Why carry out this study?

Many patients with paroxysmal nocturnal hemoglobinuria (PNH) experience ongoing symptoms that necessitate blood transfusions even during treatment with eculizumab; however, real-world studies on the burden of transfusion-dependent (TD) eculizumab users compared to transfusion-free (TF) eculizumab users are currently lacking.

To address this knowledge gap, we conducted a retrospective, longitudinal cohort study evaluating the economic burden and treatment patterns of TD versus TF eculizumab users based on nationally representative, administrative claims data from a US population.

What was learned from this study?

TD eculizumab users, who comprised more than 36% of the overall study sample, showed evidence of greater disease severity (e.g., high rates of aplastic anemia), earlier discontinuation of eculizumab treatment, higher resource utilization, and higher direct medical costs and medical-related absenteeism costs driven by increased hospitalizations, compared to those who were TF.

The findings suggest that blood transfusion dependence may serve as an important indicator of uncontrolled disease and high economic burden among patients with PNH.

In future research investigating PNH management strategies, an improved understanding of the risks and burden among TD eculizumab users may help to inform novel therapies targeting this patient population.