title: Fludarabine, low-dose busulfan and antithymocyte globulin as conditioning for Fanconi anemia patients receiving bone marrow transplantation from HLA-compatible related donors.
abstract: Allogeneic hematopoietic stem cell transplantation (SCT) from unaffected donors remains the only modality for the correction of hematological abnormalities in Fanconi anemia (FA) patients. We performed four HLA-matched related donor SCT using a novel irradiation and cyclophosphamide-free conditioning regimen. The protocol included fludarabine 150 mg/m(2), busulfan 4 mg/kg, and antithymocyte globulin 90 mg/kg. Graft-versus-host disease (GVHD) prophylaxis was cyclosporin A, MTX, and daclizumab. The engraftment and occurrence of full stable donor hemopoiesis was rapid in all cases with minimal short-term toxic complications. There were no infections or febrile episodes during the inpatient phase. Three patients developed acute GVHD grade I-II involving gut and skin and one patient progressed to extensive chronic GVHD. The preparative conditioning regimen is safe and associated with low organ toxicity and effective immunosupression for the stable engraftment in FA patients undergoing SCT with matched related donors.
authors.affiliation: Bone Marrow Transplantation Unit, Russian Institute for Pediatric Hematology, Russian Children's Hospital, Moscow, Russia.
authors.forename: A A
authors.initials: AA
authors.lastname: Maschan
authors.forename: P E
authors.initials: PE
authors.lastname: Trakhtman
authors.forename: D N
authors.initials: DN
authors.lastname: Balashov
authors.forename: I P
authors.initials: IP
authors.lastname: Shipicina
authors.forename: E V
authors.initials: EV
authors.lastname: Skorobogatova
authors.forename: Y V
authors.initials: YV
authors.lastname: Skvortsova
authors.forename: Z M
authors.initials: ZM
authors.lastname: Dyshlevaja
authors.forename: E V
authors.initials: EV
authors.lastname: Samochatova
authors.forename: A G
authors.initials: AG
authors.lastname: Rumiantsev
mesh_terms: D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation / Q000276:immunology; D002066:Busulfan / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003586:Cytomegalovirus Infections / Q000175:diagnosis; D004359:Drug Therapy, Combination; D005199:Fanconi Anemia / Q000276:immunology / Q000601:surgery*; D005260:Female; D006650:Histocompatibility Testing; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D019520:Living Donors; D008297:Male; D011183:Postoperative Complications / Q000821:virology; D033581:Stem Cell Transplantation; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000627:therapeutic use*
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine