PCSK9 inhibitor overcomes muscle-related statin intolerance

Dr. Nissen and his associates did a great job in this study of bringing much more clarity to the issue of muscle-related statin intolerance. Results from observational studies have suggested that this occurs in roughly 10%-20% of patients who start treatment on a statin. The rate has often been much lower in randomized statin trials because statin-intolerant patients are often identified and excluded from participation during a run-in phase before the randomized phase begins.

The first phase of GAUSS-3 showed that significant and treatment-limiting myalgia in response to statin treatment is real, and affects about 40% of patients who have a history of reporting muscle pain while taking statins. This part of the study provides clinicians with an important message about how to determine whether a patient really has muscle-related statin intolerance, and also showed that controlled rechallenge with a statin can identify many patients who can tolerate a statin despite a history of intolerance.

Dr. Roger Blumenthal

The second phase of GAUSS-3 showed that most patients with a history of muscle-related statin intolerance could nicely tolerate treatment with an effective regimen of either ezetimibe or the PCSK9 inhibitor evolocumab. Evolocumab was especially effective, reducing patient levels of LDL cholesterol by more than 50%.

Currently, the Food and Drug Administration–approved indications for treatment with PCSK9 inhibitors are limited to patients with familial hypercholesterolemia or with poorly-controlled LDL cholesterol levels and clinical atherosclerotic cardiovascular disease. That’s because we still await reports of longer-term follow-up of studies designed to confirm the clinical benefits of lowering LDL cholesterol using a PCSK9 inhibitor. Results from these studies should be available within the next year.

Dr. Roger Blumenthal is professor of medicine and director of the Ciccarone Center for the Prevention of Heart Disease at Johns Hopkins University in Baltimore. He had no disclosures. He made these comments in an interview.