Abstract

Background Mitochondrial dysfunction affects multiple organs with varying severity and can be either primary or secondary to other diseases (e.g.: Alzheimer’s disease) or medications. Statins may be beneficial in patients with mitochondrial disease (mitoD) who have a higher prevalence of cardiac risk factors including diabetes and sedentary lifestyle. Therefore, it would be useful to know if statins are effective in patients with mitoD for prevention of cardiovascular disease. Methods We identified adult patients with confirmed mitoD in our clinic. Clinical and laboratory parameters were compared between the statin and non-statin groups. Results 56 patients had confirmed mitoD, 8 of which were on statins (average of 2 year, equivalent 20 mg daily atorvastatin) for secondary prevention. The statin group had a higher risk of cardiovascular disease (5 High, 3 Moderate Framingham risk score), including higher age (mean, years = 67 (SD 15) vs. 53 (SD 15), p<0.05) and comorbidities, such as coronary heart disease (50 vs. 15%, p<0.019), obesity (33 vs. 2%, p<0.008), diabetes mellitus (38 vs. 15%, p<0.11) and hypertension (50 vs. 40%, p<0.58). At two years, the mean reduction in LDL-C was 39.8% (SD 17%, measured in 7/8). None had myopathy with an elevated CK more than x10 the upper limit of normal (ULN), or AST more than x3 ULN, either at baseline or after the initiation of statins. Conclusion Our study suggests that high-risk patients with mitoD may benefit from statins, if indicated for the prevention of cardiovascular disease. The fact that the lowering of LDL-C in mitoD patients was of the same magnitude as expected in the general population suggests that there is no difference in statin metabolism in these individuals.

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