RT Journal Article
SR Electronic
T1 Mycophenolate mofetil versus cyclophosphamide for remission induction in ANCA-associated vasculitis: a randomised, non-inferiority trial
JF Annals of the Rheumatic Diseases
JO Ann Rheum Dis
FD BMJ Publishing Group Ltd and European League Against Rheumatism
SP 399
OP 405
DO 10.1136/annrheumdis-2018-214245
VO 78
IS 3
A1 Jones, Rachel B
A1 Hiemstra, Thomas F
A1 Ballarin, Jose
A1 Blockmans, Daniel Engelbert
A1 Brogan, Paul
A1 Bruchfeld, Annette
A1 Cid, Maria C
A1 Dahlsveen, Karen
A1 de Zoysa, Janak
A1 Espigol-Frigolé, Georgína
A1 Lanyon, Peter
A1 Peh, Chen Au
A1 Tesar, Vladimir
A1 Vaglio, Augusto
A1 Walsh, Michael
A1 Walsh, Dorothy
A1 Walters, Giles
A1 Harper, Lorraine
A1 Jayne, David
YR 2019
UL http://ard.bmj.com/content/78/3/399.abstract
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.