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Epidemiological aspects of studying outcome in rheumatoid arthritis.

Knowledge of the outcome of rheumatoid arthritis (RA) is important both to the patient and to the rheumatologist. However, little data exist on the long-term outcome and effects of treatment on the disease. Consideration is given to the problems that arise in the choice of methods and measures to be used in an outcome study. Patient selection is an underestimated factor, which often produces biases and problems of comparability. Useful lessons can be learnt from examining outcome studies in other chronic diseases.

Arthritis, Rheumatoid

Inpatient psychiatric treatment of children and adolescents: a review of outcome studies.

All outcome studies of child and adolescent residential treatment and inpatient psychiatric hospitalization reported in the literature from 1975 to the present were examined. A set of statistical procedures was developed to integrate the findings of the 34 studies, which provided a weighted predictive value for 10 variables. Psychiatric hospitalization was often beneficial, particularly if a specialized treatment program and aftercare were available and if the child presented with a less pathological clinical picture. Age at admission and sex bore no relationship to favorable outcome, and IQ and length of stay yielded only a modest relationship to outcome. Recommendations to guide future researchers are offered that will provide new insights into better understanding inpatient psychiatric treatment.

Adolescent

Specific components in the etiology, assessment, and treatment of male sexual dysfunctions: controlled outcome studies.

Controlled outcome studies investigating specific components in the etiology, assessment, and treatment of male sexual disorders are reviewed. Premature ejaculators appear just as accurate in assessing their levels of sexual arousal as are men not suffering from this condition. Absolute levels of sexual activity or desire do not seem to be as important for these individuals as has been suggested. Attempts to distinguish organic versus functional erectile failure using the MMPI or historical data have generally proved unsuccessful. Treatment effectiveness in premature ejaculation may be due to elevating the sensory threshold or may be an artifact of simply prescribing more frequent sexual activities. Removing performance demands through cognitive restructuring and sexual communication training shows promise in the treatment of erectile failure.

Ejaculation

Outcome studies of schizoaffective disorders.

Outcome studies of schizoaffective disorder have taught us much about the long-term consequences of the syndrome, and they have provided some indication of the potential usefulness of maintaining "schizoaffective disorder" as a diagnostic category separate from schizophrenia and major affective disorder. In a review of outcome studies that compared schizoaffective patients to schizophrenic or affective patients, we found consistent results despite wide variations in diagnostic criteria, length of followup, and demographic characteristics. Global measures of outcome show that schizophrenic patients are more impaired than schizoaffective patients, who in turn are more impaired than affective patients. However, studies of specific outcome domains such as symptomatology, social functioning, and occupational functioning indicate that schizoaffective disorder is heterogeneous and that subtyping by polarity (e.g., schizoaffective-manic vs. schizoaffective-depressed) accounts for some of this variance. The consistency of these findings in the face of methodological variability suggests that it would be premature to classify schizoaffective patients with schizophrenia or affective disorder, but also that strict diagnostic criteria for schizoaffective disorder are at best preliminary and need to be thoroughly validated.

Bipolar Disorder

An introduction to the evaluation of alcoholism outcome studies.

The results of outcome studies in the field of alcoholism have been challenged as not providing clear and definitive demonstrations of the effectiveness of alcoholism treatment. This has not been due to studies showing negative results, or to a lack of studies reporting positive results. The difficulty has been that the studies failed to satisfy the standards of experimentally minded methodologists. Many methodological problems with respect to patient, treatment, and criterion variables were described which will require a great deal of basic research for their solution. Others may not be soluble. Drawing a representative sample of alcoholics may not be feasible. Assigning patients at random to treatments and no treatment who are uninterested, who desire particular treatments, or who are in need of specific treatments is impractical and socially unacceptable. Matching or randomly assigning therapists to provide consistent, measured, and equal amounts of specific therapies is similarly unrealistic. Determining what constitutes a good therapeutic outcome may be more of a philosophic than a scientific issue. Applying experimental standards to human outcome studies is inappropriate. The state of the art with respect to evaluating the effectiveness of alcoholism treatment is not ready for experimental investigations or cost-benefits analyses. Despite all these problems, a review of 384 studies indicates that two-thirds of the patients improve following alcoholism treatment. Research studies are increasingly sophisticated. A great deal has been and is continuing to be learned about patient characteristics, treatment methods, and matching patients to treatments.

