Development and Validation of a Canine Health-Related Quality of Life Questionnaire and a Human-Canine Bond Questionnaire for Use in Veterinary Practice.

The use of valid questionnaires to assess dogs' health-related quality of life (HRQoL) in veterinary practice can improve canine health outcomes and communications between veterinarians and caretakers of dogs. The Canine HRQoL Questionnaire (Canine HRQoL-Q) and the Human-Canine Bond Questionnaire (HCBQ) were developed and validated to fulfill this need. A literature review, interviews with veterinarians, and focus groups with caretakers were conducted to generate questionnaire items and develop draft questionnaires, which were piloted with caretakers to establish their content validity. Measurement properties were evaluated using data from a prospective survey study (N = 327). Draft Canine HRQoL-Q and HCBQ measures were developed, including a domain structure, items, recall period, and scale/response options. Refinements were made via iterative cognitive interviews with caretakers. When no additional revisions were indicated and content validity was established, the questionnaires were psychometrically tested. Ceiling effects were observed for all items, and factor analyses indicated that the pre-specified domains are appropriate. Internal consistency was demonstrated for the HCBQ (α = 0.79-0.86) and all but the social functioning domain of the Canine HRQoL-Q (α = 0.60). Test-retest reliability for the Canine HRQoL-Q was generally moderate-to-good (with intraclass correlation coefficients (ICCs) > 0.79). Test-retest reliability for the HCBQ was moderate (ICCs: 0.70-0.79) except for the trust domain (ICC: 0.58). Known-groups validity was demonstrated via significant differences (p < 0.05) in scores for health/bonding groups. Convergent validity was supported (r > 0.40) between all domains and the total scores for both questionnaires. The Canine HRQoL-Q and the HCBQ are valid, reliable measures of canine HRQoL for use in veterinary clinics and appear to measure related but distinct concepts that contribute to canine health and wellness.

Finalization and Validation of Questionnaire and Algorithm of SPUR, a New Adherence Profiling Tool.

Purpose: The SPUR (Social, Psychological, Usage and Rational) Adherence Profiling Tool is a recently developed adaptive instrument for assessing key patient-level drivers for non-adherence. This study describes the SPUR questionnaire's finalization and psychometric evaluation. Patients and Methods: Data were collected through an online survey among patients with type 2 diabetes included by general practitioners and diabetologists in France. The survey included four questionnaires, SPUR and three validated adherence measures: BMQ, MARS and ACCEPT. Item-level analysis and a partial credit model (PCM) were performed to refine the response option coding of SPUR items. The final item selection of SPUR was defined using a PCM and a principal component analysis (PCA). Construct validity, concurrent validity and known-groups validity were assessed on the final SPUR questionnaire. Results: A total of 245 patients (55% men, mean age of 63 years) completed the survey remotely and were included in this analysis. Refining response option coding allowed a better discrimination of patients on the latent trait. After item selection, a short, an intermediate, and a long form composed the final SPUR questionnaire. The short form will be used to screen patients for risk and then the other forms will allow the collection of further information to refine the risk assessment and decide the best levers for action. Results obtained were supportive of the construct validity of the forms. Their concurrent validity was demonstrated: moderate to high significant correlations were obtained with BMQ, MARS and ACCEPT scores. Their known-groups validity were shown with a logical pattern of higher scores obtained for patients considered non-adherent and significant differences between the scores obtained for patients considered adherent versus non-adherent. Conclusion: SPUR is a valid tool to evaluate the risk of non-adherence of patients, allowing effective intervention by providing insights into the respective individual reasons for lack of adherence.

The SPUR adherence profiling tool: preliminary results of algorithm development.

OBJECTIVE: The SPUR (Social, Psychological, Usage, and Rational) Adherence Profiling Tool is a recently developed adaptive instrument for measuring key patient-level risk factors for adherence problems. This study describes the SPUR questionnaire's psychometric refinement and evaluation. METHODS: Data were collected through an online survey among individuals with type 2 diabetes in the United States. 501 participants completed multiple questionnaires, including SPUR and several validated adherence measures. A Partial Credit Model (PCM) analysis was performed to evaluate the structure of the SPUR tool and verify the assumption of a single underlying latent variable reflecting adherence. Partial least-squares discriminant analyses (PLS-DA) were conducted to identify which hierarchically-defined items within each dimension needed to be answered by a given patient. Lastly, correlations were calculated between the latent trait of SPUR adherence and other patient-reported adherence measures. RESULTS: Of the 45 candidate SPUR items, 39 proved to fit well to the PCM confirming that SPUR responses reflected one underlying latent trait hypothesized as non-adherence. Correlations between the latent trait of the SPUR tool and other adherence measures were positive, statistically significant, and ranged from 0.32 to 0.48 (p-values < .0001). The person-item map showed that the items reflected well the range of adherence behaviors from perfect adherence to high levels of non-adherence. The PLS-DA results confirmed the relevance of using four meta-items as filters to open or close subsequent items from their corresponding SPUR dimensions. CONCLUSIONS: The SPUR tool represents a promising new adaptive instrument for measuring adherence accurately and efficiently using the digital behavioral diagnostic tool.

