Deflazacort Oral Suspension: Not for Infants Due to Preservative Risk
It is critical for parents and caregivers to understand that deflazacort oral suspension is not indicated for use in infants. The product's label explicitly warns against using it in children younger than 2 years of age. The primary reason for this is the inclusion of a preservative called benzyl alcohol in the oral suspension formula.
The Risk of Benzyl Alcohol in Infants
Benzyl alcohol is a known preservative that can be toxic to very young or premature infants. The US Food and Drug Administration (FDA) has issued warnings regarding the potential for serious and fatal adverse reactions associated with benzyl alcohol, a condition known as "gasping syndrome". This syndrome is characterized by central nervous system depression, metabolic acidosis (an acid-base imbalance in the body), and gasping respirations. While deflazacort oral suspension is approved for use in children aged 2 and older, the presence of benzyl alcohol makes it unsafe for use in babies and infants.
The Approved Use of Deflazacort for Older Children
For children aged 2 years and older, deflazacort oral suspension is an approved treatment for Duchenne muscular dystrophy (DMD). DMD is a rare, inherited genetic disorder that leads to progressive muscle damage and weakness. Deflazacort, a corticosteroid, helps to manage the condition by reducing inflammation and suppressing the immune system, which in turn helps to preserve muscle strength and delay the progression of the disease.
By easing the inflammatory response that is believed to drive muscle damage in DMD, deflazacort can improve motor function and prolong the age at which a child can walk independently. Long-term use in older children has also shown potential benefits in delaying other complications associated with DMD, such as pulmonary and cardiac issues.
Key Considerations for Deflazacort Treatment
Before a child begins treatment with deflazacort, a healthcare provider will conduct a thorough evaluation. This includes ensuring the child is up-to-date on all immunizations, as corticosteroids can suppress the immune system and increase the risk of infection. Patients on this medication must be regularly monitored for potential side effects, which can be significant, especially with long-term use.
Common side effects in children include:
- Cushingoid appearance (facial puffiness)
- Weight gain and increased appetite
- Changes in mood and behavior, including irritability
- Upper respiratory tract infections
- Cough
- Frequent urination
- Gastrointestinal issues, such as constipation or stomach pain
Long-term and more serious side effects may include:
- Slower growth and development
- Development of cataracts or glaucoma
- Weakened bones (osteoporosis) leading to increased fracture risk
- Elevated blood pressure and blood sugar levels
- Adrenal gland suppression
Deflazacort vs. Prednisone for Duchenne Muscular Dystrophy
Deflazacort is not the only corticosteroid used to manage DMD. Prednisone is another commonly prescribed steroid, and they have some differences in their side effect profiles.
| Feature | Deflazacort | Prednisone | References |
|---|---|---|---|
| Mechanism | Corticosteroid prodrug, metabolized to an active form that acts through the glucocorticoid receptor. | Corticosteroid prodrug, metabolized to prednisolone. | |
| FDA Approval (for DMD) | Approved for DMD in patients aged 2 and older. | Used off-label for DMD; not specifically FDA-approved for this condition. | |
| Effect on Weight Gain | Often associated with less weight gain, especially in the first year of treatment. | Associated with a greater risk of significant weight gain. | |
| Risk of Cataracts | May carry a higher risk of developing cataracts with long-term use. | Risk of cataracts is generally lower compared to deflazacort. | |
| Behavioral Side Effects | Patients may experience more withdrawn behavior. | May be associated with more aggressive behavioral changes. | |
| Bone Mineral Density | May have a relatively bone-sparing effect compared to prednisone in some studies, but long-term monitoring is still necessary. | Greater risk of bone demineralization, requiring regular monitoring. |
How Deflazacort Oral Suspension is Administered
For children aged 2 and older, deflazacort oral suspension is taken orally, typically once a day. The dose is based on the child's body weight, and a measuring device provided with the medication should be used for accuracy. To help with administration, the suspension can be mixed with 3 to 4 ounces of milk or juice (not grapefruit juice) and should be taken immediately after mixing.
Conclusion
In summary, it is vital to be aware that deflazacort oral suspension is not for use in babies due to the serious risks associated with its benzyl alcohol preservative. The medication is only approved for children aged 2 years and older for the treatment of Duchenne muscular dystrophy. While it offers a valuable therapeutic option for managing DMD symptoms and delaying disease progression in older children, it is a potent corticosteroid with a range of potential side effects, requiring close medical supervision. Parents should always discuss the risks and benefits of all medications with their healthcare provider to determine the safest and most appropriate treatment course.
For more detailed information on Duchenne muscular dystrophy, resources like the Muscular Dystrophy Association are valuable sources of information. https://www.mda.org/disease/duchenne-muscular-dystrophy
