By Jill Stein
BOSTON, MA -- May 2, 2007 -- When infantile spasm is managed by a short course of high-dose adrenocorticotropic hormone (ACTH) followed by rapid taper, severe adverse events are no more frequent than with other medications, according to data presented here at the American Academy of Neurology (AAN) 59th Annual Meeting.
The treatment may lead to weight gain and an increase in blood pressure, but these effects are reversible, said researcher Arthur Partikian, MD, senior resident, pediatric neurology division, Children's Hospital of Los Angeles, Los Angeles, California.
Dr. Partikian reported on the results of a retrospective chart review of all cases of infantile spasm treated at his institution during a recent 16-year period.
"The well-documented side effects of ACTH have been a deterrent to its general use as first-line therapy for infantile spasm, a relatively rare but devastating pediatric epileptic encephalopathy," he said in his presentation on May 1st.
"Although the recent AAN and Child Neurology Society practice parameter for the medical treatment of infantile spasms concluded that ACTH is probably effective for the short-term treatment of infantile spasms, it also noted that there is a lack of prospective trials regarding the optimal dosage and duration of treatment," he added.
He also said that few studies have focused on the safety, tolerability, and adverse events associated with ACTH for infantile spasms.
Overall, 43 patients with infantile spasms in this series received ACTH as initial treatment, 16 received vigabatrin, 6 received prednisone, and 11 received other anti-epileptic drugs.
ACTH treatment was associated with statistically significant weight gain and increase in blood pressure compared with other treatments. However, these relatively minor effects were readily reversed as the dose was reduced, Dr. Partikian noted.
There were no statistically significant differences in major adverse events with ACTH compared with other medications. One death from sepsis occurred, and 1 patient had spontaneous vertebral fractures. However, both infants had been treated with uncharacteristically prolonged courses with high doses of ACTH (> 6 weeks at full daily dose), he said.
Overall, few patients required a medication change or dosage reduction because of intolerable adverse effects.
Dr. Partikian said the study is the largest reported retrospective cohort of infantile spasm patients in North America.
The study was supported by Questcor Pharmaceuticals, Inc.
[Presentation title: Adverse Events Associated With Treatment of Infantile Spasms. Abstract P02.002]
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