Trial reportBritish journal of haematology2011
Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease.
Trial report in British journal of haematology, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 1 of them a synthesis 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
20 citing papers in PubMed, 1 synthesis or guideline pooled it, 78 citations in OpenAlex.
- Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia.The Cochrane database of systematic reviews · 2018Pooled it
- Deferiprone for transfusional iron overload in sickle cell disease and other anemias: open-label study of up to 3 years.Blood advances · 2023Trial
- Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias.Health and quality of life outcomes · 2018Trial
- Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia.The Cochrane database of systematic reviews · 2023Review
- Characterisation of individual ferritin response in patients receiving chelation therapy.British journal of clinical pharmacology · 2022Article
- The nephropathy of sickle cell trait and sickle cell disease.Nature reviews. Nephrology · 2022Review
- Emerging Mechanisms and Disease Implications of Ferroptosis: Potential Applications of Natural Products.Frontiers in cell and developmental biology · 2021Review
- Why, Who, When, and How? Rationale for Considering Allogeneic Stem Cell Transplantation in Children with Sickle Cell Disease.Journal of clinical medicine · 2019Review
- Current challenges in the management of patients with sickle cell disease - A report of the Italian experience.Orphanet journal of rare diseases · 2019Review
- Early Kidney Damage Markers after Deferasirox Treatment in Patients with Thalassemia Major: A Case-Control Study.Oxidative medicine and cellular longevity · 2019Article
- Transient Elastography (TE) is a Useful Tool for Assessing the Response of Liver Iron Chelation in Sickle Cell Disease Patients.Mediterranean journal of hematology and infectious diseases · 2018Article
- How we manage iron overload in sickle cell patients.British journal of haematology · 2017Review
- Deferasirox nephrotoxicity-the knowns and unknowns.Nature reviews. Nephrology · 2014Review
- Clinical pharmacology of deferasirox.Clinical pharmacokinetics · 2014Review
- Deferasirox for managing transfusional iron overload in people with sickle cell disease.The Cochrane database of systematic reviews · 2014Article
- Organizing national responses for rare blood disorders: the Italian experience with sickle cell disease in childhood.Orphanet journal of rare diseases · 2013Article
- Effect of deferasirox chelation on liver iron and total body iron concentration.Indian journal of pediatrics · 2013Article
- Deferasirox: appraisal of safety and efficacy in long-term therapy.Journal of blood medicine · 2013Article
- Article
- Efficacy and safety of deferasirox at low and high iron burdens: results from the EPIC magnetic resonance imaging substudy.Annals of hematology · 2013Article
Corrections and comments
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Authors and funding
17 authors at 13 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
To date, there is a lack of long-term safety and efficacy data for iron chelation therapy in transfusion-dependent patients with sickle cell disease (SCD). To evaluate the long-term safety and efficacy of deferasirox (a once-daily oral iron chelator), patients with SCD completing a 1-year, Phase II, randomized, deferoxamine (DFO)-controlled study entered a 4-year extension, continuing to receive deferasirox, or switching from DFO to deferasirox. Average actual deferasirox dose was 19·4 ± 6·3 mg/kg per d. Of 185 patients who received at least one deferasirox dose, 33·5% completed the 5-year study. The most common reasons for discontinuation were withdrawal of consent (23·8%), lost to follow-up (9·2%) and adverse events (AEs) (7·6%). Investigator-assessed drug-related AEs were predominantly gastrointestinal [including nausea (14·6%), diarrhoea (10·8%)], mild-to-moderate and transient in nature. Creatinine clearance remained within the normal range throughout the study. Despite conservative initial dosing, serum ferritin levels in patients with ≥ 4 years deferasirox exposure significantly decreased by -591 μg/l (95% confidence intervals, -1411, -280 μg/l; P = 0·027; n = 67). Long-term deferasirox treatment for up to 5 years had a clinically acceptable safety profile, including maintenance of normal renal function, in patients with SCD. Iron burden was substantially reduced with appropriate dosing in patients treated for at least 4 years.
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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.