Abstract
Introduction: Patients with familial hypercholesterolemia (FH) suffer from premature coronary heart disease (CHD) due to extreme elevations in low-density lipoprotein cholesterol (LDL-C). As such, they require high doses of potent statins, a major risk factor for developing statin-induced myopathy. Little is known, though, about other underlying factors predisposing them to statin-induced myopathy. Methods: We studied 272 genetically screened FH patients - ascertained from Dallas, TX, lipid specialty clinics - with and without a documented history of statin-induced myopathy (defined as developing muscle symptoms or elevated serum creatine kinase (CK) levels while taking a statin). Results: Statin-induced myopathy was diagnosed in 35% of FH patients; however, at the time of participation in our study, 73% of myopathy patients were taking statins (vs 79% of statin-tolerant patients; p = 0.37). Statin dosages were similar in the two groups except for rosuvastatin (20 mg/day in myopathy patients vs 40 mg/day in statin-tolerant patients; p < 0.001). Myopathy patients were older (57 ± 11 vs 51 ± 14 years; p = 0.001), had lower BMI (29 ± 6 vs 31 ± 7 kg/m2; p = 0.01), suffered from less premature CHD (22% vs 34%; p = 0.006), and had less hypertension (57% vs 67%; p = 0.05). On-treatment LDL-C was higher in myopathy patients (141 ± 37, n = 63, vs 128 ±40 mg/dL, n= 148; p < 0.01). No differences were identified in pretreatment LDL-C (253 ± 53 vs 263 ± 83 mg/dL; p = 0.57), gender (63% vs 57 % female; p = 0.3), % with diabetes (23% vs 29%; p = 0.38), % with LDL-receptor or apolipoprotein B mutations (26% vs 36%; p = 0.09), or baseline CK levels (median 103 vs 115; p = 0.10). Conclusions: Despite a history of statin-induced myalgia, most FH patients eventually tolerated statins and achieved up to 44% reduction in LDL-C levels. FH patients with increased age and lower BMI were more likely to be diagnosed with myopathy, consistent with prior reports that age and smaller body frame are risk factors. Also, FH patients lacking hypertension or premature CHD were diagnosed more often with myopathy, suggesting that patients without CHD risk factors complain more about muscle symptoms. In conclusion, statin-induced myopathy adds an additional - but manageable - challenge to the treatment of FH patients.
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