A phase 1/2, dose‐escalation trial of deferasirox for the treatment of iron overload in HFE‐related hereditary hemochromatosis

• Published in: Hepatology (Baltimore, Md.) Vol. 52; no. 5; pp. 1671 - 1779
• Main Authors: Phatak, Pradyumna, Brissot, Pierre, Wurster, Mark, Adams, Paul C, Bonkovsky, Herbert L., Gross, John, Malfertheiner, Peter, McLaren, Gordon D., Niederau, Claus, Piperno, Alberto, Powell, Lawrie W., Russo, Mark W., Stoelzel, Ulrich, Stremmel, Wolfgang, Griffel, Louis, Lynch, Nicola, Zhang, Yiyun, Pietrangelo, Antonello
• Format: Journal Article
• Language: English
• Published: Hoboken Wiley Subscription Services, Inc., A Wiley Company 01.11.2010; Wiley; Wiley Subscription Services, Inc
• Subjects: Adult; Aged; Amino Acid Substitution; Anemia; Benzoates - adverse effects; Benzoates - therapeutic use; Biological and medical sciences; Creatinine - blood; Dose-Response Relationship, Drug; Female; Ferritins - blood; Ferritins - genetics; Gastroenterology. Liver. Pancreas. Abdomen; Hemochromatosis - blood; Hemochromatosis - complications; Hemochromatosis - genetics; Hemochromatosis - therapy; Hepatology; Homozygote; Humans; Iron; Iron Chelating Agents - adverse effects; Iron Chelating Agents - therapeutic use; Iron Overload - blood; Iron Overload - drug therapy; Iron Overload - etiology; Iron Overload - therapy; Liver. Biliary tract. Portal circulation. Exocrine pancreas; Male; Medical sciences; Metabolic diseases; Metals (hemochromatosis...); Middle Aged; Other metabolic disorders; Phlebotomy - methods; Safety; Steatohepatitis/Metabolic Liver Disease; Transferrin - metabolism; Triazoles - adverse effects; Triazoles - therapeutic use; Deferasirox; Iron overload; Hemochromatosis; Treatment; Gastroenterology; Chelating agent; Metabolic diseases; Iron; Enzymopathy; Dose

Hereditary hemochromatosis (HH) is characterized by increased intestinal iron absorption that may result in iron overload. Although phlebotomy is widely practiced, it is poorly tolerated or contraindicated in patients with anemias, severe heart disease, or poor venous access, and compliance can vary...