MedicalResearch.com: What are the main findings of the study?
Dr. Stein: The study which is the first 52 week randomized double blind trial of a PCSK9 to report results (all others have been 12 weeks) demonstrated that the excellent LDL-C reductions of 55-60% seen at 12 weeks are maintained through 52 weeks, with no fall off in patient compliance, tolerability of efficacy. It also demonstrated that with longer treatment no new or unexpected side effects.
The study also had a unique design in that prior to randomization to the PCSK9 inhibitor (evolocumab) or placebo patients had a run in period during which time they were assigned, based on NCEP-ATP III criteria, to appropriated background therapy which ranged from diet only, to atorvastatin 10 mg a day, to atorvatatin 80 mg a day or atorvastatin 80 mg a day plus ezetimibe – reflecting how these patients are treated in practice. Only then if their LDL-C was still above 75 mg/dL were they randomized into the treatment part of the study with the new drug. The study showed that irrespective of background therapy the reduction with evolocumab was consistent.
MedicalResearch.com: What should clinicians and patients take away from your report?
Dr. Stein: That despite having to be given sub-cutaneously every month after 1 year of treatment patients tolerated the medications as well as any oral cholesterol medications, including statins – in comparison with prior trials with statins and ezetimibe the number of patients who completed the 52 week study with evolocumab was as good, or better, and the LDL-C reduction greater than that seen with even the highest dose of the most effective statins, such as rosuvastatin 40 mg or atorvastatin 80 mg.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Dr. Stein: There have been a large number of studies, 5 others released at ACC, recently completed addressing a variety of other questions – but all very consistent in terms of LDL-C reduction and showing no new or unexpected side effects. The next and perhaps most important study is the one currently underway which is recruiting 22,000 patients with heart disease to assess if long term treatment will reduce not just LDL-C by heart disease itself.
Dirk J. Blom, M.D., Ph.D., Tomas Hala, M.D., Michael Bolognese, M.D., Michael J. Lillestol, M.D., Phillip D. Toth, M.D., Lesley Burgess, M.B., B.Ch., M.Med., Ph.D., Richard Ceska, M.D., Ph.D., Eli Roth, M.D., Michael J. Koren, M.D., Christie M. Ballantyne, M.D., Maria Laura Monsalvo, M.D., Kate Tsirtsonis, M.Sc., Jae B. Kim, M.D., Rob Scott, M.D., Scott M. Wasserman, M.D., and Evan A. Stein, M.D., Ph.D. for the DESCARTES Investigators