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High-dose cyclophosphamide effective treatment for severe aplastic anemia
High-dose cyclophosphamide was effective at treating severe aplastic anemia in treatment-naive patients, according to long-term follow-up results.
Researchers assessed the response, survival and long-term follow-up of patients with severe aplastic anemia assigned daily IV cyclophosphamide at a dose 50 mg/kg of ideal body weight for four consecutive days. Forty-four patients were treatment-naive; 23 received at least one course of either antithymocyte globulin and cyclosporine (n=21) or cyclosporine alone (n=2).
IV 2-mercaptoethane sulfonate 10 mg/kg was administered 30 minutes before and three, six and eight hours after cyclophosphamide. On day 10, all patients received daily granulocyte colony-stimulating factor 5 mg/kg until neutrophil count was 1 × 109 cells/L for two consecutive days.
At 10-year follow-up, OS was 88% (95% CI, 75%-95%), hematologic response rate was 70.5% and event-free survival was 58% among treatment-naive patients.
Patients with refractory severe aplastic anemia did not manage as well after high-dose cyclophosphamide therapy; at 10 years, OS was 61.8%, response rate was 47.8% and event-free survival was 27.7%.
Median time to neutrophil count of 0.5 × 109 cells/L was 60 days for treatment-naive patients; median time to final platelet transfusion was 117 days; median time to final red cell transfusion was 186 days.
Patients with refractory severe aplastic anemia had a median time to a neutrophil count of 54 days; median time to final platelet transfusion of 103 days; and median time to final red cell transfusion of 210 days.
Median time to complete remission was 20 months.
The researchers suggested a “large multi-institutional study to compare high-dose cyclophosphamide vs. traditional immunosuppressive therapy to determine the best approach for managing severe aplastic anemia patients who are not candidates for bone marrow transplant.”
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- Brodsky RA. Blood. 2010;115:2136-2141.