What are the detailed instructions for Deflazacort?

Deflazacort is a synthetic glucocorticoid drug that belongs to the Corticosteroid family. Compared with traditional prednisone and methylprednisolone, deflazacort has certain advantages in pharmacological activity and safety, especially showing better tolerance in long-term neuromuscular diseases. The drug is branded Emflaza and was approved by the US FDA in 2017 for the treatment of patients 2 years old and older with Duchenne Muscular Dystrophy (DMD)**. Currently on the market in Europe, the United States and some Latin American countries, it is considered an important oral hormone option to improve the quality of life of DMD patients.

【Mechanism of action】

From a pharmacological mechanism, deflazacort inhibits the release of inflammatory mediators by binding to intracellular glucocorticoid receptors, thereby reducing muscle inflammation and tissue damage. It can regulate muscle fiber metabolism, reduce the rate of muscle degeneration, and delay the progression of muscle weakness and loss of mobility in patients. AlthoughDeflazacort cannot cure Duchenne muscular dystrophy, but long-term regular use can significantly improve patients' muscle strength, lung function and walking time. It is currently one of the irreplaceable core drugs in the management of DMD internationally.

【Indications】

Deflazacort's primary indication is the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. It slows down muscle atrophy by reducing chronic inflammatory responses and protecting muscle fiber structure. In clinical practice in some countries, Deflazacort is also used "off-label" to treat immune diseases such as systemic lupus erythematosus, asthma, rheumatoid arthritis, nephrotic syndrome, etc. However, these uses must be strictly followed by medical advice because the dosage and risks in different diseases vary greatly.

【Usage and Dosage】

Deflazacort dosage is usually calculated based on patient weight. The recommended dose for adults is 0.9 mg/kg once daily, usually in the morning. Doses may be rounded to the nearest tablet dose based on body weight. For children 2 years and older, the dosage is the same as for adults (based on body weight). The medication should be swallowed whole and taken after meals to reduce stomach upset. If patients are concurrently using moderate or strong CYP3A4 inhibitors (such as ketoconazole, clarithromycin, etc.), the dose needs to be reduced to one-third of the recommended dose to avoid adverse reactions caused by increased plasma concentrations. If you forget to take a dose, you should take it as soon as possible, but do not double the dose.

[Adverse reactions and precautions]

Deflazacort has similar adverse effects to other corticosteroids, but is generally better tolerated. Common reactions include weight gain, increased appetite, mild mood swings, facial swelling, and sleep disturbances. Long-term use may lead to decreased bone density, increased blood sugar, and weakened immunity, so it is recommended to regularly monitor bone metabolism indicators and blood sugar levels. Compared with prednisone, deflazacort has relatively mild effects on bone metabolism and blood sugar and is considered a "milder" hormone alternative. For pediatric patients, it is necessary to pay close attention to growth rate and weight changes to prevent the use of hormones from affecting bone development.

[Drug interactions]

Deflazacort is metabolized by hepatic CYP3A4 and may interact with other drugs that affect the activity of this enzyme. For example, CYP3A4 inhibitors such as ketoconazole and clarithromycin can increase the plasma concentration of deflazacort, while inducers such as phenytoin and rifampicin can reduce its efficacy. The combined use of alcohol and nonsteroidal anti-inflammatory drugs (NSAIDs) increases the risk of gastric ulcers, so use them together with caution.

【Storage and specifications】

Deflazacort is usually sold in tablet form, with strengths including 6mg, 18mg, 30mg, 36mg, etc. Should be stored in a cool, dry place away from direct sunlight.

Reference: https://www.drugs.com/mtm/deflazacort.html