Santoro Jonathan D, Waltz Michael, Aaen Greg, Belman Anita, Benson Leslie, Gorman Mark, Goyal Manu S, Graves Jennifer S, Harris Yolanda, Krupp Lauren, Lotze Timothy, Mar Soe, Moodley Manikum, Ness Jayne, Rensel Mary, Rodriguez Moses, Schreiner Teri, Tillema Jan-Mendelt, Waubant Emmanuelle, Weinstock-Guttman Bianca, Hurtubise Brigitte F, Roalstad Shelly, Rose John, Casper T Charles, Chitnis Tanuja
From Partners Pediatric Multiple Sclerosis Center (J.D.S., T.C.), Massachusetts General Hospital; Harvard Medical School (J.D.S.), Boston, MA; Pediatric Multiple Sclerosis and Related Disorders Program at Boston Children's Hospital (J.D.S., L.B., M.G.), MA; Children's Hospital Los Angeles (J.D.S.); Keck School of Medicine at the University of Southern California (J.D.S.), Los Angeles; Data Coordinating and Analysis Center (M.W., S.R., J.R., T.C.C.), University of Utah, Salt Lake City; Pediatric Multiple Sclerosis Center (G.A.), Loma Linda University Children's Hospital, CA; Pediatric MS Center at NYU Langone Health (A.B., L.K.), New York, NY; Washington University (M.S.G., S.M.), St. Louis, MO; Pediatric Multiple Sclerosis Center (J.S.G.), University of California San Diego; UAB Center for Pediatric-Onset Demyelinating Disease (Y.H., J.N.), University of Alabama at Birmingham; The Blue Bird Circle Clinic for Multiple Sclerosis (T.L.), Texas Children's Hospital, Baylor College of Medicine, Houston; Mellen Center for Multiple Sclerosis (M.M., M. Rensel), Cleveland Clinic, OH; Mayo Clinic Pediatric Multiple Sclerosis Center (M. Rodriguez, J.-M.T.), Mayo Clinic, Rochester, MN; Rocky Mountain Multiple Sclerosis Center (T.S.), Children's Hospital Colorado, University of Colorado at Denver, Aurora; Pediatric Multiple Sclerosis Center (E.W.), University of California San Francisco; Jacobs Pediatric Multiple Sclerosis Center (B.W.-G.), State University of New York at Buffalo; and Department of Neurology (B.F.H.), Stanford University School of Medicine, Palo Alto, CA.
Neurology. 2020 Sep 29;95(13):e1844-e1853. doi: 10.1212/WNL.0000000000010414. Epub 2020 Jul 20.
To characterize disease severity and distribution of disability in pediatric-onset multiple sclerosis (POMS) and to develop an optimized modeling scale for measuring disability, we performed a multicenter retrospective analysis of disability scores in 873 persons with POMS over time and compared this to previously published data in adults with multiple sclerosis (MS).
This was a retrospective analysis of prospectively collected data collected from 12 centers of the US Network of Pediatric MS Centers. Patients were stratified by the number of years from first symptoms of MS to Expanded Disability Status Scale (EDSS) assessment and an MS severity score (Pediatric Multiple Sclerosis Severity Score [Ped-MSSS]) was calculated per criteria developed by Roxburgh et al. in 2005.
In total, 873 patients were evaluated. In our cohort, 52%, 19.4%, and 1.5% of all patients at any time point reached an EDSS of 2.0, 3.0, and 6.0. Comparison of our Ped-MSSS scores and previously published adult Multiple Sclerosis Severity Scores (MSSS) showed slower progression of Ped-MSSS with increasing gaps between higher EDSS score and years after diagnosis. Decile scores in our POMS cohort for EDSS of 2.0, 3.0, and 6.0 were 8.00/9.46/9.94, 7.86/9.39/9.91, and 7.32/9.01/9.86 at 2, 5, and 10 years, respectively. Notable predictors of disease progression in both EDSS and Ped-MSSS models were ever having a motor relapse and EDSS at year 1. Symbol Digit Modalities Test (SDMT) scores were inversely correlated with duration of disease activity and cerebral functional score.
Persons with POMS exhibit lower EDSS scores compared to persons with adult-onset MS. Use of a Ped-MSSS model may provide an alternative to EDSS scoring in clinical assessment of disease severity and disability accrual.
为了描述儿童多发性硬化症（POMS）的疾病严重程度和残疾分布情况，并开发一种优化的残疾测量模型量表，我们对873例POMS患者的残疾评分进行了多中心回顾性分析，并将其与先前发表的成年多发性硬化症（MS）患者的数据进行比较。
这是一项对前瞻性收集的数据进行的回顾性分析，这些数据来自美国儿童MS中心网络的12个中心。患者根据从MS首次症状到扩展残疾状态量表（EDSS）评估的年数进行分层，并根据Roxburgh等人在2005年制定的标准计算MS严重程度评分（儿童多发性硬化症严重程度评分[Ped-MSSS]）。
总共评估了873例患者。在我们的队列中，在任何时间点，所有患者中有52%、19.4%和1.5%的EDSS分别达到2.0、3.0和6.0。我们的Ped-MSSS评分与先前发表的成人多发性硬化症严重程度评分（MSSS）的比较显示，随着EDSS评分升高和诊断后年数增加，两者差距增大，Ped-MSSS进展较慢。在我们的POMS队列中，EDSS为2.0、3.0和6.0时，在2年、5年和10年的十分位数评分分别为8.00/9.46/9.94、7.86/9.39/9.91和7.32/9.01/9.86。在EDSS和Ped-MSSS模型中，疾病进展的显著预测因素是曾有运动性复发和第1年的EDSS。符号数字模态测试（SDMT）评分与疾病活动持续时间和脑功能评分呈负相关。
与成人发病的MS患者相比，POMS患者的EDSS评分较低。在疾病严重程度和残疾累积的临床评估中，使用Ped-MSSS模型可能为EDSS评分提供一种替代方法。
