title: Non-myeloablative bone marrow transplantation in an adult with Wiskott-Aldrich syndrome.
abstract: Early bone marrow transplant is now standard treatment for infants with severe immunodeficiencies such as Wiskott-Aldrich Syndrome (WAS), but results in older children and adults are poor. Non-myeloablative transplant has shown promise in the treatment of older children, who are likely to have active infections and organ damage. We describe a non-myeloablative transplant of a 26-year-old man with WAS, undertaken because of severe infections and vasculitis. Partial engraftment and immunorestoration were achieved. The patient is well 1 year post transplantation.
authors.affiliation: Department of Immunopathology, St Bartholomew's Hospital, London, UK. hjlonghurst@mds.qmw.ac.uk
authors.forename: Hilary J
authors.initials: HJ
authors.lastname: Longhurst
authors.forename: David
authors.initials: D
authors.lastname: Taussig
authors.forename: Tanzina
authors.initials: T
authors.lastname: Haque
authors.forename: Denise
authors.initials: D
authors.lastname: Syndercombe-Court
authors.forename: Jamie
authors.initials: J
authors.lastname: Cavenagh
authors.forename: J David
authors.initials: JD
authors.lastname: Edgar
authors.forename: Matthew R
authors.initials: MR
authors.lastname: Helbert
mesh_terms: D000328:Adult; D000074323:Alemtuzumab; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D061067:Antibodies, Monoclonal, Humanized; D000912:Antibodies, Neoplasm / Q000627:therapeutic use; D016026:Bone Marrow Transplantation / Q000379:methods*; D003520:Cyclophosphamide / Q000627:therapeutic use; D006801:Humans; D016756:Immunoglobulins, Intravenous; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D010404:Penicillin V / Q000008:administration & dosage; D018183:Transplantation Chimera; D015662:Trimethoprim, Sulfamethoxazole Drug Combination / Q000008:administration & dosage; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use; D014923:Wiskott-Aldrich Syndrome / Q000628:therapy*
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000912:Antibodies, Neoplasm; D016756:Immunoglobulins, Intravenous; D007166:Immunosuppressive Agents; D000074323:Alemtuzumab; D015662:Trimethoprim, Sulfamethoxazole Drug Combination; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine; D010404:Penicillin V