Emflaza 22.75mg/ml Suspension
It is used to treat Duchenne muscular dystrophy (DMD).
Drug Class
Corticosteroid
Pharmacologic Class
Glucocorticoid receptor agonist
Pregnancy Category
Not available
FDA Approved
Feb 2017
DEA Schedule
Not Controlled
Overview
What is this medicine?
Emflaza is a medicine called a corticosteroid, similar to natural hormones your body makes. It's used to treat Duchenne muscular dystrophy (DMD) in patients 2 years and older. It works by reducing inflammation and slowing down the damage to muscles, which can help improve muscle strength and function in people with DMD.
How to Use This Medicine
Taking Your Medication Correctly
To ensure you get the most benefit from your medication, follow these steps:
1. Follow Your Doctor's Orders: Take this medication exactly as directed by your doctor. Read all the information provided with your medication and follow the instructions carefully.
2. Administration: You can take this medication with or without food.
3. Preparing the Bottle: If the adapter is not already attached to the bottle, prepare it according to the instructions you received or as described in the package insert.
4. Measuring Your Dose: Shake the bottle well before use. Measure the liquid dose carefully using the measuring device provided with the medication. If no device is included, ask your pharmacist for one.
5. Mixing with Juice or Milk: Slowly add the measured dose to 3 to 4 ounces (90 to 120 mL) of juice or milk. Mix well, but do not use grapefruit juice.
6. Taking Your Dose: Take your dose immediately after mixing. Do not store the mixture for later use.
7. Continuing Your Medication: Continue taking this medication as directed by your doctor or healthcare provider, even if you start feeling well.
Storing and Disposing of Your Medication
1. Storage: Store the medication at room temperature in a dry place, away from the bathroom.
2. Disposal: After opening, discard any unused medication after 1 month.
3. Safety Precautions: Keep all medications in a safe place, out of the reach of children and pets.
What to Do If You Miss a Dose
1. Taking a Missed Dose: If you miss a dose, take it as soon as you remember.
2. Skipping a Missed Dose: If it's close to the time for your next dose, skip the missed dose and return to your regular schedule.
3. Avoiding Extra Doses: Do not take two doses at the same time or take extra doses.
To ensure you get the most benefit from your medication, follow these steps:
1. Follow Your Doctor's Orders: Take this medication exactly as directed by your doctor. Read all the information provided with your medication and follow the instructions carefully.
2. Administration: You can take this medication with or without food.
3. Preparing the Bottle: If the adapter is not already attached to the bottle, prepare it according to the instructions you received or as described in the package insert.
4. Measuring Your Dose: Shake the bottle well before use. Measure the liquid dose carefully using the measuring device provided with the medication. If no device is included, ask your pharmacist for one.
5. Mixing with Juice or Milk: Slowly add the measured dose to 3 to 4 ounces (90 to 120 mL) of juice or milk. Mix well, but do not use grapefruit juice.
6. Taking Your Dose: Take your dose immediately after mixing. Do not store the mixture for later use.
7. Continuing Your Medication: Continue taking this medication as directed by your doctor or healthcare provider, even if you start feeling well.
Storing and Disposing of Your Medication
1. Storage: Store the medication at room temperature in a dry place, away from the bathroom.
2. Disposal: After opening, discard any unused medication after 1 month.
3. Safety Precautions: Keep all medications in a safe place, out of the reach of children and pets.
What to Do If You Miss a Dose
1. Taking a Missed Dose: If you miss a dose, take it as soon as you remember.
2. Skipping a Missed Dose: If it's close to the time for your next dose, skip the missed dose and return to your regular schedule.
3. Avoiding Extra Doses: Do not take two doses at the same time or take extra doses.
Lifestyle & Tips
- Take Emflaza exactly as prescribed, usually once daily. Do not stop taking it suddenly without talking to your doctor, as this can cause serious withdrawal symptoms.
