Drugdocs | Deflazacort

Drug updated on 3/28/2024

Dosage Form	Tablet (oral: 6 mg, 18 mg, 30 mg, 36 mg); Suspension (oral: 22.75 mg/ml)
Drug Class	Corticosteroids
Ongoing and Completed Studies	ClinicalTrials.gov

Indication

Deflazacort (Emflaza) is used to treat Duchenne muscular dystrophy in patients aged 5 years and above.
A total of one systematic review/meta-analysis document was reviewed, which included 12 studies involving 667 participants with definite DMD diagnosis.
The treatment involves corticosteroids such as deflazacort given for a minimum of three months, however the optimal timing for starting this treatment remains unclear.
Moderate quality evidence from randomized controlled trials indicates that corticosteroid therapy improves muscle strength and function in the short term (twelve months), and strength up to two years; there's no clear evidence on prolongation of walking ability based solely on these trials.
Adverse effects like excessive weight gain, behavioural abnormalities, cushingoid appearance, and excessive hair growth were more common with corticosteroids than placebo; very low quality evidence suggests less weight gain with deflazacort compared to prednisone after a year's treatment.
Non-randomised studies suggest sustained benefit from corticosteroids for up to 66 months but also identify clinically significant adverse effects of long-term treatment; daily regimen appears more effective than intermittent regimens over longer periods but has greater frequency of side effects according to these non-randomized studies.

Document Title	Year	Source
Emflaza (deflazacort) Prescribing Information.	2021	PTC Therapeutics, Inc., South Plainfield, NJ

Document Title	Year	Source
Deflazacort, eteplirsen, and golodirsen for Duchenne muscular dystrophy: effectiveness and value.	2019	ICER
Corticosteroids for the treatment of Duchenne muscular dystrophy	2016	The Cochrane Database of Systematic Reviews