ESCAPE to the Real World: A Retrospective Review of Clinical use of Alirocumab in Lipoprotein Apheresis Patients

Background: The recent ODYSSEY ESCAPE trial demonstrated a significant reduction in the use of lipoprotein apheresis (LA) for heterozygous familial hypercholesterolemia (HeFH) patients when placed on alirocumab 75/150 mg SC every 2 weeks (Q2W). HeFH patients often have low-density lipoprotein cholesterol (LDL-C) values >100 mg/dL despite treatment with maximally tolerated lipid lowering therapies. We present clinical experience with LA and alirocumab for patients not in a clinical trial.
Methods: We examined data from five LA centers that started alirocumab for their LA patients. This retrospective review included patients who were on LA and alirocumab initiated Q2W. In addition to LDL-C levels, total cholesterol, high-density lipoprotein cholesterol (HDL-C), non-HDL-C, triglycerides, lipoprotein(a) and particle numbers were included when available.
Results: Eleven out of 25 patients were able to discontinue LA completely after initiation of alirocumab therapy, due to achieving LDL-C 50% reduction from baseline levels (Table). Fourteen patients remained on LA; however, seven patients were able to decrease the frequency of LA sessions (Table). No significant safety problems were reported.
Conclusion: Alirocumab was able to significantly lower LDL-C levels for some patients receiving LA for elevated LDL-C. Many patients on alirocumab therapy were able to discontinue LA treatment while others were able to reduce its frequency. However, some patients continued to require LA due to extremely elevated LDL-C.