What are the indications for Daflazacort (deflazacort)?

Indications of Deflazacort

Deflazacort is primarily indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 6 years and older, and as an alternative glucocorticoid for various inflammatory and autoimmune conditions when prednisone side effects are concerning. 1

Primary Indication

• Duchenne Muscular Dystrophy (DMD): Recommended for patients aged 6 years and older who are at a plateau or declining phase of the disease, with a standard dosing of 0.9 mg/kg/day 1

• Deflazacort should be considered as first-line therapy in DMD patients with pre-existing weight or behavioral issues, as it has a more favorable side effect profile compared to prednisone 1

Secondary Indications

• Allergic Bronchopulmonary Aspergillosis/Mycosis (ABPA/ABPM): Used at a dose of 0.75 mg/kg/day for 4 weeks, then decreased by half every 4 weeks for 2 months, followed by tapering by 6 mg every 2 weeks until discontinuation 1

• Autoimmune Hepatitis: Used as an alternative glucocorticoid in patients who are intolerant to standard therapy, with fewer reported steroid-related side effects 1

• Nephrotic Syndrome in Children: Alternative to prednisolone in pediatric patients with first episode of nephrotic syndrome 1

• Inflammatory Bowel Disease: May be associated with a lower risk of osteoporosis compared to other systemic steroids 1

Comparative Advantages

• Reduced Side Effect Profile: Deflazacort appears to have:

  • Less impact on weight gain compared to prednisone 1, 2
  • Less negative impact on growth rate in children requiring corticosteroid therapy 2
  • Less effect on parameters associated with corticosteroid-induced osteoporosis 2, 3
  • Fewer diabetogenic effects at moderate dosages 2

• Immunomodulatory Effects: More efficient than prednisone in inducing immunomodulatory effects in rheumatoid arthritis patients 4

Dosing Considerations

• DMD: 0.9 mg/kg/day (maximum 36 mg/day when weight reaches 40 kg) 1

• ABPA/ABPM: 0.75 mg/kg/day for 4 weeks, then tapered over 4 months 1

• Equivalent Potency: Deflazacort has approximately 83% of the therapeutic potency of prednisone, requiring slightly higher doses for equivalent effect 3

Monitoring and Precautions

• Annual ophthalmological examination: Deflazacort use requires monitoring for cataracts, which may develop more frequently than with prednisone 1

• Bone health monitoring: Annual DEXA scans and vitamin D level monitoring recommended despite potentially lower risk of osteoporosis 1

• Growth monitoring: Height should be monitored at least every 6 months, especially in pediatric patients 1

• Immunization status: Ensure complete immunization schedule before initiating therapy, particularly varicella immunization 1

Clinical Decision Algorithm

1. For DMD patients:

   • If patient is ≥6 years old and at plateau or declining phase → Initiate deflazacort 0.9 mg/kg/day 1
   • If pre-existing weight or behavioral concerns exist → Choose deflazacort over prednisone 1
   • If cataracts develop that affect vision → Consider switching from deflazacort to prednisone 1

2. For inflammatory conditions:

   • If patient has risk factors for steroid-induced osteoporosis → Consider deflazacort over prednisone 1, 2
   • If patient has diabetes or risk factors for diabetes → Consider deflazacort for potentially fewer diabetogenic effects 2
   • If patient is a child requiring long-term steroid therapy → Consider deflazacort to minimize growth impairment 2

Important Caveats

• Despite potentially fewer metabolic side effects, deflazacort still requires careful monitoring for all typical glucocorticoid adverse effects 1, 2

• The FDA-approved indication in the US is specifically for DMD, though it is used off-label for other inflammatory conditions based on clinical evidence 5, 2

• While deflazacort may have advantages over prednisone for certain side effects, it is not completely devoid of glucocorticoid-related adverse effects and requires appropriate monitoring 1, 2