- Avoid exposure to people with infections, especially chickenpox or measles, as Emflaza weakens your immune system.
- Report any signs of infection (fever, chills, sore throat) to your doctor immediately.
- Monitor blood sugar levels if advised by your doctor, especially if you have diabetes or are at risk.
- Maintain good bone health through adequate calcium and vitamin D intake, and regular weight-bearing exercise if appropriate.
- Attend all scheduled doctor appointments and monitoring tests (blood pressure, blood sugar, eye exams, bone density scans).
- Discuss any new medications, including over-the-counter drugs, supplements, or herbal products, with your doctor or pharmacist.
- Carry a steroid emergency card or medical alert identification indicating you are taking a corticosteroid.
Available Forms & Alternatives
Dosing & Administration
Adult Dosing
Standard Dose:
0.9 mg/kg once daily
Condition-Specific Dosing:
Duchenne Muscular Dystrophy:
0.9 mg/kg once daily. Dose should be rounded to the nearest 0.1 mg/kg. Administer with or without food.
Pediatric Dosing
Neonatal:
Not established
Infant:
Not established (approved for patients 2 years of age and older)
Child:
0.9 mg/kg once daily (for patients 2 years and older with Duchenne Muscular Dystrophy)
Adolescent:
0.9 mg/kg once daily (for patients with Duchenne Muscular Dystrophy)
Dose Adjustments
Renal Impairment:
Mild:
No dose adjustment needed
Moderate:
No dose adjustment needed
Severe:
No dose adjustment needed
Dialysis:
No specific recommendations; deflazacort is not significantly removed by dialysis.
Hepatic Impairment:
Mild:
Reduce dose by approximately 1/3 (e.g., if standard dose is 0.9 mg/kg, reduce to 0.6 mg/kg)
Moderate:
Reduce dose by approximately 1/3 (e.g., if standard dose is 0.9 mg/kg, reduce to 0.6 mg/kg)
Severe:
Not studied; avoid use in patients with severe hepatic impairment.
Pharmacology
Mechanism of Action
Deflazacort is a corticosteroid that acts as a glucocorticoid receptor agonist. It exerts anti-inflammatory and immunosuppressive effects by binding to glucocorticoid receptors, modulating gene expression, and inhibiting the release of inflammatory mediators. In Duchenne muscular dystrophy, it is thought to reduce inflammation and slow the progression of muscle damage.
Pharmacokinetics
Absorption:
Bioavailability:
Not explicitly quantified, but rapidly absorbed.
Tmax:
0.5 to 2 hours (for active metabolite 21-desacetyl deflazacort)
FoodEffect:
Food decreases Cmax and AUC of the active metabolite by approximately 30-35% and 15% respectively, but this is not considered clinically significant. Can be taken with or without food.
Distribution:
Vd:
Not readily available
ProteinBinding:
Approximately 40% (for active metabolite)
CnssPenetration:
Limited, but corticosteroids can cross the blood-brain barrier and affect the central nervous system.
Elimination:
HalfLife:
1.7 to 2.7 hours (for active metabolite)
Clearance:
Not readily available
ExcretionRoute:
Primarily renal (approximately 70%) and fecal (approximately 30%)
Unchanged:
Negligible
Pharmacodynamics
OnsetOfAction:
Variable, depends on the specific effect being measured (e.g., anti-inflammatory effects may be seen within hours to days).
PeakEffect:
Variable, depends on the specific effect.
DurationOfAction:
Once-daily dosing suggests a prolonged duration of action sufficient for daily administration.
Safety & Warnings
Side Effects
Urgent Side Effects: Seek Medical Help Right Away
Although rare, some people may experience severe and potentially life-threatening side effects while taking this medication. If you notice any of the following symptoms, contact your doctor immediately or seek emergency medical attention:
Signs of an allergic reaction: rash, hives, itching, red, swollen, blistered, or peeling skin (with or without fever), wheezing, tightness in the chest or throat, difficulty breathing, swallowing, or talking, unusual hoarseness, or swelling of the mouth, face, lips, tongue, or throat.
