Background/Purpose: Avacopan (AVP) is a recently approved adjunct therapy for remission induction of ANCA-associated vasculitis (AAV). Data on real-world use of AVP in AAV are lacking. The objective of this study was to describe the current practice in the use of AVP for the treatment of AAV and associated outcomes.

Methods: We performed a multi-center retrospective cohort study of 80 adult patients with new and relapsing AAV treated with AVP. Follow-up time was up to 52 weeks from diagnosis. The primary outcome measure was clinical remission, as determined by the investigator. Secondary outcome measures included Birmingham Vasculitis Activity Score (BVAS) 3, cumulative glucocorticoid dose, estimated glomerular filtration rate (eGFR) (mL/min/1.73m²), proteinuria (g/g), hematuria, disease relapses, hospitalizations, end-stage kidney disease (ESKD), infections, and death. Data are presented as mean (±SD), median (IQR), or number (percent).

Results: Mean age was 59 years (±17), 65% were female, 59 (74%) had MPO ANCA, and 76 (95%) had kidney involvement. At diagnosis, 19 patients (24%) had eGFR < 15 mL/min/1.73m^2, and 8 (10%) were dialysis-dependent. Rituximab plus cyclophosphamide was the most common induction regimen (49%), followed by rituximab only (46%). AVP was started 8.8 weeks (SD ± 19.6) after glucocorticoid initiation with 58 patients (73%) discontinuing prednisone 7.5 weeks (±18) after starting AVP. Outcomes are summarized in Table 1. Among the 60 patients (75%) with hematuria at diagnosis, 70% had resolution of hematuria 14 weeks (±14) after AVP initiation. Nadir proteinuria of 0.3 g/g (0.1 – 0.7) was achieved 10 weeks (±21) after AVP initiation. The cumulative dose of IV methylprednisolone was 2.4 g (±1.4), and 12-week oral prednisone was 1.8 g (±1.1). At week 26, 5 of 59 patients (8%) remained on prednisone. Of the 80 patients, 60 were started on remission maintenance therapy with rituximab (n = 56), azathioprine (n = 3), and intravenous immunoglobulin (n = 1).

AVP was stopped in 25 patients (31%): 11 (14%) after completing 52 weeks of treatment, and 14 (18%) before 52 weeks due to adverse events, including 4 patients with transaminitis. At a mean follow-up time of 8 months (±6), 5 (6%) had a disease relapse, 7 (9%) had infections requiring hospitalization, 3 (4%) progressed to end-stage kidney disease, and 3 (4%) patients died.

Conclusion: Patients with AAV treated with AVP in conjunction to standard remission-induction therapy have a high rate of clinical remission at weeks 26 and 52 and demonstrate a sustained improvement in eGFR. There was variability in the time till initiation of AVP and glucocorticoid discontinuation. AVP was discontinued in a group of patients due to adverse events or after 1-year of continuous treatment. Further data on the longer-term use of AVP is needed.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/real-world-experience-with-avacopan-in-anca-vasculitis-a-multi-center-retrospective-cohort-analysis/