Efficacy of long term weekly ACTH therapy for intractable epilepsy

Takehiko Inui, Tomoko Kobayashi, Satoru Kobayashi, Ryo Sato, Wakaba Endo, Atsuo Kikuchi, Tojo Nakayama, Mitsugu Uematsu, Masaru Takayanagi, Mitsuhiro Kato, Hirotomo Saitsu, Naomichi Matsumoto, Shigeo Kure, Kazuhiro Haginoya

Research output: Contribution to journalArticlepeer-review

12 Citations (Scopus)


Background: Adrenocorticotropic hormone (ACTH) therapy is the first-line therapy for infantile spasms, and is effective for many other intractable epilepsies. While spasms may respond to ACTH for weeks, a substantial proportion of patients develop recurrent seizures over a yearly period. To maintain efficacy, we treated two children with intractable epilepsy with weekly ACTH therapy for 1. year and described the changes in clinical seizures, electroencephalograms, developmental assessments and side effects. Subjects and methods: A girl with infantile spasms due to lissencephaly and a boy with atypical absence seizures were studied. In both cases, seizures were frequent and resistant to antiepileptic drugs; electroencephalograms showed continuous epileptiform activities, and the patients' development was delayed and stagnant prior to ACTH treatment. The initial ACTH therapy (daily 0.015. mg/kg for 2. weeks, 0.015. mg/kg every 2. days for 1. week, 0.0075. mg/kg every 2. days for 1. week), was transiently effective in both cases. The second-round ACTH therapy consisted of the initial ACTH therapy protocol followed by weekly ACTH injections (0.015. mg/kg or 0.0075. mg/kg) for 1. year. Both cases were followed for at least 1. year after therapy. Results: In both patients, clinical seizures were completely controlled during and 1. year after the second-round AHCH therapy. Continuous epileptiform discharges disappeared, while intermittent interictal epileptiform discharges remained. Both patients showed some developmental gains after achieving seizure control. No serious side effects were recorded. Conclusion: Further studies are warranted to determine if a long-term weekly ACTH is a safe and effective treatment for intractable epilepsy.

Original languageEnglish
Pages (from-to)449-454
Number of pages6
JournalBrain and Development
Issue number4
Publication statusPublished - 2015 Apr 1


  • ACTH
  • Atypical absence seizure
  • Infantile spasms
  • Intractable epilepsy
  • LIS1
  • Lissencephaly


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