[儿童脐带血造血祖细胞移植]。

Anales Espanoles De Pediatria Pub Date : 2000-12-01
Badell Serra I, Olivé Oliveras T, Madero López L, Muñoz Villa A, Martínez Rubio A, Verdeguer Miralles A, Díaz De Heredia Rubio C, Díaz Perez M, Cubells Rieró J, Maldonado Regalado M, Ortega Aramburu J
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引用次数: 0

摘要

目的:回顾性研究西班牙儿童脐带血移植(CBT)的预后。方法:28例患者(平均年龄6.5岁;1994年7月至1998年5月在西班牙儿童骨髓移植组的几个中心接受了CBT治疗。2例患者的供体是相同的人类白细胞抗原(HLA)同胞,2例供体是不匹配的家庭供体。在24例患者中,供体是不相关的,其中21例接受了hla不匹配的CBT。21例(75%)接受了以晚期白血病为主的CBT治疗。7例患者因遗传病接受移植。其中5人患有先天性免疫缺陷。调理治疗包括10例全身照射,其余患者联合化疗。在所有的移植物抗宿主病(GVHD)患者中,使用环孢素进行预防,在hla不匹配的供者中添加皮质类固醇或甲氨蝶呤。平均灌注有核细胞数为53.4 × 106/kg。结果:9例患者出现移植物衰竭。18例(64.3%)发展为iii级急性GVHD。8例(28.6%)发展为严重GVHD。3年时,所有患者的精算无事件生存率(EFS)为34.4±9%,平均随访时间为16.6个月。EFS在遗传性疾病患者(71>=6 17%)和HLA (A, B和DR)相同供者(6/6)(66>=6 19%)中更有利。结论:对遗传性疾病患者的治疗效果最好。我们观察到EFS与HLA相同供者之间呈负相关。严重急性GVHD的高发病率可能与某些患者HLA分型不准确有关。
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[Transplantation of umbilical cord blood hematopoietic progenitor cells in children].

Aim: Retrospective study of the outcome of cord blood transplantation (CBT) in children in Spain.

Methods: Twenty-eight patients (mean age 6.5 years; mean weight 25 kg) received a CBT between July 1994 and May 1998 in several centres of the Spanish Pediatric Bone Marrow Transplant Group. In 2 patients the donor was an identical human leukocyte antigen (HLA)-sibling and in two the donor was a mismatched family donor. In 24 patients the donor was unrelated, and 21 of these received an HLA-mismatched CBT. Twenty-one patients (75 %) received a CBT for leukemia mainly in advanced phase. Seven patients were transplanted for genetic disease. Of these, five had congenital immunodeficiency. The conditioning treatment included total body irradiation in ten patients and combined chemotherapy in the remaining patients. In all patients graft-versus-host disease (GVHD) prophylaxis was performed with cyclosporine, and corticosteroids or methotrexate were added in patients with HLA-mismatched donors. The mean number of nucleated cells infused was 53.4 x 106/kg.

Results: Graft failure was observed in nine patients. Eighteen patients (64.3%) developed grade IIIV acute GVHD. Eight patients (28.6%) developed severe GVHD. Actuarial event free survival (EFS) of all the patients was 34.4 +/- 9% at 3 years, with a mean followup of 16.6 months. EFS was more favorable in patients with genetic disease (71>=6 17%) and in those with an HLA (A, B and DR) identical donor (6/6) (66>=6 19%).

Conclusions: The most favorable results were obtained in patients with genetic diseases. We observed an inverse correlation between EFS and patients with HLA identical donors. The high incidence of severe acute GVHD could have been related to a lack of accuracy in the HLA typography of some patients.

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