脐血移植治疗小儿恶性和非恶性血液疾病的临床研究  

Clinical study on umbilical cord blood transplantation in children with malignant and nonmalignant hematopathy

作　　者：孙新[1] 刘莎[1] 赵喆[1] 郝文革[1] 廖灿[1] 郭乃榄[2] 

机构地区：[1]广东广州市妇婴医院儿科广州市脐血库,510180 [2]北京大学人民医院血液研究所

出　　处：《中国基层医药》2003年第6期484-487,共4页Chinese Journal of Primary Medicine and Pharmacy

基　　金：广东省科委重点科技攻关课题;广东省卫生厅"五个一工程"重大科研项目 (穗卫科学 1996- 2 3 );广州市科委重点攻关课题 ( 96- Z- 64 - 1和 99 -Z -0 0 8 -0 1) ;<脐血造血干细胞库的建立及其应用>课题临床应用部分

摘　　要：目的　探讨脐血移植治疗小儿恶性和非恶性血液病的可行性 ,重点了解造血重建的特点和移植相关并发症的发生情况。方法　用HLA全相合与不全相合的同胞和非血缘脐血移植治疗小儿急性白血病患儿 6例 ,重型 β 地中海贫血 8例 ,再生障碍性贫血患儿 1例。移植的预处理方案HLA全相合同胞与非血缘脐血患儿采用马利兰 +环磷酰胺 +抗胸腺细胞球蛋白方案 ;HLA 2个位点不全相合 β 地中海患儿采用高剂量输血 +去铁胺 +羟基脲 +氟达拉宾 +马利兰 +环磷酰胺 +抗胸腺细胞球蛋白方案。结果　 14例患儿白细胞达到植活标准 ,12例患儿血小板达到植活标准水平 ,其血小板 >5 0× 10 9/L的平均时间为 5 8 6 7d。 13例患儿发生急性移植物抗宿主病 ,其中Ⅰ度aGVHD 9例 ,Ⅱ度aGVHD 3例 ,Ⅳ度aGVHD 1例 ,2例慢性移植物抗宿主病。 9例患儿出现巨细胞病毒感染。 12例患儿长期存活 ;3例分别死于颅内出血、Ⅳ度aGVHD、Ⅲ度出血性膀胱炎。结论　脐血移植可有效治疗小儿恶性和非恶性血液疾病 ;如何有效防治GVHD、病毒与细菌感染等并发症至关重要。Objective To investigate the feasibility of umbilical cord blood transplantation for children with malignant and nonmalignant hematopathy and emphatically to understand the characteristics of hematopoietic reconstitution and the related complications.Methods HLA-identical and HLA mismatched,sib and non-sib umbilical cord blood had been used to reconstitute hematopoiesis in 15 patients with acute leukemia(n=6),β-thalassemia major(n=8),and severe aplastic anemia(n=1).The HLA-identical,sib and non-sib patients accepted the combined regimen consisting of busulfan,cyclophosphamide,antithymocyteglobulin,the HLA 2 locus-mismatch patients accepted the combined regimen consisting of hypertransfusions,desferrioxamine,hydroxyurea,fludarabine,busulfan,cyclophosphamide,antithymocyteglobulin.Results Fourteen patients had neutrophil recovery and 12 patients had platelet recovery(PLT>50×10 9/L),the median time was 58.67 days.Thirteen patients accomplished with acute graft-versus-host disease(aGVHD) including in grade Ⅰ 9 patients,grade Ⅱ in 3 patients,grade Ⅳ in 1 patient and 2 patients accomplished chronic graft-versus-host disease(cGVHD),9 patients infected with cytomegalovirus.Twelve patients had long-term survival,3 patients died of aGVHD grade Ⅳ,grade Ⅲ hemorrhage cystitis and intracranial hemorrhage respectively.Conclusion Umbilical cord blood transplantation is an effective way to treat children's malignant and nonmalignant hematopathy.It is very important to prevent and treat complications,for example,GVHD,bacterial and viral infection.

关 键 词：脐血移植 治疗 小儿 恶性血液疾病 非恶性血液疾病 并发症 

分 类 号：R457[医药卫生—治疗学]