The challenges of iron chelation therapy in thalassemia: how do we overcome them?

Packed red blood cell (pRBC) transfusions are the primary treatment for thalassemia. However, chronic transfusions ultimately result in iron overload, causing heart, liver, and endocrine complications along with other comorbidities. Although iron chelation is routinely initiated to remove excess iron, adherence remains a challenge, and iron overload still contributes to significant morbidity and early mortality in thalassemia.

We review the evidence for iron overload and its complications in thalassemia. We also assess iron chelation strategies with possible adherence challenges categorized as patient-, medication-, and system-related barriers. Evidence suggests that lower adherence rates have been associated with more endorsed barriers. Further, patient-related barriers could be internal or external, and taking a patient-centered approach is key to addressing these challenges. Choosing the right iron chelator could help overcome some medication-related barriers. Finally, insurance coverage and access to specialized centers could affect initiation of iron chelation.

A critical and routine assessment of adherence barriers is key to optimizing patients' adherence to iron chelation. Adherence is often a multifactorial process, and it varies over time. Shared decision making with patients and/or caregivers is an important next step to improving adherence to iron chelation, and ultimately health outcomes.