Signs of infection: fever, chills, severe sore throat, ear or sinus pain, cough, increased or discolored sputum, painful urination, mouth sores, or a wound that won't heal.
Signs of high blood sugar: confusion, drowsiness, excessive thirst or hunger, frequent urination, flushing, rapid breathing, or fruity-smelling breath.
Signs of Cushing's syndrome: weight gain in the upper back or abdomen, moon face, severe headache, or slow wound healing.
Signs of adrenal gland problems: severe nausea or vomiting, extreme dizziness or fainting, muscle weakness, fatigue, mood changes, decreased appetite, or weight loss.
Signs of high blood pressure: severe headache or dizziness, fainting, or changes in vision.
Signs of electrolyte imbalance: mood changes, confusion, muscle pain, cramps, or spasms, weakness, shakiness, balance problems, abnormal heartbeat, seizures, loss of appetite, or severe nausea or vomiting.
Shortness of breath, sudden weight gain, or swelling in the arms or legs.
Skin changes: acne, stretch marks, slow healing, or excessive hair growth.
Changes in vision.
Bone or joint pain.
Black, tarry, or bloody stools.
Vomiting blood or coffee ground-like material.
New or worsening mental, mood, or behavioral changes.
Purple, red, blue, brown, or black bumps or patches on the skin or in the mouth.
Signs of a blood clot: chest pain or pressure, coughing up blood, shortness of breath, swelling, warmth, numbness, color changes, or pain in a leg or arm, or difficulty speaking or swallowing.
A severe skin reaction (toxic epidermal necrolysis) may occur, causing serious health problems or even death. Seek immediate medical attention if you experience: red, swollen, blistered, or peeling skin (with or without fever), red or irritated eyes, or sores in your mouth, throat, nose, or eyes.
Other Possible Side Effects
Like all medications, this drug can cause side effects. Many people experience no side effects or only mild ones. If you're concerned about any of the following side effects or if they persist, contact your doctor:
Weight gain.
Increased appetite.
Common cold symptoms.
Nose and throat irritation.
Frequent urination.
Stomach pain.
Constipation.
Back pain.
Upset stomach.
* Headache.
This is not an exhaustive list of possible side effects. If you have questions or concerns, consult your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch.
Although rare, some people may experience severe and potentially life-threatening side effects while taking this medication. If you notice any of the following symptoms, contact your doctor immediately or seek emergency medical attention:
Signs of an allergic reaction: rash, hives, itching, red, swollen, blistered, or peeling skin (with or without fever), wheezing, tightness in the chest or throat, difficulty breathing, swallowing, or talking, unusual hoarseness, or swelling of the mouth, face, lips, tongue, or throat.
Signs of infection: fever, chills, severe sore throat, ear or sinus pain, cough, increased or discolored sputum, painful urination, mouth sores, or a wound that won't heal.
Signs of high blood sugar: confusion, drowsiness, excessive thirst or hunger, frequent urination, flushing, rapid breathing, or fruity-smelling breath.
Signs of Cushing's syndrome: weight gain in the upper back or abdomen, moon face, severe headache, or slow wound healing.
Signs of adrenal gland problems: severe nausea or vomiting, extreme dizziness or fainting, muscle weakness, fatigue, mood changes, decreased appetite, or weight loss.
Signs of high blood pressure: severe headache or dizziness, fainting, or changes in vision.
Signs of electrolyte imbalance: mood changes, confusion, muscle pain, cramps, or spasms, weakness, shakiness, balance problems, abnormal heartbeat, seizures, loss of appetite, or severe nausea or vomiting.
Shortness of breath, sudden weight gain, or swelling in the arms or legs.
Skin changes: acne, stretch marks, slow healing, or excessive hair growth.
Changes in vision.
Bone or joint pain.
