Powered By
If you’ve been diagnosed with congenital adrenal hyperplasia (CAH), you may have been told your body isn’t making enough cortisol. This important hormone helps your body handle stress. It also regulates your metabolism and blood sugar.
One treatment for people with CAH and low cortisol is glucocorticoid therapy. This type of medication is similar to naturally occurring cortisol to help your body have all the cortisol it needs.
In this article, we’ll explain how glucocorticoid medications are used to treat CAH, how they work in the body, and the possible benefits and risks of this treatment.
Over 95 percent of people with CAH are born with a gene mutation (a variant or change) that affects the protein 21-hydroxylase. This protein is necessary for making cortisol in the body. When you have a changed version of 21-hydroxylase, your body can’t produce enough cortisol on its own, so you need medication to supplement it. That’s where glucocorticoids come in.
Cortisol is a steroid hormone made in the adrenal glands, which sit on top of your kidneys. Cortisol affects how your body regulates blood sugar, responds to inflammation, and manages blood pressure. It’s also produced during periods of stress. Cortisol affects almost every organ system in your body. It’s very important that the level of cortisol in your body isn’t too low, especially when you need it.
Depending on the form of CAH, low cortisol levels might cause your body to produce high amounts of adrenal androgens (sex hormones often thought of as “male hormones”). High androgen levels cause some of the symptoms of CAH.
Glucocorticoids are medications that mimic naturally occurring cortisol. They’re also called corticosteroids, or just steroids. If you have CAH and low levels of cortisol, your healthcare provider will prescribe glucocorticoid therapy.
Glucocorticoids can replace the cortisol that your body can’t make. The goal of this treatment is to provide enough cortisol for day-to-day functioning and times of illness, reduce the symptoms caused by low cortisol levels, and — at higher doses — prevent high androgen levels in CAH. The type and dosing of glucocorticoids for CAH depend on a person’s age and the type of CAH they have.
For children with classic CAH, glucocorticoid therapy is a long-term treatment that can help with normal growth and development. Hydrocortisone is the first choice for glucocorticoid treatment in pediatric CAH. It tends to have fewer side effects than longer-acting glucocorticoids, and it’s usually taken as pills at least three times a day.
A healthcare provider will regularly measure hormone levels in the blood. The dose may need to be adjusted to find the amount that replaces cortisol, provides control of androgens, and minimizes side effects.
When children with CAH become sick, they need to take increased amounts of hydrocortisone (also known as “stress dosing”) to mimic what the body would produce during times of illness. For emergencies, including trauma, passing out, or repetitive vomiting (twice in 20 minutes), there is a hydrocortisone injection.
During the teenage years, puberty affects how much cortisol replacement the body needs. Maintaining normal amounts of androgen hormones is important for growth and pubertal development. To help reduce high androgen levels, doses of glucocorticoids might be increased during this time.
Frequent lab tests are needed, especially while teenagers are still growing. After adult height is reached and puberty is complete, different treatment strategies can be considered.
There are no standard guidelines on which medications are best to treat adults with CAH. A combination of glucocorticoids can be prescribed. Hydrocortisone is still a common treatment option. In addition, longer-acting glucocorticoids, like prednisolone and dexamethasone, might be more convenient in maintaining control over excess androgens.
With longer-acting medication, fewer doses per day are needed. For example, dexamethasone is usually only taken once a day. However, the side effects of dexamethasone tend to be stronger. The choice of medication will be up to you and your endocrinologist.
For people who have a form of CAH called nonclassic CAH, glucocorticoid therapy isn’t usually required. People who develop nonclassic CAH as adolescents or adults may be able to take antiandrogen therapies that are not glucocorticoids. Children who develop nonclassic CAH may still require glucocorticoid therapy to regulate growth and pubertal development.
Most adults with nonclassic CAH do not take regular glucocorticoid therapy, but it may depend on how much cortisol their body makes. They might require temporary treatment with glucocorticoids at certain times. These include:
When cortisol levels are too low for what the body needs, it’s possible to develop an adrenal crisis. This can happen when someone with CAH is sick, has a fever or infection, has surgery, or stops medication. The symptoms of an adrenal crisis include weakness, nausea, and vomiting. In children, vomiting more than once within 20 minutes is a common symptom of an adrenal crisis.
A salt-wasting adrenal crisis involves an additional component of dehydration due to salt loss.
An adrenal crisis is life-threatening, and immediate medical attention is necessary. Be sure to ask your healthcare provider about warning signs and what to do in case of a suspected adrenal crisis. If your healthcare team knows about a planned surgery, they may increase your glucocorticoid dosage to prevent an adrenal crisis. If you’re sick, your endocrinologist can also provide oral stress dosing to help prevent an adrenal crisis.
Glucocorticoids are powerful medications that affect the entire body. They can have several known adverse effects when used over long periods of time. In general, the higher the dose of glucocorticoid, the greater the risk of developing side effects. Longer-acting glucocorticoids in particular can have side effects that include gradual bone loss and mood changes.
Your healthcare team will work with you to find the lowest dose that brings your hormones to levels that align with your treatment goals. This may help minimize the side effects.
Ongoing research is investigating different forms of glucocorticoid therapy. Reducing the side effects of long-term use is one major goal. For example, researchers are currently studying modified-release hydrocortisone, which could allow for lower dosages and fewer side effects.
Continuous subcutaneous hydrocortisone infusion — a method that delivers hydrocortisone through a small pump under the skin throughout the day — is another new approach under investigation. In one trial, it showed promising results in controlling high androgen levels and reducing side effects like bone loss. We still need more data on whether these newer treatments are as safe and effective as existing medications.
Treating CAH can feel challenging. Adults navigating fertility or body image concerns might have a tough time. Parents of children with CAH may feel overwhelmed by their role as caregivers. Dealing with frequent medical visits and laboratory tests can be frustrating.
It’s important to remember that overall, glucocorticoid therapy treats symptoms and improves quality of life. Working closely with your healthcare team and communicating any challenges and issues you face is the best way to manage CAH treatment.
On CAHteam, people share their experiences with congenital adrenal hyperplasia, get advice, and find support from others who understand.
If you’re on glucocorticoid therapy for CAH, how has it affected your symptoms? Let others know in the comments below.
Get updates directly to your inbox.
Continue with Facebook
Sign up with your email
Become a member to get even more
Join
We'd love to hear from you! Please share your name and email to post and read comments.
You'll also get the latest articles directly to your inbox.