EFFECTS OF CORNSTARCH TREATMENT IN ADOLESCENT PATIENTS WITH GLYCOGEN STORAGE DISEASE TYPE 1

Abstract

Glycogen storage disease (GSD) type la is known to cause progressive, severe renal dysfunction and growth retardation unless adequately treated. Although dietary therapy has been successful in promoting growth, improving metabolic control and renal function, data in Pediatrics has remained limited. We report on the beneficial effects of long-term cornstarch therapy in 17 adolescent patients (17±2.3 yrs.) with GSD type 1. Patients: After 8±0.9yrs. (X±SEM) of continuous nocturnal nasogastric tube feedings (NNG) with elemental formula, 11 patients had their dietary therapy supplemented with 2gms/Kg of uncooked cornstarch (UCS) for 5.9±0.7yrs, and 6 were managed with UCS alone for 5±0.4yrs. Biochemical, growth and renal function parameters were compared pre and post cornstarch therapy. Results:Table Patients maintained normoglycemia on all forms of dietary therapy. Liver adenoma was present in 5 patients and disappeared in one after 2 years of UCS therapy. In 2 of 8 patients, epistaxis improved on UCS therapy. Conclusions: Corn starch therapy improves growth and blood lipid profiles. Normoglycemia and normalized serum uric acids reflects improved metabolic control. Renal hyperfiltration was refractory to all forms of dietary management, and may reflect the need for early institution of effective cornstarch therapy. Long-term trials will be needed to assess whether dietary cornstarch therapy will prevent the progression of renal disease.