Defzort6 mg

FDA-Approved Indication: Duchenne Muscular Dystrophy

Defzort is FDA-approved (established indication) for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. Defzort is used to help slow the decline in muscle strength associated with this genetic muscle-wasting disease.

Broader Anti-Inflammatory and Immunosuppressive Uses (Guideline-Supported/Off-Label Outside the US)

Outside the United States - including in Bangladesh, where Defzort is widely prescribed - Defzort is also used as a general corticosteroid for a broad range of inflammatory, allergic, and autoimmune conditions. These uses are guideline-supported or established in regional clinical practice but are not part of the US FDA-approved label for Defzort:

• Allergic/hypersensitivity conditions: anaphylaxis (adjunct), severe hypersensitivity reactions, acute and chronic bronchial asthma exacerbations
• Rheumatologic/connective tissue disorders: rheumatoid arthritis, juvenile chronic (idiopathic) arthritis, polymyalgia rheumatica, systemic lupus erythematosus, dermatomyositis, mixed connective tissue disease, polyarteritis nodosa, sarcoidosis
• Dermatologic conditions: pemphigus, bullous pemphigoid, pyoderma gangrenosum
• Renal disorders: minimal change nephrotic syndrome, acute interstitial nephritis
• Cardiac: rheumatic carditis
• Gastrointestinal: ulcerative colitis, Crohn's disease (adjunct)
• Ophthalmic: uveitis, optic neuritis
• Hematologic: autoimmune hemolytic anemia, idiopathic thrombocytopenic purpura
• Oncologic (adjunct): acute lymphoblastic leukemia, malignant lymphoma, multiple myeloma, as part of combination chemotherapy regimens
• Transplantation: immunosuppression to prevent/treat organ transplant rejection

Dosing and duration for each condition should be individualized by a qualified physician.

Each Deflazacort tablet contains Deflazacort INN as the active ingredient, available in strengths of 6 mg, 18 mg, 24 mg, 30 mg, and 36 mg. Deflazacort is also available as an oral suspension (22.75 mg/mL) and, in some markets, an oral syrup (6 mg/5 mL).

Defzort is a corticosteroid (glucocorticoid) medicine used to reduce inflammation and calm an overactive immune system. In the United States, Defzort is FDA-approved specifically for Duchenne muscular dystrophy in children aged 2 years and older, where Defzort helps preserve muscle strength. In many other countries, including Bangladesh, Defzort is also used more broadly for allergic, autoimmune, and inflammatory conditions such as asthma, arthritis, and certain kidney and blood disorders. Defzort works by binding to glucocorticoid receptors and suppressing inflammatory pathways throughout the body. Because Defzort affects multiple body systems, Defzort should only be used under close medical supervision, with the dose tailored to each patient's condition.

Corticosteroid (Glucocorticoid)

Deflazacort is a prodrug that is rapidly converted by esterases in the body to Deflazacort's pharmacologically active metabolite, 21-desDFZ. This active metabolite binds to the glucocorticoid receptor, producing anti-inflammatory and immunosuppressive effects, including inhibition of phospholipase A2 (which reduces the production of inflammatory mediators) and suppression of the release of pro-inflammatory cytokines and other immune mediators. Through these actions, Deflazacort reduces inflammation, edema, and immune-mediated tissue damage across multiple organ systems.

• Take Defzort by mouth, generally once daily, at approximately the same time each day.
• Defzort tablets and oral suspension may be taken with or without food; taking Defzort with food or a light meal may help reduce stomach upset.
• Do not take Defzort with grapefruit or grapefruit juice, as grapefruit can increase Defzort levels in the body.
• Shake the Defzort oral suspension well before measuring each dose, and use an accurate oral dosing syringe or cup, not a household spoon.
• Swallow Defzort tablets whole, or as directed by your doctor or pharmacist.
• Do not stop taking Defzort suddenly, especially after using Defzort for more than a few days - the dose of Defzort must be tapered down gradually under medical supervision.
• If a dose of Defzort is missed, take the missed dose as soon as remembered unless it is almost time for the next dose of Defzort; do not double the dose.
• Keep all follow-up appointments so your doctor can monitor your response to Defzort and adjust the dose of Defzort as needed.

