Research review finds support for some off-label uses of atypical antipsychotic medications

Research Activities, May 2012, No. 381

A typical antipsychotic medications, approved by the Food and Drug Administration to treat schizophrenia, are also used to treat such conditions as agitation in dementia, generalized anxiety, and obsessive-compulsive disorder (called off-label use). Although some patients appear to benefit from off-label treatment with specific atypical antipsychotic drugs, the drugs are nevertheless associated with serious adverse events. These are the findings of a systematic review of studies on the topic.

The researchers examined controlled trials that compared the effect of specific atypical antipsychotic drugs versus placebo, another atypical antipsychotic drug, or other medications for off-label use in adults. From 14 placebo-controlled trials of elderly patients with dementia, the researchers found small but statistically significant improvement in psychosis, mood, and aggression for aripiprazole, olanzapine, and risperdone. For treatment of general anxiety, evidence from three pooled trials showed that quetiapine had a 26 percent greater likelihood of improving a patient's anxiety scores than did placebo. Risperidone was found 3.9 times more likely to produce a favorable response in patients with obsessive-compulsive disorder than did placebo.

In elderly patients, the antipsychotics were associated with an increased risk of death, stroke, uncontrolled body or facial movements, and urinary tract symptoms, depending on the specific drug used. The major problems observed in nonelderly adults were weight gain, fatigue, sedation, and uncontrollable facial or body movements. The study was funded with support from the Agency for Healthcare Research and Quality to the RAND Health Southern California Evidence-based Practice Center (Contract No. 290-07-1006).

More details are in "Efficacy and comparative effectiveness of atypical antipsychotic medications for off-label uses in adults," by Alicia Ruelaz Maher, M.D., Margaret Maglione, M.P.P., Steven Bagley, M.D., and others in the September 2011 Journal of the American Medical Association 306(12), pp. 1359-1369.

Page last reviewed May 2012
Internet Citation: Research review finds support for some off-label uses of atypical antipsychotic medications: Research Activities, May 2012, No. 381. May 2012. Agency for Healthcare Research and Quality, Rockville, MD.