A Prospective Observational Study for the Predictors of Clinical Outcomes of Deep Brain Stimulation in Parkinson's Disease
概览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Hong Kong University of Science and Technology
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Blood proteomic profile
概览
简要总结
The goal of this observational study is to identify factors in blood that are associated with response to deep brain stimulation (DBS) surgery in patients with Parkinson's disease. The main questions it aims to answer are:
- What factors in blood (proteins and RNA) are associated with good vs poor response to DBS?
- Are these factors able to predict response to DBS?
- How do these factors change before and after DBS?
Blood and leftover brain tissue (which spontaneously adhere to the surgical instruments) will be taken and routine clinical data (including scores from routine assessments) will be collected from consenting participants who undergo DBS.
详细描述
This is a prospective observational study that investigates the clinical, proteomic, transcriptomic, and genomic profiles that are associated with DBS response with regards to motor symptoms, axial symptoms, non-motor symptoms, change in medications, activities of daily living (ADLs), and quality of life (QOL). The investigators will take blood samples from participants but will not take extra brain tissue; only leftover brain tissue (which is routinely discarded) that spontaneously adheres to surgical instruments will be collected. Study participation will not change the surgical procedure in any way. The investigators also aim to characterize the change in these multiomic profiles before versus after DBS treatment, to evaluate the ability of these multiomic profiles to predict and stratify response to DBS, and to identify modifiable factors to improve response to DBS. Additionally, the investigators will use these multiomic profiles, patient demographics, and clinical presentation to develop a DBS prediction model with advanced explainable AI (XAI) techniques.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of "Clinically Established PD" as defined by the Movement Disorder Society Clinical Diagnostic Criteria for Parkinson's disease (MDS-PD criteria)
- •Referred for DBS according to standard local clinical guidelines 2a. Significant motor complications despite optimized pharmacological treatment 2ai. UPDRS motor score \>30/108 in the off-medication state 2aii. Hoehn and Yahr staging \>2.5/5 in the off-medication state 2b. Dopamine responsive 2bi. \>33% improvement in UPDRS motor score after levodopa administration 2c. Age ≤75 years 2d. No contraindication to surgery or other significant comorbidity with limited life expectancy 2e. No significant psychiatric problems or cognitive impairment 2f. No structural lesions or features suggestive of atypical parkinsonism or other mimickers of idiopathic PD on neuroimaging
排除标准
- •Unwilling to undergo blood sampling for study purposes
- •Evidence of Parkinsonism due to heavy metal exposure
- •History of neurodevelopmental disorder, neurodegenerative disease other than PD, CNS infection, neuroinflammatory disease (e.g. multiple sclerosis, CNS lupus), malignancy within the last 10 years, cerebrovascular accident, HIV infection, systemic autoimmune disease, alcohol dependence or other substance use
- •Unable to pass DBS pre-operative assessment or unwilling to undergo DBS
研究组 & 干预措施
Treatment
Parkinson's disease patients who have received deep brain stimulation
干预措施: DBS (Procedure)
结局指标
主要结局
Blood proteomic profile
时间窗: From pre-operative to 5 years post-operative
Plasma proteins measured using high-throughput ultra-sensitive proteomics platform
Blood transcriptomic profile
时间窗: From pre-operative to 5 years post-operative
Bulk RNA-sequencing of blood cells
Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
时间窗: From pre-operative to 5 years post-operative
Rating scale that measures non-motor experiences of daily living (part I), motor experiences of daily living (part II), motor function via physical examination (part III), and motor complications (part IV) Higher scores indicate a worse outcome Range: Part I: 0-52 Part II: 0-52 Part III: 0-132 Part IV: 0-24
Hoehn and Yahr (H&Y) stage
时间窗: From pre-operative to 5 years post-operative
Staging of overall functional disability in Parkinson's disease Higher score indicates a worse outcome Range: 0-5 Stage 0: No signs of disease Stage 1: Unilateral disease Stage 1.5: Unilateral plus axial involvement Stage 2: Bilateral disease without impairment of balance Stage 2.5: Mild bilateral disease with recovery on pull test Stage 3: Mild to moderate bilateral disease; some postural instability; physically independent Stage 4: Severe disability; still able to walk / stand unassisted Stage 5: Wheelchair bound or bedridden unless aided
次要结局
- Montreal Cognitive Assessment (MoCA)(From pre-operative to 5 years post-operative)
- Non-Motor Symptoms Scale (NMSS)(From pre-operative to 5 years post-operative)
- Parkinson's Disease Sleep Scale - 2 (PDSS-2)(From pre-operative to 5 years post-operative)
- Parkinson's Disease Questionnaire - 39 (PDQ-39)(From pre-operative to 5 years post-operative)
- Depression, Anxiety, and Stress Scale - 21 (DASS-21)(From pre-operative to 5 years post-operative)
- Schwab and England Activities of Daily Living Scale (S&E ADL scale)(From pre-operative to 5 years post-operative)
研究者
Danise Au
Assistant scientific officer
Hong Kong University of Science and Technology