Healy Brian C, Glanz Bonnie I, Swallow Elyse, Signorovitch James, Hagan Kaitlin, Silva Diego, Pelletier Corey, Chitnis Tanuja, Weiner Howard
Brigham and Women's Hospital, Boston, MA, USA.
Analysis Group, Boston, MA, USA.
Mult Scler J Exp Transl Clin. 2021 Apr 11;7(2):2055217321999070. doi: 10.1177/2055217321999070. eCollection 2021 Apr-Jun.
Although confirmed disability progression (CDP) is a common outcome in multiple sclerosis (MS) clinical trials, its predictive value for long-term outcomes is uncertain.
To investigate whether CDP at month 24 predicts subsequent disability accumulation in MS.
The Comprehensive Longitudinal Investigation of Multiple Sclerosis at Brigham and Women's Hospital includes participants with relapsing-remitting MS or clinically isolated syndrome with Expanded Disability Status Scale (EDSS) scores ≤5 (N = 1214). CDP was assessed as a predictor of time to EDSS score 6 (EDSS 6) and to secondary progressive MS (SPMS) using a Cox proportional hazards model; adjusted models included additional clinical/participant characteristics. Models were compared using Akaike's An Information Criterion.
CDP was directionally associated with faster time to EDSS 6 in univariate analysis (HR = 1.61 [95% CI: 0.83, 3.13]). After adjusting for month 24 EDSS, CDP was directionally associated with slower time to EDSS 6 (adjusted HR = 0.65 [0.32, 1.28]). Models including CDP had worse fit statistics than those using EDSS scores without CDP. When models included clinical and magnetic resonance imaging measures, T2 lesion volume improved fit statistics. Results were similar for time to SPMS.
CDP was less predictive of time to subsequent events than other MS clinical features.
尽管确诊的残疾进展(CDP)在多发性硬化症(MS)临床试验中是常见的结果,但其对长期预后的预测价值尚不确定。
研究24个月时的CDP是否能预测MS患者随后的残疾累积情况。
布莱根妇女医院的多发性硬化症综合纵向研究纳入了复发缓解型MS或临床孤立综合征且扩展残疾状态量表(EDSS)评分≤5的参与者(N = 1214)。使用Cox比例风险模型评估CDP作为达到EDSS评分6(EDSS 6)和继发进展型MS(SPMS)时间的预测指标;调整后的模型纳入了其他临床/参与者特征。使用赤池信息准则比较模型。
在单变量分析中,CDP与达到EDSS 6的时间更快呈方向性相关(HR = 1.61 [95% CI:0.83, 3.13])。在调整24个月时的EDSS后,CDP与达到EDSS 6的时间较慢呈方向性相关(调整后的HR = 0.65 [0.32, 1.28])。包含CDP的模型的拟合统计量比不包含CDP的EDSS评分模型更差。当模型纳入临床和磁共振成像测量指标时,T2病变体积改善了拟合统计量。对于达到SPMS的时间,结果相似。
与其他MS临床特征相比,CDP对后续事件发生时间的预测性较差。