Deflazacort

Approved Indication

• Duchenne Muscular Dystrophy (DMD):
  Indicated for the treatment of Duchenne muscular dystrophy in patients 2 years of age and older.

Other Indications (Recognized in Clinical Practice)

• Rheumatologic Disorders:
  Rheumatoid arthritis, juvenile idiopathic arthritis, ankylosing spondylitis, systemic lupus erythematosus, and various forms of vasculitis.
• Allergic and Respiratory Conditions:
  Severe bronchial asthma, perennial or refractory allergic rhinitis, atopic dermatitis, and other steroid-responsive allergic conditions.
• Gastrointestinal Diseases:
  Inflammatory bowel diseases, including Crohn’s disease and ulcerative colitis.
• Renal Disorders:
  Nephrotic syndrome (particularly steroid-responsive types).
• Hematologic and Immunologic Diseases:
  Autoimmune hemolytic anemia, idiopathic thrombocytopenic purpura, hemophagocytic syndrome, and other immune-mediated hematologic disorders.
• Dermatologic Disorders:
  Severe psoriasis, pemphigus vulgaris, bullous pemphigoid, and other severe inflammatory skin diseases requiring systemic corticosteroid therapy.
• Ophthalmic Conditions:
  Severe ocular inflammation such as uveitis and optic neuritis.
• Neurological Disorders:
  Used in certain inflammatory neurological conditions (e.g., multiple sclerosis relapses) as a corticosteroid alternative.

রেজিস্টার্ড চিকিৎসকের নির্দেশনা অনুযায়ী ঔষধ সেবন করুন।

Adults

• Initial Dose: Typically 6 mg to 90 mg orally once daily, depending on the disease severity and indication.
• Maintenance Dose: Adjust according to clinical response; use the lowest effective dose to control symptoms.
• Divided Doses: For acute or severe conditions, the total daily dose may be divided into 2–3 doses to optimize efficacy.

Children

• Recommended Dose: 0.25 – 1.5 mg/kg/day orally, individualized according to disease severity.
• Titration: Adjust dose based on clinical response and tolerability.
• Monitoring: Long-term therapy requires careful monitoring for growth suppression, bone health, and adrenal function.

Elderly

• No specific dose adjustment is generally required.
• Caution: Elderly patients are more susceptible to corticosteroid-related side effects, including osteoporosis, diabetes, hypertension, and infections, so careful monitoring is recommended.

Special Populations

• Renal Impairment: Usually no dose adjustment is needed, but monitor for fluid retention and electrolyte imbalance.
• Hepatic Impairment: Since deflazacort is metabolized in the liver, dose reduction may be necessary in moderate to severe hepatic dysfunction.
• Stress Conditions (e.g., surgery, trauma, severe infection): Temporary increase in dose may be required to meet the body’s increased corticosteroid demands.

Administration Tips:

• Take with food to minimize gastrointestinal irritation.
• Tablets should be swallowed whole with water.
• Avoid abrupt discontinuation after long-term therapy; taper gradually to prevent adrenal insufficiency.
• Alternate-day therapy may be considered in chronic use to reduce side effects.

রেজিস্টার্ড চিকিৎসকের নির্দেশনা অনুযায়ী ঔষধ সেবন করুন।

Deflazacort is a synthetic glucocorticoid prodrug that is rapidly converted by plasma esterases into its active metabolite, 21‑desacetyl deflazacort. It binds to the glucocorticoid receptor, which translocates to the nucleus and modulates gene expression—upregulating anti-inflammatory proteins and downregulating pro-inflammatory cytokines. Compared to prednisone, deflazacort has a favorable therapeutic index with reduced effects on glucose and lipid metabolism and less impact on bone mineral density and growth in children.

Absorption:

• Rapid and complete absorption after oral administration; peak active metabolite levels reached within 1–2 hours.

Distribution:

• Volume of distribution ~0.5 L/kg.
• Plasma protein binding ~40–60%.

