Receiving a diagnosis of Duchenne Muscular Dystrophy (DMD) for your child is a life-altering moment, marking the beginning of a journey focused on preserving strength, mobility, and quality of life. DMD is a progressive genetic disorder that causes muscle weakness, and managing its effects is a daily priority. In the landscape of DMD treatments, corticosteroids are a cornerstone of care, and one important option in this class is Deflazacort.

Deflazacort is a prescription corticosteroid, a type of medication that mimics the effects of hormones your body produces naturally to fight inflammation. While not a cure, it is a standard-of-care treatment specifically approved to help manage the symptoms of DMD in patients two years of age and older. For families navigating the complexities of DMD, understanding how Deflazacort works can empower them to be active partners in their child’s healthcare.

What does Deflazacort do?

Deflazacort is approved by the U.S. Food and Drug Administration (FDA) for the treatment of Duchenne Muscular Dystrophy. Its primary purpose is to help patients retain muscle strength and function for as long as possible. By slowing the progression of muscle weakness, it can have a meaningful impact on a child’s life.

Patients taking Deflazacort may experience:

• A slower decline in muscle strength
• The ability to continue walking independently for a longer period of time
• Improved pulmonary (lung) function
• A delay in the development of scoliosis (curvature of the spine)

Daily corticosteroid treatment, such as Deflazacort, significantly alters disease progression, delaying the loss of ambulation for several years and promoting independence in children. (U.S. Food and Drug Administration, 2017).

How does Deflazacort work?

The exact way Deflazacort helps in DMD is complex, but its main benefits are tied to its powerful anti-inflammatory and immunosuppressive properties. In Duchenne Muscular Dystrophy, the absence of a crucial protein called dystrophin makes muscle cells fragile and easily damaged. This damage triggers a chronic inflammatory response from the body’s immune system, which in turn causes even more harm to the muscles over time.

Deflazacort works by acting like cortisol, a natural hormone produced by the adrenal glands. It enters cells throughout the body and switches off the genes that produce inflammatory substances. Think of it as a firefighter that dampens the flames of inflammation that are constantly attacking the muscles.

By broadly suppressing this inflammatory process, Deflazacort helps to protect the already vulnerable muscle fibers from further injury. This protective effect is what helps preserve muscle function and slow the progression of the disease. It is a derivative of another common steroid, prednisone, but is thought to have a different profile regarding certain side effects, such as weight gain.

Deflazacort side effects

As a corticosteroid, Deflazacort affects the entire body and can cause a range of side effects, particularly with long-term use. Your child’s healthcare team will monitor them closely to manage these effects.

Common side effects may include:

• Increased appetite and weight gain
• Cushingoid appearance (facial puffiness, often called “moon face”)
• Unwanted hair growth
• Skin problems like acne or easy bruising
• Mood changes, such as irritability or mood swings

Long-term use can lead to more serious side effects, such as:

• Weakened immune system, leading to an increased risk of infections
• Stunted growth in children
• Reduced bone density (osteoporosis), which increases the risk of fractures
• Increased blood sugar levels and risk of diabetes
• Cataracts or glaucoma (increased pressure in the eyes)

Inform your doctor of any active infections before treatment. Do not stop Deflazacort abruptly; taper dose slowly under a doctor’s supervision to avoid withdrawal. Seek immediate medical attention if your child shows signs of a severe allergic reaction, such as hives, difficulty breathing, or swelling of the face and throat (Mayo Clinic, 2024).

Deflazacort dosage

Deflazacort is available as an oral tablet and an oral suspension. It is typically taken once a day, and the dose is based on the patient’s body weight. The tablets can be crushed and mixed with soft food like applesauce if your child has difficulty swallowing them whole.

Given the potential for long-term side effects, regular and thorough monitoring is a critical part of treatment. Your child’s healthcare provider will likely schedule routine check-ups to:

• Monitor growth and weight.
• Check blood pressure and blood sugar levels.
• Perform eye exams to screen for cataracts and glaucoma.
• Assess bone health using bone density scans (DEXA scans).
• Monitor for any signs of infection.

These regular evaluations ensure continued treatment benefits outweigh risks, allowing doctors to adjust the care plan as needed.

Does Deflazacort have a generic version?

Yes, there are FDA-approved generic versions of Deflazacort available in the United States. The brand name for deflazacort is Emflaza. The FDA requires that generic medications be bioequivalent to their brand-name counterparts, meaning they have the same active ingredient, work the same way, and are just as safe and effective (FDA, 2019). The availability of generics often makes treatment more accessible and affordable for families.

Conclusion

Deflazacort is a vital tool in the management of Duchenne muscular dystrophy. While it is not a cure, its ability to reduce inflammation and protect muscle fibers can significantly slow the progression of the disease, helping children maintain strength, mobility, and a better quality of life for longer. The decision to use a corticosteroid involves carefully balancing these profound benefits against the well-known side effects.

With close supervision from a dedicated healthcare team, the risks associated with Deflazacort can be effectively monitored and managed. By staying informed and working in partnership with your doctor, you can make the best decisions for your child’s health and help them navigate their journey with DMD with strength and resilience.

References

1. Mayo Clinic. (2024). Deflazacort (Oral Route). https://www.mayoclinic.org/drugs-supplements/deflazacort-oral-route/side-effects/drg-20150536
2. U.S. Food and Drug Administration. (2017). EMFLAZA® (deflazacort) Prescribing Information. https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/208684s000,208685s000lbl.pdf
3. U.S. Food and Drug Administration. (2019). FDA approves first generics of Emflaza. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-generics-emflaza