Black, tarry, or bloody stools.
Vomiting blood or coffee ground-like material.
New or worsening mental, mood, or behavioral changes.
Purple, red, blue, brown, or black bumps or patches on the skin or in the mouth.
Signs of a blood clot: chest pain or pressure, coughing up blood, shortness of breath, swelling, warmth, numbness, color changes, or pain in a leg or arm, or difficulty speaking or swallowing.
A severe skin reaction (toxic epidermal necrolysis) may occur, causing serious health problems or even death. Seek immediate medical attention if you experience: red, swollen, blistered, or peeling skin (with or without fever), red or irritated eyes, or sores in your mouth, throat, nose, or eyes.
Other Possible Side Effects
Like all medications, this drug can cause side effects. Many people experience no side effects or only mild ones. If you're concerned about any of the following side effects or if they persist, contact your doctor:
Weight gain.
Increased appetite.
Common cold symptoms.
Nose and throat irritation.
Frequent urination.
Stomach pain.
Constipation.
Back pain.
Upset stomach.
* Headache.
This is not an exhaustive list of possible side effects. If you have questions or concerns, consult your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch.
Seek Immediate Medical Attention If You Experience:
- Signs of infection (fever, chills, body aches, cough, sore throat, unusual fatigue, skin redness/swelling)
- High blood sugar (increased thirst, frequent urination, increased hunger, blurred vision)
- High blood pressure (severe headache, dizziness, blurred vision)
- Swelling in hands, ankles, or feet
- Unusual weight gain or changes in body shape (e.g., moon face, buffalo hump)
- Mood changes (irritability, anxiety, depression, euphoria, difficulty sleeping)
- Severe stomach pain, black or tarry stools, or vomiting blood (signs of GI bleeding)
- Muscle weakness or pain that worsens
- Vision changes (blurred vision, eye pain, halos around lights)
- Signs of adrenal insufficiency if dose is missed or stopped abruptly (severe fatigue, weakness, nausea, vomiting, dizziness, low blood pressure)
Before Using This Medicine
Before Taking This Medication: Important Information to Share with Your Doctor
It is essential to inform your doctor about the following conditions to ensure safe treatment:
Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Describe the allergic reaction you experienced, including the symptoms that occurred.
The presence of any infection, including bacterial, viral, or fungal infections, as well as specific conditions like:
+ Amoeba infection (e.g., traveler's diarrhea)
+ Herpes infection of the eye
+ Malaria infection in the brain
+ Threadworm infestation
+ Other types of infections
Certain health problems, such as:
+ Diverticulitis
+ Hole in the gastrointestinal (GI) tract
+ Stomach or bowel infection
+ Ulcers
Recent stomach or bowel surgery
* Any medications you are currently taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins, as some may interact with this medication. Specifically, inform your doctor about:
+ Certain drugs used to treat HIV, infections, seizures, and other conditions
+ Other medications that may have adverse interactions with this drug
Please note that this is not an exhaustive list of all potential interactions. To ensure your safety, it is crucial to discuss all your medications and health problems with your doctor and pharmacist. Before starting, stopping, or changing the dose of any medication, consult with your doctor to confirm that it is safe to do so.
It is essential to inform your doctor about the following conditions to ensure safe treatment:
Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Describe the allergic reaction you experienced, including the symptoms that occurred.
The presence of any infection, including bacterial, viral, or fungal infections, as well as specific conditions like:
+ Amoeba infection (e.g., traveler's diarrhea)
+ Herpes infection of the eye
+ Malaria infection in the brain
+ Threadworm infestation
+ Other types of infections
Certain health problems, such as:
+ Diverticulitis
+ Hole in the gastrointestinal (GI) tract
+ Stomach or bowel infection
+ Ulcers
Recent stomach or bowel surgery
* Any medications you are currently taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins, as some may interact with this medication. Specifically, inform your doctor about:
+ Certain drugs used to treat HIV, infections, seizures, and other conditions
+ Other medications that may have adverse interactions with this drug
Please note that this is not an exhaustive list of all potential interactions. To ensure your safety, it is crucial to discuss all your medications and health problems with your doctor and pharmacist. Before starting, stopping, or changing the dose of any medication, consult with your doctor to confirm that it is safe to do so.
