Langford Carol A, Khalidi Nader, Springer Jason, Friedman Marcia, Hellmich Bernhard, Pagnoux Christian, Dehghan Natasha, Gewurz-Singer Ora, Koening Curry L, Lin Yih Chang, Monach Paul A, Moreland Larry W, Fifi-Mah Aurore, Flossmann Oliver, Forbess Lindsy J, Lanyon Peter, Molloy Eamonn, Specks Ulrich, Spiera Robert, Yacyshyn Elaine, McAlear Carol A, Burroughs Cristina, Jones Rachel B, Rhee Rennie L, Hajj-Ali Rula, Warrington Kenneth J, Cuthbertson David, Krischer Jeffrey P, Jayne David, Merkel Peter A
Cleveland Clinic, Cleveland, Ohio.
St Joseph's Healthcare and McMaster University, Hamilton, Ontario, Canada.
Arthritis Rheumatol. 2025 Jun 12. doi: 10.1002/art.43272.
To compare the efficacy of abatacept to placebo for the treatment of relapsing, nonsevere granulomatosis with polyangiitis (GPA).
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.
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.
In patients with relapsing, nonsevere GPA, abatacept did not reduce the risk of relapse, severe worsening, or failure to achieve remission.
比较阿巴西普与安慰剂治疗复发性、非重症肉芽肿性多血管炎（GPA）的疗效。
在这项多中心试验中，符合条件的复发性、非重症GPA患者被随机分为两组，一组每周皮下注射一次125mg阿巴西普，另一组接受安慰剂，两组均联合每日30mg泼尼松（或等效药物），在第12周逐渐减量并停药。已服用甲氨蝶呤、硫唑嘌呤、霉酚酸酯或来氟米特的患者继续以稳定剂量服用该药物。达到缓解的患者继续接受随机分配的治疗，直至复发、提前终止或最后一名患者入组12个月后的共同结束日期。在第6个月时出现非重症复发、非重症病情恶化或未缓解的患者可选择接受开放标签的阿巴西普治疗。主要终点是治疗失败率，定义为复发、疾病恶化或在6个月时未达到韦格纳肉芽肿病伯明翰血管炎活动评分（BVAS/WG）为0或1。
65例患者被随机分组；34例接受阿巴西普治疗，31例接受安慰剂治疗。接受阿巴西普治疗的患者与接受安慰剂治疗的患者在治疗失败率上无统计学差异（P = 0.853）。在关键次要终点方面，包括完全缓解时间（BVAS/WG = 0）、无糖皮质激素缓解持续时间、复发严重程度、损伤预防以及患者报告的生活质量结果，阿巴西普治疗与安慰剂相比均未显示出任何统计学差异。治疗组之间不良事件的频率或严重程度无差异，包括感染。
在复发性、非重症GPA患者中，阿巴西普并未降低复发、严重恶化或未达到缓解的风险。
