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| __NOTOC__ | | __NOTOC__ |
| {{Glycogen storage disease type II}} | | {{Glycogen storage disease type II}} |
| {{CMG}}; {{AE}} | | {{CMG}}; {{AE}} {{Anmol}} |
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| ==Overview== | | ==Overview== |
| There is no treatment for [disease name]; the mainstay of therapy is supportive care.
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| Supportive therapy for [disease name] includes [therapy 1], [therapy 2], and [therapy 3].
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| The majority of cases of [disease name] are self-limited and require only supportive care.
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| [Disease name] is a medical emergency and requires prompt treatment.
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| The mainstay of treatment for [disease name] is [therapy].
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| The optimal therapy for [malignancy name] depends on the stage at diagnosis.
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| [Therapy] is recommended among all patients who develop [disease name].
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| Pharmacologic medical therapy is recommended among patients with [disease subclass 1], [disease subclass 2], and [disease subclass 3].
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| Pharmacologic medical therapies for [disease name] include (either) [therapy 1], [therapy 2], and/or [therapy 3].
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| Empiric therapy for [disease name] depends on [disease factor 1] and [disease factor 2].
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| Patients with [disease subclass 1] are treated with [therapy 1], whereas patients with [disease subclass 2] are treated with [therapy 2].
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| | Pharmacologic medical therapy is recommended among patients with infantile onset glycogen storage type 2 (GSD type 2). Pharmacologic medical therapies for GSD type 2 include [[enzyme replacement therapy]] (ERT) with recombinant human [[acid alpha-glucosidase]]. |
| ==Medical Therapy== | | ==Medical Therapy== |
| *Pharmacologic medical therapy is recommended among patients with [disease subclass 1], [disease subclass 2], and [disease subclass 3]. | | *Pharmacologic medical therapy is recommended among patients with infantile onset glycogen storage type 2 (GSD type 2). |
| *Pharmacologic medical therapies for [disease name] include (either) [therapy 1], [therapy 2], and/or [therapy 3]. | | *Pharmacologic medical therapies for GSD type 2 include [[enzyme replacement therapy]] (ERT) with recombinant human [[acid alpha-glucosidase]].<ref name="pmid17151339">{{cite journal| author=Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL et al.| title=Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. | journal=Neurology | year= 2007 | volume= 68 | issue= 2 | pages= 99-109 | pmid=17151339 | doi=10.1212/01.wnl.0000251268.41188.04 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=17151339 }}</ref> |
| *Empiric therapy for [disease name] depends on [disease factor 1] and [disease factor 2].
| | *In 2006, ERT was approved by [[US Food and Drug Administration]] ([[FDA]]) for treatment of infantile onset GSD type 2.<ref name="urlDrugs@FDA: FDA Approved Drug Products">{{cite web |url=https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=BasicSearch.process |title=Drugs@FDA: FDA Approved Drug Products |format= |work= |accessdate=}}</ref> |
| *Patients with [disease subclass 1] are treated with [therapy 1], whereas patients with [disease subclass 2] are treated with [therapy 2].
| | ===Glycogen storage disease type 2=== |
| ===Disease Name===
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| * '''1 Stage 1 - Name of stage'''
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| ** 1.1 '''Specific Organ system involved 1'''
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| *** 1.1.1 '''Adult'''
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| **** Preferred regimen (1): [[drug name]] 100 mg PO q12h for 10-21 days '''(Contraindications/specific instructions)'''
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| **** Preferred regimen (2): [[drug name]] 500 mg PO q8h for 14-21 days
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| **** Preferred regimen (3): [[drug name]] 500 mg q12h for 14-21 days
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| **** Alternative regimen (1): [[drug name]] 500 mg PO q6h for 7–10 days
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| **** Alternative regimen (2): [[drug name]] 500 mg PO q12h for 14–21 days
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| **** Alternative regimen (3): [[drug name]] 500 mg PO q6h for 14–21 days
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| *** 1.1.2 '''Pediatric'''
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| **** 1.1.2.1 (Specific population e.g. '''children < 8 years of age''')
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| ***** Preferred regimen (1): [[drug name]] 50 mg/kg PO per day q8h (maximum, 500 mg per dose)
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| ***** Preferred regimen (2): [[drug name]] 30 mg/kg PO per day in 2 divided doses (maximum, 500 mg per dose)
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| ***** Alternative regimen (1): [[drug name]]10 mg/kg PO q6h (maximum, 500 mg per day)
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| ***** Alternative regimen (2): [[drug name]] 7.5 mg/kg PO q12h (maximum, 500 mg per dose)
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| ***** Alternative regimen (3): [[drug name]] 12.5 mg/kg PO q6h (maximum, 500 mg per dose)
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| ****1.1.2.2 (Specific population e.g. ''''''children < 8 years of age'''''')
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| ***** Preferred regimen (1): [[drug name]] 4 mg/kg/day PO q12h(maximum, 100 mg per dose)
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| ***** Alternative regimen (1): [[drug name]] 10 mg/kg PO q6h (maximum, 500 mg per day)
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| ***** Alternative regimen (2): [[drug name]] 7.5 mg/kg PO q12h (maximum, 500 mg per dose)
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| ***** Alternative regimen (3): [[drug name]] 12.5 mg/kg PO q6h (maximum, 500 mg per dose)
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| ** 1.2 '''Specific Organ system involved 2'''
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| *** 1.2.1 '''Adult'''
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| **** Preferred regimen (1): [[drug name]] 500 mg PO q8h
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| *** 1.2.2 '''Pediatric'''
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| **** Preferred regimen (1): [[drug name]] 50 mg/kg/day PO q8h (maximum, 500 mg per dose)
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| * 2 '''Stage 2 - Name of stage'''
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| ** 2.1 '''Specific Organ system involved 1 '''
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| **: '''Note (1):'''
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| **: '''Note (2)''':
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| **: '''Note (3):'''
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| *** 2.1.1 '''Adult'''
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| **** Parenteral regimen
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| ***** Preferred regimen (1): [[drug name]] 2 g IV q24h for 14 (14–21) days
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| ***** Alternative regimen (1): [[drug name]] 2 g IV q8h for 14 (14–21) days
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| ***** Alternative regimen (2): [[drug name]] 18–24 MU/day IV q4h for 14 (14–21) days
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| **** Oral regimen
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| ***** Preferred regimen (1): [[drug name]] 500 mg PO q8h for 14 (14–21) days
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| ***** Preferred regimen (2): [[drug name]] 100 mg PO q12h for 14 (14–21) days
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| ***** Preferred regimen (3): [[drug name]] 500 mg PO q12h for 14 (14–21) days
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| ***** Alternative regimen (1): [[drug name]] 500 mg PO q6h for 7–10 days
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| ***** Alternative regimen (2): [[drug name]] 500 mg PO q12h for 14–21 days
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| ***** Alternative regimen (3):[[drug name]] 500 mg PO q6h for 14–21 days
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| *** 2.1.2 '''Pediatric'''
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| **** Parenteral regimen
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| ***** Preferred regimen (1): [[drug name]] 50–75 mg/kg IV q24h for 14 (14–21) days (maximum, 2 g)
