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Deflazacort

Duchenne Muscular Dystrophy, Inflammation

DRUG STATUS

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Approvals

US(FDA), UK(BNF)

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Essential Medicine

NO

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Teratogenicity

N/A

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Pharmaceutical Class

Corticosteroid

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Drug Ingredient

Deflazacort

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Summary

  • Deflazacort is a medication used to treat Duchenne muscular dystrophy (DMD), a muscle weakening disease, in children aged 5 and older.

  • Deflazacort works by reducing inflammation and suppressing the immune system. It turns into an active form that attaches to a part of cells called the glucocorticoid receptor. This triggers a series of events that reduce production of inflammatory substances and suppress the immune response.

  • Deflazacort is taken once daily by mouth. The recommended dose is about 0.9 mg for each kilogram of body weight. It can be taken with or without food, but not with grapefruit juice.

  • Common side effects include weight gain, increased appetite, a round face (Cushingoid appearance), upper respiratory infections (common cold), frequent urination, nose and throat inflammation, excessive hair growth, and irritability.

  • Deflazacort should not be taken by people who are allergic to it or any of its ingredients. It should be used with caution during pregnancy and breastfeeding. Stopping Deflazacort suddenly can lead to steroid withdrawal syndrome, causing symptoms like loss of appetite, nausea, vomiting, tiredness, and headache.

Indications and Purpose

What is Deflazacort used for?

Deflazacort tablets are a medication used to treat a condition called Duchenne muscular dystrophy (DMD) in children aged 5 and older. 

DMD is a muscle weakening disease which mainly affects boys due to the way the disease is inherited (it's linked to the X chromosome). 

Deflazacort tablets work by reducing inflammation and slowing down muscle damage, which can help improve muscle function and strength.

How does Deflazacort work?

Deflazacort is a medication that reduces inflammation and suppresses the immune system. It works by turning into an active form that attaches to a specific part of cells, called the glucocorticoid receptor. This attachment triggers a series of events that lead to the reduced production of inflammatory substances and the suppression of the immune response.

Is Deflazacort effective?

In a study, people taking a daily dose of 0.9 mg/kg of deflazacort had a bigger increase in average muscle strength over 12 weeks than those taking a placebo (inactive substance).Studies show it improves strength, slows disease progression, and may delay wheelchair use. It's not a cure, but it can improve quality of life.

How does one know if Deflazacort is working?

Deflazacort helps treat Duchenne muscular dystrophy (DMD) by reducing inflammation and protecting muscles. Doctors track its benefits by checking muscle strength with tests like the 6-minute walk test and by monitoring lung function

Regular checkups are important to track progress and manage any side effects, like changes in growth, blood pressure, or bone health.

Directions for Use

How do I take Deflazacort?

You can take Deflazacort tablets with or without food. However, avoid taking them with grapefruit juice.

For how long do I take Deflazacort?

The typical duration of deflazacort use varies widely depending on the condition being treated and individual patient factors. For its primary indication—Duchenne Muscular Dystrophy (DMD)—deflazacort is often used as long-term therapy to manage symptoms and slow disease progression. Patients may remain on deflazacort for months to years, depending on their response to treatment and side effect profile. If used for other inflammatory or autoimmune conditions, the duration can range from short courses (days to weeks) to longer periods, tailored to the patient's specific needs. Deflazacort is a corticosteroid medication. When taken for more than a few days, it's important to gradually reduce the dosage to avoid increasing the risk of osteoporosis, a condition that makes bones weak and brittle.  If you take deflazacort for more than 6 weeks , your doctor may monitor you for cataracts or glaucoma, eye conditions that can be caused by prolonged corticosteroid use.

How long does it take for Deflazacort to start working?

Deflazacort may take a few weeks to show initial effects and several months for more noticeable benefits. This is because the drug primarily works by slowing the progression of muscle damage rather than directly repairing existing damage.

How should I store Deflazacort?

Medications should be stored at room temperature, between 68° to 77°F (20° to 25°C). They can be stored at temperatures ranging from 59° to 86°F (15° to 30°C) for short periods. Avoid storing medications in extreme heat or cold, as this can damage them.

Warnings and Precautions

Who should avoid taking Deflazacort?

Deflazacort tablets should not be taken by people who are allergic to deflazacort or any of its ingredients. 

Some people who have taken corticosteroids, including deflazacort, have had allergic reactions, including anaphylaxis (a severe, life-threatening allergic reaction).

Can I take Deflazacort with other prescription drugs?

Some medicines can affect how well deflazacort tablets work. Tell your doctor about all the medicines you are taking, including those you buy without a prescription.

Can I take Deflazacort with vitamins or supplements?

When taking Deflazacort, check with your doctor before taking any vitamins or supplements. 

Some can affect how the medicine works or increase side effects. This is especially important for Vitamin D and calcium, as Deflazacort can impact bone health. 

Avoid St. John's Wort and be cautious with grapefruit.

Can Deflazacort be taken safely while pregnant?

Corticosteroids are a type of medication that can have harmful effects on a baby if taken during pregnancy. These effects can include birth defects like cleft lip or cleft palate, as well as low birth weight and stunted growth.

Can Deflazacort be taken safely while breastfeeding?

Corticosteroids are medicines that can be passed through breast milk. While these medicines may be needed by the mother, they can potentially harm the baby by affecting their growth, hormone production, or causing other problems. 

Doctors weigh the benefits of breastfeeding to the baby with the risks of the medicine to the baby when deciding if a mother should breastfeed while taking corticosteroids. 

Is Deflazacort safe for the elderly?

Deflazacort is a drug used to treat Duchenne muscular dystrophy (DMD), a condition that affects children and young adults. There is no experience with using deflazacort in older adults because DMD usually affects younger people.