Treatment patterns in patients with Familial hypercholesterolemia: evidence from real-world studies in Germany and the UK

Abstract

Abstract Background Familial hypercholesterolemia (FH) includes a spectrum of disease as per the number and effect of mutations in specific proteins involved in low-density lipoprotein cholesterol (LDL-C) metabolism, together with other genetic factors. Elevated LDL-C levels have been strongly associated with risk of cardiovascular and coronary heart disease, with up to 10-fold risk in patients (pts) with FH than without FH. The aim of lipid-lowering treatments (LLTs) is to reduce the LDL-C levels, although there is limited research describing treatment patterns and LDL-C outcomes in FH pts in routine care. Purpose To characterize the treatment patterns and LDL-C outcomes of FH pts in the real-world setting in Germany (GER) and the UK. Methods We conducted two descriptive, non-interventional and retrospective cohort studies. Pts in GER were identified from General Physician (GP) and Cardiology practices available in electronic medical records database Disease Analyzer (January 1992-June 2020). Pts in the UK were identified from the Clinical Practice Research Datalink linked to the Hospital Episode Statistics admitted pts care and Office of National Statistics datasets. Pts were included if they had diagnosis of FH (index date [ID]) and data available within 6-month before and 3-month after the ID. The first diagnosis of FH in the identification period (GER, 1/07/2015–30/06/2019; UK, 01/01/2010–31/05/2018) was considered the ID. Persistence and adherence to the recorded LLT at ID was analyzed for pts with at least 12 months and 24 months of follow-up. Persistence was measured as the duration (in days) with allowed gap of 60 days and adherence as proportion of days covered (PDC). Results Analysis included 2,105 FH pts from GER and 9,846 from the UK. Data are presented as GER/UK. The mean (SD) age of pts was 60 (15)/52 (14) years, and 60%/61% were females. Hypertension (53%/27%) and depression (31%/38%) were the common comorbidities. At ID, statin monotherapy (29%/68%) was the most commonly prescribed LLT. The use of ezetimibe, fibrates and PCSK9 inhibitors was very low in both countries (Table 1). Of note, LDL-C measurements at ID (−6m/+3m) were available for 31%/73% of pts. In pts with uncontrolled LDL-C (≥55 mg/dL), 34%/64% were receiving statin monotherapy, whereas there was no use of LLT in 62%/29% of pts. During the 24 months follow-up, the mean (SD) persistence and PDC to statins monotherapy was 471 (264)/489 (289) days and 0.65 (0.36)/0.69 (0.46), respectively, with 50%/70% of pts being adherent (PDC ≥0.80). Conclusions In our study, in GER, the rate of LDL-C measurements was low. In both GER and UK, almost all measured patients had LDL-C ≥55mg/dL at ID. Findings indicate low prescriptions of LLTs in GP setting, particularly non-statin LLTs in both countries. The mean adherence (PDC) in GER and the UK was 65% and 69%, respectively within 24 months after ID. Improved LDL-C monitoring and new therapies with potential to lower LDL-C are warranted. Funding Acknowledgement Type of funding sources: Private company. Main funding source(s): Novartis Pharma AG, Basel, Switzerland