Journal
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
Volume 129, Issue 1, Pages 176-183Publisher
MOSBY-ELSEVIER
DOI: 10.1016/j.jaci.2011.10.005
Keywords
Chronic granulomatous disease; primary immunodeficiencies; bone marrow transplantation; graft-versus-host disease
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Funding
- National Institutes of Health Primary Immune Deficiency Treatment Consortium [AI082979]
- National Institutes of Health/National Heart Lung and Blood Institute
- National Institutes of Health/National Cancer Institute
- National Institutes of Health
- Leukemia and Lymphoma Society
- Allergy and Immunology Section
- Hematology and Oncology Section, Department of Pediatrics, Baylor College of Medicine
- Stem Cell Transplant Unit at Texas Children's Hospital
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Background: Matched related donor (MRD) hematopoietic stem cell transplantation (HSCT) is a successful treatment for chronic granulomatous disease (CGD), but the safety and efficacy of HSCT from unrelated donors is less certain. Objective: We evaluated the outcomes and overall survival in patients with CGD after HSCT. Methods: We report the outcomes for 11 children undergoing HSCT from an MRD (n = 4) or an HLA-matched unrelated donor (MUD) (n = 7); 9 children were boys, and the median age was 3.8 years (range, 1-13 years). We treated both X-linked (n = 9) and autosomal recessive (n = 2) disease. Nine children had serious clinical infections before transplantation. The conditioning regimens contained busulfan, cyclophosphamide, cytarabine, or fludarabine according to the donor used. All patients received alemtuzumab (anti-CD52 antibody). Additional graft-versus-host disease (GvHD) prophylaxis included cyclosporine and methotrexate for MUD recipients and cyclosporine and prednisone for MRD recipients. Results: Neutrophil recovery took a median of 16 days (range, 12-40 days) and 18 days (range, 13-24 days) for MRD and MUD recipients, respectively. Full donor neutrophil engraftment occurred in 9 patients, and 2 had stable mixed chimerism; all patients had sustained correction of neutrophil oxidative burst defect. Four patients had grade I skin acute GVHD responding to topical treatment. No patient had grade II to IV acute GvHD or chronic GvHD. All patients are alive between 1 and 8 years after HSCT. Conclusion: For CGD, equivalent outcomes can be obtained with MRD or MUD stem cells, and HSCT should be considered an early treatment option. (J Allergy Clin Immunol 2012; 129: 176-83.)
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