Real-world experience with iron chelation therapy in transfusion-dependent thalassemia: impact of the oral chelators’ era

Iron overload is a common complication in patients with transfusion-dependent-thalassemia that can lead to end-organ damage. Management of iron overload has considerably evolved since the early 2000s with the approval of oral iron chelators and widespread use of MRI monitoring. We conducted a retrospective cohort study of 144 patients with transfusion-dependent-thalassemia treated at a single center in the US and followed since initiation of regular transfusion therapy. Patients who were receiving deferoxamine monotherapy and then switched to/added an oral chelator had a mean decrease in liver iron concentration (LIC) by 0.02 mg/g dry weight (dw) per month (0.24 mg/g dw per year) and a mean increase in cardiac T2* by 0.07 ms per month (1.68 ms per year) after starting an iron chelator (p < 0.001 for both). There was a statistically significant decrease in the proportion of patients with clinically-relevant cardiac iron overload (cardiac T2* < 20 ms and < 10 ms) from 2006–2010 to 2016–2020, with a trend towards a decrease in the proportion of patients with clinically-relevant hepatic iron overload (LIC > 15 mg/g dw). The introduction of oral chelators has transformed management in patients with transfusion-dependent thalassemia and led to persistent improvements in iron burden over the years.