Abstract: SA-PO0798
Real-World Effectiveness of Ravulizumab Among Patients with Atypical Hemolytic Uremic Syndrome (aHUS) in Underrepresented Minority Subgroups: Physician Panel-Based Chart Review Study
Session Information
- Glomerular Research: Design, Registries, Surveys, and Epidemiology
November 08, 2025 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics
Authors
- Hanna, Ramy Magdy, University of California Irvine, Irvine, California, United States
- Chaturvedi, Shruti, Johns Hopkins University, Baltimore, Maryland, United States
- Nag, Arpita, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
- Huynh, Lynn, Analysis Group Inc, Boston, Massachusetts, United States
- Burdeau, Jordan, Analysis Group Inc, Boston, Massachusetts, United States
- Young-Xu, Leili, Analysis Group Inc, Boston, Massachusetts, United States
- Duh, Mei Sheng, Analysis Group Inc, Boston, Massachusetts, United States
- Wang, Yan, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
Background
aHUS is a form of thrombotic microangiopathy caused by complement dysregulation. Ravulizumab (RAV), a complement C5 inhibitor (C5i), is approved for aHUS based on registered clinical studies; real-world evidence in patients (pts) who are underrepresented in clinical trials is limited.
Methods
Retrospective, longitudinal chart review of C5i-naive pts in the USA treated with RAV. Participating physicians randomly selected 1–5 pts with aHUS with ≥6 months of medical records after RAV initiation, or <6 months if the pt had died; among these, Black/African American (AA) and Hispanic pts were analyzed.
Results
Of 79 adult pts in the study, 18 were Black/AA and 19 were Hispanic. Median (IQR) age at RAV initiation was 36 (24, 52) years for Black/AA pts and 37 (23, 51) for Hispanic pts. Mean (±SD) time from first aHUS manifestation to diagnosis was 0.6 (±0.8) and 2.6 (±5.6) months for Black/AA and Hispanic pts, respectively. In total, two (11%) Black/AA and seven (37%) Hispanic pts received dialysis 12 months before to ≤2 weeks after RAV initiation. Laboratory parameters over time are shown in the Figure. Statistically significant changes from baseline (all p<0.01) occurred in platelet count, lactate dehydrogenase and serum creatinine (SCr) as early as Day 4 in both subgroups. Among Black/AA and Hispanic pts, respectively, the proportion with ≥25% improvement in SCr was 2/15 (13%) and 8/16 (50%) at Day 8 and 9/10 (90%) and 9/10 (90%) at 12 months after RAV initiation.
Conclusion
RAV initiation demonstrated immediate and sustained response in two underrepresented aHUS populations, similar to the broader aHUS population previously reported.
Funding
- Commercial Support – Alexion, AstraZeneca Rare Disease.