Precautions & Cautions
Important Warnings and Cautions
It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication. Regular blood tests, as directed by your doctor, are crucial to monitor your health. Additionally, if you are taking this medication long-term, you may need to undergo eye pressure and bone density checks.
Long-term Use Risks
Prolonged use of this medication may increase the risk of developing cataracts or glaucoma. Discuss this potential risk with your doctor. Furthermore, long-term use may cause weak bones (osteoporosis). Consult your doctor to determine if you are at a higher risk of osteoporosis or if you have any concerns.
Blood Pressure and Sugar Monitoring
This medication may cause high blood pressure. Regular blood pressure checks, as advised by your doctor, are necessary. If you have diabetes, it is crucial to closely monitor your blood sugar levels. You may need to reduce your salt intake and take extra potassium supplements; consult your doctor for guidance.
Dietary Restrictions and Vaccine Precautions
Avoid consuming grapefruit and grapefruit juice while taking this medication. If you are not up to date with all recommended vaccines, discuss your vaccination status with your doctor. You may need to receive certain vaccines before starting treatment with this medication. However, some vaccines may not be effective or may increase the risk of infection when taken with this medication. Consult your doctor before receiving any vaccines.
Infection Risks
Steroid medications, including this one, can increase the risk of infection. They can also worsen existing infections and make it more challenging to detect signs of infection. Infections can range from mild to severe and potentially life-threatening. The risk of infection is typically higher with higher doses of steroids. To minimize this risk, wash your hands frequently, avoid close contact with people who have infections, colds, or flu, and inform your doctor if you experience any signs of infection.
Reactivation of Infections
In some cases, this medication may reactivate latent infections, such as tuberculosis or hepatitis B. Inform your doctor if you have a history of these infections.
Adrenal Insufficiency and Stress
This medication may decrease the natural production of steroids in your body. If you experience a fever, infection, surgery, or injury, consult your doctor, as you may require additional oral steroid doses to help your body cope with stress. Carry a warning card indicating that you may need extra steroids in certain situations.
Rare but Serious Risks
Long-term use of this medication may lead to rare but potentially life-threatening conditions, including changes in adrenal gland function and the development of a tumor on the adrenal gland (pheochromocytoma). Discuss these risks with your doctor.
Blood Clots and Cancer Risks
This medication may increase the risk of blood clots. Inform your doctor if you have a history of blood clots. Additionally, long-term use of this medication may increase the risk of a type of cancer called Kaposi's sarcoma. Discuss this risk with your doctor.
Discontinuation and Missed Doses
Do not stop taking this medication abruptly without consulting your doctor, as this may increase the risk of side effects. If you need to discontinue this medication, your doctor will guide you on how to gradually stop taking it. If you miss a dose or recently stopped taking this medication and experience fatigue, weakness, shakiness, rapid heartbeat, confusion, sweating, or dizziness, inform your doctor.
Pediatric Use and Growth Monitoring
This medication may affect growth in children and teenagers. Regular growth checks may be necessary; consult your doctor for guidance.
Benzyl Alcohol Warning
This medication contains benzyl alcohol, which can cause serious side effects in newborns and infants. If possible, avoid using products containing benzyl alcohol in these age groups. If you have concerns, discuss them with your doctor.
Pregnancy and Breastfeeding
Inform your doctor if you are pregnant, plan to become pregnant, or are breastfeeding. You will need to discuss the benefits and risks of this medication to you and your baby.
It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication. Regular blood tests, as directed by your doctor, are crucial to monitor your health. Additionally, if you are taking this medication long-term, you may need to undergo eye pressure and bone density checks.
