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

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