Lack of benefit of a single dose of synthetic human secretin in the treatment of autism and pervasive developmental disorder

Background: Secretin is a peptide hormone that stimulates pancreatic secret ion. After recent publicity about a child with autism whose condition marke dly improved after a single dose of secretin, thousands of children with au tistic disorders may have received secretin injections. Methods: We conducted a double-blind, placebo-controlled trial of a single intravenous dose of synthetic human secretin in 60 children (age, 3 to 14 y ears) with autism or pervasive developmental disorder. The children were ra ndomly assigned to treatment with an intravenous infusion of synthetic huma n secretin (0.4 microg per kilogram of body weight) or saline placebo. We u sed standardized behavioral measures of the primary and secondary features of autism, including the Autism Behavior Checklist, to assess the degree of impairment at base line and over the course of a four-week period after tr eatment. Results: Of the 60 children, 4 could not be evaluated -- 2 received secreti n outside the study, and 2 did not return for follow-up. Thus, 56 children (28 in each group) completed the study. As compared with placebo, secretin treatment was not associated with significant improvements in any of the ou tcome measures. Among the children in the secretin group, the mean total sc ore on the Autism Behavior Checklist at base line was 59.0 (range of possib le values, 0 to 158, with a larger value corresponding to greater impairmen t), and among those in the placebo group it was 63.2. The mean decreases in scores over the four-week period were 8.9 in the secretin group and 17.8 i n the placebo group (mean difference, -8.9; 95 percent confidence interval, -19.4 to 1.6; P = 0.11). None of the children had treatment-limiting adver se effects. After they were told the results, 69 percent of the parents of the children in this study said they remained interested in secretin as a t reatment for their children. Conclusions: A single dose of synthetic human secretin is not an effective treatment for autism or pervasive developmental disorder. (N Engl J Med 199 9;341:1801-6.) (C)1999, Massachusetts Medical Society.