Background The majority of patients with Wegener's granulomatosishave disease flares after conventional medications are tapered.There is no consistently safe, effective treatment for the maintenanceof remission.
Methods We conducted a randomized, placebo-controlled trialat eight centers to evaluate etanercept for the maintenanceof remission in 180 patients with Wegener's granulomatosis.The primary outcome was sustained remission, defined as a BirminghamVasculitis Activity Score for Wegener's Granulomatosis of 0for at least six months (scores can range from 0 to 67, withhigher scores indicating more active disease). In addition toetanercept or placebo, patients received standard therapy (glucocorticoidsplus cyclophosphamide or methotrexate). After remission, standardmedications were tapered according to the protocol.
Results The mean follow-up for the overall cohort was 27 months.Of the 174 patients who could be evaluated, 126 (72.4 percent)had a sustained remission, but only 86 (49.4 percent) remainedin remission for the remainder of the trial. There were no significantdifferences between the etanercept and control groups in therates of sustained remission (69.7 percent vs. 75.3 percent,P=0.39), sustained periods of low-level disease activity (86.5percent vs. 90.6 percent, P=0.32), or the time required to achievethose measures. Disease flares were common in both groups, with118 flares in the etanercept group (23 severe and 95 limited)and 134 in the control group (25 severe and 109 limited). Therewas no significant difference between the etanercept and controlgroups in the relative risk of disease flares per 100 person-yearsof follow-up (0.89, P=0.54). During the study, 56.2 percentof patients in the etanercept group and 57.1 percent of thosein the control group had at least one severe or life-threateningadverse event or died (P=0.90). Solid cancers developed in sixpatients in the etanercept group, as compared with none in thecontrol group (P=0.01).
Conclusions Etanercept is not effective for the maintenanceof remission in patients with Wegener's granulomatosis. Durableremissions were achieved in only a minority of the patients,and there was a high rate of treatment-related complications.
Source Information
As chairman of the WGET Research Group, Dr. John H. Stone accepts full responsibility for the integrity of the article.
Address reprint requests to Dr. John H. Stone at the Johns Hopkins Vasculitis Center, 5501 Hopkins Bayview Cir., JHAAC 1B.23, Baltimore, MD 21224, or at jstone{at}jhmi.edu.
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