Abstract
Simple SummaryThalassemia is a hereditary anemia characterized by defect in hemoglobin synthesis. Patients with severe forms of the disease require regular blood transfusions, as well as iron chelation. Available chelators have demonstrated long-term efficacy and safety, but present various limitations. A new deferasirox film-coated tablet (DFX FCT) was designed to offer a more convenient mode of administration and better tolerability. The present study aimed to assess the efficacy and safety of DFX FCT in thalassemic pediatric patients. DFX FCT proved safe for older patients, but demonstrated increased frequency of adverse events in younger patients, resulting in drug discontinuation. As for efficacy, DFX FCT succeeded in maintaining a stable iron load, though it required use of relatively high doses.Thalassemic syndromes are characterized by clinical heterogenicity. For severe disease forms, lifelong blood transfusions remain the mainstay of therapy, while iron overload monitoring and adequate chelation treatment are required in order to ensure effective disease management. Compared to previous chelators, the new deferasirox film-coated tablet (DFX FCT) is considered to offer a more convenient and well-tolerated treatment scheme, aiming at better treatment-related and patient-related outcomes. The present study’s objective was to prospectively evaluate the safety and efficacy of DFX FCT in children and adolescents with transfusion-dependent thalassemia. Data collected included patient demographics, hematology and biochemistry laboratory work up, magnetic resonance imaging of heart and liver for iron load, as well as ophthalmological and audiological examination prior to and a year following drug administration. Study results confirmed DFX FCT safety in older children in a manner similar to adults, but demonstrated increased frequency of adverse events in younger patients, mainly, involving liver function. With regards to efficacy, study results confirmed the preventive role of DFX FCT in iron loading of liver and heart, however, higher doses than generally recommended were required in order to ensure adequate chelation.
Highlights
Thalassemia syndromes are recessively inherited hemoglobinopathies, characterized by defects in hemoglobin production [1,2]
Adequate chelation during childhood, i.e., dose tailoring based on changing body weight, as well as treatment modifications guided by iron overload markers, is of great importance in order to achieve complication free survival-even, normal life expectancy [7]
Prior to deferasirox film-coated tablet (DFX film-coated tablet (FCT)) administration, 14 (56%) patients were on the previous DFX formulation (DFX dispersible tablet (DT)), 7 (28%) were on a different iron chelator and 4 (16%) were chelation-naive (Table 1)
Summary
Thalassemia syndromes are recessively inherited hemoglobinopathies, characterized by defects in hemoglobin production [1,2]. Mutations within globin genes, located in chromosomes 16 and 11, result in reduced production of globin chains and, hemoglobin A [3]. Subsequent parenchymal iron deposition, primarily in the heart, liver and endocrine glands, results in severe organ damage [6]. Besides safe transfusions, iron overload monitoring and iron chelation treatment are necessary in order to ensure effective disease management [2]. Adequate chelation during childhood, i.e., dose tailoring based on changing body weight, as well as treatment modifications guided by iron overload markers, is of great importance in order to achieve complication free survival-even, normal life expectancy [7]
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