Tuberous Sclerosis Complex Natural History Study: Renal Manifestations
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 450
- 试验地点
- 1
研究概览
简要总结
Eighty percent of patients with tuberous sclerosis complex (TSC) have renal angiomyolipomata. These lesions grow and can lead to significant morbidity by hemorrhage or renal failure. Understanding the natural history of these lesions and understanding which lesions may be more prone to grow quickly or develop aneurysms that predispose to hemorrhage will greatly assist clinical care of patients with TSC. The objective is to test the hypothesis that serial MR and CT imaging will allow objective, reproducible quantification of angiomyolipoma growth by volumetric analysis, and analysis of lesions characteristics will identify angiomyolipomata with rapid growth potential that would require intervention. The specific aim of this proposal is to collect clinically obtained serial abdominal imaging from the Tuberous Sclerosis Natural History Consortium Centers and analyze the volume and adiposity of the individual angiomyolipomata. The growth rate is hypothesized to have an inverse relationship to adiposity. Yearly renal MR or CT imaging will be performed of patients with TSC. The images will be coded at the site of acquisition, and transferred via VPN to a secure server at Cincinnati Children's Hospital Medical Center. Using innovative imaging processing software (Cincinnati Children's Hospital Image Processing Software (CCHIPS), the image data will be segmented to reveal various tissue components based on signal intensities. Different signal intensities can differentiate normal renal parenchyma, and renal angiomyolipomata. Using the imaging data and the novel software, the volume of an individual angiomyolipoma, as well as the adiposity will be determined. Imaging at enrollment (year 1) will serve as baseline. At years two and three, the lesions will undergo repeat analysis. Angiomyolipoma growth rates and adiposity over three years will be analyzed to test the hypothesis above.
详细描述
Objectives.
Our objective is to test the hypothesis that serial MR and CT imaging will allow objective, reproducible quantification of angiomyolipoma growth by volumetric analysis, and analysis of lesions characteristics will identify angiomyolipomata with rapid growth potential that would require intervention.
The specific aim of this proposal is to collect clinically obtained serial abdominal imaging from the Tuberous Sclerosis Natural History Consortium Centers and analyze the volume and adiposity of the individual angiomyolipomata. The growth rate is hypothesized to have an inverse relationship to adiposity. The prognostic value of identifying lesions with aggressive growth characteristics is very large, and intervention can be instituted early in order to reduce the renal damage.
Study Population.
- The target population for this study will be patients with tuberous sclerosis who attend a tuberous sclerosis clinic that is part of the consortium. The data collected will include routine imaging data, age, gender, and if know, the genotype. Approximately 855 patients throughout the United States will be asked to participate in this natural history study and 450 of those are anticipated to consent and have complete data on 3 years of CT and MR Imaging Scans to measure angiomyolipoma growth and adiposity.
- Patients attending the tuberous sclerosis clinics that are members of the consortium will be asked if they would be involved in the study.
- Imaging will be obtained as part of the standard of care at the Consortium Centers. Imaging done on pregnant patients will not be excluded. MRI has been used now extensively for pregnant patients.
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
- 7 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who receive yearly follow-up at their respective tuberous sclerosis clinic. Such clinics have a director who is knows their patients well.
- •Patients known to have an angiomyolipoma between the ages of 7-
- •Pregnant women to be included if tested with MR
排除标准
- •Patients who are not seen annually and therefore do not have yearly imaging.
- •Patients who do not have an angiomyolipoma.
- •Patients who are not likely to follow-up as recommended.
- •Use of an investigational drug, including rapamycin, within the last 30 days.
- •Pregnant women to be excluded if they cannot be tested with MR