CAJ | 학술논문

目的观察FBCA(氟达拉滨十白消安+环磷酰胺+ATG)预处理方案对接受异基因造血干细胞移植(allo-HSCT)治疗重型再生障碍性贫血(SAA)患者的供者造血干细胞持续植入的影响.方法 2000年1月至2011年6月,接受allo-HSCT的22例SAA患者纳入研究,男12例、女10例,中位年龄21(5～52)岁.外周血造血干细胞移植9例,外周血与骨髓造血干细胞共移植13例;HLA全相合亲缘供者14例,HLA不全相合亲缘供者8例.预处理方案:氟达拉滨30 mg·m-2·d-1×5 d,白消安3mg/kg×2 d,环磷酰胺60 mg·kg-1·d-1×2 d,兔ATG 2.5 mg·kg-1·d-1×5 d.采用环孢素+短程甲氨蝶呤方案预防移植物抗宿主病(GVHD).于移植后1、3、6、12、24个月通过短串联重复序列(STR)-PCR法观察患者异基因干细胞植入.分析患者长期生存及移植并发症情况.结果 22例SAA患者中性粒细胞植入与血小板植入的中位时间分别为15(11～22)、16(12～27)d.22例患者中2例(9.1％)发生Ⅰ～Ⅲ度急性GVHD,3例(15.8％)发生慢性局限型GVHD.中位随访24(1～141)个月,移植相关死亡3例(巨细胞病毒肺炎、急性GVHD、重症肺感染各1例),19例患者无病生存,中位生存时间为24.0(0.5～140.5)个月.存活的19例患者在allo-HSCT后不同时间点STR-PCR检测结果均为完全供者嵌合体.结论在SAA患者allo-HSCT中,采用免疫抑制较强的FBCA方案进行预处理可获得供者造血干细胞稳定持续植入.
목적 관찰FBCA(불체랍빈십백소안+배린선알+ATG)예처리방안대접수이기인조혈간세포이식(allo-HSCT)치료중형재생장애성빈혈(SAA)환자적공자조혈간세포지속식입적영향.방법 2000년1월지2011년6월,접수allo-HSCT적22례SAA환자납입연구,남12례、녀10례,중위년령21(5～52)세.외주혈조혈간세포이식9례,외주혈여골수조혈간세포공이식13례;HLA전상합친연공자14례,HLA불전상합친연공자8례.예처리방안:불체랍빈30 mg·m-2·d-1×5 d,백소안3mg/kg×2 d,배린선알60 mg·kg-1·d-1×2 d,토ATG 2.5 mg·kg-1·d-1×5 d.채용배포소+단정갑안접령방안예방이식물항숙주병(GVHD).우이식후1、3、6、12、24개월통과단천련중복서렬(STR)-PCR법관찰환자이기인간세포식입.분석환자장기생존급이식병발증정황.결과 22례SAA환자중성립세포식입여혈소판식입적중위시간분별위15(11～22)、16(12～27)d.22례환자중2례(9.1％)발생Ⅰ～Ⅲ도급성GVHD,3례(15.8％)발생만성국한형GVHD.중위수방24(1～141)개월,이식상관사망3례(거세포병독폐염、급성GVHD、중증폐감염각1례),19례환자무병생존,중위생존시간위24.0(0.5～140.5)개월.존활적19례환자재allo-HSCT후불동시간점STR-PCR검측결과균위완전공자감합체.결론 재SAA환자allo-HSCT중,채용면역억제교강적FBCA방안진행예처리가획득공자조혈간세포은정지속식입.
Objective To observe the effects of increased-intensity conditioning regimen with FBCA (Fludarabine,Busulfan,Cyclophosphamide,and Antithymocyte globublin) for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in acquired severe aplastic anemia (SAA).Methods From January 2000 to June 2011,twenty-two patients (male 12,female 10)with SAA underwent allo-HSCT with FBCA conditioning regimen which consisted of fludarabine (30 mg·m-2· d-1× 5d),busulfan (3 mg/kg×2 d),cyclophosphamide (60 mg·kg-1 ·d-1×2 d) and ATG (2.5 mg·kg-1 ·d-1×5 d).GVHD prophylaxis was performed by cyclosporine and short-term course methotrexate.Nine patients received mobilized peripheral blood stem cells transplantation and 13 patients underwent mobilized peripheral blood combined with bone marrow stem cells.Fourteen cases were human leukocyte antigen (HLA)-matched related donors,while the other 8 cases were HLA-haploidentical transplantation.Engraftment was documented by short tandem repeats with polymerase chain reaction (STR-PCR) on approximately day + 30,+ 90,+ 180,+ 1 year and + 2 year,respectively.Long-term survival and transplantation-related complications were analyzed.Results All patients obtained prompt and sustained hematopoietic reconstitution.Median time for neutrophil and PLT engraftment was 15 (range:11-22) days and 16 (range:12-27) days,respectively.All patients were full donor chimerism identified by STR-PCR.2 of the total 22 cases (9.1％) had grade Ⅰ-Ⅲ acute GVHD and 3 (15.8％) was chronic GVHD.Three patients (13.6％) died of transplantation related mortality and the other 19 cases were disease-free survival with a median time of 24 (range:0.5-140.5) months.The causes of death were cytomegalovirus pneumonia (n=1),acute GVHD (n=1) and severe pulmonaw infection (n=1).Conclusion Increasedintensity of FBCA conditioning regimen could favor donor stem cell sustained engraftment for allo-HSCT in SAA.