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JIMD Rep. 2016;26:85-90. doi: 10.1007/8904_2015_488. Epub 2015 Aug 25.

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

Author information

1
Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.
2
Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA. weinsda@peds.ufl.edu.

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.

KEYWORDS:

Glycogen storage disease; Ketotic hypoglycemia; Uncooked cornstarch; Waxy maize extended release cornstarch

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