What is the recommended use and dosage of Deflazacort (Deflazacort) for inflammatory and autoimmune conditions?

Deflazacort Use and Dosage for Inflammatory and Autoimmune Conditions

Deflazacort is primarily indicated for Duchenne muscular dystrophy at a dose of 0.9 mg/kg/day, with limited but promising evidence for use in other inflammatory and autoimmune conditions where its improved metabolic profile may offer advantages over traditional glucocorticoids. 1, 2

FDA-Approved Indication and Dosing

• Deflazacort is FDA-approved for Duchenne muscular dystrophy (DMD) in patients 5 years and older at a recommended dose of 0.9 mg/kg/day 2
• Complete immunization schedule before initiating therapy, particularly live vaccines (4-6 weeks prior) 2
• Dose must be tapered gradually if administered for more than a few days to prevent adrenal insufficiency 2
• Dose modification required with CYP3A4 inhibitors: reduce to one-third of recommended dose 2
• Avoid use with moderate or strong CYP3A4 inducers 2

Use in Autoimmune Conditions

Autoimmune Hepatitis

• In an open-label trial, deflazacort 7.5 mg/day was effective for maintaining remission in autoimmune hepatitis patients previously stabilized on prednisolone 3
• Conversion ratio from prednisolone to deflazacort is approximately 5 mg prednisolone to 7.5 mg deflazacort 4
• May offer advantages in bone health compared to traditional steroids, though not first-line therapy 3

Rheumatoid Arthritis and Other Inflammatory Disorders

• Studies show deflazacort is as effective as prednisone or methylprednisolone in rheumatoid arthritis at doses equivalent to 83% of prednisone potency 5, 6
• Effective in juvenile chronic arthritis with potentially less impact on growth rate in children 5
• Overall incidence of adverse events (16.5%) is lower than with prednisone (20.5%) or methylprednisolone (32.7%) 5

Systemic Autoimmune Rheumatic Disease with ILD

• Not specifically mentioned in guidelines for interstitial lung disease in systemic autoimmune rheumatic diseases, where mycophenolate, rituximab, cyclophosphamide, and azathioprine are conditionally recommended as first-line options 3
• Traditional glucocorticoids are conditionally recommended for non-SSc-ILD but should be used at the lowest effective dose (ideally <15 mg/day) 3

Advantages of Deflazacort

• Less negative impact on calcium metabolism compared to prednisone, potentially reducing osteoporosis risk 5, 6
• Less effect on growth rate in children requiring corticosteroid therapy 5, 7
• Fewer diabetogenic effects at moderate doses in short-term studies 5
• May have fewer weight and behavioral side effects compared to prednisone 1

Side Effects and Monitoring

• Monitor for Cushing's syndrome, hyperglycemia, and adrenal insufficiency 2
• Annual ophthalmological examination for cataracts; consider switching to prednisone if cataracts develop 3
• Monitor bone density with DEXA scans and vitamin D levels 3
• Watch for hypertension, glucose intolerance, and gastroesophageal reflux disease 3
• May have important immunosuppressive activity potentially increasing risk of opportunistic infections 8

Practical Considerations

• Can be taken with or without food; do not administer with grapefruit juice 2
• Tablets can be administered whole or crushed and mixed with applesauce 2
• Available in 6 mg, 18 mg, 30 mg, and 36 mg tablet strengths 2
• Consider deflazacort as first-line when pre-existing weight or behavioral concerns exist 1
• Maximum weight-based dose corresponds to 36 mg/day for a 40 kg patient 3

Caution and Contraindications

• Contraindicated in patients with known hypersensitivity to deflazacort or inactive ingredients 2
• Limited data on long-term safety profile compared to other glucocorticoids 7
• Higher cost may limit widespread use despite potential metabolic advantages 7
• Requires careful monitoring for side effects, especially within initial 6 months of treatment 3