Alcoholism

Psychotherapy outcome studies do not accurately represent current models of psychotherapy. A proposed remedy.

It is argued that the design of contemporary psychotherapy outcome studies is conceptually incompatible with the models of psychotherapy evaluated in those studies. Contemporary outcome studies are incompatible with psychotherapy models because the outcome studies treat patients with standardized treatments that are assigned on the basis of psychiatric diagnosis rather than with individualized treatments based on a theory-driven psychological assessment of the individual's difficulties. One possible remedy, idiographic outcome studies using a case formulation model of assessment and treatment, is proposed here. It is suggested that this research strategy may narrow the scientist-practitioner gap and make it easier to demonstrate differential outcomes of different treatments.

Follow-Up Studies

Small sample studies: unique contributions for large sample outcome studies.

There are many important issues relevant to the course of illness and outcome of chronic psychiatric disorders that are not readily amenable to investigation with existing large sample, quantitative methodology. For example, complex interactive phenomena that change over time, such as the longitudinal impact of changes in social and work function on levels of symptomatology, are particularly difficult to quantify and assess in large n studies. Small sample studies are advantageous because they offer the ability to examine the longitudinal interplay of a wide range of variables in individual patients. The small n approach is useful for delineating important new course variables, developing models of course change, and generating unique research hypotheses. In this article, small sample "qualitative" methodology is reviewed, and data derived from recent small n, intensive longitudinal studies are presented. Methodological issues related to addressing qualitative data in large sample, controlled studies are addressed, and specific research hypotheses generated by small sample studies for consideration in future large sample outcome studies are suggested. It is concluded that small sample studies offer unique contributions to the understanding of the determinants of course of illness.

Follow-Up Studies

Neglected factors in chronic pain treatment outcome studies--referral patterns, failure to enter treatment, and attrition.

An increasing number of chronic pain treatment outcome studies have appeared in the literature. In general, these studies support the efficacy of multidisciplinary pain programs, as well as specific treatment modalities such as biofeedback and relaxation. Reviews of this literature have tended to be cautiously optimistic. Some concerns, however, have been raised about the methodological adequacy of these studies, particularly in terms of the lack of control groups, the brief duration of follow-up periods, and the vague criteria used for establishing the success of the therapeutic interventions. Other factors that mitigate conclusions regarding the generalizability of the favorable results reported need to be considered. In this paper 3 rarely discussed topics that are implicit within most treatment outcome studies and that need to be given greater attention are examined. These topics include: (1) referral patterns to pain clinics (who are referred to pain clinics, when, and how representative is the referred sample?); (2) failure to enter treatment (e.g., exclusion criteria, lack of available financial support to cover the cost of treatment, patient's refusal to accept recommendations), and consequently, the representativeness of the treated sample; and (3) patient's attrition. In this paper we discuss each of these factors as they underscore important qualifications that have to be made in evaluating treatment outcome studies.

Chronic Disease

Nonoperative treatment of herniated lumbar intervertebral disc with radiculopathy. An outcome study.

The functional outcome of patients with lumbar herniated nucleus pulposus without significant stenosis was analyzed in a retrospective cohort study. Inclusion criteria were as follows: 1) a chief complaint of leg pain, primarily; 2) a positive straight leg raising (SLR) at less than 60 degrees reproducing the leg pain; 3) a computed tomography (CT) scan demonstrating a herniated nucleus pulposus without significant stenosis by a radiologist's reading, which was also confirmed by the authors; 4) a positive electromyogram (EMG) demonstrating evidence of radiculopathy; and 5) response to a follow-up questionnaire. All patients had undergone an aggressive physical rehabilitation program consisting of back school and stabilization exercise training. Of a total of 347 consecutively identified patients, 64 patients with an average follow-up time of 31.1 months met the inclusion criteria and constituted the study population. They were sent questionnaires that inquired about activity level, pain level, work status, and further medical care. The patients with neurologic loss, extruded discs, and those seeking a second opinion regarding surgery were identified and subgrouped. Results for the total group included 90% good or excellent outcome with a 92% return to work rate. For the subgroups with extruded discs and second opinions, 87% and 83% had good or excellent outcomes, respectively, all (100%) of whom returned to work. Sick leave time for these subgroups was 2.9 months (+/- 1.4 months) and 3.4 months (+/- 1.7 months), respectively. These results compared favorably with previously published surgical studies. Four of six patients who required surgery were found to have stenosis at operation.(ABSTRACT TRUNCATED AT 250 WORDS)

Adult

Handicraft or interactional groups: a comparative outcome study of neurotic inpatients.

