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Deferiprone for transfusional iron overload in sickle cell disease and other anemias: open-label study of up to 3 years.
Elalfy, Mohsen S; Hamdy, Mona; El-Beshlawy, Amal; Ebeid, Fatma S E; Badr, Mohamed; Kanter, Julie; Inusa, Baba; Adly, Amira A M; Williams, Suzan; Kilinc, Yurdanur; Lee, David; Fradette, Caroline; Rozova, Anna; Temin, Noemi Toiber; Tricta, Fernando; Kwiatkowski, Janet L.
Afiliación
  • Elalfy MS; Pediatric Hematology Oncology Department, Faculty of Medicine, Ain Shams University, Cairo, Egypt.
  • Hamdy M; Department of Pediatrics, Faculty of Medicine, Cairo University, Cairo, Egypt.
  • El-Beshlawy A; Department of Pediatric Hematology, Pediatric Hospital of Cairo University, Cairo, Egypt.
  • Ebeid FSE; Pediatric Hematology Oncology Department, Faculty of Medicine, Ain Shams University, Cairo, Egypt.
  • Badr M; Department of Pediatrics, Faculty of Medicine, Zagazig University, Zagazig, Egypt.
  • Kanter J; Division of Hematology and Oncology, Department of Medicine, University of Alabama, Birmingham, AL.
  • Inusa B; Paediatric Haematology, Evelina Children's Hospital, Guy's and St. Thomas National Health Service Foundation Trust, London, United Kingdom.
  • Adly AAM; Pediatric Hematology Oncology Department, Faculty of Medicine, Ain Shams University, Cairo, Egypt.
  • Williams S; Department of Haematology and Oncology, The Hospital for Sick Children, University of Toronto, Toronto, Canada.
  • Kilinc Y; Department of Pediatric Hematology, Faculty of Medicine, Cukurova University, Adana, Turkey.
  • Lee D; Hematology/Immunology Program, Chiesi Canada Corporation, Toronto, Canada.
  • Fradette C; Hematology/Immunology Program, Chiesi Canada Corporation, Toronto, Canada.
  • Rozova A; Hematology/Immunology Program, Chiesi Canada Corporation, Toronto, Canada.
  • Temin NT; Hematology/Immunology Program, Chiesi Canada Corporation, Toronto, Canada.
  • Tricta F; Hematology/Immunology Program, Chiesi Canada Corporation, Toronto, Canada.
  • Kwiatkowski JL; Division of Hematology, The Children's Hospital of Philadelphia, and the Department of Pediatrics, Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA.
Blood Adv ; 7(4): 611-619, 2023 02 28.
Article en En | MEDLINE | ID: mdl-36018224
Long-term safety and efficacy data on the iron chelator deferiprone in sickle cell disease (SCD) and other anemias are limited. FIRST-EXT was a 2-year extension study of FIRST (Ferriprox in Patients With Iron Overload in Sickle Cell Disease Trial), a 1-year, randomized noninferiority study of deferiprone vs deferoxamine in these populations. Patients who entered FIRST-EXT continued to receive, or were switched to, deferiprone. Altogether, 134 patients were enrolled in FIRST-EXT (mean age: 16.2 years), with mean (SD) exposure to deferiprone of 2.1 (0.8) years over the 2 studies. The primary end point was safety. Secondary end points were change in liver iron concentration (LIC), cardiac T2∗, serum ferritin (SF), and the proportion of responders (≥20% improvement in efficacy measure). The most common adverse events considered at least possibly related to deferiprone were neutropenia (9.0%) and abdominal pain (7.5%). LIC (mg/g dry weight) decreased over time, with mean (SD) changes from baseline at each time point (year 1, -2.64 [4.64]; year 2, -3.91 [6.38]; year 3, -6.64 [7.72], all P < .0001). Mean SF levels (µg/L) decreased significantly after year 2 (-771, P = .0008) and year 3 (-1016, P = .0420). Responder rates for LIC and SF increased each year (LIC: year 1, 46.5%; year 2, 57.1%; year 3, 66.1%; SF: year 1, 35.2%; year 2, 55.2%; year 3, 70.9%). Cardiac T2∗ remained normal in all patients. In conclusion, long-term therapy with deferiprone was not associated with new safety concerns and led to continued and progressive reduction in iron load in individuals with SCD or other anemias. The trial was registered at www.clinicaltrials.gov as #NCT02443545.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Sobrecarga de Hierro / Anemia de Células Falciformes Tipo de estudio: Clinical_trials Límite: Adolescent / Humans Idioma: En Revista: Blood Adv Año: 2023 Tipo del documento: Article País de afiliación: Egipto Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Sobrecarga de Hierro / Anemia de Células Falciformes Tipo de estudio: Clinical_trials Límite: Adolescent / Humans Idioma: En Revista: Blood Adv Año: 2023 Tipo del documento: Article País de afiliación: Egipto Pais de publicación: Estados Unidos