Background: Terminal complement inhibitors eculizumab and ravulizumab are approved treatments for atypical hemolytic uremic syndrome (aHUS). Ravulizumab was engineered from eculizumab to reduce dosing frequency from every 2 weeks to every 4–8 weeks (bodyweight dependent).

Aims: To evaluate treatment preference for eculizumab and ravulizumab, and treatment impact on daily life, in adults with aHUS.

Methods: Eligible US adults (aged ≥18 years) with a confirmed diagnosis of aHUS who had previously received eculizumab and ≥3 doses of ravulizumab were included. Treatment preference and impacts were evaluated using a web-based survey that included questions on demographics, aHUS disease and management history, and ravulizumab versus eculizumab preference. Variables were compared using paired t-tests (continuous) or McNemar-Bowker tests (dichotomous) at a significance level of p < 0.05 (descriptive purposes only).

Results: Fifty patients completed the survey (mean [standard deviation] age, 46.5 [13.9] years; 80% female). Mean (standard deviation) duration of eculizumab and ravulizumab treatment was 46.9 (33.4) and 12.9 (6.0) months, respectively. Overall, 94% of patients preferred ravulizumab (6% either had no preference or preferred eculizumab; p < 0.0001). Regarding factors related to daily life, patients showed significant preference for ravulizumab over eculizumab (Figure 1); when considering infusion frequency, all patients preferred ravulizumab. When asked to select up to five factors that were most important when deciding overall treatment preference, 80% of patients selected infusion frequency. Significantly more patients (p≤0.05) responded that they were able to enjoy life more with ravulizumab than with eculizumab, and that ravulizumab infusion frequency had minimal impact on daily life and work (Figure 2). When considering factors such as treatment effectiveness, safety and financial wellbeing, there were no significant differences between treatment preferences.

Conclusion(s): Survey results suggest that less frequent infusions strongly influences patient preference for ravulizumab over eculizumab, and demonstrates the benefit of ravulizumab treatment on patients’ quality of life and completion of daily activities.

Figure 1

Treatment preference based on factors that may affect patient daily life

Figure 2

Comparison of treatment-specific impacts

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/preference-for-ravulizumab-over-eculizumab-a-real-world-patient-preference-and-life-impact-study-on-the-treatment-of-atypical-hemolytic-uremic-syndrome/