(15) Real-world safety profile of twice-daily deferiprone for iron overload in US patients with thalassemia, sickle cell disease or other anemias

Location: Exhibit Hall D

First Author Presenting(s)

Mengru Chen, MD MSc (she/her/hers)

Dr
Chiesi UK Limited.
Manchester, England, United Kingdom

Co-Author Not Presenting(s)

Sujit Sheth, MD

Professor
New York-Presbyterian Hospital-Weill Cornell Medical Center
New York, New York, United States
AL

Ashutosh Lal, MD

Dr
UCSF Benioff Children’s Hospital Oakland
Oakland, California, United States
Abena O. Appiah-Kubi, MD, MPH

Dr
Cohen Children’s Medical Center New York; Northwell Health New York
New York, New York, United States
Scott A. Peslak, MD PhD (he/him/his)

Dr
Department of Medicine, Division of Hematology/Oncology, University of Pennsylvania Perelman School of Medicine
Philadelphia, Pennsylvania, United States
NT

Noemi Toiber Temin, MA Stats P. Stat

Ms
Chiesi Canada Corp.
Woodbridge, Ontario, Canada
TC

Thomas Coates, MD

Professor
Children’s Hospital Los Angeles
Los Angeles, California, United States

Title: Real-world safety profile of twice-daily deferiprone for iron overload in US patients with thalassemia, sickle cell disease or other anemias

Abstract : Basis of inquiry:
Iron overload is a significant cause of transfusion-related morbidity and mortality. Deferiprone (DFP) is an oral iron chelator with >28 years of safety and efficacy data. Initially approved for administration 3 times a day (TID), a twice-a-day (BID) formulation was developed to improve patient adherence. The Food and Drug Administration (FDA) approved DFP BID for transfusional iron overload in patients with thalassemia in 2020, and DFP TID and BID for sickle cell disease (SCD) or other anemias in 2021. To evaluate the real-world safety profile of DFP, the Ferriprox® Total Care Registry was established in the United States (US).

Purpose/objective:
Assess the safety of DFP BID in patients with thalassemia, SCD, or other anemias in real-world clinical practice.

Methods:
Data were obtained from US patients receiving DFP BID between July 1st 2020–August 31st 2023. DFP exposure: time active in the registry. The frequency of adverse events (AEs) and serious AEs (SAEs) by Medical Dictionary for Regulatory Activities (MedDRA) Preferred Term was assessed.

Findings or outcomes:
A total of 425 patients were referred to the Ferriprox® Total Care Registry. Primary diagnoses included “thalassemia” (n=133, 31.3%), “SCD” (n=197, 46.4%), “other anemias” (n=30, 7.1%), and “other indications” (n=65, 15.3%). Overall, 61 patients (14.4%) were < 18 years of age. An estimated total exposure of 839 patient-years was observed in 412 patients who received ≥1 month DFP BID treatment. In total, 1250 AEs and 437 SAEs were reported. All reports of agranulocytosis (2 events) and neutropenia (7 events) recovered. None of the 33 fatal outcomes reported were assessed as possibly or probably related to DFP treatment.

DFP BID is well-tolerated in real-world clinical practice in patients with thalassemia, SCD, or other anemias, with no new safety concerns identified compared to DFP TID. Limitations associated with real-world data collection must be acknowledged.