先天性尿素サイクル異常症に伴う高アンモニア血症治療薬L-アルギニン, 安息香酸ナトリウムの錠剤化とその臨床応用

Hyperammonemia which is caused by inherited urea cycle enzyme deficiencies, is often fatal when acute symptoms of hyperammonemic coma (vomiting, unconsciousnessf convulsion, respiratory paralysis, et al.) appear and continue without any medical treatment. L-Arginine or sodium benzoate has been used for the treatment of hyperammonemia. However, it is difficult for children to take such reagents orally in powder form due to the bulky dose volume and their offensive bitter taste. A significant decrease in medication compliance has thus led to hyperammonemic coma or liver transplantation.In this study, we prepared tablets containing a high percentage of L-arginine or sodium benzoate by the wetgranulation method, and administered them to several children with hyperammonemia. Remarkable improvements in the compliance and clinical effects, as well as decreases in the occurance of comas and the serum ammonium levels, have been observed in all patients.