PMID- 38429922
OWN - NLM
STAT- Publisher
LR  - 20240302
IS  - 1096-8652 (Electronic)
IS  - 0361-8609 (Linking)
DP  - 2024 Mar 1
TI  - Real-world evidence: Long-term safety of deferiprone in a large cohort of 
      patients with sickle cell disease enrolled in a registry for up to 10 years.
LID - 10.1002/ajh.27276 [doi]
AB  - Patients with sickle cell disease (SCD) and other anemias who receive blood 
      transfusions are at risk of organ damage due to transfusional iron overload. 
      Deferiprone is an iron chelator with a well-established safety and efficacy 
      profile that is indicated for the treatment of transfusional iron overload. Here, 
      we report safety data from the large-scale, retrospective Ferriprox(R) Total Care 
      Registry, which involved all patients with SCD taking deferiprone following the 
      2011 approval of deferiprone in the United States through August 2020. A total of 
      634 patients who had initiated deferiprone treatment were included. The mean (SD) 
      duration of deferiprone exposure in the registry was 1.6 (1.6) years (range 0 to 
      9.7 years). In the overall patient population (N = 634), 64.7% (n = 410) of 
      patients reported a total of 1885 adverse events (AEs). In subgroup analyses, 
      54.6% (n = 71) of pediatric patients and 67.3% (n = 339) of adult patients 
      reported AEs. The most common AEs reported in patients receiving deferiprone were 
      sickle cell crisis (22.7%), nausea (12.1%), vomiting (8.7%), abdominal discomfort 
      (5.4%), and fatigue (5.4%). Neutropenia was reported in four (0.6%) patients and 
      severe neutropenia/agranulocytosis (defined as absolute neutrophil count 
      <0.5 x 10(9) /L) was reported in two (0.3%) patients. Of patients with evaluable 
      data, all cases of neutropenia and severe neutropenia/agranulocytosis resolved 
      with deferiprone discontinuation. Results from the nearly 10 years of real-world 
      data collected in the Ferriprox(R) Total Care Registry demonstrate that deferiprone 
      is safe and well tolerated in patients with SCD or other anemias who have 
      transfusional iron overload.
CI  - (c) 2024 Wiley Periodicals LLC.
FAU - Kwiatkowski, Janet L
AU  - Kwiatkowski JL
AUID- ORCID: 0000-0001-7103-3406
AD  - Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, 
      Pennsylvania, USA.
AD  - Department of Pediatrics, Perelman School of Medicine of the University of 
      Pennsylvania, Philadelphia, Pennsylvania, USA.
FAU - Thompson, Alexis A
AU  - Thompson AA
AD  - Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, 
      Pennsylvania, USA.
AD  - Department of Pediatrics, Perelman School of Medicine of the University of 
      Pennsylvania, Philadelphia, Pennsylvania, USA.
FAU - Tricta, Fernando
AU  - Tricta F
AD  - Chiesi Canada Corporation, Toronto, Ontario, Canada.
FAU - Temin, Noemi Toiber
AU  - Temin NT
AD  - Chiesi Canada Corporation, Toronto, Ontario, Canada.
FAU - Rozova, Anna
AU  - Rozova A
AD  - Chiesi Canada Corporation, Toronto, Ontario, Canada.
FAU - Fradette, Caroline
AU  - Fradette C
AD  - Chiesi Canada Corporation, Toronto, Ontario, Canada.
FAU - Badawy, Sherif M
AU  - Badawy SM
AUID- ORCID: 0000-0002-4739-265X
AD  - Division of Hematology, Oncology and Stem Cell Transplantation, Ann & Robert H. 
      Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.
AD  - Department of Pediatrics, Northwestern University Feinberg School of Medicine, 
      Chicago, Illinois, USA.
LA  - eng
GR  - Chiesi Global Rare Diseases (formerly ApoPharma Inc.)/
PT  - Journal Article
DEP - 20240301
PL  - United States
TA  - Am J Hematol
JT  - American journal of hematology
JID - 7610369
SB  - IM
EDAT- 2024/03/02 12:45
MHDA- 2024/03/02 12:45
CRDT- 2024/03/02 01:22
PHST- 2024/02/16 00:00 [revised]
PHST- 2023/11/27 00:00 [received]
PHST- 2024/02/19 00:00 [accepted]
PHST- 2024/03/02 12:45 [medline]
PHST- 2024/03/02 12:45 [pubmed]
PHST- 2024/03/02 01:22 [entrez]
AID - 10.1002/ajh.27276 [doi]
PST - aheadofprint
SO  - Am J Hematol. 2024 Mar 1. doi: 10.1002/ajh.27276.