Reliability and validity of the Advanced Basal Cell Carcinoma Index (aBCCdex).

BACKGROUND: Patient-reported outcome (PRO) questionnaires were recently developed specifically for use with patients with advanced basal cell carcinoma (aBCC) and basal cell carcinoma naevus syndrome (BCCNS). OBJECTIVES: To evaluate the measurement properties of PRO questionnaires for use in patients with aBCC or BCCNS. METHODS: In total 129 patients from 10 clinical sites in the U.S.A. and the BCCNS Support Network completed the two newly developed questionnaires multiple times over 3 months. Patients also completed the Skindex-16 and the 12-Item Short-Form Health Survey as collateral measures. Psychometric properties of the questionnaires were evaluated, including internal consistency and test-retest reliability, construct and known-groups validity, and responsiveness. RESULTS: Based on the results of exploratory factor analysis and clinical input, the two newly developed questionnaires were combined into a single questionnaire, called the aBCCdex, which is relevant for patients with both aBCC and BCCNS. The internal consistency reliability was acceptable, and all aBCCdex scale scores correlated significantly with conceptually similar scales. When divided into groups that differed based on scores from collateral measures, aBCCdex scale scores differentiated between groups (known-groups validity) and were responsive to change. CONCLUSIONS: The aBCCdex is a brief and comprehensive questionnaire appropriate for use with patients with aBCC and BCCNS. Its reliability and validity have been confirmed. Further research is necessary to estimate the minimally important difference in a larger patient population.

Relationships between symptoms, symptom bother, and health-related quality of life in patients with overactive bladder taking solifenacin or placebo in the VIBRANT study.

AIMS: Studies of antimuscarinics for overactive bladder (OAB) typically use objective and subjective measures to assess efficacy, as each provides unique information about patient outcome. We evaluated the relationships between changes in diary-documented OAB symptoms and other patient-reported outcome (PRO) measures. METHODS: In VIBRANT, OAB patients received solifenacin (5/10 mg) or placebo for 12 weeks. During the study, patients completed 3-day bladder diaries and other generic and disease-specific PRO measures. Data from both treatment groups were combined (n=738). Categorical changes in diary variables were compared with changes in PRO measures. Partial correlations controlling for treatment and Spearman correlations were also calculated. RESULTS: Categorical improvements in diary variables were significantly associated with greater improvements in PRO measures. Pair-wise comparisons showed that patients with major symptomatic improvements had significantly greater improvements on PROs vs. those with some or no improvement. Odds ratios ranged from 1.52 to 4.09 (p≤0.002). Linear relationships between changes in PRO measures and diary variables were low to moderate but statistically significant (p<0.001). Partial correlations were highest for diary variables and OAB-Questionnaire Symptom Bother. Spearman correlations ranged from 0.170 to 0.450 (p<0.001). CONCLUSIONS: In patients with OAB, changes in objectively measured symptoms of urgency, incontinence and frequency showed low-to-moderate correlations to changes in PRO measures. While providing evidence for similar change patterns in symptoms and patient perceptions, correlations were not high, lending support to the concept that in OAB clinical trials, both bladder diaries and PRO measures are important independent measures of efficacy.

Patient experience in systemic lupus erythematosus: development of novel patient-reported symptom and patient-reported impact measures.

BACKGROUND: Comprehensive assessment of systemic lupus erythematosus (SLE) and its treatment requires patient-reported outcome (PRO) measures to capture impacts and fluctuating symptoms. The objective of this study was to develop PROs, in accordance with the Food and Drug Administration (FDA) PRO Guidance, to assess fluctuations in SLE symptoms and its impact. METHODS: Following independent review board approval, six US rheumatology practices recruited patients with SLE to participate in concept elicitation (CE) interviews, in order to identify important SLE symptoms and their impacts. The SLE Symptom Severity Diary (SSD) and SLE Impact Questionnaire (SIQ) were drafted based on CE interview results and clinician input. The PROs were revised based on patient feedback from cognitive debriefing (CD) interviews, clinician feedback, and a translatability assessment. RESULTS: Forty-one patients completed CE interviews. Commonly-reported symptoms included fatigue (98%), joint pain (93%), and rash (88%). The most frequently reported impact was difficulty with chores/housework (61%). Eighteen patients completed CD interviews. The PROs were considered comprehensive, clear, and relevant.The SSD contains 17 items assessing energy/vitality, joint and muscle pain/stiffness/swelling, flu-like symptoms, cognition, numbness/tingling, skin symptoms and hair loss using an 11-point numeric response scale and a 24-h recall period (with the exception of hair loss). It also evaluates steroid status and dose. The SIQ contains 50 items, uses a 5-point Likert scale and a 7-day recall period, to assess disease impacts including patients' ability to make plans, work, and physical/social/emotional functioning. CONCLUSION: The SSD and SIQ are comprehensive SLE-specific PROs developed in accordance with the FDA PRO Guidance. Following assessment of their measurement properties, they may be useful in clinical studies and clinical practice to measure fluctuations in, and the impact of, symptoms in patients with SLE.

Quality of life and cancer pain: satisfaction and side effects with transdermal fentanyl versus oral morphine.

PURPOSE: To compare pain-related treatment satisfaction, patient-perceived side effects, functioning, and well-being in patients with advanced cancer who were receiving either transdermal fentanyl (Duragesic, Janssen Pharmaceuticals, Titusville, NJ) or sustained-release oral forms of morphine (MS Contin, Perdue Frederick Co, Norwalk, CT, or Oramorph SR, Roxanne Laboratories, Columbus, OH). PATIENTS AND METHODS: A total of 504 assessable cancer patients participated in this cross-sectional, quality-of-life study. Relevant elements of four validated scales were used--the Functional Assessment of Cancer Therapy-General (FACT-G) scale, the Brief Pain Inventory (BPI), the Medical Outcomes Study (MOS) questionnaire, and the Memorial Symptom Assessment Scale (MSAS)--as well as original scales that were developed and validated for this study. RESULTS: The majority of patients in both treatment groups had late-stage (IV/D) cancer. Patients who received transdermal fentanyl were more satisfied overall with their pain medication than those who received sustained-release oral forms of morphine (P = .035). Fentanyl patients also experienced a significantly lower frequency (P < .002) and impact (P < .001) of pain medication side effects. These results occurred despite the fact that cancer patients who received fentanyl were significantly older (P < .001) and had significantly lower functioning and well-being scores (P = .001). Measures of pain intensity, sleep adequacy, and symptoms demonstrated no significant differences between treatment groups. CONCLUSION: These data suggest that patients are more satisfied with transdermal fentanyl compared with sustained-release oral forms of morphine. A lower frequency and reduced impact of side effects with transdermal fentanyl may be one reason cancer patients who receive fentanyl are more satisfied with their pain management.

Untreated anxiety among adult primary care patients in a Health Maintenance Organization.

BACKGROUND: Untreated anxiety may be particularly difficult for primary care physicians to recognize and diagnose because there are no reliable demographic or medical profiles for patients with this condition and because these patients present with a high rate of comorbid psychological conditions that complicate selection of treatment. METHOD: A prospective assessment of untreated anxiety symptoms and disorders among primary care patients. RESULTS: Approximately 10% of eligible patients screened in clinic waiting rooms of a mixed-model health maintenance organization reported elevated symptoms and/or disorders of anxiety that were unrecognized and untreated. These patients with untreated anxiety reported significantly worse functioning on both physical and emotional measures than "not anxious" comparison patients; in fact these patients reported reduced functioning levels within ranges that would be expected for patients with chronic physical diseases, such as diabetes and congestive heart failure. The most severe reductions in functioning were reported by untreated patients whose anxiety was mixed with depression symptoms or disorders. CONCLUSION: Primary care physicians may benefit from screening tools and consultations by mental health specialists to assist in recognition and diagnosis of anxiety symptoms and disorders alone and mixed with depression.

Health-related quality-of-life results from multinational clinical trials of insulin lispro. Assessing benefits of a new diabetes therapy.

OBJECTIVE: To compare health-related quality of life (HRQOL) in patients with diabetes receiving insulin lispro with patients receiving regular human insulin (Humulin R). RESEARCH DESIGN AND METHODS: We performed two randomized comparative studies over a 6-month period (3 months per treatment). Primary analyses used crossover baseline to 3-month changes in HRQOL scores. Ninety-three principal investigators in Canada, France, Germany, and the U.S. participated in these studies. One HRQOL crossover study included 468 patients with type I diabetes; the other HRQOL crossover study included 474 patients with type II diabetes. In both studies, patients were taking insulin at least 2 months before enrollment. Primary outcomes included two generic HRQOL domains, energy/fatigue and health distress, and two diabetes-specific domains, treatment satisfaction and treatment flexibility. Thirty secondary outcomes included both generic and diabetes-specific measures. Secondary outcome domains were controlled for multiplicity in the analyses. RESULTS: Primary analyses showed that treatment satisfaction scores (P < 0.001) and treatment flexibility scores (P = 0.001) were higher for insulin lispro in type I diabetic patients. No other significant treatment differences were detected using the data from these 6-month crossover studies. CONCLUSIONS: Treatment satisfaction and treatment flexibility were significantly improved in patients with type I diabetes using insulin lispro. Other HRQOL findings were comparable for insulin lispro and regular human insulin. Insulin lispro appears to have a measurable impact on lifestyle benefits in patients with type I diabetes, as demonstrated by increased treatment satisfaction and treatment flexibility.

Chronic pelvic pain: prevalence, health-related quality of life, and economic correlates.

