Impact of Acute ITTP Therapies on Long Term Neurologic and Cognitive Outcomes in ITTP Survivors
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 116
- 试验地点
- 1
- 主要终点
- Cognitive impairment
研究概览
简要总结
- We expect to find that the silent cerebral infarct (SCI) rate is two fold higher in patients treated without caplacizumab. We also expect to find that the rate of mild and major cognitive impairment in patients treated with caplacizumab within 3 days of starting plasma exchange will be lower than patients treated without caplacizumab.
- We expect that the differences in cognitive impairment in cases (caplacizumab) versus controls (no caplacizumab) will persist on serial evaluation 1 year later. We also expect that there will be differences in these groups even after adjusting for time since episode and severity of presentation.
- We expect to find that SCI and cognitive impairment is associated with worse scores on the health related quality of life instrument (SF-36)
- Based on studies in non-TTP populations, we expect to find that the rate of incident stroke over the period of follow up is at least 2 fold higher in patients that have SCI compared with patients who do not have SCI
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age >= 18 years
- •Confirmed iTTP based on ADAMS13 activity < 10 % (or 10-20% with positive inhibitor or antibody) during an acute iTTP episode
- •Only 1 episode of iTTP that was treated with plasma exchange and caplacizumab started within 3 days of diagnosis (or if more than 1 episode, then all episodes treated with plasma exchange and caplacizumab started within 3 days of diagnosis)
排除标准
- •Any contraindication for MRI (metallic implants, shrapnel, MRI incompatible stents, etc)
- •Unable to speak, read or understand instructions in English (for NIH ToolBox)
- •Combination of iTTP episodes treated without caplacizumab or with caplacizumab.
- •Caplacizumab started at >= 4 days from diagnosis or for refractory iTTP
研究组 & 干预措施
Cases
干预措施: Caplacizumab (Drug)
结局指标
主要结局
Cognitive impairment
时间窗: 12 months
Cognitive impairment (co-primary endpoint) - measured using NIH ToolBox Cognition Battery. Mild and major cognitive impairment are defined as T scores that are 1-2 SD below mean and \> 2SD below mean for any domain, respectively
Silent cerebral infarction
时间窗: 12 months
Silent cerebral infarction (primary endpoint) - SCI is derived from quantitation (number and total volume) of ischemic (infarct-like) lesions shown as foci of T2 and FLAIR hyperintensity. SCI is diagnosed if the participant has an infarct like lesion, at least 3 mm, with normal neurologic examination or an abnormality on examination that is not explained by the location of the brain lesion. We have assembled a panel of 3 neuroradiologists headed by Dr. Doris Lin. Each MRI will be read by 2 radiologists. A third reviewer will serve as a tie breaker in case of disagreement.
次要结局
- SF-36 scores for quality of life(12 months)
- Patient reported cognitive performance(12 months)
- Depression scores on BDI-II(12 months)