New and Emerging Therapies for Congenital Adrenal Hyperplasia (CAH)

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By Staff
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Other new CAH treatments attempt to address the challenges of steroid timing, which can be tough to get right.

Cortisol levels normally fluctuate throughout the day. But traditional oral steroids don’t always align to those changes, producing cortisol peaks that are too high and leaving levels too low before your next dose, says Sasan Mirfakhraee, MD, an associate professor in the division of endocrinology at UT Southwestern Medical Center in Dallas.

“They don’t replicate the body’s natural rhythm,” he says.

Modified-Release Steroids

Only available in Europe, oral medications Efmody and Plenadren are modified-release forms of hydrocortisone. Compared with regular steroids, the body takes longer to break them down and to clear them from your system. This can allow for one or two daily doses, with a more gradual rise and drop in cortisol levels that more closely mimics what your body would do naturally.

There are no known plans for the FDA to approve either medication for use in the United States, Auchus says.

Steroid Pump Therapy

Continuous subcutaneous hydrocortisone infusion (CSHI), or steroid pumps, are another therapy that can mimic the ebbs and flows of cortisol. The FDA has not approved these devices, but doctors may prescribe them off-label.

These devices work like an insulin pump, delivering liquid hydrocortisone through a small tube that goes under your skin.

“The pump can be programmed to more closely match the body’s natural rhythm, which generally allows for a lower total daily dose of steroid and improved symptoms such as energy and mood,” Dr. Mirfakhraee says.

CSHI can improve well-being and quality of life in people with CAH. But it comes with a major risk: If a steroid pump fails without you noticing, it can interrupt how hydrocortisone is administered, which could lead to life-threatening complications, such as adrenal crisis.

Mirfakhraee says that more research is needed to determine whether the benefits of CSHI outweigh its risks, compared with other treatment types.

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