Assessment of the Structure and Function of Heart and Selected Cardiovascular Risk Factors in Patients With Acute Intermittent Porphyria
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 180
- 试验地点
- 2
- 主要终点
- Rate of cardiac arrhythmias.
研究概览
简要总结
This study aims to assess the changes in the cardiovascular system in patients with acute intermittent porphyria (AIP).
详细描述
Porphyrias are heterogeneous group of the disorders of heme biosynthesis. Acute intermittent porphyria (AIP) is the most common acute hepatic porphyria, caused by the mutations in the gene encoding hydroxymethylbilane synthase. Clinical symptoms i.e. abdominal pain, nausea, vomiting, paresis or paralysis, coma and/or mental abnormalities may be induced by many porphyrinogenic factors, such as drugs, alcohol, starvation or stress. The symptoms are often accompanied by tachycardia and elevated blood pressure. Due to the non-specific clinical picture, AIP is often diagnosed too late and causes a threat to the patients' lives.
There is a scarcity of data regarding the changes in cardiovascular system in patients with AIP. The aim of this study is to assess the structure and function of heart in patients with this disease. The prevalence of hypertension, cardiac arrhythmias and selected cardiovascular risk factors in patients with AIP will also be evaluated.
This is a case-control study with prospective observation of the subgroup of patients examined during the exacerbations of AIP.
Specific goals:
- assessment of the cardiac morphology and function,
- assessment of the concentrations of markers of myocardial injury (troponin T) and heart failure (NT-proBNP),
- assessment of the prevalence of cardiac arrhythmias and electrocardiographic abnormalities,
- assessment of the blood pressure profiles,
- assessment of selected cardiovascular risk factors,
- assessment of quality of life,
- assessment of clinical and biochemical factors associated with the pathological findings in patients with AIP.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •acute intermittent porphyria (AIP),
- •age 18-65 years,
- •at least one hospitalization due to exacerbation of AIP.
排除标准
- •previous myocardial infarction,
- •heart failure of established (other than porphyria) etiology,
- •severe heart valve disease,
- •congenital heart defects,
- •history of myocarditis,
- •pacemaker,
- •hyperthyroidism / hypothyroidism (except for adequate thyroid hormone replacement therapy),
- •chronic advanced lung diseases,
- •lack of consent to participate in the study.
结局指标
主要结局
Rate of cardiac arrhythmias.
时间窗: day 1 and up to 2 years in case of the exacerbation of AIP
Cardiac arrhythmias observed in 24-hour ECG monitoring.
Number of participants with hypertension.
时间窗: day 1 and up to 2 years in case of the exacerbation of AIP
Arterial hypertension diagnosed by ambulatory 24-hour blood pressure monitoring.
Number of participants with heart failure and left ventricular dysfunction.
时间窗: day 1 and up to 2 years in case of the exacerbation of AIP
Heart failure and left ventricular dysfunction assessed by symptoms and echocardiography.
次要结局
- Number of participants with dyslipidemia.(day 1)
- Number of participants with increased concentrations of markers of heart failure.(day 1 and up to 2 years in case of the exacerbation of AIP)
- Number of participants with chronic kidney disease.(day 1 and up to 2 years in case of the exacerbation of AIP)
- Number of participants with increased concentrations of markers of myocardial injury.(day 1 and up to 2 years in case of the exacerbation of AIP)
- Number of participants with diabetes.(day 1)
- Level of quality of life.(day 1)
研究者
Krzysztof Jaworski
Principal Investigator
National Institute of Cardiology, Warsaw, Poland