Mark L. Willenbring, MD
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DEVELOPMENT OF MODERN TREATMENT APPROACHES
Professional treatment for alcohol use disorder (AUD) supported by basic and clinical research is a relatively new field that for most of its history only provided custodial treatment. Modern behavioral approaches grew initially out of the success of Alcoholics Anonymous (AA) on the one hand and the growth of academic psychiatry and psychology after World War II on the other. AA was established in 1935, and its Big Book was published in 1939, spreading rapidly. The Minnesota Model of treatment, initially conceived in the 1950s and to this day the most prevalent form of treatment available in the United States, uses both professional staff and staff recovering from alcohol dependence; patient and family education; strong linkage to AA; and belief that alcoholism is a primary, progressive disease that cannot be cured, although it can be arrested through abstinence. It was initially provided only in 28-day programs in hospitals or residential facilities but is now provided in outpatient settings as well. The primary modalities used in most programs are group counseling and education. Twelve-Step facilitation is a manualized version of the Minnesota Model adapted for an individual outpatient approach.
At about the same time, the fields of psychology and psychiatry were undergoing substantial development. Albert Ellis developed the first type of cognitive–behavior therapy, rational-emotive therapy, in the mid-1950s, and Aaron Beck developed cognitive therapy for depression in the 1960s. Specific therapies for alcohol dependence based on such earlier psychological theories include therapeutic communities, aversion therapy, cognitive–behavior therapy, skills training, community reinforcement, and contingency management. More recently, William Miller and colleagues developed an approach based on stages of change and motivation.
Over the same period, research developed on the nature, causes, consequences, course, and treatment of AUD, and pharmacotherapy for alcohol dependence was attempted with many new psychiatric medications. All except disulfiram—approved for use as a deterrent or aversive agent in 1949—failed to substantiate early claims. It took 46 years for the next medication, naltrexone, to be approved for treatment of alcohol dependence. More recently, acamprosate and topiramate have been shown to be effective. It is very likely that there will be additional medications available in the future.
Despite considerable progress toward enlightened, humane, and effective treatment that has been made in a relatively short time, unique obstacles must be overcome for progress to continue. In more than 90% of community programs (not including the Veterans Affairs health care system), group counseling and referral to AA provided by counselors with minimal education are the only modalities offered. In most cases, there is no physician involvement in treatment other than treating withdrawal. Furthermore, treatment is time-limited, focused on inducing and maintaining remission, and offers little except repetition for patients who do not respond. Because of the lack of integration of addiction treatment programs with medical and psychiatric treatment, few programs are able to identify and treat coexisting mental and physical disorders in their patients, even though these are very common in a treatment-seeking population. The quality of counseling in community programs is poor.
HOW SHOULD TREATMENT OUTCOME BE DETERMINED?
Until relatively recently, total, continuous, permanent abstinence was considered by most to be the only goal of treatment and the only measure of outcome. Reasons for this are complex but include the strongly held belief of AA members and Minnesota Model treatment providers that anything less than a commitment to total lifetime abstinence would result in failure. Additionally, abstinence was easier for researchers to measure and verify. On the other hand, some researchers believed that drinking (including heavy drinking) was a learned behavior and that it might therefore be possible for patients to learn new ways of (moderate) drinking. Treatment methods based on this idea were developed and eventually compared to abstinence-based approaches, which generated considerable controversy and outright animosity.
The research community has developed increasingly sophisticated ways to measure outcome, although they are far from perfect. There are three broad categories of outcome: drinking behavior, symptoms or diagnostic criteria of dependence, and functioning in multiple life areas such as social function and psychological and physical health.
For the outcome of treatment of dependence, and using the Diagnostic and Statistical Manual of the American Psychiatry Association, fourth edition (DSM-IV) criteria, recovery is defined as no longer meeting any of the seven criteria of dependence (full remission), irrespective of drinking. Partial remission means meeting one or two dependence criteria, but not enough to qualify for the dependence diagnosis, and nonremission is continuing to meet three or more criteria. In some studies, meeting one or more abuse criteria, but not three or more dependence criteria, is also considered partial remission (recurrence of symptoms but not syndrome). Importantly, although drinking quantity and frequency alone are sufficient to determine whether a patient’s drinking constitutes a risk factor for future problems, they are not included in the diagnosis and thus are not considered in DSM-IV. This lack of attention to actual drinking patterns was continued in DSM-5.
