The long-term cognitive and seizure outcomes of 37 patients with cryptogenic infantile spasms (onset, age 3 to 9 months) treated with high-dose synthetic adrenocorticotropic hormone (ACTH) (1 mg IM every 48 hrs for 2 weeks, 8-10-week slow taper, followed by oral prednisone, 10 mg/d for 1 month, and slow taper for 5 months or until age 1 year) were evaluated at Schneider Children’s Medical Center of Israel, Petah Tiqva, Israel. Development was assessed before treatment and cognitive outcomes were determined after 6 to 21 years and analyzed in relation to treatment lag (after 1 month from onset) and pretreatment regression. Cognitive outcome was normal in all 22 (100%) infants treated within 1 month of onset of infantile spasms (early treatment group), and in 40% of 15 treated after 1 to 6.5 months (late-treatment group). Normal cognitive outcome occurred in all 25 (100%) with no or mild mental deterioration at onset, but in only 3 (25%) of 12 with severe pre-treatment deterioration. The prognosis for normal long-term cognitive outcome is poor if development has severely regressed for 1 month or more before treatment begins. [1]

COMMENT. In this series of cryptogenic cases, infantile spasms were permanently controlled in 92% of the early treated group and in 80% of the late treated group. Adverse effects were common with the high dose ACTH regimen: Cushingoid features developed in 100%, hypertension in >50%, and hypokalemia and infections were frequent occurrences. The authors justify the high-dose ACTH based on the results of a previous study showing poor results in patients treated with low-dose ACTH or corticosteroids. [2]

The debate regarding low vs high dose ACTH for infantile spasms continues. My own preference like that of researchers in Japan has been for the low dose regimen, so that serious side effects are avoided [3, 4]. High doses are favored by colleagues in the UK and also by some in the US [5]. The earlier diagnosis is made and ACTH treatment begun, the better the results. This is true with either regimen. Whether the incidence of relapse is less and cognitive outcome superior with large dose cf low dose treatment remains to be proven by a controlled trial.