title: Better posttransplant outcome with fludarabine based conditioning in multitransfused fanconi anemia patients who underwent peripheral blood stem cell transplantation.
abstract: Several investigators have been looking for less toxic conditioning regimen for stem cell transplantation in Fanconi anemia (FA) patients because of sensitivity to DNA cross-linking agents and tendency to malignancy. We report 16 multitransfused FA patients who underwent peripheral stem cell transplantation from 13 related and 3 unrelated donors. Although the first 6 patients received thoraco-abdominal irradiation + cyclophosphamide + antithymocyte globulin (regimen A) for conditioning, fludarabine (FLU) + cyclophosphamide + antithymocyte globulin (regimen B) were used in the last 10 patients in which 3 of them received unrelated graft. Cyclosporin A was given alone for the related allografts but also included mycophenolate mofetil for the unrelated allograft as graft versus host disease prophylaxis. We observed a lower risk of peritransplant morbidity and mortality with fewer and milder graft versus host disease in FLU based group. We lost 3 patients in regimen A group and 1 of them from secondary acute myeloid leukemia. Three patients are alive with transfusion independent. In regimen B group, 9 of 10 patients are alive with normal hematologic parameters and full donor chimerism. The longest follow-up durations are 90 and 60 months in regimen A and B, respectively. In conclusion, FLU based conditioning is more effective and successful with lower toxicity in multitransfused FA patients. However, it needs more experience and longer follow up duration.
authors: Department of Pediatric Hematology-Oncology, Akdeniz University School of Medicine, Antalya, Turkiye. yesilipek@akdeniz.edu.tr
authors: Mehmet Akif
authors: MA
authors: Yesilipek
authors: Gulsun Tezcan
authors: GT
authors: Karasu
authors: Alphan
authors: A
authors: Kupesiz
authors: Vedat
authors: V
authors: Uygun
authors: Volkan
authors: V
authors: Hazar
mesh_terms: D000293:Adolescent; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D005199:Fanconi Anemia / Q000628:therapy*; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D006801:Humans; D008297:Male; D036102:Peripheral Blood Stem Cell Transplantation; D011878:Radiotherapy; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives*
chemical_list: D000961:Antilymphocyte Serum; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine