Deferasirox: an orphan drug for chronic iron overload in non-transfusion dependent thalassemia syndromes

Patients with non-transfusion dependent thalassemia (NTDT) are at a significant risk of developing iron overload due to increased iron absorption from the gastrointestinal tract, resulting in a host of comorbidities. Deferasirox, the only oral iron chelator approved for use in NTDT, has been evaluated in several prospective studies and clinical trials. Areas covered: In this review, we will summarize the studies investigating the use of deferasirox in NTDT, focusing on the two largest clinical trials available thus far. THALASSA, the first prospective, randomized and placebo-controlled trial has demonstrated significant reduction in iron burden with deferasirox treatment along with a favorable side effect profile. THETIS, an ongoing clinical trial, has confirmed the findings of THALASSA and highlighted the efficacy and safety of using higher doses of deferasirox along with an earlier dose escalation protocol. Expert opinion: Longer follow-up and continued investigations will further tailor the treatment approach to iron chelation in patients with NTDT, which will likely affect their comorbidity profile and impact their quality of life scores. Since the iron chelation treatment algorithm in NTDT relies on MRI technology, further understanding of the molecular processes governing iron overload in this population may help establish diagnostic surrogates in resource limited countries in order to guide treatment with deferasirox.