Limited data regarding erythrocytapheresis in children, adolescents, and young adults have been published. The aim of this study was to evaluate erythrocytapheresis, either as a standalone
therapy or in combination with
iron chelation therapy, in children and young adults with
hemoglobinopathies in whom current
iron chelation therapy is not sufficient in decreasing the
iron overload during management. We retrospectively analysed erythrocytapheresis in 19 patients with
hemoglobinopathies in need of
iron chelation therapy diagnosed with
sickle cell disease (SCD) or β-
thalassemia major. Patients were divided into (1) a case cohort who received erythrocytapheresis alone or in combination with
iron chelation therapy and (2) a control cohort who received oral
iron chelation therapy alone. Serum
ferritin and haemoglobin levels were compared at five different time points over a one-year period. In the erythrocytapheresis cohort, there was a significant decrease in serum
ferritin (p < 0.001). In the
iron chelation therapy alone cohort, there was no significant decrease in serum
ferritin over time (p = 0.156). Comparing the evolution of median serum
ferritin between
therapy with erythrocytapheresis and
iron chelation therapy showed a statistically significant difference (p = 0.008). Patients with β-
thalassemia major receiving erythrocytapheresis showed a greater reduction in serum
ferritin compared to patients without (p = 0.036). A difference could not be shown between the erythrocytapheresis and
iron chelation single
therapies (p = 0.100). This study showed an overall significant reduction in serum
ferritin in patients with
hemoglobinopathies treated with erythrocytapheresis in addition to
iron chelation. A clinical, although not statistical, trend of higher haemoglobin levels was maintained. Erythrocytapheresis in paediatric patients with β-
thalassemia major was as effective in decreasing
ferritin levels as in previously reported studies with SCD. Erythrocytapheresis is a promising
therapy for treating and preventing transfusion-related
iron overload.