Caitlyn T. Wilke

Health Utilities Using the EQ-5D in Studies of Cancer

Cancer is one of the most frequent disease-specific applications of the EQ-5D. The objective of this review was to summarise evidence to support the validity and reliability of the EQ-5D in cancer, and to provide a catalogue of utility scores based on the use of the EQ-5D in clinical trials and in studies of patients with cancer.A structured literature search was conducted in EMBASE and MEDLINE to identify papers using key words related to cancer and the EQ-5D. Original research studies of patients with cancer that reported EQ-5D psychometric properties, responses and/or summary scores were included.Of 57 identified articles, 34 were selected for inclusion, where 12 studies reported evidence of validity or reliability and 31 reported EQ-5D responses or summary scores. The majority of investigations using the EQ-5D concerned patients with prostate cancer (n = 4), breast cancer (n = 4), cancers of the digestive system (n = 7) and Hodgkin and/or non-Hodgkin lymphoma (n = 3). Mean index based scores ranged from 0.33 (SD 0.4) to 0.93 (SD 0.12) and visual analogue scale scores ranged from 43 (SD 13.3) to 84 (SD 12.0) across subtypes of cancer.A substantial and growing body of literature using the EQ-5D in cancer that supports the validity and reliability of EQ-5D in cancer has emerged. This review provides utility estimates for cancer patients across a wide range of cancer subtypes, treatment regimens and tumour stage(s) that may inform the modelling of outcomes in economic evaluations of cancer treatment.

Use of a preference-based measure of health (EQ-5D) in COPD and asthma.

BACKGROUND EQ-5D is a generic preference-based measure of health that can help to understand the impact of asthma and chronic obstructive pulmonary disease (COPD). The purpose of this paper was to synthesize literature on the validity and reliability of EQ-5D use in studies of asthma and COPD, and estimate EQ-5D utility scores associated with stage of disease. METHODS A structured search was conducted in EMBASE and MEDLINE (1988-2007) using keywords relevant to respiratory disease and EQ-5D. Original research studies in asthma or COPD that reported EQ-5D results and/or psychometric properties were included. RESULTS Studies that reported psychometric properties supported the construct validity, test-retest reliability, and responsiveness of EQ-5D in asthma (seven studies) and COPD (nine studies), although some evidence of ceiling effects were observed in asthma studies. In asthma studies that reported summary scores (n=11), EQ-5D index-based scores ranged from 0.42 (SD 0.30) to 0.93 (SD not reported). In COPD studies (n=8), scores ranged from 0.52 (SD 0.16) to 0.84 (SD 0.15). While few asthma studies reported scores by severity level, sufficient studies in COPD were available to calculate pooled mean utility scores according to GOLD stage: stage I=0.74 (0.62-0.87), stage II=0.74 (0.66-0.83), stage III=0.69 (0.60-0.78) and stage IV=0.61 (0.44-0.77) (most severe). CONCLUSIONS Evidence generally supported the validity and reliability of EQ-5D in asthma and COPD. Utility scores associated with COPD stage may be useful for modeling health outcomes in economic evaluations of treatments for COPD.

Disease-Specific Patient Reported Outcome Tools for Systemic Lupus Erythematosus

PURPOSE Systemic lupus erythematosus (SLE) can significantly affect both health and non-health-related quality of life (HRQOL and non-HRQOL). However, of the existent published patient-reported outcome (PRO) tools, none were developed from US patients, an ethnically diverse population. Furthermore, these tools do not address men with SLE or assess non-HRQOL issues. Herein, we present the development and validation of the Lupus Patient-Reported Outcome tool (LupusPRO) and discuss its clinical utility and research value compared with other PRO tools currently available for SLE. METHODS Beginning with a conceptual framework, items for LupusPRO were generated using feedback from women and men with SLE. The tool underwent iterations based on patient feedback and clinimetric and psychometric analyses. Validity (content, construct, and criterion) and reliability (internal consistency and test-retest) for the 44-item LupusPRO tool are presented. RESULTS Consistent with the conceptual framework, items were identified that were related to HRQOL and non-HRQOL constructs. HRQOL domains included (1) lupus symptoms; (2) physical health (physical function, role physical); (3) pain-vitality; (4) emotional health (emotional function and role emotional); (5) body image; (6) cognition; (7) procreation; and (8) lupus medications. Non-HRQOL domains were (1) available social support and coping; (2) desires-goals; and (3) satisfaction with medical care. Internal consistency reliability (0.68-0.94), test-retest reliability (0.55-0.92), content, construct (r > 0.50 with SF-36), and criterion (r > -0.35 with disease activity) validity were fair to good. CONCLUSIONS LupusPRO is a valid and reliable disease-targeted patient-reported health outcome tool that is generalizable to SLE patients in the United States of varied ethnic backgrounds and either gender.

