(A-121) Real-World Effectiveness of Ravulizumab in Adults With aHUS who Switched to Ravulizumab Within 3 Months of Eculizumab Treatment (aHUS IMPACT)

Sunday, August 24, 2025

12:00 PM - 1:30 PM ET

Co-Author(s)

SC

Shruti Chaturvedi

Johns Hopkins University, Baltimore, MD, USA, United States
YW

Yan Wang

Alexion, AstraZeneca Rare Disease, Boston, MA, USA, United States
MO

Moh-Lim Ong

Alexion, AstraZeneca Rare Disease, Boston, MA, USA, United States
AN

Arpita Nag

Alexion, AstraZeneca Rare Disease, Boston, MA, USA, United States
RS

Rui Song

Analysis Group, Inc., Boston, MA, USA, United States

Co-Author(s)

LH

Lynn Huynh

Analysis Group, Inc., Boston, MA, USA, United States

Co-Author(s)

JB

Jordan A. Burdeau

Analysis Group, Inc., Boston, MA, USA, United States

Presenting Author(s)

MD

Mei Sheng Duh

Analysis Group, Inc., Boston, MA, USA, United States

Co-Author(s)

RH

Ramy M. Hanna

University of California, Irvine, CA, USA, United States

Background: Atypical haemolytic uraemic syndrome (aHUS) is a progressive rare disease caused by complement dysregulation that, if untreated, can lead to severe organ damage and death. Ravulizumab, a next-generation terminal complement C5 inhibitor, has demonstrated immediate, complete, and sustained terminal complement inhibition in clinical trials.

Objectives: To determine ravulizumab effectiveness following short-term eculizumab treatment (≤ 3 months) among patients (pts) with aHUS.

Methods: This was a retrospective, longitudinal, physician panel-based chart review of adults (aged ≥ 18 years) with aHUS in the USA who switched from eculizumab to ravulizumab between 1 November 2019 and 30 September 2022, and with ≤ 3 months of eculizumab treatment. Physicians with medical and laboratory records for ≥ 1 pt(s) with aHUS randomly selected ≤ 5 pts with ≥ 6 months of medical records after ravulizumab initiation; pts with < 6 months of medical records could be included if the pt died during these 6 months. Laboratory parameters (platelet count, lactate dehydrogenase [LDH], and serum creatinine [SCr]) were collected at eculizumab and ravulizumab initiation (or at the point closest to eculizumab or ravulizumab initiation, respectively, within the prior 6 months), and at 1, 3, 6 and 12 months after ravulizumab initiation.

Results: Overall, 54 pts with aHUS (enrolled by 33 physicians) were included in the analysis; 35 (64.8%) were male, 39 (72.2%) were White, 9 (16.7%) were Black or African American and 6 (11.1%) were Asian. Median (interquartile range) age at ravulizumab initiation was 43.0 (32.5, 57.0) years. Mean (standard deviation) time from diagnosis to eculizumab initiation was 1.8 (3.7) months, and from eculizumab initiation to ravulizumab initiation was 1.7 (0.9) months. Two pts (3.7%) had a kidney transplant prior to eculizumab initiation, and 6 pts (11.1%) received acute (n = 4) or chronic (n = 2) dialysis during the baseline period. Compared with pre-ravulizumab initiation, mean (95% confidence interval) platelet count increased by 54.1 × 109/L (36.0, 72.3; p < 0.001) at Month 1 and 111.3 × 109/L (87.7, 135.0; p < 0.001) at Month 12; LDH decreased by 151.0 U/L (91.6, 210.4; p < 0.001) at Month 1 and 245.4 U/L (145.8, 344.9; p < 0.001) at Month 12; serum creatinine decreased by 24.7% (15.4%, 33.0%; p < 0.001) at Month 1 and 41.9% (32.4%, 50.0%; p < 0.001) at Month 12.

Conclusions: This study provides real-world evidence that switching to ravulizumab after short-term eculizumab is achievable in pts with aHUS and may result in significant haematological and renal improvements in this cohort. This was demonstrated by both an early response and continued improvement through 1 year after ravulizumab initiation.