AVACOPAN IN THE REAL-LIFE PRACTICE: EVALUATION OF EFFICACY, SAFETY, AND IMPACT ON QUALITY OF LIFE IN THE EARLY STAGES OF TREATMENT
Abstract: AB0789
Authors: E. Treppo et al.
Avacopan, a selective complement 5a-receptor antagonist, is already approved in several countries for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). It does not replace but complement treatment with cyclophosphamide (CYC) and/or rituximab (RTX). It significantly reduces cumulative glucocorticoid use and shows positive effects on kidney function.
Key content:
The aim of this prospective multicenter study was to assess efficacy and safety of avacopan in the real-life practice in GPA or MPA patients in the setting of a compassionate use program. A total of 12 patients (7 females, 5 males) with a median age of 64 (IQR 48-66) were recruited. Avacopan was mainly used in relapsing disease (8/12, 66.7%) and in GPA (9/12, 75%), compared with new-onset disease (4/12, 33.3%) and MPA (3/12, 25%). The relapsed patients had a median disease duration of 12 (IQR 4.3-15.3) years and had been previously treated with other immunosuppressants, including RTX (5/8) CYC (5/8), AZA (3/8), MTX (2/8), and MMF (1/8).
At the start of avacopan treatment, the median BVASv3 and VDI were 12 (IQR 8-20.8) and 2.5 (IQR 1.5-3.3), respectively. 9 out of 12 (75%) patients had renal involvement, of which 5/9 (55.6%) had rapidly progressive glomerulonephritis. Other disease involvements were ENT (5/12) and pulmonary (5/12) of which 3/5 had alveolar haemorrhage. Treatment was initiated with a median steroid dose of 50 (IQR 6.9-50) mg prednisone equivalent and in combination with RTX at a dose of 1 gram every 2 weeks (9/12) or 375 mg/m2 weekly for 4 consecutive weeks (3/12). Two patients also received 1 gram of CYC before RTX.
After month 3, 10/12patients were in clinical remission and the median steroid dose had decreased to 5 (IQR 2.2-10.6) mg prednisone equivalent. The median value of creatinine did not differ from baseline, whereas the median value of 24-hour urine protein had decreased from 680.5mg per day to 254mg per day. A total of 3 adverse events (AEs) were recorded.
Relevance:
The data are in agreement with the results of the prospective studies. They show the anticipated steroid-sparing effect and a good tolerability. Regarding preservation of kidney function and long-term benefit large registries are needed such as the European or the Swiss registry (EUVAS and VASAS, respectively).
