Skip to content
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Menu
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Menu
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
biochemistry, genetics and molecular biology
Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: Efficacy and safety during 5 years' follow-up
Blood, Volume 118, No. 4, Year 2011
Notification
URL copied to clipboard!
Description
Patients with β-thalassemia require lifelong iron chelation therapy from early childhood to prevent complications associated with transfusional iron overload. To evaluate long-term efficacy and safety of once-daily oral iron chelation with deferasirox, patients aged ≥ 2 years who completed a 1-year, phase 3, randomized trial entered a 4-year extension study, either continuing on deferasirox (deferasirox cohort) or switching from deferoxamine to deferasirox (crossover cohort). Of 555 patients who received ≥ 1 deferasirox dose, 66.8% completed the study; 43 patients (7.7%) discontinued because of adverse events. In patients with ≥ 4 years' deferasirox exposure who had liver biopsy, mean liver iron concentration significantly decreased by 7.8 ± 11.2 mg Fe/g dry weight (dw; n = 103; P < .001) and 3.1 ± 7.9 mg Fe/g dw (n = 68; P < .001) in the deferasirox and crossover cohorts, respectively. Median serum ferritin significantly decreased by 706 ng/mL (n = 196; P < .001) and 371 ng/mL (n = 147; P < .001), respectively, after ≥ 4 years'exposure. Investigator-assessed, drug-related adverse events, including increased blood creatinine (11.2%), abdominal pain (9.0%), and nausea (7.4%), were generally mild to moderate, transient, and reduced in frequency over time. No adverse effect was observed on pediatric growth or adolescent sexual development. This first prospective study of long-term deferasirox use in pediatric and adult patients with β- thalassemia suggests treatment for ≤ 5 years is generally well tolerated and effectively reduces iron burden. This trial was registered at www.clinicaltrials-.gov as #NCT00171210. © 2011 by The American Society of Hematology.
Authors & Co-Authors
Cappellini, Maria Teresa
Italy, Milan
Università Degli Studi Di Milano
Béjaoui, Mohamed
Tunisia, Tunis
Centre National de Greffe de Moelle Osseuse
Agaoglu, Leyla
Turkey, Istanbul
İstanbul Tıp Fakültesi
Canatan, Duran
Turkey, Isparta
Süleyman Demirel Üniversitesi
Capra, Marcello
Italy, Vicenza
Ospedale Civile Di Vicenza
Cohen, Alan R.
United States, Philadelphia
The Children's Hospital of Philadelphia
Drelichman, Guillermo I.
Argentina, Buenos Aires
Hospital de Ninos Ricardo Gutierrez
Economou, Marina P.
Greece, Athens
Hippokration General Hospital
Fattoum, Slaheddine
Tunisia, Tunis
Université de Tunis el Manar, Hôpital D'enfants Béchir Hamza de Tunis
Kattamis, Antonis C.
Greece, Athens
National and Kapodistrian University of Athens
Kılınç, Yurdanur
Turkey, Adana
Çukurova Üniversitesi Tip Fakültesi
Perrotta, Silverio
Italy, Naples
Università Degli Studi Di Napoli Federico Ii
Piga, Antonio Giulio
Italy, Turin
Università Degli Studi Di Torino
Porter, John B.
United Kingdom, London
University College London
Griffel, Louis
Switzerland, Basel
Novartis International ag
Dong, Victor
Switzerland, Basel
Novartis International ag
Clark, Joan
Switzerland, Basel
Novartis International ag
Aydınok, Yeşim
Turkey, Izmir
Ege University Medical School
Statistics
Citations: 207
Authors: 18
Affiliations: 16
Identifiers
Doi:
10.1182/blood-2010-11-316646
ISSN:
00064971
Research Areas
Health System And Policy
Maternal And Child Health
Sexual And Reproductive Health
Study Design
Cohort Study