Real-world experience with iron chelation therapy in transfusion-dependent thalassemia: Impact of the oral chelators’ era Journal Article


Authors: Pines, M.; Kleinert, D.; Thomas, C.; Mensah, C.; Musallam, K. M.; Sheth, S.
Article Title: Real-world experience with iron chelation therapy in transfusion-dependent thalassemia: Impact of the oral chelators’ era
Abstract: Iron overload is a common complication in patients with transfusion-dependent-thalassemia that can lead to end-organ damage. Management of iron overload has considerably evolved since the early 2000s with the approval of oral iron chelators and widespread use of MRI monitoring. We conducted a retrospective cohort study of 144 patients with transfusion-dependent-thalassemia treated at a single center in the US and followed since initiation of regular transfusion therapy. Patients who were receiving deferoxamine monotherapy and then switched to/added an oral chelator had a mean decrease in liver iron concentration (LIC) by 0.02 mg/g dry weight (dw) per month (0.24 mg/g dw per year) and a mean increase in cardiac T2* by 0.07 ms per month (1.68 ms per year) after starting an iron chelator (p < 0.001 for both). There was a statistically significant decrease in the proportion of patients with clinically-relevant cardiac iron overload (cardiac T2* < 20 ms and < 10 ms) from 2006–2010 to 2016–2020, with a trend towards a decrease in the proportion of patients with clinically-relevant hepatic iron overload (LIC > 15 mg/g dw). The introduction of oral chelators has transformed management in patients with transfusion-dependent thalassemia and led to persistent improvements in iron burden over the years. © The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature 2024.
Keywords: adult; controlled study; middle aged; major clinical study; nuclear magnetic resonance imaging; follow up; prevalence; cohort analysis; retrospective study; electronic medical record; liver; thalassemia; blood transfusion; heart; deferoxamine; iron chelation; deferasirox; iron overload; chelating agent; deferiprone; iron chelating agent; human; male; female; article
Journal Title: Annals of Hematology
Volume: 103
Issue: 12
ISSN: 0939-5555
Publisher: Springer  
Date Published: 2024-12-01
Start Page: 5229
End Page: 5234
Language: English
DOI: 10.1007/s00277-024-06092-1
PUBMED: 39672943
PROVIDER: scopus
DOI/URL:
Notes: The MSK Cancer Center Support Grant (P30 CA008748) is acknowledged in the PDF -- Source: Scopus
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  1. Morgan Joshua Pines
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