Effect of simvastatin in addition to chenodeoxycholic acid in patients with cerebrotendinous xanthomatosis

The effects of combination therapy with chenodeoxycholic acid (CDCA) and si mvastatin on serum cholestanol, low-density lipoprotein (LDL) cholesterol, and lathosterol levels were investigated in seven adult patients with cereb rotendinous xanthomatosis (CTX) who were on long-term treatment with CDCA. The patients were treated with a combination of CDCA 750 mg daily and an in creasing dose of simvastatin from 10 mg to 40 mg daily for a period of 6 mo nths. We found a significant effect of this combination therapy compared wi th CDCA alone in terms of decreasing the serum cholestanol and LDL choleste rol levels, particularly with a daily dose of 40 mg simvastatin. The mean c holestanol level decreased from 9.27 mu mol/L (baseline) to 6.69 mu mol/L ( 40 mg simvastatin), while the mean LDL cholesterol level decreased from 5.0 8 mmol/L (baseline) to 3.04 mmol/L (40 mg simvastatin). No side effects wer e reported, and there were no effects on the clinical condition, cerebral m agnetic resonance imaging (MRI), visual evoked potentials, and electroencep halographic features. We conclude that a combination of 750 mg CDCA and 40 mg simvastatin daily is effective to further reduce serum cholestanol, LDL cholesterol, and lathosterol in adult CTX patients treated with long-term C DCA. Whether this combination treatment will be effective for the long-term prevention of neurological deterioration and atherosclerosis remains to be established. Copyright (C) 1999 by W.B. Saunders Company.