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| ***** Alternative regimen (1): [[drug name]] 150–200 mg/kg/day IV q6–8h for 14 (14–21) days (maximum, 6 g per day)
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| ***** 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)''''''
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| **** Oral regimen
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| ***** Preferred regimen (1): [[drug name]] 50 mg/kg/day PO q8h for 14 (14–21) days (maximum, 500 mg per dose)
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| ***** 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)
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| ***** Preferred regimen (3): [[drug name]] 30 mg/kg/day PO q12h for 14 (14–21) days (maximum, 500 mg per dose)
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| ***** Alternative regimen (1): [[drug name]] 10 mg/kg PO q6h 7–10 days (maximum, 500 mg per day)
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| ***** Alternative regimen (2): [[drug name]] 7.5 mg/kg PO q12h for 14–21 days (maximum, 500 mg per dose)
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| ***** Alternative regimen (3): [[drug name]] 12.5 mg/kg PO q6h for 14–21 days (maximum,500 mg per dose)
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| ** 2.2 '<nowiki/>'''''Other Organ system involved 2''''''
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| **: '''Note (1):''' | |
| **: '''Note (2)''':
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| **: '''Note (3):'''
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| *** 2.2.1 '''Adult'''
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| **** Parenteral regimen
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| ***** Preferred regimen (1): [[drug name]] 2 g IV q24h for 14 (14–21) days
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| ***** Alternative regimen (1): [[drug name]] 2 g IV q8h for 14 (14–21) days
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| ***** Alternative regimen (2): [[drug name]] 18–24 MU/day IV q4h for 14 (14–21) days
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| **** Oral regimen
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| ***** Preferred regimen (1): [[drug name]] 500 mg PO q8h for 14 (14–21) days
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| ***** Preferred regimen (2): [[drug name]] 100 mg PO q12h for 14 (14–21) days
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| ***** Preferred regimen (3): [[drug name]] 500 mg PO q12h for 14 (14–21) days
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| ***** Alternative regimen (1): [[drug name]] 500 mg PO q6h for 7–10 days
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| ***** Alternative regimen (2): [[drug name]] 500 mg PO q12h for 14–21 days
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| ***** Alternative regimen (3):[[drug name]] 500 mg PO q6h for 14–21 days
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| *** 2.2.2 '''Pediatric'''
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| **** Parenteral regimen
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| ***** Preferred regimen (1): [[drug name]] 50–75 mg/kg IV q24h for 14 (14–21) days (maximum, 2 g)
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| ***** Alternative regimen (1): [[drug name]] 150–200 mg/kg/day IV q6–8h for 14 (14–21) days (maximum, 6 g per day)
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| ***** 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)
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| **** Oral regimen
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| ***** Preferred regimen (1): [[drug name]] 50 mg/kg/day PO q8h for 14 (14–21) days (maximum, 500 mg per dose)
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| ***** Preferred regimen (2): [[drug name]] 4 mg/kg/day PO q12h for 14 (14–21) days (maximum, 100 mg per dose)
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| ***** Preferred regimen (3): [[drug name]] 30 mg/kg/day PO q12h for 14 (14–21) days (maximum, 500 mg per dose)
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| ***** Alternative regimen (1): [[drug name]] 10 mg/kg PO q6h 7–10 days (maximum, 500 mg per day)
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| ***** Alternative regimen (2): [[drug name]] 7.5 mg/kg PO q12h for 14–21 days (maximum, 500 mg per dose)
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| ***** Alternative regimen (3): [[drug name]] 12.5 mg/kg PO q6h for 14–21 days (maximum,500 mg per dose)
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| | * '''1 Infantile onset GSD type 2''' |
| | ** '''1.1 Enzyme replacement therapy''' |
| | *** Preferred regimen (1): recombinant human [[acid alpha-glucosidase]] 20 mg/Kg IV every two week |
| | **: '''NOTE (1):''' If there is poor response to initial therapy, the dose can be increased to 20 mg/kg every week. |
| ==References== | | ==References== |
| {{reflist|2}} | | {{reflist|2}} |
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| [[Category:Endocrinology]] | | [[Category:Endocrinology]] |
| [[Category:Hepatology]] | | [[Category:Hepatology]] |
| | [[Category:Gastroenterology]] |
| | [[Category:Pediatrics]] |
| | [[Category:Up-To-Date]] |
| | [[Category:Genetic disorders]] |
| | [[Category:Metabolic disorders]] |
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| {{WS}} | | {{WS}} |
| {{WH}} | | {{WH}} |