As with other medical disorders such as cardiovascular disease, outcomes with chronic alcohol dependence can be measured in terms of reduction in disease-related adverse consequences. Heavy drinking is the third leading preventable cause of death in the United States, after smoking and obesity/lack of exercise. In middle age, chronic dependence is associated with a host of physical disorders such as liver fibrosis and cirrhosis, cancer, and cardiovascular disease; social consequences such as unemployment and divorce; and mental illness such as depression and suicide. The relationship between these outcomes and alcohol consumption is complex, however, mediated by genetic vulnerability, other lifestyle factors such as smoking, social factors, and possibly choice of beverage. Moreover, most mortality data on people with AUDs have been obtained with samples of people seeking or receiving treatment for alcohol dependence, populations with higher annual mortality than that of matched controls. Because treatment seekers have more severe dependence, a higher prevalence of comorbid conditions, and less social capital than do non–treatment seekers, information obtained by studying treatment populations applies only to the 20% or less of people with AUDs with the most severe and recurring form of dependence.
In short, quantity and frequency alone are sufficient to determine whether a patient’s drinking constitutes a risk factor for future problems. However, drinking becomes an AUD when it causes clinically significant distress or impairment, and these variables are more difficult to measure. Similarly, the relationship between social and occupational function is complex and bidirectional. Because abstinence and low-risk drinking without symptoms of an AUD (remission) are most robustly predictive of continued recovery as well as function, yet are easier to measure, they may serve as the best single outcome measure.
THE SPECTRUM OF HEAVY DRINKING AND AUDS
New research has provided a new and more complete view of the range of drinking, AUD, and alcohol-related harms. However, some definitions are needed to describe drinking behavior and relate it to adverse events. In the United States, a single alcohol serving is defined as the amount of ethanol in 1.5 oz (45 mL) of 80 proof spirits, 12 oz of beer, or 5 oz of table wine, each containing about 14 g of absolute ethanol. Because actual alcohol levels in beer and wine vary, these amounts are meant to be approximate. The National Institute of Alcohol Abuse and Alcoholism of the National Institutes of Health recommends that men drink no more than 4 alcohol servings per day and 14 servings per week and that women drink no more than 3 servings per day and 7 servings per week. Drinking within these limits is considered “low-risk” drinking. Lower limits or abstinence may be indicated in the presence of coexisting medical or psychiatric disorders, in older people, or when medication interactions are a concern. Women who are pregnant or at risk of becoming pregnant are advised to abstain. In this chapter, a day on which the limit is exceeded is considered a “heavy drinking day,” and “heavy drinking” is defined as drinking in excess of the maximum limits on a regular basis, such as exceeding the daily limits weekly or more often. “At-risk drinking” is heavy drinking in the absence of meeting any criteria for an AUD, whereas heavy drinking includes both at-risk drinking and symptomatic drinking (AUDs).
About 70% of the U.S. adult population report either being abstinent or engaging in low-risk drinking in any given year, about 21% are at-risk drinkers, and 9% have an AUD (5% abuse and 4% alcohol dependence.) At-risk drinking places an individual at higher risk for developing alcohol-related problems, such as an AUD, liver disease, or a mental disorder.
Most heavy drinkers are without current symptoms or problems but are at increased risk for physical, mental, and substance use disorders developing over time. Most with an abuse-only diagnosis qualify only by virtue of drinking and driving. Importantly, more than 40% of daily or near daily heavy drinkers do not meet any criteria for AUD. Similarly, only 20% to 40% of people with alcoholic liver cirrhosis also have alcohol dependence. Finally, not all alcohol-related harms occur in people who have AUDs, in part because there are twice as many at-risk drinkers as there are people with AUDs.
Drinking quantity and frequency patterns vary considerably among people who meet minimum criteria for alcohol dependence but most often involve drinking 4 to 7 days/week. People with alcohol dependence vary in many other ways as well, suggesting that the clinical diagnosis is actually comprised of an unknown number of subgroups. This heterogeneity presents a serious problem for treatment research. Although there are exceptions, most studies of treatment have included all people meeting criteria for dependence. Similarly, most treatment programs essentially provide the same treatment to everyone entering the program. Attempts to subtype dependence according to clinical and demographic characteristics have not been productive in terms of predicting differential response to treatment. Therefore, there is a pressing need to identify more robust subtypes of heavy drinkers, in particular subtypes that predict treatment response. Until that occurs, the individual physician must attempt to assimilate the latest information from randomized controlled trials and skillfully apply that information to an individual patient’s situation.
