The effect of cholesterol-lowering agents on hepatic and plasma cholesterol in Niemann-Pick disease type C

M. C. Patterson; A. M. Di Bisceglie; J. J. Higgins; R. B. Abel; R. Schiffmann; C. C. Parker; C. E. Argoff; R. P. Grewal; K. Yu; P. G. Pentchev; R. O. Brady; N. W. Barton

The effect of cholesterol-lowering agents on hepatic and plasma cholesterol in Niemann-Pick disease type C

M. C. Patterson, A. M. Di Bisceglie, J. J. Higgins, R. B. Abel, R. Schiffmann, C. C. Parker, C. E. Argoff, R. P. Grewal, K. Yu, P. G. Pentchev, R. O. Brady, N. W. Barton

Neurology

Research output: Contribution to journal › Article › peer-review

86 Scopus citations

Abstract

Niemann-Pick disease type C (NP-C) is a neurovisceral lipidosis characterized by defective intracellular trafficking of cholesterol and lysosomal accumulation of unesterified cholesterol, believed to be an offending metabolite. We studied the effect of cholesterol-lowering agents on hepatic and plasma cholesterol levels in NP-C by randomly assigning 25 patients with NP-C to one of five treatment regimens containing different combinations of cholestyramine, lovastatin, nicotinic acid, or dimethyl sulfoxide (DMSO). Unesterified cholesterol content was measured in liver biopsies before and after 4 months' treatment. All drug regimens except DMSO alone reduced hepatic and plasma cholesterol levels. Toxicity was limited and did not prevent any patient from completing the study. The combination of cholestyramine, lovastatin, and nicotinic acid lowered cholesterol levels in liver and blood with minimal side effects. A controlled clinical study will be necessary to determine if this regimen influences the rate of neurologic progression.

Original language	English (US)
Pages (from-to)	61-64
Number of pages	4
Journal	Neurology
Volume	43
Issue number	1
State	Published - Jan 1993

ASJC Scopus subject areas

Clinical Neurology

Cite this

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title = "The effect of cholesterol-lowering agents on hepatic and plasma cholesterol in Niemann-Pick disease type C",

abstract = "Niemann-Pick disease type C (NP-C) is a neurovisceral lipidosis characterized by defective intracellular trafficking of cholesterol and lysosomal accumulation of unesterified cholesterol, believed to be an offending metabolite. We studied the effect of cholesterol-lowering agents on hepatic and plasma cholesterol levels in NP-C by randomly assigning 25 patients with NP-C to one of five treatment regimens containing different combinations of cholestyramine, lovastatin, nicotinic acid, or dimethyl sulfoxide (DMSO). Unesterified cholesterol content was measured in liver biopsies before and after 4 months' treatment. All drug regimens except DMSO alone reduced hepatic and plasma cholesterol levels. Toxicity was limited and did not prevent any patient from completing the study. The combination of cholestyramine, lovastatin, and nicotinic acid lowered cholesterol levels in liver and blood with minimal side effects. A controlled clinical study will be necessary to determine if this regimen influences the rate of neurologic progression.",

author = "Patterson, {M. C.} and {Di Bisceglie}, {A. M.} and Higgins, {J. J.} and Abel, {R. B.} and R. Schiffmann and Parker, {C. C.} and Argoff, {C. E.} and Grewal, {R. P.} and K. Yu and Pentchev, {P. G.} and Brady, {R. O.} and Barton, {N. W.}",

year = "1993",

month = jan,

language = "English (US)",

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journal = "Neurology",

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AU - Patterson, M. C.

AU - Di Bisceglie, A. M.

AU - Higgins, J. J.

AU - Abel, R. B.

AU - Schiffmann, R.

AU - Parker, C. C.

AU - Argoff, C. E.

AU - Grewal, R. P.

AU - Yu, K.

AU - Pentchev, P. G.

AU - Brady, R. O.

AU - Barton, N. W.

PY - 1993/1

Y1 - 1993/1

N2 - Niemann-Pick disease type C (NP-C) is a neurovisceral lipidosis characterized by defective intracellular trafficking of cholesterol and lysosomal accumulation of unesterified cholesterol, believed to be an offending metabolite. We studied the effect of cholesterol-lowering agents on hepatic and plasma cholesterol levels in NP-C by randomly assigning 25 patients with NP-C to one of five treatment regimens containing different combinations of cholestyramine, lovastatin, nicotinic acid, or dimethyl sulfoxide (DMSO). Unesterified cholesterol content was measured in liver biopsies before and after 4 months' treatment. All drug regimens except DMSO alone reduced hepatic and plasma cholesterol levels. Toxicity was limited and did not prevent any patient from completing the study. The combination of cholestyramine, lovastatin, and nicotinic acid lowered cholesterol levels in liver and blood with minimal side effects. A controlled clinical study will be necessary to determine if this regimen influences the rate of neurologic progression.

AB - Niemann-Pick disease type C (NP-C) is a neurovisceral lipidosis characterized by defective intracellular trafficking of cholesterol and lysosomal accumulation of unesterified cholesterol, believed to be an offending metabolite. We studied the effect of cholesterol-lowering agents on hepatic and plasma cholesterol levels in NP-C by randomly assigning 25 patients with NP-C to one of five treatment regimens containing different combinations of cholestyramine, lovastatin, nicotinic acid, or dimethyl sulfoxide (DMSO). Unesterified cholesterol content was measured in liver biopsies before and after 4 months' treatment. All drug regimens except DMSO alone reduced hepatic and plasma cholesterol levels. Toxicity was limited and did not prevent any patient from completing the study. The combination of cholestyramine, lovastatin, and nicotinic acid lowered cholesterol levels in liver and blood with minimal side effects. A controlled clinical study will be necessary to determine if this regimen influences the rate of neurologic progression.

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JF - Neurology

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The effect of cholesterol-lowering agents on hepatic and plasma cholesterol in Niemann-Pick disease type C

Abstract

ASJC Scopus subject areas

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