Deferasirox film-coated tablet (FCT) is a favorable option for long-term management of iron overload with iron chelation therapy (ICT), according to research from a phase 3 trial published in Experimental Hematology & Oncology.
Patients with chronic transfusion-dependent refractory anemias may require ICT for life to avoid complications from iron overload. However, compliance with ICT is a challenge. Deferasirox is 1 of 3 main iron chelators. Deferasirox FCT has a simpler oral administration and improved gastrointestinal tolerability compared to the dispersible tablet (DT) formulation.
The phase 2 ECLIPSE study evaluated the safety and efficacy of deferasirox DT and FCT in patients with transfusion-dependent thalassemia (TDT) or very low-, low-, or intermediate-risk myelodysplastic syndrome (MDS). The current study is a phase 3 trial studying the long-term safety and efficacy of the FCT formulation in a subset of patients treated for 2 years after the 24-week treatment in the ECLIPSE study.
A total of 53 patients were enrolled, and 19 discontinued treatment early. Out of these, 49 patients had TDT and 4 patients had MDS. A total of 50 patients were treated for at least 20 weeks. Out of these patients, 80% of patients received an average actual dose of 17.5mg/kg/day of deferasirox FCT.
A total of 98.1% of patients experienced at least 1 adverse event, with the most common being headache, diarrhea, pyrexia, nausea, vomiting, cough, and upper abdominal pain. This phase 3 study found no new safety signals and the adverse events seen in the study were consistent with those expected with deferasirox.
In the study, patients had a consistent reduction in serum ferritin levels across all post-baseline time points. From month 9 and after, researchers observed a greater than 25% decrease in serum ferritin.
Patients had a treatment compliance of about 90% and persistence (continuous use for 30 days or longer) of more than 95%. The study authors concluded that the FCT formulation of deferasirox is a favorable choice of ICT.
Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.
Tartaglione I, Origa R, Kattamis A, et al. Two-year long safety and efficacy of deferasirox film-coated tablets in patients with thalassemia or lower/intermediate risk MDS: phase 3 results from a subset of patients previously treated with deferasirox in the ECLIPSE study. Exp Hematol Oncol. Published online August 10, 2020. doi:10.1186/s40164-020-00174-2