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)

• Published in: Pediatric hematology and oncology 2007-01, Vol.24 (6), p.393-402
• Main Authors: Muñoz, A., Olivé, T., Martinez, A., Bureo, E., Maldonado, M. S., de Heredia, C. Diaz, Sastre, A., Gonzalez-Vicent, M.
• Format: Article
• Language: English
• Subjects: Antilymphocyte Serum - therapeutic use; Busulfan - therapeutic use; Child; Child, Preschool; Cyclophosphamide - therapeutic use; Cyclosporine - therapeutic use; Graft vs Host Disease - mortality; Graft vs Host Disease - prevention & control; haploidentical donors; Haplotypes; Hematopoietic Stem Cell Transplantation - mortality; Hematopoietic Stem Cell Transplantation - statistics & numerical data; hemopoietic stem cell transplantation; Histocompatibility; HLA Antigens - genetics; HLA Antigens - immunology; Humans; Immunosuppressive Agents - therapeutic use; Infant; Living Donors; Lymphocyte Depletion; Male; matched donors; Melphalan - therapeutic use; mismatched donors; Multiple Organ Failure - mortality; Postoperative Complications - mortality; Reoperation; Retrospective Studies; Spain - epidemiology; T-Lymphocytes; Transplantation Conditioning; Transplantation, Homologous - mortality; Transplantation, Homologous - statistics & numerical data; Treatment Outcome; Vidarabine - analogs & derivatives; Vidarabine - therapeutic use; Wiskott-Aldrich syndrome; Wiskott-Aldrich Syndrome - epidemiology; Wiskott-Aldrich Syndrome - surgery
• ISSN: 0888-0018; 1521-0669
• DOI: 10.1080/08880010701454404

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.