Development and validation of the Methotrexate Experience Questionnaire, a new methotrexate oral treatment adherence tool in rheumatoid arthritis.

OBJECTIVE: Despite the development of new biologic therapies, methotrexate (MTX) remains the preferred initial disease-modifying anti-rheumatic drug to treat rheumatoid arthritis (RA). Adherence to disease-modifying anti-rheumatic drugs is suspected to be highly variable potentially leading to reduced treatment effectiveness. This work aimed to develop and validate the Methotrexate Experience Questionnaire (MEQ), a tool to identify and characterize non-adherence to oral MTX. METHODS: MEQ development included a literature review and qualitative interviews with RA patients and physicians in the United States. A retrospective, cross-sectional study using data from Optimum Patient Care Research Database, a large primary care database of electronic medical records in the United Kingdom, was conducted to finalize the MEQ and evaluate its psychometric properties. RESULTS: Three hundred seven e-consented subjects (66% women, mean age of 65 years) completed the MEQ remotely, and were included in this analysis. Item-convergent and divergent validity were generally supportive of the construct validity of the MEQ and Cronbach's alpha of 0.87 supported its reliability. The MEQ Total score presented statistically significant correlations of small to medium size with all selected concurrent scales, as expected; the highest correlation was obtained between the general acceptance score of ACCEPT and the MEQ Total score (0.55, p < 0.001). Known-groups validity was demonstrated as a logical pattern of higher MEQ scores was obtained for patients considered adherent with both the 6- and 12-month Proportion of Days Covered (mean MEQ total score 82.7 for 12-month PDC ≥ 80% against 76.3 for 12-month PDC < 80%, p< 0.0001). Additionally, a pattern of lower MEQ scores was obtained for patients with more severe disease assessed with Routine Assessment of Patient Index Data 3. CONCLUSION: The 24-item MEQ is a reliable and valid instrument to assess the adherence of RA patients taking MTX, potentially improving over historical refill rate metrics by providing insights into the individual reasons for lack of adherence. This information should facilitate clinician-patient discussions and help inform treatment decisions.

Comparing Physician and Patient Perspectives on Prophylactic Treatment with BAY 94-9027 for Severe Haemophilia A: A Post Hoc Analysis.

INTRODUCTION: BAY 94-9027 is a newly developed extended half-life product to treat haemophilia, allowing for fewer injections than with standard products. This post hoc analysis aimed to compare physicians' and patients' opinions on BAY 94-9027 prophylaxis, and explore how qualitative interview data is aligned with the data from the Haemophilia-specific Quality of Life questionnaire for Adults (Haemo-QoL-A). METHODS: Exploratory qualitative interviews were conducted with physicians and patients by phone upon the exit of patients from the PROTECT VIII extension phase following a semi-directed guide. In this post hoc analysis, all transcripts were reviewed and reported concepts were compared to assess the level of concordance between physicians and patients. These qualitative data were compared with the Haemo-QoL-A mean global and subscale scores at baseline and end of main phase (36 weeks later). RESULTS: Ten physicians and 16 patients (mean age 47 years) from Israel, the Netherlands and the USA were interviewed. Significant improvements were reported by all physicians from baseline [e.g. lower frequency of bleeds (80%), improvement in emotional functioning (90%)], which is in concordance with patients' reports. The improved confidence reported by physicians cascaded to greater participation in various activities, resulting in a better perceived emotional state and a significant improvement on the Haemo-QoL-A emotional impact subscale score (p = 0.04) between baseline and end of main phase. Most physicians (80%) reported improvement in bleed frequency, as patients did (88%). Improvement in physical functioning or mobility was not consistently reported in this 8-month study. CONCLUSION: Interviewed physicians and patients generally agreed on the beneficial impact of BAY 94-9027, specifically regarding the increased level of self-confidence in patients and its subsequent positive impact on patients' lives. These findings supported the observed improvement on the Haemo-QoL-A emotional impact subscale. Overall, this study highlights the concordance between physician and patient perspective on the positive experience with BAY 94-9027.

The multidimensional nature of dyspnoea in amyotrophic lateral sclerosis patients with chronic respiratory failure: Air hunger, anxiety and fear.

