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儿童多发性硬化症队列中早期严重程度的预后因素。

Prognostic factors for early severity in a childhood multiple sclerosis cohort.

作者信息

Mikaeloff Yann, Caridade Guillaume, Assi Saada, Suissa Samy, Tardieu Marc

机构信息

Service de Neurologie Pédiatrique, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris, INSERM U802, Paris, France.

出版信息

Pediatrics. 2006 Sep;118(3):1133-9. doi: 10.1542/peds.2006-0655.

Abstract

OBJECTIVE

The goal was to identify prognostic factors for an early severe course in a cohort of patients with childhood-onset multiple sclerosis, for the construction of a predictive tool.

METHODS

The cohort consisted of 197 children from the French Kid Sclérose en Plaques neuropediatric cohort with relapsing/remitting multiple sclerosis beginning before the age of 16 years. Patients were included from 1990 to 2003. We used multivariate survival analysis (Cox model) to evaluate the prognostic value of clinical, MRI, and biological covariates at onset for the occurrence of a third attack or severe disability ("severity" outcome).

RESULTS

The cohort was monitored for a mean of 5.5 +/- 3.6 years. The "severity" outcome was recorded for 144 patients (73%). The risk of severity was higher for girls, for a time between the first and second attacks of < 1 year, for childhood-onset multiple sclerosis MRI criteria at onset, for an absence of severe mental state changes at onset, and for a progressive course. A derived childhood-onset multiple sclerosis potential index for early severity was found to have a positive predictive value for severity of > 35% for the upper 2 quartiles.

CONCLUSIONS

The clinical and MRI prognostic factors for early severity that were identified were used as the basis of a predictive tool, which will be validated in another cohort. This tool should make it possible to identify subgroups at risk of early severe disease and should facilitate therapeutic studies.

摘要

目的

本研究旨在确定儿童期起病的多发性硬化症患者早期病情严重的预后因素,以构建一种预测工具。

方法

该队列由来自法国儿童多发性硬化症神经儿科队列的197名儿童组成,这些儿童患有复发/缓解型多发性硬化症,发病年龄在16岁之前。患者纳入时间为1990年至2003年。我们使用多变量生存分析(Cox模型)来评估发病时临床、MRI和生物学协变量对第三次发作或严重残疾(“严重程度”结局)发生的预后价值。

结果

该队列平均随访5.5±3.6年。144名患者(73%)记录到了“严重程度”结局。女孩、首次发作与第二次发作间隔时间<1年、发病时符合儿童期起病的多发性硬化症MRI标准、发病时无严重精神状态改变以及病程呈进展性的患者,病情严重的风险更高。发现一种推导得出的儿童期起病的多发性硬化症早期严重程度潜在指数,对于上两个四分位数的严重程度具有>35%的阳性预测价值。

结论

所确定的早期严重程度的临床和MRI预后因素被用作一种预测工具的基础,该工具将在另一个队列中进行验证。此工具应能够识别有早期严重疾病风险的亚组,并应有助于开展治疗研究。

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