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: 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)