Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia

Geneen, Louise J; Dorée, Carolyn; Estcourt, Lise J

doi:10.1002/14651858.cd012349.pub3

Cited by 4 publications

(3 citation statements)

References 147 publications

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“…Pharmacological interventions, such as vamiferport, a ferroportin inhibitor, have also proven effective in mitigating the severity of SCD by reducing stress erythropoiesis and the presence of circulating mitochondria-rich RBCs ( Nyffenegger et al, 2022 ). By limiting this risk, iron chelation therapy significantly improves overall patient outcome ( Meerpohl et al, 2014 , Ribeiro et al, 2019 , Geneen et al, 2023 ).…”

Section: Ferroptosis – Vaso-occlusion and Hemolysis In Scdmentioning

confidence: 99%

“…The decision to initiate therapy is based on transfusion frequency, iron deposition levels, and hepatic/cardiac dysfunction, typically starting when serum ferritin exceeds 1000–1500 µg/L or liver iron concentration surpasses 3–5 mg/g dry weight. Despite the benefits, deferiprone's risks, such as agranulocytosis and liver dysfunction, underscore the challenges of chelation therapy, often affecting patient adherence ( Ferraresi et al, 2023 , Geneen et al, 2023 ).…”

Section: Therapeutic Strategy Targeting Ferroptosis In Scdmentioning

confidence: 99%

See 1 more Smart Citation

Ferroptosis as an emerging target in sickle cell disease

Fortuna,

Lima,

Oliveira

et al. 2024

Current Research in Toxicology

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Section: Ferroptosis – Vaso-occlusion and Hemolysis In Scdmentioning

confidence: 99%

Section: Therapeutic Strategy Targeting Ferroptosis In Scdmentioning

confidence: 99%

Ferroptosis as an emerging target in sickle cell disease

Fortuna,

Lima,

Oliveira

et al. 2024

Current Research in Toxicology

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“…They are also important for considering and planning the design of new therapeutic strategies, including its potential use for the treatment of many new iron-loading and other non-iron-loading diseases, as well in medical diagnostics and other applications ( Table 1 and Table 2 ) [ 301 , 302 , 303 , 304 , 305 , 306 , 307 , 308 , 309 , 310 , 311 , 312 , 313 , 314 , 315 ]. In all these cases, a risk/benefit assessment should be considered for the use of L1 in any new disease or patient case, including different factors related to the underlying disease, the present rate of morbidity and mortality, existing treatments, and the introduction of possible combination therapies [ 316 , 317 , 318 , 319 , 320 , 321 , 322 , 323 , 324 , 325 , 326 , 327 , 328 , 329 , 330 ]. The selection of the appropriate dose protocols, the duration of treatment, and monitoring methods are also very important parameters for the assessment of L1 in such new repurposing cases.…”

Section: Future Challenges and Potential New Clinical Uses Of Deferip...mentioning

confidence: 99%

The Vital Role Played by Deferiprone in the Transition of Thalassaemia from a Fatal to a Chronic Disease and Challenges in Its Repurposing for Use in Non-Iron-Loaded Diseases

Kontoghiorghes¹

2023

Pharmaceuticals

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The iron chelating orphan drug deferiprone (L1), discovered over 40 years ago, has been used daily by patients across the world at high doses (75–100 mg/kg) for more than 30 years with no serious toxicity. The level of safety and the simple, inexpensive synthesis are some of the many unique properties of L1, which played a major role in the contribution of the drug in the transition of thalassaemia from a fatal to a chronic disease. Other unique and valuable clinical properties of L1 in relation to pharmacology and metabolism include: oral effectiveness, which improved compliance compared to the prototype therapy with subcutaneous deferoxamine; highly effective iron removal from all iron-loaded organs, particularly the heart, which is the major target organ of iron toxicity and the cause of mortality in thalassaemic patients; an ability to achieve negative iron balance, completely remove all excess iron, and maintain normal iron stores in thalassaemic patients; rapid absorption from the stomach and rapid clearance from the body, allowing a greater frequency of repeated administration and overall increased efficacy of iron excretion, which is dependent on the dose used and also the concentration achieved at the site of drug action; and its ability to cross the blood–brain barrier and treat malignant, neurological, and microbial diseases affecting the brain. Some differential pharmacological activity by L1 among patients has been generally shown in relation to the absorption, distribution, metabolism, elimination, and toxicity (ADMET) of the drug. Unique properties exhibited by L1 in comparison to other drugs include specific protein interactions and antioxidant effects, such as iron removal from transferrin and lactoferrin; inhibition of iron and copper catalytic production of free radicals, ferroptosis, and cuproptosis; and inhibition of iron-containing proteins associated with different pathological conditions. The unique properties of L1 have attracted the interest of many investigators for drug repurposing and use in many pathological conditions, including cancer, neurodegenerative conditions, microbial conditions, renal conditions, free radical pathology, metal intoxication in relation to Fe, Cu, Al, Zn, Ga, In, U, and Pu, and other diseases. Similarly, the properties of L1 increase the prospects of its wider use in optimizing therapeutic efforts in many other fields of medicine, including synergies with other drugs.

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For people with sickle cell disease or thalassemia, how do different iron chelators compare for improving adherence?

Tort¹,

Ciapponi²

2023

Cochrane Clinical Answers

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Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia

Abstract: Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia.

Cited by 4 publications

References 147 publications

Ferroptosis as an emerging target in sickle cell disease

Ferroptosis as an emerging target in sickle cell disease

The Vital Role Played by Deferiprone in the Transition of Thalassaemia from a Fatal to a Chronic Disease and Challenges in Its Repurposing for Use in Non-Iron-Loaded Diseases

For people with sickle cell disease or thalassemia, how do different iron chelators compare for improving adherence?

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