Verrips A, Wevers R A, Van Engelen B G, Keyser A, Wolthers B G, Barkhof F, Stalenhoef A, De Graaf R, Janssen-Zijlstra F, Van Spreeken A, Gabreëls F J
Dutch Cerebrotendinous Xanthamatosis Research Group, University Hospital Nijmegen, The Netherlands.
Metabolism. 1999 Feb;48(2):233-8. doi: 10.1016/s0026-0495(99)90040-9.
The effects of combination therapy with chenodeoxycholic acid (CDCA) and simvastatin on serum cholestanol, low-density lipoprotein (LDL) cholesterol, and lathosterol levels were investigated in seven adult patients with cerebrotendinous xanthomatosis (CTX) who were on long-term treatment with CDCA. The patients were treated with a combination of CDCA 750 mg daily and an increasing dose of simvastatin from 10 mg to 40 mg daily for a period of 6 months. We found a significant effect of this combination therapy compared with CDCA alone in terms of decreasing the serum cholestanol and LDL cholesterol levels, particularly with a daily dose of 40 mg simvastatin. The mean cholestanol level decreased from 9.27 micromol/L (baseline) to 6.69 micromol/L (40 mg simvastatin), while the mean LDL cholesterol level decreased from 5.08 mmol/L (baseline) to 3.04 mmol/L (40 mg simvastatin). No side effects were reported, and there were no effects on the clinical condition, cerebral magnetic resonance imaging (MRI), visual evoked potentials, and electroencephalographic 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 CDCA. Whether this combination treatment will be effective for the long-term prevention of neurological deterioration and atherosclerosis remains to be established.
在7名长期接受鹅去氧胆酸（CDCA）治疗的成人脑腱性黄瘤病（CTX）患者中，研究了CDCA与辛伐他汀联合治疗对血清胆甾烷醇、低密度脂蛋白（LDL）胆固醇和羊毛甾醇水平的影响。患者接受每日750 mg CDCA与每日剂量从10 mg递增至40 mg辛伐他汀联合治疗，为期6个月。我们发现，与单独使用CDCA相比，这种联合治疗在降低血清胆甾烷醇和LDL胆固醇水平方面有显著效果，尤其是每日服用40 mg辛伐他汀时。平均胆甾烷醇水平从9.27微摩尔/升（基线）降至6.69微摩尔/升（40 mg辛伐他汀），而平均LDL胆固醇水平从5.08毫摩尔/升（基线）降至3.04毫摩尔/升（40 mg辛伐他汀）。未报告有副作用，对临床状况、脑磁共振成像（MRI）、视觉诱发电位和脑电图特征也无影响。我们得出结论，对于长期接受CDCA治疗的成年CTX患者，每日联合使用750 mg CDCA和40 mg辛伐他汀可有效进一步降低血清胆甾烷醇、LDL胆固醇和羊毛甾醇。这种联合治疗对长期预防神经功能恶化和动脉粥样硬化是否有效仍有待确定。
