Continuous Nocturnal Intragastric Feeding for Management of Type 1 Glycogen-Storage Disease

Abstract

The clinical and biochemical abnormalities associated with Type 1 glycogen-storage disease can be reversed by avoidance of hypoglycemia and secondary hormonal flux. Three patients with Type 1 disease were treated with intragastric infusions of a high glucose formula at night with three-hour starch feedings during the day. This regimen stabilized blood glucose levels above 70 mg per deciliter and decreased serum uric acid, triglyceride, lactate and serum oxalacetic transaminase levels, as well as hepatic size, in all patients. Increased linear growth rate (mean 1 cm per month) was associated with a decrease in mean plasma glucagon (from 190 to 40 pg per milliliter) and an increase in mean plasma insulin (from 19 to 43 muU per milliliter, [two patients]). These changes occurred within four weeks of beginning of treatment and continued with home treatment for 13 months. No complications resulted from tube placement daily by the patients. Type 1 disease can be managed by nighttime intragastric feeding and frequent daytime high starch meals.

Necrotic foci, elevated chemokines and infiltrating neutrophils in the liver of glycogen storage disease type Ia

“…GSD-Ib patients exhibit additional clinical manifestations of neutropenia and myeloid dysfunctions (1)(2)(3)(4). In the last two and half decades, effective dietary therapies (5,6) have significantly alleviated the metabolic abnormalities of GSD-I. However, hepatocellular adenoma (HCA) with risk for spontaneous hemorrhage and malignant transformation remains a long-term presentation of unknown etiology (7)(8)(9)(10)(11)(12)(13).…”

Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer

“…1,2 Over the last 25 years, dietary therapy consisting of nocturnal nasogastric infusion of glucose 3 or frequent oral administration of uncooked cornstarch 4 has significantly alleviated the metabolic abnormalities of GSD-Ia and greatly improved the prognosis. However, the underlying disease remains untreated and patients continue to suffer from hyperlipidemia, hyperuricemia, hypercalciuria, hypocitraturia, and lactic acidemia.…”

Glucose-6-phosphate transporter gene therapy corrects metabolic and myeloid abnormalities in glycogen storage disease type Ib mice

“…The current treatment for GSD-Ib consists of a dietary therapy, including continuous nasogastric infusion of glucose 7 or frequent oral administration of uncooked cornstarch, 8 to correct the loss of glucose homeostasis, augmented with granulocyte colonystimulating factor (G-CSF) therapy, 9,10 to restore myeloid functions. The combined dietary and G-CSF therapies significantly alleviate the metabolic and myeloid abnormalities of GSD-Ib patients and greatly improve their prognosis.…”