natgillin/translations-raw huggingface dataset:
Desuden forøger simvastatin i moderat grad high - density lipoprotein - (HDL) kolesterol og reducerer plasmatriglycerider.
In addition, simvastatin moderately increases high density lipoprotein (HDL) cholesterol and reduces plasma triglycerides.
Som resultat af disse ændringer reduceres forholdene mellem total til HDL- kolesterol og LDL til HDL- kolesterol.
As a result of these changes the ratios of total to HDL cholesterol and LDL to HDL cholesterol are reduced.
• dysbetalipoproteinæmi
• dysbetalipoproteinemia
19/ 39
25/ 46
En indikation for dysbetalipoproteinæmi (Fredrickson, type III hyperlipidæmi) er blevet godkendt i 6 medlemsstater (Østrig, Frankrig, Island, Irland, Nederlandene og Portugal).
evaluate the efficacy of simvastatin in patients with combined hyperlipidaemia (elevated cholesterol and triglycerides).
Denne tilstand omfatter et antal sygdomme såsom familiær kombineret hyperlipidæmi, andre dårligt karakteriserede polygene syndromer og dysbetalipoproteinæmi.
This condition includes a number of diseases such as familial combined hyperlipidemia, other poorly characterised polygenic syndromes, and dysbetalipoproteinemia.
Undersøgelsen omfattede mænd og kvinder i alderen 21 til 70 år med kombineret hyperlipidæmi, defineret som LDL- kolesterol højere end 130 mg/ dl, og triglycerider mellem 300 og 700 mg/ dl ved uge - 4 og - 2.
The study included men and women 21 to 70 years with combined hyperlipidemia defined as LDL cholesterol greater than 130 mg/ dl, and triglycerides between 300 and 700 mg/ dl at Weeks -4 and -2.
Patienter med type 2 diabetes mellitus var kvalificerede til at deltage i undersøgelsen, forudsat at hæmoglobin A1C ved uge - 4 ikke var ≤10%.
Patients with type 2 diabetes mellitus were eligible to participate provided hemoglobin A1C at Week -4 was not ≤ 10%.
Et hundrede og tredive patienter blev randomiseret til 80 mg simvastatin, 40 mg simvastatin eller placebo.
One hundred and thirty patients were randomised to 80 mg simvastatin, 40 mg simvastatin or placebo.
Det primære mål var at bestemme den LDL- kolesterolreducerende effekt af simvastatin 80 mg/ dag plus diæt og simvastatin 40 mg/ dag plus diæt i forhold til diæt plus placebo hos patienter med kombineret hyperlipidæmi ved slutningen af en behandlingsperiode på 6 uger.
The primary objective were to determine the LDL cholesterol lowering efficacy of simvastatin 80 mg/ day plus diet, and simvastatin 40 mg/ day plus diet relative to diet plus placebo in patients with combined hyperlipidaemia at the end of a 6-week treatment period.
De sekundære mål var, hos patienter med kombineret hyperlipidæmi behandlet i 6 uger at evaluere:
To evaluate in patients with combined hyperlipidaemia treated for 6 weeks:
(1) effekten af simvastatin 80 mg/ dag + diæt og simvastatin 40 mg/ dag + diæt i forhold til diæt + placebo på VLDL- kolesterol, triglycerider, non- HDL- kolesterol og HDL- kolesterol, (2) effekten af simvastatin 80 mg/ dag + diæt og simvastatin 40 mg/ dag + diæt i forhold til diæt + placebo på LDL- kolesterol, VLDL- kolesterol, triglycerider og non- HDL- kolesterol i en undergruppe af patienter med kombineret hyperlipidæmi og non- insulin- dependent diabetes mellitus (NIDDM; type 2 diabetes mellitus), og (3) sikkerheden og tolerabiliteten af simvastatin 80 mg/ dag hos patienter med kombineret hyperlipidæmi.
(1) the effects of S 80 mg/ day + diet and simvastatin 40 mg/ day + diet relative to diet + placebo on VLDL cholesterol, triglycerides, non HDL cholesterol, and HDL cholesterol; (2) the effects of simvastatin 80 mg/ day plus diet and simvastatin 40 mg/ day plus diet relative to diet plus placebo on LDL cholesterol, VLDL cholesterol, triglycerides, and non HDL cholesterol, in a subgroup of patients with combined hyperlipidaemia and non-insulin dependent diabetes mellitus (NIDDM; type 2 diabetes mellitus); and (3) the safety and tolerability of simvastatin 80 mg/ day in patients with combined hyperlipidaemia.
