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Congenital Adrenal Hyperplasia Treatment Options
If you have classic CAH or nonclassic CAH with symptoms, medications are available to help fix your body’s imbalance of hormone levels — namely, a lack of cortisol and aldosterone and excess androgen. People who have nonclassic CAH without symptoms won’t need treatment.
Mineralocorticoids
Fludrocortisone (Florinef Acetate) is a steroid medication that replaces the hormone aldosterone. This helps boost development and growth, counter sodium loss, and prevent adrenal crises (a life-threatening emergency that occurs when cortisol and aldosterone levels are too low).
Glucocorticoids
Glucocorticoids (aka corticosteroids or simply steroids) replace the hormone cortisol. This helps manage adrenal insufficiency, control androgen levels, and aid in development and growth. Options include:
- dexamethasone
- hydrocortisone
- methylprednisolone
- prednisolone
- prednisone
- Increased appetite and weight gain
- Mood changes
- More body hair
- Muscle weakness
- Risk of infection
- Signs of high blood sugar (i.e., feeling hungry or thirsty or urinating more frequently than normal)
- Skin changes (including acne)
- Stomach irritation
In children, glucocorticoids may also cause growth delays, says Phyllis Speiser, MD, a pediatric endocrinologist at Cohen Children’s Medical Center of New York and an associate investigator at the Feinstein Institutes for Medical Research at Northwell Health in Manhasset, New York.
Because of these side effects, “We usually recommend the least long-acting or least potent glucocorticoid, which is hydrocortisone, for daily maintenance,” says Dr. Speiser. “For children, we are especially vigilant about maintaining the lowest possible dose to achieve effective adrenal control.”
The other drugs are used “for situations in which a patient doesn’t respond as expected to the hydrocortisone regimen,” she says.
Corticotropin-Releasing Factor Type 1 Receptor Antagonists
A medication called crinecerfont (Crenessity) is a corticotropin-releasing factor type 1 receptor antagonist that can also be used with glucocorticoids to better control androgen levels in adults and children over age 4 who have classic CAH. This allows for a reduced daily glucocorticoid dose while maintaining control of androgen levels.
Salt Supplements
Medication Considerations
People who take medication for CAH will be monitored regularly, says Speiser. For example, follow-up visits may be:
- Every 1 to 2 months for infants
- Every 4 to 6 months for children
- Every 6 to 12 months for adults
In people with CAH, those with the classic type will need to take medication for life, says Speiser, whereas those with the nonclassic type may not need medication forever — if at all.
If you have CAH, you may need to take more glucocorticoids during times of physical stress, such as after an injury or when sick. This is called “stress dosing.” Your doctor will give you instructions about using higher and more frequent hydrocortisone doses. Or, if you’re unable to take oral medications, they may prescribe an injectable form of glucocorticoids, along with intravenous fluids, if you go to urgent care, says Speiser.
Other Medication Considerations for Women
Some women who have nonclassic CAH may also be given other treatments, including:
- Oral contraceptives, to help regulate periods and treat acne and facial hair
- spironolactone (Aldactone), to help treat acne and facial hair
- Corticosteroids, to help with fertility and decrease the risk of miscarriage
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