Glycogen storage disease type III medical therapy: Difference between revisions

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**Calories from [[fats]]: 25-30% (including MCT)
**Calories from [[fats]]: 25-30% (including MCT)


===Nutritional therapy===


* '''1 Asymptomatic or mild hypoglycemia'''
** '''1.1 Infants'''
**** 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 3. 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


 
* '''2 Severe hypoglycemia
*Pharmacologic medical therapy is recommended among patients with [disease subclass 1], [disease subclass 2], and [disease subclass 3].
** '''2.1 Infants or young child'''
*Pharmacologic medical therapies for [disease name] include (either) [therapy 1], [therapy 2], and/or [therapy 3].
**** Preferred treatment (1): Cornstarch (CS) 1.6 g/kg body weight PO q4h
*Empiric therapy for [disease name] depends on [disease factor 1] and [disease factor 2].
**** Preferred treatment (2): Overnight gastric feedings (OGFs)
*Patients with [disease subclass 1] are treated with [therapy 1], whereas patients with [disease subclass 2] are treated with [therapy 2].
** '''2.2 Older child'''
===Disease Name===
**** Preferred treatment (1): Cornstarch (CS) 1.7–2.5 g/kg body weight PO q6h
 
* '''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 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)
****1.1.2.2 (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)
** 1.2 '''Specific Organ system involved 2'''
*** 1.2.1 '''Adult'''
**** Preferred regimen (1): [[drug name]] 500 mg PO q8h
*** 1.2.2  '''Pediatric'''
**** Preferred regimen (1): [[drug name]] 50 mg/kg/day PO q8h (maximum, 500 mg per dose)
 
* 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
***** Preferred regimen (1): [[drug name]] 2 g IV q24h for 14 (14–21) days
***** Alternative regimen (1): [[drug name]] 2 g IV q8h for 14 (14–21) days
***** Alternative regimen (2): [[drug name]] 18–24 MU/day IV q4h for 14 (14–21) days
**** 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)
** 2.2  '<nowiki/>'''''Other Organ system involved 2''''''
**: '''Note (1):'''
**: '''Note (2)''':
**: '''Note (3):'''
*** 2.2.1 '''Adult'''
**** Parenteral regimen
***** Preferred regimen (1): [[drug name]] 2 g IV q24h for 14 (14–21) days
***** Alternative regimen (1): [[drug name]] 2 g IV q8h for 14 (14–21) days
***** Alternative regimen (2): [[drug name]] 18–24 MU/day IV q4h for 14 (14–21) days
**** 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)


==References==
==References==

Revision as of 14:58, 14 December 2017

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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]

Nutritional therapy

  • 1 Asymptomatic or mild hypoglycemia
    • 1.1 Infants
        • 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 3. 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
  • 2 Severe hypoglycemia
    • 2.1 Infants or young child
        • Preferred treatment (1): Cornstarch (CS) 1.6 g/kg body weight PO q4h
        • Preferred treatment (2): Overnight gastric feedings (OGFs)
    • 2.2 Older child
        • Preferred treatment (1): Cornstarch (CS) 1.7–2.5 g/kg body weight PO q6h

References

  1. ↑ 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.
  2. ↑ 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.
  3. ↑ Goldberg T, Slonim AE (1993). "Nutrition therapy for hepatic glycogen storage diseases". J Am Diet Assoc. 93 (12): 1423–30. PMID 8245377.

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