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Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency.
- Source :
-
Blood [Blood] 2012 Oct 25; Vol. 120 (17), pp. 3615-24; quiz 3626. Date of Electronic Publication: 2012 Jul 12. - Publication Year :
- 2012
-
Abstract
- Deficiency of the purine salvage enzyme adenosine deaminase leads to SCID (ADA-SCID). Hematopoietic cell transplantation (HCT) can lead to a permanent cure of SCID; however, little data are available on outcome of HCT for ADA-SCID in particular. In this multicenter retrospective study, we analyzed outcome of HCT in 106 patients with ADA-SCID who received a total of 119 transplants. HCT from matched sibling and family donors (MSDs, MFDs) had significantly better overall survival (86% and 81%) in comparison with HCT from matched unrelated (66%; P < .05) and haploidentical donors (43%; P < .001). Superior overall survival was also seen in patients who received unconditioned transplants in comparison with myeloablative procedures (81% vs 54%; P < .003), although in unconditioned haploidentical donor HCT, nonengraftment was a major problem. Long-term immune recovery showed that regardless of transplant type, overall T-cell numbers were similar, although a faster rate of T-cell recovery was observed after MSD/MFD HCT. Humoral immunity and donor B-cell engraftment was achieved in nearly all evaluable surviving patients and was seen even after unconditioned HCT. These data detail for the first time the outcomes of HCT for ADA-SCID and show that, if patients survive HCT, long-term cellular and humoral immune recovery is achieved.
- Subjects :
- Adenosine Deaminase deficiency
Adenosine Deaminase immunology
Agammaglobulinemia immunology
Agammaglobulinemia mortality
Agammaglobulinemia pathology
Child
Child, Preschool
Female
Graft Survival
Histocompatibility Testing
Humans
Immunity, Cellular
Immunity, Humoral
Infant
Infant, Newborn
Kaplan-Meier Estimate
Lymphocyte Count
Male
Myeloablative Agonists therapeutic use
Retrospective Studies
Severe Combined Immunodeficiency immunology
Severe Combined Immunodeficiency mortality
Severe Combined Immunodeficiency pathology
Siblings
T-Lymphocytes immunology
Treatment Outcome
Unrelated Donors
Agammaglobulinemia drug therapy
Hematopoietic Stem Cell Transplantation
Severe Combined Immunodeficiency drug therapy
Transplantation Conditioning
Subjects
Details
- Language :
- English
- ISSN :
- 1528-0020
- Volume :
- 120
- Issue :
- 17
- Database :
- MEDLINE
- Journal :
- Blood
- Publication Type :
- Academic Journal
- Accession number :
- 22791287
- Full Text :
- https://doi.org/10.1182/blood-2011-12-396879