Long-term Use Risks
Prolonged use of this medication may increase the risk of developing cataracts or glaucoma. Discuss this potential risk with your doctor. Furthermore, long-term use may cause weak bones (osteoporosis). Consult your doctor to determine if you are at a higher risk of osteoporosis or if you have any concerns.
Blood Pressure and Sugar Monitoring
This medication may cause high blood pressure. Regular blood pressure checks, as advised by your doctor, are necessary. If you have diabetes, it is crucial to closely monitor your blood sugar levels. You may need to reduce your salt intake and take extra potassium supplements; consult your doctor for guidance.
Dietary Restrictions and Vaccine Precautions
Avoid consuming grapefruit and grapefruit juice while taking this medication. If you are not up to date with all recommended vaccines, discuss your vaccination status with your doctor. You may need to receive certain vaccines before starting treatment with this medication. However, some vaccines may not be effective or may increase the risk of infection when taken with this medication. Consult your doctor before receiving any vaccines.
Infection Risks
Steroid medications, including this one, can increase the risk of infection. They can also worsen existing infections and make it more challenging to detect signs of infection. Infections can range from mild to severe and potentially life-threatening. The risk of infection is typically higher with higher doses of steroids. To minimize this risk, wash your hands frequently, avoid close contact with people who have infections, colds, or flu, and inform your doctor if you experience any signs of infection.
Reactivation of Infections
In some cases, this medication may reactivate latent infections, such as tuberculosis or hepatitis B. Inform your doctor if you have a history of these infections.
Adrenal Insufficiency and Stress
This medication may decrease the natural production of steroids in your body. If you experience a fever, infection, surgery, or injury, consult your doctor, as you may require additional oral steroid doses to help your body cope with stress. Carry a warning card indicating that you may need extra steroids in certain situations.
Rare but Serious Risks
Long-term use of this medication may lead to rare but potentially life-threatening conditions, including changes in adrenal gland function and the development of a tumor on the adrenal gland (pheochromocytoma). Discuss these risks with your doctor.
Blood Clots and Cancer Risks
This medication may increase the risk of blood clots. Inform your doctor if you have a history of blood clots. Additionally, long-term use of this medication may increase the risk of a type of cancer called Kaposi's sarcoma. Discuss this risk with your doctor.
Discontinuation and Missed Doses
Do not stop taking this medication abruptly without consulting your doctor, as this may increase the risk of side effects. If you need to discontinue this medication, your doctor will guide you on how to gradually stop taking it. If you miss a dose or recently stopped taking this medication and experience fatigue, weakness, shakiness, rapid heartbeat, confusion, sweating, or dizziness, inform your doctor.
Pediatric Use and Growth Monitoring
This medication may affect growth in children and teenagers. Regular growth checks may be necessary; consult your doctor for guidance.
Benzyl Alcohol Warning
This medication contains benzyl alcohol, which can cause serious side effects in newborns and infants. If possible, avoid using products containing benzyl alcohol in these age groups. If you have concerns, discuss them with your doctor.
Pregnancy and Breastfeeding
Inform your doctor if you are pregnant, plan to become pregnant, or are breastfeeding. You will need to discuss the benefits and risks of this medication to you and your baby.
Overdose Information
Overdose Symptoms:
- Exaggerated side effects of corticosteroids, such as severe fluid retention, electrolyte imbalances (e.g., hypokalemia), hyperglycemia, hypertension, and signs of Cushing's syndrome (e.g., moon face, central obesity, muscle weakness).
What to Do:
There is no specific antidote for deflazacort overdose. Management is supportive and symptomatic. Contact a poison control center immediately (Call 1-800-222-1222 in the US) or seek emergency medical attention.
Drug Interactions
Contraindicated Interactions
- Live or live attenuated vaccines (due to immunosuppression)
Major Interactions
- Strong CYP3A4 inhibitors (e.g., ketoconazole, ritonavir, clarithromycin) - may significantly increase deflazacort exposure, requiring dose reduction.
