Which medications in the drug class Adrenocorticotropic Agents are used in the treatment of Tuberous Sclerosis?

Updated: Aug 21, 2018
  • Author: David Neal Franz, MD; Chief Editor: Amy Kao, MD  more...
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Answer

Adrenocorticotropic Agents

These agents cause profound and varied metabolic effects. Corticosteroids modify the body's immune response to diverse stimuli.

Corticotropin (HP Acthar, ACTH)

Used in infants with infantile spasms (West syndrome) due to TSC. Estimated overall efficacy (percentage of infants with infantile spasms due to any cause reaching seizure freedom) is 50-67%. Associated with serious, potentially life-threatening adverse effects.

Must be administered IM, which is painful to infant and unpleasant for parent to perform. Daily dosages expressed as U/d (most common), U/m2/d, or U/kg/d.

Prospective single-blind study demonstrated no difference in effectiveness of high-dose, long-duration corticotropin (150 U/m2/d for 3 wk, tapering over 9 wk) versus low-dose, short-duration corticotropin (20-30 U/d for 2-6 wk, tapering over 1 wk) with respect to spasm cessation and improvement in patient's EEG. Hypertension was more common with larger doses.

Prednisone (Rayos)

Like ACTH, has been used for infants with infantile spasms (West syndrome) due to TSC. Few studies comparing ACTH and prednisone have been performed; one double-blind, placebo-controlled, crossover study demonstrated no difference between low-dose ACTH (20-30 U/d) and prednisone (2 mg/kg/d), while a second prospective, randomized, single-blinded study demonstrated high-dose ACTH at 150 U/m2/d was superior to prednisone (2 mg/kg/d) in suppressing clinical spasms and hypsarrhythmic EEG in infants with infantile spasms.


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