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Faculty of Medicine, Macau University of Science and Technology, Macau, China
Copyright © 2021 Korean Endocrine Society
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (https://creativecommons.org/licenses/by-nc/4.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
CONFLICTS OF INTEREST
Brian Tomlinson has acted as consultant or speaker for Amgen Inc., Kowa, and Merck Serono, for which he received honoraria. The other authors have no conflicts to disclose.
Study, duration, and reference | Patients, number and type | Treatment | Change in LDL-C, %a |
---|---|---|---|
RUTHERFORD, 12 weeks, Raal et al. (2012) [56] | 56 HeFH | Placebo Q4W | 1.1±2.9 |
55 HeFH | E 350 mg Q4W | −42.7±2.9 | |
56 HeFH | E 420 mg Q4W | −55.2±2.9 | |
|
|||
RUTHERFORD-2, 12 weeks, Raal et al. (2015) [58] | 54 HeFH | Placebo Q2W | −1.1 (−5.8 to 3.7) |
110 HeFH | E 140 mg Q2W | −61.2 (−64.6 to −57.9) | |
55 HeFH | Placebo QM | 2.3 (−2.5 to 7.1) | |
110 HeFH | E 420 mg QM | −63.3 (−66.6 to −59.9) | |
|
|||
HAUSER-RCT, 24 weeks, Santos et al. (2020) [60] | 53 HeFHb | Placebo QM | −5.9 (−11.1 to −0.6) |
104 HeFHb | E 420 mg QM | −48.0 (−51.7 to −44.2) | |
|
|||
TESLA, 12 weeks, Stein et al. (2013) [61] | 8 HoFH | E 420 mg Q4W | −16.5±19.0c |
8 HoFH | E 420 mg Q2W | −13.9±27.2c | |
|
|||
TESLA Part B, 12 weeks, Raal et al. (2015) [62] | 16 HoFH | Placebo QM | 7.9 (−2.7 to 18.5) |
33 HoFH | E 420 mg Q4W | −23.1 (30.7 to −15.4) | |
|
|||
TAUSSIG, 12 weeks, Raal et al. (2017) [63] | 106 HoFH | E 420 mg Q4W | −20.6±24.4c |
LDL-C, low-density lipoprotein cholesterol; RUTHERFORD, Reduction of LDL-C with PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder; HeFH, heterozygous familial hypercholesterolemia; Q4W, every 4 weeks; E, evolocumab; Q2W, every 2 weeks; QM, every month; TESLA, The Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities; HoFH, homozygous familial hypercholesterolemia; TAUSSIG, Trial Assessing Long-Term Use of PCSK9 Inhibition in Subjects with Genetic LDL Disorders.
a Values are expressed as least-squares mean±standard error or (95% CI) unless indicated;
b Pediatric heterozygous familial hypercholesterolemia;
c Values are expressed as mean±standard deviation changes.
