Randomized, Single-Blind, Parallel Clinical Trial on Efficacy of Oral Prednisolone Versus Intramuscular...

We earlier completed a single-blind, parallel-group, randomized clinical trial to test the null hypothesis that adrenocorticotropic hormone (ACTH) is not superior to high-dose prednisolone for short-term control of West syndrome. We now present long-term follow-up data for spasm control for individuals who completed this earlier trial.

Infants with untreated West syndrome were randomized to receive 14 days of prednisolone (40 to 60 mg/day) or intramuscular long-acting ACTH (40 to 60 IU every other day). They were evaluated at three, six, and 12 months to evaluate long-term spasm control.

The total number of infants treated was 97 (48 prednisolone; 49 ACTH). All completed the treatment course. Eighty-five, 82, and 76 children were available for follow-up at three, six, and 12 months. The number lost to follow-up at each interval was not statistically different. Likelihood of spasm freedom at three months was significantly higher for prednisolone (64.6%) than for ACTH (38.8%) (P = 0.01; odds ratio = 2.9; 95% confidence interval = 1.3 to 6.6). At six months (P = 0.19) and twelve months (P = 0.13), the control of spasms was not statistically different, although a trend in favor of prednisolone was documented at both of these time points (58.3% versus 44.9% for ACTH at six months and 56.2% versus 40.8% with ACTH at 12 months). After initial remission by day 14 (n = 46), the likelihood of a relapse within the next 12 months was not statistically different between the two treatment groups (P = 0.1).

Control of spasms at three months was significantly better if initially treated with prednisolone. Control of spasms at six and 12 months was not significantly different despite a trend favoring prednisolone. Risk of relapse following initial remission was similar in the two groups.