Advances in the treatment of antineutrophil cytoplasmic antibody associated vasculitis: current recommendations, clinical trials and real word data

Antineutrophil cytoplasmic antibody (ANCA) associated vasculitis is a group of rare, autoimmunological diseases that can manifest as a vital organ and/or life-threatening disorder. They are classified as small vessel vasculitis (SVV). Inflammatory infiltrations cause destruction and necrosis of the vessel wall, as well as occlusion of the vessel lumen; thus, organs supplied by these vessels become ischemic and damaged. According to the 2012 Revised International Chapel Hill Consensus Conference Nomenclature, AAV comprises three distinct diseases, but this review focuses on granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). Quick diagnosis and prompt initiation of intensive immunosuppressive therapy are crucial to prevent an unfavorable outcome in AAV patients. Recent years have brought development in therapeutic strategies, new immunosuppressive agents, and future perspectives. Our review starts with the description of current diagnostic and long-term follow-up strategies, including the role of ANCA, IgG, and CD19/CD20 cells monitoring, as well as remission assessment. The following sections present possible induction and maintenance therapies, including cyclophosphamide, rituximab, glucocorticoids, avacopan, and therapeutic plasma exchange, based on current recommendations from EULAR, KIDIGO, and the British Society of Rheumatology, as well as results from recent randomized clinical trials and real-world data. These therapies are particularly relevant for specific clinical conditions, such as rapidly progressive glomerulonephritis and/or pulmonary hemorrhage. We describe in detail a new, promising molecule, avacopan, which acts by blocking the C5a receptor on neutrophils and other cell types. Since July 2025, this drug has also been available in Poland.