Drug updated on 4/29/2024
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
- • Indicated to treat Duchenne muscular dystrophy in patients ≥ 2 years of age.
Summary
- 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.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Emflaza (deflazacort) Prescribing Information. | 2021 | PTC Therapeutics, Inc., South Plainfield, NJ |
Systematic Reviews / Meta-Analyses
Document Title | Year | Source |
---|---|---|
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy. | 2021 | Journal of Comparative Effectiveness Research |
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: a meta-analysis of disease progression rates in recent multicenter clinical trials. | 2020 | Muscle and Nerve |
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Clinical practice guidelines for the diagnosis and management of Duchenne muscular dystrophy: a scoping review. | 2024 | Frontiers in Medicine |
Management of adrenal insufficiency risk after long-term systemic glucocorticoid therapy in Duchenne muscular dystrophy: Clinical Practice Recommendations. | 2019 | Journal of Neuromuscular Diseases |