Promoting LDL Cholesterol Clearance in Pediatric Patients with Heterozygous Familial Hypercholesterolemia with Alirocumab
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Reviewed by Dat Tien Nguyen, B.A, ScM.
Translated by Nhi Phuong Quynh Le, B.A |
Posted on June 14th, 2024
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Statin and other lipid-lowering therapies are frequently used to control the cholesterol-carrying low-density lipoprotein (LDL-C) in pediatric patients with familial cholesterolemia. PSCK9 plays an important role in maintaining a high LDL-C level by destroying the cellular receptor that would clear LDL-C from blood. Alirocumab is an antibody that can inhibit PSCK9, and a study was conducted to assess its capability in promoting LDL-C clearance to manage heterozygous familial hypercholesterolemia in children.
The phase 3 clinical trial included 153 participants between the age of 8 and 17 years old. These patients had been diagnosed with heterozygous familial hypercholesterolemia and have an LDL-C level above 130 mg/dL despite being treated with other therapies such as statin and ezetimibe. The participants were randomly assigned to be injected with either placebo or 2 dose schedules of alirocumab: every 2 weeks or every 4 weeks. For patients who receive an injection every 2 weeks, those who weigh below 50 kg will receive 40 mg of alirocumab; the dose is increased to 75 mg for those who weigh above 50 kg. In the group that was injected with alirocumab every 4 weeks, the dose is increased to 150 mg and 300 for those who weigh below and above 50 kg respectively. After 24 weeks of treatment, there was minimal fluctuation in the LDL-C level in those in the placebo group, but there was a 43.3% and 33.8% reduction in those who were treated with alirocumab every 2 weeks and every 4 weeks. When adverse reactions was analyzed, there was little difference between the 2 dose schedules and the placebo group.
The phase 3 clinical trial included 153 participants between the age of 8 and 17 years old. These patients had been diagnosed with heterozygous familial hypercholesterolemia and have an LDL-C level above 130 mg/dL despite being treated with other therapies such as statin and ezetimibe. The participants were randomly assigned to be injected with either placebo or 2 dose schedules of alirocumab: every 2 weeks or every 4 weeks. For patients who receive an injection every 2 weeks, those who weigh below 50 kg will receive 40 mg of alirocumab; the dose is increased to 75 mg for those who weigh above 50 kg. In the group that was injected with alirocumab every 4 weeks, the dose is increased to 150 mg and 300 for those who weigh below and above 50 kg respectively. After 24 weeks of treatment, there was minimal fluctuation in the LDL-C level in those in the placebo group, but there was a 43.3% and 33.8% reduction in those who were treated with alirocumab every 2 weeks and every 4 weeks. When adverse reactions was analyzed, there was little difference between the 2 dose schedules and the placebo group.