CAJ | 학술논문

目的探讨异基因造血干细胞移植治疗噬血细胞综合征的预处理方案、疗效及移植相关副作用.方法 2010年2月至2012年9月于北京市道培医院1 1例接受异基因造血干细胞移植术的噬血细胞综合征患儿.其中8例为原发性噬血细胞综合征(PRF1基因突变2例,UNC13D基因突变3例,STXBP2基因突变2例,XIAP基因突变1例),3例没有查到免疫缺陷相关基因,诊断EB病毒相关的噬血细胞综合征.移植前达到缓解期的患儿仅1例.选用非血缘异基因造血干细胞移植供者8例,HLA配型7/10 ～10/10相合,半相合异基因造血干细胞移植3例.采用以依托泊苷/马利兰/氟达拉滨/抗淋巴细胞球蛋白(VP-16/Bu/Flu/ATG)为主的预处理方案7例,采用以依托泊苷/氟达拉滨/马法兰/抗淋巴细胞球蛋白(VP-16/Flu/Mel/ATG)为主预处理方案4例.11例患儿预防移植物抗宿主病(GVHD)采用环孢菌素或普乐可复、骁悉、短疗程甲氨蝶呤,其中4例加用抗CD25单抗,7例辅助输注非血缘脐带血,1例辅助输注半相合骨髓预防GVHD.结果 11例患儿中有3例死亡.可评估的8例患儿总生存中位时间585 d(154～1 115 d).11例患儿均骨髓顺利植入.供受者嵌合率均为100％供者型,其中2例在首次骨髓评估供受者嵌合率时分别为80.14％、97.76％.急性移植物抗宿主病(aGVHD)8例,其中aGVHD Ⅰ～Ⅱ° 3例,aGVHDⅢ～Ⅳ° 5例.慢性移植物抗宿主病(cGVHD)4例.巨细胞病毒血症7例,其中发展为巨细胞病毒病3例.结论异基因造血干细胞移植是治疗原发性及难治、复发性噬血细胞综合征的有效手段.缓解期患儿的移植疗效明显高于非缓解期患儿,重视移植后病毒的激活,合理的预处理方案可以减少移植风险.
목적 탐토이기인조혈간세포이식치료서혈세포종합정적예처리방안、료효급이식상관부작용.방법 2010년2월지2012년9월우북경시도배의원1 1례접수이기인조혈간세포이식술적서혈세포종합정환인.기중8례위원발성서혈세포종합정(PRF1기인돌변2례,UNC13D기인돌변3례,STXBP2기인돌변2례,XIAP기인돌변1례),3례몰유사도면역결함상관기인,진단EB병독상관적서혈세포종합정.이식전체도완해기적환인부1례.선용비혈연이기인조혈간세포이식공자8례,HLA배형7/10 ～10/10상합,반상합이기인조혈간세포이식3례.채용이의탁박감/마리란/불체랍빈/항림파세포구단백(VP-16/Bu/Flu/ATG)위주적예처리방안7례,채용이의탁박감/불체랍빈/마법란/항림파세포구단백(VP-16/Flu/Mel/ATG)위주예처리방안4례.11례환인예방이식물항숙주병(GVHD)채용배포균소혹보악가복、효실、단료정갑안접령,기중4례가용항CD25단항,7례보조수주비혈연제대혈,1례보조수주반상합골수예방GVHD.결과 11례환인중유3례사망.가평고적8례환인총생존중위시간585 d(154～1 115 d).11례환인균골수순리식입.공수자감합솔균위100％공자형,기중2례재수차골수평고공수자감합솔시분별위80.14％、97.76％.급성이식물항숙주병(aGVHD)8례,기중aGVHD Ⅰ～Ⅱ° 3례,aGVHDⅢ～Ⅳ° 5례.만성이식물항숙주병(cGVHD)4례.거세포병독혈증7례,기중발전위거세포병독병3례.결론 이기인조혈간세포이식시치료원발성급난치、복발성서혈세포종합정적유효수단.완해기환인적이식료효명현고우비완해기환인,중시이식후병독적격활,합리적예처리방안가이감소이식풍험.
Objective To observe the conditioning regimen,efficacy and side effects of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for hemophagocytic lymphohistiocytosis (HLH).Method From 2010 to 2012,a total of 11 cases after allo-HSCT were evaluated including 8 cases with familial hemophagocytic lymphohistiocytosis (FHL) and 3 cases with Epstein-Barr virus (EBV) related HLH.Allo-HSCT from HLA haploidentical HSCT was performed for 3 cases and unrelated allo-HSCT for 8 cases; 7 cases underwent allo-HSCT with conditioning regimen of etoposide (VP16),busulphan (Bu),fludarabine(Flu) and antilymphocyte globulin (ATG) and 4 cases with Flu,melphalan (Mel) and ATG.Cyclosporine(CsA) or tacrolimus,mycophenolate(MMF) and methorexate (MTX) were used for prevention of graft versus host disease (GVHD).Four cases received anti-CD25 MoAbs,7 cases received cord blood and 1 of them received haploidentical bone marrow to prevent GVHD.Result Three cases died after alloHSCT.The median overall survival time of the 8 cases evaluated was 585 days (154-1 115 d).All the patients were successfully engrafted.Acute GVHD (aGVHD) occurred in 8 cases,including 3 cases of grade Ⅰ/Ⅱ and 5 cases of grade Ⅲ/Ⅳ.Chronic GVHD (cGVHD) occurred in 4 cases.Seven cases had cytomegalovirus (CMV) reactivation.Conclusion The allo-HSCT was successful in treating primary and refractory hemophagocytic syndrome.