Phase II clinical evaluation of deferasirox, a once-daily oral chelating agent, in pediatric patients with beta-thalassemia major

• Published in: Haematologica (Roma) Vol. 91; no. 10; pp. 1343 - 1351
• Main Authors: Galanello, R, Piga, A, Forni, GL, Bertrand, Y, Foschini, ML, Bordone, E, Leoni, G, Lavagetto, A, Zappu, A, Longo, F, Maseruka, H, Hewson, N, Sechaud, R, Belleli, R, Alberti, D
• Format: Journal Article
• Language: English
• Published: Pavia Haematologica 01.10.2006
• Subjects: Administration, Oral; Adolescent; Anemias. Hemoglobinopathies; Benzoates - administration & dosage; Benzoates - blood; beta-Thalassemia - blood; beta-Thalassemia - drug therapy; Biological and medical sciences; Child; Child, Preschool; Diseases of red blood cells; Drug Administration Schedule; Female; Hematologic and hematopoietic diseases; Humans; Iron Chelating Agents - administration & dosage; Iron Chelating Agents - metabolism; Male; Medical sciences; Triazoles - administration & dosage; Triazoles - blood; Human; Hemoglobinopathy; Pediatrics; thalassemia; Hematology; Hemopathy; Iron; Genetic disease; pediatric; Deferasirox; Hemolytic anemia; β-Thalassemia; Chelating agent; ICL670; iron chelation; Daily dose; Child
• ISSN: 0390-6078; 1592-8721

Ospedale Regionale Microcitemie, Dipartimento di Scienze Biomediche e Biotecnologie, Universita di Cagliari, Via Jenner s/n, 09121 Cagliari, Italy. renzo.galanello@mcweb.unica.it BACKGROUND AND OBJECTIVES: Deferasirox (ICL670) is a novel once-daily oral iron chelator developed for the treatment of chronic iron overload from blood transfusions. This study evaluated the safety and tolerability of deferasirox in pediatric patients with transfusion-dependent beta-thalassemia major. Efficacy and pharmacokinetic assessments were secondary objectives. DESIGN AND METHODS: Forty patients equally stratified into two age groups--children (2 to