Abstract: SA-PO804
Real-World Effectiveness of Ravulizumab among C5 Inhibitor-Naive Patients with Atypical Hemolytic Uremic Syndrome (aHUS): A Physician Panel-Based Chart Review Study (aHUS IMPACT)
Session Information
- C3G, TMA, MGRS, Amyloidosis, and More
October 26, 2024 | 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, California, United States
- Chaturvedi, Shruti, John Hopkins University, Baltimore, Maryland, United States
- Ong, Moh-Lim, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
- Nag, Dr. Arpita, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
- Song, Rui, Analysis Group, Inc., Boston, Massachusetts, United States
- Huynh, Lynn, Analysis Group, Inc., Boston, Massachusetts, United States
- Burdeau, Jordan, 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 rare disease and form of thrombotic microangiopathy (TMA) caused by complement dysregulation. Ravulizumab (RAV; a C5 inhibitor [C5i]) is approved for aHUS based on registrational clinical studies, but real-world evidence is limited.
Methods
This was a retrospective, longitudinal, physician panel-based chart review of C5i-naive patients (pts) in the USA treated with RAV. Physicians with medical and laboratory records for ≥1 pt with aHUS randomly selected 1–5 pts with aHUS who had ≥6 months of medical records after RAV initiation, unless the pt died.
Results
Overall, 79 C5i-naive pts with aHUS (enrolled by 31 physicians) initiated RAV. Median (IQR) pt age at RAV initiation was 44 (27–54) years; 3 pts (4%) had a kidney transplant before RAV initiation and 20 (25%) 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 occurred as early as Day 4 (LDH and percent change in serum creatinine [SCr]; both p<0.001) and Day 8 (platelet count, p<0.001). The proportions of pts with normalization of platelets, LDH and ≥25% improvement in SCr were 19/59 (32%), 17/51 (33%) and 19/58 (33%) at Day 8 and 40/48 (83%), 35/38 (92%) and 42/48 (88%) within 12 months after RAV initiation. Complete TMA response rates were 61% and 70% within 6 and 12 months after RAV initiation, respectively, and the median (IQR) time to complete TMA response was 3 months (1–13).
Conclusion
This study supports the immediate and sustained benefits of initiating with RAV in pts with aHUS as seen by the early response and continued improvement.
Funding
- Commercial Support – Alexion, AstraZeneca Rare Disease.