People with paroxysmal nocturnal hemoglobinuria (PNH) taking eculizumab or ravulizumab experience improvements in quality of life, according to research in Orphanet Journal of Rare Diseases.
Eculizumab is a complement component-5 (C5) inhibitor and has been the standard of care for PNH since 2007. It is given in biweekly doses. Ravulizumab is a newer C5 inhibitor, with an 8-week dosing interval, making it a little easier for patients.
Clinical trials have found ravulizumab to be non-inferior to eculizumab. The study evaluated the quality of life of patients with PNH when treated with eculizumab or ravulizumab. The researchers measured the European Organisation for Research and Treatment of Cancer (EORTC)—QLQ-C30 Global Health Status/QOL score after 26 weeks of treatment.
The researchers also investigated response-shift effects, which relates to how a person with chronic illness evaluates their quality of life over time. This may be different than how the general population evaluates their quality of life.
The study included 441 people with PNH from previous trials of both drugs. The EORTC general population sample included 15,386 people.
Patients taking either drug for 26 weeks reported better physical, emotional, and cognitive functioning, and lower nausea/vomiting, pain, insomnia, appetite loss, constipation, and diarrhea than the general population sample.
Patients taking ravulizumab had higher global health status and quality of life, as well as lower fatigue and less financial difficulty than the general population. For most measures, the patients taking ravulizumab had higher effect sizes.
The study authors also evaluated quality of life scores at baseline. Patients with PNH had worse function and symptoms scores before treatment than the general population.
Overall, the researchers found that both treatments for PNH improved patients’ quality of life. Over time, patients with PNH were less likely to endorse, or give lower scores, for poor health.
“These recalibration and reprioritization effects reflect adaptive response shifts,” the authors wrote in their report. “This pattern of responses suggests that ravulizumab and eculizumab enabled patients not only to achieve a better QOL but also to adapt to their condition.”
Disclosure: One or more study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference fo ra full list of authors’ disclosures.
Schwartz CE, Stark RB, Borowiec K, Nolte S, Myren KJ. Norm-based comparison of the quality-of-life impact of ravulizumab and eculizumab in paroxysmal nocturnal hemoglobinuria. Orphanet J Rare Dis. 2021;16(1):389. doi:10.1186/s13023-021-02016-8