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Deferiprone vs deferoxamine for transfusional iron overload in SCD and other anemias: a randomized, open-label, noninferiority study

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States. Cairo University, Cairo, Egypt. Cairo University, Giza, Egypt. Ain Shams University, Cairo, Egypt. Zagazig University, Zagazig, Egypt. Assir Central Hospital, Abha, Saudi Arabia. University of Alabama Birmingham, Birmingham, Alabama, United States. Guy's and St Thomas' Hospital, London, Wyoming, United Kingdom. Hospital for Sick Children, Toronto, Canada. Cukurova University Medical Facility, Adana, Turkey. Chiesi Canada Corporation, Woodbridge, Canada. Chiesi Canada Corporation, Toronto, Canada.

Blood advances. 2021
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PICO Summary

Population

Patients with sickle cell disease or other anaemias receiving chronic transfusion therapy (n= 228).

Intervention

Oral deferiprone (n= 152).

Comparison

Subcutaneous deferoxamine (n= 76).

Outcome

The least squares mean (standard error) change in liver iron concentration was -4.04 (0.48) mg/g dry weight for deferiprone vs. -4.45 (0.57) mg/g dry weight for deferoxamine, with noninferiority of deferiprone to deferoxamine demonstrated by analysis of covariance. Non-inferiority of deferiprone was also shown for both cardiac T2* MRI and serum ferritin. Treatment-related adverse events (AEs), serious AEs, and AEs leading to withdrawal did not differ significantly between the groups.
Abstract
Many people with sickle cell disease (SCD) or other anemias require chronic blood transfusions, which often causes iron overload and requires chelation therapy. The iron chelator deferiprone is often used in individuals with thalassemia syndromes, but data in patients with SCD are limited. This open-label study (NCT02041299) assessed the efficacy and safety of deferiprone in patients with SCD or other anemias receiving chronic transfusion therapy. A total of 228 patients (mean age: 16.9 [range 3-59] years; 46.9% female) were randomized to receive either oral deferiprone (n = 152) or subcutaneous deferoxamine (n = 76). The primary endpoint was change from baseline at 12 months in liver iron concentration (LIC), assessed by R2* magnetic resonance imaging (MRI). The least squares mean (standard error) change in LIC was -4.04 (0.48) mg/g dry weight for deferiprone vs -4.45 (0.57) mg/g dry weight for deferoxamine, with noninferiority of deferiprone to deferoxamine demonstrated by analysis of covariance (least squares mean difference 0.40 [0.56]; 96.01% confidence interval, -0.76, 1.57). Noninferiority of deferiprone was also shown for both cardiac T2* MRI and serum ferritin. Rates of overall adverse events (AEs), treatment-related AEs, serious AEs, and AEs leading to withdrawal did not differ significantly between the groups. AEs related to deferiprone treatment included abdominal pain (17.1% of patients), vomiting (14.5%), pyrexia (9.2%), increased alanine transferase (9.2%) and aspartate transferase levels (9.2%), neutropenia (2.6%), and agranulocytosis (0.7%). The efficacy and safety profiles of deferiprone were acceptable and consistent with those seen in patients with transfusion-dependent thalassemia.
Study details
Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine