Validity and responsiveness of the euroqol as a measure of health-related quality of life in people enrolled in an AIDS clinical trial.

OBJECTIVE: Brief utility measures are needed in clinical trials in addition to existing descriptive measures of health-related quality of life (HRQOL). We examined the reliability and validity of the EuroQol (EQ-SD) and MOS-HIV and their responsiveness to HIV-related clinical events. METHODS: Subjects with advanced HIV disease (CD4 < 100) were enrolled in a randomized trial for CMV prophylaxis (n = 990). The EQ-5D includes a weighted sum of five domains (EQ-5D Index) and a visual analog scale (EQ-VAS). The MOS-HIV has 10 subscales and physical (PHS) and mental health summary scores (MHS). Construct validity of the EQ-5D was tested based on hypothesized relationships to subscales of the MOS-HIV. Relative precision and responsiveness to adverse experiences and opportunistic infections (Ols) were compared for the two instruments. RESULTS: Mean age of the patients was 38, 94% were male, 80% white, and 7% had injected drugs. Mean baseline scores for EQ-5D Index and EQ-VAS were 0.80 and 76.0, respectively, 28 and 4% reported maximum scores. Mean MOS-HIV subscales score ranged from 55 (role) to 84 (cognitive); mean PHS and MHS were 47.4 and 49.5, respectively. Correlations between MOS-HIV subscales and EQ-5D Index ranged from 0.45 (role) to 0.63 (pain); correlations with EQ-VAS ranged from 0.33 (cognitive) to 0.66 (health perceptions). Correlations between MOS-HIV PHS and MHS with EQ-5D Index were 0.61 and 0.58; and with EQ-VAS, 0.57 and 0.60, respectively. Responsiveness to adverse experiences was highest for MOS-HIV pain and PHS (effect sizes = 0.9 and 0.4); pain had the highest relative precision (2.4) for adverse experiences: EQ-VAS had the greatest relative precision (1.6) for developing an OI. CONCLUSION: In these patients with advanced HIV disease. EQ-5D showed good construct validity, but there may be a ceiling effect for its EQ-5D Index component. EQ-5D was less responsive to adverse events than the MOS-HIV. However, the EQ-VAS was most sensitive to developing an OI and is likely to be a useful measure of HRQOL for generating QALYs in cost-utility studies involving patients with advanced HIV disease.

Stratification of flare intensity identifies placebo responders in a treatment efficacy trial of patients with osteoarthritis.

OBJECTIVE: Studies evaluating osteoarthritis treatment often use increased arthritis activity ("flare") as a selection criterion, although no standardized assessments are available to quantify flare intensity and little is known about how this criterion affects treatment comparisons. This study evaluated the reliability of a flare assessment and how pretreatment flare intensity impacts conclusions on treatment efficacy. METHODS: Using data from a double-blind, randomized, controlled trial (n = 182), we compared 3 osteoarthritis treatments with placebo in patients who met 3 of 4 flare criteria. The Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) questionnaire was used to document levels of pain, stiffness, and physical functioning at baseline and at the final visit. Following factor analytic evaluation, the flare items were standardized and summed to create a flare intensity index, which was used to identify patient subgroups. Analysis of covariance was applied to compare change in WOMAC scale scores from baseline to final visit for assessment of treatment differences among the flare intensity subgroups. RESULTS: The flare indicators appeared unidimensional. Analyses were stratified by tertiles of flare intensity. Mean WOMAC scores improved in the patients receiving active treatment who were categorized into the 2 lowest flare intensity subgroups, but mean WOMAC scores improved in patients in all 4 treatment groups (active and placebo) in the most intense flare subgroup. CONCLUSION: Patients with higher intensity flares may be more likely to report substantial improvement in functional status regardless of treatment. Failure to account for flare intensity in analyses of data from pain trials with flare-based designs may inflate the risk of Type I and Type II errors in the interpretation of study results.

Reliability, validity and responsiveness of a revised scoring system for the Liverpool Seizure Severity Scale.

This report examines the reliability, validity and responsiveness of a revised scoring system for the Liverpool Seizure Severity Scale (LSSS). The revised scoring system was validated using archival data from an observational study and a randomized controlled study. Factor analyses confirmed that a single dimension captured how patients evaluate the severity of their most severe seizures occurring during a recall period. The revised scoring system repositions the severity score to range from 0 (no seizures) to 100 (most severe possible). Scores based on the new system were reliable, had construct validity (known-groups validity), and were responsive to changes in the patients' epilepsy as noted by their physicians. Results suggest that future epilepsy studies assessing seizure severity should incorporate the revised LSSS scoring system and a modified version of the questionnaire that simplifies self-assessment and analyses. The modified version of the LSSS and its scoring system are appended to this report.

The impact of women's family status on completion of substance abuse treatment.

This study examines the role of family status and demographic characteristics in explaining the nearly 60% dropout rate for women in substance abuse treatment. Data from the administrative record files of the Illinois Office of Alcoholism and Substance Abuse (OASA) for the fiscal year 1996-97 were analyzed for women age 12 or older who completed intake for publicly funded substance abuse treatment and whose outpatient treatment records were closed at year-end. Multivariate logistic regression models found that the likelihood of not completing treatment was greatest for women who were African American, pregnant, had custody of minor children, or were younger than age 21. However, African American women who had children in foster care were more likely to complete treatment. Implications for treatment and research are discussed.

Quality of life and treatment satisfaction after the addition of lamivudine or lamivudine plus loviride to zidovudine-containing regimens in treatment-experienced patients with HIV infection.

BACKGROUND: Assessments of health-related quality of life and treatment satisfaction were conducted as part of a randomised, double-blind, placebo-controlled 52-week trial conducted in Canada, Australia, Europe, and South Africa (CAESAR). The Medical Outcomes Study HIV Health Survey (MOS-HIV) was self-administered during 3 scheduled clinic visits (baseline, week 28 and the end-of-treatment/withdrawal visit). A single question was used at the end of treatment to assess patient satisfaction with study medications. METHODS: Patients were randomly allocated to receive placebo, lamivudine (150 mg twice daily) or lamivudine (150 mg twice daily) plus loviride (100 mg 3 times daily) in addition to their current treatment regimen, which could be either zidovudine monotherapy, or zidovudine in combination with didanosine or zalcitabine at standard dosages. RESULTS: Statistically significant differences across treatment groups were demonstrated for the Physical and Mental Health Summary scores, and for 5 of 10 MOS-HIV subscales (physical functioning, vitality, cognitive functioning, general health perceptions, social functioning). These differences favoured the lamivudine and lamivudine plus loviride groups over the placebo group (p < 0.05). No significant difference was found between the 3 treatment groups with regard to the percentages of patients who were satisfied with their study medication. CONCLUSION: The results suggest that, for treatment-experienced patients with HIV infection and CD4+ counts < 250 cells/mm3, the addition of lamivudine or lamivudine plus loviride to antiretroviral regimens containing zidovudine maintained patient-reported mental and physical health.

Reliability and validity of French, German, Italian, Dutch, and UK English translations of the Medical Outcomes Study HIV Health Survey.

OBJECTIVES: Test the reliability and validity of 5 translations of the 34-item version of the MOS HIV for use in multinational clinical trials. RESEARCH DESIGN: Investigators in five countries followed a standardized protocol and recruited HIV+ patients stratified by disease stage: asymptomatic; symptomatic; and AIDS. During routine clinic visits, patients completed the MOS HIV and a checklist of HIV-related symptoms. Clinicians reported patients' demographics, most recent CD4+ count and disease stage. SUBJECTS: Three hundred and sixty three HIV+ outpatients attending AIDS clinics in The Netherlands, France, Germany, Italy, and England. MEASURES: Dutch, French, German, Italian, and UK English translations of the MOS HIV CD4+ cell count and the SCL-57. RESULTS: All translations recruited roughly equal proportions of each disease stage, although the number of patients recruited differed by translation (n: German = 92, French = 86; Italian = 88; UK English = 72; and Dutch = 25). Internal consistency reliability was similar across translations and adequate (alpha >.70) for all scales except for Mental Health in the French sample. Multi-trait analyses supported structural validity of the MOS HIV scales in each translation. Principal component analysis of scale scores identified 2 dimensions for all translations except German. For all translations, scores were significantly correlated with symptom severity scores but were uncorrelated with CD4+ cell counts. CONCLUSIONS: In general, the 5 translations of the MOS HIV had similar psychometric properties to those reported in the validation study for the original US English version of the MOS HIV. With some revision, these translations promise to provide useful quality of life data from HIV+ subjects in clinical trials.

Impact of zidovudine plus lamivudine or zalcitabine on health-related quality of life.

OBJECTIVE: To assess the impact of treatment with zidovudine plus lamivudine or zalcitabine on health-related quality of life (HRQOL) in patients with HIV. DESIGN: HRQOL assessments were conducted as part of a double-blind, randomized, 24-week (extended to 52 wk) efficacy and safety study. The Medical Outcomes Study HIV Health Survey (MOS-HIV), which assesses 10 physical and psychological domains of HRQOL, was self-administered by patients at baseline and at weeks 16, 32, 52, or at treatment discontinuation. SETTING: Twenty-one outpatient centers in the US, Canada, and Puerto Rico. PATIENTS: The study enrolled 254 HIV-positive patients (CD4+ 100-300 cells/mm3); 206 patients completed the MOS-HIV at baseline and at least once during treatment. Post hoc analyses stratified patients into two subgroups: AIDS (CD4+ < 200 cells/mm3) and non-AIDS (CD4+ > or = 200 cells/mm3). INTERVENTIONS: Patients received zidovudine 200 mg three times daily plus one of the following: lamivudine 150 mg twice daily, lamivudine 300 mg twice daily, or zalcitabine 0.75 mg three times daily. MAIN OUTCOME MEASURE: Change in MOS-HIV scores from baseline to last completed questionnaire. RESULTS: Following an average of 36 weeks of treatment, there were statistically significant differences across treatment groups in mean change scores on the physical functioning, role functioning, and vitality scales, with stable or increased (improved) scores in the zidovudine plus lamivudine 150 mg group and decreased scores in the zidovudine plus zalcitabine and zidovudine plus lamivudine 300 mg groups for most scales. Post hoc analyses found that in the non-AIDS subgroup, only the zidovudine plus lamivudine 150 mg group had increases in mean MOS-HIV scores (on 8 of 10 scales); in the AIDS subgroup, all but two MOS-HIV scores (in the zidovudine plus zalcitabine group) decreased in all three treatment groups. CONCLUSIONS: These results suggest that, of the three combination therapies studied, zidovudine plus lamivudine 150 mg was most likely to maintain or improve HRQOL in HIV-positive patients.

Clinical effectiveness and quality of life with ranitidine vs placebo in gastroesophageal reflux disease patients: a clinical experience network (CEN) study.

BACKGROUND: Gastroesophageal reflux disease (GERD), often characterized as heartburn, is a highly common presenting complaint to family physicians. This study is the first large, prospective, nationwide family practice outpatient evaluation of the effectiveness of the histamine (H2)-receptor antagonist ranitidine as medical therapy for this disorder. METHODS: This randomized, double-blind, placebo-controlled, parallel group, 6-week study was designed to evaluate the effect of ranitidine on clinical outcomes and quality of life in patients with GERD. Eligible patients included those who were at least 18 years old and had at least a 3-month history of heartburn or heartburn therapy and a minimum of 4 days with at least one heart-burn episode in the week preceding the baseline visit. Quality-of-life effects were measured using a general health status instrument and a previously validated heartburn-specific questionnaire. RESULTS: Ranitidine treatment conferred clinically and statistically significant reductions in mean heartburn pain scores within the first 24 hours (P < or = .001) and mean number of heartburn episodes within the first 48 hours (P < or = .001). These reductions were maintained throughout the 6-week trial, during both daytime and nighttime. Compared with patients receiving placebo, patients treated with ranitidine also used significantly fewer doses of antacids (P < or = .003). Further, both ranitidine-treated patients' and their physicians' global assessments of decreases in heartburn severity, as well as clinical improvement on ranitidine, proved superior to those of controls (P < or = .001). The rate of adverse events associated with ranitidine and placebo was low and similar. Ranitidine-treated patients had more favorable scores on the general health status dimensions of physical functioning, bodily pain, and vitality (P < .05), and more favorable scores on all dimensions of the heartburn-specific questionnaire (P < .05). CONCLUSIONS: Twice-daily treatment with ranitidine 150 mg is a valuable therapy for GERD in a typical family practice setting. It reduces the frequency and severity of symptoms within the first 24 to 48 hours of treatment and diminishes the use of nonprescription antacids while improving the quality of life as measured by both a general health status instrument and a disease-specific instrument.

The cost of depression-complicated alcoholism: health-care utilization and treatment effectiveness.

Clinical and epidemiologic evidence suggests that alcoholism complicated by concurrent or a lifetime history of depression is slower to remit and more prone to relapse than uncomplicated alcoholism. Consequently, alcoholics with a history of depressive illness, on average, are likely to use more health care and to have higher treatment costs than those without depression complications. This article contrasts evidence of the suitability of three models for predicting the impact of depression on an alcoholic's health-care use: a null model (assuming no differences), a cumulative-effect model (arguing for a linear increase associated with comorbid depression), and a synergistic model (wherein alcoholism complicated with depression is qualitatively as well as quantitatively different than uncomplicated alcoholism). To test these models, health-care costs and utilization of 491 "pure" alcoholics (those with no history of depression diagnosis) and 199 depression-complicated alcoholics, who received alcohol treatment while enrolled in a self-insured health-care program of a major U.S. manufacturing company, were compared. Results are discussed in terms of the implications for cost containment and the likelihood of relapse among the depression-complicated alcoholism group.

Substance abuse and family illness: evidence from health care utilization and cost-offset research.

Although the substance abuse treatment community recognizes that physical and psychological problems are common among families with a substance-abusing member, third-party funding for comprehensive treatment of the families of substance abusers is limited. Failure to provide treatment for these collateral effects of substance abuse on the family is thought to reduce the efficacy of substance abuse treatment, increase the risk of relapse, and leave untreated secondary pathology among family members. This article presents a review of health care utilization and cost-offset studies of the collateral effects of substance abuse on the family to aid administrators and planners in documenting the economic advantages of comprehensive treatment for the families of substance abusers.