Validation of the Multi-INdependence Dimensions (MIND) questionnaire for prolonged mechanically ventilated subjects.

BACKGROUND: Evaluating severity of illness of patients with prolonged mechanical ventilation (PMV) is important to adopt the best appropriate care management for each individual. Yet, no severity-of-illness scoring system has been specifically designed for this type of patients. The aim of this study was to develop and validate a new instrument, the Multi-INdependence Dimensions (MIND) questionnaire designed to comprehensively measure the severity of illness of patients under PMV. METHODS: The validation of the MIND questionnaire was performed during a longitudinal observational study conducted with PMV subjects in weaning facilities in three countries (Argentina, Colombia and Germany). The questionnaire validity was tested in 3 stages: 1) Specification of components, with description of item responses, inter-item and Cronbach alpha correlations; 2) Creation of the composite scores; 3) Measurement properties determination including test-retest reliability after 30 days, clinical validity (Medical Research Council (MRC) muscle strength score, Sepsis-related Organ Failure Assessment (SOFA), Glasgow Coma Scale (GCS), Dependence Nursing Scale and EuroQol-5 Dimension evaluated at inclusion), and ability to detect change. RESULTS: A total of 128 subjects participated in the validation study. Eleven component scores and four composite scores were created. MIND scores significantly correlated with MRC muscle strength, SOFA, DNS, GCS and EQ-5D, supporting the validity of the new scores. Intraclass Correlation Coefficient greater than 0.82 were observed for all composite scores, indicating good test-retest reliability. MIND scores were able to detect improvement in subject severity of illness. CONCLUSION: The MIND questionnaire is a valid and reliable instrument for measuring comprehensively the multiple dimensions characterizing the severity of illness of PMV patients. TRIAL REGISTRATION: NCT02255058 .

Assessing patients' acceptance of their medication to reveal unmet needs: results from a large multi-diseases study using a patient online community.

BACKGROUND: Patients with chronic conditions are required to take long-term treatments for their disease itself or to prevent any potential health risks. Measuring patient acceptance of their medication should help to better understand and predict patients' behavior toward treatment. This study aimed to describe the level of patient acceptance toward various long-term treatments in real life using an online patient community. METHODS: This was an observational, cross-sectional study conducted through the French Carenity platform. All Carenity patient members were invited to complete an online questionnaire including the 25-item ACCEptance by the Patients of their Treatment (ACCEPT©) questionnaire. ACCEPT© measures patient acceptance toward their medication and includes one general acceptance dimension (Acceptance/General) and six treatment-attribute specific dimensions (scores 0-100; lowest to highest acceptance): Acceptance/Medication Inconvenience, Acceptance/Long-term Treatment, Acceptance/Regimen Constraints, Acceptance/Side effects, Acceptance/Effectiveness, and Acceptance/Numerous Medications. Patients included in the analysis were treated adults experiencing any chronic diseases and who responded to at least one ACCEPT© item. RESULTS: Among the 4193 patients included in the analysis, more than 270 chronic diseases were represented, amidst which 19 included more than 30 patients. Mean ACCEPT© Acceptance/General score for those 19 diseases were 61.2 (SD = 31.9) for type 1 diabetes, 59.8 (SD = 32.3) for asthma, 56.3 (SD = 34.3) for hypertension, 52.0 (SD = 32.2) for chronic obstructive pulmonary disease, 51.7 (SD = 27.0) for epilepsy, 50.1 (SD = 33.1) for bipolar disorder, 49.9 (SD = 33.1) for type 2 diabetes, 48.6 (SD = 31.6) for multiple sclerosis, 46.1 (SD = 34.5) for Crohn's disease/ulcerative colitis, 44.3 (SD = 31.5) for depression, 42.8 (SD = 31.5) for lupus, 42.3 (SD = 33.0) for arthrosis, 41.8 (SD = 32.6) for Parkinson's disease, 40.5 (SD = 32.2) for rheumatoid arthritis, 38.6 (SD = 31.7) for breast cancer, 36.4 (SD = 36.4) for myocardial infarction, 35.8 (SD = 32.0) for ankylosing spondylitis, 34.1 (SD = 32.3) for psoriasis, and 33.7 (SD = 31.7) for fibromyalgia. CONCLUSIONS: This first of its kind study enabled ACCEPT© data to be collected in real life for a variety of chronic diseases. These data may help in evaluating and interpreting levels of acceptance in future studies and provide valuable insights about patient priorities and current unmet needs.

