Excellent survival after sibling or unrelated donor stem cell transplantation for chronic granulomatous disease - 24/12/11
, Sweta Shah, MD c, William T. Shearer, MD, PhD c, Howard M. Rosenblatt, MD d, Mary E. Paul, MD c, Javier Chinen, MD, PhD c, Kathryn S. Leung, MD a, b, Alana Kennedy-Nasser, MD a, b, Malcolm K. Brenner, MD, PhD a, b, Helen E. Heslop, MD, PhD a, b, Hao Liu, PhD e, Meng-Fen Wu, MS e, Imelda C. Hanson, MD c, 
, Robert A. Krance, MD a, b, Corresponding author: Caridad A. Martinez, MD, Texas Children’s Cancer Center, Center for Cell and Gene Therapy, Baylor College of Medicine, 6621 Fannin St, MC3-3320, Houston, TX 77030.Abstract |
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.
Le texte complet de cet article est disponible en PDF.Key words : Chronic granulomatous disease, primary immunodeficiencies, bone marrow transplantation, graft-versus-host disease
Abbreviations used : CGD, ConA, GvHD, HSCT, MRD, MUD, SI
Plan
| Supported by grants from the National Institutes of Health Primary Immune Deficiency Treatment Consortium (AI082979). |
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| Disclosure of potential conflict of interest: M. K. Brenner receives research support from the National Institutes of Health/National Heart Lung and Blood Institute and the National Institutes of Health/National Cancer Institute. H. E. Heslop receives research support from the National Institutes of Health and the Leukemia and Lymphoma Society. The rest of the authors declare that they have no relevant conflicts of interest. |
Vol 129 - N° 1
P. 176-183 - janvier 2012 Retour au numéro
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