Evidence Lacking to Support Many Off-Label Uses of Atypical Antipsychotics

ROCKVILLE, Md., January 17, 2007 /PRNewswire-USNewswire/ -- Some newer antipsychotic medications approved to treat schizophrenia and bipolar disorder are being prescribed to millions of Americans for depression, dementia, and other psychiatric disorders without strong evidence that such off-label uses are effective, according to a new analysis by HHS' Agency for Healthcare Research and Quality.

The federally funded comparative effectiveness review of these drugs -- called atypical antipsychotics -- identified the medications' potential for serious side effects while pointing to an "urgent need" for more research into new treatments for the growing population of dementia patients who display severe agitation.

"This report emphasizes the importance of understanding the risks and benefits of different medicines," said AHRQ Director Carolyn M. Clancy, M.D. "Caution is necessary in the off-label use of atypical antipsychotics, especially when used in the elderly and when the evidence for effectiveness is not good."

Atypical antipsychotics are second-generation medicines designed to cause fewer neurological complications than conventional antipsychotics. They include aripiprazole (sold as Abilify), olanzapine (Zyprexa), quetiapine (Seroquel), risperidone (Risperdal), and ziprasidone (Geodon). Each is approved by the Food and Drug Administration to treat schizophrenia and bipolar disorder, and risperidone is also approved to treat irritability in children ages 5 to 16 who have autism.

Some studies suggest that atypical antipsychotics may help patients with mental health conditions for which there are no FDA-approved alternatives. Risperidone and quetiapine, for example, help certain patients with obsessive- compulsive disorder when used in conjunction with antidepressants. Risperidone and olanzapine improve sleep problems, depression, and other symptoms in men with combat-related post-traumatic stress disorder when used to augment therapy with antidepressants or other psychotropic medications.

Overall, however, researchers found that much of the scientific evidence for off-label use of antipsychotics was of insufficient quality because studies were too small or lacked scientific rigor.

Review authors evaluating the potential benefits and risks of the medications also found strong evidence that atypical antipsychotics can increase chances of adverse events. Some of the drugs increase risks of stroke, tremors, significant weight gain, sedation, and gastrointestinal problems.

The new review was produced by AHRQ's Effective Health Care program. It was authored by AHRQ's Southern California/RAND Evidence-based Practice Center. The center examined 84 published studies on atypical antipsychotics and summarized evidence about several conditions:

    * Dementia: One analysis showed a small benefit for risperidone and

      aripiprazole in the treatment of agitation and psychosis. Another

      suggested olanzapine may help treat psychosis. But a large clinical

      trial that explored whether risperidone, olanzapine, and quetiapine

      controlled behavioral disturbances in Alzheimer's patients concluded

      that the risks of adverse events offset the potential benefits.

      Overall, analyses identified potential harms as a small increase in the

      risk of death and increased chances of stroke, neurological problems

     (such as tremors or muscle contractions), and weight gain.


    * Depression: For patients who don't benefit from selective serotonin

      reuptake inhibitors (SSRIs), the supplemental use of atypical

      antipsychotics was not helpful, according to research. No studies showed

      the drugs provided a clear benefit for patients with major depressive

      disorder with psychotic features. Evidence is conflicting for bipolar

      depression.


    * Obsessive-Compulsive Disorder: Atypical antipsychotics significantly

      helped patients who don't respond adequately to SSRI therapy, studies

      showed. Overall, patients taking the drugs were about 2.7 times as

      likely to improve as patients taking placebo. The chances of benefiting

      were best for risperidone and quetiapine.


    * Post-Traumatic Stress Disorder: Studies of men with combat-related PTSD

      showed risperidone and olanzapine, when used with antidepressants or

      other psychotropic medications, improved sleep quality, anxiety, and

      other symptoms. Studies were inconclusive when measuring benefits for

      women.


    * Personality Disorders: For patients with borderline personality

      disorder, one study suggested olanzapine was more effective than placebo

      but showed little benefit when used to augment talk therapy. All studies

      of olanzapine were very small, however, and patients experienced

      significant weight gain. Two other small trials suggested risperidone

      may benefit patients with schizotypal personality disorder, and

      aripiprazole may help patients with borderline personality disorder.


    * Tourette's Syndrome: Risperidone is more effective than placebo,

      according to a small body of research. The benefits of ziprasidone are

      uncertain.

Off-label prescribing is a common but relatively understudied practice in health care. A 2001 AHRQ-funded study concluded that about 21 percent of prescribed drug use was for conditions not indicated on the label. Cardiac medications and anticonvulsants were the drugs most commonly used off label. Most off-label use occurs without scientific support, the study said.

The report released today, Efficacy and Comparative Effectiveness of Off- Label Use of Atypical Antipsychotics, is the newest analysis from AHRQ's Effective Health Care program. That program represents the first federal effort to compare alternative treatments for significant health conditions and make the findings public. The program is intended to help patients, doctors, nurses, and others choose the most effective treatments. Information on the program, including full reports, can be found at http://www.effectivehealthcare.ahrq.gov.

CONTACT: AHRQ Public Affairs, +1-301-427-1998, or +1-301-427-1855

Web site: http://www.effectivehealthcare.ahrq.gov/

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Posted: January 2007

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