Koelman Diederik L H, Chahin Salim, Mar Soe S, Venkatesan Arun, Hoganson George M, Yeshokumar Anusha K, Barreras Paula, Majmudar Bittu, Klein Joshua P, Chitnis Tanuja, Benkeser David C, Carone Marco, Mateen Farrah J
From the Department of Neurology (D.L.H.K., T.C., F.J.M.), Massachusetts General Hospital, Boston; Department of Neurology (D.L.H.K.), Academic Medical Center, Amsterdam, the Netherlands; Department of Neurology (S.C.), University of Pennsylvania, Philadelphia; Department of Neurology (S.S.M., G.M.H., B.M.), Washington University School of Medicine, St. Louis, MO; Department of Neurology (A.V., A.K.Y., P.B.), Johns Hopkins University School of Medicine, Baltimore, MD; Department of Neurology (J.P.K.), Brigham and Women's Hospital, Boston; Harvard Medical School (J.P.K., F.J.M.), Boston, MA; and Department of Biostatistics (D.C.B., M.C.), University of Washington, Seattle.
Neurology. 2016 May 31;86(22):2085-93. doi: 10.1212/WNL.0000000000002723. Epub 2016 May 4.
To analyze the range of demographic, clinical, MRI, and CSF features of acute disseminated encephalomyelitis (ADEM), a rare, typically monophasic demyelinating disorder, and analyze long-term outcomes including time and risk factors for subsequent clinical events as well as competing diagnoses.
We performed a retrospective, multicenter study in 4 US academic medical centers of all patients clinically diagnosed with ADEM. Initial presentation of pediatric and adult ADEM and monophasic and multiphasic disease were compared. The Aalen-Johansen estimator was used to produce estimates of the probability of transitioning to a multiphasic diagnosis as a function of time since initial diagnosis, treating death and alternative diagnoses as competing risks.
Of 228 patients (122 children, age range 1-72 years, 106 male, median follow-up 24 months [25th-75th percentile 6-67], 7 deaths), approximately one quarter (n = 55, 24%) experienced at least one relapse. Relapsing disease in children was more often diagnosed as multiphasic ADEM than in adults (58% vs 21%, p = 0.007), in whom MS was diagnosed more often. Encephalopathy at initial presentation (hazard ratio [HR] 0.383, p = 0.001), male sex (HR 0.394, p = 0.002), and increasing age at onset (HR 0.984, p = 0.035) were independently associated with a longer time to a demyelinating disease relapse in a multivariable model. In 17 patients, diagnoses other than demyelinating disease were concluded in long-term follow-up.
Relapsing disease after ADEM is fairly common and associated with a few potentially predictive features at initial presentation. Age-specific guidelines for ADEM diagnosis and treatment may be valuable, and vigilance for other, mostly rare, diseases is imperative.
分析急性播散性脑脊髓炎（ADEM）这一罕见的、通常为单相脱髓鞘疾病的人口统计学、临床、磁共振成像（MRI）及脑脊液特征范围，并分析长期预后，包括后续临床事件发生的时间和风险因素以及鉴别诊断。
我们在美国4家学术医疗中心对所有临床诊断为ADEM的患者进行了一项回顾性多中心研究。比较了儿童和成人ADEM的初始表现以及单相和多相疾病。使用Aalen-Johansen估计量来估计自初始诊断以来随时间转变为多相诊断的概率，将死亡和替代诊断视为竞争风险。
在228例患者中（122例儿童，年龄范围1至72岁，106例男性，中位随访24个月[第25至75百分位数为6至67]，7例死亡），约四分之一（n = 55，24%）经历了至少一次复发。儿童复发性疾病被诊断为多相ADEM的情况比成人更常见（58%对21%，p = 0.007），成人中更常诊断为多发性硬化（MS）。在多变量模型中，初始表现为脑病（风险比[HR] 0.383，p = 0.001）、男性（HR 0.394，p = 0.002）以及发病年龄增加（HR 0.984，p = 0.035）与脱髓鞘疾病复发时间延长独立相关。在17例患者中，长期随访得出了脱髓鞘疾病以外的诊断结论。
ADEM后复发性疾病相当常见，且与初始表现时的一些潜在预测特征相关。针对ADEM诊断和治疗的年龄特异性指南可能很有价值，并且必须警惕其他大多数罕见疾病。
