- Last Updated on 14 December 2016
There is strong evidence in the scientific literature for treatment of infantile spasms with hormonal therapies. These treatments include multiple agents such as corticosteroids (e.g. prednisolone, prednisone, hydrocortisone), ACTH (adrenocorticotropic hormone, also called corticotropin, and marketed as Acthar® as well as tetracosactide (Synacthen®), a modified synthetic form of ACTH used frequently outside the United States). ACTH is a naturally occurring hormone produced in the brain which causes the adrenal glands to produce cortisol (another natural hormone which is very similar to prednisolone). This in part explains why these treatments have similar effectiveness and side effects. The exact mechanism by which they impact infantile spasms is unknown. There has been considerable ongoing debate as to which particular form of hormonal therapy is most effective. Although it is clear that traditionally high-dose ACTH is superior to typical dosages of prednisolone (2 mg/kg/day), very high doses of prednisolone (4-8 mg/kg/day) appear to exhibit similar response rates in comparison to ACTH. Furthermore, there is continued debate regarding the equivalence of various dosages of ACTH.
Prednisolone is supplied as a liquid, with a reliably unpleasant taste. The taste can be masked by pharmacists using a variety of flavorings, with variable success. In contrast, ACTH is supplied as an injectable gel. ACTH must be injected into muscle, and as such, parents must be trained to administer the intramuscular injections.
Prednisone (not to be confused with prednisolone) is a similar corticosteroid drug that is often used in place of prednisolone. The liver converts prednisone to prednisolone, the latter being the active drug. Prednisone and prednisolone are generally considered equivalent and interchangeable, though prednisolone exhibits somewhat better taste and aftertaste.
For prednisolone, dosage ranges from 2 to 8 mg/kg/day (with a maximum daily dosage of 60 mg). The schedule of administration varies considerably, from every other day, to multiple daily doses. The most successful regimen in published studies is 6 to 8 mg/kg/day, divided in three doses each day. The duration of therapy is short, and successful response is usually achieved within 2 weeks. A typical course of treatment lasts 2 to 4 weeks (including a taper of the medication).
Regarding ACTH, dosage ranges from 80 to 150 international units per square meter of body surface area. (This is calculated based on a patient's weight and length). The most successful regimen in scientific publications is 150 IU/m2, divided in 2 doses each day. Like prednisolone, the treatment course is short, and successful responses are usually seen in less than 2 weeks. Similarly, a typical duration of treatment is 2 to 4 weeks.
Prednisolone and ACTH have similar side effect profiles. Common side effects include irritability, increased appetite, weight gain, hypertension (high blood pressure), and hyperglycemia (high blood sugar). Frequent physician visits (e.g. twice a week) to check for hypertension and hyperglycemia are crucial to identify these potential side effects early so as to avoid serious complications. Immune system suppression is a common and severe side effect which poses a risk of very dangerous and potentially life-threatening infections such as pneumonia or sepsis (infection in the blood). Infants treated with these medications should avoid exposure to other sick children and adults as much as possible, as seemingly mild infections can become quite severe with immune system suppression. Hospital and pediatric clinic waiting rooms should be avoided. Other serious side effects include stomach ulcers, electrolyte abnormalities (especially low potassium), glaucoma, myopathy (muscle disorder leading to weakness), and impaired wound healing. Gastrointestinal protectants such as ranitidine (Zantac®) and famotidine (Pepcid®) are routinely prescribed with these medications to prevent stomach ulcers. Vaccinations should be delayed for several months following the completion of a hormonal therapy course. See our vaccination page.
These medications should be administered only under the direct supervision of a physician.