OBJECTIVE: To determine the prevalence of chronic pelvic pain in U.S. women aged 18-50 years, and to examine its association with health-related quality of life, work productivity, and health care utilization. METHODS: In April and May 1994, the Gallup Organization telephoned 17,927 U.S. households to identify women aged 18-50 years who experienced chronic pelvic pain, ie, of at least 6 months' duration. Those who reported chronic pelvic pain were surveyed on severity, frequency, and diagnosis; quality of life; work loss and productivity; and health care utilization. RESULTS: Among 5263 eligible women who agreed to participate, 773 (14.7%) reported chronic pelvic pain within the past 3 months. Those who reported chronic pelvic pain had significantly lower mean scores for general health than those who did not (70.5 versus 78.8,P<.05), and 61% of those with chronic pelvic pain reported that the etiology was unknown. Women diagnosed with endometriosis reported the most health distress, pain during or after intercourse, and interference with activities because of pain. Estimated direct medical costs for outpatient visits for chronic pelvic pain for the U.S population of women aged 18-50 years are $881.5 million per year. Among 548 employed respondents, 15% reported time lost from paid work and 45% reported reduced work productivity. CONCLUSION: Frequently, the cause of chronic pain is undiagnosed, although it affects approximately one in seven U.S. women. Increased awareness of its cost and impact on quality of life should promote increased medical attention to this problem.

Health-related quality of life and functional status of patients with rheumatoid arthritis randomly assigned to receive etanercept or placebo.

OBJECTIVE: To compare the functional status and well-being of patients with rheumatoid arthritis (RA) who were randomly assigned to receive placebo, etanercept 10 mg, or etanercept 25 mg during a 26-week, phase III, double-blind clinical trial. BACKGROUND: No single indicator of disease activity, severity, or therapeutic efficacy has been established for RA. During the past decade, health-related quality of life, a multidimensional way to assess physical, emotional, and social aspects of a disease or its treatment, has become an important outcome in RA studies and in assessments of RA drug therapies. METHODS: A total of 234 patients completed the Health Assessment Questionnaire (HAQ), the Short-Form 36 (SF-36) (n = 48 patients), items assessing energy and mental health from the Medical Outcomes Study (MOS), and a single-item rating scale assessing current health (feeling thermometer) at baseline and several times during 6 months. RESULTS: Significant improvements from baseline to last assessment were reported with etanercept versus placebo and in the HAQ Disability Index score (ie, the total HAQ score) and all 8 HAQ categories (P < 0.05), with the exception of grip. Significant improvements with etanercept in the MOS energy and mental health subscales, current health (from the feeling thermometer), and mental and physical function components of the SF-36 were reported (P < 0.05). CONCLUSIONS: Patients receiving 10- or 25-mg doses of etanercept reported significantly better functional status and well-being than did patients receiving placebo.

The impact on unemployment of an intervention to increase recognition of previously untreated anxiety among primary care physicians.

Anxiety is a common, though often unrecognized, problem in primary care settings. This study examines the effect on employment of an intervention designed to attune primary care physicians to previously unrecognized and untreated anxiety. Primary care physicians in a mixed-model health maintenance organization (HMO) were randomized by practice site to groups with (intervention) and without (usual care) intensive one-on-one education about anxiety and periodic feedback about their patients with anxiety. All persons 21-65 years of age presenting to the offices of these primary care providers were screened for anxiety with the SCL-90-R on two occasions. Those meeting the SCL-90-R cutpoints for anxiety and whose medical records provided no evidence of recognition or treatment for a mental health condition within the last 6 months were eligible for the study (n = 637). Of these, 573 (90%) completed two follow-up assessments. The present study evaluates the impact of the intervention aimed at the primary care physicians on the labor force participation rate of the persons with anxiety after 5 months of follow-up. The study also evaluates the impact of the intervention on hours of work and the presence of days spent in bed among the persons with anxiety working at the baseline interview and after 5 months. At baseline, the patients of intervention and usual care physicians with previously unrecognized and untreated anxiety did not differ in labor force participation rates. At the conclusion of the study, the patients of the intervention group physicians had significantly lower rates of labor force participation than those of the usual care group physicians. Among those working both at the beginning and conclusion of the study, the intervention had no impact on hours of work or the presence of days spent in bed. We conclude that attuning physicians may reduce labor force participation rates.

An instrument to measure patient satisfaction with healthcare in an observational database: results of a validation study using data from CaPSURE.

OBJECTIVE: To validate a satisfaction measure for use in longitudinal, prospective studies of patient care. STUDY DESIGN: Patients with biopsy-confirmed prostate cancer (n = 228) who were enrolled in CaPSURE (Cancer of the Prostate Strategic Urologic Research Endeavor) completed a self-administered questionnaire that included a health-related quality-of-life and satisfaction measure. A subset of patients completed the questionnaire again within 30 days. METHODS: The satisfaction measure contained 6 individual subscales: overall satisfaction with care, contact with providers, confidence in providers, communication skills, humaneness, and a summary scale. Six items surveyed patients' willingness to participate in decision making (participatory style), and these were averaged into a single score. Variability, reliability, stability, and validity were evaluated. RESULTS: Responses to the items varied substantially. The overall satisfaction scale demonstrated good internal consistency reliability (Cronbach alpha = 0.82) and moderate test-retest reliability (0.62), and it could discriminate between groups of individuals expected to differ with regard to satisfaction (by age and disease stage). Subscale internal consistency reliability (0.37-0.54) and stability (0.38-0.63) were weaker, suggesting that only a single scale should be reported. The participatory scale performed poorly and could not be recommended for future use. CONCLUSION: The overall satisfaction measure developed for this study demonstrated good reliability and validity and should be useful in other population-based studies in conjunction with other outcome measures.