• CYP3A4 inhibitors (e.g., ketoconazole, clarithromycin, fluconazole, diltiazem, verapamil, grapefruit juice): These drugs can significantly increase blood levels of the active metabolite of Defzort by inhibiting the breakdown of Defzort; the dose of Defzort may need to be reduced to about one-third of the usual dose.
• CYP3A4 inducers (e.g., rifampicin, rifabutin, carbamazepine, phenytoin, phenobarbitone, efavirenz): These drugs speed up the breakdown of Defzort's active metabolite, reducing the effectiveness of Defzort; concurrent use with strong inducers should generally be avoided.
• NSAIDs (e.g., ibuprofen, diclofenac, aspirin): Combining NSAIDs with Defzort increases the risk of gastrointestinal irritation, ulceration, and bleeding.
• Potassium-depleting diuretics and other drugs affecting potassium (e.g., furosemide, thiazide diuretics, amphotericin B): Defzort increases potassium excretion, so combined use with these drugs raises the risk of hypokalemia; potassium levels should be monitored.
• Antidiabetic medications (insulin, oral hypoglycemic agents): Defzort can raise blood glucose levels and reduce the effectiveness of antidiabetic medications, so doses of diabetes medications may need to be adjusted.
• Live or live-attenuated vaccines: Defzort suppresses the immune response, so live vaccines should not be given during Defzort therapy at immunosuppressive doses; vaccination should be completed before starting Defzort where possible.
• Other immunosuppressants (e.g., other corticosteroids, methotrexate, cyclosporine): Combined use of these drugs with Defzort increases the overall risk of infection due to additive immune suppression.
• Neuromuscular blocking agents (e.g., pancuronium): Concurrent use of neuromuscular blocking agents with Defzort has been associated with an increased risk of acute myopathy, particularly affecting respiratory muscles.

Common Side Effects

• Cushingoid appearance (rounded face, fat redistribution)
• Weight gain and increased appetite
• Hirsutism (excess hair growth)
• Upper respiratory tract infection, nasopharyngitis, cough
• Irritability and behavioral changes
• Abdominal discomfort, nausea, constipation
• Frequent urination (pollakiuria)
• Skin changes: acne, erythema, striae, skin thinning
• Headache, back pain

Serious Side Effects - Seek Medical Attention

• Signs of adrenal insufficiency (severe fatigue, weakness, dizziness, low blood pressure, vomiting) - especially if Defzort is stopped abruptly
• Signs of infection (fever, sore throat, unusual fatigue) that may be masked by Defzort
• Severe stomach pain, black or bloody stools, vomiting blood (possible GI ulcer/perforation)
• Vision changes, eye pain (possible cataracts, glaucoma, or increased intraocular pressure)
• Severe mood changes, depression, confusion, or psychosis
• Signs of blood clots (leg swelling/pain, chest pain, shortness of breath)
• Bone pain or fractures (possible osteoporosis or avascular necrosis)
• Severe skin reaction (blistering, peeling skin, rash with fever) - possible toxic epidermal necrolysis
• Muscle weakness, especially affecting breathing muscles
• Signs of heart failure (swelling, shortness of breath, rapid weight gain)
• Symptoms of high blood sugar (increased thirst, frequent urination, confusion)

Pregnancy

Defzort readily crosses the placenta. As with other corticosteroids, use of Defzort during pregnancy has been associated with a small increase in the risk of orofacial clefts when Defzort is used in the first trimester, and with intrauterine growth restriction and lower birth weight when Defzort is used later in pregnancy. Defzort should be used during pregnancy only if the potential benefit to the mother justifies the potential risk to the fetus, and only under close medical supervision. Infants born to mothers who received substantial doses of Defzort during pregnancy should be monitored for signs of adrenal insufficiency.

Breastfeeding

Corticosteroids, including Defzort, pass into breast milk. Low to moderate doses of Defzort are generally considered unlikely to cause adverse effects in a breastfed infant, but higher or prolonged doses of Defzort could theoretically affect infant growth or adrenal function. A doctor should weigh the mother's need for Defzort against the possible effects on the breastfed infant, and the infant should be monitored if the mother is taking Defzort.

