加拿大麦克马斯特大学Michael Walsh团队近日取得一项新成果。他们研究了血浆置换和糖皮质激素治疗严重ANCA相关性血管炎的疗效。这一研究成果发表在2020年2月13日出版的国际学术期刊《新英格兰医学杂志》上。

抗中性粒细胞胞浆抗体（ANCA）相关性血管炎需要更有效、更安全的治疗。

研究组进行了一项随机试验，招募严重ANCA相关性血管炎患者，采用2*2因子设计，将其随机分组分别进行血浆置换或不进行血浆置换；同时随机分组接受标准剂量糖皮质激素或减量糖皮质激素进行治疗。

随访7年后，血浆置换组的352例患者中有100例（28.4%）发生全因死亡或终末期肾病（ESKD），对照组352例患者中有109例（31.0%），差异不显著。亚组和次要指标分析结果亦相差不大。

糖皮质激素减量组的330例患者中有92例（27.9%）发生全因死亡或ESKD，标准剂量组325例患者中有83例（25.5%），符合非劣效性标准。减量组1年内严重感染的发生率显著低于标准剂量组，但其他次要指标相差不大。

总之，对于严重ANCA相关性血管炎患者，血浆置换并不能降低死亡或ESKD的发生率，糖皮质激素减量治疗亦不优于标准剂量方案。

附：英文原文

Title: Plasma Exchange and Glucocorticoids in Severe ANCA-Associated Vasculitis

Author: Michael Walsh, M.D., Ph.D.,, Peter A. Merkel, M.D., M.P.H.,, Chen-Au Peh, F.R.A.C.P., Ph.D.,, Wladimir M. Szpirt, M.D.,, Xavier Puéchal, M.D., Ph.D.,, Shouichi Fujimoto, M.D., Ph.D.,, Carmel M. Hawley, M.B., B.S., M.Med.Sci.,, Nader Khalidi, M.D.,, Oliver Flomann, F.R.C.P., M.D.(Res),, Ron Wald, M.D.C.M., M.P.H.,, Louis P. Girard, M.D., M.B.T.,, Adeera Levin, M.D.,, Gina Gregorini, M.D.,, Lorraine Harper, F.R.C.P., Ph.D.,, William F. Clark, M.D.,, Christian Pagnoux, M.D., M.P.H.,, Ulrich Specks, M.D.,, Lucy Smyth, M.B., B.S.,, Vladimir Tesar, M.D., Ph.D.,, Toshiko Ito-Ihara, M.D., Ph.D.,, Janak Rashme de Zoysa, M.B., Ch.B.,, Wojciech Szczeklik, M.D., Ph.D.,, Luis Felipe Flores-Suárez, M.D., Ph.D.,, Simon Carette, M.D.,, Loc Guillevin, M.D.,, Charles D. Pusey, F.Med.Sci.,, Alina L. Casian, M.D.,, Biljana Brezina, M.Sc.,, Andrea Mazzetti, B.A.,, Carol A. McAlear, M.A.,, Elizabeth Broadhurst, B.Sc.,, Donna Reidlinger, M.P.H.,, Samir Mehta, M.Sc.,, Natalie Ives, M.Sc.,, and David R.W. Jayne, M.D.

Issue&Volume: 2020-02-12

Abstract:

Background
More effective and safer treatments are needed for antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis.

Methods
We conducted a randomized trial with a 2-by-2 factorial design to evaluate the use of plasma exchange and two regimens of oral glucocorticoids in patients with severe ANCA-associated vasculitis (defined by an estimated glomerular filtration rate of <50 ml per minute per 1.73 m2 of body-surface area or diffuse pulmonary hemorrhage). Patients were randomly assigned to undergo plasma exchange (seven plasma exchanges within 14 days after randomization) or no plasma exchange (control group). Patients were also randomly assigned to follow either a standard-dose regimen or a reduced-dose regimen of oral glucocorticoids. Patients were followed for up to 7 years for the primary composite outcome of death from any cause or end-stage kidney disease (ESKD).

Results
Death from any cause or ESKD occurred in 100 of 352 patients (28.4%) in the plasma-exchange group and in 109 of 352 patients (31.0%) in the control group (hazard ratio, 0.86; 95% confidence interval [CI], 0.65 to 1.13; P=0.27). The results were similar in subgroup analyses and in analyses of secondary outcomes. We also assessed the noninferiority of a reduced-dose regimen of glucocorticoids to a standard-dose regimen, using a noninferiority margin of 11 percentage points. Death from any cause or ESKD occurred in 92 of 330 patients (27.9%) in the reduced-dose group and in 83 of 325 patients (25.5%) in the standard-dose group (absolute risk difference, 2.3 percentage points; 90% CI, ?3.4 to 8.0), which met the criterion for noninferiority. Serious infections at 1 year were less common in the reduced-dose group than in the standard-dose group (incidence rate ratio, 0.69; 95% CI, 0.52 to 0.93), but other secondary outcomes were similar in the two groups.

Conclusions
Among patients with severe ANCA-associated vasculitis, the use of plasma exchange did not reduce the incidence of death or ESKD. A reduced-dose regimen of glucocorticoids was noninferior to a standard-dose regimen with respect to death or ESKD.

DOI: 10.1056/NEJMoa1803537

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa1803537