N-of-1 Trial of a Statin, Placebo, or No Treatment to Assess Side Effects

Abstract

Statins are often discontinued because of side effects,^1,2 even though some blinded trials have not shown an excess of symptoms with statins as compared with placebo.^3,4 Patients who had previously discontinued statins because of side effects that occurred within 2 weeks after the initiation of treatment were enrolled in a double-blind, three-group, n-of-1 trial to test whether symptoms would be induced by a statin or placebo. Details of the trial methods are provided in Section S2 in the Supplementary Appendix (available with the full text of this letter at NEJM.org); the trial protocol and statistical analysis plan are also available at NEJM.org.