Does Corticotropin work?
Partly: a small trial of 29 babies backs the main use; most other uses were never tested well.
The use corticotropin is best known for today, infantile spasms, is supported by randomized trials, but they are small: the trial in the citations randomized 29 infants. Most of the other labeled indications were approved in the 1950s on uncontrolled experience and the label itself says controlled trials in multiple sclerosis showed faster recovery from attacks without changing the disease course. That earns a B and not an A. 13
Baram and colleagues randomized 29 infants with infantile spasms. The infants also showed the chaotic EEG pattern called hypsarrhythmia. They got two weeks of high-dose corticotropin or prednisone. A response meant the spasms stopped and the EEG cleared. An investigator who did not know the treatment judged this. Thirteen of 15 infants on corticotropin responded (87%). Only 4 of 14 on prednisone did (29%). The authors note that earlier blinded studies found low-dose ACTH no better than prednisone. 1
For multiple sclerosis, the label says controlled trials showed corticotropin speeds recovery from acute flare-ups. But it says there is no evidence the drug changes the final outcome or the course of the disease. 3
Evidence checklist
- Randomised controlled trial Blinded trial vs prednisone in infants
- Human study over 100 people Infant trial had 29 participants
- More than one research group No other groups' trials described
- Clinical outcomes, not surrogate markers Spasm and EEG response measured
- Human safety data beyond case reports Steroid-type effects well documented
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More on Corticotropin
Citations
- Baram 1996, Pediatrics: high-dose corticotropin versus prednisone for infantile spasms, randomized blinded study pubmed.ncbi.nlm.nih.gov
- Acthar Gel prescribing information (manufacturer) acthar.com
- FDA label, 2021: Acthar Gel (repository corticotropin injection) accessdata.fda.gov
- Drugs@FDA: Acthar Gel (NDA 008372) approval record accessdata.fda.gov
Reference information only, not medical advice. Drawn from the Corticotropin article, fact-checked against the cited sources. No dosing anywhere on this site.