Deferasirox in patients with iron overload secondary to hereditary hemochromatosis: results of a 1‐yr Phase 2 study

• DOI: 10.1111/ejh.12530
• Publication Date: 2015-02-13T13:10:09Z
• Authors: Rodolfo Cançado; Murilo R. Melo; Roberto de Moraes...; Paulo C. J. L. Sa...; Elivira M. Guerra...; Carlos Chiattone; Samir K. Ballas
• Keywords: Adult; Erythrocyte Indices; Male; 0301 basic medicine; Iron Overload; Time Factors; Histocompatibility Antigens Class I; Membrane Proteins; Middle Aged; Triazoles; Iron Chelating Agents; Benzoates; 3. Good health; Deferasirox; 03 medical and health sciences; Treatment Outcome; Mutation; Humans; Female; Hemochromatosis; Hemochromatosis Protein; Biomarkers; Aged

AbstractThis open‐label, prospective, phase 2 study evaluated the safety and efficacy of deferasirox (10 ± 5 mg/kg/d) in patients with hereditary hemochromatosis (HH) and iron overload refractory to or intolerant of phlebotomy. Ten patients were enrolled and all completed the 12‐month treatment period. There were significant decreases from baseline to end of study (i.e., 12 months) in median serum ferritin (P < 0.001), mean transferrin saturation (P < 0.05), median liver iron concentration (P < 0.001), and mean alanine aminotransferase (P < 0.05). The median time to achieve serum ferritin reduction ≥50% compared to baseline was 7.53 months. The most common adverse events were mild, transient diarrhea (n = 5) and nausea (n = 2). No patient experienced an increase in serum creatinine that exceeded the upper limit of normal. These data confirm that deferasirox was well tolerated and effective in reducing iron burden in patients with hereditary hemochromatosis and could be a safe alternative to phlebotomy in selected patients.