Deflazacort

# Deflazacort ## Overview - **Classification**: Glucocorticoid, Corticosteroid - **Mechanism**: Prodrug converted to active metabolite (21-desacetyl deflazacort), which binds to glucocorticoid receptors, modulating gene expression for anti-inflammatory and immunosuppressive effects. ## Primary Indications 1. **Duchenne Muscular Dystrophy (DMD)** - To slow disease progression and preserve motor function. 2. **Other inflammatory/autoimmune conditions** - (e.g., asthma, rheumatoid arthritis), in some regions/settings. ## Adult Dosing ### Standard Dosing **Duchenne Muscular Dystrophy** - **Dose**: **0.9 mg/kg** - **Frequency**: Once daily - **Route**: Oral - **Maximum Dose**: **36 mg/day** ### Dose Adjustments - **Renal Impairment**: No specific dose adjustment generally needed. Monitor for adverse effects. - **Hepatic Impairment**: No specific dose adjustment generally needed. Use with caution; monitor closely for adverse effects. - **Elderly Patients**: Use with caution due to increased susceptibility to adverse effects. ## Pediatric Dosing ### Neonates (0-28 days) - **Dose**: Not established for DMD. - **Special Notes**: Use generally not recommended in this age group. ### Infants (1-12 months) - **Dose**: Not established for DMD. - **Special Notes**: Use generally not recommended in this age group. ### Children (1-12 years) - **Indication**: Duchenne Muscular Dystrophy (typically ≥2 years old). - **Dose**: **0.9 mg/kg** - **Frequency**: Once daily - **Route**: Oral - **Maximum**: **36 mg/day** - **Special Notes**: Monitor growth and development closely. ### Adolescents (13-18 years) - **Indication**: Duchenne Muscular Dystrophy. - **Dose**: **0.9 mg/kg** - **Frequency**: Once daily - **Route**: Oral - **Maximum**: **36 mg/day** - **Special Notes**: Follow adult dosing guidelines. ## Safety Information ### Contraindications - **Absolute**: Systemic fungal infections. - **Absolute**: Known hypersensitivity to deflazacort or its components. - **Relative**: Live or live-attenuated vaccine administration during high-dose therapy. ### Common Adverse Effects - **Very Common (>10%)**: Cushingoid appearance, weight gain, increased appetite, headache. - **Common (1-10%)**: Abdominal pain, nausea, vomiting, mood changes, hyperglycemia, acne. - **Serious but Rare**: Adrenal suppression, osteoporosis, cataracts, glaucoma, GI perforation, avascular necrosis. ### Key Drug Interactions - **CYP3A4 Inhibitors (e.g., Ketoconazole, Ritonavir)**: May increase deflazacort exposure; monitor for corticosteroid adverse effects. - **CYP3A4 Inducers (e.g., Rifampin, Phenytoin)**: May decrease deflazacort exposure; reduced efficacy possible. - **Live/Live-attenuated Vaccines**: Increased risk of severe infection; avoid concurrent use. - **Antidiabetic Agents**: May decrease effectiveness; monitor blood glucose levels closely. ## Monitoring & Follow-up - **Before Treatment**: Baseline weight, height, BP, glucose, electrolytes, bone mineral density, ophthalmologic exam. - **During Treatment**: Growth (children), weight, BP, blood glucose, electrolytes (periodically). Ophthalmologic exams (annually). Bone density (periodically). - **Clinical Signs**: Monitor for signs of infection, adrenal insufficiency (especially during withdrawal), Cushingoid features, behavioral/mood changes. ## Clinical Pearls - 💡 **Tip 1**: Administer deflazacort with food to minimize gastrointestinal upset. - 💡 **Tip 2**: Do not discontinue abruptly; taper dose slowly to prevent adrenal crisis. - 💡 **Tip 3**: Patients should carry a steroid emergency card and be aware of increased infection risk. - 💡 **Tip 4**: Consider calcium and vitamin D supplementation to support bone health, especially in children. > **⚠️ Important**: This information is for educational purposes only. Always consult current prescribing information, local guidelines, and clinical judgment before prescribing.