A Clinical, Structural, and Functional Neuroimaging Study of Cognition in Adults With Erdheim-Chester Disease, Langerhans Cell Histiocytosis, Rosai-Dorfman Disease, and Other Histiocytoses
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 13
- 试验地点
- 8
- 主要终点
- neurocognitive function
研究概览
简要总结
The purpose of this study is to try to understand how histopcytosis can cause symptoms or problems in the brain. The tests being done in the study will look at memory and thinking as well as brain function via MRI scan.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age greater than or equal to 18 years.
- •Fluency in English
- •Diagnosis of a histiocytic disorder as determined, in the opinion of the study PI, by a corroborating constellation of histopathology, clinical, and/or radiologic findings.
- •Will undergo Standard of Care MRI.
排除标准
- •Known intracranial involvement of histiocytosis (including dura, leptomeninges and brain parenchyma)
- •Prior stroke or intracranial hemorrhage
- •Other (non-histiocytic) intracranial neoplasm or neurological disorder deemed by the PI or Co-PI to confound neuroimaging studies (e.g., demyelinating disease)
- •Existing diagnosis of a psychiatric disorder or untreated mood disturbance
- •Existing diagnosis of a neurodegenerative disease, such as Alzheimer's disease
- •Chronic or daily excessive alcohol consumption as determined by the PI.
- •History of chronic use of corticosteroids, defined as continuous treatment for six months or longer at any time in the past
- •History of severe claustrophobia or other contraindications to patient SOC brain MRI
- •Prior intravenous cytarabine or cladribine
- •Other current or prior treatments (e.g., high-dose chemotherapy for a different cancer) deemed by the PI or Co-PI to confound imaging studies or cognitive performance
结局指标
主要结局
neurocognitive function
时间窗: 1 year
Neurocognitive tests will yield raw scores that are converted to z-scores, normalized for participant's age, sex and educational level.The proportion of participants demonstrating cognitive impairment (as defined by 2 or more z-scores less than -1.5) will be summarized. Also, the proportion of participants with impairment (z \< -1.5) for each cognitive test will be summarized. In this way the cognitive assessment yields a dichotomous outcome of impaired versus not impaired for each participant but also allow for more granular assessment of impairment in specific cognitive domains.
quality of life
时间窗: 1 year
questionnaires will yield raw scores (total score for HADS and McGill QOL, as well as anxiety and depression subscores for the HADS). QOL scores will be analyzed as raw scores. Cognitive test scores will be transformed into z-sores (based on score distributions from established normative samples with a mean of 0 and standard deviation of 1) to define the presence/severity of cognitive dysfunction. Scores will be normalized for age, sex and education.
comparing of grey matter volume
时间窗: 1 year
In a whole-brain analysis, first, cortical thickness will be compared between participants and controls and regions of statistically significant grey matter loss will be identified. This is done computionally with a whole-brain, vertex-by-vertex approach, as stated above. Also, as stated above (Section 7), statistical significance thresholding is at p\<0.001, correcting for multiple comparisons using the False Discovery Rate (FDR) method and clustering thresholds, methods used in various high-throughput contexts, including MRI analysis.
次要结局
未报告次要终点