Deferasirox for managing iron overload in people with thalassaemia

Abstract

Deferasirox offers an important treatment option for people with thalassaemia and secondary iron overload. Based on the available data, deferasirox does not seem to be superior to deferoxamine at the usually recommended ratio of 1 mg of deferasirox to 2 mg of deferoxamine. However, similar efficacy seems to be achievable depending on the dose and ratio of deferasirox compared to deferoxamine. Whether this will result in similar efficacy and will translate to similar benefits in the long term, as has been shown for deferoxamine, needs to be confirmed. Data from randomised controlled trials on rare toxicities and long-term safety are still limited. However, after a detailed discussion of the potential benefits and risks, deferasirox could be offered as the first-line option to individuals who show a strong preference for deferasirox, and may be a reasonable treatment option for people showing an intolerance or poor adherence to deferoxamine.

Recent Advances in Thalassemia Management: From Curative Therapies to Artificial Intelligence

“…Similarly, deep-learning models have been reported for automated LIC staging from multi-echo liver MRI [14]. HippoNet, a convolutional neural network framework, demonstrated strong performance for classifying iron burden categories and supporting standardized interpretation across sites [15,16]. In conventional practice, MRI-based LIC estimation using R2* relaxometry often relies on manual region-of-interest (ROI) placement, and variability can arise from differences in ROI strategy (e.g., multiple small ROIs versus whole-liver ROI); whole-liver ROI approaches have been reported to provide more consistent estimates in some settings [17].…”

Recent Advances in Thalassemia Management: From Curative Therapies to Artificial Intelligence

“…Similarly, deep-learning models have been reported for automated LIC staging from multi-echo liver MRI [14]. HippoNet, a convolutional neural network framework, demonstrated strong performance for classifying iron burden categories and supporting standardized interpretation across sites [15,16]. In conventional practice, MRI-based LIC estimation using R2* relaxometry often relies on manual region-of-interest (ROI) placement, and variability can arise from differences in ROI strategy (e.g., multiple small ROIs versus whole-liver ROI); whole-liver ROI approaches have been reported to provide more consistent estimates in some settings [17].…”

Cost-utility of new film-coated tablet formulation of deferasirox vs deferoxamine among major beta-thalassemia patients in Iran

“…One of these studies is a clinical trial comparing the Jadenu with Exjade [ 15 ] and another study is a systematic review that compares deferasirox with Desferal. [ 9 ] In fact, there have not been any studies comparing the efficacy of Jadenu with Desferal. For this reason, due to the similar efficacy of Exjade and Jadenu, we used the systematic review comparing the overall efficacy of Desferal with deferasirox.…”

“…However, after discussing the potential risks and benefits, deferasirox can be suggested as a first-line treatment option for people who tend to or who have low compliance to deferoxamine. [ 9 ]…”

Yeast Chemogenomic Profiling Reveals Iron Chelation To Be the Principle Cell Inhibitory Mode of Action of Gossypol

“…Next, we queried our in-house HIP HOP database comprising profiles of ∼4000 diverse chemical compounds and checked for closely related chemogenomic profiles. The HOP profile of gossypol clustered best with its own replicate (Pearson correlation value of 0.82) followed by the HOP profile of the cation chelator drug deferasirox which is approved for use in iron overload 37,38 (0.77) and the natural iron chelator desferricoprogen 39 (0.71) (see Figure 2). Performing a pairwise comparison between gossypol and deferasirox or desferricoprogen (Figure 1E,F) revealed not only a conserved set of hypersensitive hits (FTR1, FET3, CCC2 (including the overlapping ORFs YDR269c and YDR271c) and GEF1 but also a highly conserved sequence of hypersensitivity: FTR1 and FET3 were the most hypersensitive and GEF1 was the least with the three CCC2 associated ORFs distributed in the middle.…”

Recent Advances in Thalassemia Management: From Curative Therapies to Artificial Intelligence

“…Similarly, deep-learning models have been reported for automated LIC staging from multi-echo liver MRI [14]. HippoNet, a convolutional neural network framework, demonstrated strong performance for classifying iron burden categories and supporting standardized interpretation across sites [15,16]. In conventional practice, MRI-based LIC estimation using R2* relaxometry often relies on manual region-of-interest (ROI) placement, and variability can arise from differences in ROI strategy (e.g., multiple small ROIs versus whole-liver ROI); whole-liver ROI approaches have been reported to provide more consistent estimates in some settings [17].…”

