How can we improve the experiences of patients and families who request medical assistance in dying? A multi-centre qualitative study.

BACKGROUND: Medical assistance in dying has been available in Canada for 5 years, but it is unclear which practices contribute to high-quality care. We aimed to describe patient and family perspectives of quality of care for medical assistance in dying. METHODS: We conducted a multi-centre, qualitative descriptive study, including face to face or virtual one-hour interviews using a semi-structured guide. We interviewed 21 english-speaking patients found eligible for medical assistance in dying and 17 family members at four sites in Canada, between November 2017 and September 2019. Interviews were de-identified, and analyzed in an iterative process of thematic analysis. RESULTS: We identified 18 themes. Sixteen themes were related to a single step in the process of medical assistance in dying (MAID requests, MAID assessments, preparation for dying, death and aftercare). Two themes (coordination and patient-centred care) were theme consistently across multiple steps in the MAID process. From these themes, alongside participant recommendations, we developed clinical practice suggestions which can guide care. CONCLUSIONS: Patients and families identified process-specific successes and challenges during the process of medical assistance in dying. Most importantly, they identified the need for care coordination and a patient-centred approach as central to high-quality care. More research is required to characterize which aspects of care most influence patient and family satisfaction.

High-frequency oscillation in early acute respiratory distress syndrome.

BACKGROUND: Previous trials suggesting that high-frequency oscillatory ventilation (HFOV) reduced mortality among adults with the acute respiratory distress syndrome (ARDS) were limited by the use of outdated comparator ventilation strategies and small sample sizes. METHODS: In a multicenter, randomized, controlled trial conducted at 39 intensive care units in five countries, we randomly assigned adults with new-onset, moderate-to-severe ARDS to HFOV targeting lung recruitment or to a control ventilation strategy targeting lung recruitment with the use of low tidal volumes and high positive end-expiratory pressure. The primary outcome was the rate of in-hospital death from any cause. RESULTS: On the recommendation of the data monitoring committee, we stopped the trial after 548 of a planned 1200 patients had undergone randomization. The two study groups were well matched at baseline. The HFOV group underwent HFOV for a median of 3 days (interquartile range, 2 to 8); in addition, 34 of 273 patients (12%) in the control group received HFOV for refractory hypoxemia. In-hospital mortality was 47% in the HFOV group, as compared with 35% in the control group (relative risk of death with HFOV, 1.33; 95% confidence interval, 1.09 to 1.64; P=0.005). This finding was independent of baseline abnormalities in oxygenation or respiratory compliance. Patients in the HFOV group received higher doses of midazolam than did patients in the control group (199 mg per day [interquartile range, 100 to 382] vs. 141 mg per day [interquartile range, 68 to 240], P<0.001), and more patients in the HFOV group than in the control group received neuromuscular blockers (83% vs. 68%, P<0.001). In addition, more patients in the HFOV group received vasoactive drugs (91% vs. 84%, P=0.01) and received them for a longer period than did patients in the control group (5 days vs. 3 days, P=0.01). CONCLUSIONS: In adults with moderate-to-severe ARDS, early application of HFOV, as compared with a ventilation strategy of low tidal volume and high positive end-expiratory pressure, does not reduce, and may increase, in-hospital mortality. (Funded by the Canadian Institutes of Health Research; Current Controlled Trials numbers, ISRCTN42992782 and ISRCTN87124254, and ClinicalTrials.gov numbers, NCT00474656 and NCT01506401.).

Coenrollment in a randomized trial of high-frequency oscillation: prevalence, patterns, predictors, and outcomes*.

