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Updated on April 18, 2025
Congenital adrenal hyperplasia (CAH) is a group of genetic disorders that affect hormone production in the adrenal glands, a pair of walnut-sized glands located on top of the kidneys.
Hormones are chemicals that travel through the blood and regulate many processes throughout the body. When a person has CAH, gene mutations (small sections of DNA) interfere with the adrenal glands’ ability to produce hormones that regulate stress response, sodium and potassium levels, and growth and development.
Different types of CAH can cause different symptoms, and the severity of symptoms can vary from person to person. Some types cause few or no symptoms. Some affect growth, development, and fertility. Other types can cause life-threatening complications, such as severe dehydration and adrenal crisis (very low levels of adrenal hormones that can lead to severely low blood pressure and shock).
Medications that treat CAH
While there is no cure for CAH, the condition is manageable. Treatment will be individualized to a person’s needs, and will focus on managing hormone levels, preventing complications, and addressing any health concerns and needs. People with mild forms of CAH may not require treatment or may only require low doses of medications.
Glucocorticoid (also called corticosteroid) replacement medications are a mainstay of treatment. These medications raise the amount of the hormone cortisol and help reduce androgen production (which will help to promote normal growth and development).
A person may also need therapy with mineralocorticoid replacement medications that replace the hormone aldosterone to balance sodium/potassium levels, as well as therapies to control blood pressure, salt replacement medications, and medications to promote development during puberty. Again, different people will have different treatment needs, and a healthcare team will be your best source of information.
New therapies are under development, with the goal of therapies to reduce excess androgen production and reduce the need for glucocorticoid therapy. A new medication became available in 2024.
Questions when prescribed a new medication
CAH is a lifelong condition and treatment needs will change over a person’s lifetime. This means a person will need to change dosages and/or medications at different times and for a variety of reasons.
One of the most common examples is stress dosing. Stress dosing is an increase in medication dosage during times of physical stress, such as illness, injury, pregnancy, and to prepare for medical procedures like surgery. Stress dosing helps avoid adrenal crisis during times when the body has a greater-than-normal need for adrenal hormones.
It helps to know what to ask when prescribed a new medication or a change in medication.
What to ask when prescribed a new medication:
- What is the name of the medication?
- What dosage will I be prescribed?
- What is the goal of taking this medication?
- How is the medication taken? How often? How long will I be taking this medication?
- How will we know if the medication is working? What happens if this medication does not work?
- What potential side effects can this medication cause? Is there a risk of serious side effects?
- How will I feel while taking this medication?
- Can this medication interact with other medications?
- Is there anything I need to avoid while taking this medication? For example, certain foods or activities?
- What is the out-of-pocket cost of this medication? Who can I talk to if I have concerns about the cost?
It’s also important that your healthcare providers know about all the medications that you are taking, including prescription medications (for CAH and any other illnesses or conditions), any over-the-counter medications, and any supplements.
