Real-World Characteristics and Treatment Patterns of Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM): A Multi-Country, Non-Interventional, Disease Registry
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- Pfizer
- 入组人数
- 350
- 试验地点
- 1
- 主要终点
- Demographic data
研究概览
简要总结
This study is a multi-center, non-interventional, disease registry to characterize the natural history of ATTR-CM and treatment patterns in clinically diagnosed ATTR-CM patients. Data will be collected in the course of routine clinical practice or following local standard practice guidelines. No procedures or treatments will be mandated by this study, patients will receive usual clinical care.
The patient population will include all adult patients with a confirmed diagnosis of ATTR-CM after 01 June 2019 and who meet eligibility criteria. The index date of each patient will be the date of the first documented ATTR-CM diagnosis. The observation period for each patient will range from the index date to whichever occurs first of death, patient withdrawal of consent, loss to follow-up, or end of data collection. The end of data collection (ie, the end of study) is planned for 12 months after the end of the enrollment period.
As this study is descriptive in nature with no hypothesis testing, the study size will be based on the number of eligible ATTR-CM cases identified in the medical records and meeting the eligibility criteria. Approximately 350 patients diagnosed with ATTR-CM across approximately 17 sites in Taiwan, Hong Kong, and Malaysia are planned to be enrolled.
详细描述
Study variables will include demographic and clinical characteristics of the ATTR-CM patients at diagnosis, including age, sex, physical measurement, relevant medical history and comorbidities, New York Heart Association (NYHA) functional class, prior prescription of heart and CV medication, type of ATTR-CM (ATTRwt or ATTRm), and diagnosis parameters. Exposure variables will include treatment type, treatment regimen/patterns, and concomitant medications. Outcome variables will provide information on death (status and reasons), hospitalizations (status and reasons), imaging and lab assessments, Kansas City Cardiomyopathy Questionnaire (KCCQ), and healthcare resource utilization (eg, days of hospitalization, frequency of emergency department visits). All relevant data will be collected from the index date to the end of the observation period.
Data will be obtained from all information that is available at the site in the patients' electronic medical records and medical charts (eg, consultation notes, discharge summaries, laboratory test results, recorded prescription data, and any other documentation of communication with other health care providers). All data will be collected by trained site personnel and entered directly into web-based electronic case report forms (eCRFs).
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
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
- •Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study. Waivers of informed consent for deceased patients will be pursued if permitted by local regulation
- •Patients aged ≥18 years at first ATTR-CM diagnosis
- •Patients with confirmed diagnosis of ATTR-CM after 01 June 2019
排除标准
- 未提供
研究组 & 干预措施
ATTR-CM patients in Taiwan, Hong Kong, and Malaysia
case-only
干预措施: Observational (Other)
结局指标
主要结局
Demographic data
时间窗: From Enrollment/Baseline to 12 months
Age (measured in years) at enrollment, sex/gender (male/female), race/ethnicity
Physical measurements (in all patients whether receiving treatment or not)
时间窗: index date (closest available measurement) to 12 months
Height (in cm), weight (in kg), smoking status (never/prior/current), blood pressure (diastolic/systolic \[in mmHg\], heart rate (in beats/minute)
Relevant medical history and comorbidities
时间窗: enrollment/baseline to 12 months
The following information will be collected: First clinical cardiac symptom related to ATTR-CM, symptom onset date, age at onset of symptom; treatment received for the first symptom (with start and end date); Relevant medical history and comorbidities of interest including: amyloidosis, CV conditions (HFpEF, HFrEF, left ventricular hypertrophy/increased wall thickness, ischemic heart disease, pulmonary embolism, pericarditis, aortic stenosis, cardiac arrhythmias, atrial fibrillation and flutter, atrioventricular and left bundle branch block, conduction disorders, pacemaker or implantable cardioverter-defibrillator, other) Other vascular comorbidities: hypertensive diseases, diabetes mellitus, cerebrovascular disease, peripheral vascular disease, venous thrombosis, other. Nervous system conditions: carpal tunnel syndrome, lumbar spinal stenosis, peripheral neuropathy, autonomic neuropathy, other. Genitourinary system conditions: chronic kidney disease, erectile dysfunction, other.
NYHA Functional Class
时间窗: index date (closest available measurement to enrollment)
Patients' Physical activities wil be graded I, II, III, IV. I being asymptomatic, II being slight limitation, III being marked limitation, and IV being symptoms at rest.
Prescription of heart and cardiovascular medication
时间窗: within 6 months prior to the index date to 12 months
The following information will be collected: Medication type (eg. beta-blockers, ACE inhibitors, ARBs, ARNi, digoxin, calcium channel blockers, diuretics, antiplatelets, lipid-lowering agents, anticoagulants, other), medication name, start date, end date, reason of discontinuation
Type of ATTR-CM
时间窗: enrollment/index date
described by whether it is wild type or hereditary (including genetic variant if identified)
Family History
时间窗: index date/baseline
Describe whether the patient has family history of known cardiomyopathy, polyneuropathy, and sudden cardiac death (among parents, siblings, and 2nd/3rd grade family)
Diagnosis of ATTR-CM
时间窗: enrollment/baseline
Date of first ATTR-CM diagnosis; Age (in years) at first ATTR-CM diagnosis; Method of diagnosis of ATTR-CM including any of the following: cardiological assessments, MRI, ECG, ECHO, PYP scan with SPECT/CT (for definite confirmatory testing), nerve conduction studies, plasma neurofilament light (pNfL), and any other methods
次要结局
- Treatment for ATTR-CM(enrollment, every 3 month or as per standard of care, 12 months)
- Concomitant medications (including heart and CV medications and other concomitant medications)(from index date until 12 months)