OBJECTIVE: Enrollment of individual patients into more than one study has been poorly evaluated. The objective of this study was to describe the characteristics of patients, researchers and centers involved in coenrollment, studies precluding coenrollment, and the prevalence, patterns, predictors, and outcomes of coenrollment in a randomized clinical trial. DESIGN, SETTING, METHODS: We conducted an observational study nested within the OSCILLation for Acute Respiratory Distress Syndrome Treated Early Trial, which compared high-frequency oscillatory ventilation to conventional ventilation. We collected patient, center, and study data on coenrollment in randomized patients. Multilevel regression examined factors independently associated with coenrollment, considering clustering within centers. We examined the effect of coenrollment on safety and the trial outcome. INTERVENTIONS: None. MEASUREMENTS AND MAIN RESULTS: Overall, 127 of 548 randomized patients (23.2%) were coenrolled in 25 unique studies. Coenrollment was reported in 17 of 39 centers (43.6%). Patients were most commonly coenrolled in one additional randomized clinical trial (76; 59.8%). Coenrollment was less likely in older patients (odds ratio, 0.87; 95% CI, 0.76-0.997), and in ICUs with greater than 26 beds (odds ratio, 0.56; 95% CI, 0.34-0.94), and more likely by investigators with more than 11 years of experience (odds ratio, 1.73; 95% CI, 1.06-2.82), by research coordinators with more than 8 years of experience (odds ratio, 1.87; 95% CI, 1.11-3.18) and in Canada (odds ratio, 4.66; 95% CI, 1.43-15.15). Serious adverse events were similar between coenrolled high-frequency oscillatory ventilation and control patients. Coenrollment did not modify the treatment effect of high-frequency oscillatory ventilation on hospital mortality. CONCLUSIONS: Coenrollment occurred in 23% of patients, commonly in younger patients, in smaller centers with more research infrastructure, and in Canada. Coenrollment did not influence patient safety or trial results.

Barriers, Solutions, and Opportunities for Adapting Critical Care Clinical Trials in the COVID-19 Pandemic.

Importance: The COVID-19 pandemic created unprecedented challenges for clinical trials worldwide, threatening premature closure and trial integrity. Every phase of research operations was affected, often requiring modifications to protocol design and implementation. Objectives: To identify the barriers, solutions, and opportunities associated with continuing critical care trials that were interrupted during the pandemic, and to generate suggestions for future trials. Design, Setting, and Participants: This mixed-methods study performed an explanatory sequential analysis involving a self-administered electronic survey and focus groups of principal investigators (PIs) and project coordinators (PCs) conducting adult and pediatric individual-patient randomized trials of the Canadian Critical Care Trials Group during the COVID-19 pandemic. Eligible trials were actively enrolling patients on March 11, 2020. Data were analyzed between September 2023 and January 2024. Main Outcomes and Measures: Importance ratings of barriers to trial conduct and completion, solutions employed, opportunities arising, and suggested strategies for future trials. Quantitative data examining barriers were analyzed using descriptive statistics. Data addressing solutions, opportunities, and suggestions were analyzed by qualitative content analysis. Integration involved triangulation of data sources and perspectives about 13 trials, synthesized by an interprofessional team incorporating reflexivity and member-checking. Results: A total of 13 trials run by 29 PIs and PCs (100% participation rate) were included. The highest-rated barriers (on a 5-point scale) to ongoing conduct during the pandemic were decisions to pause all clinical research (mean [SD] score, 4.7 [0.8]), focus on COVID-19 studies (mean [SD] score, 4.6 [0.8]), and restricted family presence in hospitals (mean [SD] score, 4.1 [0.8]). Suggestions to enable trial progress and completion included providing scientific leadership, implementing technology for communication and data management, facilitating the informed consent process, adapting the protocol as necessary, fostering site engagement, initiating new sites, streamlining ethics and contract review, and designing nested studies. The pandemic necessitated new funding opportunities to sustain trial enrollment. It increased public awareness of critical illness and the importance of randomized trial evidence. Conclusions and Relevance: While underscoring the vital role of research in society and drawing the scientific community together with a common purpose, the pandemic signaled the need for innovation to ensure the rigor and completion of ongoing trials. Lessons learned to optimize research procedures will help to ensure a vibrant clinical trials enterprise in the future.

How We Can Improve the Quality of Care for Patients Requesting Medical Assistance in Dying: A Qualitative Study of Health Care Providers.