Lupus-specific health outcome measure for US patients: the LupusQoL-US version

Background: Patient-reported outcomes are valuable for the management of chronic diseases like systematic lupus erythematosus (SLE), but no measures have been validated for use in US-based patients with SLE. Objectives: To adapt and assess the validity and reliability of an SLE-specific quality of life (QoL) measure developed in the United Kingdom, the LupusQoL, for use in US-based patients with SLE. Methods: Debriefing interviews of subjects with SLE guided the language modifications of the tool. The LupusQoL-US, SF-36 and EQ5D were administered. Internal consistency (ICR) and test–retest (TRT) reliability, convergent and discriminative validity were examined. Factor analyses were performed. Results: The mean (SD) age of the 185 subjects with SLE was 42.5 (12.9) years. ICR and TRT of the eight domains ranged from 0.85 to 0.94 and 0.68 to 0.92, respectively. Related domains on the SF-36 correlated with the LupusQoL domains (physical health and physical function r = 0.73, physical health and role physical r = 0.57, emotional health and mental health r = 0.72, emotional health and role emotional r = 0.48, pain and bodily pain r = 0.66, fatigue and vitality r = 0.70, planning and social functioning r = 0.58). Most LupusQoL-US domains could discriminate between subjects with varied disease activity and damage. Principal component analysis disclosed five factors in the US version, with physical function, pain and planning items loading on one factor. Conclusions: These data provide evidence to support the psychometric properties of the LupusQoL-US, suggesting its utility as an assessment tool for patients with SLE in the USA.

Psychometric Properties of the EuroQol-5D and Short Form-6D in Patients with Systemic Lupus Erythematosus

Objective. Health related quality of life (HRQOL) is an important patient-reported outcome in systemic lupus erythematosus (SLE). We evaluated the psychometric properties of 2 widely used preference-based generic HRQOL measures, EuroQol-5D (EQ-5D) and Short Form-6D (SF-6D), among United States patients with SLE. Methods. Patients with SLE enrolled at an academic institution were assessed for self-reported generic HRQOL (EQ-5D, Medical Outcomes Study SF-36), disease activity, and disease damage SF-6D. Physical Component Score (PCS) and Mental Component Score (MCS) were calculated from SF-36. Criterion validity, convergent validity, and known-groups comparisons were evaluated for EQ-5D and SF-6D. Sensitivity to change (t tests, effect size) was evaluated in a subset of the cohort followed longitudinally. Results. One hundred sixty-seven patients with SLE were enrolled. Related domains on the EQ-5D and SF-36 correlated strongly, e.g., mobility and physical functioning (r = 0.60), whereas unrelated domains showed weak to moderate correlation. EQ-5D index, EQ-5D visual analog scale, and SF-6D score correlated strongly among each other as well as with most domains of SF-36. Both EQ-5D and SF-6D indices differentiated among patients of varied disease severity. EQ-5D and SF-6D were found to be sensitive to self-reported change in health but insensitive to change in disease activity longitudinally. Disease activity and damage showed weak correlation with HRQOL measures. Conclusion. The SF-6D and EQ-5D exhibited satisfactory psychometric properties for use among US patients with SLE. Measures of disease activity and damage were weakly correlated with HRQOL, suggesting that HRQOL is an important complementary source of information about patients with SLE.

Outcomes associated with tiotropium use in patients with chronic obstructive pulmonary disease.

