Alirocumab efficacy in patients with double heterozygous, compound heterozygous, or homozygous familial hypercholesterolemia  Merel L. Hartgers, MD, Joep.

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Alirocumab efficacy in patients with double heterozygous, compound heterozygous, or homozygous familial hypercholesterolemia  Merel L. Hartgers, MD, Joep C. Defesche, PhD, Gisle Langslet, MD, Paul N. Hopkins, MD, MSPH, John J.P. Kastelein, MD, PhD, FESC, Marie T. Baccara-Dinet, MD, MSc, Werner Seiz, MD, Sara Hamon, MA, PhD, Poulabi Banerjee, PhD, Claudia Stefanutti, MD, PhD  Journal of Clinical Lipidology  Volume 12, Issue 2, Pages 390-396.e8 (March 2018) DOI: 10.1016/j.jacl.2017.12.008 Copyright © 2018 National Lipid Association Terms and Conditions

Figure 1 Percentage change from baseline in LDL-C at (A) week 12 and (B) week 24 for individual patients. Data were not available for patient 2 (APOB defective/LDLR negative). Patient 7 was from the 12-week phase 2 study therefore no data were available at week 24. Patient 11 (PCSK9 GOF and LDLR negative) received a different alirocumab administration regimen and is not included in this figure. APOB, apolipoprotein B; LDL-C, low-density lipoprotein cholesterol; LDLR, low-density lipoprotein receptor; LDLRAP1, LDLR adaptor protein 1; N/A, not available. Journal of Clinical Lipidology 2018 12, 390-396.e8DOI: (10.1016/j.jacl.2017.12.008) Copyright © 2018 National Lipid Association Terms and Conditions

Supplementary Figure 1 Percentage change from baseline in ApoB at weeks 12 and 24. Data were not available for patient 2 (APOB defective/LDLR negative). Patient 7 was from the 12-week phase 2 study therefore no data were available at week 24. Patient 11 (PCSK9 GOF and LDLR negative) received a different alirocumab administration regimen and is not included in this figure. APOB, apolipoprotein B; LDLR, low-density lipoprotein receptor; LDLRAP1, LDLR adaptor protein 1; NA, not available. Journal of Clinical Lipidology 2018 12, 390-396.e8DOI: (10.1016/j.jacl.2017.12.008) Copyright © 2018 National Lipid Association Terms and Conditions

Supplementary Figure 2 Percentage change from baseline in Lp(a) at weeks 12 and 24. Data were not available for patient 2 (APOB defective/LDLR negative). Patient 7 was from the 12-week phase 2 study therefore no data were available at week 24. Patient 11 (PCSK9 GOF and LDLR negative) received a different alirocumab administration regimen and is not included in this figure. APOB, apolipoprotein B; LDLR, low-density lipoprotein receptor; LDLRAP1, LDLR adaptor protein 1; Lp(a), lipoprotein (a); NA, not available. Journal of Clinical Lipidology 2018 12, 390-396.e8DOI: (10.1016/j.jacl.2017.12.008) Copyright © 2018 National Lipid Association Terms and Conditions

Supplementary Figure 3 Percentage change from baseline in non-HDL-C at weeks 12 and 24. Data were not available for patient 2 (APOB defective/LDLR negative). Patient 7 was from the 12-week phase 2 studytherefore no data were available at week 24. Patient 11 (PCSK9 GOF and LDLR negative) received a different alirocumab administration regimen and is not included in this figure. APOB, apolipoprotein B; LDLR, low-density lipoprotein receptor; LDLRAP1, LDLR adaptor protein 1; non-HDL-C, non-high-density lipoprotein cholesterol. Journal of Clinical Lipidology 2018 12, 390-396.e8DOI: (10.1016/j.jacl.2017.12.008) Copyright © 2018 National Lipid Association Terms and Conditions

Supplementary Figure 4 Percentage change from baseline in triglycerides at weeks 12 and 24. Data were not available for patient 2 (APOB defective/LDLR negative). Patient 7 was from the 12-week phase 2 study therefore no data were available at week 24. Patient 11 (PCSK9 GOF and LDLR negative) received a different alirocumab administration regimen and is not included in this figure. APOB, apolipoprotein B; LDLR, low-density lipoprotein receptor; LDLRAP1, LDLR adaptor protein 1; NA, not available. Journal of Clinical Lipidology 2018 12, 390-396.e8DOI: (10.1016/j.jacl.2017.12.008) Copyright © 2018 National Lipid Association Terms and Conditions