A comparative outcome study of 2 contrasting activity-based groups with 80 hospitalized nonpsychotic patients is described. One group focused on activities designed to evoke emotional or interpersonal reactions followed by a subsequent reflection. The other focused on handicrafts and non-emotionally challenging activities. Ego strength was also measured with an instrument developed in conjunction with the study. The 2 groups were demonstrated to be consistent with the preconditions and significantly different by independent scoring of videotaped sessions. There was a greater rated therapeutic gain in the interactional group at discharge, but patient ratings did not differ between groups. There were no differences at follow-up between the groups. Measured ego strength strongly predicted outcome after correcting for the initial symptom levels. Ego strength did not interact with activity type. Clinical diagnosis did not predict differential outcome. The groups had no differential effects on specific symptom clusters or social functioning.

Adult

EMG biofeedback treatment of torticollis: a controlled outcome study.

Successful treatment of torticollis with electromyographic (EMG) biofeedback has been reported in a number of single case and single group studies. The present investigation represents the first controlled outcome study. Twelve torticollis patients were randomly assigned to EMG biofeedback or relaxation training and graded neck exercises (RGP). The procedure involved three sessions of baseline assessment, 15 sessions of EMG BF or RGP, 6 sessions of EMG BF or RGP plus home-management, 6 sessions of home-management alone, and follow-up 3 months after the end of treatment. A variety of outcome measures were used including physiological (EMG from the two sternocleidomastoid muscles, skin conductance level), behavioral (angle of head deviation, range of movement of the head), and self-report (depression, functional disability, body concept), therapist and "significant other" reports and independent observer assessment of videos. In both groups, neck muscle activity was reduced from pre- to posttreatment. This reduction was greater in the EMG biofeedback group. There was evidence of feedback-specific neck muscle relaxation in the EMG biofeedback group. Therefore, the outcome was not due to nonspecific factors and could be attributed to feedback-specific effects. Changes in skin conductance level showed that neck muscle relaxation was not simply mediated by a general reduction of "arousal." Significant improvements of extent of head deviation, and range of movement of the head, as well as reductions of depression were present, which were not different in the two groups. At the end of treatment, no patient was asymptomatic. Any therapeutic benefit was generally maintained at follow-up. The results and the procedural simplicity of RGP make the issue of cost-efficacy of EMG biofeedback a pertinent one. Further controlled outcome studies of EMG biofeedback treatment of torticollis with larger samples are required.

Adult

The Medical Outcomes Study. An application of methods for monitoring the results of medical care.

The Medical Outcomes Study was designed to (1) determine whether variations in patient outcomes are explained by differences in system of care, clinician specialty, and clinicians' technical and interpersonal styles and (2) develop more practical tools for the routine monitoring of patient outcomes in medical practice. Outcomes included clinical end points; physical, social, and role functioning in everyday living; patients' perceptions of their general health and well-being; and satisfaction with treatment. Populations of clinicians (n = 523) were randomly sampled from different health care settings in Boston, Mass; Chicago, Ill; and Los Angeles, Calif. In the cross-sectional study, adult patients (n = 22,462) evaluated their health status and treatment. A sample of these patients (n = 2349) with diabetes, hypertension, coronary heart disease, and/or depression were selected for the longitudinal study. Their hospitalizations and other treatments were monitored and they periodically reported outcomes of care. At the beginning and end of the longitudinal study, Medical Outcomes Study staff performed physical examinations and laboratory tests. Results will be reported serially, primarily in The Journal.

Adult

Control groups for psychosocial intervention outcome studies.

In psychosocial outcome research, as contrasted to pharmacologic research, control groups receiving inert treatment, designed to raise expectations but otherwise provide no service, are almost never indicated; this is true because of methodologic as well as ethical reasons. Four types of comparisons suffice as alternatives: treatment versus no treatment, treatment versus minimal treatment, treatment A versus treatment B, and dismantling. When choices are made among these types of comparisons with power analysis and eight other factors taken into account, the questions of outcome research should be answerable with maximum economic efficiency, with maximum benefit to subjects, and without deception.

