Abstract: FR-PO0659
Real-World Outcomes with Avacopan in ANCA-Associated Vasculitis: A Single-Center Experience
Session Information
- Glomerular Diseases: Clinical, Outcomes, and Therapeutics Research - ANCA/FSGS
October 23, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics
Authors
- Chadha, Ashima, Mass General Brigham Inc, Boston, Massachusetts, United States
- Chung, James L., Mass General Brigham Inc, Boston, Massachusetts, United States
- Schaefer, Emma R., Mass General Brigham Inc, Boston, Massachusetts, United States
- Aaron, Sydney, Mass General Brigham Inc, Boston, Massachusetts, United States
- Park, Sarah, Mass General Brigham Inc, Boston, Massachusetts, United States
- Laliberte, Karen A., Mass General Brigham Inc, Boston, Massachusetts, United States
- Efe, Orhan, Mass General Brigham Inc, Boston, Massachusetts, United States
- Niles, John L., Mass General Brigham Inc, Boston, Massachusetts, United States
- Al Jurdi, Ayman, Mass General Brigham Inc, Boston, Massachusetts, United States
- Seethapathy, Harish Shanthanu, Mass General Brigham Inc, Boston, Massachusetts, United States
Background
Complement activation plays a central role in the inflammatory injury characteristic of ANCA-associated vasculitis (AAV), driving the development of avacopan, an oral C5a receptor inhibitor, as an adjunct to standard immunosuppression. Recent regulatory reassessment of avacopan has prompted closer evaluation of its efficacy and safety in routine practice. We evaluated clinical and safety outcomes in a large single-center cohort treated with avacopan.
Methods
In this retrospective cohort study of all adults with AAV treated with avacopan at the MGB Vasculitis and Glomerulonephritis Center, we included patients with 12 months of follow-up from treatment initiation for index AAV episode or follow-up until death if earlier. Primary endpoints were remission at 26 and 52 weeks. Secondary endpoints assessed over 12-month follow-up were duration of steroid exposure, relapse or need for rescue therapy, adverse events, and longitudinal renal outcomes.
Results
104 patients met inclusion criteria (mean age 61 ± 15 years; 66.3% female). 98.1% received induction with rituximab- and/or cyclophosphamide-based regimens followed by rituximab maintenance. Median duration of avacopan therapy was 6 months (IQR 4-11), with physician practice favoring a planned 6-12 month course. 30.8% discontinued avacopan early due to side effects; 5 patients had transaminitis which resolved with cessation of the drug. 65.4% achieved remission by ~26 weeks, increasing to 83.7% by ~52 weeks. Median steroid duration was 15.3 weeks (IQR 7.5-26.4), relative to the clinic’s standard 21-week taper typically prescribed to those not on avacopan. Relapse or need for rescue therapy occurred in 28.8% of patients. Infections requiring hospitalization occurred in 19.2%. 6 patients died over 12-month follow-up. Among those with renal involvement (65.4%), median eGFR improved from 24 (IQR 14.5-48) to 34 (IQR 24.5-59.5) mL/min/1.73 m2 over 11-15 months (Wilcoxon signed-rank test, p=0.003); 4 progressed to end-stage renal disease.
Conclusion
Patients treated with avacopan exhibited high remission rates and favorable renal outcomes. However, many did not tolerate a full course of therapy, and the observed reduction in steroid exposure compared to standard taper was modest. Comparative analysis with an internal control cohort will further clarify clinical and safety outcomes and help define the role for complement inhibition in AAV.