Deferasirox and children: From clinical trials to the real world

Since its commercialization, deferasirox has become the most widely used iron chelator in children with thalassemia. Nevertheless, data on its efficacy in young patients are conflicting. In addition, several reports have linked deferasirox use with acute renal failure, Fanconi syndrome, and other tubulopathies, with a prevalence of up to 90% in small cohorts 1, 2. We retrospectively reviewed 77 thalassemia major subjects, who received at least 1 dose of deferasirox before age 18 at Ospedale Microcitemico, Cagliari (Italy).