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

Research output: Contribution to journalArticle

85 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 languageEnglish (US)
Pages (from-to)61-64
Number of pages4
JournalNeurology
Volume43
Issue number1
DOIs
StatePublished - 1993

ASJC Scopus subject areas

  • Clinical Neurology

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