Trade Name(s)

  • Emflaza

Ther. Class.
none assigned

Pharm. Class.


Duchenne muscular dystrophy (DMD).


Exerts anti-inflammatory and immunosuppressive effects. Mechanism for therapeutic effect in DMD not fully known.

Therapeutic Effect(s):

Improved muscle strength.


Absorption: Rapidly absorbed.

Distribution: Unknown.

Metabolism and Excretion: Parent drug (prodrug) is rapidly metabolized by esterases to active metabolite (21-desDFZ), which is then further metabolized by CYP3A4 to inactive metabolites. Primarily excreted in urine.

Half-life: Unknown.

TIME/ACTION PROFILE (plasma concentrations)

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Contraindicated in:

  • Hypersensitivity;
  • Active untreated infections.

Use Cautiously in:

  • Hypopituitarism, primary adrenal insufficiency, congenital adrenal hyperplasia, thyroid dysfunction, or pheochromocytoma (↑ risk of endocrine abnormalities);
  • HF, hypertension, recent MI, or renal impairment;
  • Peptic ulcer disease, diverticulitis, recent intestinal anastomoses, or ulcerative colitis (↑ risk of GI perforation);
  • Myasthenia gravis (↑ risk of myopathy);
  • OB:  Use only if potential maternal benefit outweighs potential fetal risk;
  • Lactation: Use only if potential maternal benefit outweighs potential risks to child;
  • Pedi:  Children <5 yr (safety and effectiveness not established); oral suspension contains benzyl alcohol which can cause gasping syndrome in premature neonates and low birth weight infants.

Adverse Reactions/Side Effects

CNS: headache, depression, euphoria, insomnia, irritability, mood swings

CV: THROMBOEMBOLIC EVENTS, hypertension, peripheral edema

Derm: TOXIC EPIDERMAL NECROLYSIS, acne, hirsutism, erythema, rash, skin striae

EENT: cataracts, epistaxis, glaucoma, rhinorrhea

Endo: Cushingoid appearance, adrenal suppression, ↓ bone density, ↓ growth (children), hyperglycemia

GI: PEPTIC ULCERATION, abdominal pain, constipation, ↑ appetite, nausea

Metabolic: ↑ weight

MS: avascular necrosis of joints, back pain, myopathy

Resp: cough


* CAPITALS indicate life-threatening.
Underline indicate most frequent.



  • May ↑ requirement for  insulins  or oral hypoglycemic agents.
  • May ↓ antibody response to and ↑ the risk of adverse reactions from  live virus vaccines.
  • Concurrent use with  neuromuscular blocking agents  may ↑ risk of acute myopathy.
  •  Moderate CYP3A4 inhibitors  and strong CYP3A4 inhibitors, including clarithromycin, fluconazole, diltiazem, or  verapamil  may ↑ levels and risk of toxicity; ↓ deflazacort dose.
  •  Moderate CYP3A4 inducers  and strong CYP3A4 inducers, including rifampin, efavirenz, carbamazepine, or  phenytoin  may ↓ levels and therapeutic effect; avoid concurrent use.


Grapefruit juice may ↑ levels and risk of toxicity; ↓ deflazacort dose.


PO (Adults and Children ≥5 yr): 0.9 mg/kg once daily.  Concurrent use of moderate or strong CYP3A4 inhibitors– Give 1/3 the recommended dose.


Oral suspension: 22.75 mg/mL

Tablets: 6 mg, 18 mg, 30 mg, 36 mg


  • Monitor for signs and symptoms of corticosteroid withdrawal syndrome (anorexia, nausea, vomiting, lethargy, headache, fever, joint pain, desquamation, myalgia, weight loss) following therapy. May occur months after discontinuation of therapy. May need to reinstate corticosteroids.
  • Monitor for signs and symptoms of Cushing's syndrome (hypertension, truncal obesity and thinning of the limbs, purple striae, facial rounding, facial plethora, muscle weakness, easy and frequent bruising with thin fragile skin, posterior neck fat deposition, osteopenia, acne, amenorrhea, hirsutism, psychiatric abnormalities. May occur with prolonged use of corticosteroids.
  • Monitor for signs and symptoms of infection (fever, chills, sore throat, painful urination) during therapy. May require dose reduction or discontinuation of deflazacort.  If patient is exposed to chickenpox,  consider prophylaxis with varicella zoster immune globulin (VZIG).  If exposed to measles,  prophylaxis with immunoglobulin (IG) may be indicated.  If chickenpox develops,  consider treatment with antiviral agents.  If fungal infections occur,  may require dose reduction or discontinuation of therapy.  May activate latent amebiasis;  rule out amebiasis in patient with unexplained diarrhea or who has spent time in the tropics before starting therapy.  If Strongyloides (threadworm) infestation occurs,  reduce dose or discontinue therapy.
  • Monitor BP and assess for signs and symptoms of fluid overload (dyspnea, edema) periodically during therapy. Treat symptoms as needed.
  • Monitor for signs and symptoms of peptic ulceration (abdominal pain, black, tarry stools) periodically during therapy. Symptoms may be masked by deflazacort.
  • Assess for signs and symptoms of behavioral and mood disturbances (euphoria, insomnia, mood swings) during therapy. May require dose reduction or discontinuation.
  • Monitor intraocular pressure periodically during therapy if treatment lasts more than 6 wk.
  • Assess for signs and symptoms of toxic epidermal necrolysis (rash, itching, skin blistering) during therapy; usually begin within 8 wks of starting therapy. Discontinue deflazacort at 1st sign of rash.
  • Monitor for signs and symptoms of Kaposi's sarcoma (slightly elevated purple, pink, brown, or red blotches or bumps anywhere on the skin or in the mouth and/or throat, lymphedema, unexplained cough or chest pain, unexplained stomach or intestinal pain) periodically during therapy. Clinical improvement may occur with discontinuation of deflazacort.

Lab Test Considerations: Monitor blood glucose levels periodically during therapy. May cause hyperglycemia. Treat or adjust treatment as needed.

  • May cause changes in thyroid levels. If levothyroxine and corticosteroids are required, initiate corticosteroids first to reduce risk of adrenal crisis.
  • If pheochromocytoma is suspected, deflazacort may cause crisis.
  • May cause reactivation of hepatitis B virus.
  • Monitor serum potassium levels during therapy; may require supplementation.
  • Monitor bone mineral density in patients on long-term therapy.

Potential Diagnoses


  • PO Administer without regard to food. Tablets may be taken whole or crushed and mixed with applesauce.
  • Shake suspension well prior to administration. Use disperser provided for accurate dose. Add suspension slowly to 3–4 ounces of juice or milk; mix well. Administer immediately. Do not administer with grapefruit juice. Discard bottle 1 month after first opened.

Patient/Family Teaching

  • Instruct patient and parents to take as directed. Do not stop taking without consulting health care professional; must be discontinued gradually.
  • Advise patient to avoid live virus vaccines during therapy.
  • Instruct patient to notify health care professional if signs and symptoms of infection, BP or fluid retention, behavioral or mood disturbances (manic or depressive symptoms), or rash occur.
  • Inform patient of need for periodic bone mineral density and ophthalmalogic exams.
  • Advise female patients to notify health care professional if pregnancy is planned or suspected or if breast feeding.

Evaluation/Desired Outcomes

Improved muscle strength.

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