Dosisafhængige procentvise ændringer fra basislinjen blev observeret i samtlige grupper, der modtog behandling med placebo, simvastatin 40 mg og simvastatin 80 mg for det primære endepunkt (LDL - kolesterol) og for de sekundære endepunkter (VLDL- kolesterol, triglycerider, non- HDL- kolesterol, HDL- kolesterol, apolipoprotein B, og apolipoprotein AI).
Dose-dependent percent changes from baseline were observed across the placebo, simvastatin 40 mg and simvastatin 80 mg treatments for the primary end point (LDL cholesterol) and for the secondary end points (VLDL cholesterol, triglycerides, non HDL cholesterol, HDL cholesterol, apolipoprotein B, and apolipoprotein A-I).
Hos patienter med kombineret hyperlipidæmi og type 2 diabetes mellitus (n=24 randomiseret) var de observerede dosisafhængige procentvise ændringer fra basislinjen ikke forskellige fra dem, der observeredes hos ikke- diabetikere.
In patients with combined hyperlipidemia and type 2 diabetes mellitus (n=24 randomized), observed dose-dependent percent changes from baseline were not different from those observed in nondiabetic patients.
Otte patienter viste sig retrospektivt at have dysbetalipoproteinæmi baseret på forhøjet kolesterol og forhøjede triglycerider, tilstedeværelse af apolipoprotein E2/ 2 genotype og et forhold mellem VLDL og triglycerider ≥0, 25.
Eight patients were retrospectively found to have dysbetalipoproteinemia, based on elevated cholesterol and triglycerides, the presence of the apolipoprotein E2/ 2 genotype, and a VLDL/ triglycerides ratio ≥ 0.25.
Indehaveren af markedsføringstilladelsen præsenterer data for 7 patienter (randomiseret til tre grupper) og argumenterer, at der blev fundet en stor og signifikant effekt af simvastatin for alle de undersøgte variabler med undtagelse af HDL- kolesterol (se tabel 1).
The MAH presents data of 7 patients (randomised to three groups) and argues that a large and significant effect of simvastatin was found for all of the variables considered except HDL cholesterol (see table 1).
Median procentvis (%) ændring (95% IC)
Median percent (%) change (95% IC)
Simvastatin 40 mg
Simvastatin 40 mg
Simvastatin 80 mg
Simvastatin 80 mg
Total- c LDL- c VLDL- c HDL- c triglycerider
Total-c LDL-c VLDL-c HDL-c triglycerides
- 7, 6 (- 24, 8; 9, 5) - 8, 1 (- 44, 8; 28, 6) - 3, 8 (- 12, 7; 5, 1) - 1, 6 (- 14, 5; 11, 2) 3, 5 (- 15, 3; 22, 4)
-7.6 (-24.8; 9.5) -8.1 (-44.8; 28.6) -3.8 (-12.7; 5.1) -1.6 (-14.5; 11.2) 3.5 (-15.3; 22.4)
- 49, 6 (- 69, 0; - 30, 3) - 49, 8 (- 63, 1; - 36, 6) - 58, 3 (- 85, 7; - 30, 9) 6, 7 (- 10, 6; 23, 9) - 40, 5 (- 64, 1; - 16, 8)
-49.6 (-69.0; -30.3) -49.8(-63.1; -36.6) -58.3 (-85.7; -30.9) 6.7 (-10.6; 23.9) -40.5 (-64.1; -16.8)
- 51, 6 (- 57, 9; - 45, 3) - 50, 5 (- 57, 5; - 43, 4) - 59, 5 (- 72, 9; - 46, 1) 6, 7 (8- 12, 5; 25, 8) 38, 2 (- 50, 2; - 26, 1)
-51.6 (-57.9; -45.3) -50.5 (-57.5; -43.4) -59.5 (-72.9; -46.1) 6.7 8-12.5; 25.8) 38.2 (-50.2; -26.1)
En undersøgelse gennemført af Stuyt et al. var en undersøgelse med kendskab til det administrerede lægemiddel, der omfattede 12 patienter med familiær dysbetalipoproteinæmi.
