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Microsomal triglyceride transfer protein inhibitor (lomitapide) efficacy in the treatment of patients with homozygous familial hypercholesterolemia phenotype

Session Poster Session 6

Speaker Epameinondas Fountas

Event : ESC Congress 2019

  • Topic : cardiovascular pharmacology
  • Sub-topic : Lipid-Lowering Agents
  • Session type : Poster Session

Authors : E Fountas (Athens,GR), O Diakoumakou (Athens,GR), V Kolovou (Athens,GR), S Stratakis (Heraklion,GR), E Zacharis (Heraklion,GR), E Liberopoulos (Ioannina,GR), F Matsouka (Athens,GR), A Tsoutsinos (Athens,GR), I Mastorakou (Athens,GR), T Katsikas (Athens,GR), S Mavrogeni (Athens,GR), G Hatzigeorgiou (Athens,GR), G Kolovou (Athens,GR)

E Fountas1 , O Diakoumakou1 , V Kolovou1 , S Stratakis2 , E Zacharis3 , E Liberopoulos4 , F Matsouka1 , A Tsoutsinos1 , I Mastorakou1 , T Katsikas1 , S Mavrogeni1 , G Hatzigeorgiou1 , G Kolovou1 , 1Onassis Cardiac Surgery Center - Athens - Greece , 2University Hospital of Heraklion, Nephrology Department - Heraklion - Greece , 3University Hospital of Heraklion, Department of Cardiology - Heraklion - Greece , 4University of Ioannina, Department of Internal Medicine - Ioannina - Greece ,


Introduction: The majority of patients with homozygous familial hypercholesterolemia (HoFH) phenotype are treated additionally to lipid lowering (LL) drugs (statin + ezetimibe ± colesevelam) with lipoprotein apheresis (LA) sessions. The aim of this study was to evaluate the effect of microsomal triglyceride transfer protein inhibitor (lomitapide) in HoFH patients.

Patients and Methods: In 12 HoFH patients treated with LL drugs ± biweekly LA sessions (9 patients) the 5-40 mg daily of lomitapide was added. Lipid profile [total cholesterol (TC), low density lipoprotein cholesterol (LDL-C), triglycerides (TG) and high density lipoprotein cholesterol (HDL-C)] before (LL drugs ± LA) and after lomitapide treatment were evaluated.

Results: The follow-up period of lomitapide treatment for 12 patients was 3-24 months (13.8 ± 7.9). The median baseline LDL-C levels was 1000 mg/dL (339-1150). During LL drug therapy, patients showed a median LDL-C level of 383.5 mg/dL (Range: 214-866 mg/dL) and with LL drugs + Time-averaged levels (CAVG) the median was 288.1 mg/dL (Range: 183.69-716.65 mg/dL). The addition of lomitapide at the average dosage of 21.4 mg/day lowered LDL-C levels by 56.8% comparing to LL drugs alone therapy [mean reduction 262.12, 95% CI (105.53, 418.71), p = 0.005] and by 54% [mean reduction 182.89, 95% CI (-342.35, -23.43), p = 0.031] comparing to LL drugs + LA (CAVG). The CAVG of LDL-C in LL drugs + LA patients compared with LL drugs + lomitapide was 54% in favour of lomitapide (p=0.031).  After lomitapide administration to LL drugs + LA treatment, 78% patients discontinued LA and 2 patients reduced their LA frequency by 50%. During follow-up, 2 patients (16.6%) reported side effects (transient diarrhea, 1 patient had liver transaminase >5× ULN and had to decrease dose of lomitapide). Two patients stopped lomitapide due to diet and alcohol restrictions.  

Conclusions: Treatment with lomitapide in HoFH patients has beneficial effect with constant decrease of LDL-C by 57% compared with classical LL therapy and by 54% compared with classical LL therapy and CAVG and seems to be with good safety profile. Although, in some cases the hybrid (all availably drug treatment and LA) therapy may be needed.

Total Cholesterol




Before any intervention

1000 (339-1150)



34.5 (25-50)

125 (87-314)

After LL drugs

434 (278-915)



36 (27-51)

113 (62-198)

LL drugs + LA(CAVG)



288.07 (183.69-716.65)





LL drugs + Lomitapide

228.5 (118-554)

173.5 (74-515)

37.5 (29-50)

83.5 (23-316)

Median and ranges of lipid and lipoprotein values before any intervention, after LL drugs, LL drugs plus LA, LL drugs plus LA(CAVG) and LL drugs plus lomitapide.

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