Published in:
Open Access
01-06-2011
Allogeneic Haematopoietic Stem Cell Transplantation as Therapy for Chronic Granulomatous Disease—Single Centre Experience
Authors:
Jolanta Goździk, Anna Pituch-Noworolska, Szymon Skoczeń, Wojciech Czogała, Jarosław Baran, Aleksandra Krasowska-Kwiecień, Oktawiusz Wiecha, Marek Zembala
Published in:
Journal of Clinical Immunology
|
Issue 3/2011
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Abstract
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) × 108 nucleated cells, 3.8 (2.0–8.0) × 106 CD34+ cells and 45 (27–64) × 106 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.