BACKGROUND To date, there is mixed evidence on the safety and effectiveness of tiotropium. Our objective was to evaluate the comparative effectiveness of regimens containing tiotropium bromide vs other medication regimens for chronic obstructive pulmonary disease (COPD) in real-world clinical settings. METHODS We conducted a cohort study on 2 separate cohorts with a diagnosis of COPD in the Veterans Affairs health care system. Patients with a diagnosis of COPD prescribed tiotropium and patients in a historic cohort prior to the introduction of tiotropium were selected for comparison using propensity scores, with the base case including scores from 0.1 to 0.4. Outcomes identified during follow-up were all-cause mortality, COPD exacerbations, and COPD hospitalizations. Exposure to COPD medication regimens was defined in a time-varying manner and Cox proportional hazards regression were used to evaluate outcomes. RESULTS For 42 090 patients in the base case, the regimen of tiotropium + inhaled corticosteroids (ICS) + long-acting beta-agonists (LABA) was associated with 40% reduced risk of death (hazard ratio [HR], 0.60; 95% confidence interval [CI], 0.45-0.79) compared with ICS + LABA. This combination was associated with reduced rates of COPD exacerbations (HR, 0.84; 95% CI, 0.73-0.97) and COPD hospitalizations (HR, 0.78; 95% CI, 0.62-0.98). Tiotropium in combination with 2 other medications was associated with increased risk of mortality, exacerbations, and hospitalizations. CONCLUSIONS When used with ICS and LABA, tiotropium use was associated with a decreased risk of mortality compared with treatment with ICS and LABA. However, this result was not consistent in other medication regimens that included tiotropium [corrected].

Economic evaluation of major knee surgery with recombinant activated factor VII in hemophilia patients with high titer inhibitors and advanced knee arthropathy : exploratory results via literature-based modeling

ABSTRACT Objectives: People with severe hemophilia suffer from frequent intra-articular hemorrhages, leading to pain, swelling, reduced flexion, and arthropathy. Elective orthopedic surgery using factor VIII (FVIII) replacement to prevent uncontrolled bleeding has been endorsed as an effective treatment option for patients with severe or advanced hemophilic arthropathy. These surgeries reduce pain, restore mobility and function, and reduce the frequency of recurrent joint bleeds. Unfortunately, some patients with hemophilia develop inhibitors to FVIII, which neutralize FVIII activity and render the use of even massive amounts of FVIII replacement ineffective and surgery very risky. For this reason, elective surgical procedures in high-titer inhibitor patients had largely been abandoned until the introduction of new agents, such as recombinant activated factor VII (rFVIIa, NovoSeven, Novo Nordisk A/S, Denmark). rFVIIa has been shown effective for prophylaxis during elective surgery and has therefore improved the feasibility of orthopedic surgery in hemophilia patients with high-titer inhibitors. The present research explored, from a modified US payer perspective, the direct economic and quality of life benefits of four different elective knee surgeries (total knee replacement [TKR], knee arthrodesis [KA], proximal tibial osteotomy, and distal femoral osteotomy) with rFVIIa coverage in hemophilia patients with high-titer inhibitors. Methods: An exploratory literature-based life-table model was developed to compare the direct medical costs and quality of life of two hypothetical cohorts of high-titer inhibitor patients with frequent bleeding episodes: one undergoing and the other not undergoing elective knee surgery. Knee surgery costs included perioperative rFVIIa costs, inpatient and rehabilitation care, and repeat procedures due to surgery failure, prosthesis loosening or deep infection. Based on efficacy studies, knee surgery was assumed to reduce mean annual bleeding episodes at the affected joint from 9.13 to 1.64. The cost of managing each bleeding episode was estimated at

Exposure and safety of higher doses of recombinant factor VIIa ≥250 μg kg−1 in individuals with congenital haemophilia complicated by alloantibody inhibitors: the Haemophilia and Thrombosis Research Society Registry experience (2004–2008)

15 298. Thus, by reducing bleeding episodes, surgery was expected to result in related cost offsets. All costs were expressed in 2006 US dollars. Surgery was also assumed to result in gains in quality of life by reducing pain and reducing bleeding episodes. The impact of pain reduction on quality of life and utility was estimated by simulating EQ-5D scores for a typical patient with and without knee surgery. Results: Based on the model, average knee surgery costs are predicted to range from a low of

Statistical implications of utility weighted and equally weighted HRQL measures: an empirical study

694 000 (for KA) to a high of

Risk Of Stroke Associated With Ipratropium And Tiotropium Use In Patients With COPD

855 000 (for TKR). However, knee surgery is also expected to reduce the subsequent number of bleeding episodes and resultant costs, leading to long-term costs savings. Due to improvement in pain levels, surgical patients are expected to experience improvements in quality-adjusted life-years (QALYs). Thus, surgery appears to be the preferred strategy (i.e., saves costs and increases QALYs). Based on the assumptions used in the model, the initial cost of knee surgery was offset during the 8th and 10th years for KA and TKR, respectively, with intermediate break-even time for the other surgeries. As expected, cost savings and gains in QALYs increased over time, as well as the cost effectiveness of knee surgery. Specifically, the cost per QALY with KA and TKR fell under