山东医药2016，Vol.56Issue(21)：4-6,3.DOI:10.3969/j.issn.1002-266X.2016.21.002
单倍体造血干细胞移植治疗阵发性睡眠性血红蛋白尿症1例临床观察
Haplo-hematopoietic stem cell transplantation in treatment of paroxysmal nocturnal hemoglobinuria:A case report
摘要
Abstract
Objective To observe the effect of human leukocyte antigen (HLA)haplo-hematopoietic stem cell trans-plantation in the treatment of patient with paroxysmal nocturnal hemoglobinuria (PNH).Methods A 43-year-old male pa-tient with PNH received haploidentical hematopoietic stem cell transplantation.The conditioning regimen was Fludarabine /cyclophosphamide (Flu/Cy)+anti-human T lymphocyte rabbit immunoglobulin (ATG-fresen).Prophylaxis of GVHD was performed with cyclosporine (CsA),mycophenolate mofetil (MMF)and short-term methotrexate (MTX).Alprostadil and Fufang Danshen injection was use to prevent hepatic veno-occlusive disease.The patient received 6.51 ×108 mononucleat-ed cells/kg and 4.62 ×106 CD34 +cells/kg.The blood routine and liver function after transplantation were detected,and the hemolysis was observe by using sucrose hemolysis test and Ham 's test.Using flow cytometry to monitor CD55 and CD59 on the surface of peripheral blood cells as well as FLAER loss rate.The bone marrow liquid was extracted to do bone mar-row cytology and chromosome examination.Results On day 15 and 19 after transplantation,the neutrophils reached 0.5 × 109 /L and the platelets reached 20 ×109 /L.Ham test and sucrose hemolysis test were negative,and CD55,CD59, FLAER were normal on day 35 after transplantation.The patient had no acute GVHD.Limited chronic skin GVHD devel-oped,which improved after treatment.At the last follow-up of 406-days,the patient showed normal hemogram and myelo-gram,which indicated persistent hematopoietic reconstitution.Conclusion Haploidentical hematopoietic stem cell trans-plantation is successful and safe in treatment of PNH.关键词
阵发性睡眠性血红蛋白尿症/单倍体造血干细胞移植/非清髓预处理Key words
paroxysmal nocturnal hemoglobinuria/haplo-hematopoietic stem cell transplantation/non-myeloablative conditioning分类
医药卫生引用本文复制引用
张银银,姜中兴,郭荣,谢新生,万鼎铭,孙玲,孙慧,何飞..单倍体造血干细胞移植治疗阵发性睡眠性血红蛋白尿症1例临床观察[J].山东医药,2016,56(21):4-6,3.基金项目
国家自然科学基金资助项目（81070445）；河南省医学科技攻关计划（201403029）。 （）
