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ACTH therapy in infantile spasms: side effects

Insights

Adrenocorticotropic hormone (ACTH) treatment for infantile spasms caused significant side effects in 37% of children, including infections and electrolyte disturbances. Careful monitoring and reconsidering high dosages are crucial due to potential severe complications and mortality.

Area of Science:

  • Pediatric Neurology
  • Endocrinology

Background:

  • Infantile spasms (IS) are a severe form of epilepsy in infants.
  • Adrenocorticotropic hormone (ACTH) has been a treatment option for IS.

Observation:

  • A retrospective study analyzed 162 children treated with ACTH for infantile spasms between 1960-1976.
  • Treatment outcomes and adverse events were documented.

Findings:

  • 37% of children experienced pronounced side effects, with a 4.9% mortality rate.
  • Common complications included infections (septic, pneumonia, urinary, gastrointestinal), hypertension, osteoporosis, and electrolyte disturbances.
  • Higher ACTH doses (120 units) were associated with significantly more infections than lower doses (40 units).
  • Severe complications like intracerebral hemorrhages, oliguria, hyperkalemia, and tubular necrosis were observed.

Implications:

  • ACTH treatment for infantile spasms carries a substantial risk of severe side effects, potentially underestimated in previous assumptions.
  • Close patient monitoring before, during, and after ACTH therapy is essential.
  • The efficacy versus risk of very high-dose ACTH regimens warrants critical re-evaluation.

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