MedicalResearch.com Interview with:
Professor F. J. Raal
FRCP, FRCPC, FCP(SA), Cert Endo, MMED, PhD
Director, Carbohydrate & Lipid Metabolism Research Unit
Professor & Head, Division of Endocrinology & Metabolism
Faculty of Health Sciences, University of the Witwatersrand
Johannesburg Hospital Johannesburg South Africa
Medical Research: What are the main findings of the study?
Dr. Raal: Heterozygous familial hypercholesterolaemia (HeFH) is one of the most common inherited disorder in man affects between 1:250 to 1:300 persons worldwide. Thus, there are likely more than 3 million patients with heterozygous familial hypercholesterolaemia in the United States and Europe alone. The RUTHERFORD-2 study was a large world-wide multinational study of the use of the PCSK9-inhibitor, evolocumab, in over 300 patients with heterozygous familial hypercholesterolaemia (HeFH). Evolocumab administered either 140 mg biweekly or 420 mg monthly as a subcutaneous injection, much like an insulin injection, was well tolerated with minimal side effects, and markedly reduced levels of LDL cholesterol or “bad cholesterol” by over 60% compared to placebo.
Medical Research: What was most surprising about the results?
Dr. Raal: Within 12 weeks nearly two thirds of subjects has reduced their LDL-cholesterol levels to below 1.8 mmol/L which was never thought to be possible in subjects with this condition.
Medical Research: What should clinicians and patients take away from your report?
Dr. Raal: With the addition of evolocumab we are now able to get the majority of HeFH patients to LDL-cholesterol target and are able to “cure” HeFH. However the challenge remains to identifying these patients with an asymptomatic condition which often results in sudden death from a heart attack in the prime of their lives.
Medical Research: What recommendations do you have for future research as a result of this study?
Dr. Raal: This study was a relatively short term study but if the reductions in LDL-cholesterol can be maintained in the long-term and if the therapy is found to reduce cardiovascular events as is being studied in a large cardiovascular outcome study (FOURIER study), the addition of evolocumab with allow patients with HeFH to life, healthier, longer lives.
Prof Frederick J Raal PhD,Prof Evan A Stein PhD,Robert Dufour MD,Traci Turner MD,Fernando Civeira MD,Prof Lesley Burgess MB,Gisle Langslet MD,Prof Russell Scott MD,Prof Anders G Olsson MD,David Sullivan MD,G Kees Hovingh MD,Bertrand Cariou MD,Ioanna Gouni-Berthold MD,Ransi Somaratne MD,Ian Bridges MSc,Rob Scott MD,Scott M Wasserman MD,Prof Daniel Gaudet MD,for the RUTHERFORD-2 Investigators
The Lancet – 2 October 2014