title: Fludarabine-based stem cell transplantation protocol for Fanconi's anaemia in myelodysplastic transformation.
abstract: Allogeneic stem cell transplantation (SCT) represents the treatment of choice for severe bone marrow (BM) failure in patients with Fanconi's anaemia (FA). However, for FA patients developing leukaemic or myelodysplastic transformation, the results of SCT are much less encouraging. We present a 17-year-old girl with myelodysplastic transformation of FA (refractory anaemia with excess blasts) and oculocutaneous albinism, who was treated by sibling SCT using conditioning with fludarabine, cyclophosphamide (CY) and anti-lymphocyte globulin (ALG). She had rapid engraftment with no toxicity and no graft-versus-host disease (GVHD). Twenty-two months after SCT, she had 100% donor chimaerism on Southern blot analysis.
authors.affiliation: Department of Haematology, Hammersmith Hospital, London, UK. m.mccloy@ic.ac.uk
authors.forename: M
authors.initials: M
authors.lastname: McCloy
authors.forename: A
authors.initials: A
authors.lastname: Almeida
authors.forename: P
authors.initials: P
authors.lastname: Daly
authors.forename: T
authors.initials: T
authors.lastname: Vulliamy
authors.forename: I A
authors.initials: IA
authors.lastname: Roberts
authors.forename: I
authors.initials: I
authors.lastname: Dokal
mesh_terms: D000293:Adolescent; D000328:Adult; D000754:Anemia, Refractory, with Excess of Blasts / Q000601:surgery*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D003520:Cyclophosphamide / Q000008:administration & dosage; D016572:Cyclosporine / Q000627:therapeutic use; D005199:Fanconi Anemia / Q000601:surgery*; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007136:Immunoglobulins / Q000627:therapeutic use; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives*
chemical_list: D007136:Immunoglobulins; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine