Glycogen storage disease type III medical therapy

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Anmol Pitliya, M.B.B.S. M.D.[2]

Overview

The medical therapy of glycogen storage disease type 3 is directed towards management of hypoglycemia. However, hypoglycemic episodes are less marked in glycogen storage disease type 3 than glycogen storage disease type 1. A high protein diet is recommended, especially for children with growth failure and myopathy. A metabolic dietician should be consulted once a case of GSD type 3 is diagnosed. Preferred treatment is use of cornstarch. Careful monitoring of blood glucose is needed during illness, if diet or schedule is changed, start of exercise routine, and randomly to detect asymptomatic hypoglycemia.

Medical Therapy

• The primary concern in infants and young children with GSD type 3 is hypoglycemia.
• However hypoglycemic episodes are less marked in glycogen storage disease type 3 than glycogen storage disease type 1.
• A metabolic dietician should be consulted once a case of GSD type 3 is diagnosed.
• A high protein diet is recommended, especially for children with growth failure and myopathy due to reasons including:
  • Protein provides alanine which may be used in gluconeogenesis as a substitute source of glucose during fasting.
  • High dietary protein increase synthesis of proteins in muscles and improves muscle function.
  • Replacement of carbohydrates with protein reduces storage of glycogen.
• A low fat diet is recommended due to reasons including:
  • High protein and high carbohydrates necessitate reduction of fats.
  • Young age group individuals may be inefficient in oxidizing long chain fatty acids.
  • Moderate amount of medium chain triglycerides (MCT) are preferred as they are ketogenic and may provide additional energy source for brain.
• It is not needed to avoid sucrose (fructose and glucose) and lactose (galactose and glucose).
• Distribution of calories:
  • Calories from carbohydrate: 35-55%
  • Calories from protein: 20-30%
  • Calories from fats: 20-35 (including MCT)

Nutritional therapy

• 1 Infants or young child
  • 1.1 Asymptomatic or mild hypoglycemia

    • Preferred treatment (1): Cornstarch (CS) 1 g/kg body weight PO q4h

    NOTE (1): Digestive enzymes such as amylase are not fully functional during infancy. So, CS may not be well tolerated leading to gas, diarrhea, and bloating. Addition of pancrelipase with CS may help with digestion and reduces side effects.

    NOTE (2): Pancrealipase consists of three enzymes including lipase, protease, and amylase.

    NOTE (3): Typically, CS requirement of glycogen storage disease type 3 is less than glycogen storage disease type 1. So, starting CS at a lower dose and increasing the dose as per requirement is recommended.

    NOTE (4): During the initiation of treatment, blood glucose should be monitored every hour to establish the adequacy of CS dosage. As the dosage of CS is fixed, the frequency of blood glucose monitoring may be decreased thereafter.

    NOTE (5): Careful monitoring of blood glucose is needed during:

    • Illness
    • Diet or schedule is changed
    • During start of exercise routine
    • Randomly to detect asymptomatic hypoglycemia

  • 1.2 Severe hypoglycemia

    • Preferred treatment (1): Cornstarch (CS) 1.6 g/kg body weight PO q4h
    • Preferred treatment (2): Overnight gastric feedings (OGFs)

• 2 Older child
  • 2.1 Asymptomatic or mild hypoglycemia

    • Preferred treatment (1): Cornstarch (CS) 1 g/kg body weight PO q4h

  • 2.2 Severe hypoglycemia

    • Preferred treatment (1): Cornstarch (CS) 1.7–2.5 g/kg body weight PO q6h

• 3 Adults
  • 3.1 Glycogen storage disease type 3a

    • Increase protein in diet

  • 3.2 Glycogen storage disease type 3b

    • No specific treatment is needed

Contraindications

Medications and supplements contraindicated in glycogen storage disease type 3 include:

• 1 Absolute contraindication
  • High sugar intake
  • Steroid-based drugs
  • Growth hormone replacement therapy
• 2 Relative contraindication
  • Hormonal contraceptives in women
  • Statins for control of hyperlipidemia
  • Beta blockers