What is your approach to managing iron overload in children with transfusion-dependent beta thalassemia who have adherence challenges or toxicity with standard chelation regimens?

I would divide the adherence issues into two populations. The younger children where a caregiver is responsible for administering the chelation, and adolescents where caregivers have passed on the responsibility to the patient. For the former, adherence is reinforced with an explanation of the possible long-term effects of iron on tissues and the resulting complications. Most caregivers take this seriously, and it is worth having them witness the actual ingestion of each dose, particularly if it...

I believe adherence and toxicity are two separate, but potentially related, issues. Chelation non-adherence (like any medication non-adherence) can result from system-level and patient-level barriers and identifying which are present guides the approach.

For instance, if system-level barriers are present, evaluating if there are ways to make these medications more affordable or accessible (e.g., talking with pharmacies to improve medication shipping, investigating if there may be rebates/copay a...