A study conducted by Stuyt et al. was an open-label study including 12 patients with familial dysbetalipoproteinemia.
Efter en placebo- periode på tre uger blev de behandlet med stigende doser simvastatin (10 mg to gange om dagen, 20 mg to gange om dagen og 40 mg to gange om dagen) i seks uger.
After a three-week placebo period, they were treated with increasing doses (10 mg twice/ day, 20 mg twice/ day, and 40 mg twice/ day) of simvastatin in six-week.
Med 80 mg dosen faldt middel serum kolesterolniveauet fra 12, 30±4, 96 til 5, 29±1, 24 mmol/ l (middel reduktion 54%) og middel serum triglyceridniveauet faldt fra 8, 77 ± 7, 16 til 3, 61±1, 33 mmol/ l (- 48%); dette skyldtes et fald i VLDL - CPMP/ 459/ 04
With the 80 mg dose, the mean serum cholesterol level decreased from 12.30±4.96 to 5.29±1.24 mmol/ l (mean reduction, 54%) and the mean serum triglycerides level decreased from 8.77 ± 7.16 to 3.61±1.33 mmol/ l (-48%); this was due to a decrease in VLDL and LDL lipids.
Der var et fald i forholdet mellem VLDL- kolesterol og serum triglycerider og i apolipoprotein B og E, hvilket tyder på en reduktion af mængden af cirkulerende atherogene restpartikler.
There was a decrease in the ratio of VLDL cholesterol to serum triglycerides and in the apolipoproteins B and E, suggesting a reduction in the amount of circulating atherogenic remnant particles.
En undersøgelse gennemført af Feussner et al. omfattede 19 patienter med dysbetalipoproteinæmi, som blev behandlet med simvastatin (20 eller 40 mg per dag) alene eller i kombination med gemfibrozil (450 mg per dag) i en undersøgelse på 30 uger med ambulante patienter.
Study conducted by Feussner et al. included 19 patients with dysbetalipoproteinemia who were treated with simvastatin (20 or 40 mg per day) alone or in combination with gemfibrozil (450 mg per day) during a 30-week outpatient study.
Med 20 mg dosen (n=19) faldt middel plasma kolesterolniveauet med 39, 3% (p < 0, 05), og middel plasma triglyceridniveauet faldt med 41, 8% (ns); der var et fald i VLDL- kolesterol på 44, 8% (ns), et fald i LDL- kolesterol på 36, 5% (p < 0, 01) og en stigning i HDL- kolesterol på 18, 1% (ns).
With the 20-mg dose (n=19) the mean plasma cholesterol level decreased by 39.3% (p < 0.05), and the mean plasma triglycerides level decreased by 41.8% (ns); there was a decrease in VLDL cholesterol of 44.8% (ns), a decrease in LDL cholesterol of 36.5% (p < 0.01), and an increase in HDL cholesterol of 18.1% (ns).
Tretten (13) patienter blev behandlet med 40 mg simvastatin per dag.
Thirteen (30) patients were treated with 40 mg simvastatin per day.
Under dette regime var der et signifikant fald i LDL- kolesterol på 22, 3% (p < 0, 01).
Under this regimen there was a significant decrease in LDL cholesterol 22.3% (p < 0.01).
Hos seks patienter, som fortsatte med hyperlipidæmi på monoterapi, blev der givet kombinationsterapi med simvastatin (40 mg/ dag) og gemfibrozil (450 mg/ dag).
In six patients who remained with a hyperlipidaemia on monotherapy combination drug therapy with simvastatin (40 mg/ day) and gemfibrozil (450 mg/ day) was given.
Sammenlignet med simvastatin alene sænkede supplementet med gemfibrozil yderligere plasmakoncentrationerne af totalt kolesterol med 14, 9%, VLDL- kolesterol med 23, 5% og triglycerider med 17, 1%, selv om dette ikke var statistisk signifikant.
Compared to simvastatin alone the addition of gemfibrozil further lowered plasma concentrations of total cholesterol by 14.9%, VLDL cholesterol by 23.5%, and triglycerides by 17.1%, although this was not statistically significant.