CONTEXT: Since Canada decriminalized medical assistance in dying (MAID) in 2015, clinicians and organizations have developed policies and protocols to implement assisted dying in clinical practice. Five years on, there is little consensus as to what constitutes high-quality care in MAID. OBJECTIVES: To describe MAID clinicians' perspectives on quality of care in MAID, including challenges, successes, and clinical practice suggestions. METHODS: We conducted an exploratory, multicenter, and qualitative study at four Canadian centers. Using a semistructured interview guide, we conducted interviews with 20 health care providers. Interviews were transcribed and deidentified before analysis. Adopting a qualitative descriptive approach, we used a thematic analysis to identify primary and secondary themes in the interviews and practice suggestions to improve quality of care to patients who request MAID. RESULTS: We identified three major themes. 1) Improving access and patient experience: clinicians described struggles in ensuring equitable access to MAID and supporting MAID patients and their families. 2) Supporting providers and sustainability: clinicians described managing MAID workload, remuneration, educational needs, and the emotional impact of participating in assisted dying. 3) Institutional support: descriptions of MAID communication tools and training, use of standardized care pathways, interprofessional collaboration, and human resource planning. Clinicians also described suggestions for clinical practice to improve quality of care. CONCLUSION: Canadian health care providers described unique challenges in caring for patients who request MAID, along with practices to improve the quality of care.

Evolution of practice patterns in the management of acute respiratory distress syndrome: A secondary analysis of two successive randomized controlled trials.

PURPOSE: We sought to examine changes in acute respiratory distress syndrome (ARDS) management over a 12-year period of two successive randomized trials. METHODS: Analyses included baseline data, from eligible patients, prior to influence of trial protocols, and daily study data, from randomized patients, of variables not determined by trial protocols. Mixed linear regressions examined changes in practice year-on-year. RESULTS: A total of 2376 patients met the inclusion criteria. Over the 12-year period, baseline tidal volume index decreased (9.0 to 7.0 ml/kg, p < 0.001), plateau pressures decreased (30.8 to 29.0 cmH2O, p < 0.05), and baseline positive end-expiratory pressures increased (10.8 to 13.2 cmH2O, p < 0.001). Volume-controlled ventilation declined from 29.4 to 14.0% (p < 0.01). Use of corticosteroids increased (baseline: 7.7 to 30.3%; on study: 32.6 to 61.2%; both p < 0.001), as did neuromuscular blockade (baseline: 12.3 to 24.5%; on study: 55.5 to 70.0%; both p < 0.01). Inhaled nitric oxide use increased (24.9 to 65.8%, p < 0.05). We observed no significant change in prone positioning (16.2 to 18.9%, p = 0.70). CONCLUSIONS: Clear trends were apparent in tidal volume, airway pressures, ventilator modes, adjuncts and rescue therapies. With the exception of prone positioning, and outside the context of rescue therapy, these trends appear consistent with the evolving literature on ARDS management.

Ventilation strategy using low tidal volumes, recruitment maneuvers, and high positive end-expiratory pressure for acute lung injury and acute respiratory distress syndrome: a randomized controlled trial.

