Deferiprone (DFP) is non-inferior to deferoxamine (DFO) for treating iron-overloaded patients with sickle cell disease (SCD) or other transfusion-dependent anemias, according to research presented at the 2021 American Society of Pediatric Hematology/Oncology (ASPHO) meeting.
DFO and deferasirox are iron chelators approved in the United States to treat chronic iron overload in patients with SCD or other anemias. However, they are contraindicated in patients with severe renal disease. DFP is an iron chelator approved for patients with thalassemia.
The FIRST study (ClinicalTrials.gov Identifier NCT02041299) was a randomized, open-label noninferiority trial of DFP compared to DFO in iron-overloaded patients with SCD and other transfusion-dependent anemias. 152 patients were assigned to DFP, and 76 were assigned to DFO for 12 months. The researchers measured liver iron concentration (LIC), serum ferritin, and cardiac iron overload.
Both treatments had similar efficacy at 12 months. The mean change in LIC was -4.13 for the DFP group and -4.38 for the DFO group. Both groups also had similar patient-reported quality of life scores.
At the end of the trial, 84% of patients who completed FIRST (89 of 106 patients) enrolled in an extension study to continue receiving DFP. A total of 45 of 58 patients in the DFO group switched to DFP for the extension study.
The authors concluded that DFP is not inferior to DFO in this patient population, and that DFP presented no new safety concerns.
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.
Elalfy MS, Hamdy M, El-Beshlawy A, et al. Safety and efficacy of deferiprone vs deferoxamine for transfusion-dependent anemias. Poster presented at: 2021 American Society of Pediatric Hematology/Oncology meeting; April 21-23, 2021; virtual.