En undersøgelse gennemført af Civeira et al. sammenlignede i en dobbeltblind, randomiseret og placebokontrolleret undersøgelse virkningerne af gemfibrozil (1200 mg/ dag) og simvastatin (20 mg/ dag) på lipider, apolipoprotein AI, apolipoprotein B, apolipoprotein E og på lipider og apolipoprotein B- indholdet i VLDL, intermediate- density lipoprotein (IDL), LDL og HDL hos 10 patienter med type III hyperlipoproteinæmi.
Study conducted by Civeira et al. compared in a double-blind, randomised and placebo-controlled trial the effects of gemfibrozil (1200 mg/ day) and simvastatin (20 mg/ day) on lipids, apolipoprotein AI, apolipoprotein B, and apolipoprotein E and on lipids and apolipoprotein B content in VLDL, intermediate-density lipoprotein (IDL), LDL, and HDL in 10 patients with type III hyperlipoproteinemia.
Niveauer af totalt kolesterol, VLDL- kolesterol, IDL- kolesterol og apolipoprotein B faldt med begge lægemidler.
Levels of total cholesterol, VLDL cholesterol, IDL cholesterol, and apolipoprotein B decreased with both drugs.
Større reduktioner i triglycerider (109±28, 2 mg/ dl, p=0, 005), VLDL- kolesterol (24, 7±10, 9 mg/ dl, p=0, 05) og VLDL triglycerider (86, 3±20, 2 mg/ dl, p=0, 003) blev opnået med gemfibrozil sammenlignet med simvastatin.
Larger reductions in triglycerides (109±28.2 mg/ dl, p=0.005), VLDL cholesterol (24.7±10.9 mg/ dl, p=0.05), and VLDL triglycerides (86.3±20.2 mg/ dl, p=0.003) were obtained with gemfibrozil compared with simvastatin.
Reduktionen af LDL- kolesterol var mere effektiv med simvastatin end med gemfibrozil (44, 3±17, 1 mg/ dl, p=0, 03).
LDL cholesterol reduction was more effective with simvastatin than with gemfibrozil (44.3±17.1 mg/ dl, p=0.03).
HDL- kolesterol efter gemfibrozil var 5, 71±2, 37 mg/ dl højere end efter simvastatin.
HDL cholesterol after gemfibrozil was 5.71±2.37 mg/ dl higher than after simvastatin.
• hypertriglyceridæmi
• hypertriglyceridemia
En indikation for hypertriglyceridæmi (Fredrickson, type IV hyperlipidæmi) blev godkendt af 4 medlemsstater (Østrig, Tyskland, Island og Portugal).
An indication for hypertriglyceridemia (Fredrickson’ s Type IV hyperlipidaemia) was granted by 4 Member States (Austria, Germany, Iceland, and Portugal).
(1) den triglyceridsænkende effekt af doser på 20, 40 eller 80 mg/ dag, (2) effekten af triglyceridernes niveau ved basislinjen på den triglyceridsænkende effekt, (3) effekten af simvastatin CPMP/ 459/ 04
This indication was mainly based on study CPMP/ 459/ 04
21/ 39
27/ 46
20, 40 eller 80 mg/ dag på LDL- kolesterol, VLDL- kolesterol, non- HDL- kolesterol, HDL- kolesterol, apolipoproteiner (apos) B, A- I, E, C- III og fibrinogen, (4) effekterne af 20, 40 eller 80 mg/ dag på lipoprotein restmetabolisme og (5) tolerabiliteten af simvastatin ved doser på 20, 40 eller 80 mg/ dag.
(1) the triglycerides-lowering efficacy of 20, 40, or 80mg/ day doses; (2) the effect of baseline triglycerides levels on triglycerides-lowering efficacy; (3) the effects of simvastatin 20, 40, or 80 mg/ day on LDL cholesterol, VLDL cholesterol, non-HDL cholesterol, HDL cholesterol, apolipoproteins (apos) B, A-I, E, C-III, and fibrinogen; (4) the effects of simvastatin 20, 40, or 80 mg/ day on lipoprotein remnant metabolism; and (5) the tolerability of simvastatin at doses of 20, 40, or 80 mg/ day.