CONTEXT: Low-tidal-volume ventilation reduces mortality in critically ill patients with acute lung injury and acute respiratory distress syndrome. Instituting additional strategies to open collapsed lung tissue may further reduce mortality. OBJECTIVE: To compare an established low-tidal-volume ventilation strategy with an experimental strategy based on the original "open-lung approach," combining low tidal volume, lung recruitment maneuvers, and high positive-end-expiratory pressure. DESIGN AND SETTING: Randomized controlled trial with concealed allocation and blinded data analysis conducted between August 2000 and March 2006 in 30 intensive care units in Canada, Australia, and Saudi Arabia. PATIENTS: Nine hundred eighty-three consecutive patients with acute lung injury and a ratio of arterial oxygen tension to inspired oxygen fraction not exceeding 250. INTERVENTIONS: The control strategy included target tidal volumes of 6 mL/kg of predicted body weight, plateau airway pressures not exceeding 30 cm H2O, and conventional levels of positive end-expiratory pressure (n = 508). The experimental strategy included target tidal volumes of 6 mL/kg of predicted body weight, plateau pressures not exceeding 40 cm H2O, recruitment maneuvers, and higher positive end-expiratory pressures (n = 475). MAIN OUTCOME MEASURE: All-cause hospital mortality. RESULTS: Eighty-five percent of the 983 study patients met criteria for acute respiratory distress syndrome at enrollment. Tidal volumes remained similar in the 2 groups, and mean positive end-expiratory pressures were 14.6 (SD, 3.4) cm H2O in the experimental group vs 9.8 (SD, 2.7) cm H2O among controls during the first 72 hours (P < .001). All-cause hospital mortality rates were 36.4% and 40.4%, respectively (relative risk [RR], 0.90; 95% confidence interval [CI], 0.77-1.05; P = .19). Barotrauma rates were 11.2% and 9.1% (RR, 1.21; 95% CI, 0.83-1.75; P = .33). The experimental group had lower rates of refractory hypoxemia (4.6% vs 10.2%; RR, 0.54; 95% CI, 0.34-0.86; P = .01), death with refractory hypoxemia (4.2% vs 8.9%; RR, 0.56; 95% CI, 0.34-0.93; P = .03), and previously defined eligible use of rescue therapies (5.1% vs 9.3%; RR, 0.61; 95% CI, 0.38-0.99; P = .045). CONCLUSIONS: For patients with acute lung injury and acute respiratory distress syndrome, a multifaceted protocolized ventilation strategy designed to recruit and open the lung resulted in no significant difference in all-cause hospital mortality or barotrauma compared with an established low-tidal-volume protocolized ventilation strategy. This "open-lung" strategy did appear to improve secondary end points related to hypoxemia and use of rescue therapies. TRIAL REGISTRATION: clinicaltrials.gov Identifier: NCT00182195.

The Work Productivity and Activity Impairment Questionnaire for Patients with Gastroesophageal Reflux Disease (WPAI-GERD): responsiveness to change and English language validation.

BACKGROUND: A validated productivity questionnaire, the Work Productivity and Activity Impairment questionnaire for Gastroesophageal Reflux Disease (WPAI-GERD), exists for Swedish patients with GERD. OBJECTIVE: To assess responsiveness to change of the WPAI-GERD and construct validity of the English language version. METHODS: We used the WPAI-GERD in a before-after treatment clinical study of Canadian GERD patients with moderate or severe symptoms treated with esomeprazole 40 mg once daily for 4 weeks. We measured productivity variables including GERD-specific absence from work, reduced productivity while at work and reduced productivity while carrying out regular daily activities other than work during the preceding week. RESULTS: The analysis included 217 patients, of whom 71% (n = 153) were employed. Before treatment, employed patients reported an average 0.9 hours of absence from work due to GERD and 14.0% reduced work productivity (5.8 hours equivalent) in the previous week, as well as 21.0% reduced productivity in daily activities (all patients). After treatment, the corresponding figures decreased to 0.3 hours, 3.0% (1.1 hours equivalent) and 4.9%, respectively. Thus, the improvement (difference from start of treatment) in productivity was 0.6 hours (p = 0.011) for absence from work and 11.0% units (p < 0.001) for reduced work productivity (4.7 hours equivalent, p < 0.001). This translated into an avoided loss of work productivity of 5.3 hours in total on a weekly basis per employed patient. In addition, a 16.1% unit (p < 0.001) improvement for reduced productivity in activities was observed. Cross-sectional correlation coefficients of WPAI variables with symptoms (range 0.04-0.63) and health-related quality of life (HR-QOL; range 0.02-0.65) supported cross-sectional construct validity. Corresponding change score correlations between WPAI variables and HR-QOL (range 0.05-0.56) supported longitudinal construct validity of the WPAI-GERD while low change score correlations between productivity variables and relevant symptoms (range 0.06-0.34) did not. CONCLUSION: The English version of the WPAI-GERD showed good cross-sectional construct validity, and results indicated that the WPAI-GERD is responsive to change. Although the results also indicated that longitudinal construct validity may be poor, the overall findings suggest that further study of the instrument remains warranted.

Fluids in Sepsis and Septic Shock (FISSH): protocol for a pilot randomised controlled trial.

