Dynamics of clinical and laboratory indices in children with glycogen storage disease type I on comprehensive therapy

Abstract

Treatment of the glycogen storage disease (GSD) type I with uncooked cornstarch (UCS) allows to maintain long-term normoglycemia, however, the efficacy of the treatment declines due to the insufficient compliance of children patients and their parents. There were determined clinical and laboratory features of the course of GSD type I in children on the long-term comprehensive therapy. There was performed a retrospective analysis of case histories data on 19 GSD children, including 8 patients with subtype Ia and 11 cases - with subtype Ib. All children received the treatment with both UCS and hepatoprotectors. It was established that during the treatment liver sizes did not change significantly, the fasting blood glucose concentration increased by 2.8 times to 4.21 mmol/l, lactate content decreased by 1.9 times to 5.1 mmol/L, ALT activity decreased by 2.5 times, AST - by 3.3 times, and GGT - by 1.8 times if compared to the level before the treatment. In children with GSD subtype Ib blood concentrations of triglycerides prior to the treatment were 2 times higher than baseline values andfailed to change on the comprehensive therapy. Blood levels of cholesterol and uric acid after the treatment also were not significantly dependent on the duration of the therapy. The authors suggest that comprehensive course therapy of GSD type I by means of prescription to children UCS in combination with hepatoprotectors contributes to the compensation of carbohydrate metabolism and provides the relief of cytolysis and cholestasis, but disturbances of the purine and lipid metabolism in patients persist.