What Is the Drug Deflazacort Used For? A Comprehensive Guide

Deflazacort, a corticosteroid marketed under the brand name Emflaza, is primarily used to treat Duchenne muscular dystrophy (DMD) in patients aged 2 years and older. What is the drug Deflazacort used for? Beyond DMD, it has also been used for various inflammatory and autoimmune conditions, particularly outside the U.S..

Quick Summary

Deflazacort is a corticosteroid indicated for treating Duchenne muscular dystrophy and other inflammatory and autoimmune diseases. It works by suppressing inflammation and immune activity, but requires careful management.

Key Points

• Primary Indication: Deflazacort is FDA-approved to treat Duchenne muscular dystrophy (DMD) in patients aged 2 years and older.

• Mechanism: As a corticosteroid prodrug, it reduces inflammation and suppresses the immune system after being metabolized to its active form.

• Less Weight Gain and Mood Swings: Compared to prednisone, deflazacort may cause less weight gain and fewer behavioral side effects in DMD patients.

• Increased Risk of Cataracts: Some studies indicate a higher incidence of cataracts and potential negative effects on bone growth with long-term deflazacort use.

• Administration: It is a once-daily oral medication available in tablet or suspension form.

• Tapering is Crucial: Abruptly stopping deflazacort can cause serious adrenal problems, so a gradual reduction under medical guidance is essential.

• Immunosuppression Risk: The medication increases the risk of infections, and live vaccines should be avoided during treatment.

What is Deflazacort?

Deflazacort is a synthetic glucocorticoid belonging to the class of medications called corticosteroids. It is a prodrug, which means it is inactive when administered and is then converted by the body into its active form, 21-desacetyldeflazacort. This active metabolite exerts powerful anti-inflammatory and immunosuppressive effects by binding to glucocorticoid receptors in cells, which alters gene expression to reduce inflammation and immune system activity. First developed for use in inflammatory conditions, its most prominent approved use today, particularly in the United States, is for Duchenne muscular dystrophy.

Primary Use in Duchenne Muscular Dystrophy (DMD)

Since its FDA approval in 2017 under the brand name Emflaza, deflazacort has become an important treatment for Duchenne muscular dystrophy (DMD) in patients two years of age and older. DMD is a progressive and severe inherited disease characterized by muscle degeneration and weakness due to a genetic mutation affecting the dystrophin protein. Treatment with deflazacort has been shown to provide several significant benefits in managing the condition, including:

• Delayed progression: Studies have shown that deflazacort can delay the progression of muscle weakness, prolonging ambulation and other motor function milestones.
• Preserved function: It helps preserve cardiac and respiratory function, which are often compromised in the later stages of DMD.
• Reduced scoliosis: Long-term treatment can help reduce the severity of scoliosis, potentially delaying or eliminating the need for spinal surgery.
• Improved quality of life: By slowing functional decline, the medication can significantly improve the overall quality of life for patients and their families.

Use in Other Inflammatory and Autoimmune Conditions

Before its specific approval for DMD in the U.S., deflazacort was used internationally for a range of other inflammatory and autoimmune diseases. Its potent anti-inflammatory and immunosuppressive properties make it effective for many steroid-responsive conditions. These include:

• Rheumatoid arthritis: A chronic inflammatory disorder affecting the joints.
• Juvenile chronic arthritis: A form of arthritis affecting children.
• Bronchial asthma: Used to control inflammation and improve symptoms.
• Nephrotic syndrome: A kidney disorder causing the body to exc

Frequently Asked Questions

Is deflazacort a chemotherapy drug?

No, deflazacort is a corticosteroid, not a chemotherapy drug. While some corticosteroids are used in certain cancer treatment regimens, deflazacort itself is primarily used for its anti-inflammatory and immunosuppressive properties in conditions like Duchenne muscular dystrophy.

How long does it take for deflazacort to show results?

For Duchenne muscular dystrophy, some studies have shown improvements in muscle strength within a few months of starting treatment. However, the long-term benefits of slowing disease progression are more significant over extended treatment periods.

Can deflazacort be stopped suddenly?

No, it is dangerous to stop deflazacort abruptly. Sudden discontinuation can lead to adrenal insufficiency, a potentially fatal condition. The use of this medication must be tapered gradually under the supervision of a healthcare provider.

What are the most common side effects of deflazacort?

Common side effects include weight gain, increased appetite, facial puffiness, frequent urination, upper respiratory tract infections, increased hair growth, and mood changes.

How does deflazacort differ from prednisone?

While both are corticosteroids used for similar conditions, studies suggest deflazacort may cause less weight gain and behavioral side effects than prednisone. However, deflazacort has been associated with a higher risk of cataracts and potential bone loss in some patient groups.

Is deflazacort safe for children with Duchenne muscular dystrophy?

Deflazacort is FDA-approved for children aged 2 and older with DMD and is a standard treatment option. However, careful monitoring is necessary to manage potential side effects such as growth suppression and cataracts.

What precautions should be taken regarding vaccinations?

Live-attenuated vaccines should not be given to patients receiving immunosuppressive doses of deflazacort. All necessary immunizations should be completed before starting treatment, and patients should be cautioned to avoid exposure to illnesses like chickenpox or measles.

Can deflazacort be taken with or without food?

Deflazacort tablets and oral suspension can be taken with or without food. Mixing the oral suspension with milk or juice is an option, but grapefruit juice should be avoided.

What happens if a dose of deflazacort is missed?

If a dose is missed, it should be taken as soon as it is remembered. However, if it is almost time for the next dose, the missed dose should be skipped to avoid a double dose.