Use of Avacopan outside the ADVOCATE Inclusion Criteria

Background: The ADVOCATE (Avacopan Development in Vasculitis to Obtain Corticosteroid elimination and Therapeutic Efficacy) trial was a large randomized controlled trial which has demonstrated the beneficial use of Avacopan in the management of Antineutrophil Cytoplasmic antibody (ANCA)-associated Vasculitis (AAV). However, patients with the most severe disease including those with estimated glomerular filtration rate (eGFR) <15, significant pulmonary haemorrhage and dual positive with anti-GBM antibody were excluded. We report 3- and 6-months outcomes of the use of Avacopan outside the ADVOCATE inclusion criteria in AAV patients with severe and life-threatening disease. Methods: We reviewed patients with AAV and renal involvement treated with Avacopan outside the ADVOCATE inclusion criteria. The response to treatment was measured on change in eGFR at diagnosis, 3 and 6 months, as well as the resolution of lung haemorrhage. Results: A total of 37 patients were treated with Avacopan with 15 patients falling outside the ADVOCATE inclusion criteria. The demographics and the serology of the patients is shown in table 1. Two were dialysis dependent at presentation and later regained renal function to become independent of renal replacement therapy (RRT) and two had dual positive antibodies with ANCA and anti-GBM. Two patients had diffuse pulmonary haemorrhage and one was ANCA negative. All patients showed an improvement in their renal functions at 3 and 6 months. Conclusion: This small case series demonstrate successful use of Avacopan outside ADVOCATE inclusion criteria. Further real-life data is likely to support use of Avacopan for a wider and more severe presentations of AAV.