Yazarlar : Cappellini MD, Bejaoui M, Agaoglu L, Canatan D, Capra M, Cohen A, Drelichman G, Economou M, Fattoum S, Kattamis A, Kilinc Y, Perrotta S, Piga A, Porter JB, Griffel L, Dong V, Clark J, Aydinok Y.
Yayın : Blood
Yayın Yılı : 2011
Pubmed Linki : http://www.ncbi.nlm.nih.gov/pubmed/21628399
Konu : Talasemi
Literatür İçeriği :
Abstract
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 III, 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 (AEs). In patients with ≥4 years' deferasirox exposure who had liver biopsy, mean liver iron concentration significantly decreased by 7.8±11.2 (n=103;P<0.001) and 3.1±7.9 (n=68;P<0.001) mg Fe/g dw in the deferasirox and crossover cohorts, respectively. Median serum ferritin significantly decreased by 706 (n=196;P<0.001) and 371 (n=147;P<0.001) ng/mL, respectively, after ≥4 years exposure. Investigator-assessed, drug-related AEs, 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. There was no adverse effect on pediatric growth or adolescent sexual development. This first prospective study of long-term deferasirox use in pediatric and adult β-thalassemia patients suggests treatment for up to 5 years is generally well tolerated and effectively reduces iron burden. This study is registered at www.clinicaltrials.gov as NCT0017210.
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