DEFLAZACORT

Background and Date of Approval

Deflazacort is a synthetic glucocorticoid medication containing deflazacort as the active ingredient. It is used to treat various conditions including inflammation, allergies, autoimmune disorders, and certain cancers. The medication works by suppressing the immune system and reducing inflammation in the body.

Deflazacort received approval from the United States Food and Drug Administration in 2017 for the treatment of Duchenne muscular dystrophy, a genetic disorder characterised by progressive muscle weakness and wasting. In patients with DMD, it is believed to improve muscle strength and delay disease progression. The medication has also received approval in other countries for various indications, including asthma and allergic rhinitis.

Uses

In particular, deflazacort is commonly used to treat Duchenne muscular dystrophy (DMD). It is also used to treat a range of inflammatory and autoimmune conditions. These include asthma, allergies, and inflammatory bowel disease (IBD), such as Crohn's disease and ulcerative colitis. It also treats rheumatoid arthritis, lupus (systemic lupus erythematosus), and certain skin conditions.

How is it administered?

Deflazacort is usually taken orally in the form of tablets. The tablets should be swallowed whole with water or another liquid and not crushed, chewed, or broken. It is generally recommended to take it on an empty stomach, either one hour before or two hours after meals, to help maximize its absorption and effectiveness. Do not take a double dose to make up for a missed one.

Side Effects

Deflazacort, like all medications, can cause side effects. Common side effects may include nausea, vomiting, indigestion, and changes in appetite. Long-term use can also cause more serious side effects, such as osteoporosis, high blood pressure, and increased risk of infection. Other possible side effects include mood changes, difficulty sleeping, and weight gain. It is important to promptly report any unusual or severe side effects to a healthcare provider.

Warnings

Deflazacort may increase the risk of infections due to its effects on the immune system. Long-term use of this medication may suppress the function of the adrenal glands, which can cause a condition called adrenal insufficiency. The symptoms include fatigue, weakness, and low blood pressure. The extended use may also increase the risk of developing cataracts, which is a clouding of the lens in the eye, and the risk of osteoporosis (weak and brittle bone). This medication may increase the risk of gastrointestinal bleeding, particularly in individuals with a history of stomach ulcers or bleeding. Long-term use may affect growth in children. This medication may cause fluid retention, leading to swelling of arms, legs, or abdomen. This can also lead to an increase in blood pressure.

Precautions

It is important to have regular eye exams while taking deflazacort. You must notify your healthcare provider immediately if you experience any signs of infection, such as fever, sore throat, or chills. You should discuss with your healthcare provider the potential risks and benefits of medicine for an extended period. It is important to monitor for signs of fluid retention and to notify your healthcare provider if you experience swelling or weight gain. Monitor the growth of children who are taking this medicine. Tell your doctor immediately if you experience stomach pain, nausea, vomiting, or blood in your stool to avoid further gastrointestinal bleeding complications.

Word of Advice

Deflazacort is a medication requiring healthcare provider supervision. Follow your doctor's dosage and administration instructions precisely, and report any side effects or concerns promptly. Patients with known allergy to deflazacort or any of its components should not take this medication. Deflazacort is not recommended during pregnancy and breastfeeding. Women of reproductive potential should use contraceptives during treatment and for several weeks after completion. Do not receive live or attenuated vaccines whilst undergoing this treatment. Inform your doctor before starting deflazacort if you have high blood pressure, diabetes, weak bones, or problems affecting your eyes, heart, thyroid gland, or blood vessels. Contact your doctor immediately if you experience mood disturbances, severe stomach pain, or allergic reactions after taking this tablet.

References

Sanofi, Electronic Medicines Compendium (EMC), [Revised on Nov 2020] [Accessed on 29th April 2023], https://www.medicines.org.uk/emc/files/pil.6287.pdf PTC Therapeutics, Inc., [Revised on Jun 2021] [Accessed on 29th April 2023], https://www.emflaza.com/prescribing-information.pdf Parente, Deflazacort: therapeutic index, relative potency and equivalent doses versus other corticosteroids, BMC Pharmacology and Toxicology, 2017, 18:1, https://bmcpharmacoltoxicol.biomedcentral.com/track/pdf/10.1186/s40360-016-0111-8.pdf

Frequently Asked Questions

Can deflazacort be taken long-term?

Deflazacort can be taken long-term but may increase the risk of side effects such as osteoporosis, weight gain, and high blood pressure. It is important to discuss the risks and benefits of long-term use with your healthcare provider and to monitor for any potential side effects regularly.

Can I take Deflazacort if I have diabetes?

Deflazacort may increase blood sugar levels. So, caution should be exercised when administering the medication to individuals with diabetes. Your healthcare provider may need to monitor your blood sugar levels more closely while you are taking it.

Can I take Deflazacort if I have tuberculosis?

Deflazacort should be used with caution in patients with active or latent tuberculosis (TB) infection because it may suppress the immune system, which can worsen TB infection. Therefore, it is important to inform your healthcare provider if you have TB or a history of TB before starting this medication. In some cases, your healthcare provider may recommend monitoring for signs and symptoms of TB.

Can I take Deflazacort if I have a kidney problem?

Deflazacort should be used cautiously in patients with kidney problems because it can affect kidney function. Your healthcare provider may need to adjust the dosage or monitor your kidney function more closely if you have kidney problems while taking this medicine.

How can Deflazacort affect my mental health?

Deflazacort, like many other corticosteroids, may cause changes in mood, behavior, or thinking, particularly in individuals taking high doses or having a history of mental health conditions. These changes may include irritability, anxiety, or depression. Sometimes, this medicine may also cause more severe mental health effects, such as psychosis or mania.

Can deflazacort cause weight gain?

Yes, weight gain is a common side effect of deflazacort. It is important to follow a healthy diet and exercise regularly to help manage weight gain while taking it.

Available Brands 5

Written By: Muskan Patel, M.Pharm

Reviewed By: Dr. Vishwa Patel, MBBS