Item Reduction, Scoring, and First Validation of the ACCEPTance by the Patients of their Treatment (ACCEPT©) Questionnaire.

OBJECTIVES: The objectives of this study were to finalize, develop the scoring, and explore the psychometric properties of the ACCEPTance by the Patients of their Treatment (ACCEPT©) questionnaire, as well as to provide the first elements for its interpretation and guidance for its future use. METHODS: ACCEPT© was finalized according to reference methods including testing in a pilot study, i.e., a multi-center, observational, longitudinal study conducted in France, in collaboration with a network of pharmacists. Principal component analysis using Varimax rotation was performed. The loadings of items on components in the principal component analysis were used to inform item selection. Validity of the measurement model of ACCEPT© was confirmed using Multi-trait/Multi-item Analysis based on item-scale Spearman correlations. Internal consistency reliability of the questionnaire was assessed by determining the Cronbach's α coefficient. Linear and logistic regressions were used to identify predictors of general acceptance, and to study predictors of persistence. RESULTS: A total of 189 patients were included. The final version of ACCEPT© is composed of 25 items, distributed in seven dimensions providing a comprehensive appraisal of acceptance of long-term medication, with six scores measuring acceptance of treatment specific attributes and one score measuring general treatment acceptance. The measurement properties of ACCEPT© were overall fairly satisfactory. Regressions showed that Acceptance/Effectiveness is a predictor of general acceptance. However, no predictor of persistence could be identified. CONCLUSION: The self-administered ACCEPT© questionnaire is a valid and reliable instrument for the assessment of patients' acceptance of long-term medication. Disease-specific and large prospective studies are needed to assess the ability of ACCEPT

Real-life assessment of the multidimensional nature of dyspnoea in COPD outpatients.

Dyspnoea is a prominent symptom of chronic obstructive pulmonary disease (COPD). Recent multidimensional dyspnoea questionnaires like the Multidimensional Dyspnea Profile (MDP) individualise the sensory and affective dimensions of dyspnoea. We tested the MDP in COPD outpatients based on the hypothesis that the importance of the affective dimension of dyspnoea would vary according to clinical characteristics.A multicentre, prospective, observational, real-life study was conducted in 276 patients. MDP data were compared across various categories of patients (modified Medical Research Council (mMRC) dyspnoea score, COPD Assessment Test (CAT) score, Global Initiative for Chronic Obstructive Lung Disease (GOLD) airflow obstruction categories, GOLD "ABCD" categories, and Hospital Anxiety and Depression Scale (HADS)). Univariate and multivariate regressions were conducted to explore factors influencing the affective dimension of dyspnoea. Cluster analysis was conducted to create homogeneous patient profiles.The MDP identified a more marked affective dimension of dyspnoea with more severe mMRC, CAT, 12-item Short-Form Health Survey mental component, airflow obstruction and HADS. Multivariate analysis identified airflow obstruction, depressive symptoms and physical activity as determinants of the affective dimension of dyspnoea. Patients clustered into an "elderly, ex-smoker, severe disease, no rehabilitation" group exhibited the most marked affective dimension of dyspnoea.An affective/emotional dimension of dyspnoea can be identified in routine clinical practice. It can contribute to the phenotypic description of patients. Studies are needed to determine whether targeted therapeutic interventions can be designed and whether they are useful.

Measuring patients' satisfaction with their anti-TNF treatment in severe Crohn's disease: scoring and psychometric validation of the Satisfaction for PAtients in Crohn's diseasE Questionnaire (SPACE-Q(©)).

