Allogeneic Haematopoietic Stem Cell Transplantation as Therapy for Chronic Granulomatous Disease-Single Centre Experience

被引:15
作者
Gozdzik, Jolanta [1 ,2 ]
Pituch-Noworolska, Anna [2 ]
Skoczen, Szymon [1 ,2 ]
Czogala, Wojciech [1 ]
Baran, Jaroslaw [2 ]
Krasowska-Kwiecien, Aleksandra [1 ,2 ]
Wiecha, Oktawiusz [1 ]
Zembala, Marek [2 ]
机构
[1] Univ Childrens Hosp, Transplantat Ctr, PL-30633 Krakow, Poland
[2] Jagiellonian Univ, Coll Med, Polish Amer Inst Paediat, Dept Clin Immunol & Transplantat, Krakow, Poland
关键词
Immunological deficiency; chronic granulomatous disease; haematopoietic stem cell transplantation; children; CLINICAL-FEATURES; DONOR; ALLOGRAFT; REGISTRY; PATIENT; CHILD;
D O I
10.1007/s10875-011-9513-y
中图分类号
R392 [医学免疫学]; Q939.91 [免疫学];
学科分类号
100102 ;
摘要
Chronic granulomatous disease (CGD) is phagocytic cell metabolic disorder resulting in recurrent infections and granuloma formation. This paper reports the favourable outcome of allogeneic transplantation in six high-risk CGD patients. The following donors were used: HLA-matched, related (two) and unrelated (three), and HLA-mismatched, unrelated (one). One patient was transplanted twice using the same sibling donor because of graft rejection at 6 months after reduced-intensity conditioning transplant (fludarabine and melphalan). Myeloablative conditioning regimen consisted of busulphan and cyclophosphamide. Stem cell source was unmanipulated bone marrow containing: 5.2 (2.6-6.5) x 10(8) nucleated cells, 3.8 (2.0-8.0) x 10(6) CD34+ cells and 45 (27-64) x 10(6) CD3+ cells per kilogramme. Graft-versus-host disease prophylaxis consisted of cyclosporine A and, for unrelated donors, short course of methotrexate and anti-T-lymphocyte globulin. Mean neutrophile and platelet engraftments were observed at day 22 (20-23) and day 20 (16-29), respectively. Pre-existing infections and inflammatory granulomas resolved. With the follow-up of 4-35 months (mean, 20 months), all patients are alive and well with full donor chimerism and normalized superoxide production.
引用
收藏
页码:332 / 337
页数:6
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