异基因造血干细胞移植治疗经典型阵发性睡眠性血红蛋白尿症5例临床观察  

Clinical observation of allogeneic hematopoietic stem cell transplantation for treating five cases of classic paroxysmal nocturnal hemoglobinuria

作　　者：赵小利 张利宁 张潇予 郝梦泽 陈书连 魏嘉璘 何祎 韩明哲 姜尔烈 Zhao Xiaoli;Zhang Lining;Zhang Xiaoyu;Hao Mengze;Chen Shulian;Wei Jialin;He Yi;Han Mingzhe;Jiang Erlie(State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Haihe Laboratory of Cell Ecosystem,Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Tianjin 300020,China;Tianjin Institutes of Health Science,Tianjin 301600,China)

机构地区：[1]中国医学科学院血液病医院(中国医学科学院血液学研究所),实验血液学国家重点实验室,国家血液系统疾病临床医学研究中心,细胞生态海河实验室,天津300020 [2]天津医学健康研究院,天津301600

出　　处：《中华血液学杂志》2024年第12期1125-1128,共4页Chinese Journal of Hematology

摘　　要：2019年至2023年期间,5例经典型阵发性睡眠性血红蛋白尿症(cPNH)患者在中国医学科学院血液病医院接受异基因造血干细胞移植。5例患者均为男性,中位年龄26(26~46)岁,诊断到移植的中位间隔时间为5.5(3.6~18.0)年,移植前粒细胞中位PNH克隆为96.3%(90.0%~99.7)%,中位乳酸脱氢酶2224(1244~4753)U/L,骨髓增生活跃至极度活跃。单倍体移植4例,同胞全相合移植1例。清髓性预处理(MAC)3例,减低剂量预处理(RIC)2例。未发生原发性植入失败及移植后早期死亡。中位中性粒细胞植入时间为15(13~21)d,中位血小板植入时间为24(13~60)d。3例患者移植后发生Ⅱ度急性移植物抗宿主病(GVHD),2例患者发生局限型慢性GVHD。5例患者移植后PNH克隆均转阴,中位转阴时间为移植后2(1~3)个月。中位随访16(6~34)个月,1例患者死于疾病复发合并感染。存活的4例患者中,2例已停用所有药物,1例口服环孢素A治疗皮肤慢性GVHD,另外1例口服环孢素A常规预防GVHD。This study enrolled five patients with classic paroxysmal nocturnal hemoglobinuria(cPNH)who underwent allogeneic hematopoietic stem cell transplantation in our hospital from 2019 to 2023.All five patients were male,with a median age of 26(range:26-46)years.The median time from diagnosis to allo-HSCT was 5.5(range:3.6-18.0)years.The median PNH granulocyte clone size was 96.3%(ranged 90.0%-99.7%),and the median lactate dehydrogenase(LDH)level was 2224 IU/L(8896-fold of the upper limit of normal).All patients were detected to have bone marrow hyperplasia by trephine biopsy.The stem cell source came from four haploidentical donors and one HLA-identical sibling donor.Among the five patients,three underwent myeloablative conditioning(MAC)and two underwent reduced-intensity conditioning(RIC)treatment.None of the patients experienced primary implantation failure.The neutrophil implantation time was 15(range:13-21)days,and the platelet implantation time was 24(range:13-60)days.The three patients developed grade II acute graft-versus-host disease(aGVHD).No patients developed gradeⅢ/ⅣaGVHD.The two patients developed localized chronic GVHD(cGVHD),and no patients developed extensive cGVHD.PNH clones turned negative in all patients after 2(range:1-3)months of transplantation.At a median follow-up of 16(range:6-34)months,one patient died of relapse and infection,and the remaining four patients survived,of which two patients had discontinued all drugs.

关 键 词：骨髓增生活跃 转阴时间 中国医学科学院 异基因造血干细胞移植 局限型 环孢素A 乳酸脱氢酶 PNH 

分 类 号：R556.6[医药卫生—血液循环系统疾病]