Gender-based effect of statins on functional decline in amyotrophic lateral sclerosis.

Recently an association between statins and the onset and more rapid disease course of amyotrophic lateral sclerosis (ALS) was reported, while other studies rejected such a link. The role of gender in that controversy is unclear. We evaluated the gender-specific effect of statins on the rate of functional decline in patients with ALS, based on data retrieved from the medical records of all ALS patients who participated in two previously reported clinical trials on the efficacy of topiramate and of celecoxib in ALS. The topiramate trial enrolled 294 patients, 28 (9.5%) of whom were statin users (20 males). The celecoxib trial enrolled 300 patients, 25 (8.3%) of whom were statin users (17 males). Statins had no effect on the functional decline in the celecoxib trial, but they did have a negative impact on disease course in the topiramate trial. When males and females were analyzed separately, the functional decline of females taking statins was significantly greater than that of males in both trials. Our results indicate that statins affect possibly negatively ALS progression among females but not males. They emphasize the need to consider gender in future analyses of drug effects.