Fludarabine-Based Protocol for Human Umbilical Cord Blood Transplantation in Children With Fanconi Anemia

Publisher Website
Google Scholar
Abstract
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. 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 he-matopoietic stem cells. 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. 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.