A small multicentre clinical prospective study of AMD patients has suggested that intensive statin therapy may bring about a regression of large soft drusen and an improvement in visual acuity in approximately half the patients examined. While a Cochrane report in 2015 indicated there was insufficient evidence for statins having a role in preventing or delaying the onset or progression of AMD, a small randomized controlled study on the impact of simvastatin on AMD suggested that 40mg simvastatin may slow progression of the disease, especially in patients harbouring the at-risk complement factor H (CFH) genotype CC (Y402H). Several additional studies have been performed on statin use and AMD but researchers at the Massachusetts Eye and Ear Infirmary, Harvard Medical School, wanted to investigate if high dose atorvastatin resulted in any regression of drusen or improvement of visual acuity (VA) in AMD patients.
The pilot multicenter open-label prospective study was conducted among 26 patients with AMD, including patients with many large, soft drusenoid deposits. The patients were administered with 80 mg of atorvastatin (Lipitor) daily with monitoring and follow-up at baseline and every 3 months for a 12-18 month period. Twenty-three of the patients were able to complete the minimum follow-up of 12 months among which 10 patients experienced regression of drusen deposits and an associated with vision gain of +3.3 letters (p = 0.06). Responders appeared to be slightly older than non-responders (70.6 ± 6.2 vs. 66.2 ± 5.5, p = 0.081) while women appeared to have a higher odds ratio (7.71) of being responders (95% confidence limits 0.746–79.7746, p = 0.0886).
The study had several limitations, being an open label, non-randomized pilot study with a small and homogeneous sample size (n=26) and an average duration of 18 months follow-up. As such, far larger randomized blinded prospective studies will be required to build a more robust picture of the role of statins in AMD. Despite such limitations the authors cautiously concluded that, “our open-label pilot safety/efficacy study suggests that intensive statin treatment may cause regression of high-risk features of AMD, such as drusen, without progression to advanced AMD and without the vision loss that often accompanies spontaneous disappearance of drusen.” In addition, a news release from Harvard Medical School reported one of the study’s co-author. Dr. Demetrios Vavvas, MD, PhD, stating that “Not all cases of dry AMD are the exactly the same, and our findings suggest that if statins are going to help, they will be most effective when prescribed at high dosages in patients with an accumulation of soft, lipid material. These data suggest that it may be possible to eventually have a treatment that not only arrests the disease but also reverses its damage and improves the visual acuity in some patients.”