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:
Overview
There is no treatment for [disease name]; the mainstay of therapy is supportive care.
OR
Supportive therapy for [disease name] includes [therapy 1], [therapy 2], and [therapy 3].
OR
The majority of cases of [disease name] are self-limited and require only supportive care.
OR
[Disease name] is a medical emergency and requires prompt treatment.
OR
The mainstay of treatment for [disease name] is [therapy].
OR The optimal therapy for [malignancy name] depends on the stage at diagnosis.
OR
[Therapy] is recommended among all patients who develop [disease name].
OR
Pharmacologic medical therapy is recommended among patients with [disease subclass 1], [disease subclass 2], and [disease subclass 3].
OR
Pharmacologic medical therapies for [disease name] include (either) [therapy 1], [therapy 2], and/or [therapy 3].
OR
Empiric therapy for [disease name] depends on [disease factor 1] and [disease factor 2].
OR
Patients with [disease subclass 1] are treated with [therapy 1], whereas patients with [disease subclass 2] are treated with [therapy 2].
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.[1]
- A high protein diet is recommended, especially for children with growth failure and myopathy due to reasons including:[2]
- Protein provides alanine which may be used in gluconeogenesis as a substitute source of glucose during fast.
- 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).
- A metabolic dietician should be consulted once a case of GSD type 3 is diagnosed.
- Distribution of calories:[3]
- Calories from carbohydrate: 45-55%
- Calories from protein: 25%
- Calories from fats: 25-30% (including MCT)
- Pharmacologic medical therapy is recommended among patients with [disease subclass 1], [disease subclass 2], and [disease subclass 3].
- Pharmacologic medical therapies for [disease name] include (either) [therapy 1], [therapy 2], and/or [therapy 3].
- Empiric therapy for [disease name] depends on [disease factor 1] and [disease factor 2].
- Patients with [disease subclass 1] are treated with [therapy 1], whereas patients with [disease subclass 2] are treated with [therapy 2].
Disease Name
- 1 Stage 1 - Name of stage
- 1.1 Specific Organ system involved 1
- 1.1.1 Adult
- Preferred regimen (1): drug name 100 mg PO q12h for 10-21 days (Contraindications/specific instructions)
- Preferred regimen (2): drug name 500 mg PO q8h for 14-21 days
- Preferred regimen (3): drug name 500 mg q12h for 14-21 days
- Alternative regimen (1): drug name 500 mg PO q6h for 7–10 days
- Alternative regimen (2): drug name 500 mg PO q12h for 14–21 days
- Alternative regimen (3): drug name 500 mg PO q6h for 14–21 days
- 1.1.2 Pediatric
- 1.1.2.1 (Specific population e.g. children < 8 years of age)
- Preferred regimen (1): drug name 50 mg/kg PO per day q8h (maximum, 500 mg per dose)
- Preferred regimen (2): drug name 30 mg/kg PO per day in 2 divided doses (maximum, 500 mg per dose)
- Alternative regimen (1): drug name10 mg/kg PO q6h (maximum, 500 mg per day)
- Alternative regimen (2): drug name 7.5 mg/kg PO q12h (maximum, 500 mg per dose)
- Alternative regimen (3): drug name 12.5 mg/kg PO q6h (maximum, 500 mg per dose)
- 1.1.2.2 (Specific population e.g. 'children < 8 years of age')
- Preferred regimen (1): drug name 4 mg/kg/day PO q12h(maximum, 100 mg per dose)
- Alternative regimen (1): drug name 10 mg/kg PO q6h (maximum, 500 mg per day)
- Alternative regimen (2): drug name 7.5 mg/kg PO q12h (maximum, 500 mg per dose)
- Alternative regimen (3): drug name 12.5 mg/kg PO q6h (maximum, 500 mg per dose)
- 1.1.2.1 (Specific population e.g. children < 8 years of age)
- 1.1.1 Adult
- 1.2 Specific Organ system involved 2
- 1.1 Specific Organ system involved 1
- 2 Stage 2 - Name of stage
- 2.1 Specific Organ system involved 1
- Note (1):
- Note (2):
- Note (3):
- 2.1.1 Adult
- Parenteral regimen
- Oral regimen
- Preferred regimen (1): drug name 500 mg PO q8h for 14 (14–21) days
- Preferred regimen (2): drug name 100 mg PO q12h for 14 (14–21) days
- Preferred regimen (3): drug name 500 mg PO q12h for 14 (14–21) days
- Alternative regimen (1): drug name 500 mg PO q6h for 7–10 days
- Alternative regimen (2): drug name 500 mg PO q12h for 14–21 days
- Alternative regimen (3):drug name 500 mg PO q6h for 14–21 days
- 2.1.2 Pediatric
- Parenteral regimen
- Preferred regimen (1): drug name 50–75 mg/kg IV q24h for 14 (14–21) days (maximum, 2 g)
- Alternative regimen (1): drug name 150–200 mg/kg/day IV q6–8h for 14 (14–21) days (maximum, 6 g per day)
- Alternative regimen (2): drug name 200,000–400,000 U/kg/day IV q4h for 14 (14–21) days (maximum, 18–24 million U per day) '(Contraindications/specific instructions)'
- Oral regimen
- Preferred regimen (1): drug name 50 mg/kg/day PO q8h for 14 (14–21) days (maximum, 500 mg per dose)
- Preferred regimen (2): drug name (for children aged ≥ 8 years) 4 mg/kg/day PO q12h for 14 (14–21) days (maximum, 100 mg per dose)
- Preferred regimen (3): drug name 30 mg/kg/day PO q12h for 14 (14–21) days (maximum, 500 mg per dose)
- Alternative regimen (1): drug name 10 mg/kg PO q6h 7–10 days (maximum, 500 mg per day)
- Alternative regimen (2): drug name 7.5 mg/kg PO q12h for 14–21 days (maximum, 500 mg per dose)
- Alternative regimen (3): drug name 12.5 mg/kg PO q6h for 14–21 days (maximum,500 mg per dose)
- Parenteral regimen
- 2.2 'Other Organ system involved 2'
- Note (1):
- Note (2):
- Note (3):
- 2.2.1 Adult
- Parenteral regimen
- Oral regimen
- Preferred regimen (1): drug name 500 mg PO q8h for 14 (14–21) days
- Preferred regimen (2): drug name 100 mg PO q12h for 14 (14–21) days
- Preferred regimen (3): drug name 500 mg PO q12h for 14 (14–21) days
- Alternative regimen (1): drug name 500 mg PO q6h for 7–10 days
- Alternative regimen (2): drug name 500 mg PO q12h for 14–21 days
- Alternative regimen (3):drug name 500 mg PO q6h for 14–21 days
- 2.2.2 Pediatric
- Parenteral regimen
- Preferred regimen (1): drug name 50–75 mg/kg IV q24h for 14 (14–21) days (maximum, 2 g)
- Alternative regimen (1): drug name 150–200 mg/kg/day IV q6–8h for 14 (14–21) days (maximum, 6 g per day)
- Alternative regimen (2): drug name 200,000–400,000 U/kg/day IV q4h for 14 (14–21) days (maximum, 18–24 million U per day)
- Oral regimen
- Preferred regimen (1): drug name 50 mg/kg/day PO q8h for 14 (14–21) days (maximum, 500 mg per dose)
- Preferred regimen (2): drug name 4 mg/kg/day PO q12h for 14 (14–21) days (maximum, 100 mg per dose)
- Preferred regimen (3): drug name 30 mg/kg/day PO q12h for 14 (14–21) days (maximum, 500 mg per dose)
- Alternative regimen (1): drug name 10 mg/kg PO q6h 7–10 days (maximum, 500 mg per day)
- Alternative regimen (2): drug name 7.5 mg/kg PO q12h for 14–21 days (maximum, 500 mg per dose)
- Alternative regimen (3): drug name 12.5 mg/kg PO q6h for 14–21 days (maximum,500 mg per dose)
- Parenteral regimen
- 2.1 Specific Organ system involved 1
References
- ↑ Fernandes J, Leonard JV, Moses SW, Odièvre M, di Rocco M, Schaub J; et al. (1988). "Glycogen storage disease: recommendations for treatment". Eur J Pediatr. 147 (3): 226–8. PMID 3292244.
- ↑ Slonim AE, Coleman RA, Moses WS (1984). "Myopathy and growth failure in debrancher enzyme deficiency: improvement with high-protein nocturnal enteral therapy". J Pediatr. 105 (6): 906–11. PMID 6438290.
- ↑ Goldberg T, Slonim AE (1993). "Nutrition therapy for hepatic glycogen storage diseases". J Am Diet Assoc. 93 (12): 1423–30. PMID 8245377.