title: Deferasirox in patients with iron overload secondary to hereditary hemochromatosis: results of a 1-yr Phase 2 study.
abstract: 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) 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.
authors: Department of Hematology/Oncology, Santa Casa Medical School, Sao Paulo, Brazil.
authors: Rodolfo
authors: R
authors: Cançado
authors: Molecular Medicine Laboratory, Santa Casa Medical School, Sao Paolo, Brazil.
authors: Murilo R
authors: MR
authors: Melo
authors: Radiology Department, Santa Casa Medical School, Sao Paolo, Brazil.
authors: Roberto
authors: R
authors: de Moraes Bastos
authors: Laboratory of Genetics and Molecular Cardiology, Heart Institute (InC0r), University of Sao Paolo Medical School, Sao Paolo, Brazil.
authors: Paulo C J L
authors: PC
authors: Santos
authors: Department of Clinical Chemistry and Toxicology, Pharmaceutical Sciences School, University of Sao Paolo, Sao Paolo, Brazil.
authors: Elivira M
authors: EM
authors: Guerra-Shinohara
authors: Department of Hematology/Oncology, Santa Casa Medical School, Sao Paulo, Brazil.
authors: Carlos
authors: C
authors: Chiattone
authors: Cardeza Foundation for Hematological Research Jefferson Medical College, Thomas Jefferson University Philadelphia, Philadelphia, PA, USA.
authors: Samir K
authors: SK
authors: Ballas
mesh_terms: D000328:Adult; D000368:Aged; D001565:Benzoates / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D015415:Biomarkers; D000077588:Deferasirox; D004909:Erythrocyte Indices; D005260:Female; D006432:Hemochromatosis / Q000150:complications* / Q000175:diagnosis / Q000235:genetics; D000071020:Hemochromatosis Protein; D015395:Histocompatibility Antigens Class I / Q000235:genetics; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology*; D008297:Male; D008565:Membrane Proteins / Q000235:genetics; D008875:Middle Aged; D009154:Mutation; D013997:Time Factors; D016896:Treatment Outcome; D014230:Triazoles / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D001565:Benzoates; D015415:Biomarkers; C103673:HFE protein, human; D000071020:Hemochromatosis Protein; D015395:Histocompatibility Antigens Class I; D007502:Iron Chelating Agents; D008565:Membrane Proteins; D014230:Triazoles; D000077588:Deferasirox
keywords: chelation therapy; deferasirox; erythrocytapheresis; hereditary hemochromatosis; iron overload; phlebotomy; transfusional siderosis