Susan D. Mathias

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 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 Cronbachs 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.

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.

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 (Cronbachs 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.

A Phase 3, Randomized, Double‐Blind, Placebo‐Controlled Study to Determine the Effect of Romiplostim on Health‐Related Quality of Life in Children with Primary Immune Thrombocytopenia and Associated Burden in Their Parents

Chronic immune thrombocytopenia (ITP) in children can negatively impact their health‐related quality of life (HRQoL) and impose a burden on their parents. This study sought to examine the effect of romiplostim on HRQoL and parental burden in children with primary ITP.

The Impact of Infusion Reactions on Oncology Patients and Clinicians in the Inpatient and Outpatient Practice Settings: Oncology Nursesʼ Perspectives

Cancer patients treated with parenteral chimeric or humanized monoclonal antibody agents or chemotherapy are at risk of experiencing an infusion reaction. The impact of infusion reactions can be substantial. Infusion reactions, even when mild, can be disruptive and stressful. In-person interviews with 202 nurses were conducted at the 2005 Annual Oncology Nursing Society Congress to evaluate the impact of infusion reactions on patients and nurses in the inpatient and outpatient practice settings. Results from this survey study suggest that infusion reactions have a significant negative impact on both patients and nurses.

The minimal important difference for measures of urticaria disease activity: Updated findings.

BACKGROUND The Urticaria Patient Daily Diary (UPDD) is a validated patient-reported outcome that captures key measures of urticaria disease activity. OBJECTIVE To update estimates of the minimal important difference (MID) for urticaria disease activity measures in the UPDD, including the weekly itch severity score, weekly number of hives score, weekly average size of largest hive score, and the composite measure of itch severity and number of hives over 7 days, or urticaria activity score 7 (UAS7). METHODS A total of 975 subjects with chronic idiopathic urticaria from three randomized, double-blind, placebo-controlled studies completed the UPDD and other patient-reported outcome assessments (the Dermatology Life Quality Index, Medical Outcomes Study Sleep Scale, the Chronic Urticaria Quality-of-Life Questionnaire, the EuroQoL-5 Dimension Questionnaire) multiple times. MIDs were estimated through a combination of distribution- and anchor-based methods. RESULTS MID estimates ranged from 4.5 to 5.0 for the weekly itch severity score, 5.0 to 5.5 for weekly hives count score, 9.5 to 10.5 for the UAS7, and 4.0 to 4.5 for the weekly size of the largest hive score. CONCLUSION This analysis provided confirmation of the previous MID estimates for the urticaria disease activity measures in the UPDD.

Quality-Days Incrementally Gained: A New Approach for Short-Term Evaluations Based on the Extended Q-Twist Method Illustrated in a Clinical Study of Reflux Disease Patients*

Objective: The objective of this study was to derive a measure of quality-time to be used in treatment evaluations for gastroesophageal reflux disease (reflux disease). Specifically, we sought to refine the Extended Quality-adjusted Time without Symptoms and Toxicities (Q-TWiST) approach and propose a new measure, called Quality-Days Incrementally Gained (QDIG). Design: One hundred and sixty-seven patients with reflux disease randomized to one of two treatments completed a health-related quality-of-life questionnaire at baseline and after four and eight weeks of treatment. The questionnaire contains generic and reflux disease-specific measures and corresponding importance items. The weighted assessment score, fundamental to calculating both the Extended Q-TWiST and the QDIG, was computed using several weighting schemes to determine the most robust and appropriate statistic. Main Outcome Measures and Results: The Extended Q-TWiST was affected by the relative weighting of baseline and follow-up weighted assessment score. The QDIG directly discounts the baseline score and showed the greatest sensitivity to treatment differences. The variance in the Extended Q-TWiST and QDIG were both reduced by using importance weights carried forward from baseline rather than time-varying importance weights, and by using population-weights rather than individual person weights. Conclusions: Careful consideration should be made when deciding to use the Extended Q-TWiST or QDIG approach. Our data suggest the QDIG approach is superior in studies of short duration with heterogeneous populations.

Treatment Satisfaction in Systemic Lupus Erythematosus: Development of a Patient-Reported Outcome Measure

ObjectiveThe aim of this study was to develop a patient-reported outcome measure specific for systemic lupus erythematosus (SLE) to assess patient satisfaction with treatment, treatment options, and medical care. MethodsPatients with SLE were recruited from four US rheumatology practices. Concept elicitation interviews identified aspects that patients considered important and relevant regarding satisfaction with treatment and medical care. Concept elicitation interviews and clinical input were used to draft the Lupus Satisfaction Questionnaire (LSQ). A second cohort of patients with SLE participated in combined concept elicitation/cognitive debriefing interviews, after which the LSQ was revised. ResultsFourteen patients completed concept elicitation interviews: 93% were female, 57% were white, and 85% had moderate/severe SLE. Current treatments included hydroxychloroquine (93%), steroids (79%), and belimumab (57%), and 43% were biologic naive. Patients were generally satisfied with their treatment and medical care; however, they were dissatisfied with treatment adverse effects and the number of available treatment options. Cognitive debriefing interviews (n = 8) demonstrated that the LSQ was comprehensive, clear, and relevant; therefore, only minor revisions were made to the questionnaire. The LSQ assesses satisfaction with current SLE treatments (25 items), medical care (11 items), and insurance coverage (3 items). The draft LSQ was evaluated in 195 adults with SLE. Fifty-eight percent of patients reported that they were “somewhat satisfied” with their SLE treatment. ConclusionsThe LSQ has been developed to assess treatment satisfaction among patients with SLE. Following further testing to support its validity and reliability, it will provide a useful tool to facilitate assessment of satisfaction with treatments for SLE and help inform treatment decisions.