Use of Deferasirox Film-Coated Tablets in Pediatric Patients with Transfusion Dependent Thalassemia: A Single Center Experience

Abstract

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.

Efficacy and safety of early‐start deferiprone in infants and young children with transfusion‐dependent beta thalassemia: Evidence for iron shuttling to transferrin in a randomized, double‐blind, placebo‐controlled, clinical trial (START)

“…No patients reported AEs involving increased liver enzymes, a common AE observed with iron chelators, including deferiprone 14 . In contrast, the deferasirox film‐coated tablet has been reported to have an increased frequency of AEs in younger patients, including high liver enzyme activity 29 . Furthermore, the frequent AEs in children treated with deferasirox contributed to high dropout rates, with all patients aged 2–6 years discontinuing 29 .…”

“…14 In contrast, the deferasirox film-coated tablet has been reported to have an increased frequency of AEs in younger patients, including high liver enzyme activity. 29 Furthermore, the frequent AEs in children treated with deferasirox contributed to high dropout rates, with all patients aged 2-6 years discontinuing. 29 Agranulocytosis and the less severe neutropenia-are the most concerning safety issues associated with iron chelators.…”

“…29 Furthermore, the frequent AEs in children treated with deferasirox contributed to high dropout rates, with all patients aged 2-6 years discontinuing. 29 Agranulocytosis and the less severe neutropenia-are the most concerning safety issues associated with iron chelators. The rates of agranulocytosis and neutropenia observed in START were low and consistent with rates seen in other age groups receiving deferiprone.…”

Switching from Deferasirox Dispersible Tablets to Deferasirox Film-Coated Tablets: Is There an Effect on Ferritin Levels in Children and Adolescents with Transfusion-Dependent Anemia?

“… 2 , 3 In practice, ICT is started after the first 10-20 transfusions or when the serum ferritin (SF) level is >1000 ng/mL. 4 , 5 The necessity of lifelong and daily use requires compliance and commitment to treatment. Therefore, minimizing the side effects of ICT drugs and ease of use are important for patients.…”

“…Compared to DFO, which must be used parenterally, and DFP, which is used orally 3 times a day, DFX provides ease of use and thus improves compliance in patients because it is taken orally once a day. 5 It has been in use in Turkey since 2007 and is used as the first-choice iron chelator in our center. 7 The film-coated tablet (FCT) form of DFX was approved by the FDA in 2015 and promises a more comfortable use than the original form.…”