BACKGROUND: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder leading to chronic respiratory failure. Few studies have investigated ALS-related dyspnoea, and none have characterised the emotional distress it inflicts. We hypothesised that ALS-related dyspnoea has a strong affective component that relates to quality of life. METHODS: This prospective, observational study was conducted in 41 ALS patients >18 with chronic respiratory failure and an indication for noninvasive ventilation (NIV). Dyspnoea was assessed using the Multidimensional Dyspnea Profile (MDP) at baseline and 1 month after NIV initiation. Correlations between scores evaluating the sensory and affective dimensions of dyspnoea and other patient-reported outcomes and pulmonary function tests were analysed. RESULTS: Dyspnoea was described as intense (median [IQR] score on a 0-10 scale: 6.5 [4.0-7.5]). The sensory dimension of dyspnoea was polymorphic, but «air hunger¼ was the most common (48.8%) and the most intense (6 [4-8]) sensory descriptor. In the affective domain, most patients rated «anxious¼ (85.4%) and «afraid¼ (60.9%) above 0. The MDP affective dimension correlated significantly with other patient-reported outcomes, with the strongest correlation being between MDP «anxious¼ and the anxiety component of the Hospital Anxiety Depression Scale (Pearson's R = 0.70). One month after initiation of NIV, dyspnoea during unassisted breathing was described in virtually the same terms, particularly the affective dimension. DISCUSSION: ALS-related dyspnoea is intense and fear-provoking, persists during unassisted breathing between NIV sessions, and significantly impacts health-related quality of life. This study highlights the need for increased awareness of and research into ALS-related dyspnoea.

Sustained quality of life improvement after intracoronary injection of autologous bone marrow cells in the setting of acute myocardial infarction: results from the BONAMI trial.

PURPOSE: Cardiac cell therapy is a promising treatment for acute myocardial infarction (AMI), leading to cardiac function improvement. However, whether it translates into quality of life (QoL) improvement is unclear. We hypothesized that administration of bone marrow cells (BMC) to patients with AMI improves QoL. METHODS: In the multicenter BONAMI trial (NCT00200707), patients with reperfused AMI and decreased myocardial viability were randomized to intracoronary autologous BMC infusion (n = 52) or state-of-the-art therapy (n = 49). QoL data, derived from the Minnesota Living with Heart Failure questionnaire (MLHFQ), were obtained 1, 3, and 12 months after AMI and analyzed using a Rasch-family model. RESULTS: Using this model, QoL improved over time in the BMC group (p = 0.025) but not in the control group. Furthermore, the BMC-group patients displayed a better QoL than the control-group patients at 3 and 12 months post-AMI (p = 0.034 and p = 0.003, respectively). These findings were not detected when analyzing MLHFQ data using a standard method. Cardiac function, myocardial viability, mortality, and number of major adverse cardiac events did not differ between treatment groups. CONCLUSION: Our results suggest that BMC therapy can improve QoL, stressing the need for confirmation trials and for systematic QoL assessment in cardiac cell therapy trials .

Rasch-family models are more valuable than score-based approaches for analysing longitudinal patient-reported outcomes with missing data.

The objective was to compare classical test theory and Rasch-family models derived from item response theory for the analysis of longitudinal patient-reported outcomes data with possibly informative intermittent missing items. A simulation study was performed in order to assess and compare the performance of classical test theory and Rasch model in terms of bias, control of the type I error and power of the test of time effect. The type I error was controlled for classical test theory and Rasch model whether data were complete or some items were missing. Both methods were unbiased and displayed similar power with complete data. When items were missing, Rasch model remained unbiased and displayed higher power than classical test theory. Rasch model performed better than the classical test theory approach regarding the analysis of longitudinal patient-reported outcomes with possibly informative intermittent missing items mainly for power. This study highlights the interest of Rasch-based models in clinical research and epidemiology for the analysis of incomplete patient-reported outcomes data.

Assessment of score- and Rasch-based methods for group comparison of longitudinal patient-reported outcomes with intermittent missing data (informative and non-informative).

PURPOSE: The purpose of this study was to identify the most adequate strategy for group comparison of longitudinal patient-reported outcomes in the presence of possibly informative intermittent missing data. Models coming from classical test theory (CTT) and item response theory (IRT) were compared. METHODS: Two groups of patients' responses to dichotomous items with three times of assessment were simulated. Different cases were considered: presence or absence of a group effect and/or a time effect, a total of 100 or 200 patients, 4 or 7 items and two different values for the correlation coefficient of the latent trait between two consecutive times (0.4 or 0.9). Cases including informative and non-informative intermittent missing data were compared at different rates (15, 30 %). These simulated data were analyzed with CTT using score and mixed model (SM) and with IRT using longitudinal Rasch mixed model (LRM). The type I error, the power and the bias of the group effect estimations were compared between the two methods. RESULTS: This study showed that LRM performs better than SM. When the rate of missing data rose to 30 %, estimations were biased with SM mainly for informative missing data. Otherwise, LRM and SM methods were comparable concerning biases. However, regardless of the rate of intermittent missing data, power of LRM was higher compared to power of SM. CONCLUSIONS: In conclusion, LRM should be favored when the rate of missing data is higher than 15 %. For other cases, SM and LRM provide similar results.