• Adrenal suppression and tapering (do not stop abruptly): Long-term use of Defzort suppresses the body's natural cortisol production (HPA axis suppression). Stopping Defzort suddenly after prolonged use can cause acute adrenal insufficiency, which can be life-threatening; the dose of Defzort must always be reduced gradually under medical supervision.
• Increased risk of infection: Defzort suppresses the immune system, increasing susceptibility to viral, bacterial, fungal, and parasitic infections, and can mask the usual signs of infection (such as fever), delaying diagnosis and treatment.
• Osteoporosis and growth suppression: Long-term Defzort use decreases bone mineral density, increasing the risk of fractures in adults, and can suppress linear growth in children; bone health and growth in children should be monitored during prolonged Defzort therapy.
• Cushingoid effects: Prolonged use of Defzort commonly causes a Cushingoid appearance (facial rounding, central weight gain, purple striae) and, in severe cases, Cushing's syndrome with associated metabolic and psychiatric effects.
• Hyperglycemia: Defzort can raise blood glucose levels and unmask or worsen diabetes mellitus; blood glucose should be monitored, particularly in patients with existing diabetes or risk factors for diabetes.
• Ophthalmic effects: Prolonged Defzort use (more than 6 weeks) can cause posterior subcapsular cataracts, glaucoma, and increased intraocular pressure; regular eye examinations are recommended during long-term Defzort therapy.
• Cardiovascular and fluid effects: Defzort can cause fluid and salt retention, elevated blood pressure, and potassium loss; Defzort should be used with caution in patients with hypertension, heart failure, or renal impairment.
• Gastrointestinal risk: Defzort should be used with caution in patients with active or prior peptic ulcer disease, diverticulitis, or ulcerative colitis, due to an increased risk of GI perforation or bleeding with Defzort.
• Psychiatric effects: Defzort may cause mood swings, insomnia, anxiety, depression, or, rarely, psychosis, particularly at higher doses of Defzort.
• Serious skin reactions: Rare but serious skin reactions, including toxic epidermal necrolysis, have been reported with Defzort; discontinue Defzort and seek medical attention if a severe skin reaction occurs.
• Live vaccines: Live or live-attenuated vaccines should not be given to patients receiving immunosuppressive doses of Defzort.
• Use with caution in specific conditions: Defzort should be used cautiously in patients with myasthenia gravis, epilepsy, hypothyroidism, ocular herpes simplex, or a history of psychiatric illness.

There is no specific antidote for Defzort overdose. In cases of acute overdose of Defzort, treatment with gastric lavage or induced emesis may be considered, but only if directed by a healthcare professional or poison control - do not induce vomiting on your own unless instructed to do so. Management is otherwise supportive and symptomatic. Chronic overexposure to Defzort (excessive dosing over time) may require temporary dose reduction or a switch to alternate-day dosing under medical guidance. If Defzort overdose is suspected, contact emergency medical services or a poison control center immediately.

Store Defzort according to the product label. In general, keep Defzort in a cool, dry place below 25°C, protected from light and moisture, and out of the reach of children. Do not use Defzort after the expiry date printed on the pack.

Children

Defzort is FDA-approved for Duchenne muscular dystrophy in children 2 years of age and older, and is also used more broadly for pediatric inflammatory and autoimmune conditions. Long-term corticosteroid use, including use of Defzort, can suppress growth in children; height and growth should be monitored regularly during prolonged Defzort therapy, and growth typically resumes after Defzort is discontinued in most cases. The oral suspension formulation of Defzort (which may contain benzyl alcohol) should not be used in infants under 2 years of age.

Elderly

No specific dose adjustment of Defzort is required based on age alone in elderly patients, but elderly patients receiving Defzort should be monitored closely, as age-related conditions such as osteoporosis, diabetes, and hypertension can make the adverse effects of Defzort more serious.

Renal Impairment

No specific dose adjustment of Defzort is generally required in renal impairment; standard precautions for corticosteroid use apply.

Hepatic Impairment

In patients with significant hepatic impairment, blood levels of Defzort's active metabolite may increase; Defzort should be used with caution and the dose of Defzort monitored carefully in these patients.

Diabetic Patients

Defzort can raise blood glucose levels; patients with diabetes may need closer glucose monitoring and adjustment of their antidiabetic medications while taking Defzort.

For acute conditions (such as a severe allergic reaction or an asthma exacerbation), Defzort is typically used as a short course at a higher dose, then tapered and stopped once the condition resolves. For chronic conditions, including Duchenne muscular dystrophy and long-term autoimmune or inflammatory diseases, Defzort is often continued for months to years at the lowest effective dose. Whenever Defzort has been used for more than a few days, Defzort must be tapered off gradually under medical supervision rather than stopped suddenly, to allow the body's own cortisol production to recover and to avoid adrenal insufficiency.

Corticosteroid; Glucocorticoid; Anti-inflammatory and immunosuppressive agent

Deflazacort is a prodrug converted to Deflazacort's active metabolite, 21-desDFZ, which binds to the glucocorticoid receptor. This receptor binding modulates gene transcription, reducing the production of inflammatory mediators (including inhibition of phospholipase A2) and suppressing the migration and activity of immune cells, resulting in anti-inflammatory and immunosuppressive effects.

Defzort is FDA-approved for the treatment of Duchenne muscular dystrophy in children 2 years of age and older, based on clinical trial data in boys aged 5 to 15 years, with safety and efficacy in younger children (2 to 5 years) extrapolated from this data. Defzort is also used more broadly in pediatric patients outside the US for conditions such as juvenile chronic (idiopathic) arthritis, nephrotic syndrome, and acute severe asthma, generally at doses of 0.25-1.5 mg/kg/day depending on the condition. Because long-term use of Defzort and other corticosteroids can suppress linear growth in children, height, weight, and growth velocity should be monitored regularly throughout Defzort treatment, and a pediatric specialist should be involved in long-term management.