BACKGROUND: Severe Crohn's disease management includes anti-tumor necrosis factor (anti-TNF) drugs that differ from early-stage treatments regarding efficacy, safety, and convenience. This study aimed to finalize and psychometrically validate the Satisfaction for PAtients in Crohn's diseasE Questionnaire (SPACE-Q(©)), developed to measure satisfaction with anti-TNF treatment in patients with severe Crohn's disease. METHODS: A total of 279 patients with severe Crohn's disease receiving anti-TNF therapy completed the SPACE-Q 62-item pilot version at inclusion and 12 and 13 weeks after first anti-TNF injection. The final SPACE-Q scoring was defined using multitrait and regression analyses and clinical relevance considerations. Psychometric validation included clinical validity against Harvey-Bradshaw score, concurrent validity against Treatment Satisfaction Questionnaire for Medication (TSQM), internal consistency reliability, test-retest reliability, and responsiveness against the patient global impression of change (PGIC). RESULTS: Quality of completion was good (55%-67% of patients completed all items). Four items were removed from the questionnaire. Eleven scores were defined within the final 58-item SPACE-Q: disease control; symptoms, anal symptoms, and quality of life transition scales; tolerability; convenience; expectation confirmation toward efficacy, side effects, and convenience; satisfaction with treatment; and motivation. Scores met standards for concurrent validity (correlation between SPACE-Q satisfaction with treatment and TSQM satisfaction scores =0.59), internal consistency reliability (Cronbach's α=0.67-0.93), test-retest reliability (intraclass correlations =0.62-0.91), and responsiveness (improvement in treatment experience assessed by the SPACE-Q for patients reporting improvement on the PGIC). Significantly different mean scores were observed between groups of patients with different Harvey-Bradshaw disease severity scores. CONCLUSION: The SPACE-Q is a valid, reliable, and responsive instrument to measure satisfaction with anti-TNF treatment in patients with severe Crohn's disease and for use in future studies.

Improving the primary care physicians' decision making for fibromyalgia in clinical practice: development and validation of the Fibromyalgia Detection (FibroDetect®) screening tool.

BACKGROUND: Fibromyalgia diagnosis is a challenging and long process, especially among primary care physicians (PCPs), because of symptom heterogeneity, co-morbidities and clinical overlap with other disorders. The purpose was to develop and validate a screening tool in French (FR), German (DE) and English (UK) to help PCPs identify patients with fibromyalgia. METHODS: The FibroDetect questionnaire was simultaneously developed in FR, DE and UK based on information obtained from a literature review, focus groups conducted with clinicians, and face-to-face interviews with fibromyalgia patients (FR, DE and UK, n = 23). The resulting tool was comprehension-tested in patients with diagnosed or suspected fibromyalgia (n = 3 and n = 2 in each country, respectively). Acceptability and applicability were assessed and the tool modified accordingly, then assessed in clinical practice. A scoring method was created using an iterative process based on statistical and clinical considerations with American College of Rheumatology + (ACR+) patients and ACR- patients (n = 276), and validated with fibromyalgia and non-fibromyalgia patients (n = 312). RESULTS: The FibroDetect included 14 questions assessing patients' pain and fatigue, personal history and attitudes, symptoms and impact on lives. Six questions were retained in the final scoring, demonstrating satisfactory discriminative power between ACR + and ACR- patients with area under the Receiver Operating Characteristic curve of 0.74. The predictive accuracy of the tool increased to 0.86 for fibromyalgia and non-fibromyalgia patient detection, with a sensitivity of 90% and a specificity of 67% for a cut-off of 6 on the score. CONCLUSIONS: The FibroDetect is a self-administered tool that can be used as a screening classification surrogate to the ACR criteria in primary care settings to help PCPs detect potential fibromyalgia patients among a population complaining of chronic widespread pain.

Impact of human papillomavirus-related genital diseases on quality of life and psychosocial wellbeing: results of an observational, health-related quality of life study in the UK.

