Alirocumab in patients with heterozygous familial hypercholesterolaemia undergoing lipoprotein apheresis: The ODYSSEY ESCAPE trial

Patrick M. Moriarty, Klaus G. Parhofer, Stephan P. Babirak, Marc Andre Cornier, P. Barton Duell, Bernd Hohenstein, Josef Leebmann, Wolfgang Ramlow, Volker Schettler, Vinaya Simha, Elisabeth Steinhagen-Thiessen, Paul D. Thompson, Anja Vogt, Berndt Von Stritzky, Yunling Du, Garen Manvelian

Research output: Contribution to journalArticlepeer-review

126 Scopus citations


Aim: To evaluate the effect of alirocumab on frequency of standard apheresis treatments [weekly or every 2 weeks (Q2W)] in heterozygous familial hypercholesterolaemia (HeFH). Methods and results: ODYSSEY ESCAPE (NCT02326220) was a double-blind study in 62 HeFH patients undergoing regular weekly or Q2W lipoprotein apheresis. Patients were randomly assigned (2:1, respectively) to receive alirocumab 150mg (n=41) or placebo (n=21) Q2W subcutaneously for 18 weeks. From day 1 to week 6, apheresis rate was fixed according to the patient's established schedule; from weeks 7 to 18, apheresis rate was adjusted based on the patient's low-density lipoprotein cholesterol (LDL-C) response in a blinded fashion. Apheresis was not performed when the LDL-C value was-30% lower than the baseline (pre-apheresis) value. The primary efficacy endpoint was the rate of apheresis treatments over 12 weeks (weeks 7-18), standardized to number of planned treatments. In the alirocumab group the least square (LS) mean6SE (95% confidence interval [CI]) per cent change in pre-apheresis LDL-C from baseline at week 6 was-53.762.3 (-58.2 to-49.2) compared with 1.663.1 (-4.7 to 7.9) in the placebo group. The primary efficacy endpoint showed statistically significant benefit in favour of alirocumab (Hodges-Lehmann median estimate of treatment difference: 0.75; 95% CI 0.67-0.83; P<0.0001). Therefore, alirocumabtreated patients had a 0.75 (75%) additional reduction in the standardized rate of apheresis treatments vs. placebo-treated patients. During this period, 63.4% of patients on alirocumab avoided all and 92.7% avoided at least half of the apheresis treatments. Adverse event rates were similar (75.6% of patients on alirocumab vs. 76.2% on placebo). Conclusions: Lipoprotein apheresis was discontinued in 63.4% of patients on alirocumab who were previously undergoing regular apheresis, and the rate was at least halved in 92.7% of patients. Alirocumab was generally safe and well tolerated.

Original languageEnglish (US)
Pages (from-to)3588-3595
Number of pages8
JournalEuropean heart journal
Issue number48
StatePublished - Dec 21 2016


  • Alirocumab
  • Familial hypercholesterolaemia
  • Low-density lipoprotein cholesterol
  • Low-density lipoprotein receptor
  • Monoclonal antibody
  • Proprotein convertase subtilisin/kexin type 9

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine


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