Overview
This multicenter prospective observational cohort study aims to develop and validate individualized risk prediction models for stroke and cognitive decline in patients with moyamoya disease or moyamoya syndrome by integrating clinical characteristics, laboratory data, multimodal neuroimaging, genetic information, and other available multidimensional data. Approximately 3,000 participants will be enrolled and followed for up to 24 months.
Description
This is a multicenter prospective observational cohort study enrolling approximately 3,000 patients with moyamoya disease or moyamoya syndrome.
At baseline, demographic and clinical characteristics, neurological and cognitive assessments, laboratory data, multimodal neuroimaging, and peripheral blood samples will be collected according to the study protocol. Baseline vascular imaging will include at least one analyzable CTA, MRA, or DSA examination. Additional structural MRI and perfusion imaging, including CTP, ASL, or DSC, will be collected when available.
The study does not assign treatment. Participants will receive conservative management or cerebral revascularization according to their clinical condition, current standards of care, and shared decision-making between treating physicians and patients.
Participants will be followed at 3-6 months, 12 months, and 24 months after enrollment. The primary outcome is incident stroke within 24 months. Secondary outcomes include stroke at earlier time points, transient ischemic attack, functional deterioration, mortality, cognitive change assessed by the Montreal Cognitive Assessment, disease progression, and subsequent cerebral revascularization among participants initially managed conservatively.
Prediction models for ischemic stroke, intracerebral hemorrhage, and cognitive decline will be developed and validated using baseline clinical, imaging, laboratory, genetic, and other available multidimensional data. Twelve-month prediction and dynamically updated models will be considered secondary or exploratory analyses.
Eligibility
Inclusion Criteria
- Age 12 to 70 years.
- Diagnosis of moyamoya disease or moyamoya syndrome by DSA, CTA or MRA.
- Ability to complete neurological and cognitive assessments.
- Ability to complete at least 12 months of clinical follow-up and willingness, in principle, to complete 24 months of follow-up.
- At least one analyzable baseline vascular imaging examination in original DICOM format, including CTA, MRA, or DSA.
- Written informed consent provided by the participant and/or legally authorized representative.
Exclusion Criteria
- Refusal to participate or inability to complete follow-up.
- Pregnancy or breastfeeding.
- Coexisting cerebrovascular malformation, intracranial tumor, hydrocephalus, or another disorder that may independently affect stroke or cognitive outcomes.
- Severe visual impairment, aphasia, impaired consciousness, psychiatric illness, or another disorder that prevents valid cognitive assessment, including Alzheimer's disease, Parkinson's disease, or frontotemporal dementia.
- Cardiac conditions that may cause cardioembolic stroke, including atrial fibrillation, patent foramen ovale or other right-to-left shunting cardiac disease, intracardiac mural thrombus, or infective endocarditis; or severe hepatic or renal dysfunction, coagulation disorders, malignancy, or severe cardiopulmonary disease.
- Receipt of an investigational drug or device within 30 days before screening, or current participation in another interventional clinical trial that may interfere with this study.
- Previous cerebral revascularization surgery.
