Long-term use of deferiprone was shown to be a safe and effective treatment to control iron overload in patients with sickle cell disease, according to results of the FIRST-EXT study presented at the 2021 American Society of Pediatric Hematology/Oncology (ASPHO) meeting.

FIRST-EXT (ClinicalTrials.gov Identifier NCT02443545) was a prospective, multicenter, open-label extension of the FIRST study (ClinicalTrials.gov Identifier NCT02041299). The objective was to assess the long-term safety and efficacy of deferiprone in patients with sickle cell disease. Safety was assessed by measuring adverse events (AEs), serious AEs, and the number of discontinuations due to AEs.

A total of 134 patients who completed the FIRST trial were invited to participate in the extension trial and all received deferiprone. Of these participants, 85.8% had sickle cell disease and 14.2% had other anemias. The mean patient age was 16.2±8.6 years. At baseline, all patients had a liver iron concentration (LIC) above normal. All patients except 1 had serum ferritin (SF) levels above normal, and all patients had normal cardiac iron levels as assessed by magnetic resonance imaging.

Continue Reading

The change from baseline was determined at 3 different times after treatment with deferiprone: 1 year, 2 years, and 3 years. A total of 122 patients completed treatment with deferiprone for 1 year, 69 patients for 2 years, and 50 patients for 3 years.

The most common reason for withdrawal or discontinuation from the study was due to sponsor decision (31 patients, 23.1%). This was because the study was stopped early after existing methods of surveillance for safety were deemed sufficient.

LIC decreased significantly over time, with the mean values decreasing from 14.93 mg/g dw at baseline to 12.30 mg/g dw after 1 year of treatment, to 11.19 mg/g dw after 2 years, and to 10.45 mg/g dw after 3 years (P <.001 for all). SF levels did not decrease significantly after 1 year of treatment (P =.995), but were significantly lower after 2 years (P <.001) and 3 years (P =.042) of treatment. Cardiac iron levels did not change over time, and no patients had excess cardiac iron levels during treatment.

Overall, 41 patients (30.6%) reported at least 1 AE during the study, with the most common including neutropenia (12 patients), decreased neutrophil count (12 patients), and abdominal pain (10 patients). A total of 13 patients (9.7%) experienced serious AEs, including neutropenia (12 patients), agranulocytosis (2 patients), thrombocytopenia (1 patient), and generalized edema (1 patient). Of these, 3 AEs were determined “definitely related” to deferiprone treatment.

Only 4 patients discontinued treatment due to AEs, 2 of which were due to pregnancy.

The study presenters concluded that deferiprone treatment was well tolerated because most AEs were mild and not determinably related to deferiprone. No new safety concerns were observed.

Disclosure: This clinical trial was supported by ApoPharma (now Chiesi) and writing support was funded by Chiesi and Oxford PharmaGenesis. Please see the original reference for a full list of authors’ disclosures.


Elafly MS, Hamdy M, El-Beshlawy A, et al. Long-term efficacy and safety of deferiprone for patients with sickle cell disease or other anemias. Poster presented at: 2021 American Society of Pediatric Hematology/Oncology virtual meeting; April 21-23, 2021.