In randomized clinical trials (RCTs) proprotein convertase subtilisin/kexin 9 (PCSK9) inhibitors showed a favorable safety profile, however, "real-world" data on adverse events (AEs) is scarce. Three datasets, a hospital registry (n = 164), and two Pharmacovigilance databases, Lareb (n = 149) and VigiLyze (n = 15,554), reporting AEs attributed to PCSK9 inhibitors (alirocumab or evolocumab) prescribed in clinical practice were analyzed. In the hospital registry, 41.5% of the patients reported any AE, most often injection-site reactions (33.8%) and influenza-like illness (27.9%). Twelve patients (7%) discontinued PCSK9 inhibitor treatment. Most common AE reported in the Lareb and VigiLyze database was myalgia (12.8% and 8.3%, respectively). No clinically relevant differences in gender or between drugs were observed. No specific subgroup of patients could be identified at risk of developing AEs. During follow-up, AEs resolved in most patients (71.1%). In a real-world setting, PCSK9 inhibitors are well tolerated with an overall safety profile comparable to RCTs.

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FH girls have higher levels of TC, LDL-C, and non-HDL-C levels than boys from birth up to 19 years of age. This may contribute significantly to the total lifelong cholesterol burden in FH women.

A health-related outcomes set has been developed for familial hypercholesterolemia (FH) patients. • This set contained both outcomes defined by health care professionals as patient-related outcomes (PROMS). • The response rate of the PROMS was 81.4%, implicating high acceptance.• Sufficient knowledge of a patient was associated with better health-outcomes. A R T I C L E I N F O Keywords:Familial hypercholesterolemia Value-based health care Patient-reported outcome measures Prevention Cardiovascular disease A B S T R A C TBackground and aims: Familial hypercholesterolemia (FH) is the most common hereditary lipid disorder requiring life-long treatment to prevent cardiovascular disease. A recent concept in healthcare is not only to focus on outcomes defined by healthcare professionals, but also take Patient-Reported Outcomes Measures (PROMs) into account. The aim of this study is (1) to describe the development and first results of a health-related outcomes set including PROMs for FH patients and (2) investigate the influence of patient knowledge on healthrelated outcomes. Methods: A multidisciplinary group of FH experts, in collaboration with a sounding board of FH patients (n = 166), developed a health-related outcomes set containing the domains: medication adherence (MARS-5), smoking, self-efficacy and self-management, quality of life (QOL) (EQ-5D-5L), reported adverse drug reactions, lipid outcome measures, and FH and cardiovascular risk factor knowledge. Knowledge scores ranged from 0 to 10. Two groups were created: Insufficient knowledge (INSUF) (< 7.5), and Sufficient knowledge (SUF) (≥7.5). Results: The response rate of the questionnaires was 81.4% (n = 429), implicating acceptance of PROMs. In general, FH patients showed good knowledge, high QOL and were adherent to medication. However, the INSUF group had higher triglycerides levels (1.0 vs 0.9, p < 0.05), lower QOL (0.89 [0.79, 1.00] vs 0.89 [0.85, 1.00], p < 0.05), were more often smokers (14% vs 7%, p < 0.05) and reported more adverse drug reactions (62% vs. 49%, p < 0.05). Conclusions: A health-related outcomes set for FH patients, including PROMs, has been developed, which shows that insufficient knowledge of FH is negatively related to health outcomes. Improving patients' knowledge of FH may lead to better health.

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