Abstract |
Allogeneic stem cell transplantation is the only curative treatment for Wiskott-Aldrich syndrome. The authors retrospectively analyzed the outcome with this procedure in 13 patients with severe Wiskott-Aldrich syndrome transplanted in 5 Spanish centers from 1989 to 2006. A patient was transplanted twice from the same donor due to a late engraftment failure. Age at transplant ranged from 7 to 192 months (median 30 months). There were 10 matched donors (3 related and 7 unrelated), 2 mismatched unrelated, and 1 haploidentical. Conditioning regimen consisted of busulfan and cyclophosphamide (BuCy) in 11 cases and fludarabine and melfalan (1) or BuCy (1). ATG was added in transplants from non-genetically matched donors. GvHD prophylaxis consisted of cyclosporine and methotrexate in most patients plus T-cell depletion in the haploidentical HSCT. Nine of the 13 transplanted patients are alive with complete clinical, immunologic, and hematologic recovery 8-204 months (median 101 months) after HSCT. Eight surviving patients had been transplanted from matched donors (3 related and 5 unrelated) and 1 from a haploidentical donor. Four patients died, 2 transplanted from matched donors (1 from acute GvHD and organ failure, 1 from a lymphoproliferative disorder after a second transplant), and 2 transplanted from mismatched unrelated donors (1 from acute GvHD and organ failure, 1 from graft failure and infection). Allogeneic hemopoietic stem cell transplantation must be utilized in all patients with severe Wisckott- Aldrich syndrome, using the most suitable graft variant for each patient.
|
Authors | A Muñoz, T Olivé, A Martinez, E Bureo, M S Maldonado, C Diaz de Heredia, A Sastre, M Gonzalez-Vicent, Spanish Working Party for Blood and Marrow Transplantation in Children (GETMON) |
Journal | Pediatric hematology and oncology
(Pediatr Hematol Oncol)
Vol. 24
Issue 6
Pg. 393-402
(Sep 2007)
ISSN: 1521-0669 [Electronic] England |
PMID | 17710656
(Publication Type: Evaluation Study, Journal Article, Multicenter Study)
|
Chemical References |
- Antilymphocyte Serum
- HLA Antigens
- Immunosuppressive Agents
- Cyclosporine
- Cyclophosphamide
- Vidarabine
- Busulfan
- fludarabine
- Melphalan
|
Topics |
- Antilymphocyte Serum
(therapeutic use)
- Busulfan
(therapeutic use)
- Child
- Child, Preschool
- Cyclophosphamide
(therapeutic use)
- Cyclosporine
(therapeutic use)
- Graft vs Host Disease
(mortality, prevention & control)
- HLA Antigens
(genetics, immunology)
- Haplotypes
- Hematopoietic Stem Cell Transplantation
(mortality, statistics & numerical data)
- Histocompatibility
- Humans
- Immunosuppressive Agents
(therapeutic use)
- Infant
- Living Donors
- Lymphocyte Depletion
- Male
- Melphalan
(therapeutic use)
- Multiple Organ Failure
(mortality)
- Postoperative Complications
(mortality)
- Reoperation
- Retrospective Studies
- Spain
(epidemiology)
- T-Lymphocytes
- Transplantation Conditioning
- Transplantation, Homologous
(mortality, statistics & numerical data)
- Treatment Outcome
- Vidarabine
(analogs & derivatives, therapeutic use)
- Wiskott-Aldrich Syndrome
(epidemiology, surgery)
|