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New review finds pharmacotherapies are moderately effective for treating alcohol use disorder in combination with behavioral treatment

Findings indicate that acamprosate and naltrexone are most effective in reducing a return to drinking among the nine medications studied

RESEARCH TRIANGLE PARK, N.C. – A new systematic review from researchers at RTI International, a nonprofit research institute, has found that two medications — acamprosate and oral naltrexone — help prevent individuals with an alcohol use disorder (AUD) from returning to drinking and can reduce the amount of drinking in people who continue to drink alcohol. The study updated a 2014 systematic review on the benefits and harms of pharmacotherapies for AUD and was published in the Journal of the American Medical Association on Tuesday.

In 2020, approximately 28.3 million Americans met the criteria for an AUD, according to data from the National Survey on Drug Use and Health. Excessive alcohol consumption is the third leading cause of mortality in the U.S., resulting in more than 140,000 deaths each year. Despite the availability of pharmacotherapies for AUD, only 0.9% of diagnosed individuals received treatment in 2021. 

“Alcohol use disorders are associated with many health implications, from increases in cancer risk to liver disease and others," said Melissa McPheeters, Ph.D., senior director for analytics at RTI and lead author of the paper. “They can affect quality of life — as well as productivity and safety — by increasing risk of car crashes and other accidents and affecting the ability to engage in work and relationships. The public health implications of alcohol use disorders are enormous, given the number of people who struggle with these disorders. Even a moderate effectiveness of medications in treating AUDs could be very helpful across the population in improving health.” 

The research team, which included experts from RTI, The Ohio State University and the Kaiser Permanente Center for Health Research, used data from 118 randomized clinical trials that were conducted in outpatient settings. Researchers compared the efficacy of nine pharmacotherapies, including three approved by the U.S. Food and Drug Administration (FDA) and six off-label medications commonly used for AUD treatment. 

According to the study findings, return to drinking was reduced by 12% with the use of acamprosate and 7% with oral naltrexone. Topiramate, which is not an FDA-approved AUD treatment, also had moderate strength of evidence in reducing alcohol consumption but was associated with significant side effects, including cognitive disfunction, dizziness and taste abnormalities. 

“These results provide evidence that certain medications for alcohol use disorder are moderately effective,” said co-author and RTI senior research scientist Mark Edlund, M.D., Ph.D. “Patients and clinicians should decide together what will be the best treatment. Importantly, these medications are studied along with other forms of treatment — such as behavioral interventions — that should be in active use, with or without medication. The relatively moderate effectiveness of treatments also highlights the need for better primary and secondary prevention efforts.”

The systematic review also aimed to determine if the use of pharmacotherapies improved quality of life and prevented motor vehicle crashes and mortality among AUD patients, but the data were insufficient to make conclusions. Data were also insufficient to assess the effectiveness of these medications in primary care settings. 

The research was funded by the Agency for Healthcare Research and Quality (AHRQ) and findings are available in a report published by the agency.

Read the full paper 

Learn more about RTI’s substance use treatment research