Off-Label Drug Use
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Questions and Answers

What is the title of the Clinical Practice Guideline for the Treatment of Coccidioidomycosis mentioned in the content?

Infectious Diseases Society of America (IDSA) Clinical Practice Guideline

Which type of infections are discussed in the article by Spampinato and Leonardi?

  • Candida infections (correct)
  • Bacterial infections
  • Protozoal infections
  • Viral infections
  • The article by Spampinato and Leonardi mainly discusses traditional antifungal agents.

    False

    What does off-label prescribing refer to?

    <p>Both a and b</p> Signup and view all the answers

    What is one of the off-label uses of Metformin?

    <p>Weight loss</p> Signup and view all the answers

    Lactulose is sometimes used off-label to reduce blood ammonia levels in patients with hepatic ______________.

    <p>encephalopathy</p> Signup and view all the answers

    Match the drug with its off-label use:

    <p>Salbutamol = Second line management in the treatment of hyperkalemia Domperidone = Increasing breast milk supply Viagra (sildenafil) = Treating high-altitude pulmonary edema Aspirin = Preeclampsia</p> Signup and view all the answers

    Warfarin is used off-label to treat certain types of cancer.

    <p>False</p> Signup and view all the answers

    Study Notes

    Off-Label Clinical Use of Drugs

    • The FDA approves drugs for specific health conditions, but doctors often prescribe them for off-label use, which accounts for around 20% of all prescriptions in the US.
    • Off-label use can be safe and effective, but it also carries certain risks.

    Dexamethasone

    • Used off-label during pregnancy to treat congenital adrenal hyperplasia (CAH) in female babies.
    • Accelerates fetal lung maturation, decreases respiratory distress syndrome, and improves survival in preterm neonates.

    Thiazide

    • Used off-label to treat osteoporosis.

    Metformin

    • Used off-label for weight loss and breast cancer treatment.

    Silymarin

    • Used off-label as a liver supplement and iron chelating agent in thalassemia.
    • Decreases oxidative stress and frequency of blood transfusions.

    Salbutamol

    • On-label use: Relieves symptoms of asthma and chronic obstructive pulmonary disease (COPD).
    • Off-label use: Second-line management in treating hyperkalemia.
    • Mechanism of action: Reduces serum potassium levels by increasing the shift of extracellular potassium into the intracellular space.

    Lactulose

    • On-label use: Treats constipation.
    • Off-label use: Treats hepatic coma.
    • Mechanism of action:
      • On-label: Not absorbed in the small intestine, broken down in the colon, and increases fecal bulk, leading to bowel movements.
      • Off-label: Acidifies colonic contents, converting ammonia to its ionized form, which is less readily absorbed, reducing blood ammonia levels and improving neurological symptoms.

    Domperidone (Motilium)

    • On-label use: Treats nausea, vomiting, indigestion, and gastric reflux.
    • Off-label use: Increases milk supply in lactating women.
    • Mechanism of action: Stimulates gastric muscle contraction, releasing prolactin, which stimulates breast milk production.

    Colchicine

    • On-label use: Treats and prevents gout.
    • Off-label use: Treats acute pericarditis, preventing recurrent episodes, and other conditions like actinic keratosis, amyloidosis, Peyronie's disease, and psoriasis.
    • Mechanism of action: Inhibits microtubule self-assembly, decreasing leucocyte motility and phagocytosis.

    Spironolactone

    • On-label use: Treats heart failure, hypertension, and hypokalemia.
    • Off-label use: Treats acne vulgaris and hirsutism in female patients.
    • Mechanism of action: Affects androgen receptors in the sebaceous glands, reducing sebum production, and blocks the conversion of weaker androgens to more potent androgens in peripheral tissues.

    Viagra (Sildenafil)

    • On-label use: Treats erectile dysfunction.
    • Off-label use: Treats high-altitude pulmonary edema (HAPE).
    • Mechanism of action: Dilates blood vessels in the lungs, improving oxygenation.

    Warfarin

    • On-label use: Treats and prevents hypercholesteremia, atherosclerosis, and reduces the risk of heart disease and stroke.
    • Off-label use: Treats certain types of cancer.

    Azathioprine

    • On-label use: Prevents kidney transplant rejection.
    • Off-label use: Treats inflammatory bowel disease, active rheumatoid arthritis, multiple sclerosis, myasthenia gravis, and autoimmune hepatitis.
    • Mechanism of action: Inhibits purine synthesis, disrupting DNA function and triggering cell death.

    Statin

    • On-label use: Treats and prevents hypercholesteremia, atherosclerosis, and reduces the risk of heart disease and stroke.
    • Off-label use: Treats certain types of cancer, neurological diseases like Alzheimer's and Parkinson's, and autoimmune diseases like HIV and tuberculosis.

    Propranolol

    • On-label use: Treats hypertension, angina, arrhythmias, and anxiety.
    • Off-label use: Treats essential tremor, ventricular fibrillation, and pulseless ventricular tachycardia.

    Semaglutide (Wegovy)

    • On-label use: Treats type 2 diabetes.
    • Off-label use: Treats obesity.
    • Mechanism of action: Increases insulin secretion and reduces glucagon secretion, mimicking the effects of glucagon-like peptide-1.

    Adrenaline

    • On-label use: Treats type 1 hypersensitivity reactions, including anaphylaxis, acute asthmatic attacks, and symptomatic bradycardia.
    • Off-label use: Treats ventricular fibrillation, pulseless ventricular tachycardia, cardiac arrest, and pulseless electrical activity.

    Aspirin (Antiplatlets)

    • On-label use: Treats pain, fever, rheumatic disease, coronary syndrome, cerebrovascular disease, and angina.
    • Off-label use: Treats preeclampsia in gynecology and obstetrics outpatients.
    • Mechanism of action: Inhibits the activity of cyclooxygenase, leading to the formation of prostaglandins that cause inflammation, swelling, pain, and fever.

    Quetiapine (Atrolak, Sondate)

    • On-label use: Treats schizophrenia, bipolar disorder, and major depressive disorder.
    • Off-label use: Treats insomnia.
    • Mechanism of action: Antagonizes the D2 receptor in the mesolimbic pathway, relieving positive symptoms, and antagonizes 5HT2A receptors, relieving negative symptoms of schizophrenia.

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    Learn about the FDA approval process and the common practice of off-label prescribing in the US. Discover the risks and benefits of taking a drug for an off-label use.

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