Resultater vedrørende effekt:
Efficacy results:
For det primære endepunkt, triglycerider, de sekundære endepunkter, LDL- kolesterol, apo B, HDL- kolesterol, non- HDL- kolesterol og VLDL- kolesterol var trend- testene signifikante med hensyn til respons i samtlige behandlingsgrupper med placebo, simvastatin 20, simvastatin 40 og simvastatin 80 mg.
For the primary endpoint, triglycerides, the secondary endpoints, LDL cholesterol, apo B, HDL cholesterol, non-HDL cholesterol, and VLDL cholesterol, the trends tests were significant in the response across placebo, simvastatin 20, simvastatin 40, and simvastatin 80 mg.
I undergruppen af patienter med isoleret hypertriglyceridæmi blev der observeret mediane procentvise ændringer i triglycerider på henholdsvis - 13, 3%, - 20, 7%, - 20, 6% og - 33, 0% for placebo, simvastatin 20, 40 og 80 mg behandlingsgrupperne (se tabel 3).
In the subgroup of patients with isolated hypertriglyceridemia, median triglycerides percent changes of -13.3, -20.7, -20.6, and -33.0% were observed for the placebo, simvastatin 20, 40, and 80 mg treatment groups, respectively (see Table 3).
Trend- testen var signifikant med hensyn til triglycerid- respons over hele området af doser fra placebo til simvastatin 80 mg (p=0, 001).
The trend test was significant in the triglycerides response over the range of doses from placebo through simvastatin 80 mg (p=0.001).
Behandlingseffekten af den triglyceridsænkende respons var signifikant i grupperne fra placebo til simvastatin 40 mg (p=0, 041).
The treatment effect of the triglycerides-lowering response was significant between the placebo through simvastatin 40 mg (p=0.041).
Der blev ikke opnået nogen signifikant forskel mellem placebo og 20 mg simvastatin- behandlingen (p > 0, 100).
No significant difference between the placebo and 20-mg treatment was achieved (p > 0.100).
Total- kolesterol, LDL- kolesterol og apolipoprotein B faldt signifikant.
Total cholesterol, LDL cholesterol and apolipoprotein B were significantly reduced.
Tabel 2, Middel1/ median2 procentvis ændring fra basislinjen
Mean1/ Median2 percent change from baseline
TG²
TG²
LDL- c1
LDL-c1
Apo B1
Apo B1
HDL- c1
HDL-c1
VLDL- c2
VLDL-c2
- 13, 3( 35, 3)
-13.3(35.3)
3, 4( 16, 1)
3.4(16.1)
- 1, 6( 8, 7)
-1.6(8.7)
2, 7( 9, 9)
2.7(9.9)
- 10, 3( 39, 0)
-10.3(39.0)
S 20 mg S 40 mg S 80 mg
S 20 mg S 40 mg S 80 mg
- 20, 7( 38, 6) - 20, 6( 26, 1) - 33, 0( 19, 6)
-20.7(38.6) -20.6(26.1) -33.0(19.6)
- 22, 6( 15, 5) - 25, 2( 18, 2) - 34, 5( 14, 5)
-22.6(15.5) -25.2(18.2) -34.5(14.5)
- 22, 2( 15, 8) - 23, 5( 10, 1) - 31, 2( 11, 7)
-22.2(15.8) -23.5(10.1) -31.2(11.7)
9, 0( 16, 8) 9, 1( 11, 3) 10, 8( 9, 5)
9.0(16.8) 9.1(11.3) 10.8(9.5)
- 33, 4( 27, 6) - 34, 9( 29, 5) - 43, 8( 22, 1)
-33.4(27.6) -34.9(29.5) -43.8(22.1)
Ingen patient oplevede forhøjelse af ALT og/ eller AST > 3 X ULN.
No patient experienced any elevation of ALT and/ or AST > 3 X ULN.
Én patient i simvastatin 40 mg gruppen havde en forhøjelse af CK > 5 X ULN i løbet af undersøgelsen, men blev ikke betragtet som en AE af investigatoren.
One patient in the simvastatin 40-mg group had an elevation of CK > 5 X ULN during the study, but was not considered an AE by the investigator.
