Carcinoid Heart Disease National Registry of Poland
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- Characterization of patients with carcinoid heart disease
研究概览
简要总结
Carcinoid tumors are neuroendocrine tumors (NETs), most commonly originating from the gastrointestinal tract, that may secrete bioactive substances such as serotonin. Persistent exposure to these mediators can lead to carcinoid syndrome and, in up to 50% of patients, carcinoid heart disease (CHD). CHD is characterized by fibrotic degeneration of cardiac valves, predominantly right-sided, resulting in progressive valvular dysfunction and a significant increase in mortality.
The mechanisms underlying selective cardiac involvement and predictors of disease progression remain incompletely understood. In addition, optimal timing and selection of surgical versus transcatheter valve interventions, particularly in high-risk patients, require further clarification.
This multicenter, retrospective observational registry aims to identify and characterize patients with carcinoid heart disease and at least moderate valvular involvement. CHD is defined by confirmed neuroendocrine tumor, echocardiographic evidence of ≥ moderate valvular disease with features of fibrosis, elevated NT-proBNP (>260 ng/L), and histopathological confirmation when available.
Approximately 100-120 consecutive patients will be enrolled over 12 months. Standardized data collection will include detailed echocardiographic assessment, demographic and clinical characteristics, cardiovascular risk factors, comorbidities, tumor features, oncological treatment history, and cardiac management strategies.
The primary objective is to describe the clinical profile and management of patients with CHD in a real-world multicenter setting. Secondary objectives include identifying factors associated with advanced valvular dysfunction and evaluating eligibility and outcomes of transcatheter valve therapies. The registry is expected to improve risk stratification and support clinical decision-making in carcinoid heart disease.
详细描述
Carcinoid tumors are well-differentiated neuroendocrine neoplasms (NETs) capable of secreting vasoactive and fibrogenic substances, including serotonin, tachykinins, histamine, and prostaglandins. Persistent exposure to these mediators, particularly in the presence of hepatic metastases bypassing first-pass metabolism, may lead to the development of carcinoid syndrome and, in a substantial proportion of patients, carcinoid heart disease (CHD). Cardiac involvement is characterized by plaque-like fibrotic thickening of the endocardium, predominantly affecting right-sided heart valves, leading to leaflet retraction, reduced mobility, and progressive valvular regurgitation and/or stenosis. Over time, this process results in right ventricular volume overload, chamber dilation, heart failure symptoms, and reduced survival.
Although CHD represents a major determinant of prognosis in patients with carcinoid syndrome, important gaps remain in understanding its natural history, predictors of progression, and optimal management strategies. The incidence and severity of cardiac involvement vary considerably between individuals with similar oncologic profiles. Biomarkers such as NT-proBNP are widely used for screening and monitoring, but their integration with imaging findings and clinical variables in risk stratification requires further refinement. In addition, advances in oncological therapy have improved overall survival in NET patients, thereby increasing the clinical relevance of long-term cardiovascular complications.
Surgical valve replacement has traditionally been the standard treatment for advanced valvular dysfunction in CHD. However, many patients are at elevated operative risk due to metastatic disease burden, hepatic dysfunction, malnutrition, or other comorbidities. Emerging transcatheter valve therapies, including bicaval valve implantation and transcatheter tricuspid valve replacement or repair, offer potential alternatives for selected high-risk patients. Data regarding patient selection, anatomical suitability, procedural feasibility, and outcomes in the specific context of CHD remain limited.
This multicenter, retrospective, observational registry is designed to provide a comprehensive characterization of patients with carcinoid heart disease and at least moderate valvular involvement. Participating centers will identify consecutive eligible patients treated within predefined study periods to minimize selection bias. The registry reflects real-world clinical practice and does not mandate any additional diagnostic or therapeutic interventions beyond standard of care.
The study population consists of adult patients with a confirmed diagnosis of neuroendocrine tumor consistent with carcinoid tumor and evidence of carcinoid-related cardiac involvement. Cardiac involvement is established by echocardiographic documentation of at least moderate valvular dysfunction in conjunction with morphological features typical of fibrotic degeneration. Elevated NT-proBNP levels are required to support the presence of hemodynamic significance. In patients undergoing cardiac surgery, histopathological confirmation of carcinoid-related fibrosis will be recorded when available.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
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 ≥ 18 years.
- •Confirmed diagnosis of a neuroendocrine tumor consistent with carcinoid heart disease.
- •Echocardiographic evidence of at least moderate valvular disease associated with endocardial fibrosis.
- •Elevated NT-proBNP levels (>260 ng/L) consistent with cardiac dysfunction.
- •Availability of relevant clinical, echocardiographic, and laboratory data.
- •For patients who underwent cardiac surgery, histopathological confirmation of carcinoid-related cardiac tissue (if available).
- •Ability to provide informed consent for participation in the registry (or waiver as per local ethics approval for retrospective data).
排除标准
- •Patients with insufficient clinical or imaging data to confirm carcinoid heart disease.
- •Presence of other primary cardiac conditions causing significant valvular disease unrelated to carcinoid syndrome (e.g., rheumatic heart disease, congenital valve disease)
- •Active participation in interventional clinical trials that would conflict with registry data collection
- •Patients younger than 18 years
结局指标
主要结局
Characterization of patients with carcinoid heart disease
时间窗: At baseline (time of registry enrollment or most recent echocardiogram)
Characterization of patients with carcinoid heart disease defined as: presence of neuroendocrine tumor with hepatic metastases, echocardiographic evidence of right-sided valvular disease (tricuspid and/or pulmonary valve involvement), and biochemical evidence of serotonin excess (elevated urinary 5-HIAA or serum serotonin)
次要结局
- Identification of patients eligible for transcatheter valve interventions(Baseline)
- Assessment of clinical factors associated with advanced valvular dysfunction(Baseline)
- Assessment of echocardiographic factors associated with advanced valvular dysfunction(Baseline)