Cardiac Modifications in Transthyretin Cardiac Amyloidosis
试验速览
- 阶段
- 不适用
- 入组人数
- 25
- 试验地点
- 2
- 主要终点
- Change of Atrial and Ventricular Walls Dimensions
研究概览
简要总结
Cardiac Amyloidosis (CA) is characterized by a long subclinical phase characterized by deposition of amyloid fibrils in atria, valves and walls of ventricles. Longitudinal dysfunction of the left ventricle (LV) with preserved ejection fraction (EF) is the early phase of CA.
Longitudinal dysfunction mainly involves the LV basal and middle segments with less involvement of the distal segments (apical sparing).
Strain echocardiography (STE) measures myocardial deformation. The technique has been shown to be sensitive for early detection of impaired systolic function and for the study of CA. Additionally, cardiac efficiency (myocardial work) can be derived from myocardial strain data analysis.
In the year 2018, "RNA interferences" (patisiran and inotersen) were included in the list of compassionate therapeutic use programs for exclusive use for the treatment of adult patients with hereditary amyloidosis neuropathy. The aim of our study is to evaluate the morpho-functional modifications with RNA interferences.
详细描述
Transthoracic echocardiographic images are recorded in the available ultrasound units (Vivid 7 and Vivid 9, GE Ultrasound, Horten, Norways and MyLab - Esaote, Genoa, Italy). The diameters and the wall thicknesses are measured according to the insurance of the American Society of Echocardiography. The images obtained with apical view and in format 4 bedrooms with frame rates (70-80 frames / s) - stored for three cardiac cycles in cine-loop format - will be used for evaluate off-line to evaluate left ventricle deformation in the longitudinal and radial direction, basalelateral, and top segments (post processing) through software already in use and available (Suitestenza, Esaote, Florence, Italy and GE Ultrasound, Horten, Norway).
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •presence of data obtained from historical records of patients who retrospectively met the prescriptive (neurological) criteria and received the prescription of the drugs in question (RNA interferences) having
- •Cardiological evaluation (examination ECG, echocardiography) at the time of prescription
- •Cardiological evaluation (examination ECG, echocardiography) evaluated six months after therapy,
- •Cardiological evaluation (examination ECG, echocardiography) evaluated 12-18 months after therapy,
排除标准
- •it wasn't possible to putatively confirm the reasonable shipment of the innovative therapy prescribed
- •it wasn't possible to putatively confirm the reasonable shipment of conventional therapy prescribed (background therapy)
结局指标
主要结局
Change of Atrial and Ventricular Walls Dimensions
时间窗: Change from Baseline Wall Thickness to 18 months
Measurements (mm) of Interatrial Septum, Ventricular Walls, Coumadin Ridge, Mitro-aortic Lamina, Valves
Change of Atrial and Ventricular Strain
时间窗: Change from Baseline Atrial and Ventricular Strain to 18 months
Mean paired change in myocardial strain (percent) by echocardiographic strain-rate imaging (unitless)
次要结局
- Change of Myocardial Work index of Left Ventricle(Change from Baseline Myocardial work to 18 months)
研究者
Gianluca Di Bella
Associate Professor Cardiovascular Diseases
University of Messina