I’m Roberto’s dog, Teddy. I don’t get walked or played with as often on days when Roberto is gone a long time at the infusion center. Despite his current C5i treatment, many PNH patients like Roberto still require regular blood transfusions.1,‡
Based on a patient survey (N=122), many patients treated with a C5i remained anemic and were unable to achieve Hb normalization, despite displaying Hb stabilization.1,2
Ongoing transfusion dependence1
In patients who had a lifetime history of a transfusion
and were on C5i treatment for at least 1 year:
Possible limitations of the survey included1:
- A small sample size
- Selection bias from patients dissatisfied with their current C5i therapy
- Subjectivity of patient-reported outcomes
- Convenience sampling methodology rendering results not generalizable to total population
C5i=complement component 5 inhibitor.
‡US patient-reported data from a survey investigating the symptom burden of PNH in patients (N=122) currently treated with C5 inhibitors eculizumab or ravulizumab to determine possible unmet needs within PNH treatment. Study results were presented at the 2021 International Society for Pharmacoeconomics and Outcomes Research Europe (ISPOR-EU) Virtual Conference.1
References: 1. Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol. 2022;101(2):251-263. 2. Risitano AM, Marotta S, Ricci P, et al. Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT. Front Immunol. 2019;10:1157.