Treatment Inertia in Patients With Familial Hypercholesterolemia

Abstract

Background We studied care gap in patients with familial hypercholesterolemia (FH) with respect to lipid-lowering therapy. Methods and Results We enrolled patients with cardiovascular disease (CVD) or FH and low-density lipoprotein-cholesterol >2.0 mmol/L despite maximally tolerated statin therapy. During follow-up physicians received online reminders of treatment recommendations of 2009 patients (median age, 63 years, 42% women), 52.4% had CVD only, 31.7% FH only, and 15.9% both CVD and FH. Patients with FH were younger and more likely to be women and non-White with significantly higher baseline low-density lipoprotein-cholesterol level (mmol/L) as compared with patients with CVD (FH 3.92±1.48 versus CVD 2.96±0.94, P<0.0001). Patients with FH received less statin (70.6% versus 79.2%, P=0.0001) at baseline but not ezetimibe (28.1% versus 20.4%, P=0.0003). Among patients with FH only, 45.3% were at low-density lipoprotein target (≥ 50% reduction from pre-treatment level or low-density lipoprotein <2.5 mmol/L) at baseline and increasing to 65.8% and 73.6% by visit 2 and 3, respectively. Among patients with CVD only, none were at recommended level (≤2.0 mmol/L) at baseline and 44.3% and 53.3% were at recommended level on second and third visit, respectively. When primary end point was analyzed as a difference between baseline and last available follow-up observation, only 22.0% of patients with FH only achieved it as compared with 45.8% with CVD only (P<0.0001) and 55.2% with both FH+CVD (P<0.0001). Conclusions There is significant treatment inertia in patients with FH including those with CVD. Education focused on patients with FH should continue to be undertaken.

Keywords: familial hypercholesterolemia; lipid lowering; treatment inertia.

Figure 1. Proportion of patients achieving the recommended low‐density lipoprotein cholesterol (LDL‐C) level during follow up and change from baseline.

A, Proportion of patients achieving recommended LDL‐C level* at each visit. *Recommended LDL‐C for cardiovascular disease and cardiovascular disease+familial hypercholesterolemia is <2.0 mmol/L while for familial hypercholesterolemia only <2.5 mmol/L or 50% reduction from pre‐treatment level. **Inclusion criteria for all was LDL‐C >2 mmol/L. These patients with familial hypercholesterolemia had LDL‐C >2 but <2.5 mmol/L. B, Change in treatment from baseline to last available observation during follow‐up. CVD indicates cardiovascular disease; FH, familial hypercholesterolemia; LDL‐C, low‐density lipoprotein cholesterol; and PCSK9, proprotein convertase subtilisin/kexin type 9.

Figure 2. Reasons for not prescribing recommended therapy.

A, Reasons provided by physicians for not following the guidelines with respect to the use of ezetimibe (%) at baseline (visit 1). B, Reasons provided by physicians for not following the guidelines with respect to the use of PCSK9 inhibitor (%) at baseline. CVD indicates cardiovascular disease; FH, familial hypercholesterolemia; PCSK9, proprotein convertase subtilisin/kexin type 9.