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目的探讨非清髓异基因造血干细胞移植(NST)治疗骨髓增生异常综合征(MDS)的疗效。方法 14例有血缘相关HLA相合的MDS患者接受NST治疗。骨髓增生异常综合征-难治性贫血(MDS-RA)患者非清髓预处理方案为:氟达拉滨(Flud)+环磷酰胺(CTX)+抗人胸腺细胞免疫球蛋白(ATG);骨髓异常综合征-难治性贫血伴原始细胞过多(MDSRAEB)患者非清髓预处理方案为:在MDS-RA患者非清髓预处理方案基础上加用马利兰(Bu)。移植物抗宿主病(GVHD)预防采用环孢素(CSA)联合霉酚酸酯(MMF)。结果 13例患者造血均获得快速重建,1例造血未恢复。中性粒细胞在术后第9～12天恢复到0.5×109/L以上,12例患者血小板在术后第11～21天恢复到30×109/L以上。14例中12例完全植入,2例混合嵌合植入,其中1例经过二次移植后达到完全植入,另1例在移植后2个月发生移植排斥,移植后70d因脑出血死亡。1例发生急性Ⅱ度肠道GVHD,经静脉给予他克莫司(FK506)和甲泼尼龙治疗后病情控制,但患者在后期应用FK506治疗过程中出现癫痫持续发作,经多种抗癫痫药物治疗未见好转,家属放弃治疗自动出院后死亡。14例中未见慢性GVHD和间质性肺炎发生。随访36～133个月,11例患者现无病存活,其中10例存活60个月以上,3例死亡。结论 NST治疗MDS安全可行,可为治愈MDS提供新手段。
Objective To investigate the efficacy of nonmyeloablative allogeneic hematopoietic stem cell transplantation (NST) in the treatment of myelodysplastic syndrome (MDS). Methods Fourteen patients with MDS associated with HLA-matched HLA were treated with NST. Myelodysplastic syndrome-refractory anemia (MDS-RA) patients with non-myelo-preconditioning regimen: fludarabine (CTX) + anti-human thymocyte immunoglobulin (ATG) Myelodysplastic Syndromes - Non-myeloablative preconditioning in patients with refractory anemia and hyperproliferation (MDSRAEB) is the use of non-myeloablative preconditioning with MD. Prevention of graft-versus-host disease (GVHD) uses cyclosporine (CSA) in combination with mycophenolate mofetil (MMF). Results Thirteen patients achieved rapid reconstruction of hematopoiesis and no hematopoiesis in one. Neutrophils returned to above 0.5 × 109 / L on the 9th to 12th postoperative days. Platelets of 12 patients returned to above 30 × 109 / L on the 11th to 21st days after operation. Of the 14 cases, 12 cases were completely implanted and 2 cases were implanted by mixed chimerism. One case achieved complete engraftment after secondary transplantation and another case had graft rejection 2 months after transplantation, and died of cerebral hemorrhage 70 days after transplantation . One patient developed acute grade II GVHD and was treated with intravenous administration of tacrolimus (FK506) and methylprednisolone. However, patients undergoing FK506 treatment experienced persistent epileptic seizures and were treated with various antiepileptic drugs No improvement, family members give up treatment and died after being discharged from the hospital automatically. No cases of chronic GVHD and interstitial pneumonia occurred in 14 cases. During the follow-up of 36 to 133 months, 11 patients survived without disease, of whom 10 survived for more than 60 months and 3 died. Conclusion The treatment of MDS by NST is safe and feasible, which can provide a new means to cure MDS.