背景:Susac综合征(SuS)是一种罕见的以脑病为特征的疾病,听力障碍和视觉障碍。免疫抑制治疗是基于自身免疫性内皮病驱动疾病的假设而使用的。然而,缺乏可靠的循证治疗方法.这篇综述的目的是提供患者特征的概述,自2013年以来文献报道的与成功结局相关的病程和治疗模式.
方法:三位审稿人在2022年2月进行了系统的文献检索。主要结果是使用的治疗,来自分类为可能或明确的SuS的病例,描述成功的治疗结果(即≥1个月没有疾病活动迹象)。次要结果是复发时间和随访时间。包括已发布的病例报告和病例系列。提取各种临床特征和治疗并分类为不同的治疗阶段。
结果:共识别出810条记录。120篇符合纳入标准,抽取161例。其中,151例被归类为可能或明确的SuS,并包括在最终分析中。每个治疗阶段使用的治疗组合数量为:6个经验,35确诊后,43用于维持治疗,22复发后,18在复发后维持期间。中位随访时间为12.3个月(0.5;120),中位复发时间为4个月(1;120)。
结论:本范围综述总结了SuS患者的治疗方法,突出可变性。国际努力收集临床,需要注册的SuS患者的影像学和治疗数据,为了提供关于治疗反应的较少偏见和长期随访信息,复发和患者预后的预测因素。这可能导致更多基于证据的治疗方法。
BACKGROUND: Susac syndrome (SuS) is a rare disease characterized by encephalopathy, hearing impairment and visual disturbances. Immunosuppressive treatments are used based on the hypothesis that an autoimmune endotheliopathy drives the disease. However, a solid evidence-based treatment approach is lacking. The aim of this
review is to provide an overview of patient characteristics, disease course and treatment patterns related to successful outcome that have been reported in literature since 2013.
METHODS: Three reviewers conducted a systematic literature search in February 2022. The primary outcome was treatment used, derived from cases classified as probable or definite SuS, describing successful treatment outcome (i.e. no signs of disease activity for ≥ 1 month). Secondary outcomes were time-to-relapse and follow-up time. Published case reports and case series were included. Various clinical characteristics and treatment(s) were extracted and categorized into different phases of treatment.
RESULTS: A total of 810 records was identified. 120 articles met inclusion criteria and 161 cases were extracted. Of these, 151 cases were classified as probable or definite SuS and included in the final analysis. Number of combinations of treatments used per treatment phase were: 6 empirically, 35 after confirmed diagnosis, 43 for maintenance treatment, 22 after relapse, 18 during maintenance post-relapse. Median follow-up time was 12.3 months (0.5; 120) and median time to relapse was 4 months (1; 120).
CONCLUSIONS: This scoping
review summarizes treatment approaches in patients with SuS, highlighting variability. International efforts to collect clinical, imaging and treatment data from patients with SuS in registries are needed, in order to provide less biased and long-term follow-up information on treatment response, predictors of relapse and patient outcomes. This may lead to more evidence-based therapeutic approaches.