Corticosteroid replacement therapy is a daily treatment that provides the hormones cortisol and aldosterone for people who cannot produce it normally (as in the case of adrenoleukodystrophy – ALD). Corticosteroid replacement therapy is not a cure but can reduce the symptoms of adrenal insufficiency (when the adrenal glands are not producing enough of these two hormones).

How corticosteroid replacement therapy can help ALD patients

ALD is a serious genetic disorder characterized by neurodegeneration and adrenal insufficiency. There are few treatments available, and the only potential cure is a stem cell transplant, which not all patients are able to receive.

Corticosteroids are medications that resemble the hormone cortisol. In the case of ALD, the adrenal glands do not produce enough cortisol or aldosterone.

To replace cortisol, most corticosteroid replacement medications contain hydrocortisone. Other possible medicines are prednisolone or dexamethasone, but these are less commonly used.

Aldosterone is replaced with a medication called fludrocortisone.

Most patients receiving corticosteroid replacement therapy find that the medication allows them to return to a normal routine of activity and exercise, though some patients may find they are more easily fatigued.

Other information

It is very important for patients to take their medication, as missing multiple doses may lead to an adrenal crisis, a potentially life-threatening condition. It is also important for patients to take their medication on time, as taking a dose late may make them more tired or cause insomnia.

Corticosteroid replacement therapy may cause some patients to develop related conditions such as diabetes or underactive thyroid, which must be monitored carefully. Most patients will meet once or twice a year with an endocrinologist, who will evaluate their medication regimen and make adjustments as necessary.

Corticosteroids may cause side effects, including weight gain, increased appetite, stomach pain, headache, and nervousness or restlessness.

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