What is the use of Deflazacort (corticosteroid)?

Deflazacort: Primary Use and Clinical Applications

Deflazacort is a corticosteroid primarily indicated for treating Duchenne muscular dystrophy (DMD) in patients 5 years and older, where it slows muscle strength decline, prolongs ambulation, and reduces respiratory and cardiac complications. 1

Primary Indication: Duchenne Muscular Dystrophy

When to Initiate Treatment

• Start deflazacort at 0.9 mg/kg/day in DMD patients aged 6 years or older who are at a plateau or declining phase of disease. 2, 3
• Deflazacort should be considered as first-line therapy over prednisone when pre-existing weight gain or behavioral issues exist, as it has a more favorable side effect profile for these specific concerns. 2, 3
• Do not initiate in children under 2 years who are still improving (typical trajectory). 2
• For children aged 2-6 years at plateau or declining, monitor closely and consider early initiation. 2

Clinical Benefits in DMD

Deflazacort provides multiple disease-modifying effects:

• Slows decline in muscle strength and function, which is the only pharmacological intervention proven to alter DMD natural history. 2, 3
• Prolongs ambulation by approximately 2 years compared to untreated patients. 3
• Reduces risk of scoliosis and need for spinal surgery, particularly when continued after loss of ambulation. 2, 3
• Stabilizes pulmonary function and delays respiratory decline. 2, 3
• May slow cardiac dysfunction progression, though data on cardiac mortality reduction remain limited. 2, 3

Dosing and Administration

• Standard dose: 0.9 mg/kg/day (maximum 36 mg/day at 40 kg body weight). 2
• Continue treatment even after loss of ambulation to prevent scoliosis progression and stabilize pulmonary function. 2
• Complete all immunizations, particularly varicella, before initiating therapy. 3
• Provide families with a steroid card listing emergency-care considerations. 2, 3

Secondary Indications

Allergic Bronchopulmonary Aspergillosis/Mycosis (ABPA/ABPM)

• Dose: 0.75 mg/kg/day for 4 weeks, then decrease by half every 4 weeks for 2 months, followed by tapering by 6 mg every 2 weeks until discontinuation. 3

Autoimmune Hepatitis

• Alternative glucocorticoid for patients intolerant to standard therapy, with fewer reported steroid-related side effects. 3

Nephrotic Syndrome

• Alternative to prednisolone in pediatric patients with first episode of nephrotic syndrome. 3

Inflammatory Bowel Disease

• May be associated with lower risk of osteoporosis compared to other systemic steroids. 3

Monitoring Requirements

Essential Surveillance

• Annual ophthalmological examination to monitor for cataracts, which develop more frequently with deflazacort than prednisone. 2, 3
• Annual DEXA scans to monitor bone density despite potentially lower osteoporosis risk. 2, 3
• Annual vitamin D level monitoring (ideally late winter in seasonal climates); supplement with vitamin D3 if level is <32 nmol/L. 2
• Height monitoring at least every 6 months in pediatric patients, as growth retardation may be more pronounced with deflazacort. 3
• Blood pressure monitoring at each clinic visit as percentile for height and sex. 2
• Glucose monitoring via urine dipstick at clinic visits; if positive, obtain fasting or post-prandial blood glucose. 2

Side Effect Profile Compared to Prednisone

Deflazacort demonstrates a different chronic risk profile:

• Less weight gain and fewer behavioral side effects compared to prednisone/prednisolone. 4
• Greater risk of cataracts requiring annual ophthalmology screening. 2, 4
• Potentially greater impact on bone health and growth parameters despite lower overall osteoporosis risk. 4
• Similar efficacy to prednisone 0.75 mg/kg/day in slowing functional decline. 5, 4

Common Pitfalls to Avoid

• Do not use deflazacort for disc prolapse or low back pain—it lacks evidence for this indication and would expose patients to systemic corticosteroid risks without benefit. 6
• Do not stop deflazacort when patients lose ambulation—continued use prevents scoliosis and stabilizes pulmonary function. 2
• Do not initiate without completing immunizations—particularly varicella vaccination is critical before starting therapy. 3
• Do not ignore cataract screening—annual ophthalmology exams are mandatory as cataracts may affect vision and require switching to prednisone. 2, 3