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CC March 2016 Heterozygous Familial hypercholesterolemia (HeFH) and Alirocumab (Praluent)

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echoecho's version from 2016-04-19 03:54

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Question Answer
What is arcus cornealis?it is lipid depositis within the periphery of the cornea seen in familial hypercholesterolemia
While arcus cornealis may have no implications for lipid siorders in the older patient, would this be significant if seen in a 30 year old?yes
What is familial hypercholesterolemia caused by?a genetic defect that results in reduced clearance of LDL cholesterol from the circulation with a resultant rise in serum LDL levels
Many mutations exist. What are the two forms?1) homozygous form 2) heterozygous form
The homozygous form is very rare, but it is estimated that 1 in ____ to ____ individuals in North American and Europe have the heterozygous familial hypercholesterolemia (HeFH)?200; 500
List the diagnostic criteria for HeFH as listed in the Dutch Lipid Network criteria endorsed by the WHO?1) Family hx (first degree relative with premature coronary artery disease and/or first-degree relative with LDL level > 95% = 1 point. For first-degree relative with tendon xanthomata and/or children < 18 years of age with LDL level > 95% for sex and age = 2 points 2) clinical hx (for patients with premature CAD = 2 points; for patients with premature cerebral or peripheral vascular disease = 1 point) 3) Physical findings (tendon xanthomata = 6 points; for arcus cornealis < 45 years of age = 4 points) 4) LDL cholesterol (> 330 mg/dl = 8 points; 250-329 mg/dL = 5 points; 190-240 mg/dl = 3 points; 155-189 mg/dL = 1 point
What two levels found in the lipid panel are not part of the diagnostic criteria?HDL and total cholesterol
What does the score from the Dutch Lipid Network criteria interpreted?Total score > 8 = defiinite dx of HeFH. 6-7 points = probable for HeFH. 3-5 points = possible dx of HeFH. < 3 points = unlikely dx of HeFH
Define premature CAD in men? In women?1) men before age 55 years of age 2) women before age 60 years of age
What is the initial treatment for patients with HeFH?lifestyle modification (weight reduction, exercise, diet modification, smoking cessation)
Is there any consensus regarding when to start pharmacologic therapy, particularly in children?no
However, the higher ____ levels and the presence of risk fxs for cardiovascular disease should prompt stronger consideration of pharmacotherapy, even at a young age?LDL
What is the main intervention for HeFH if diet and lifestyle modifications do not result in sufficient lowering of LDL levels?statin therapy
If necesary, what additional agents may be added to statin therapy?1) ezetimibe (Zetia) 2) bile-acid sequestrants
In July 2015, the FDA approved what medication for the treatment of HeFH?alirocumab (Praluent)
Explain what is the mechanism of action of Alirocumab (Praluent)?1) alirocumab is a proportein convertase subtilisin / kexin 9 (PCSK9) monoclonal antibody that targets the PCSK9 protein 2) this PCSK9 protein REDUCES the number of receptors in the liver that remove circulating LDL cholesterol thus increasing LDL levels 3) Alirocumab blocks PCSK9, making more receptors available to remove LDL cholesterol, thus lowering serum levels
List the 2 patient types that Alirocumab (Praluent) is indicated for?1) patients with HeFH who do NOT reach goal LDL levels (<100 mg/dL) with maximally-tolerated statin therapy and diet OR 2) patients with clinical atherosclerotic cardiovascular disease (on statin and diet thearpy) who require additional LDL lowering
*** A meta-analysis of 20 randomized controlled trials found that alirocumab (Praluent) was very effective in lowering LDL levels and also lowered what levels while increasing what levels?1) total cholesterol 2) HDL
The average lowering of LDL was ___ to ___% compared to placebo?36; 59
Was there any serious treatment-emergent adverse events with alirocumab?no
How is Alirocumab given?subcutaneous injection once every 2 weeks (initial dose is 75 mg, however, dose can be increased to 150 mg if LDL levels checked 4-8 weks after initiation of therapy do not show an adequate response)
What are the most common reported adverse effects of Alirocumab?nasopharyngitis, inection site reactions and influenza-like symptoms, allergic reactions (including hypersensitivity vasculitis)
What is the cost of one injection? one year of injections?1) $560 per injection 2) $14,600 per year
While Alirocumab is very effective at lowering LDL cholesterol, are there any data regarding the effect of alirocumab on cardiovascular (MI, CVA) outcomes or morbidity?no
*** SUMMARY = What new medication is approved for the treatment of HeFH?Alirocumab (Praluent)
*** SUMMARY = List the two patient types that Alirocumab (Praluent) is indicated for?1) patients with HeFH who do NOT reach goal LDL levels (<100 mg/dL) with maximally-tolerated statin therapy and diet OR 2) patients with clinical atherosclerotic cardiovascular disease (on statin and diet thearpy) who require additional LDL lowering
*** SUMMARY = How is Alirocumab (Praluent) administered and comment on it's price?1) subcutaneous injection 2) very expensive
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