BACKGROUND Cerebellar and brainstem symptoms are common in early stages of multiple sclerosis (MS) yet their prognostic values remain unclear. OBJECTIVE The aim of this study was to investigate long-term disability outcomes in patients with early cerebellar and brainstem symptoms. METHODS This study used data from MSBase registry. Patients with early cerebellar/brainstem presentations were identified as those with cerebellar/brainstem relapse(s) or functional system score ⩾ 2 in the initial 2 years. Early pyramidal presentation was chosen as a comparator. Andersen-Gill models were used to compare cumulative hazards of (1) disability progression events and (2) relapses between patients with and without early cerebellar/brainstem symptoms. Mixed effect models were used to estimate the associations between early cerebellar/brainstem presentations and expanded disability status scale (EDSS) scores. RESULTS The study cohort consisted of 10,513 eligible patients, including 2723 and 3915 patients with early cerebellar and brainstem symptoms, respectively. Early cerebellar presentation was associated with greater hazard of progression events (HR = 1.37, p < 0.001) and EDSS (β = 0.16, p < 0.001). Patients with early brainstem symptoms had lower hazard of progression events (HR = 0.89, p = 0.01) and EDSS (β = -0.06, p < 0.001). Neither presentation was associated with changes in relapse risk. CONCLUSION Early cerebellar presentation is associated with unfavourable outcomes, while early brainstem presentation is associated with favourable prognosis. These presentations may be used as MS prognostic markers and guide therapeutic approach.

中文翻译：

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多发性硬化症早期小脑和脑干症状的残疾结果

背景 小脑和脑干症状在多发性硬化症 (MS) 的早期阶段很常见，但它们的预后价值仍不清楚。目的 本研究的目的是调查有早期小脑和脑干症状的患者的长期残疾结果。方法 本研究使用来自 MSBase 注册中心的数据。早期小脑/脑干表现的患者被确定为在最初 2 年内有小脑/脑干复发或功能系统评分 ⩾ 2 的患者。选择早期金字塔形呈现作为比较。Andersen-Gill 模型用于比较 (1) 残疾进展事件和 (2) 有和没有早期小脑/脑干症状的患者复发的累积风险。混合效应模型用于估计早期小脑/脑干表现与扩展残疾状态量表 (EDSS) 评分之间的关​​联。结果 该研究队列由 10,513 名符合条件的患者组成，分别包括 2723 名和 3915 名具有早期小脑和脑干症状的患者。早期小脑表现与更大的进展事件风险（HR = 1.37，p < 0.001）和 EDSS（β = 0.16，p < 0.001）相关。具有早期脑干症状的患者发生进展事件（HR = 0.89，p = 0.01）和 EDSS（β = -0.06，p < 0.001）的风险较低。两种表现均与复发风险的变化无关。结论 早期小脑表现与不良结局相关，而早期脑干表现与良好预后相关。