BACKGROUND: Data on the psychosocial burden of human papillomavirus (HPV)-related diseases other than cervical cancer are scarce. The objectives of this study were to measure and compare the psychosocial burden and the impact on health-related quality of life (HRQoL) of HPV-related lower genital tract diseases and genital warts (GW) using several generic and disease-specific instruments. METHODS: Overall, 842 individuals with normal cervical cytology (n = 241), borderline nuclear abnormalities and/or mild dyskaryosis (n = 23), cervical intraepithelial neoplasia (CIN)1 (n = 84), CIN2/3 (n = 203), vulval intraepithelial neoplasia (VIN)2/3 (n = 43), GW (n = 186) and a history of GW (non-current) (n = 62) were included. The generic European Quality of Life Index Version 5D (EQ-5D) questionnaire was completed by patients with GW and VIN2/3. Sexual functioning was evaluated using the Change in Sexual Functioning Questionnaire (CSFQ). Psychosocial impact was measured in women using the HPV Impact Profile (HIP) questionnaire. HRQoL was assessed using a GW-specific questionnaire, the Cuestionario Especifico en Condilomas Acuminados (CECA) (completed by patients with GW and history of GW). For each instrument, scores were compared between groups using the Student's t-test. In addition, utility loss due to GW and VIN2/3 was evaluated by comparing mean EQ-5D scores weighted by age and sex with the UK general population normal values. RESULTS: A significant psychosocial impact was found in women diagnosed with HPV-related genital diseases, particularly in those with GW. The health state of younger adults with GW was significantly impaired compared with UK normal values (mean EQ-5D index score 0.86 vs 0.94, p < 0.001 for 18-24-year-olds; 0.87 vs 0.93, p = 0.030 for 25-34-year-olds). VIN2/3 was found to have a significant negative impact on sexual functioning, and women with VIN2/3 had a highly impaired health state compared with women in the UK general population (weighted mean EQ-5D index score 0.72 vs 0.89, p < 0.001; weighted mean Visual Analogue Scale score 62 vs 85, p < 0.001). CONCLUSIONS: HPV-related lower genital tract lesions and GW significantly impair psychosocial wellbeing and HRQoL. The psychosocial aspects of HPV-related diseases need to be considered when evaluating the potential benefit of HPV vaccination.

Demonstrating the burden of hypoglycemia on patients' quality of life in diabetes clinical trials: measurement considerations for hypoglycemia.

OBJECTIVES: To evaluate the association between hypoglycemia and health-related quality of life (HRQoL) in the context of a clinical trial using both an objectively confirmed and a patient-reported measure of hypoglycemia. METHODS: During a phase III, double-arm, randomized study, patients completed the short form 36 health survey (SF-36), a generic HRQoL questionnaire, at baseline and at weeks 24, 52, and 104. The objectively confirmed measure of hypoglycemia was based on a combination of plasma glucose measure and presence of hypoglycemia-related symptoms. The patient-reported frequency of hypoglycemia was defined as the following item: "How often have you felt that your blood sugars have been unacceptably low recently?" The association between hypoglycemia and HRQoL was evaluated in intent-to-treat patients (N = 3059) by using repeated-measurements analyses, with SF-36 scores used as explained variables and baseline SF-36 score, age, sex, country, time, and either number of objectively confirmed hypoglycemic events (0, ≥1) or patient-reported frequency of hypoglycemia (continuous variable 0-6) as explanatory variables. RESULTS: During study duration, less than 6% of patients experienced at least one objectively confirmed hypoglycemic event and about half the patients reported unacceptably low blood sugars "none of the time." The association between the number of objectively confirmed hypoglycemic events and HRQoL was not statistically significant, while the patient-reported frequency of hypoglycemia was statistically significantly related to all SF-36 scores (P < 0.001), except physical functioning; patients reporting greater perceived frequency of hypoglycemia had worse HRQoL. CONCLUSIONS: Using a patient-reported measure of hypoglycemia in the context of a clinical trial could enable the burden of hypoglycemia for patients to be demonstrated.

Impact on parents of bronchiolitis hospitalization of full-term, preterm and congenital heart disease infants.

