Iron chelation therapy in transfusion-dependent thalassemia patients: current strategies and future directions

Saliba, Antoine N.; Harb, Afif; Taher, Alì

doi:10.2147/jbm.s72463

Cited by 47 publications

(33 citation statements)

References 89 publications

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“…In large randomized studies on TM, DFX has been shown to have an efficacy similar to DFO with regard to the reduction in liver iron concentration and serum ferritin levels ( 14 , 15 ). Nevertheless, some patients fail to respond adequately to monotherapy with any of these agents ( 16 ). Comparative effectiveness trials may help to determine the ideal strategy for treating various scenarios of organ-specific iron loading.…”

Section: Introductionmentioning

confidence: 99%

Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine

Hassan¹,

Tolba²

2016

Electron physician

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IntroductionIron overload is the primary cause of mortality and morbidity in thalassemia major (TM) despite advances in chelation therapy. The aim of this study was to compare the effectiveness and safety of deferasirox (DFX) and deferoxamine (DFO) as iron-chelating agents in patients with transfusion-dependent β-thalassemia major.MethodsThis prospective randomized study included 60 patients with transfusion-dependent β-TM during the period from September 2014 to September 2015. Their ages were ≥ 6 years, and they had serum ferritin above 1500 μg/L and were on irregular DFO therapy. Patients had regular packed red cell transfusion in a dose of 10 mL/kg/session. They were randomized to receive DFX (single oral daily dose of 20–40 mg/kg/day) or DFO (20–50 mg/kg/day via subcutaneous infusion over 8–10 hours, 5 days a week). Iron overload was determined by serum ferritin level. The primary endpoint was decrease of serum ferritin level below 1500 μg/L. The secondary endpoint was drug safety.ResultsBoth drugs significantly reduced serum ferritin (p < 0.001). At the end of follow-up, there were no significant differences between the two groups in serum ferritin levels (p = 0.673) and in percent reduction of ferritin (p = 0.315). There were no significant differences between the two groups in the total amount of blood transfusion (p = 0.166) and average iron intake (p = 0.227). There were no mortalities or any serious adverse effects, neutropenia, arthropathy, or pulmonary toxicity. Gastrointestinal upset and skin rash occurred more frequently with DFX than with DFO (p = 0.254 and 0.095, respectively).ConclusionWith appropriate dosing and compliance with drugs, both DFX and DFO are generally well tolerated, safe, and effective in reducing serum ferritin levels in iron-overloaded, regularly-transfused thalassemia major patients. Therefore, oral DFX is recommended for more convenience and adherence to the treatment regimen.

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Section: Introductionmentioning

confidence: 99%

Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine

Hassan¹,

Tolba²

2016

Electron physician

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“…Patients with transfusional iron overload usually require iron chelation therapy (ICT) to help decrease the iron burden and to prevent and/or delay long-term complications associated with iron deposition in tissues [14]. There are currently three available iron chelators.…”

Section: New Formulation Of Iron Chelatorsmentioning

confidence: 99%

“…The dose ranges from 20 to 50 mg/kg/day. Though it is an effective drug, limited compliance was reported due to the inconvenience of parenteral administration as well as infectious complications [14].…”

Section: Deferoxaminementioning

confidence: 99%

Updates in Thalassemia

Hassan¹,

Zakaria²

2020

Beta Thalassemia

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Beta-thalassemia is a genetic disease caused by mutations in the β-globin gene, resulting in partial or complete deficiency of β-chain. Deficiency of β-chain was accompanied by excess unmatched α-globin chains with subsequent dyserythropoiesis, oxidative stress, and chronic anemia. The main therapeutic option is blood transfusion that improves the anemic status but unfortunately exacerbates iron overload status. Till now, the only curative measure is allo-hematopoietic stem cell transplantation. New diagnostic and therapeutic modalities are now available. These include the preimplantation genetic diagnosis and new tools in the assessment of iron overload. Also, new therapeutic options aimed at different targets are being developed; for example, therapies that stimulate the synthesis of γ-globin and reduce the synthesis of α-globin, as well as the iron excess, dyserythropoiesis, and oxidative stress. However, the most likely ideal approach is efficient prevention, through genetic counseling, carrier detection, and prenatal diagnosis.

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“…Briefly, it has been assessed that DFX at a dose of 20 mg/kg per day can stabilize serum ferritin levels and LIC, while at doses of 30–40 mg/kg per day is able to reduce these parameters and achieve negative iron balance in patients with transfusional iron overload. 36 …”

Section: Chelation In Ntdtmentioning

confidence: 99%

Profile of deferasirox for the treatment of patients with non-transfusion-dependent thalassemia syndromes

Ricchi¹,

Marsella²

2015

DDDT

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It has been clearly shown that iron overload adds progressively significant morbidity and mortality in patients with non-transfusion-dependent thalassemia (NTDT). The lack of physiological mechanisms to eliminate the excess of iron requires effective iron chelation therapy. The reduced compliance to deferoxamine and the risk of severe hematological adverse events during deferiprone treatment have limited the use of both these drugs to correct iron imbalance in NTDT. According to the principles of evidence-based medicine, following the demonstration of the effectiveness and the safety of deferasirox (Exjade®) in a prospective, randomized, controlled trial, deferasirox was approved by the US Food and Drug Administration in May 2013 for the treatment of iron overload associated with NTDT. This review, assessing the available scientific literature, will focus on the profile of DFX in the treatment of non-transfusional hemosiderosis in patients with NTDT.

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Iron chelation therapy in transfusion-dependent thalassemia patients: current strategies and future directions

Cited by 47 publications

References 89 publications

Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine

Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine

Updates in Thalassemia

Profile of deferasirox for the treatment of patients with non-transfusion-dependent thalassemia syndromes

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