Long-Term Study of Heart Problems and Sudden Cardiac Death Risks in Adults with Fabry Disease

What this Trial Is About

Fabry disease is a rare lysosomal storage disorder with a natural history that leads to poor survival beyond age 50, highlighting the need to understand cardiac symptoms and outcomes in these patients. Researchers are studying predictors for heart rhythm problems, sudden cardiac death, and terminal heart failure in people with Fabry disease. This is a long-term, prospective cohort study that has been observing patients since 2001 to estimate these risks. The study includes all patients treated at the Fabry Center Wuerzburg who provide informed consent. It follows patients over time to monitor occurrences of cardiac death, heart transplants, and dangerous heart rhythm disturbances. The observation period extends until the year 2032 or until the first event of interest occurs for each participant. Participants will be regularly evaluated for heart-related events and outcomes throughout the study period. Researchers will track the time from enrollment to cardiac death, heart transplantation, or malignant arrhythmias. This ongoing monitoring aims to improve understanding of heart involvement in Fabry disease and guide future care. Total participation time varies based on individual timing of events or study end in 2032.

Evaluation of HEArt invoLvement in Patients With FABRY Disease