Metabolism:

• Deflazacort is a prodrug hydrolyzed by esterases to active metabolite 21-desacetyl deflazacort; further metabolized via hepatic CYP3A4 to inactive metabolites.

Elimination:

• Half‑life of active metabolite ~1.5 to 3 hours.
• Excreted primarily via urine (approximately 60%) and feces.

• Pregnancy:
• FDA Category C. Animal studies show fetal effects at high doses; human data limited. Use only if maternal benefit outweighs potential risks. Monitor fetal growth and amniotic fluid.
• Lactation:
• Active metabolite is excreted in breast milk; caution advised. Breastfeeding may continue if low dose and infant monitored for adrenal suppression or growth impact.

• Primary Class: Glucocorticoid
• Subclass: Synthetic corticosteroid prodrug with anti‑inflammatory and immunosuppressive properties

• Known hypersensitivity to deflazacort or any excipients.
• Systemic fungal infections unless concurrently treated.
• Live or attenuated vaccines during high-dose therapy.
• Uncontrolled infections (relative contraindication).

• Adrenal Suppression: Gradually taper dose; abrupt cessation may precipitate adrenal crisis.
• Growth Suppression in Children: Monitor growth periodically; dose sparing strategies advised.
• Osteoporosis and Bone Health: Long-term use decreases bone density; monitor bone markers and supplement calcium/vitamin D; consider bisphosphonates in high-risk patients.
• Hyperglycemia and Diabetes: Monitor blood glucose; use caution in diabetics.
• Hypertension and Fluid Retention: Monitor blood pressure and electrolytes.
• Risk of Infection: Immunosuppression increases susceptibility; avoid live vaccines; monitor for opportunistic infections.
• Psychiatric Effects: Monitor for mood changes, euphoria, insomnia, or depression.
• GI Effects: Risk of peptic ulcer disease; consider gastroprotective agents in high-risk individuals.

Common:

• Weight gain (due to appetite increase and fluid retention)
• Gastrointestinal upset (dyspepsia)
• Insomnia or mood changes
• Hypertension
• Increased blood glucose
• Acne and facial rounding (“moon face”)

Less Common:

• Growth retardation in children
• Osteopenia/osteoporosis
• Gastric ulceration or GI bleeding
• Muscle weakness (myopathy)

Rare/Serious:

• Adrenal insufficiency if stopped abruptly
• Severe infections (e.g., tuberculosis, fungal sepsis)
• Psychiatric disturbances (e.g., psychotic reaction)
• Pancreatitis
• Cataracts or ocular hypertension with prolonged use

Timing and Dose‑Dependence:

• Metabolic and GI effects often manifest with moderate to high doses and over weeks to months.
• Some effects (e.g., mood, insomnia) may appear early.
• Growth and bone effects accumulate with chronic use.

• CYP3A4 Inhibitors (e.g., ketoconazole, ritonavir): May increase deflazacort levels; monitor for corticosteroid excess; consider dose reduction.
• CYP3A4 Inducers (e.g., rifampin, carbamazepine): May decrease deflazacort efficacy; may require dose increase.
• NSAIDs or Anticoagulants: Additive GI bleeding risk; monitor closely.
• Live Vaccines: Contraindicated in immunosuppressed; risk of disseminated infection.
• Diabetic Medications: May require adjustment due to hyperglycemic effects.

• DMD Clinical Guidelines (2019–2024): Deflazacort endorsed as first‑line steroid in DMD due to better growth and metabolic profile compared to prednisone/prednisolone.
• Endocrine Society: Updated recommendations emphasize bone-protective strategies and growth monitoring with pediatric steroid use.
• EMA & FDA advisories: Highlight risk of adrenal suppression and recommend standardized tapering protocols.

• Store at 20°C to 25°C (68°F to 77°F).
• Protect from moisture and light; keep in original container.
• Do not freeze.
• Handling Precautions: No special handling needed; wash hands after handling tablets if dose splitting.
• Use: Tablets should be swallowed whole; do not crush or chew.