Duchenne muscular dystrophy medical therapy: Difference between revisions

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Revision as of 18:17, 7 May 2019

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Fahimeh Shojaei, M.D.

Overview

Pharmacologic medical therapies for Duchenne muscular dystrophy include glucocorticoids and disease-modifying treatment.

Medical Therapy

Pharmacologic medical therapies for Duchenne muscular dystrophy include glucocorticoids and disease-modifying treatment.

Duchenne muscular dystrophy

1 Glucocorticoids
- Preferred regimen : Prednisone 0.75 mg/kg PO per day

2 Disease-modifying treatment
- Preferred regimen (1): Eteplirsen 30 mg/kg IV per week (infusion over 35 to 60 minutes)
- Preferred regimen (2): Ataluren 40 mg/kg PO per day q8h (10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening, for a total daily dose of 40 mg/kg.)

References

Template:WH Template:WS

@@ Line 4: / Line 4: @@
 ==Overview==
-There is no treatment for [disease name]; the mainstay of therapy is supportive care.
+Pharmacologic medical therapies for Duchenne muscular dystrophy include glucocorticoids and disease-modifying treatment.
-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==
-*Pharmacologic medical therapy is recommended among patients with [disease subclass 1], [disease subclass 2], and [disease subclass 3].
+*Pharmacologic medical therapies for Duchenne muscular dystrophy include glucocorticoids and disease-modifying treatment.
-*Pharmacologic medical therapies for [disease name] include (either) [therapy 1], [therapy 2], and/or [therapy 3].
+===Duchenne muscular dystrophy===
-*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 Glucocorticoids'''
-** 1.1 '''Specific Organ system involved 1'''
+** Preferred regimen : Prednisone 0.75 mg/kg PO per day
-*** 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 day
-** 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 Disease-modifying treatment'''
-** 2.1 '''Specific Organ system involved 1 '''
+**Preferred regimen (1): Eteplirsen 30 mg/kg IV per week (infusion over 35 to 60 minutes)
-**: '''Note (1):'''
+**Preferred regimen (2): Ataluren 40 mg/kg PO per day q8h (10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening, for a total daily dose of 40 mg/kg.)
-**: '''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) '<nowiki/>'''''(Contraindications/specific instructions)''''''
-**
 *****