Study, duration, and reference | Patients, number and type | Treatment | Change in LDL-C, %a |
---|---|---|---|
Phase 2 study, 12 weeks, Stein et al. (2012) [70] | 15 HeFH | Placebo Q2W | −10.65±5.04 |
15 HeFH | A 150 mg Q4W | −28.87±5.08 | |
16 HeFH | A 200 mg Q4W | −31.54±4.91 | |
15 HeFH | A 300 mg Q4W | −42.53±5.09 | |
16 HeFH | A 150 mg Q2W | −67.90±4.85 | |
|
|||
ODYSSEY FH I, 24 weeks; ODYSSEY FH II, 24 weeks, Kastelein et al. (2015) [72] | 163 HeFH | Placebo Q2W | 9.1±2.2 |
322 HeFH | A 75/150 mg Q2W | −48.8±1.6 | |
55 HeFH | Placebo Q2W | 2.8±2.8 | |
166 HeFH | A 75/150 mg Q2W | −48.7±1.9 | |
|
|||
ODYSSEY HIGH FH, 24 weeks, Ginsberg et al. (2016) [73] | 35 HeFH | Placebo Q2W | −6.6±4.9 |
71 HeFH | A 150 mg Q2W | −45.7±3.5 | |
|
|||
ODYSSEY KIDS, 8 weeks, Daniels et al. (2020) [79] | 10 HeFHb | A 30/50 mg Q2W | −21.2±7.9 |
10 HeFHb | A 40/75 mg Q2W | −46.1±8.3 | |
11 HeFHb | A 75/150 mg Q4W | −7.8±7.6 | |
11 HeFHb | A 150/300 mg Q4W | −44.5±7.6 | |
|
|||
ODYSSEY HoFH, 12 weeks, Blom et al. (2020) [80] | 24 HoFH | Placebo Q2W | 8.9±19.0 |
45 HoFH | A 150 mg Q2W | −26.9±4.6 |
LDL-C, low-density lipoprotein cholesterol; HeFH, heterozygous familial hypercholesterolemia; Q2W, every 2 weeks; A, alirocumab; Q4W, every 4 weeks; ODYSSEY FH I, Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy; ODYSSEY FH II, Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy); ODYSSEY HIGH FH, Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia; ODYSSEY KIDS, An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia; HoFH, homozygous familial hypercholesterolemia.
a Values are expressed as least-squares mean±standard error;
b Pediatric heterozygous familial hypercholesterolemia.
Study, duration, and reference | Patients, number and type | Treatment | Change in LDL-C, % |
---|---|---|---|
ORION-2, 180 days, Hovingh et al. (2020) [89] | 4 HoFH | I 300 mg on days 1 and 90/104 | −37.0 to 3.3 |
|
|||
ORION-5, 24 months, (NCT03851705) [90] | 56 HoFH randomized 2:1 to I or placebo | Placebo on days 1, 90 | Awaited |
I 300 mg on days 1, 90, 270, 450, 630 | |||
|
|||
ORION-9, 18 months, Raal et al. (2020) [73] | 240 HeFH | Placebo on days 1, 90, 270, 450 | 8.2 (4.3 to 12.2) |
242 HeFH | I 300 mg on days 1, 90, 270, 450 | −39.7 (−43.7 to −35.7) |
Study, duration, and reference | Patients, number and type | Treatment | Change in LDL-C, % |
---|---|---|---|
RUTHERFORD, 12 weeks, Raal et al. (2012) [56] | 56 HeFH | Placebo Q4W | 1.1±2.9 |
55 HeFH | E 350 mg Q4W | −42.7±2.9 | |
56 HeFH | E 420 mg Q4W | −55.2±2.9 | |
| |||
RUTHERFORD-2, 12 weeks, Raal et al. (2015) [58] | 54 HeFH | Placebo Q2W | −1.1 (−5.8 to 3.7) |
110 HeFH | E 140 mg Q2W | −61.2 (−64.6 to −57.9) | |
55 HeFH | Placebo QM | 2.3 (−2.5 to 7.1) | |
110 HeFH | E 420 mg QM | −63.3 (−66.6 to −59.9) | |
| |||
HAUSER-RCT, 24 weeks, Santos et al. (2020) [60] | 53 HeFH |
Placebo QM | −5.9 (−11.1 to −0.6) |
104 HeFH |
E 420 mg QM | −48.0 (−51.7 to −44.2) | |
| |||
TESLA, 12 weeks, Stein et al. (2013) [61] | 8 HoFH | E 420 mg Q4W | −16.5±19.0 |
8 HoFH | E 420 mg Q2W | −13.9±27.2 | |
| |||
TESLA Part B, 12 weeks, Raal et al. (2015) [62] | 16 HoFH | Placebo QM | 7.9 (−2.7 to 18.5) |
33 HoFH | E 420 mg Q4W | −23.1 (30.7 to −15.4) | |
| |||
