ODYSSEY ESCAPE: Alirocumab Could Minimize Need For Apheresis Treatments in HeFH Patients
Use of the PCSK9 inhibitor alirocumab has the potential to reduce or eliminate the need for apheresis treatments in patients with heterozygous familial hypercholesterolemia (HeFH), according to results from the phase 3 ODYSSEY ESCAPE Trial presented Aug. 29 at ESC Congress 2016 in Rome, and published simultaneously in the European Heart Journal.
The study was based on 62 HeFH patients from 14 centers in the U.S. and Germany who were undergoing apheresis either weekly or every two weeks. These patients were randomized to receive subcutaneous injections of either alirocumab (150 mg) (n=41) or placebo (n=21) every two weeks for 18 weeks while still continuing their regular lipid-lowering medications (LLT). The rate of apheresis treatments was fixed according to the patient’s established schedule through week six, but was then adjusted between weeks seven through 18 based on individual needs. If a patient’s LDL-C dropped by 30 percent or more from the start of the study, apheresis was skipped.
Results found that alirocumab-treated patients had a 75 percent greater reduction in apheresis compared to those on placebo (P<0.0001). According to study investigators, 63.4 percent of patients in the alirocumab group eliminated apheresis altogether, compared to none in the placebo group. Additionally, 92.7 percent of alirocumab patients avoided at least half of the procedures compared to 14.3 percent in the placebo group. Adverse events were generally not serious, and were similar across both groups (75.6 percent of alirocumab patients vs. 76.2 percent of placebo patients).
“Reasons why apheresis rates reduced with placebo are unclear, but may reflect individual variation in LDL-C values, perhaps due to changes in diet or adherence to LLT, the small sample size, and the fact that patients were in a supervised clinical trial,” said lead investigator Patrick M. Moriarty, MD, FACC, from the University of Kansas Medical Center in Kansas City.
He noted that the findings could be a “major breakthrough” for HeFH patients who can spend up to $75,000 a year and several hours per week on apheresis treatments. “If our results are confirmed in other studies this could mark a new era for patients with familial hypercholesterolemia who have uncontrolled cholesterol levels and resistance to normal medical management,” he said. “In the future, lipoprotein-apheresis centers may now add alirocumab to a patient’s LLT and possibly not have to treat them with apheresis or at least treat them less often.”
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