Cardiac Involvement in Adult Patients With Fabry Disease; Relation to Enzyme Replacement Therapy - A Nationwide Danish Clinical Cohort Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 66
- 主要终点
- Left Ventricular Mass Index
研究概览
简要总结
The purpose of this study was to assess the progression of cardiac involvement in adult patients with Fabry Disease (FD), in the unique Danish Fabry cohort and comparing those FD patients receiving primary therapy vs. those that did not.
The hypothesis is, that we will not be able to see a significant positive difference in cardiac involvement in those FD patient who received FD specific therapy vs. those that did not.
详细描述
Enzyme replacement therapy and concomitant treatment:
FD specific treatment (ERT, enzyme replacement therapy) have been available since 2001 and patients have been treated with either agalsidase alpha (Replagal®) or agalsidase beta (Fabrazyme®) every other week at recommended doses, 0.2 mg/kg and 1.0 mg/kg, respectively.
Concomitant treatment with cardiovascular medication including angiotensin-converting-enzyme-inhibitor, angiotensin-II-receptor-blocker, acetylsalicylic acid, beta-blocker, calcium-channel-blocker, diuretics and statins, was registered for all patients.
Statistical analysis:
Primarily, we compared the progression of cardiac involvement from baseline to follow-up, according to ERT including subgroup analysis according to gender.
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
- •Genetically verified Fabry disease
- •Age at baseline >18 years
- •Baseline cardiac examination performed
排除标准
- •Switch from FD specific treatment (Fabrazyme or Replagal) to no FD specific treatment
结局指标
主要结局
Left Ventricular Mass Index
时间窗: Up to 13 years follow-up
Measured by Transthoracic Echocardiography using a Philips IE 33. Two-dimensional parasternal images were used to determine left ventricular chamber dimensions and wall thickness; LV mass was calculated by the American Society of Echocardiography (ASE) equation and indexed to body surface area. Median \[range\] is evaluated and presented.
Left Ventricular Hypertrophy - Sokolow-Lyon voltage criteria
时间窗: Up to 13 years follow-up
12-lead electrocardiography (ECG) was performed using a Schiller Cardiovit AT-2 (Schiller AG, Dietikon, Switzerland). Left ventricular hypertrophy was evaluated by Sokolow-Lyon voltage criteria (S in V1 + R in V5/V6 ≥ 35 mm). Median \[range\] and frequency (%) of hypertrophy is evaluated and presented.
Left Ventricular Hypertrophy - Cornell voltage product criteria
时间窗: Up to 13 years follow-up
12-lead ECG was performed using a Schiller Cardiovit AT-2 (Schiller AG, Dietikon, Switzerland). Left ventricular hypertrophy was evaluated by Cornell product criteria (R in aVL + S in V3 (+6 mm for women) x QRS duration \> 2440 mm·ms). Median \[range\] and frequency (%) of hypertrophy is evaluated and presented.
次要结局
- Clinical outcome(Up to 13 years follow-up)
- Arrhythmias(Up to 13 years follow-up)
研究者
Ulla Feldt-Rasmussen
Professor, Chief physician
Rigshospitalet, Denmark