Normal Cholesterol Levels With Lovastatin (Mevinolin) Therapy in a Child With Homozygous Familial Hypercholesterolemia Following Liver Transplantation

East, Cara; Grundy, Scott M.; Bilheimer, David W.

doi:10.1001/jama.1986.03380200081026

Cited by 42 publications

(11 citation statements)

References 28 publications

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“…Liver transplantation provides a source of normal LDL‐Rs, which may clear cholesterol from plasma so effectively that the disease may be completely cured without additional medication (13–19) (Table 4). The liver is not the only source of LDL‐Rs; therefore, some cures may be incomplete, and patients may require additional medications to control the high cholesterol levels (11, 20–22). The success of treatment depends on the number of normal receptors transplanted.…”

Section: Resultsmentioning

confidence: 99%

Liver transplantation as a treatment option for three siblings with homozygous familial hypercholesterolemia

Tevfik

Yankol

Kanmaz

et al. 2011

Pediatric Transplantation

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Section: Resultsmentioning

confidence: 99%

Liver transplantation as a treatment option for three siblings with homozygous familial hypercholesterolemia

Tevfik

Yankol

Kanmaz

et al. 2011

Pediatric Transplantation

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“…Cholesterol-lowering drugs, such as HMG-CoA reductase inhibitors [3,6,81, are ineffective because LDL receptors are almost absent in patients with HFH. Selective LDL plasmapheresis can decrease the plasma concentration of cholesterol to approximately 50% of the initial level [l, 91.…”

Section: Discussionmentioning

confidence: 99%

Living-donor liver transplantation for homozygous familial hypercholesterolemia from a donor with heterozygous hypercholesterolemia

Shirahata¹,

Ohkohchi²,

Kawagishi³

et al. 2003

Transplant Int

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Familial hypercholesterolemia is a rare inherited disease with an incidence of approximately one per million. Severe hypercholesterolemia is observed from the time of birth onwards. It is associated with severe atherosclerosis in childhood, leading to death from myocardial infarction before the age of 20 years. Liver transplantation is the only effective treatment for this disease. We experienced the case of an infant aged 2 years 5 months who had homozygous familial hypercholesterolemia and who received a liver graft from his father, who had familial heterozygous hypercholesterolemia. The pre-operative plasma cholesterol level was > 800 mg/dl. After liver transplantation, the recipient's cholesterol level decreased to 250 mg/dl after we administered the HMC-CoA reductase inhibitor. At present, 6 months after transplantation, the patient is doing well and free from a special diet. We can thus conclude that the combination therapy of liver transplantation from a donor with heterozygous familial hypercholesterolemia on cholesterol-lowering drugs is an effective therapy for a patient with the homozygous type of hypercholesterolemia.

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“…However, no major side effects have resulted from its use in children with familial hypercholesterolemia (28)(29)(30)(31)(32). Based on our and other's (1, 10-12) experience with safety and efficacy of lovastatin in children with CESD, we speculate that ongoing controlled clinical trials in children with severe heterozygous familial hypercholesterolemia will demonstrate that lovastatin is not only more effective therapeutically than currently approved treatment alternatives (bile acid binding resins) but also safely produces these effects without interfering with normal growth and development.…”

Section: Discussionmentioning

confidence: 99%

Safety and Efficacy of Treatment of Pediatric Cholesteryl Ester Storage Disease with Lovastatin

et al. 1992

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ABSTRACT. The aim of this study was to prospectively assess the safety and efficacy of lovastatin in the treatment of cholesteryl ester storage disease in siblings who were ages 11.6 and 5 y at the beginning of treatment. Mean total and LDL cholesterol in the male proband, 7.40 and 5.68 mmol/L, respectively, on diet alone, fell 30% to 5.2 ( p 5 0.001) and 31% to 3.9 mmol/L ( p 5 0.001) on lovastatin 40 mg/d over 3.3 y, with simultaneous resolution of hepatosplenomegaly. In his sister, on lovastatin 20 mg/d for 1.5 y, total and LDL cholesterol fell, but not significantly; her hepatosplenomegaly was also reduced on treatment. Lovastatin was well tolerated without overt side effects or complications and without adverse changes in liver function tests or creatine phosphokinase. Normal and expected accretion of height and weight occurred during the treatment period for both children. Lovastatin appears to be a safe and effective treatment for pediatric cholesteryl ester storage disease. (Pediatr Res 32: 559-565, 1992) Abbreviations CESD, cholesteryl ester storage disease LDLC, low density lipoprotein cholesterol HDLC, high density lipoprotein cholesterol CPK, creatine phosphokinase GGT, 7-glutamyl transferase SGPT, serum glutamate pyruvate transaminase CESD is a rare, recessively inherited disorder characterized by very low or unmeasurable levels of the hepatic lysosomal enzyme cholesteryl ester hydrolase (1-14). Patients with CESD usually have hepatosplenomegaly because of hepatic lysosomal sequestration of cholesteryl ester and commonly have high plasma cholesterol, LDLC, and triglycerides and very low levels of HDLC. High LDLC and low HDLC in CESD patients puts them at markedly increased risk for premature coronary artery disease. Moreover, ongoing storage of cholesteryl esters in the hepatic lysosomes, augmented by hyperlipidemia, leads to progressive hepatosplenomegaly. Consequent complications may include hepatic fibrosis, cirrhosis, and liver failure, with associated portal hypertension, varices, and hypersplenism.Until the recent availability of lovastatin (1, 10, 1 l), CESD could not be satisfactorily treated because diet and other lipid lowering drugs were not effective. In 1987, successful therapy of CESD with the newly available hydroxymethylglutaryl-CoA reductase inhibitor, lovastatin, was reported by Ginsberg et al. (I).

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Normal Cholesterol Levels With Lovastatin (Mevinolin) Therapy in a Child With Homozygous Familial Hypercholesterolemia Following Liver Transplantation

Cited by 42 publications

References 28 publications

Liver transplantation as a treatment option for three siblings with homozygous familial hypercholesterolemia

Liver transplantation as a treatment option for three siblings with homozygous familial hypercholesterolemia

Living-donor liver transplantation for homozygous familial hypercholesterolemia from a donor with heterozygous hypercholesterolemia

Safety and Efficacy of Treatment of Pediatric Cholesteryl Ester Storage Disease with Lovastatin

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