Trial reportAnnals of hematology2013
Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients: 1-year extension results from the THALASSA study.
Trial report in Annals of hematology, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 2 of them syntheses that pooled it.
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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
28 citing papers in PubMed, 2 syntheses or guidelines pooled it, 72 citations in OpenAlex.
- [Guidelines for iron chelation therapy in thalassemia in China (2025)].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2025Guideline
- Deferasirox for managing iron overload in people with thalassaemia.The Cochrane database of systematic reviews · 2017Pooled it
- Proton pump inhibition for secondary hemochromatosis in hereditary anemia: a phase III placebo-controlled randomized cross-over clinical trial.American journal of hematology · 2022Trial
- Efficacy and safety of deferasirox in non-thalassemic patients with elevated ferritin levels after allogeneic hematopoietic stem cell transplantation.Bone marrow transplantation · 2016Trial
- Approaching low liver iron burden in chelated patients with non-transfusion-dependent thalassemia: the safety profile of deferasirox.European journal of haematology · 2014Trial
- Bridging Pharmacokinetics and Pharmacodynamics: A PBPK/PD Model-Based Approach for Deferasirox Dosing in Transfusion-Dependent Thalassemia.Clinical and translational science · 2025Article
- Cardiac injury caused by iron overload in thalassemia.Frontiers in pediatrics · 2025Review
- Article
- Non-Transfusion-Dependent Thalassemia: A Panoramic Review.Medicina (Kaunas, Lithuania) · 2022Review
- Thalassemia Intermedia: Chelator or Not?International journal of molecular sciences · 2022Review
- The Oral Ferroportin Inhibitor VIT-2763 Improves Erythropoiesis without Interfering with Iron Chelation Therapy in a Mouse Model of β-Thalassemia.International journal of molecular sciences · 2021Article
- Evaluation of endocrine complications in beta-thalassemia intermedia (β-TI): a cross-sectional multicenter study.Endocrine · 2020Article
- Final Height and Endocrine Complications in Patients with β-Thalassemia Intermedia: Our Experience in Non-Transfused Versus Infrequently Transfused Patients and Correlations with Liver Iron Content.Mediterranean journal of hematology and infectious diseases · 2019Article
- Non-Transfusion-Dependent Thalassemia: An Update on Complications and Management.International journal of molecular sciences · 2018Review
- Deferasirox: Over a Decade of Experience in Thalassemia.Mediterranean journal of hematology and infectious diseases · 2018Review
- Iron Overload and Chelation Therapy in Non-Transfusion Dependent Thalassemia.International journal of molecular sciences · 2017Review
- Iron overload in thalassemia: different organs at different rates.Hematology. American Society of Hematology. Education Program · 2017Review
- Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.Advances in experimental medicine and biology · 2017Review
- Current Standards of Care and Long Term Outcomes for Thalassemia and Sickle Cell Disease.Advances in experimental medicine and biology · 2017Review
- Efficacy and safety of iron-chelation therapy with deferoxamine, deferiprone, and deferasirox for the treatment of iron-loaded patients with nontransfusion-dependent thalassemia syndromes.Drug design, development and therapy · 2016Article
Corrections and comments
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Authors and funding
14 authors at 11 institutions in 8 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Patients with non-transfusion-dependent thalassemia (NTDT) often develop iron overload that requires chelation to levels below the threshold associated with complications. This can take several years in patients with high iron burden, highlighting the value of long-term chelation data. Here, we report the 1-year extension of the THALASSA trial assessing deferasirox in NTDT; patients continued with deferasirox or crossed from placebo to deferasirox. Of 133 patients entering extension, 130 completed. Liver iron concentration (LIC) continued to decrease with deferasirox over 2 years; mean change was -7.14 mg Fe/g dry weight (dw) (mean dose 9.8 ± 3.6 mg/kg/day). In patients originally randomized to placebo, whose LIC had increased by the end of the core study, LIC decreased in the extension with deferasirox with a mean change of -6.66 mg Fe/g dw (baseline to month 24; mean dose in extension 13.7 ± 4.6 mg/kg/day). Of 166 patients enrolled, 64 (38.6 %) and 24 (14.5 %) patients achieved LIC <5 and <3 mg Fe/g dw by the end of the study, respectively. Mean LIC reduction was greatest in patients with the highest pretreatment LIC. Deferasirox progressively decreases iron overload over 2 years in NTDT patients with both low and high LIC. Safety profile of deferasirox over 2 years was consistent with that in the core study.
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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.