Thoracic Disease Research Unit, Division of Pulmonary and Critical Care Medicine, Mayo Clinic Rochester, MN, USA.
European University of Brittany and Brest University Hospital, Brest, France.
Nephrol Dial Transplant. 2017 Jan 1;32(suppl_1):i37-i47. doi: 10.1093/ndt/gfw384.
The prognosis of the antineutrophil cytoplasmic antibody associated vasculitides (AAV), microscopic polyangiitis (MPA), granulomatosis with polyangiitis (GPA) and eosinophilic GPA (EGPA), has been fundamentally improved over the last five decades by the use of glucocorticoids and immunosuppressants, turning them from consistently fatal diseases into chronic conditions. The long-term course is now largely determined by the frequency of disease flares and by accruing damage caused by disease activity and treatment-related comorbidities. This review summarizes the evidence derived from clinical trials performed during the last 30 years and the remaining clinical unmet needs that new studies aim to address. In MPA and GPA, ongoing studies assess (i) different strategies to reduce cumulative glucocorticoid doses currently used for induction and maintenance of remission, (ii) the efficacy of new drugs and (iii) the optimal duration of immunosuppression and the use of biomarkers to individualize therapy. Prospective randomized trials also target disease-associated cardiovascular risk and infections. The first prospective controlled trials specifically designed for EGPA have recently been launched and could lead to new therapeutic options for patients diagnosed with this rare disease. This is an exciting time for researchers in the field of AAV, and for patients as collaborative efforts raise the hope of developing new therapies and more individualized approaches to the management of the diseases, maximizing efficacy while minimizing treatment toxicities.
抗中性粒细胞胞浆抗体相关性血管炎（AAV）、显微镜下多血管炎（MPA）、肉芽肿性多血管炎（GPA）和嗜酸性肉芽肿性多血管炎（EGPA）的预后在过去五十年中通过使用糖皮质激素和免疫抑制剂得到了根本改善，使这些疾病从致命性疾病转变为慢性疾病。目前，长期病程主要取决于疾病复发的频率以及由疾病活动和治疗相关合并症引起的累积损伤。本文综述了过去 30 年进行的临床试验中获得的证据，以及新研究旨在解决的尚未满足的临床需求。在 MPA 和 GPA 中，正在进行的研究评估了（i）不同策略以减少目前用于诱导和维持缓解的累积糖皮质激素剂量，（ii）新药的疗效，以及（iii）最佳免疫抑制持续时间和使用生物标志物进行个体化治疗。前瞻性随机试验还针对与疾病相关的心血管风险和感染。最近专门为 EGPA 设计的前瞻性对照试验已经启动，这可能为诊断为这种罕见疾病的患者提供新的治疗选择。这是 AAV 领域研究人员和患者的激动人心时刻，因为合作努力带来了开发新疗法和更个体化疾病管理方法的希望，最大限度地提高疗效，同时最小化治疗毒性。
