High Dose Steroids in Children With Stroke and Unilateral Focal Arteriopathy: Paediatric Arteriopathy Steroid Aspirin (PASTA) Project. A Multicentre Randomized Trial.
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 主要终点
- Time to recovery up to 12 months
研究概览
简要总结
Arterial ischemic stroke (AIS) is a devastating condition, affecting 1.6-5/100,000 children/year. Although their outcome is different, children with stroke do not recover better than adults, with at least 2/3 suffering long term sequels such as developmental (motor, global intellectual, language...) and behavioral disabilities, epilepsy, and low adaptative and academic skills...
Stenotic cerebral arteriopathy is identified as AIS etiology in 60-80% of previously healthy children and the course of this arteriopathy is the strongest predictor of recurrent events. 30-40% of these children have a focal unilateral cerebral arteriopathy (FCA). Childhood FCA is suspected to be an inflammatory vessel wall pathology triggered by varicella and other (viral) infections. As recurrences occur for the great majority in the first 6 months after the index event, aspirin 5 mg/kg/day is recommended for at least 18 months to 2 years.
As there is a rational for using immunomodulatory drugs at the acute stage of FCA, immunotherapies are currently used by neuropaediatricians in AIS, mainly as steroids for children with stenosing arteriopathies. However, due to weak evidences, the literature cannot either encourage or discourage this practice.
The long term course of children with FCA is only approach to date by retrospective studies and controversies about outcome remain (for example, the recurrence risk on antithrombotic treatment varies notably from quasi zero to 25%). And finally, it is shown in childhood stroke, as well as in the global field of longstanding impairment, that parental and medical points of view do not match consistently. Longitudinal studies are needed to deserve this familial approach.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
盲法说明
The outcome assessor don't know patient treatment
入排标准
- 年龄范围
- 6 Months 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •- Aged 6 months to <15 years
- •AIS ≤ 48 hours
- •Newly acquired focal neurological deficit with confirmation by magnetic resonance imaging (MRI) of ischaemic lesion in an arterial territory corresponding with the clinical features (definition of Arterial ischemic stroke).
- •Magnetic resonance arteriography showing unilateral proximal stenosis or irregularities of the corresponding carotid trifurcation (i.e. terminal carotid and/or M1-M2 and/or A1 segments) or of the posterior circulation (P1-P2 segments).
- •No evidence of an underlying systemic disorder (e.g. lupus erythematodes) explaining the features.
- •Informed and signed consent of parents or legal guardians.
- •French Social Security (Sécurité sociale; i.e. national health coverage) affiliation
排除标准
- •Children with secondary central nervous system angiitis due to infections (meningitis, endocarditis, borreliosis), rheumatic or other systemic inflammatory diseases (e.g. lupus erythematodes). These children are already under immune suppression or need other co-medications regarding their underlying disease.
- •Children with known syndromal and/or genetic vasculopathies such as phaces syndrome, Neurofibromatosis type 1, trisomy
- •Children with moyamoya or sickle cell disease.
- •Children with a progressive large to medium vessel childhood primary angiitis of the central nervous system with two out of the following three criteria : Children with progressive neurocognitive dysfunction; Children with bilateral lesions/vessel involvement; Children with distal arterial stenosis (beyond the M2, A1 or P2 segment).
- •Children already on steroid treatment at disease onset or with a contraindication to receive steroid treatment (e.g. congenital or acquired immunodeficiency).
- •Children with delayed diagnosis ≥3 days as treatment start is not allowed to be more ≥5 day-delayed.
- •Contraindications to steroids (see also summary of product characteristics in chapter 1.1) and notably: Not-manageable infectious, hydro-electrolytic or metabolic (e.g. diabetes mellitus) disorders, or elevated blood pressure, Serious behavioral disorders, Current vaccination with live or attenuated live strains, Allergy/sensibility to any ingredient, Association with some medications such as antiarrhythmic drugs.
研究组 & 干预措施
experimental group
Children will be treated by methylprednisolone + prednisolone and standard of care
干预措施: Methylprednisolone + prednisolone (Drug)
结局指标
主要结局
Time to recovery up to 12 months
时间窗: Up to 12 months
Evaluate of time to recovery up to 12 months by score paediatric Recurrence and Recovery Questionnaire (RRQ )
次要结局
- Improvement of functional outcome by face-to-face visits(Months:1,6,12, 24 and 36)
- Recurrence of stroke, epilepsy, neurological and developmental sequels, and academic achievement(Months: 1, 3, 6,12, 24 and 36)
- Number of serious adverse events(Months 72)
- Arteriopathic course along time(Months: 1, 6, 24)
- Outcome by age group(Months 72)
- Familial impact(Months 72)