Study | Location | Population | Measures reported | Clinical outcomes |
---|---|---|---|---|
Spinelli et al. [9] | Italy | Genetically proven FD with normal left ventricular ejection fraction | eGFR, LVMI | Cardiac events (defined as cardiac death, malignant ventricular tachycardia, atrial fibrillation and severe cardiac failure) |
Italy | Genetically confirmed FD patients belonging to 20 different families | eGFR, LVMI | Composite measure of major events: cardiac failure, atrial fibrillation, stroke, progression to dialysis or eGFR decline | |
Canada | Patients aged ≥ 18 years with gene-positive FD | LVMI | Cardiac failure, cardiac death | |
USA | Male and female patients aged ≥ 18 years with FD treated with agalsidase alfa | eGFR, LVMI | Myocardial infarction/cardiac failure, renal failure | |
Lenders et al. [10] | Germany | Classical or late-onset clinical phenotype form of FD including FD-typical signs and symptoms (patients with genetic variants of unknown significance and polymorphisms were not included) | eGFR | Myocardial infarction, ESRD |
Switzerland | Genetically confirmed FD patients | eGFR, LVMI | Composite measure including: cardiac failure, cerebrovascular events or death | |
Patel et al. [11] | Multiple | Previously untreated FD patients | eGFR | Mortality: cardiac events, stroke |
Netherlands, UK, and Germany | Adults with a definite FD diagnosis who were treatment-naïve and subsequently treated with either agalsidase alfa or agalsidase beta for ≥ 9 months | eGFR, LVMI | Clinical events: Renal event, cardiac arrest, cerebral events |