PT  - JOURNAL ARTICLE
AU  - Rachel B Jones
AU  - Thomas F Hiemstra
AU  - Jose Ballarin
AU  - Daniel Engelbert Blockmans
AU  - Paul Brogan
AU  - Annette Bruchfeld
AU  - Maria C Cid
AU  - Karen Dahlsveen
AU  - Janak de Zoysa
AU  - Georgína Espigol-Frigolé
AU  - Peter Lanyon
AU  - Chen Au Peh
AU  - Vladimir Tesar
AU  - Augusto Vaglio
AU  - Michael Walsh
AU  - Dorothy Walsh
AU  - Giles Walters
AU  - Lorraine Harper
AU  - David Jayne
ED  - ,
TI  - Mycophenolate mofetil versus cyclophosphamide for remission induction in ANCA-associated vasculitis: a randomised, non-inferiority trial
AID  - 10.1136/annrheumdis-2018-214245
DP  - 2019 Mar 01
TA  - Annals of the Rheumatic Diseases
PG  - 399--405
VI  - 78
IP  - 3
4099  - http://ard.bmj.com/content/78/3/399.short
4100  - http://ard.bmj.com/content/78/3/399.full
SO  - Ann Rheum Dis2019 Mar 01; 78
AB  - Objectives Cyclophosphamide induction regimens are effective for antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), but are associated with infections, malignancies and infertility. Mycophenolate mofetil (MMF) has shown high remission rates in small studies of AAV.Methods We conducted a randomised controlled trial to investigate whether MMF was non-inferior to cyclophosphamide for remission induction in AAV. 140 newly diagnosed patients were randomly assigned to MMF or pulsed cyclophosphamide. All patients received the same oral glucocorticoid regimen and were switched to azathioprine following remission. The primary endpoint was remission by 6 months requiring compliance with the tapering glucocorticoid regimen. Patients with an eGFR <15 mL/min were excluded from the study.Results At baseline, ANCA subtype, disease activity and organ involvement were similar between groups. Non-inferiority was demonstrated for the primary remission endpoint, which occurred in 47 patients (67%) in the MMF group and 43 patients (61%) in the cyclophosphamide group (risk difference 5.7%, 90% CI −7.5% to 19%). Following remission, more relapses occurred in the MMF group (23 patients, 33%) compared with the cyclophosphamide group (13 patients, 19%) (incidence rate ratio 1.97, 95% CI 0.96 to 4.23, p=0.049). In MPO-ANCA patients, relapses occurred in 12% of the cyclophosphamide group and 15% of the MMF group. In PR3-ANCA patients, relapses occurred in 24% of the cyclophosphamide group and 48% of the MMF group. Serious infections were similar between groups (26% MMF group, 17% cyclophosphamide group) (OR 1.67, 95% CI 0.68 to 4.19, p=0.3).Conclusion MMF was non-inferior to cyclophosphamide for remission induction in AAV, but resulted in higher relapse rate.Trial registration number NCT00414128.