INTRODUCTION: Observational evidence suggests physiological benefits and lower mortality with lower chloride solutions; however, 0.9% saline remains the most widely used fluid worldwide. Given uncertainty regarding the association of lower chloride on mortality, it is unlikely that practice will change without direct randomised clinical trial (RCT) evidence. This pilot RCT will investigate the feasibility of a large-scale trial directly comparing low chloride with high chloride fluids in patients with septic shock. METHODS AND ANALYSIS: This is a randomised, concealed, blinded parallel-group multicentre pilot trial. We will include adult critically ill patients with septic shock, defined as ongoing hypotension despite 1 L of fluid, or a serum lactate >4 mmol/L, who are within 6 hours of hospital presentation or rapid response team activation. We will exclude patients if they have an aetiology of shock other than sepsis, if they have acute burn injury, elevated intracranial pressure, intent to withdraw life support or previous enrolment in this or a competing trial. Following informed consent, patients will be randomised to a low chloride fluid strategy or a high chloride fluid strategy for the duration of their ICU stay or until 30 days postrandomisation. Clinicians, patients, families and research staff will be blinded. The primary outcome for this trial will be feasibility, assessed by consent rate, recruitment success and protocol adherence. Patient-important clinical outcomes include mortality, receipt of renal replacement therapy, intensive care unit and hospital lengths of stay and surrogate outcomes of incidence of acidosis, hyperkalaemia and acute kidney injury. ETHICS AND DISSEMINATION: This pilot trial will test the feasibility of conducting the main trial, which will examine the effect of high versus low chloride fluids in patients with septic shock on patient-important outcomes. TRIAL REGISTRATION NUMBER: NCT02748382, registered 8 April 2016. PROTOCOL DATE: 1 July 2016.

Management of Acute Respiratory Distress Syndrome and Refractory Hypoxemia. A Multicenter Observational Study.

RATIONALE: Clinicians' current practice patterns in the management of acute respiratory distress syndrome (ARDS) and refractory hypoxemia are not well described. OBJECTIVES: To describe mechanical ventilation strategies and treatment adjuncts for adults with ARDS, including refractory hypoxemia. METHODS: This was a prospective cohort study (March 2014-February 2015) of mechanically ventilated adults with moderate-to-severe ARDS requiring an FiO2 of 0.50 or greater in 24 intensive care units. RESULTS: We enrolled 664 patients: 222 (33%) with moderate and 442 (67%) with severe ARDS. On Study Day 1, mean Vt was 7.5 (SD = 2.1) ml/kg predicted body weight (n = 625); 80% (n = 501) received Vt greater than 6 ml/kg. Mean positive end-expiratory pressure (PEEP) was 10.5 (3.7) cm H2O (n = 653); 568 patients (87%) received PEEP less than 15 cm H2O. Treatment adjuncts were common (n = 440, 66%): neuromuscular blockers (n = 276, 42%), pulmonary vasodilators (n = 118, 18%), prone positioning (n = 67, 10%), extracorporeal life support (n = 29, 4%), and high-frequency oscillatory ventilation (n = 29, 4%). Refractory hypoxemia, defined as PaO2 less than 60 mm Hg on FiO2 of 1.0, occurred in 138 (21%) patients. At onset of refractory hypoxemia, mean Vt was 7.1 (SD = 2.0) ml/kg (n = 124); 95 patients (77%) received Vt greater than 6 ml/kg. Mean PEEP was 12.1 (SD = 4.4) cm H2O (n = 133); 99 patients (74%) received PEEP less than 15 cm H2O. Among patients with refractory hypoxemia, 91% received treatment adjuncts (126/138), with increased use of neuromuscular blockers (n = 87, 63%), pulmonary vasodilators (n = 57, 41%), and prone positioning (n = 32, 23%). CONCLUSIONS: Patients with moderate-to-severe ARDS receive treatment adjuncts frequently, especially with refractory hypoxemia. Paradoxically, therapies with less evidence supporting their use (e.g., pulmonary vasodilators) were over-used, whereas those with more evidence (e.g., prone positioning, neuromuscular blockade) were under-used. Patients received higher Vts and lower PEEP than would be suggested by the evidence.

The impact of illness in patients with moderate to severe gastro-esophageal reflux disease.