TAUSSIG, 12 weeks, Raal et al. (2017) [63] | 106 HoFH | E 420 mg Q4W | −20.6±24.4 |
Study, duration, and reference | Patients, number and type | Treatment | Change in LDL-C, % |
---|---|---|---|
Phase 2 study, 12 weeks, Stein et al. (2012) [70] | 15 HeFH | Placebo Q2W | −10.65±5.04 |
15 HeFH | A 150 mg Q4W | −28.87±5.08 | |
16 HeFH | A 200 mg Q4W | −31.54±4.91 | |
15 HeFH | A 300 mg Q4W | −42.53±5.09 | |
16 HeFH | A 150 mg Q2W | −67.90±4.85 | |
| |||
ODYSSEY FH I, 24 weeks; ODYSSEY FH II, 24 weeks, Kastelein et al. (2015) [72] | 163 HeFH | Placebo Q2W | 9.1±2.2 |
322 HeFH | A 75/150 mg Q2W | −48.8±1.6 | |
55 HeFH | Placebo Q2W | 2.8±2.8 | |
166 HeFH | A 75/150 mg Q2W | −48.7±1.9 | |
| |||
ODYSSEY HIGH FH, 24 weeks, Ginsberg et al. (2016) [73] | 35 HeFH | Placebo Q2W | −6.6±4.9 |
71 HeFH | A 150 mg Q2W | −45.7±3.5 | |
| |||
ODYSSEY KIDS, 8 weeks, Daniels et al. (2020) [79] | 10 HeFH |
A 30/50 mg Q2W | −21.2±7.9 |
10 HeFH |
A 40/75 mg Q2W | −46.1±8.3 | |
11 HeFH |
A 75/150 mg Q4W | −7.8±7.6 | |
11 HeFH |
A 150/300 mg Q4W | −44.5±7.6 | |
| |||
ODYSSEY HoFH, 12 weeks, Blom et al. (2020) [80] | 24 HoFH | Placebo Q2W | 8.9±19.0 |
45 HoFH | A 150 mg Q2W | −26.9±4.6 |
Study, duration, and reference | Patients, number and type | Treatment | Change in LDL-C, % |
---|---|---|---|
ORION-2, 180 days, Hovingh et al. (2020) [89] | 4 HoFH | I 300 mg on days 1 and 90/104 | −37.0 to 3.3 |
| |||
ORION-5, 24 months, (NCT03851705) [90] | 56 HoFH randomized 2:1 to I or placebo | Placebo on days 1, 90 | Awaited |
I 300 mg on days 1, 90, 270, 450, 630 | |||
| |||
ORION-9, 18 months, Raal et al. (2020) [73] | 240 HeFH | Placebo on days 1, 90, 270, 450 | 8.2 (4.3 to 12.2) |
242 HeFH | I 300 mg on days 1, 90, 270, 450 | −39.7 (−43.7 to −35.7) |
LDL-C, low-density lipoprotein cholesterol; RUTHERFORD, Reduction of LDL-C with PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder; HeFH, heterozygous familial hypercholesterolemia; Q4W, every 4 weeks; E, evolocumab; Q2W, every 2 weeks; QM, every month; TESLA, The Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities; HoFH, homozygous familial hypercholesterolemia; TAUSSIG, Trial Assessing Long-Term Use of PCSK9 Inhibition in Subjects with Genetic LDL Disorders. Values are expressed as least-squares mean±standard error or (95% CI) unless indicated; Pediatric heterozygous familial hypercholesterolemia; Values are expressed as mean±standard deviation changes.
LDL-C, low-density lipoprotein cholesterol; HeFH, heterozygous familial hypercholesterolemia; Q2W, every 2 weeks; A, alirocumab; Q4W, every 4 weeks; ODYSSEY FH I, Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy; ODYSSEY FH II, Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy); ODYSSEY HIGH FH, Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia; ODYSSEY KIDS, An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia; HoFH, homozygous familial hypercholesterolemia. Values are expressed as least-squares mean±standard error; Pediatric heterozygous familial hypercholesterolemia.
Values are expressed as range or mean (95% confidence interval). LDL-C, low-density lipoprotein cholesterol; HoFH, homozygous familial hypercholesterolemia; I, inclisiran; HeFH, heterozygous familial hypercholesterolemia.