Department of Neurology, University of California, San Francisco, San Francisco, CA, USA.
Department of Neurology, The University of Chicago, Chicago, IL, USA.
Mult Scler. 2019 May;25(6):837-847. doi: 10.1177/1352458518773511. Epub 2018 May 15.
Long-term follow-up from the randomized trial of interferon beta-1b (IFNB-1b) permitted the assessment of different definitions of no evidence of disease activity (NEDA) for predicting long-term outcome in multiple sclerosis (MS).
To examine the predictive validity of different NEDA definitions.
Predictive validity for negative disability outcomes (NDOs) at 16 years and survival at 21 years post-randomization were assessed. NEDA in the first 2 years was defined as follows: clinical NEDA: no relapses or Expanded Disability Status Scale (EDSS) progression from baseline to Year 2; NEDA-3a: no relapses, no confirmed ⩾1-point EDSS progression, and no new T2-active lesions; NEDA-3b: no relapses, no EDSS progression, and no increase in T2 burden of disease (T2-BOD); and NEDA-4: no relapses, no EDSS progression, and no increase in T2-BOD or atrophy. NDOs were defined as death, need for wheelchair, EDSS ⩾6, or progressive MS.
A total of 245 and 371 patients were evaluated at 16 and 21 years, respectively. Clinical NEDA predicted NDOs ( p = 0.0029), as did baseline EDSS ( p < 0.0001), baseline T2-BOD ( p < 0.0001), and change in T2-BOD ( p = 0.0033). IFNB-1b treatment ( p = 0.0251), relapse rate in the 2 years before study start ( p = 0.0260), T2-BOD at baseline ( p = 0.0014), and change in T2-BOD ( p = 0.0129) predicted survival at 21 years.
Clinical NEDA predicted long-term disability outcome. By contrast, definitions of NEDA that included on-therapy changes in magnetic resonance imaging variables did not increase the predictive validity.
干扰素β-1b（IFNB-1b）的随机试验的长期随访使我们能够评估不同的无疾病活动（NEDA）定义，以预测多发性硬化症（MS）的长期结果。
研究不同 NEDA 定义的预测有效性。
评估了 16 年时的阴性残疾结局（NDO）和 21 年后的生存预测的预测有效性。前 2 年的 NEDA 定义如下：临床 NEDA：无复发或扩展残疾状态量表（EDSS）从基线到第 2 年的进展；NEDA-3a：无复发，无确诊 ⩾1 点 EDSS 进展，无新的 T2 活跃病变；NEDA-3b：无复发，无 EDSS 进展，无 T2 疾病负担（T2-BOD）增加；和 NEDA-4：无复发，无 EDSS 进展，且 T2-BOD 或萎缩无增加。NDO 定义为死亡、需要轮椅、EDSS ⩾6 或进展性 MS。
共有 245 名和 371 名患者分别在 16 年和 21 年进行了评估。临床 NEDA 预测了 NDO（p=0.0029），基线 EDSS（p<0.0001）、基线 T2-BOD（p<0.0001）和 T2-BOD 变化（p=0.0033）也是如此。IFNB-1b 治疗（p=0.0251）、研究开始前 2 年的复发率（p=0.0260）、基线时的 T2-BOD（p=0.0014）和 T2-BOD 的变化（p=0.0129）预测了 21 年的生存。
临床 NEDA 预测了长期残疾结局。相比之下，包括治疗期间磁共振成像变量变化的 NEDA 定义并没有增加预测的有效性。
