neutrophil engraftment was at 9-15 days （median 11 days）, platelet engraftment at 8-25 days （median 13 days）. 10 patients achieved hematopoiesis reconstitution with their full donor chimerisms confirmed by STR-DNA analysis. Acute GVHD occurred in 5 cases, accounting for 38.5%. Chronic GVHD occurred in 4 cases of 10 patients could be assessed, accounting for 40.0 %. Severe GVHD more than 11 degree did not happen. 1-39 months （median time 11 months） of follow-up revealed the overall survival rate of 76.9 %（10/13）, disease-free survival of 61.5% （8/13）. The causes for death were relapse in all. Conclusion The conditioning regimen with Bu＋Flu has low toxicity, well tolerance and better effect.