CAJ | 학술논문

目的探讨含减低剂量环磷酰胺为预处理方案的HLA全相合异基因造血干细胞移植(allo-HSCT)治疗范科尼贫血(FA)的临床效果.方法以HLA匹配同胞供者allo-HSCT治疗FA伴再生障碍性贫血患者3例,3例患者的预处理方案均为抗胸腺细胞球蛋白(ATG)+氟达拉滨+减低剂量的环磷酰胺(总剂量40 mg/kg).结果 3例患者均植活,其中1例患者在移植后118 d发生晚期移植物排斥,于移植后182 d行2次移植后再次植活.3例患者已分别随访21、20和27个月,血象均正常,为完全供者嵌合状态,未发生严重移植物抗宿主病和肝静脉闭塞症等严重并发症.结论对于发生FA伴再生障碍性贫血的患者,可以采用ATG+氟达拉滨+减低剂量环磷酰胺为预处理方案的HLA全相合allo-HSCT治疗.
목적 탐토함감저제량배린선알위예처리방안적HLA전상합이기인조혈간세포이식(allo-HSCT)치료범과니빈혈(FA)적림상효과.방법 이HLA필배동포공자allo-HSCT치료FA반재생장애성빈혈환자3례,3례환자적예처리방안균위항흉선세포구단백(ATG)+불체랍빈+감저제량적배린선알(총제량40 mg/kg).결과 3례환자균식활,기중1례환자재이식후118 d발생만기이식물배척,우이식후182 d행2차이식후재차식활.3례환자이분별수방21、20화27개월,혈상균정상,위완전공자감합상태,미발생엄중이식물항숙주병화간정맥폐새증등엄중병발증.결론 대우발생FA반재생장애성빈혈적환자,가이채용ATG+불체랍빈+감저제량배린선알위예처리방안적HLA전상합allo-HSCT치료.
Objective To investigate the clinic effect of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for patients with Fanconi's anemia (FA).Method Three cases of FA were successfully subjected to matched sibling donor allo-HSCT,and a fludarabine (Flu)-based conditioning regimen was used.The regimen contained Flu,reduced-dose cyclophosphamide (40mg/kg) and antihuman thymocyte globulin.Result All 3 patients achieved engraftment with donor chimerism.One patient had secondary graft failure at the + 118th day,and a second allo-HSCT performed at + 182nd day engrafted with donor chimerism again.During a follow-up period of 21,20and 27 months,respectively,all 3 patients were transfusion-independent,and maintained full donor chimerism.None of the patients developed severe graft-versus-host disease,hepatic veno-occlusive disease or other serious complications.Conclusion Sibling donor allo-HSCT with this alternative conditioning regimen can be used to effectively treat FA patients who develop aplasia.