- Strong CYP3A4 inducers (e.g., rifampin, phenytoin, carbamazepine, phenobarbital) - may significantly decrease deflazacort exposure, reducing efficacy.
- Anticoagulants (e.g., warfarin) - may alter anticoagulant effects (increase or decrease), requiring close INR monitoring.
- NSAIDs (Nonsteroidal Anti-inflammatory Drugs) - increased risk of gastrointestinal ulceration and bleeding.
Moderate Interactions
- Diuretics (e.g., thiazide, loop diuretics) - increased risk of hypokalemia.
- Antidiabetic agents (e.g., insulin, oral hypoglycemics) - deflazacort may increase blood glucose, requiring adjustment of antidiabetic therapy.
- Cardiac glycosides (e.g., digoxin) - increased risk of toxicity due to hypokalemia.
- Cholestyramine - may decrease deflazacort absorption.
- Oral contraceptives - may increase deflazacort levels.
Minor Interactions
- Not readily available for specific minor interactions, but general corticosteroid interactions apply.
Monitoring
Baseline Monitoring
Blood pressure
Rationale: Risk of hypertension
Timing: Prior to initiation
Blood glucose
Rationale: Risk of hyperglycemia/diabetes
Timing: Prior to initiation
Serum electrolytes (especially potassium)
Rationale: Risk of hypokalemia
Timing: Prior to initiation
Bone mineral density (BMD)
Rationale: Risk of osteoporosis
Timing: Prior to initiation (DEXA scan)
Ophthalmologic exam (for cataracts/glaucoma)
Rationale: Risk of ocular adverse effects
Timing: Prior to initiation
Growth (height and weight)
Rationale: Risk of growth suppression in pediatric patients
Timing: Prior to initiation
Infection screening
Rationale: Immunosuppression increases infection risk
Timing: Prior to initiation
Routine Monitoring
Blood pressure
Frequency: Regularly (e.g., monthly or as clinically indicated)
Target: Age-appropriate normal range
Action Threshold: Sustained elevation requiring intervention
Blood glucose
Frequency: Regularly (e.g., every 3-6 months or as clinically indicated), more frequently if diabetic
Target: Fasting glucose <100 mg/dL, HbA1c <6.5%
Action Threshold: Persistent hyperglycemia requiring intervention
Serum electrolytes (especially potassium)
Frequency: Periodically (e.g., every 3-6 months or as clinically indicated)
Target: Potassium 3.5-5.0 mEq/L
Action Threshold: Hypokalemia requiring supplementation
Growth (height and weight)
Frequency: Regularly (e.g., every 3-6 months in pediatric patients)
Target: Normal growth velocity for age
Action Threshold: Significant growth deceleration
Ophthalmologic exam
Frequency: Annually or as clinically indicated
Target: Normal intraocular pressure, no cataracts
Action Threshold: Development of cataracts or glaucoma
Bone mineral density (BMD)
Frequency: Periodically (e.g., every 1-2 years) or as clinically indicated
Target: Stable BMD
Action Threshold: Significant bone loss or fractures
Signs/symptoms of infection
Frequency: Ongoing
Target: Absence of infection
Action Threshold: Fever, malaise, localized infection signs
Adrenal function (if discontinuing or tapering)
Frequency: As needed
Target: Normal adrenal response
Action Threshold: Signs of adrenal insufficiency
Symptom Monitoring
- Signs of infection (fever, chills, sore throat, cough, unusual fatigue, localized pain/redness)
- Signs of hyperglycemia (increased thirst, increased urination, increased hunger, blurred vision)
- Signs of hypertension (headache, dizziness, blurred vision)
- Signs of adrenal insufficiency (fatigue, weakness, nausea, vomiting, dizziness, low blood pressure)
- Cushingoid features (moon face, buffalo hump, central obesity, striae)
- Mood changes (irritability, anxiety, depression, euphoria)
- Gastrointestinal upset (stomach pain, nausea, vomiting, black/tarry stools)
- Muscle weakness or pain
- Bone pain or fractures
- Vision changes (blurred vision, eye pain)
- Swelling (edema)
Special Patient Groups
Pregnancy
Use during pregnancy should only be considered if the potential benefit justifies the potential risk to the fetus. Corticosteroids can cross the placenta and may be associated with adverse outcomes such as low birth weight, and potential for fetal adrenal suppression. Monitor neonates exposed to corticosteroids in utero for signs of hypoadrenalism.
