PB0793 - Preference for ravulizumab over eculizumab: a real-world patient preference and life impact study on the treatment of atypical hemolytic uremic syndrome

Monday, July 11, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Co-Author(s)

Teri Mauch, MD, PhD

Professor
University of Nebraska
Omaha, Nebraska, United States
MC

Michael Chladek

Associate Director
Clinical Outcomes Solutions, LLC (at the time of the study)
Chicago, Illinois, United States
Spero Cataland, MD

Professor
Department of Internal Medicine, Ohio State University, Columbus, OH, USA
Columbus, Ohio, United States
SC

Shruti Chaturvedi

Associate Professor
Johns Hopkins University
Baltimore, Maryland, United States
BD

Bradley Dixon

Associate Professor
University of Colorado School of Medicine
Aurora, Colorado, United States
KG

Katherine Garlo

Senior Medical Director
Alexion, AstraZeneca Rare Disease
Boston, Massachusetts, United States
CG

Christoph Gasteyger

Executive Director
Alexion, AstraZeneca Rare Disease
Boston, Massachusetts, United States
AJ

Anuja Java

Assistant Professor
Washington University School of Medicine
St. Louis, Missouri, United States

Presenting Author(s)

Jorge Leguizamo, MD

Hematology Director
Georgia Cancer Specialists/Northside Hospital Cancer Institute
Atlanta, Georgia, United States

Co-Author(s)

LL

Lucy Lloyd-Price

Research Scientist
Clinical Outcomes Solutions Ltd.
Folkestone, England, United Kingdom
TP

Tan Pham

Senior Research Scientist
Clinical Outcomes Solutions, LLC
Chicago, Illinois, United States
TS

Tara Symonds

Principal Scientific Advisor
Clinical Outcomes Solutions Ltd.
Folkestone, England, United Kingdom
IT

Ioannis Tomazos

Senior Medical Director
Alexion, AstraZeneca Rare Disease
Boston, Massachusetts, United States
YW

Yan Wang

Associate Director
Alexion, AstraZeneca Rare Disease
Boston, Massachusetts, United States

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.