Long-Term Evolution of Multiple Sclerosis Disability in the Treatment Era
Long-Term Evolution of Multiple Sclerosis Disability in the Treatment Era
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DOI：
10.1002/ana.24747
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发表时间：
2016-10-01
影响因子：
11.2
通讯作者：
Hauser, Stephen L.
中科院分区：
文献类型：
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作者：
Cree, Bruce A. C.;Gourraud, Pierre-Antoine;Hauser, Stephen L.
目的：为了描述积极治疗的多发性硬化（MS）患者队列中长期残疾的累积，并评估临床试验中使用的临床和磁共振成像（MRI）数据是否具有长期预后价值。方法：这是一项前瞻性研究，纳入了517例在单一中心积极治疗的MS患者。超过91%的患者被保留，数据在基线访视后长达10年。在最后一次评估中，与基线相比，41%的患者通过扩展残疾状态量表（EDSS）测量的神经功能残疾稳定或改善。根据临床和MRI标准，前2年内无疾病活动性证据（NEDA）的受试者的长期结局与整个队列的结局没有差异。25-OH维生素D血清水平与短期MS疾病活动呈负相关;然而，这些水平与长期残疾无关。在发病后16.8年的中位时间，（95%置信区间[CI] 57.2 - 14%）患者达到EDSS>= 6，18.1%（95% CI = 13.5 - 22.5%）从复发性MS演变为继发性进展性MS（SPMS）。与早期的自然史研究相比，恶化和演变为SPMS的发生率显著降低。值得注意的是，NEDA 2年终点不是长期稳定性的预测因子。最后，这些数据对每年MRI评估作为MS护理的靶向治疗方法的实用性提出了质疑。
Objective: To characterize the accrual of long-term disability in a cohort of actively treated multiple sclerosis (MS) patients and to assess whether clinical and magnetic resonance imaging (MRI) data used in clinical trials have long-term prognostic value.Methods: This is a prospective study of 517 actively managed MS patients enrolled at a single center.Results: More than 91% of patients were retained, with data ascertained up to 10 years after the baseline visit. At this last assessment, neurologic disability as measured by the Expanded Disability Status Scale (EDSS) was stable or improved compared to baseline in 41% of patients. Subjects with no evidence of disease activity (NEDA) by clinical and MRI criteria during the first 2 years had long-term outcomes that were no different from those of the cohort as a whole. 25-OH vitamin D serum levels were inversely associated with short-term MS disease activity; however, these levels had no association with long-term disability. At a median time of 16.8 years after disease onset, 10.7% (95% confidence interval [CI] 57.2-14%) of patients reached an EDSS >= 6, and 18.1% (95% CI=13.5-22.5%) evolved from relapsing MS to secondary progressive MS (SPMS).Interpretation: Rates of worsening and evolution to SPMS were substantially lower when compared to earlier natural history studies. Notably, the NEDA 2-year endpoint was not a predictor of long-term stability. Finally, the data call into question the utility of annual MRI assessments as a treat-to-target approach for MS care.