Proton pump inhibition for secondary hemochromatosis in hereditary anemia, a phase III placebo-controlled randomized cross-over clinical trial.

Iron overload is a severe general complication of hereditary anemias. Treatment with iron chelators is hampered by important side-effects, high costs and the lack of availability in many countries with high prevalence of hereditary anemias. Objective: to investigate the safety and efficacy of esomeprazole in treatment of iron overload in patients with non-transfusion-dependent hereditary anemias. Design: phase III randomized placebo-controlled cross-over trial. Setting: enrollment from March 2018 through April 2019 in five centers of expertise in the Netherlands. Participants: 30 adult patients with mild-to-moderate iron overload were enrolled (non-transfusion-dependent thalassemia (N=13), pyruvate kinase deficiency (N=8), congenital sideroblastic anemia (N=3), congenital dyserythropoietic anemia (N=3), sickle cell disease (N=2), and hereditary elliptocytosis (N=1)). Intervention: esomeprazole 40mg BID and placebo for 12 months in a cross-over design. Primary outcome: change of liver iron content (LIC) measured by MRI.