Cost-utility of new film-coated tablet formulation of deferasirox vs deferoxamine among major beta-thalassemia patients in Iran

“…One of these studies is a clinical trial comparing the Jadenu with Exjade [ 15 ] and another study is a systematic review that compares deferasirox with Desferal. [ 9 ] In fact, there have not been any studies comparing the efficacy of Jadenu with Desferal. For this reason, due to the similar efficacy of Exjade and Jadenu, we used the systematic review comparing the overall efficacy of Desferal with deferasirox.…”

“…However, after discussing the potential risks and benefits, deferasirox can be suggested as a first-line treatment option for people who tend to or who have low compliance to deferoxamine. [ 9 ]…”

Yeast Chemogenomic Profiling Reveals Iron Chelation To Be the Principle Cell Inhibitory Mode of Action of Gossypol

“…Next, we queried our in-house HIP HOP database comprising profiles of ∼4000 diverse chemical compounds and checked for closely related chemogenomic profiles. The HOP profile of gossypol clustered best with its own replicate (Pearson correlation value of 0.82) followed by the HOP profile of the cation chelator drug deferasirox which is approved for use in iron overload 37,38 (0.77) and the natural iron chelator desferricoprogen 39 (0.71) (see Figure 2). Performing a pairwise comparison between gossypol and deferasirox or desferricoprogen (Figure 1E,F) revealed not only a conserved set of hypersensitive hits (FTR1, FET3, CCC2 (including the overlapping ORFs YDR269c and YDR271c) and GEF1 but also a highly conserved sequence of hypersensitivity: FTR1 and FET3 were the most hypersensitive and GEF1 was the least with the three CCC2 associated ORFs distributed in the middle.…”

Recent Advances in Thalassemia Management: From Curative Therapies to Artificial Intelligence

“…Similarly, deep-learning models have been reported for automated LIC staging from multi-echo liver MRI [14]. HippoNet, a convolutional neural network framework, demonstrated strong performance for classifying iron burden categories and supporting standardized interpretation across sites [15,16]. In conventional practice, MRI-based LIC estimation using R2* relaxometry often relies on manual region-of-interest (ROI) placement, and variability can arise from differences in ROI strategy (e.g., multiple small ROIs versus whole-liver ROI); whole-liver ROI approaches have been reported to provide more consistent estimates in some settings [17].…”

Cost-utility of new film-coated tablet formulation of deferasirox vs deferoxamine among major beta-thalassemia patients in Iran

“…One of these studies is a clinical trial comparing the Jadenu with Exjade [ 15 ] and another study is a systematic review that compares deferasirox with Desferal. [ 9 ] In fact, there have not been any studies comparing the efficacy of Jadenu with Desferal. For this reason, due to the similar efficacy of Exjade and Jadenu, we used the systematic review comparing the overall efficacy of Desferal with deferasirox.…”

“…However, after discussing the potential risks and benefits, deferasirox can be suggested as a first-line treatment option for people who tend to or who have low compliance to deferoxamine. [ 9 ]…”

Yeast Chemogenomic Profiling Reveals Iron Chelation To Be the Principle Cell Inhibitory Mode of Action of Gossypol

“…Next, we queried our in-house HIP HOP database comprising profiles of ∼4000 diverse chemical compounds and checked for closely related chemogenomic profiles. The HOP profile of gossypol clustered best with its own replicate (Pearson correlation value of 0.82) followed by the HOP profile of the cation chelator drug deferasirox which is approved for use in iron overload 37,38 (0.77) and the natural iron chelator desferricoprogen 39 (0.71) (see Figure 2). Performing a pairwise comparison between gossypol and deferasirox or desferricoprogen (Figure 1E,F) revealed not only a conserved set of hypersensitive hits (FTR1, FET3, CCC2 (including the overlapping ORFs YDR269c and YDR271c) and GEF1 but also a highly conserved sequence of hypersensitivity: FTR1 and FET3 were the most hypersensitive and GEF1 was the least with the three CCC2 associated ORFs distributed in the middle.…”