Deferasirox Decreases Liver Iron Concentration in Iron-Overloaded Patients with Myelodysplastic Syndromes, Aplastic Anemia and Other Rare Anemias.

摘要: Iron overload in transfusion-dependent patients with rare anemias can be managed chelation therapy. This study evaluated deferasirox efficacy and safety myelodysplastic syndromes (MDS), aplastic anemia (AA) or other anemias. A 1-year, open-label, multicenter, single-arm, phase II trial was performed (10–40 mg/kg/day, based on transfusion frequency therapeutic goals), including an optional 1-year extension. The primary end point a change liver iron concentration (LIC) after 1 year. Secondary points included changes parameters (including ophthalmologic assessments) overall as well Japanese subpopulation. Overall, 102 (42 MDS, 29 AA 31 anemias) were enrolled; 57 continued into the Mean absolute LIC –10.9 mg Fe/g dry weight (d.w.) year (baseline: 24.5 d.w.) –13.5 d.w. 2 years. most common drug-related adverse event increased serum creatinine (23.5%), predominantly MDS patients. Four had suspected abnormalities. Outcomes generally consistent population. Results confirm anemias, profile previous studies agreed baseline values (EUDRACT 2006-003337-32).