Efficacy and safety of avacopan in antineutrophil cytoplasmic autoantibody-associated vasculitis: a retrospective cohort study in Japan
Abstract Background Avacopan, an oral C5a receptor antagonist, demonstrated efficacy as an alternative to glucocorticoid therapy in patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) in the phase 3 ADVOCATE trial. However, limited real-world data exist on the outcom...
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2025-01-01
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| Online Access: | https://doi.org/10.1186/s41927-025-00456-4 |
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| author | Genri Tagami Makoto Yamaguchi Hirokazu Sugiyama Hiroshi Kinashi Kentaro Imai Keisuke Kamiya Takayuki Katsuno Takahiro Imaizumi Shogo Banno Yasuhiko Ito Takuji Ishimoto |
| author_facet | Genri Tagami Makoto Yamaguchi Hirokazu Sugiyama Hiroshi Kinashi Kentaro Imai Keisuke Kamiya Takayuki Katsuno Takahiro Imaizumi Shogo Banno Yasuhiko Ito Takuji Ishimoto |
| author_sort | Genri Tagami |
| collection | DOAJ |
| description | Abstract Background Avacopan, an oral C5a receptor antagonist, demonstrated efficacy as an alternative to glucocorticoid therapy in patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) in the phase 3 ADVOCATE trial. However, limited real-world data exist on the outcomes and experiences associated with avacopan use for AAV in Japan. Methods We performed a single-centre retrospective analysis and evaluated 21 patients with newly diagnosed or relapsed AAV who received avacopan. The co-primary outcomes were clinical remission at 6 and 12 months. Results Among the 21 patients, 20 (95.2%) achieved clinical remission at 6 months, and 19 (90.4%) sustained remission at 12 months. The median time from initiation of immunosuppressive therapy to the start of avacopan was 12 days (interquartile range, 5–26). Adverse events were reported in 10 patients (47.6%), with elevated liver enzyme levels observed in eight patients (38.1%) as the most frequent complication. Avacopan was discontinued in nine patients (42.9%). Despite early discontinuation, these patients achieved comparable rates of clinical remission at 6 months, sustained remission at 12 months, and experienced a reduction in glucocorticoid doses relative to those who continued avacopan. Conclusions A high incidence of adverse events, particularly liver enzyme elevation, and frequent early discontinuations of avacopan were observed. Nevertheless, favourable clinical outcomes and reduced glucocorticoid doses were achieved regardless of avacopan discontinuation. Further studies are warranted to validate the optimal use of avacopan in clinical practice. |
| format | Article |
| id | doaj-art-be1643440eae4de2806853e75c31eacb |
| institution | Kabale University |
| issn | 2520-1026 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | BMC |
| record_format | Article |
| series | BMC Rheumatology |
| spelling | doaj-art-be1643440eae4de2806853e75c31eacb2025-01-26T12:57:29ZengBMCBMC Rheumatology2520-10262025-01-01911810.1186/s41927-025-00456-4Efficacy and safety of avacopan in antineutrophil cytoplasmic autoantibody-associated vasculitis: a retrospective cohort study in JapanGenri Tagami0Makoto Yamaguchi1Hirokazu Sugiyama2Hiroshi Kinashi3Kentaro Imai4Keisuke Kamiya5Takayuki Katsuno6Takahiro Imaizumi7Shogo Banno8Yasuhiko Ito9Takuji Ishimoto10Department of Nephrology and Rheumatology, Aichi Medical UniversityDepartment of Nephrology and Rheumatology, Aichi Medical UniversityDepartment of Nephrology and Rheumatology, Aichi Medical UniversityDepartment of Nephrology and Rheumatology, Aichi Medical UniversityDepartment of Nephrology and Rheumatology, Aichi Medical UniversityDepartment of Nephrology and Rheumatology, Aichi Medical UniversityDepartment of Nephrology and Rheumatology, Aichi Medical UniversityData Coordinating Center, Department of Advanced Medicine, Nagoya University HospitalDepartment of Nephrology and Rheumatology, Aichi Medical UniversityDepartment of Nephrology and Rheumatology, Aichi Medical UniversityDepartment of Nephrology and Rheumatology, Aichi Medical UniversityAbstract Background Avacopan, an oral C5a receptor antagonist, demonstrated efficacy as an alternative to glucocorticoid therapy in patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) in the phase 3 ADVOCATE trial. However, limited real-world data exist on the outcomes and experiences associated with avacopan use for AAV in Japan. Methods We performed a single-centre retrospective analysis and evaluated 21 patients with newly diagnosed or relapsed AAV who received avacopan. The co-primary outcomes were clinical remission at 6 and 12 months. Results Among the 21 patients, 20 (95.2%) achieved clinical remission at 6 months, and 19 (90.4%) sustained remission at 12 months. The median time from initiation of immunosuppressive therapy to the start of avacopan was 12 days (interquartile range, 5–26). Adverse events were reported in 10 patients (47.6%), with elevated liver enzyme levels observed in eight patients (38.1%) as the most frequent complication. Avacopan was discontinued in nine patients (42.9%). Despite early discontinuation, these patients achieved comparable rates of clinical remission at 6 months, sustained remission at 12 months, and experienced a reduction in glucocorticoid doses relative to those who continued avacopan. Conclusions A high incidence of adverse events, particularly liver enzyme elevation, and frequent early discontinuations of avacopan were observed. Nevertheless, favourable clinical outcomes and reduced glucocorticoid doses were achieved regardless of avacopan discontinuation. Further studies are warranted to validate the optimal use of avacopan in clinical practice.https://doi.org/10.1186/s41927-025-00456-4Antineutrophil cytoplasmic autoantibody-associated vasculitisAvacopanEfficacyRemissionSafety |
| spellingShingle | Genri Tagami Makoto Yamaguchi Hirokazu Sugiyama Hiroshi Kinashi Kentaro Imai Keisuke Kamiya Takayuki Katsuno Takahiro Imaizumi Shogo Banno Yasuhiko Ito Takuji Ishimoto Efficacy and safety of avacopan in antineutrophil cytoplasmic autoantibody-associated vasculitis: a retrospective cohort study in Japan BMC Rheumatology Antineutrophil cytoplasmic autoantibody-associated vasculitis Avacopan Efficacy Remission Safety |
| title | Efficacy and safety of avacopan in antineutrophil cytoplasmic autoantibody-associated vasculitis: a retrospective cohort study in Japan |
| title_full | Efficacy and safety of avacopan in antineutrophil cytoplasmic autoantibody-associated vasculitis: a retrospective cohort study in Japan |
| title_fullStr | Efficacy and safety of avacopan in antineutrophil cytoplasmic autoantibody-associated vasculitis: a retrospective cohort study in Japan |
| title_full_unstemmed | Efficacy and safety of avacopan in antineutrophil cytoplasmic autoantibody-associated vasculitis: a retrospective cohort study in Japan |
| title_short | Efficacy and safety of avacopan in antineutrophil cytoplasmic autoantibody-associated vasculitis: a retrospective cohort study in Japan |
| title_sort | efficacy and safety of avacopan in antineutrophil cytoplasmic autoantibody associated vasculitis a retrospective cohort study in japan |
| topic | Antineutrophil cytoplasmic autoantibody-associated vasculitis Avacopan Efficacy Remission Safety |
| url | https://doi.org/10.1186/s41927-025-00456-4 |
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