title: Allogeneic hemopoietic stem cell transplantation (HSCT) for Wiskott-Aldrich syndrome: a report of the Spanish Working Party for Blood and Marrow Transplantation in Children (GETMON).
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: Department of Pediatrics, Hospital Ramon y Cajal-University of Alcalá, Madrid, Spain. amvillatv@yahoo.es
authors: A
authors: A
authors: Muñoz
authors: T
authors: T
authors: Olivé
authors: A
authors: A
authors: Martinez
authors: E
authors: E
authors: Bureo
authors: M S
authors: MS
authors: Maldonado
authors: C
authors: C
authors: Diaz de Heredia
authors: A
authors: A
authors: Sastre
authors: M
authors: M
authors: Gonzalez-Vicent
mesh_terms: D000961:Antilymphocyte Serum / Q000627:therapeutic use; D002066:Busulfan / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use; D016572:Cyclosporine / Q000627:therapeutic use; D006086:Graft vs Host Disease / Q000401:mortality / Q000517:prevention & control; D006680:HLA Antigens / Q000235:genetics / Q000276:immunology; D006239:Haplotypes; D018380:Hematopoietic Stem Cell Transplantation / Q000401:mortality / Q000706:statistics & numerical data*; D006648:Histocompatibility; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D019520:Living Donors; D008212:Lymphocyte Depletion; D008297:Male; D008558:Melphalan / Q000627:therapeutic use; D009102:Multiple Organ Failure / Q000401:mortality; D011183:Postoperative Complications / Q000401:mortality; D012086:Reoperation; D012189:Retrospective Studies; D013030:Spain / Q000453:epidemiology; D013601:T-Lymphocytes; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous / Q000401:mortality / Q000706:statistics & numerical data; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use; D014923:Wiskott-Aldrich Syndrome / Q000453:epidemiology / Q000601:surgery*
chemical_list: D000961:Antilymphocyte Serum; D006680:HLA Antigens; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D003520:Cyclophosphamide; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine; D008558:Melphalan