Costs and Cost Analysis

Concordance of multiple assessments of the outcome of schizophrenia. On defining the dependent variable in outcome studies.

Concordance of multiple assessments of the outcome of schizophrenia are examined from (1) concordance of three different assessments of mental status, and (2) concordance of four different dimensions of outcome. Findings are related to the methodological issue of defining the dependent variable in outcome studies. When the assessment instruments were compared, discordance in overall assessment happened because each instrument taps varying aspects of symptomatology, suggesting that outcome is somewhat instrument-related. Although adjustment in mental status is correlated with social adjustment and role performance, a patient's status at follow-up bears little relationship to rate of rehospitalization or to consumer satisfaction with treatment. A differential impact of the same predictor variables on four dimensions of outcome underscores the discordance in multiple assessments. Since treatment outcome is multifaceted and multidertermined, multiple assessments must continue as vital procedures.

Adult

One year's experience of major trauma outcome study methodology.

OBJECTIVE: To assess the feasibility and the validity of an audit using major trauma outcome study methods in an accident and emergency department. DESIGN: Prospective audit of all cases of trauma in patients admitted to a hospital from an accident and emergency department. SETTING: Accident and emergency department in a teaching hospital. PATIENTS: 1577 Patients admitted with trauma, of whom 695 met the inclusion criteria for the study--that is, were admitted for more than three days, or admitted to intensive care, or died. 17 Patients were excluded because of failure to trace their notes. OUTCOME MEASURES: Review of case notes with TRISS (trauma score, injury severity score) methodology to compare expected and observed survival. RESULTS: Most (421/678) admissions were due to single orthopaedic injury. Serious injury was uncommon with only 43 patients having injury severity scores greater than 15. The calculated probability of survival matched the observed outcome for most of the seriously injured patients, with only two unexpected deaths. However, 36 of the 61 deaths in the 678 patients occurred in elderly patients with a fractured neck of the femur, and all of these patients had a high probability of survival predicted by TRISS methodology. CONCLUSIONS: Application of TRISS methodology seems to be valid for seriously injured patients except for elderly patients with single orthopaedic injuries, in whom there were major differences between observed and expected outcomes. Using outcome norms from the United States may not be applicable for this group. IMPLICATIONS: Audit of management of major injuries should be carried out by every hospital, and the methodology of the major trauma outcome study is an excellent system for carrying out such audit. The study of all patients admitted with trauma requires appreciable extra resources, but most hospitals should be able to monitor the care of seriously injured patients as their numbers are much fewer.

Adolescent

Acceptance, attrition, and outcome in an outpatient treatment programme for alcoholics. A comparison between a randomized and a non-randomized process-outcome study.

Acceptance, attrition and outcome of the same outpatient treatment programme for alcoholics was studied first in a non-randomized and later in a randomized study. There was a lower (15%) rate of acceptance and a higher (19%) rate of attrition in the randomized study. Among treatment completers drinking outcome during the third year after commencement of treatment did not differ between the non-randomized and the randomized study. It is concluded that the characteristics of subjects accepting a randomly assigned study should always be clearly presented. The absence of a patient-therapist matching in a randomized design could negatively effect attrition and probably also acceptance of treatment, while it seems to be less influential on outcome in those completing therapy.

Adult

The Major Trauma Outcome Study: establishing national norms for trauma care.

The Major Trauma Outcome Study (MTOS) is a retrospective descriptive study of injury severity and outcome coordinated through the American College of Surgeons' Committee on Trauma. From 1982 through 1987, 139 North American hospitals submitted demographic, etiologic, injury severity, and outcome data for 80,544 trauma patients. Motor vehicle related injuries were most frequent (34.7%). Twenty-one per cent of patients had penetrating injuries. The overall mortality rate was 9.0%. The mortality rate for direct admissions was strongly related to the presence of serious head injury, 5.0% and 40.0%, when head injuries were less than or equal to AIS (Abbreviated Injury Scale) 3 or greater than or equal to AIS 4, respectively. Survival probability norms use the Revised Trauma Score, Injury Severity Score, patient age, and injury mechanism. Patients with unexpected outcomes were identified and statistical comparisons of actual and expected numbers of survivors made for each institution. Results provide a description of injury and outcome and support evaluation and quality assurance activities.

Adolescent