Among patients with transfusion-dependent hemoglobinopathy, deferiprone appears to be cost-effective and clinically non-inferior to deferasirox for inducing iron overload control over 1 treatment year, according to research published in The Lancet Haematology.
Worldwide, up to 500,000 infants are born each year with a clinically relevant hemoglobinopathy, including alpha- and beta-thalassemia and sickle cell disease. Many of these patients become transfusion-dependent and will require iron chelation therapy throughout their lifetime.
Iron chelation options, which include deferiprone, deferasirox, and deferoxamine, differ in method of administration and in price, which inform treatment decisions in the adult population of patients. In pediatric patients, however, there are few data comparing the safety and efficacy of these options, and deferasirox is the only option currently used in this patient group.
Given that deferasirox costs more than does deferiprone, the researchers noted in their paper that any data revealing non-inferiority of the latter could help to provide iron chelation therapy to a greater percentage of a population in need. For this randomized, multi-center, phase 3 study, researchers evaluated whether deferiprone is clinically non-inferior to deferasirox among pediatric patients with hemoglobinopathy.
Of 435 patients enrolled, 393 were randomly assigned to receive deferiprone (194 patients) or deferasirox (199 patients); 140 and 170 patients, respectively, completed the trial. Baseline characteristics in the intention-to-treat population were well-balanced, with 80 (42%) and 93 (47%) female patients in the deferiprone and deferasirox groups, respectively; 87 (45%) and 92 (47%) were at least 10 years old; 175 (91%) and 177 (90%) had beta-thalassemia major; and 27 (14%) and 27 (14%) were naïve to chelation therapy, respectively.
Overall, the median follow-up periods in the deferiprone and deferasirox groups were 379 and 381 days, respectively. Among patients with primary endpoint data available at both baseline and 1 year, 69 of 125 (55.2%) patients in the deferiprone group vs 80 of 146 (54.8%) patients in the deferasirox group met the predefined criteria for treatment success (difference, 0.4%; 95% CI: -11.9-12.6).
Both compliance and adverse event rates were similar between the 2 groups.
“In conclusion, our trial supports the use of deferiprone in paediatric patients with transfusion-dependent haemoglobinopathies on the basis of data from the largest randomised clinical trial of iron chelation therapy in these patients,” the authors wrote. “The main clinical implication of this study is that paediatric patients might now have more than one efficacious and safe option for oral iron chelation therapy.”
Disclosures: Some authors have declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.
Maggio A, Kattamis A, Felisi M, et al. Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial. Lancet Haematol. 2020;7(6):e469-78. doi:10.1016/S2352-3026(20)30100-9