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.

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.

The ORTHO BC-SAT--a satisfaction questionnaire for women using hormonal contraceptives.

OBJECTIVE: To assess the reliability and validity of the ORTHO Birth Control Satisfaction Assessment Tool (ORTHO BC-SAT). DESIGN: 339 women using 1 of 4 hormonal birth control methods (oral contraceptives, transdermal patch, vaginal ring, injections), completed the questionnaire 1-2 times. MATERIALS AND METHODS: The questionnaire was developed based on findings from the literature, focus groups, and interviews. Internal consistency reliability, test-retest reliability, construct validity, and known groups validity were evaluated. RESULTS: Based on variable clustering, 8 domains were identified (Ease of Use/Convenience, Compliance, Lifestyle Impact, Symptom/Side Effect Bother, Menstrual Impact, Future Fertility Concerns, Assurance/Confidence, Overall Satisfaction). Internal consistency reliability was demonstrated with Cronbach's alpha values ranging from 0.70 to 0.89. All multi-item scales reported acceptable test-retest reliability (0.79-0.87). Construct validity was demonstrated by support of a hypothesized pattern of correlations. Known groups validity was confirmed by examining scale scores of women categorized by levels of symptom bother. As expected, women with the least amount of bother reported higher scores on all satisfaction scales than those with higher bother (p < 0.0001), except on Future Fertility Concerns (p = 0.27). CONCLUSION: Our results support the reliability and validity of the ORTHO BC-SAT. It may be used in future studies to evaluate satisfaction among hormonal contraceptive users.

Gout Assessment Questionnaire: Initial results of reliability, validity and responsiveness.

We evaluated the psychometric properties of a new gout-specific patient reported outcomes questionnaire. The Gout Assessment Questionnaire (GAQ) and the SF-36 were administered to 126 subjects in a multicenter Phase II program of febuxostat, an investigational treatment for hyperuricemia (serum uric acid >8.0 mg/dl) in patients with chronic gout. The questionnaire was administered at baseline and 1, 6 and 12 months later. The majority of subjects, mean age 54 years, were male, Caucasian and had experienced a gout flare within the last year. Seven domains were identified, all met criteria for reliability and validity. Cronbach's alpha ranged from 0.78 to 0.97. Pearson correlations between GAQ and SF-36 scales were generally low to moderate, with the highest correlation between Gout Pain and Severity and SF-36 Bodily Pain, r = 0.45. Guyatt's statistic (measure of responsiveness) ranged from 0.24 to 1.00 at 12 months. Minimal clinically important differences ranged from 2 (Gout Concern) to 10 points (Productivity). The GAQ has acceptable psychometric properties. Further research is required to confirm results, which may provide more information to improve the GAQ for use in clinical trials.

Health-Related quality-of-life and quality-days incrementally gained in symptomatic nonerosive GERD patients treated with lansoprazole or ranitidine.

Eight-hundred forty-nine patients with symptomatic nonerosive GERD from two clinical trials of lansoprazole 15 mg daily (LAN 15) and lansoprazole 30 mg daily (LAN 30) vs ranitidine 150 mg twice a day (RAN 150) completed a health-related quality-of-life (HRQoL) questionnaire at baseline and four and eight weeks after treatment. The questionnaire included the Short-Form 12, GERD symptoms, eating symptoms, social restrictions, problems with sleep, work disability, treatment satisfaction, and associated importance weights items. Both LAN groups reported greater, although not significant, improvement from baseline to week 8 versus RAN 150 in the majority of HRQoL scales. Treatment satisfaction was significantly higher at week 8 in both LAN groups. Quality-days incrementally gained analysis showed that both LAN groups gained significantly more quality days than RAN 150. Patients taking lansoprazole 15 or 30 mg daily reported better outcomes than those receiving ranitidine 150 twice a day over the eight-week study.

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.

A new treatment satisfaction measure for asthmatics: a validation study.

The Patient Satisfaction with Asthma Medication (PSAM) questionnaire was developed because no treatment satisfaction questionnaire could be identified that was comprehensive yet brief enough for use in clinical trials. Adult moderate asthmatics residing in Canada using an inhaled medication (either salmeterol, formoterol, or albuterol) self-administered the questionnaire, which also included the Asthma Quality of Life Questionnaire (AQLQ). A total of 53 asthmatics (70% female, 45% married, mean age: 47 years) completed the questionnaire. Using variable clustering, four PSAM scales were identified: Inhaler Properties, Comparison with Other Medications, Overall Perception of Medication, and Relief. Internal-consistency reliability provided evidence of reliability and lack of redundancy (Cronbach's Alpha: 0.82-0.88). Test-retest reliability was acceptable (ICC values at or near 0.70). As expected, interscale PSAM correlations were moderate to high; correlations between the PSAM and the AQLQ were low to moderate. To assess known groups validity, respondents were categorized by self-reported degree of asthma control: 'very well controlled', 'somewhat controlled', and 'not well controlled'. Significant between-groups differences were found on all PSAM scales except Inhaler Properties. Patients categorized as 'very well controlled' tended to report highest PSAM scale scores. The PSAM questionnaire demonstrated reliability and validity in moderate asthmatics. Responsiveness should be assessed in future, prospective studies.

A comparison of two approaches for assessing patient importance weights to conduct an Extended Q-TWiST analysis.

