Clinical outcome of patients with acquired bone marrow failure syndrome who received bone marrow transplantation following fludarabine-containing conditioning regimen in a single institute

Abstract

We reviewed the clinical courses of nine patients in our institute with acquired bone marrow failure syndrome. Five male and four female patients with a median age of 7 years (range: 6–19 years) were included in this study. Four patients were diagnosed with aplastic anemia, four with refractory cytopenia of childhood, and one with refractory cytopenia with multilineage dysplasia. Five patients were treated with immunosuppressive therapy (IST), including anti-thymocyte globulin and cyclosporine. IST responses were observed in only one patient with RCC (partial response). Nine patients received bone marrow transplantation (BMT) following fludarabine-containing regimen for conditioning. Engraftment was successful for all patients, although one patient received a second BMT owing to the development of a donor-type bone marrow aplasia. Acute graft-versus-host disease (GVHD) grade II or higher developed in five patients. Chronic GVHD developed in two patients, one of whom developed an extensive type. In terms of transplantation-associated complications, severe thrombotic microangiopathy, myasthenia gravis, or brain tumor developed in each patient. The IST response rate in our small cohort was lower than that in previous reports; however, all patients recovered from bone marrow failure after treatment by BMT. The development of GVHD and sequelae associated with BMT affected the patients’ quality of life.