THE SPECTRUM OF DRINKING, DISORDERS, AND TREATMENT
Recent research has shown that drinking and related problems occur along a continuum ranging from none to mild, moderate, and severe. However, studies of treatment of dependence largely have been done on middle-aged, treatment-seeking adults, who have chronic, relapsing, severe alcohol dependence and have been ill for a decade or more. The extensive study of this subgroup has created a picture of alcohol dependence as an inevitably severe, chronic disorder resistant to treatment. In fact, new research has demonstrated that 72% of U.S. adults with a lifetime diagnosis of dependence have a single episode, lasting on average 3 to 4 years. Those with more than one episode average five of decreasing length, suggesting that failure to achieve permanent remission after the first episode predicts a more chronic course.
In contrast to popular belief, most people who meet dependence criteria recover without professional treatment or even attendance at mutual help groups. It appears that most people, on recognizing a problem, attempt to change alone or with informal help, and the majority are eventually successful, albeit after several years of active dependence. Seeking help from mutual help groups or nonaddiction professionals (about 25%) or professional treatment programs (13%) occurs when informal attempts to change fail (or because of an external contingency such as a driving while intoxicated charge). A significant proportion of help-seekers respond with improvement or remission, leaving a small but important group with severe and persistent dependence.
The continuum of drinking and associated symptoms and problems suggests a corresponding continuum of care—from brief counseling and facilitated self-change to long-term care management—most of which is not yet implemented or available. The quality of care for heavy drinking and AUDs in primary care is the lowest among 30 chronic conditions, and attempts to increase screening and brief intervention for at-risk drinking have met with little success in spite of a robust evidence base for their efficacy. Treatment programs suffer from insufficient funds, resulting in poorly trained and underpaid staff and excessive turnover.
A major public health challenge is to provide earlier identification and appropriate treatment to a much broader spectrum of individuals who drink heavily or who have alcohol dependence than is currently the case. A pressing need to is develop and test treatment approaches for people with mild-to-moderate dependence and relatively few comorbidities. Such patients are unlikely to seek treatment in addiction treatment programs. Needed also are early intervention strategies for youth who begin drinking in early adolescence and are at high risk for later development of severe chronic dependence. Finally, effective and cost-effective care management strategies for managing chronic severe dependence need to be implemented with the goal of providing services intermittently or continuously for years to decades rather than weeks or months. To provide this type of comprehensive care, the specialty addiction treatment sector will require substantial development so that addiction, psychiatric, medical, and social services can be provided in an integrated way and over longer periods. Some studies suggest that for harmful drinkers with serious medical or psychiatric illnesses, addressing drinking directly in the context of medical or psychiatric treatment is preferable to referral to a standard addiction treatment program.
TYPES OF TREATMENT AND TREATMENT EFFECTIVENESS
Multiple treatment modalities have been shown to be effective in the treatment of alcohol dependence. However, the best way to match the type and intensity of treatment to the individual needs of a patient with alcohol dependence remains unclear. For example, no systematic outcome advantage has been demonstrated for residential or intensive day program treatment compared with once or twice weekly outpatient treatment. Similarly, no behavioral treatment has been shown to be better than others that are conceptually distinct and use different behavioral techniques. Attempts to match specific behavioral therapies with clinical characteristics of patients have yielded little. In practical terms, the addiction treatment offered or available likely depends on patient preference, availability, access, coercion, urgent needs such as imminent withdrawal or suicide risk, and clinician orientation rather than on scientific evidence.
The outcome of treatment varies according to the diagnosis or stage of illness. At-risk drinkers (and possibly those with abuse-only diagnoses) who are identified and offered education and brief motivational counseling on average reduce drinking about 25% over the following year. Treatment outcome for dependence is remarkably similar across studies and treatment modalities, both behavioral and pharmacologic. In a typical treatment study, about one third of subjects will be in full abstinent or nonabstinent remission for the following year, 30% to 40% will show substantial improvement but will have at least some episodes of heavy drinking, and 20% to 30% will not show an effect. However, over the course of the ensuing 5 to 10 years, most will suffer at least some recurrence.