BACKGROUND: Gastro-esophageal reflux disease (GERD) is a common disease. It impairs health related quality of life (HRQL). However, the impact on utility scores and work productivity in patients with moderate to severe GERD is not well known. METHODS: We analyzed data from 217 patients with moderate to severe GERD (mean age 50, SD 13.7) across 17 Canadian centers. Patients completed three utility instruments--the standard gamble (SG), the feeling thermometer (FT), and the Health Utilities Index 3 (HUI 3)--and several HRQL instruments, including Quality of Life in Reflux and Dyspepsia (QOLRAD) and the Medical Outcomes Short Form-36 (SF-36). All patients received a proton pump inhibitor, esomeprazole 40 mg daily, for four to six weeks. RESULTS: The mean scores on a scale from 0 (dead) to 1 (full health) obtained for the FT, SG, and HUI 3 were 0.67 (95% CI, 0.64 to 0.70), 0.76 (95% CI, 0.75 to 0.80), and 0.80 (95% CI, 0.77 to 0.82) respectively. The mean scores on the SF-36 were lower than the previously reported Canadian and US general population mean scores and work productivity was impaired. CONCLUSION: GERD has significant impact on utility scores, HRQL, and work productivity in patients with moderate to severe disease. Furthermore, the FT and HUI 3 provide more valid measurements of HRQL in GERD than the SG. After treatment with esomeprazole, patients showed improved HRQL.

The influence of demographic factors and health-related quality of life on treatment satisfaction in patients with gastroesophageal reflux disease treated with esomeprazole.

BACKGROUND: The correlation between treatment satisfaction and demographic characteristics, symptoms, or health-related quality of life (HRQL) in patients with gastroesophageal reflux disease (GERD) is unknown. The objective of this study was to assess correlates of treatment satisfaction in patients with GERD receiving a proton pump inhibitor, esomeprazole. METHODS: Adult GERD patients (n = 217) completed demography, symptom, HRQL, and treatment satisfaction questionnaires at baseline and/or after treatment with esomeprazole 40 mg once daily for 4 weeks. We used multiple linear regressions with treatment satisfaction as the dependent variable and demographic characteristics, baseline symptoms, baseline HRQL, and change scores in HRQL as independent variables. RESULTS: Among the demographic variables only Caucasian ethnicity was positively associated with treatment satisfaction. Greater vitality assessed by the Quality of Life in Reflux and Dyspepsia (QOLRAD) and worse heartburn assessed by a four-symptom scale at baseline, were associated with greater treatment satisfaction. The greater the improvement on the QOLRAD vitality (change score), the more likely the patient is to be satisfied with the treatment. CONCLUSIONS: Ethnicity, baseline vitality, baseline heartburn severity, and change in QOLRAD vitality correlate with treatment satisfaction in patients with GERD.

A comparison of the original chronic respiratory questionnaire with a standardized version.

BACKGROUND AND OBJECTIVES: The chronic respiratory questionnaire (CRQ), a widely used measure of health-related quality of life (HRQL) in patients with chronic airflow limitation, includes an individualized dyspnea domain (patients identify five important activities, and report the degree of dyspnea on a 7-point scale). Because the individualized domain is unwieldy in multicenter clinical trials, we developed a standardized version and tested its discriminative and evaluative properties. METHODS: We enrolled 51 patients who completed the standardized and individualized CRQ before starting a respiratory rehabilitation program, and again 3 months later. We calculated both cross-sectional and longitudinal correlations between the two versions and a number of other HRQL instruments, and tested the relative ability of the individualized and standardized versions of the CRQ to detect improvement with rehabilitation. RESULTS: The results of the individualized questions suggested greater dysfunction (lower scores) than did the standardized questions both at baseline (3.18 vs 3.92, p < 0.001) and follow-up (4.62 vs 4.84, p = 0.051). The standardized dyspnea domain showed superior discriminative validity. While both techniques detected important, statistically significant improvement with rehabilitation (individualized domain mean change, 1.44; 95% confidence interval [CI], 1.11 to 1.77 [p < 0.001]; standardized domain mean change, 0.92; 95% CI, 0.61 to 1.24 [p < 0.01]), the difference in effect was substantial and statistically significant (mean difference, 0.52; 95% CI, 0.22 to 0.82; p = 0.001). The two versions showed comparable longitudinal validity. CONCLUSIONS: A standardized version of the CRQ dyspnea domain improves the cross-sectional validity, maintains longitudinal validity, but reduces the responsiveness. By increasing sample size, investigators can use the more efficient standardized version of the CRQ without compromising validity.

