Piracetam Therapy for Down Syndrome: A Rush to Judgment? FREE

The recent report of Lobaugh et al¹ provides convincing evidence that piracetam is not the miracle drug for Down syndrome claimed by its proponents. The study is well controlled, the analysis detailed, and the conclusions strong. Yet an examination of their data reveals positive effects that the authors have overlooked and evidence that the negative effects are of less concern than their comments would warrant.

First, Figure 2 lists 72 primary outcome measures of attention, memory, perceptual abilities, executive function, and fine motor skills. Of these, 46 produced results that were better for piracetam than for the placebo. An outcome such as this or better by chance is highly unlikely (binomial test, P = .01).

Second, 11 of 18 parents reported cognitive improvement with piracetam and only 2 of 18 reported the same with the placebo. Such an outcome is also highly unlikely by chance (2-tailed Fisher exact test, P = .004). Since the interviews were carried out in a double-blind fashion, the parents were likely responding to real differences in their children's functioning. Finally, the well-publicized negative effects were apparently spontaneous comments by the parents rather than questionnaire responses. On the questionnaires, 5 comparisons "reached or approached significance" for improved behavior with piracetam. In particular, teachers reported that children had significantly "fewer total problems" when taking piracetam. While this is insufficient evidence that the drug was helping, it gives us confidence that its effects were not particularly harmful. It is revealing that the authors chose to feature in the "Results" section of their abstract the negative effects reported by caregivers for 7 children but failed to mention the 11 children for whom the parents indicated cognitive improvement. There seems to be no evidential basis for this focus; rather, it points to a possible bias of the authors toward these negative reports over the positive.

In summary, it is regrettable when parents are given false hope through unproven remedies, and rigorous studies such as this one are essential to counter such claims. However, we should not let our experience with worthless media-publicized claims inhibit us from identifying positive effects when they are present. A more justifiable conclusion would have been that while dramatic effects were not observed, and there were indications of adverse effects with certain children, small gains in cognition and behavior were also evident.