Treatment preference and quality of life impact: ravulizumab vs eculizumab for atypical hemolytic uremic syndrome
Summary
Aim: Ravulizumab and eculizumab are complement C5 inhibitors approved for the treatment of atypical hemolytic uremic syndrome (aHUS). Ravulizumab requires less frequent infusions than eculizumab, which may reduce treatment burden. This study investigated patients’ treatment preferences and the impact of both treatments on patient and caregiver quality of life. Materials & methods: Two surveys were conducted (one for adult patients with aHUS and one for caregivers of pediatric patients with aHUS) to quantitatively assess treatment preference and the patient- and caregiver-reported impact of ravulizumab and eculizumab on quality of life. Patients were required to have a diagnosis of aHUS, to be currently receiving treatment with ravulizumab and to have received prior treatment with eculizumab. Participants were recruited via various sources: the Alexion OneSource™ patient support program, the Rare Patient Voice recruitment agency, the aHUS Foundation and directly via a clinician involved in the study. Results: In total, 50 adult patients (mean age: 46.5 years) and 16 caregivers of pediatric patients (mean age: 10.1 years) completed the surveys. Most adult patients (94.0%) and all caregivers reported an overall preference for ravulizumab over eculizumab; infusion frequency was one of the main factors for patients when selecting their preferred treatment. Fewer patients reported disruption to daily life and the ability to go to work/school due to ravulizumab infusion frequency (4.0% and 5.7%, respectively) than eculizumab infusion frequency (72.0% and 60.0%), with similar results for caregivers. Conclusion: Adult patients and caregivers of pediatric patients indicated an overall preference for ravulizumab than eculizumab for the treatment of aHUS, driven primarily by infusion frequency. This study contributes to the emerging real-world evidence on the treatment impact and preference in patients with aHUS.
Plain language summary
What is this article about?
Atypical hemolytic uremic syndrome, also called aHUS, is a rare disease that causes kidney damage. Ravulizumab and eculizumab are medications given as intravenous (IV) infusions to treat people who have aHUS. Ravulizumab does not have to be taken as often as eculizumab. In this research, the investigators made surveys to ask people which medication they preferred to treat aHUS and how the medications affected their lives. Those who took part were either adult patients with aHUS, or people who were caring for a child with aHUS. Patients had experience being treated with both ravulizumab and eculizumab.
What were the results?
The survey responses showed that most people preferred ravulizumab over eculizumab. One of the main reasons they preferred ravulizumab was that they did not have to take ravulizumab as often as eculizumab, and their answers showed that this led to less disruption to their daily lives and their work/school attendance.
What do the results mean?
These findings should help doctors, patients and caregivers to decide whether ravulizumab or eculizumab is best for the patient.