Familial hypercholesterolemia Inherited elevated cholesterol
Conditions
Interventions
Sponsors
Amsterdam UMC
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: Patients with FH who previously participated (1997-1999) in our randomized double-blinded placebo-controlled study evaluating the two-years efficacy and safety of pravastatin in children, as well as their 95 unaffected siblings. All participants will be contacted, as in the most recent follow-up study ‘the After15’ (METC: 2017-172).
Exclusion criteria
Exclusion criteria: There are no exclusion criteria for general participation in this study. Participants will be excluded from CCTA scanning if they meet any of the following conditions:PregnancyHypersensitivity and/or allergy to contrast agentsImpaired renal function (estimated Glomerular Filtration Rate [eGFR] < 30 mL/min/1.73 m²)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Comparison of cardiovascular endpoints (CVE, death) between FH patients who initiated statin therapy in childhood versus their FH parents who started statin therapy only in adulthood.Comparison of cardiovascular endpoints (CVE, death), coronary plaque burden and plaque composition on CCTA and carotid IMT between FH patients and their unaffected siblings. | — |
Secondary
| Measure | Time frame |
|---|---|
| Comparison of cardiovascular endpoint (CVE, death) between FH patients with elevated Lp(a) versus FH patients without elevated Lp(a) who initiated statin therapy in childhood. Comparison of cardiovascular endpoint (CVE, death) between FH patients with elevated Lp(a) versus their unaffected siblings.Association between below and above median age at start statins: 1. the incidence of cardiovascular endpoints, 2. coronary plaque burden and plaque composition , and 3. cIMT. Safety: the incidence of side effects during statin therapy in FH patients (e.g. muscle pain or weakness, elevated liver enzymes, rhabdomyolysis, diabetes mellitus).Tolerability and degree of adherence to statins in FH patients.Comparison of quality of life between FH patients and their unaffected siblings.Costs-effectiveness of initiating statin therapy in childhood versus initiating in adulthood in patients with FH.Differences in gut microbiota (composition) between FH patients and their unaffected siblings.Association between long-term LLT and changes in gut microbiota (composition) in FH patients.Comparison of cardiovascular endpoints (CVE, death), coronary plaque burden and plaque composition on CCTA and carotid IMT between FH patients with calculated LDL-C exposure (cholesterol-year-score (CYS)) above and below median calculated CYS. | — |
Countries
Netherlands
Contacts
Public ContactB.A. Hutten
Amsterdam UMC
Outcome results
None listed