A comparison of narrative and table formats for presenting hypothetical health states to patients with gastrointestinal or pulmonary disease.

Traditionally, quality-of-life researchers describe states of health for patients to rate either in narrative form or in table form, but evidence about which format patients prefer is limited. The authors performed 2 randomized studies to test whether patients prefer the table or narrative format and whether the format of presentation influences how patients rate health states. Approximately three-fourths of patients with gastrointestinal disease or chronic airflow limitation preferred the table format. There were no differences in patients' ratings of 3 described health states or of their own health. Investigators should consider using the table presentation for describing health states to subjects who are not familiar with these states of health.

Do clinical marker states improve responsiveness and construct validity of the standard gamble and feeling thermometer: a randomized multi-center trial in patients with chronic respiratory disease.

BACKGROUND: Optimizing the validity and responsiveness of utility measures will enhance their usefulness in randomized trials. We evaluated the impact of clinical marker state (CMS) rating prior to patients' rating their own health on two utility instruments (feeling thermometer (FT) and standard gamble (SG)) in patients with chronic respiratory disease (CRD). METHODS: We randomized 182 patients with CRD to complete the FT (self-administered) and SG with CMS (FT+/SG+, n=91) or without marker states (FT-/SG-, n=91) before and after undergoing respiratory rehabilitation in a multi-center trial. RESULTS: Use of CMS did not influence baseline utility scores. Improvement after therapy on the scale from 0 (dead) to 1.0 (full health) was 0.04 both in FT+ (p=0.03) and FT- (p=0.02; the difference between FT+ and FT- was 0.00, p=0.83). Improvement on the SG was 0.05 in both SG+ (p=0.08) and SG- (p=0.04; difference between SG+ and SG- 0.00, p=0.95). Correlations with other health related quality of life scores were highest for FT+. CONCLUSION: Administration of CMS did not improve responsiveness of the FT but may have improved construct validity. The SG showed limited construct validity and responsiveness that was not influenced by CMS use.

A randomized multicenter trial to evaluate simple utility elicitation techniques in patients with gastroesophageal reflux disease.

BACKGROUND: Despite recommendations that patients rating their own health using utility and preference measures such as the feeling thermometer (FT) and standard gamble (SG) should also rate hypothetical marker states, little evidence supports marker state use. We evaluated whether the administration of marker states improves measurement properties of the FT and SG. METHODS: We randomized 217 patients with gastroesophageal reflux disease to complete the FT (self-administered) and SG with marker states (FT+ / SG+, n = 112) or without marker states (FT- / SG-, n = 105) before and after 4 weeks of treatment with a proton pump inhibitor, esomeprazole. Patients also completed other health-related quality of life instruments. RESULTS: The use of marker states did not influence baseline utility scores (FT+ 0.66, FT- 0.68; SG+ 0.77, SG- 0.78, on a scale from 0 [dead] to 1.0 [full health]). Improvement after therapy was 0.21 in FT+ and 0.15 in FT- (both P < 0.001; difference between FT+ and FT- = 0.06, P = 0.02). Improvement in SG+ was 0.07 (P < 0.001) and 0.06 in SG- (P = 0.003) (difference between SG+ and SG- = 0.01, P = 0.63). Correlations with other health-related quality of life scores were generally stronger, with some statistically significant differences in correlations, for FT+ compared with FT-, but tended to be weaker for SG+ compared with SG-. CONCLUSION: The administration of marker states improved the responsiveness and validity of the FT but not of the SG. Decisions about administering marker states should depend on whether the FT and SG is of primary interest and the importance of optimal validity and responsiveness relative to competing objectives such as efficiency.