Patients with paroxysmal nocturnal hemoglobinuria (PNH) undergoing treatment with eculizumab or ravulizumab have a high illness burden and relatively low quality of life (QoL), suggesting a need for improved treatments, according to research published in Annals of Hematology.

PNH, an acquired disease defined by chronic lysis of red blood cells, is linked with a high risk of thrombosis and death. The genetic issues linked with PNH are thought to cause anemia and complement-mediated hemolysis.

Aside from anemia and thrombosis, PNH is also linked with fatigue, dyspnea, abdominal pain, chest pain, hemoglobinuria, renal insufficiency, and bone marrow failure, each of which presents individual risks for patient health and QoL. Patients who are transfusion-dependent, furthermore, tend to have overall worse QoL scores.

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C5 inhibition is a mainstay in this patient population, and many patients receive either eculizumab or ravulizumab, which may help to alleviate disease symptoms. For this cross-sectional survey-based study, researchers evaluated the clinical and economic burden of PNH among patients receiving C5 inhibition therapy.

Overall, responses from 122 patients were included, among whom 35 and 87 were receiving eculizumab and ravulizumab, respectively. In the overall cohort, the mean age was 46.8 years, the mean age at diagnosis was 37.5 years, 73% of patients were female sex, and 33.6% of patients had aplastic anemia or severe aplastic anemia.

Although all patients receiving eculizumab, and the majority (95.4%) of patients receiving ravulizumab, had been on treatment for at least 3 months, more than 80% of patients in both groups had levels of hemoglobin consistent with anemia. Most patients (eculizumab, 88.6%; ravulizumab, 74.7%) also reported fatigue.

Thrombotic events (eculizumab, 10%; ravulizumab, 23.5%) and need for transfusion (eculizumab, 52.2%; ravulizumab, 22.6%) were also reported among patients receiving treatment for at least 1 year.

Patients with PNH had below average scores on both the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30).

“Overall, our results suggest that patients with PNH receiving therapy with [eculizumab] or [ravulizumab] maintain a substantial burden of illness, indicating that there is a need for novel therapeutics in PNH treatment,” the authors wrote.

Disclosure: The study author(s) declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures. 


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. doi:10.1007/s00277-021-04715-5