Observational Study on Patients With Rasmussen Encephalitis Treated With Adalimumab: Efficacy and Tolerance in the Short and Long Term
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 16
- 主要终点
- Efficiency of the treatment measured by the reduction of the number of epileptic seizures
研究概览
简要总结
Since the initial study of cases reported between 2009 and 2015, adalimumab has become in France the immunomodulatory reference treatment used after failure of corticosteroids and immunoglobulins before a possible recourse to the hemispherotomy. This observational study is intended to document the long-term efficacy and safety of Adalimumab therapy in patients with Rasmussen encephalitis.
详细描述
Rasmussen Encephalitis (ER) is a particularly severe chronic inflammatory brain disorder resulting in the progressive destruction of a hemisphere. It is a rare disease although at present no precise prevalence is available. It begins preferentially in children.
This inflammatory process is accompanied by a progressive loss of function of the affected hemisphere, associated with a pharmaco-resistant partial epilepsy. The diagnosis is based on a bundle of clinical, radiological and electroencephalographic arguments. CSF analysis directs diagnosis in 50% of cases.
No anti-epileptic treatment can stop seizures. Only hemispherotomy (surgical disconnection of a cerebral hemisphere) allows it but it is associated with definitive motor and cognitive deficits.
Over the last 20 years, new therapeutic trials have focused on immuno-modulatory treatments targeted at the T-lymphocyte pathway, including tacrolimus. Although they seem to be more effective than immunoglobulins or corticosteroids, it remains transient. In addition, the number of published cases is low. In this context, starting in 2009, it has been proposed to use adalimumab (Ab anti TNF) based on:
- 1 / study of a case index
- 2 / knowledge of the pathophysiology of Rasmussen Encephalitis. To date, very few data provide precise information on the efficacy or tolerance of the use of this product in the longer term. This information is essential to confirm the place of adalimumab in the therapeutic arsenal against Rasmussen encephalitis.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of Rasmussen Encephalitis
- •Patient aged more than 2 years
- •Compliant treatment with adalimumab
排除标准
- •Patient with a differential diagnosis
- •Patient suffering from Rassmussen Encephalitis but not treated with adalimumab
- •Patient who has not signed the informed consent
结局指标
主要结局
Efficiency of the treatment measured by the reduction of the number of epileptic seizures
时间窗: 5 years
The change of epileptic seizures (Will be considered as a responder patient any patient with a 50% decrease in the number of seizures between the time of inclusion and 6 months after the start of treatment). This response to treatment is re-evaluated at 12 months then every 6 months for 5 years.
Efficiency of the treatment measured by the stability if the cognitive assessment
时间窗: 6 months
Stability of the cognitive assessment: variation of less than 10 points on each of the different composite indices (QIT, ICV, IRF, IMT, IVT) of age-appropriate Weschler scales, between two cognitive assessments of 6 months.
次要结局
未报告次要终点