Der blev ikke observeret tegn på en forøget tendens i forekomsten af CK > 5 X ULN med stigende doser fra placebo til simvastatin 80 mg dosen.
No evidence of an increasing trend in the incidence of CK > 5 X ULN was observed with increasing doses from the placebo to the simvastatin 80-mg dose.
Der blev ikke observeret myopati eller forhøjelse af CK > 10 X ULN i løbet af undersøgelsen.
No myopathy or elevation of CK > 10 X ULN was observed during the study.
Ifølge NCEP’ s ekspertpanel III er målet for non- HDL- kolesterol med et triglyceridsænkende lægemiddel sædvanligvis inden for rækkevidde, når LDL- kolesterolniveauerne ikke er signifikant forhøjede.
According to the NCEP Expert Panel III, when LDL cholesterol levels are not significantly elevated, the goal for non-HDL cholesterol with a triglycerides-lowering drug usually is within reach.
Blandt disse er nikotinsyre normalt den mest effektive, da den reducerer triglyceriderne med 30- 50%, sædvanligvis uden at forårsage en reciprok forhøjelse i LDL- kolesterol, og samtidig forøger nikotinsyre koncentrationerne af HDL- kolesterol med 20- 30%.
Among these, nicotinic acid is usually the most effective as it reduces triglycerides by 30-50 percent usually without causing a reciprocal increase in LDL cholesterol and at the same time, nicotinic acid raises HDL cholesterol concentrations by 20-30 percent.
Hos personer med kontraindikationer over for nikotinsyre, eller hos hvem dette stof tolereres dårligt, reducerer fibrinsyrederivater triglyceriderne med 40- 60% og medfører en stigning på 15- 25% af koncentrationerne af HDL- kolesterol.
In persons with contraindications to nicotinic acid or in whom this drug is poorly tolerated, fibric acid derivatives reduce triglycerides by 40– 60%, and cause a 15– 25 percent increase in HDL-cholesterol concentrations.
• homozygot familiær hypercholesterolæmi
Nevertheless, fibrates often raise
En indikation for Zocord i homozygot familiær hypercholesterolæmi (FH) blev godkendt af 11 medlemsstater (Østrig, Belgien, Luxembourg, Frankrig, Grækenland, Storbritannien, Irland, Nederlandene, Portugal, Spanien og Island.).
CPMP/ 459/ 04
22/ 39
28/ 46
evaluering af effekten og sikkerheden af højdosis simvastatin (80 og 160 mg) hos patienter med homozygot familiær hypercholesterolæmi.
This indication is based on Study 114, a single-centre, double blind, parallel, 2-period, dose escalation study to evaluate the efficacy and safety of high-dose simvastatin (80 and 160 mg) in patients with Homozygous Familial Hypercholesterolemia.
I denne undersøgelse blev 12 patienter med homozygot familiær hypercholesterolæmi randomiseret til simvastatin 40 mg administreret som en enkelt dosis eller 80 mg/ dag (i 3 doser) efter en diæt/ placeboindkøringsfase på 4 uger.
In this study 12 patients with homozygous familial hypercholesterolemia were randomized to simvastatin 40 mg administered as a single dose or 80 mg/ day (in 3 doses), following a 4-week diet/ placebo run-in phase.
Otte patienter var randomiseret til simvastatin 80 mg/ dag og 4 patienter til 40 mg/ dag.
Eight patients randomised to simvastatin 80 mg/ day and 4 patients to 40 mg/ day.
Efter 9 ugers behandling fik de patienter, der startede med at modtage simvastatin 80 mg/ dag, deres dosis forøget til 160 mg/ dag (givet som 3 delte doser) i yderligere 9 uger, mens de patienter, der startede med simvastatin 40 mg om aftenen, modtog 40 mg givet i 3 delte doser.
After 9 weeks of treatment, patients initially receiving simvastatin 80 mg/ day had their dose increased to 160 mg/ day (given in 3 divided doses) for an additional 9 weeks while those on simvastatin 40 mg in the evening received 40 mg given in 3 divided doses.
Det primære mål var at evaluere den LDL- kolesterolsænkende effekt af simvastatin ved doser på 80 og 160 mg/ dag hos patienter med homozygot familiær hypercholesterolæmi, og det sekundære mål var at bestemme sikkerhedsprofilen på kort sigt af simvastatin ved doser på 80 og 160 mg/ dag hos denne patientpopulation.
