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

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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
StatePublished - Jan 1993
Externally publishedYes

Fingerprint

Type C Niemann-Pick Disease
Cholesterol
Liver
Cholestyramine Resin
Lovastatin
Niacin
Dimethyl Sulfoxide
Lipidoses
Nervous System
Biopsy

ASJC Scopus subject areas

  • Neuroscience(all)

Cite this

Patterson, M. C., Di Bisceglie, A. M., Higgins, J. J., Abel, R. B., Schiffmann, R., Parker, C. C., ... Barton, N. W. (1993). The effect of cholesterol-lowering agents on hepatic and plasma cholesterol in Niemann-Pick disease type C. Neurology, 43(1), 61-64.

The effect of cholesterol-lowering agents on hepatic and plasma cholesterol in Niemann-Pick disease type C. / Patterson, M. C.; Di Bisceglie, A. M.; Higgins, J. J.; Abel, R. B.; Schiffmann, R.; Parker, C. C.; Argoff, C. E.; Grewal, R. P.; Yu, K.; Pentchev, P. G.; Brady, R. O.; Barton, N. W.

In: Neurology, Vol. 43, No. 1, 01.1993, p. 61-64.

Research output: Contribution to journalArticle

Patterson, MC, Di Bisceglie, AM, Higgins, JJ, Abel, RB, Schiffmann, R, Parker, CC, Argoff, CE, Grewal, RP, Yu, K, Pentchev, PG, Brady, RO & Barton, NW 1993, 'The effect of cholesterol-lowering agents on hepatic and plasma cholesterol in Niemann-Pick disease type C', Neurology, vol. 43, no. 1, pp. 61-64.
Patterson MC, Di Bisceglie AM, Higgins JJ, Abel RB, Schiffmann R, Parker CC et al. The effect of cholesterol-lowering agents on hepatic and plasma cholesterol in Niemann-Pick disease type C. Neurology. 1993 Jan;43(1):61-64.
Patterson, M. C. ; Di Bisceglie, A. M. ; Higgins, J. J. ; Abel, R. B. ; Schiffmann, R. ; Parker, C. C. ; Argoff, C. E. ; Grewal, R. P. ; Yu, K. ; Pentchev, P. G. ; Brady, R. O. ; Barton, N. W. / The effect of cholesterol-lowering agents on hepatic and plasma cholesterol in Niemann-Pick disease type C. In: Neurology. 1993 ; Vol. 43, No. 1. pp. 61-64.
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