The following is a summary of “Avacopan for anti-neutrophil cytoplasm antibodies-associated vasculitis: a multicentre real-world study,” published in the July 2024 issue of Rheumatology by Gabilan et al.

Avacopan, a targeted C5aR1 inhibitor, has recently been recognized as a glucocorticoid-sparing treatment option for anti-neutrophil cytoplasm antibodies (ANCA)-associated vasculitis (AAV).

Researchers conducted a retrospective study assessing the safety and effectiveness of avacopan in patients outside of RCTs or patients with significant kidney involvement.

They examined clinical records of patients with AAV contraindicated for high-dose glucocorticoids receiving avacopan 30 mg b.i.d plus standard-of-care regimen under a French early access program (2020-2023). Standardized case report forms captured efficacy and safety data.

Among the 31 patients (median age 72 years), 10 had relapsing AAV, 20 were positive for anti-myeloperoxidase antibodies, and 30 exhibited kidney vasculitis. The induction therapy included rituximab (n= 27), cyclophosphamide (n= 2), or a combination of both (n= 2). Only 5 patients did not receive glucocorticoids (GCs). Following a rapid taper of GCs, discontinued in 23 patients before 3 months, 25 patients (81%) achieved favorable outcomes without severe AEs. The estimated GFR increased from 19 [15; 34] to 35 mL/min/1.73m² [23; 45] at month 12 (P<0.05), regardless of kidney biopsy findings. Development of refractory AAV was observed in 1 patient, and 2 had relapses while treated with avacopan. The ANCA persisted in over half of tested patients 10/18 (55.5%) at 12 month, 2 patients discontinued avacopan due to severe AEs (hepatitis and age-related macular degeneration).

Investigators concluded that avacopan demonstrated a glucocorticoid-sparing effect, even in patients with severe kidney vasculitis, but additional research is needed to determine the optimal glucocorticoid dosing alongside the use.

Source: academic.oup.com/rheumatology/advance-article-abstract/doi/10.1093/rheumatology/keae359/7713282