摘要
目的 评价非亲缘异基因骨髓移植 (URD BMT)治疗儿童急性和慢性白血病的临床疗效。方法 6例白血病患儿 ,其中慢性髓系白血病 2例 ,急性淋巴细胞白血病 3例 (第 1次完全缓解 ) ,急性早幼粒细胞白血病 1例 (第 2次完全缓解 ) ,由台湾慈济骨髓捐赠中心提供无关供者骨髓。预处理方案为马利兰 +环磷酰胺 (Bu/Cy2 )方案 ,急性移植物抗宿主病 (aGVHD)预防为霉酚酸酯(MMF)、环孢菌素A(CsA)加氨甲喋呤 (MTX)联合方案 ;以前列素E1预防肝静脉闭塞病 (VOD) ,以巨细胞病毒 (CMV)抗原血症监测和更昔洛韦预防CMV病。供、受者间HLA基因位点型全相合 3例 ,1个基因位点型不合 2例 ,2个基因位点型不合 1例。结果 6例患儿经DNA短串联重复序列多态性分析证明为供髓植入 ,中性粒细胞 >0 5× 10 9/L的中位天数为 14 5 (13～ 18)d ,血小板 >2 0×10 9/L的中位天数为 16 (11～ 2 3)d。发生Ⅱ～Ⅳ度aGVHD 2例 (33% ) ,局限性慢性移植物抗宿主病(cGVHD) 3例 ,未发生广泛性cGVHD。中位随访时间 4 12 (187～ 1338)d ,全部患儿均无病生存。结论非亲缘异基因骨髓移植是治疗儿童急性和慢性白血病的有效方法。
Objective Allogeneic bone m ar row transplantation has been established as a standard method for the treatment of a range of malignant and non-malignant hematologic diseases in children. Un fortunately, fewer than 30% of patients have a human leukocyte antigen (HLA)-ma tched sibling. Advances in our understanding of the HLA system and the developm ent of large international donor registries encourage the increasing use of unre lated donors as an alternative source of stem cells. The purpose of this study was to evaluate the clinical efficacy and safety of unrelated donor allogeneic b one marrow transplantation (URD-BMT) for the treatment of childhood leukemia. Methods Six patients with leukemia received URD-BMT. Tw o of them suffered from chronic myeloid leukemia (CML), 3 suffered from acute ly mphocytic leukemia (ALL) and 1 suffered from acute promyelocytic leukemia (APL) (CR2). All cases were facilitated by Tzu Chi Marrow Donor Registry (TCTMDR). T he high resolution DNA test for classⅠand Ⅱ was carried out in HLA typing of a ll donor-receiver pairs. HLA allele matched in three cases, mismatched with on e locus in two cases and with two loci in one case. All patients were prepared with cyclophosphamide (CY) 60 mg/kg/day for 2 days (total dose 120 mg/kg) and bu sulfan (Bu) 1 mg/kg×4/day for 4 days (total dose 16 mg/kg). Mycophenolate mofe til (MMF), CsA and MTX were given to prevent acute graft-versus-host-disease (aGVHD). CsA of 3 mg/kg/d was continuously given by i.v. infusion, and then 6 mg/kg/d by oral. The blood CsA concentration was 200-300 ng/ml. MTX was given at the dosage of 15 mg/m2 on d 1 and 10 mg/m2 on d 3, 6,9 or 11. MMF was given at the dosage of 0.25-0.5 g/d from day 0 to day 120. Prostaglandin E1 was given to prevent the hepatic veno-occlusive disease (VOD), Ganciclovir was used to prevent CMV infection until the CMV antigenemia became negative. Results Analysis of DNA short tandem repeats showed total en graftment of donor marrow after transplantation in all cases. The median
出处
《中华儿科杂志》
CAS
CSCD
北大核心
2004年第11期835-839,共5页
Chinese Journal of Pediatrics
基金
浙江省科技九五攻关重大项目 (110 3 0 5 8)
浙江省医药卫生重点项目 (2 0 0 0ZD0 0 1)
