Treatment preference and quality of life impact: ravulizumab vs eculizumab for atypical hemolytic uremic syndrome

Abstract

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.

Keywords: aHUS; atypical hemolytic uremic syndrome; caregiver preference; eculizumab; patient preference; patient-reported outcomes; quality of life; ravulizumab; treatment preference.

Figure 1.. Treatment preference between ravulizumab and prior eculizumab treatment by factor for (A) adult patients and (B) caregivers of pediatric patients.

For survey items relating to concerns (e.g. concern related to infusion), respondents indicated which treatment they preferred to overcome their concern. The binomial test was used to compare the proportion of those who indicated any preference for ravulizumab versus those who indicated either ‘no preference’ or any preference for eculizumab. aHUS: Atypical hemolytic uremic syndrome.

Figure 2.. Frequencies of participant responses when asked to select up to five most-important factors when deciding overall preference between treatments for (A) adult patients and (B) caregivers of pediatric patients.

Participants were asked to select up to five factors that were most important to them when deciding their overall preference between prior eculizumab treatment and ravulizumab. aHUS: Atypical hemolytic uremic syndrome.

Figure 3.. Comparison of impacts of ravulizumab and prior eculizumab treatment reported by adult patients (n = 50).

For each statement, participants were asked to select how much they agreed based on their own experience. Bar labels show the survey questions verbatim and have been grouped by negative (bar groups 1–6) and positive (bar groups 7–10) statements. It is acknowledged as a possible limitation that respondents may interpret ‘impact’ differently and not according to the intended directionality. Percentages may not sum to 100 owing to rounding. aHUS: Atypical hemolytic uremic syndrome. *p < 0.05; ***p < 0.001.

Figure 4.. Comparison of impacts of ravulizumab and prior eculizumab treatment reported by caregivers of pediatric patients (n = 16).

For each statement, participants were asked to select how much they agree based on their own experience. Bar labels show the survey questions verbatim and have been grouped by negative (bar groups 1–8) and positive (bar groups 9–13) statements. It is acknowledged as a possible limitation that respondents may interpret ‘impact’ differently and not according to the intended directionality. Percentages may not sum to 100 owing to rounding. aHUS: Atypical hemolytic uremic syndrome. *p < 0.05; **p < 0.01.