CAJ | 학술논문

目的评价异基因造血干细胞移植(allo-HSCT)治疗重型再生障碍性贫血(SAA)患者的疗效.方法回顾性分析单中心2003年1月至2012年12月接受allo-HSCT的70例SAA患者的临床资料.其中男性40例,女性30例,患者中位年龄为20岁(5～41岁).51例接受HLA匹配同胞供者allo-HSCT(MSD allo-HSCT),其中20例行骨髓移植(BMT),输注CD34+细胞中位数为3.46×106/kg;31例行外周血干细胞移植(PBSCT),输注CD34+细胞中位数为3.22×106/kg.19例接受替代供者allo-HSCT(AD allo-HSCT),其中10例行BMT,输注CD34+细胞中位数为3.10×106/kg;9例行PBSCT,输注CD34+细胞中位数为4.90×106/kg.结果 70例移植后均获得造血重建,中位随访时间为35.5个月(1～119个月),预期5年总体存活率(OS)为(79.0±5.1)％.51例MSDallo-HSCT受者移植后15例发生急性移植物抗宿主病(GVHD),10例发生慢性GVHD,6例发生移植物排斥反应；51例中死亡7例,5年OS为(85.0±5.4)％.19例AD allo-HSCT受者移植后12例发生急性GVHD,7例发生慢性GVHD,1例发生移植物排斥反应；19例中死亡7例,5年OS为(63.2±11.1)％.多因素分析结果显示:行PBSCT(P=0.049)、移植后发生严重(Ⅱ～Ⅳ度)急性GVHD(P =0.025)、严重感染(侵袭性真菌病或巨细胞病毒)(P=0.05)显著降低SAA患者allo-HSCT后疗效.结论 AlbHSCT是治疗SAA患者的有效手段,尤其MSD allo-HSCT疗效显著,但在无HLA匹配同胞供者的SAA患者中也可选择AD allo-HSCT作为替代治疗.采用BMT能有效预防移植后严重(Ⅱ～Ⅳ度)急性GVHD及严重感染(侵袭性真菌病或巨细胞病毒),对于提高SAA患者allo-HSCT疗效至关重要.
목적 평개이기인조혈간세포이식(allo-HSCT)치료중형재생장애성빈혈(SAA)환자적료효.방법 회고성분석단중심2003년1월지2012년12월접수allo-HSCT적70례SAA환자적림상자료.기중남성40례,녀성30례,환자중위년령위20세(5～41세).51례접수HLA필배동포공자allo-HSCT(MSD allo-HSCT),기중20례행골수이식(BMT),수주CD34+세포중위수위3.46×106/kg;31례행외주혈간세포이식(PBSCT),수주CD34+세포중위수위3.22×106/kg.19례접수체대공자allo-HSCT(AD allo-HSCT),기중10례행BMT,수주CD34+세포중위수위3.10×106/kg;9례행PBSCT,수주CD34+세포중위수위4.90×106/kg.결과 70례이식후균획득조혈중건,중위수방시간위35.5개월(1～119개월),예기5년총체존활솔(OS)위(79.0±5.1)％.51례MSDallo-HSCT수자이식후15례발생급성이식물항숙주병(GVHD),10례발생만성GVHD,6례발생이식물배척반응；51례중사망7례,5년OS위(85.0±5.4)％.19례AD allo-HSCT수자이식후12례발생급성GVHD,7례발생만성GVHD,1례발생이식물배척반응；19례중사망7례,5년OS위(63.2±11.1)％.다인소분석결과현시:행PBSCT(P=0.049)、이식후발생엄중(Ⅱ～Ⅳ도)급성GVHD(P =0.025)、엄중감염(침습성진균병혹거세포병독)(P=0.05)현저강저SAA환자allo-HSCT후료효.결론 AlbHSCT시치료SAA환자적유효수단,우기MSD allo-HSCT료효현저,단재무HLA필배동포공자적SAA환자중야가선택AD allo-HSCT작위체대치료.채용BMT능유효예방이식후엄중(Ⅱ～Ⅳ도)급성GVHD급엄중감염(침습성진균병혹거세포병독),대우제고SAA환자allo-HSCT료효지관중요.
Objective To evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for severe aplastic anemia (SAA).Method The clinical data of 70 cases of SAA receiving allo-HSCT from January 2003 to February 2012 were retrospectively analyzed.There ere 40 males and 30 females with median age of 20 (5-41) years old.Fifty-one patients received HLA-matched sibling donor (MSD) allo-HSCT,and 19 patients received alternative donor(AD) alloHSCT.Of 51 patients receiving MSD allo-HSCT,20 patients were subjected to bone marrow transplantation (BMT),and 31 patients to peripheral blood stem cell transplantation (PBSCT).The median reinfusion quantity of CD34+ was 3.46 (2.00～4.80) × 106/kg in BMT,and 3.22 (1.27～ 5.98) × 106/kg in PBSCT.Of patients receiving AD allo-HSCT,10 patients were subjected to BMT,and 9 to PBSCT.The median reinfusion quantity of CD34+ was 3.10 (2.11 ～4.38) × 106/kg in BMT,and 4.90 (2.08～6.88) × 106/kg in PBSCT.Result Hematopoiesis reconstitution was achieved in all 70 patients,median follow up time was 35.5 (1 ～ 119) months,and prospective overall survival (OS) for 5 years was (79.0 ± 5.1) ％.Of the patients receiving MSD allo-HSCT,acute graftversus-host disease (aGVHD) occurred 16 cases,chronic GVHD (cGVHD) in 10 cases,and graft rejection (GR) in 6 cases.There were 7 deaths.OS for 5 years was (85.0 ± 5.4) ％.In the patients receiving AD allo-HSCT,aGVHD occurred in 12 cases,cGVHD in 7 cases,and GR in one case.There were 7 deaths.OS for 5 years was (63.2 ± 11.1)％.Multiplicity analysis revealed that PBSCT (P=0.049),severe (Ⅱ-Ⅳ) aGVHD (P =0.025) and severe infection (invasive fungal disease or cytomegalovirus) (P =0.05) could reduce the efficacy of allo-HSCT for SAA significantly.Conclusion Allo-HSCT is an effective therapy for patients with SAA,especially those receiving MSD allo-HSCT.AD allo-HSCT might be feasible for SAA patients without HLA-matched sibiling donors.Bone marrow transplantation which can effectively prevent and treat severe (Ⅱ-Ⅳ) aGVHD and severe infection (invasive fungal disease or cytomegalovirus) after transplantation may improve the efficacy of allo-HSCT for SAA.