{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,29]],"date-time":"2026-08-29T04:59:17Z","timestamp":1787979557664,"version":"build-2784847793"},"reference-count":26,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2024,3,1]],"date-time":"2024-03-01T00:00:00Z","timestamp":1709251200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2024,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>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\u2010established safety and efficacy profile that is indicated for the treatment of transfusional iron overload. Here, we report safety data from the large\u2010scale, retrospective Ferriprox\u00ae 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\u2009years). In the overall patient population (<jats:italic>N<\/jats:italic>\u2009=\u2009634), 64.7% (<jats:italic>n<\/jats:italic>\u2009=\u2009410) of patients reported a total of 1885 adverse events (AEs). In subgroup analyses, 54.6% (<jats:italic>n<\/jats:italic>\u2009=\u200971) of pediatric patients and 67.3% (<jats:italic>n<\/jats:italic>\u2009=\u2009339) 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 &lt;0.5\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/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\u2009years of real\u2010world data collected in the Ferriprox\u00ae Total Care Registry demonstrate that deferiprone is safe and well tolerated in patients with SCD or other anemias who have transfusional iron overload.<\/jats:p>","DOI":"10.1002\/ajh.27276","type":"journal-article","created":{"date-parts":[[2024,3,2]],"date-time":"2024-03-02T06:13:15Z","timestamp":1709359995000},"page":"1031-1039","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Real\u2010world evidence: Long\u2010term safety of deferiprone in a large cohort of patients with sickle cell disease enrolled in a registry for up to 10\u2009years"],"prefix":"10.1002","volume":"99","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7103-3406","authenticated-orcid":false,"given":"Janet L.","family":"Kwiatkowski","sequence":"first","affiliation":[{"name":"Division of Hematology The Children's Hospital of Philadelphia  Philadelphia Pennsylvania USA"},{"name":"Department of Pediatrics Perelman School of Medicine of the University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexis A.","family":"Thompson","sequence":"additional","affiliation":[{"name":"Division of Hematology The Children's Hospital of Philadelphia  Philadelphia Pennsylvania USA"},{"name":"Department of Pediatrics Perelman School of Medicine of the University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fernando","family":"Tricta","sequence":"additional","affiliation":[{"name":"Chiesi Canada Corporation  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Noemi Toiber","family":"Temin","sequence":"additional","affiliation":[{"name":"Chiesi Canada Corporation  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Rozova","sequence":"additional","affiliation":[{"name":"Chiesi Canada Corporation  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Fradette","sequence":"additional","affiliation":[{"name":"Chiesi Canada Corporation  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4739-265X","authenticated-orcid":false,"given":"Sherif M.","family":"Badawy","sequence":"additional","affiliation":[{"name":"Division of Hematology, Oncology and Stem Cell Transplantation Ann &amp; Robert H. Lurie Children's Hospital of Chicago  Chicago Illinois USA"},{"name":"Department of Pediatrics Northwestern University Feinberg School of Medicine  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,3]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/asheducation-2013.1.447"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.14575"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0037-1963(01)90058-7"},{"key":"e_1_2_11_5_1","volume-title":"FERRIPROX\u00ae (Deferiprone) Tablets, for Oral Use Ed","author":"FERRIPROX\u00ae (deferiprone) Tablets, for oral use","year":"2021"},{"key":"e_1_2_11_6_1","unstructured":"Chiesi global rare diseases announces FDA approval of FERRIPROX\u00ae (deferiprone) for treatment of transfusional iron overload due to sickle cell disease [Press release]. PR Newswire2021."},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2021004938"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2021006778"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(20)30100-9"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1080\/17474086.2023.2178409"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.17999"},{"issue":"5","key":"e_1_2_11_12_1","first-page":"795","article-title":"Deferiprone in the treatment of transfusion\u2010dependent thalassemia: a review and perspective","volume":"3","author":"Galanello R","year":"2007","journal-title":"Ther Clin Risk Manag"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1080\/17474086.2017.1318052"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.2147\/JMDH.S160029"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.23970\/AHRQEPCREGISTRIES4"},{"key":"e_1_2_11_16_1","unstructured":"International Council for Harmonisation of technical requirements for Pharmaceuticals for Human use [ICH].Medical Dictionary for Regulatory Activities [MedDRA].2022https:\/\/www.meddra.org\/Accessed June 20th 2022."},{"key":"e_1_2_11_17_1","unstructured":"What is a serious adverse event?https:\/\/www.fda.gov\/safety\/reporting-serious-problems-fda\/what-serious-adverse-event. Accessed March 8 2023"},{"key":"e_1_2_11_18_1","volume-title":"FERRIPROX\u00ae (Deferiprone) Oral Solution, for Oral Use Ed","author":"FERRIPROX\u00ae (deferiprone) Oral Solution, for Oral Use","year":"2021"},{"key":"e_1_2_11_19_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2019-122062"},{"key":"e_1_2_11_20_1","doi-asserted-by":"publisher","DOI":"10.1177\/2040620712450252"},{"key":"e_1_2_11_21_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2011.047365"},{"key":"e_1_2_11_22_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0214355"},{"key":"e_1_2_11_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ijpam.2020.12.005"},{"key":"e_1_2_11_24_1","volume-title":"Novartis Pharmaceuticals Corporation","author":"EXJADE (Deferasirox) Tablets, for Oral Suspension","year":"2020"},{"key":"e_1_2_11_25_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2011.08720.x"},{"key":"e_1_2_11_26_1","article-title":"Deferasirox for managing transfusional iron overload in people with sickle cell disease","volume":"5","author":"Meerpohl JJ","year":"2014","journal-title":"Cochrane Database Syst Rev"},{"key":"e_1_2_11_27_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.24479"}],"container-title":["American Journal of Hematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ajh.27276","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,5,15]],"date-time":"2024-05-15T11:55:25Z","timestamp":1715774125000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ajh.27276"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,3]]},"references-count":26,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2024,6]]}},"alternative-id":["10.1002\/ajh.27276"],"URL":"https:\/\/doi.org\/10.1002\/ajh.27276","archive":["Portico"],"relation":{},"ISSN":["0361-8609","1096-8652"],"issn-type":[{"value":"0361-8609","type":"print"},{"value":"1096-8652","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,3]]},"assertion":[{"value":"2023-11-27","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-02-19","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-03-01","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}