The primary objective was to evaluate the LDL cholesterol lowering efficacy of simvastatin at doses of 80 and 160 mg/ day in patients with homozygous familial hypercholesterolemia and the secondary objective was to determine the short-term safety profile of simvastatin at doses of 80 and 160 mg/ day in this patient population.
Homozygot familiær hypercholesterolæmi var defineret ved et LDL- kolesterolniveau ≥500 mg/ dl og tilstedeværelsen af mindst 2 af følgende: sene- xanthoma, begge forældre med en diagnose af familiær hypercholesterolæmi eller en LDL- receptor genotype indicerende tilstedeværelsen af en mutation i begge LDL- receptor gener.
Homozygous familiar hypercholesterolemia was defined by an LDL cholesterol level ≥ 500 mg/ dl and the presence of at least 2 of the following: tendinous xanthoma, both parents with a diagnosis of familial hypercholesterolemia or an LDL-receptor genotype indicating the presence of a mutation in both LDL receptor genes.
Tolv patienter i alderen mellem 13 og 40 år var rekrutteret.
Twelve patients aged between 13 to 40 years were recruited.
Med hensyn til effekt blev total- kolesterol, LDL- kolesterol, HDL- kolesterol, triglycerider, Lp( a), apolipoprotein B og E vurderet.
Regarding efficacy, total cholesterol, LDL cholesterol, HDL cholesterol, triglycerides, Lp(a), apolipoproteins B and E were assessed.
Der blev foretaget evaluering af kliniske og laboratoriemæssige hændelser (AEer), vitale funktioner samt rutinemæssige laboratoriesikkerhedstest.
Evaluation of clinical and laboratory adverse experiences (AEs), vital signs, and routine laboratory safety tests were made.
Der blev udført prædefinerede ” limits of change ” - analyser for følgende laboratorietest: alkalisk fosfatase, total bilirubin, hæmatokrit, hæmoglobin, neutrofiler, blodpladetal, serumkreatinin, serumglukose, serumurinsyre og WBC- tal.
Predefined limits of change analyses were performed for the following laboratory tests: alkaline phosphatase, total bilirubin, hematocrit, hemoglobin, neutrophils, platelet count, serum creatinine, serum glucose, serum uric acid, and WBC count.
Der blev fundet en middel ændring fra basislinjen på - 24, 6% og - 30, 6% ved doserne på henholdsvis 80 mg og 160 mg for LDL- kolesterol, en middel ændring på henholdsvis - 23, 0% og - 28, 8% i total - kolesterol, og en middel ændring på henholdsvis +7, 4% og +4, 7% i HDL- kolesterol ved 80 mg og 160 mg (se tabel 3).
A mean change from baseline of – 24.6 and – 30.6% at the 80 mg and 160 mg doses for LDL cholesterol, respectively, a mean change of -23.0% and -28.8% in total cholesterol and respectively, and a mean change of +7.4% and +4.7% 24.5% at the 80 mg and 160 mg, respectively, were found (see Table 3).
Middel ændring (%) fra basislinjen (SD)
Mean change (%) from baseline (SD)
LDL- c (mg/ dl)
LDL-c (mg/ dl)
Total- c (mg/ dl)
Total-c (mg/ dl)
HDL- c (mg/ dl)
HDL-c(mg/ dl)
TG (mg/ dl)
TG (mg/ dl)
40 mg/ dag (1 dosis)
40 mg/ day (1 dose)
- 13, 7 (7, 5)
-13.7 (7.5)
- 12, 3 (6, 6)
-12.3 (6.6
9, 3 (11, 1)
9.3 (11.1)
- 3, 5 (28, 7)
-3.5 (28.7)
40 mg/ dag (3 doser) 80 mg/ dag (3 doser)
40 mg/ day (3 doses) 80 mg/ day (3 dose)
- 13, 2 (10, 3) - 24, 6 (18, 9)
-13.2 (10.3) -24.6 (18.9)
- 11, 4 (9, 4) - 23, 0 (16, 7)
-11.4 (9.4) -23.0 (16.7)