OBJECTIVE: Patient-centered methods for evaluating treatments require validated preference-elicitation techniques. We describe the validation of two preference-elicitation approaches for use in an Extended Q-TWiST treatment evaluation. The first method was an "idiographic" approach, which attempts to capture intra-individual differences in the degree to which each domain distracted from and interfered with life activities. The second method, a Likert-scaled approach, asks patients to evaluate the importance of each quality-of-life (QOL) domain. METHODS: Patient-reported QOL and preferences were assessed in participants with gastroesophageal reflux disease at baseline (n = 172), one week (n = 25), and 4 weeks after baseline (n = 100). RESULTS: Both approaches demonstrated high internal consistency and the ability to discriminate known groups based on reported pain and number of days with symptoms. The idiographic approach exhibited responsiveness, although it was more highly correlated with QOL than the Likert-scaled approach. The Likert-scaled approach had good face validity but demonstrated low reliability compared to the idiographic approach. CONCLUSIONS: Both preference-elicitation methods exhibited promise as well as limitations. Future research should focus on increasing the reliability of the Likert-scaled approach, reducing the overlap between the idiographic approach and QOL, and examining the relationship between reliability and responsiveness for a range of illness trajectories.

Development of a health-related quality-of-life questionnaire for individuals with gastroesophageal reflux disease: a validation study.

This study was conducted to test the psychometric properties of a newly developed health-related quality-of-life (HRQoL) questionnaire. A total of 172 symptomatic GERD patients completed a 57-item questionnaire (containing the SF-12; symptom frequency/bothersomeness; problems related to activities, sleep, work disability; overall HRQoL; and treatment satisfaction) at baseline, week 1 [retest (N = 25)], and week 4 [follow-up (N = 100)]. Internal-consistency reliability was acceptable for most scales (range: 0.74-0.92). Test-retest reliability was acceptable for most scales (ICC: 0.74-0.85). Construct validity was demonstrated based on observed correlations. Known-groups validity was upheld, as patients who experienced more symptom days and patients who reported higher pain reported worse HRQoL than those with less symptoms or less severe pain. When categorized according to change in pain severity, Guyatt's statistic for the "improved" and "worse" groups demonstrated responsiveness, although many of the scales for the "stable" group were also responsive. In conclusion, the HRQoL questionnaire was found to be reliable, valid, and responsive.

A health-related quality-of-life instrument for symptomatic patients with endometriosis: a validation study.

OBJECTIVE: This study was designed to evaluate the reliability, validity, and responsiveness of a newly developed, health-related quality-of-life measure. STUDY DESIGN: A total of 137 women (122 from a Phase III clinical trial and 15 from a private practice setting) with endometriosis completed the questionnaire several times. RESULTS: Reproducibility and internal-consistency reliability were acceptable with intraclass correlation coefficients ranging from 0.94 to 1.00 and Cronbach's alpha coefficients ranging from 0.84 to 0.97. Construct validity was demonstrated on the basis of correlations between items and scales. Health-related quality of life varied in a consistent manner according to clinician-rated measures of pelvic pain and dysmenorrhea and patient-reported levels of endometriosis pain, but no relationship emerged according to the revised American Fertility Society classification. In general, the questionnaire was moderately to highly responsive to change. CONCLUSION: This is the first comprehensive health-related quality-of-life questionnaire available for use with endometriosis patients that has demonstrated reliability, validity, and responsiveness.

Quality of life, health care utilization, and costs among women undergoing hysterectomy in a managed-care setting.

OBJECTIVE: We compared quality of life, utilization, and costs for women undergoing elective abdominal, laparoscopically assisted vaginal, or vaginal hysterectomy within a managed-care organization. STUDY DESIGN: A prospective study of 287 women who underwent an elective hysterectomy was performed. RESULTS: Patients undergoing a vaginal hysterectomy reported returning to normal activity levels sooner and had more favorable quality-of-life scores than did those undergoing laparoscopically assisted vaginal hysterectomy or abdominal hysterectomy. Laparoscopically assisted vaginal hysterectomy was often nearly as favorable as vaginal hysterectomy, particularly at 28 days after the operation, whereas the abdominal hysterectomy group consistently reported the poorest postoperative quality-of-life scores. No significant differences were noted in utilization or costs in the 60-day preoperative period, whereas hospitalization and postoperative costs were highest among the abdominal hysterectomy group and lowest for those undergoing a vaginal hysterectomy. CONCLUSIONS: Vaginal hysterectomy resulted in better quality-of-life outcomes and lower utilization and costs compared with laparoscopically assisted vaginal or abdominal hysterectomy.

Assessing health-related quality-of-life and health state preference in persons with obesity: a validation study.

The objective of this study was to assess the reliability, validity and responsiveness of a new health-related quality-of-life (HRQOL) measure containing global and obesity-specific domains and an obesity-specific health state preference (HSP) assessment. A total of 417 obese and 'normal' weight individuals completed these assessments. Internal consistency and test-retest reliability were demonstrated, with Cronbach's alpha, intraclass correlation coefficient and kappa values well above the acceptable level for most scales. Construct validity hypotheses were confirmed by examining scale correlations. The normal weight individuals reported statistically significantly better functioning and well-being on the majority of the HRQOL scales and HSP than obese individuals. Guyatt's statistic of responsiveness was moderate to high for all the scales and items in the weight-loss and weight-gain groups; however, many of the scales and items in the weight-stable group also displayed responsiveness. The results of this study support the reliability and validity of these assessments. However, further testing is needed to evaluate the responsiveness of both assessments in a weight-stable group.