Ravulizumab improves long-term quality-of-life in atypical haemolytic uraemic syndrome

Continued long-term therapy with ravulizumab showed sustained quality-of-life improvements in both adults and paediatric patients with atypical haemolytic uraemic syndrome enrolled in two phase 3 trials.

The analysis was based on data from two completed phase 3 trials in adult patients and paediatric patients with atypical haemolytic uraemic syndrome who were naïve to complement C5 inhibitors (NCT02949128, NCT03131219) [1]. In these trials, ravulizumab was administered intravenously every 4–8 weeks, depending on body weight, and participants were permitted to undergo dialysis as needed. Data was collected during the initial 26 weeks of the trials and through extension periods, which could last up to 4.5 years. Quality-of-life was assessed by the 3-level, 5-dimension EuroQol (EQ-5D-3L) and the Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F) questionnaires.

The analysis included a total of 56 adult and 20 paediatric participants. Overall, baseline mean values of the quality-of-life questionnaires were lower than normal population values. In adults, EQ-5D-3L scores improved from a mean 0.56 at baseline to day 29, and were maintained between 0.80–0.90 throughout the rest of the follow-up to day 1,583. Similarly, FACIT-F scores improved in both adult and paediatric participants and were maintained through follow-up. By day 743, 79.4% of the adult participants had a ≥3-point improvement from baseline in FACIT-F scores, while 100% of paediatric participants had a 3-point improvement from baseline by day 71 and remained constant through day 911.

The authors concluded that in adult and paediatric participants with atypical haemolytic uraemic syndrome, “2 quality-of-life measures showed low baseline quality-of-life scores. This analysis demonstrated that ravulizumab treatment was associated with rapid and clinically meaningful improvement in quality-of-life measures. Furthermore, improvement in quality-of-life scores after ravulizumab treatment was sustained at or near normal ranges throughout the extension period.”

1. Kavanagh D, et al. Ravulizumab in atypical haemolytic uraemic syndrome: analysis of quality of life outcomes in adult and paediatric phase 3 trials. 62nd ERA Congress, 4–7 June 2025, Vienna, Austria.

Medical writing support was provided by Mihai Surducan, PhD.

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Posted on 11 June 202522 August 2025