title: Fludarabine-based protocol for human umbilical cord blood transplantation in children with Fanconi anemia.
abstract: OBJECTIVE
  A novel conditioning regimen of fludarabine monophosphate (FLM), anti-T-lymphocyte globulin (ATG), and low-dose cyclophosphamide with no irradiation for human umbilical cord blood transplantation (HUCBT) for the treatment of Fanconi anemia (FA) is described.
  
  
  METHODS
  A 12-year-old girl with FA received a human umbilical cord blood transplant from a fully matched sibling donor. After the HUCBT, the patient was given granulocyte colony stimulating factor in combination with erythropoietin. Pretransplant conditioning consisted of FLM (30 mg/m2/d) from day -10 to day -5, cyclophosphamide (10 mg/kg/d) on day -7 and -6, and rabbit ATG (ATG-Frasenius, 10 mg/kg/d) from day -4 to day -1. Cyclosporin A (3 mg/kg/d) was administered from day -1 as graft-versus-host disease prophylaxis. Cord blood from a sibling donor was used as a source of hematopoietic stem cells.
  
  
  RESULTS
  Engraftment was normal and sustained. The regimen was well tolerated with very mild toxicity and no major transplant-related complications or >grade II graft-versus-host disease. Chimerism was 100% donor origin as determined by restriction fragment length polymorphism.
  
  
  CONCLUSIONS
  It is possible to achieve sustained engraftment and only mild toxicity in FA after HUCBT with a conditioning regimen of FLM, ATG, and cyclophosphamide with no irradiation. These preliminary results with this novel conditioning protocol are encouraging and should be evaluated in a larger group of patients with FA undergoing HUCBT.
authors.affiliation: Department of Pediatrics, Hadassah University Hospital, Jerusalem, Israel.
authors.forename: M
authors.initials: M
authors.lastname: Aker
authors.forename: G
authors.initials: G
authors.lastname: Varadi
authors.forename: S
authors.initials: S
authors.lastname: Slavin
authors.forename: A
authors.initials: A
authors.lastname: Nagler
mesh_terms: D002648:Child; D003520:Cyclophosphamide / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D005199:Fanconi Anemia / Q000628:therapy*; D005260:Female; D005312:Fetal Blood; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D019172:Transplantation Conditioning; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000627:therapeutic use
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine