TY - JOUR
T1 - The real-world use and effectiveness of avacopan in routine practice for the treatment of ANCA vasculitis. First experiences in Spain
AU - Draibe, Juliana
AU - Espigol-Frigolé, Georgina
AU - Cid, Maria Cinta
AU - Prados, M C
AU - Guillén, E
AU - Villacorta, J
AU - Vega, C
AU - Martins, J
AU - daSilva, I
AU - Martin-Gomez, Ma Adoración
AU - Huerta, A
AU - Martinez-Valenzuela, L
AU - Morales, Enrique
N1 - © The Author(s) 2024. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For permissions, please email: [email protected].
PY - 2025/4/1
Y1 - 2025/4/1
N2 - OBJECTIVES: ANCA-associated vasculitis (AAV) are chronic diseases with relapses that associate organic damage because of the disease and its treatment. Avacopan is a new treatment indicated for AAV. We present the first experiences with avacopan in Spain as part of an Early Access program.METHODS: Patients with AAV who started avacopan between June 2022-September 2023 were included. For comparison, a historical cohort of patients diagnosed with AAV around the same time and treated without avacopan was also included.RESULTS: 29 patients treated with avacopan were analyzed. Twelve patients (41.4%) were male, median age was 56 years. Most of patients were ANCA MPO positive (21/29, 72.4%). The most frequently affected organ was the kidney (23/29, 79.31%), with a mean eGFR of 23.2 ml/minMedian follow-up was 456.8 ± 181.7 days with a remission rate of 86.2%. eGFR increased from 23.2 ± 11.2-38.38 ± 18.55 ml/min after 12 months of diagnosis.2 patients had Adverse Events related to avacopan as severe neutropenia and a gastrointestinal affectation , 13 infections were reported and 1 death.Patients treated with avacopan received a significantly lower cumulative dose of prednisone at 6 and 12 months (p-values of 0.02 and <0.01, respectively) compared with historical controls. The evolution of GFR at 1 year of follow-up and the incidence of relapse were similar in both groups.CONCLUSION: The combination of avacopan in clinical practice presents a good safety profile and provides added value by contributing to the control of AAV activity, increase GFR, and removal of steroids.
AB - OBJECTIVES: ANCA-associated vasculitis (AAV) are chronic diseases with relapses that associate organic damage because of the disease and its treatment. Avacopan is a new treatment indicated for AAV. We present the first experiences with avacopan in Spain as part of an Early Access program.METHODS: Patients with AAV who started avacopan between June 2022-September 2023 were included. For comparison, a historical cohort of patients diagnosed with AAV around the same time and treated without avacopan was also included.RESULTS: 29 patients treated with avacopan were analyzed. Twelve patients (41.4%) were male, median age was 56 years. Most of patients were ANCA MPO positive (21/29, 72.4%). The most frequently affected organ was the kidney (23/29, 79.31%), with a mean eGFR of 23.2 ml/minMedian follow-up was 456.8 ± 181.7 days with a remission rate of 86.2%. eGFR increased from 23.2 ± 11.2-38.38 ± 18.55 ml/min after 12 months of diagnosis.2 patients had Adverse Events related to avacopan as severe neutropenia and a gastrointestinal affectation , 13 infections were reported and 1 death.Patients treated with avacopan received a significantly lower cumulative dose of prednisone at 6 and 12 months (p-values of 0.02 and <0.01, respectively) compared with historical controls. The evolution of GFR at 1 year of follow-up and the incidence of relapse were similar in both groups.CONCLUSION: The combination of avacopan in clinical practice presents a good safety profile and provides added value by contributing to the control of AAV activity, increase GFR, and removal of steroids.
KW - Adult
KW - Aged
KW - Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis/drug therapy
KW - Female
KW - Glomerular Filtration Rate/drug effects
KW - Humans
KW - Immunosuppressive Agents/therapeutic use
KW - Male
KW - Middle Aged
KW - Remission Induction
KW - Retrospective Studies
KW - Spain
KW - Treatment Outcome
UR - https://pubmed.ncbi.nlm.nih.gov/39352795/
U2 - 10.1093/rheumatology/keae534
DO - 10.1093/rheumatology/keae534
M3 - Article
C2 - 39352795
SN - 1462-0324
VL - 64
SP - 2019
EP - 2026
JO - Rheumatology
JF - Rheumatology
IS - 4
ER -