title: Intermediate-dose busulfan and cyclophosphamide as a conditioning regimen for bone marrow transplantation in a case of Fanconi anemia in myelodysplastic transformation.
abstract: We report an 11-year old female with myelodysplastic (refractory anemia with excess of blasts) presentation of Fanconi anemia. After failure of initial chemotherapy with low doses of 6-mercaptopurine and prednisolone she underwent allogeneic bone marrow transplantation (BMT) from her HLA-matched sibling. Busulfan 8 mg/kg and cyclophosphamide 40 mg/kg were used as conditioning. The post-transplant course was uneventful with fast trilineage engraftment and mild cutaneous acute GVHD. She is alive 17 months after BMT with full hematological reconstitution without evidence of MDS.
authors.affiliation: Bone Marrow Transplantation Department, Research Institute for Pediatric Hematology of Russian Federation, Moscow, Russia.
authors.forename: A A
authors.initials: AA
authors.lastname: Maschan
authors.forename: O I
authors.initials: OI
authors.lastname: Kryzanovskii
authors.forename: M I
authors.initials: MI
authors.lastname: Yourlova
authors.forename: E V
authors.initials: EV
authors.lastname: Skorobogatova
authors.forename: E D
authors.initials: ED
authors.lastname: Pashanov
authors.forename: Y E
authors.initials: YE
authors.lastname: Potapova
authors.forename: L A
authors.initials: LA
authors.lastname: Timonova
authors.forename: N Y
authors.initials: NY
authors.lastname: Bogatcheva
authors.forename: E V
authors.initials: EV
authors.lastname: Samochatova
authors.forename: A G
authors.initials: AG
authors.lastname: Roumjantzev
mesh_terms: D000477:Alkylating Agents / Q000008:administration & dosage*; D000754:Anemia, Refractory, with Excess of Blasts / Q000188:drug therapy / Q000473:pathology / Q000628:therapy*; D016026:Bone Marrow Transplantation; D002066:Busulfan / Q000008:administration & dosage*; D002648:Child; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage*; D004359:Drug Therapy, Combination; D005199:Fanconi Anemia / Q000188:drug therapy / Q000473:pathology / Q000628:therapy*; D005260:Female; D006801:Humans; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous
chemical_list: D000477:Alkylating Agents; D003520:Cyclophosphamide; D002066:Busulfan