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. 2025 Jun 12:10.1002/art.43272.
doi: 10.1002/art.43272. Online ahead of print.

A Randomized, Double-Blind, Placebo-Controlled Trial of Abatacept for the Treatment of Relapsing, Nonsevere Granulomatosis With Polyangiitis

Carol A Langford  1 , Nader Khalidi  2 , Jason Springer  3 , Marcia Friedman  4 , Bernhard Hellmich  5 , Christian Pagnoux  6 , Natasha Dehghan  7 , Ora Gewurz-Singer  8 , Curry L Koening  9 , Yih Chang Lin  10 , Paul A Monach  11 , Larry W Moreland  12 , Aurore Fifi-Mah  13 , Oliver Flossmann  14 , Lindsy J Forbess  15 , Peter Lanyon  16 , Eamonn Molloy  17 , Ulrich Specks  18 , Robert Spiera  19 , Elaine Yacyshyn  20 , Carol A McAlear  21 , Cristina Burroughs  10 , Rachel B Jones  22 , Rennie L Rhee  21 , Rula Hajj-Ali  1 , Kenneth J Warrington  18 , David Cuthbertson  10 , Jeffrey P Krischer  10 , David Jayne  22 , Peter A Merkel  21 ; Vasculitis Clinical Research Consortium and the European Vasculitis Society
Collaborators, Affiliations

A Randomized, Double-Blind, Placebo-Controlled Trial of Abatacept for the Treatment of Relapsing, Nonsevere Granulomatosis With Polyangiitis

Carol A Langford et al. Arthritis Rheumatol. .

Abstract

Objective: To compare the efficacy of abatacept to placebo for the treatment of relapsing, nonsevere granulomatosis with polyangiitis (GPA).

Methods: In this multicenter trial, eligible patients with relapsing, nonsevere GPA were randomized to receive abatacept 125 mg subcutaneously once a week or placebo, both together with prednisone 30 mg/day (or equivalent), tapered and discontinued at week 12. Patients already taking methotrexate, azathioprine, mycophenolate, or leflunomide continued this medication at a stable dose. Patients achieving remission remained on their randomized assignment until relapse, early termination, or the common close date 12 months after enrollment of the last patient. Those who had a nonsevere relapse, had nonsevere worsening, or were not in remission by month 6 had the option to receive open-label abatacept. The primary end point was the rate of treatment failure, defined as relapse, disease worsening, or failure to achieve a Birmingham Vasculitis Activity Score for Wegener's Granulomatosis (BVAS/WG) of 0 or 1 by six months.

Results: Sixty-five patients were randomized; 34 received abatacept and 31 received placebo. No statistical difference in the treatment failure rate was found between those who received abatacept and those who received placebo (P = 0.853). Treatment with abatacept did not demonstrate any statistical difference from placebo in key secondary end points, including time to full remission (BVAS/WG = 0), duration of glucocorticoid-free remission, relapse severity, prevention of damage, and patient-reported quality-of-life outcomes. There was no difference in the frequency or severity of adverse events between treatment arms, including infection.

Conclusion: In patients with relapsing, nonsevere GPA, abatacept did not reduce the risk of relapse, severe worsening, or failure to achieve remission.

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Figures

Figure 1.
Figure 1.
Study diagram of a randomized trial of abatacept in relapsing, non-severe granulomatosis with polyangiitis.
Figure 2.
Figure 2.
Consort diagram outlining the course of the 77 participants who signed informed consent on this trial.
Figure 3.
Figure 3.
Treatment failure in 22 of 34 patients randomized to abatacept compared to 23 of 31 who received placebo.
See this image and copyright information in PMC

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