1. Fludarabine, low-dose busulfan and antithymocyte globulin as conditioning for Fanconi anemia patients receiving bone marrow transplantation from HLA-compatible related donors
- Author
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Alexander Rumiantsev, Y.V. Skvortsova, Alexey Maschan, Pavel Trakhtman, Z M Dyshlevaja, E V Skorobogatova, I. Shipicina, E V Samochatova, and Dmitry Balashov
- Subjects
Male ,medicine.medical_specialty ,Transplantation Conditioning ,medicine.medical_treatment ,Hematopoietic stem cell transplantation ,Gastroenterology ,Postoperative Complications ,Daclizumab ,immune system diseases ,Fanconi anemia ,Internal medicine ,Cyclosporin a ,Living Donors ,medicine ,Humans ,Transplantation, Homologous ,Child ,Busulfan ,Antilymphocyte Serum ,Bone Marrow Transplantation ,Transplantation ,business.industry ,Histocompatibility Testing ,Hematology ,medicine.disease ,Fludarabine ,Fanconi Anemia ,surgical procedures, operative ,medicine.anatomical_structure ,Child, Preschool ,Cytomegalovirus Infections ,Immunology ,Drug Therapy, Combination ,Female ,Bone marrow ,business ,Immunosuppressive Agents ,Vidarabine ,Stem Cell Transplantation ,medicine.drug - 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.
- Published
- 2004
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