Trimester-Specific Risks:
First Trimester:
Potential for increased risk of cleft palate and other malformations, though data are conflicting and generally low risk.
Second Trimester:
Risk of fetal growth restriction and potential for adrenal suppression.
Third Trimester:
Risk of fetal growth restriction and potential for adrenal suppression; monitor neonate for signs of hypoadrenalism.
Lactation
Deflazacort and its active metabolite are excreted into human milk. The amount is generally low, but potential for adverse effects on the breastfed infant (e.g., growth suppression, interference with endogenous corticosteroid production) exists. The decision to breastfeed should consider the importance of the drug to the mother and the potential risks to the infant. Monitor breastfed infants for signs of adrenal suppression.
Infant Risk:
L3 (Moderate risk) - Monitor infant for adverse effects such as growth suppression, weight gain, and signs of adrenal suppression.
Pediatric Use
Approved for patients 2 years of age and older. Children are at increased risk of growth suppression, osteoporosis, and other corticosteroid-related adverse effects. Close monitoring of growth, bone mineral density, and other parameters is essential.
Geriatric Use
No specific dose adjustment is required based on age alone. However, geriatric patients may be at increased risk for common corticosteroid adverse effects such as osteoporosis, diabetes, hypertension, and cataracts. Use with caution and monitor closely.
Clinical Information
Clinical Pearls
- Emflaza is a corticosteroid specifically approved for Duchenne muscular dystrophy (DMD) in patients 2 years and older. It is NOT interchangeable with other corticosteroids like prednisone without careful dose conversion and clinical consideration.
- Administer once daily. The oral suspension can be given directly or mixed with apple juice or water. Do not mix with grapefruit juice.
- Patients should not abruptly discontinue Emflaza due to the risk of adrenal insufficiency. Tapering is required.
- Immunosuppression is a significant risk; educate patients/caregivers on signs of infection and avoidance of sick contacts. Live vaccines are contraindicated.
- Monitor for common corticosteroid side effects including hyperglycemia, hypertension, bone loss, cataracts, and behavioral changes.
- Due to its high cost, patient assistance programs and insurance coverage verification are crucial.
Alternative Therapies
- Eteplirsen (Exondys 51)
- Golodirsen (Vyondys 53)
- Viltolarsen (Viltepso)
- Casimersen (Amondys 45)
- Ataluren (Translarna - approved in some regions outside US)
- Gene therapies (e.g., Elevidys - delandistrogene moxeparvovec)
Cost & Coverage
Average Cost:
Highly variable, typically >$50,000 - $100,000+ per year
Generic Available:
Yes
Insurance Coverage:
Specialty Tier (Tier 4 or higher), often requires prior authorization and may have high co-pays/co-insurance.
General Drug Facts
If your symptoms or health issues persist or worsen, it's essential to contact your doctor for further guidance. To ensure your safety, never share your medication with others or take someone else's medication. Some medications may come with an additional patient information leaflet, so be sure to check with your pharmacist for more details. If you have any questions or concerns about your medication, don't hesitate to reach out to your doctor, nurse, pharmacist, or other healthcare provider. In the event of a suspected overdose, immediately call your local poison control center or seek medical attention. When seeking help, be prepared to provide information about the medication taken, the amount, and the time it happened.