Safety and Efficacy of Chronic Extended Release Cornstarch Therapy for Glycogen Storage Disease Type I.

ABSTRACT

BACKGROUND: Glycogen storage disease type I (GSD I) causes severe hypoglycemia during periods of fasting since both glycogenolysis and gluconeogenesis are impaired. Primary treatment in North America consists of cornstarch therapy every 3-4 h. Waxy maize extended release cornstarch was introduced for maintaining overnight glucose concentrations, but no studies have assessed long-term safety and efficacy of the product. OBJECTIVE: To demonstrate the safety and efficacy of modified cornstarch in GSD I. DESIGN: An open-label overnight trial of extended release cornstarch was performed. Subjects with a successful trial (optimal metabolic control 2 or more hours longer than with traditional cornstarch) were given the option of continuing into the chronic observational phase. Subjects were assessed biochemically at baseline and after 12 months. RESULTS: Of the 106 subjects (93 GSD Ia/13 GSD Ib), efficacy was demonstrated in 82 patients (88%) with GSD Ia and 10 patients (77%) with GSD Ib. The success rate for extending fasting was 95% for females and 78% for males. Of the patients who entered the longitudinal phase, long-term data are available for 44 subjects. Mean duration of fasting on traditional cornstarch prior to study for the cohort was 4.1 and 7.8 h on the extended release cornstarch (P < 0.001). All laboratory markers of metabolic control have remained stable in the chronically treated patients. CONCLUSION: Extended release cornstarch appears to improve the quality of life of patients with GSD I without sacrificing metabolic control. Avoiding the overnight dose of cornstarch should enhance safety in this population.