What Causes and Maintains Change in Drinking Behavior?
Help-seeking is strongly associated with increased odds of achieving recovery, but help-seekers differ systematically from non–help-seekers. Help-seekers on average are older, have more severe dependence and more coexisting mental and physical disorders as well as less social support, and are more likely to have the relapsing form of the illness. For those who do seek help, both professional treatment and Twelve-Step participation are associated with increased likelihood of recovery, especially abstinent recovery. For individuals older than 35 years, abstinence is much more stable an outcome than even light drinking without problems, whereas in younger persons, light drinking without problems (nonabstinent remission) is similar to abstinence in predicting continued remission 3 years later. In people who have been treated for alcohol dependence, recovery is in turn strongly associated with improved mortality. Thus, full remission, whether abstinent or nonabstinent, should be the goal of treatment for dependence, tempered with the recognition that full recovery cannot always be achieved.
Unfortunately, such studies are not able to establish causality. Although help-seeking and participation in treatment and Twelve-Step groups are associated with improved outcomes and decreased mortality, it cannot be ruled out that people who have decided to change will seek treatment, whereas those who do not want to change do not, or that people who respond to treatment early develop more hope and motivation to continue. That is, treatment participation or continuation may be a result of change rather than the converse. Although it appears that treatment followed by Twelve-Step participation is a frequent and effective path to recovery for many people, it is not the only one.
Integrating the Evidence and Personalizing Practice
It is a challenge to understand and incorporate new findings into practice, and there are few studies that address directly questions of central importance to clinicians. For example, should one recommend a few sessions of motivational enhancement therapy or an intensive day program for the treatment of dependence? However, certain conclusions emerge from the current body of evidence. First, although differences among different behavioral techniques tend to be minor, the quality of behavioral treatment is important. Specifically, empathic and skillful therapy is more effective than are confrontation and education. Furthermore, it is more important to engage someone with alcohol dependence in treatment than which particular treatment is used, as long as the treatment has been shown to be effective and is delivered skillfully. Therefore, it makes sense to offer a variety of treatment options, because patients are likely to vary in their preferences. Second, unless someone is unable to abstain from living in the community, there is no systematic advantage of residential versus outpatient treatment. Third, available medications offer small but clinically important benefit in early recovery, and therefore, patients should routinely be offered the opportunity to use them. However, at this time, there is no consensus that any one medication is better than another, no way to predict individual patient response, no specific sequence in which medications should be used, and no evidence at this time that combining medications is more beneficial than is monotherapy. For appropriate patients (moderate levels of dependence, little or no coexisting psychopathology, socially stable, and motivated to change drinking), medication with medical care management and encouragement to abstain, adhere to treatment, and attend mutual help groups is as effective as specialized alcohol counseling. Fourth, a social network supportive of abstinence is at least as important as whatever treatment occurs in determining outcome. Except for referral to mutual help groups, this aspect of treatment tends to be neglected, to the detriment of our patients. Behavioral marital therapy, for example, has a strong evidence base. Finally, for any given diagnosis (e.g., at-risk drinking vs. dependence), there is not yet a way to identify patient characteristics that reliably predict differential response to different treatments, although research in this area is promising.
KEY POINTS
1. Studies of treatment of dependence largely have been done on treatment-seeking adults with a chronic, severe form of alcohol dependence, creating a picture of the disorder as inevitably severe and resistant to treatment when, in fact, new research has demonstrated that 72% of U.S. adults with a lifetime diagnosis of dependence have a single episode, lasting on average 3 to 4 years.
2. In contrast to popular belief, most people who meet dependence criteria recover without professional treatment or even attendance at mutual help groups.
3. The continuum of drinking and associated symptoms and problems suggests a corresponding continuum of care—from brief counseling and facilitated self-change to long-term care management—most of which is not yet implemented or available.
4. Empathic and skillful therapy is more effective than are confrontation and education, and it is more important to engage someone with alcohol dependence in treatment than which particular treatment is used, as long as the treatment has been shown to be effective and is delivered skillfully.
REVIEW QUESTIONS
1. Which of the following correctly describes the typical course of alcohol dependence?