Trial reportJournal of cardiovascular magnetic resonance : official journal of the Society for Cardiovascular Magnetic Resonance2013
Treatment of heart failure in adults with thalassemia major: response in patients randomised to deferoxamine with or without deferiprone.
Trial report in Journal of cardiovascular magnetic resonance : official journal of the Society for Cardiovascular Magnetic Resonance, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 2 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
20 citing papers in PubMed, 2 syntheses or guidelines pooled it, 54 citations in OpenAlex.
- Efficacy and safety of deferiprone for thalassemia: a systematic review and meta-analysis of randomized controlled trials.Systematic reviews · 2025Pooled it
- Cardiovascular magnetic resonance native TJournal of cardiovascular magnetic resonance : official journal of the Society for Cardiovascular Magnetic Resonance · 2020Pooled it
- Trial
- Cardiomyocyte programmed cell death in dilated cardiomyopathy: molecular crosstalk and therapeutic implications.Frontiers in cell and developmental biology · 2026Review
- Beyond transfusions and transplants: genomic innovations rewriting the narrative of thalassemia.Annals of hematology · 2025Review
- Diagnosis, treatment, and surveillance of Diamond-Blackfan anaemia syndrome: international consensus statement.The Lancet. Haematology · 2024Article
- Cardiac complications in thalassemia throughout the lifespan: Victories and challenges.Annals of the New York Academy of Sciences · 2023Review
- An evaluation of deferiprone as twice-a-day tablets or in combination therapy for the treatment of transfusional iron overload in thalassemia syndromes.Expert review of hematology · 2023Article
- SARS-CoV-2 infection in patients with β-thalassemia: The French experience.Transfusion clinique et biologique : journal de la Societe francaise de transfusion sanguine · 2022Article
- Vasculo-toxic and pro-inflammatory action of unbound haemoglobin, haem and iron in transfusion-dependent patients with haemolytic anaemias.British journal of haematology · 2021Article
- Increasing Intracellular Levels of Iron with Ferric Ammonium Citrate Leads to Reduced P-glycoprotein Expression in Human Immortalised Brain Microvascular Endothelial Cells.Pharmaceutical research · 2021Article
- Risk factors for heart disease in transfusion-dependent thalassemia: serum ferritin revisited.Internal and emergency medicine · 2019Article
- Single-center retrospective study of the effectiveness and toxicity of the oral iron chelating drugs deferiprone and deferasirox.PloS one · 2019Article
- Pathological Roles of Iron in Cardiovascular Disease.Current drug targets · 2018Review
- Iron overload in thalassemia: different organs at different rates.Hematology. American Society of Hematology. Education Program · 2017Review
- Reduction in mitochondrial iron alleviates cardiac damage during injury.EMBO molecular medicine · 2016Article
- Review of Journal of Cardiovascular Magnetic Resonance 2014.Journal of cardiovascular magnetic resonance : official journal of the Society for Cardiovascular Magnetic Resonance · 2015Review
- Review of Journal of Cardiovascular Magnetic Resonance 2013.Journal of cardiovascular magnetic resonance : official journal of the Society for Cardiovascular Magnetic Resonance · 2014Review
- Efficacy and safety of combined oral iron chelation therapy with deferasirox and deferiprone in a patient with beta-thalassemia major and persistent iron overload.Blood research · 2014Article
- Randomized controlled trials of iron chelators for the treatment of cardiac siderosis in thalassaemia major.Frontiers in pharmacology · 2014Review
Corrections and comments
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Authors and funding
13 authors at 10 institutions in 4 countries.
Funding
Abstract
backgroundEstablished heart failure in thalassaemia major has a poor prognosis and optimal management remains unclear.
methodsA 1 year prospective study comparing deferoxamine (DFO) monotherapy or when combined with deferiprone (DFP) for patients with left ventricular ejection fraction (LVEF) <56% was conducted by the Thalassemia Clinical Research Network (TCRN). All patients received DFO at 50-60 mg/kg 12-24 hr/day sc or iv 7 times weekly, combined with either DFP 75 at mg/kg/day (combination arm) or placebo (DFO monotherapy arm). The primary endpoint was the change in LVEF by CMR.
resultsImprovement in LVEF was significant in both study arms at 6 and 12 months (p = 0.04), normalizing ventricular function in 9/16 evaluable patients. With combination therapy, the LVEF increased from 49.9% to 55.2% (+5.3% p = 0.04; n = 10) at 6 months and to 58.3% at 12 months (+8.4% p = 0.04; n = 7). With DFO monotherapy, the LVEF increased from 52.8% to 55.7% (+2.9% p = 0.04; n = 6) at 6 months and to 56.9% at 12 months (+4.1% p = 0.04; n = 4). The LVEF trend did not reach statistical difference between study arms (p = 0.89). In 2 patients on DFO monotherapy during the study and in 1 patient on combined therapy during follow up, heart failure deteriorated fatally. The study was originally powered for 86 participants to determine a 5% difference in LVEF improvement between treatments. The study was prematurely terminated due to slow recruitment and with the achieved sample size of 20 patients there was 80% power to detect an 8.6% difference in EF, which was not demonstrated. Myocardial T2* improved in both arms (combination +1.9 ± 1.6 ms p = 0.04; and DFO monotherapy +1.9 ± 1.4 ms p = 0.04), but with no significant difference between treatments (p = 0.65). Liver iron (p = 0.03) and ferritin (p < 0.001) both decreased significantly in only the combination group.
conclusionsBoth treatments significantly improved LVEF and myocardial T2*. Although this is the largest and only randomized study in patients with LV decompensation, further prospective evaluation is needed to identify optimal chelation management in these high-risk patients.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.