BACKGROUND: The objective of this work was to explore the impact on parents of the bronchiolitis hospitalization of their infant using the Impact of Bronchiolitis Hospitalization Questionnaire (IBHQ©). METHODS: Four hundred sixty-three infants aged less than 1 year and hospitalized for bronchiolitis were included in a French observational study during the 2008-2009 season. Parents were asked to complete the IBHQ at hospital discharge and 3 months later. IBHQ scores, ranging from 0 (no impact) to 100 (highest impact), were compared according to gestational age (full-term, 33-36 wGA, ≤ 32 wGA) and the presence of congenital heart disease (CHD). The potential drivers of impact were explored using multivariate linear regressions. RESULTS: The study included 332 full-terms, 71 infants born at 33-36 wGA, and 60 at ≤ 32 wGA; 28 infants had a CHD. At hospital discharge, 9 of the 12 IBHQ mean scores were above 40, indicating a marked impact on parents. Three months later, all mean scores were lower but 5 were still greater than 40. At discharge, the length of hospitalization had a significant effect on IBHQ worries and distress, fear for future, guilt and impact on daily organization scores (p<0.01); the parents' educational level had a significant effect on IBHQ worries and distress, fear for future, impact on daily organization and financial impact scores (p<0.05). The only statistically significant difference found between the parents of preterm and full-term infants was for the physical impact score at discharge (p=0.004). CONCLUSIONS: Bronchiolitis hospitalization has conspicuous emotional, physical and organizational consequences on parents and siblings, which persist 3 months after hospital discharge. The main drivers of the impact were length of hospital stay and parents' educational level, while infants' gestational age or the presence of a CHD had little influence.

Psychometric evaluation of the functional assessment of HIV Infection (FAHI) questionnaire and its usefulness in clinical trials.

PURPOSE: To evaluate psychometric properties of the Functional Assessment of Human Immunodeficiency Virus Infection (FAHI) questionnaire, a 47-item disease-specific instrument evaluating Health-Related Quality of Life (HRQL) in human immunodeficiency virus (HIV)-infected patients. METHODS: Treatment-experienced HIV-infected patients from two clinical programmes (N = 565; N = 1,096) completed the FAHI at Baseline and after 24 weeks of treatment. Psychometric properties of the FAHI were assessed in both trial populations, including minimal important differences (MIDs) calculations. Links between HRQL assessed by FAHI Total score, and biological endpoints were explored by regression analysis and mean score comparisons. RESULTS: Cronbach's alphas ranged from 0.72 to 0.94. Most items met convergent and discriminant validity criteria. Better FAHI scores were seen for patients in earlier HIV stages. Responsiveness was demonstrated with changes in FAHI scores significantly linked to change in EQ-5D score. Depending on methods used, MIDs ranged from 3.2 to 14 for FAHI Total score. Small association was found between FAHI Total score and CD4 count and viral load (r-square < 3%). Mean changes in FAHI scores were not statistically related to viral response. CONCLUSIONS: The FAHI demonstrated acceptable psychometric properties in two independent populations. HRQL assessment enabled detection of changes in patients' health status not revealed by traditional efficacy endpoints.

Effects of etravirine versus placebo on health-related quality of life in treatment-experienced HIV patients as measured by the functional assessment of human immunodeficiency virus infection (FAHI) questionnaire in the DUET trials.

PURPOSE: To study the effects of etravirine versus placebo on the health-related quality of life (HRQL) of HIV-infected patients. METHOD: HRQL of HIV-infected patients was measured using the Functional Assessment of Human Immunodeficiency Virus Infection (FAHI) questionnaire in two identically designed phase III clinical trials investigating efficacy and safety of etravirine as part of highly active antiretroviral therapy. Pooled analyses of covariance, adjusted for treatment group, baseline FAHI score, CD4 cell count, viral load, and enfuvirtide use helped investigate changes in FAHI scores between baseline and Week 24 in etravirine-treated (n = 599) and placebo-treated (n = 604) subjects. Responder analyses were also conducted. RESULTS: Significant improvements with etravirine treatment were observed for FAHI physical, functional, emotional, and total scores (P < .001). These improvements were greater and statistically different from those with placebo (.013

Scoring and psychometric properties of the Eye-Drop Satisfaction Questionnaire (EDSQ), an instrument to assess satisfaction and compliance with glaucoma treatment.

BACKGROUND: The objective of this study was to ascertain the scoring and assess the psychometric properties of the Eye-Drop Satisfaction Questionnaire (EDSQ), a 43-item Patient-Reported Outcome instrument developed to assess patients' satisfaction and compliance with glaucoma treatment. METHODS: The EDSQ was administered during an observational, retrospective study to 184 French patients treated for glaucoma. The hypothesized structure, including six dimensions (patient-clinician relationship; patient experience; patient-treatment interaction; apprehension; patient knowledge; travel), was tested by assessing the internal consistency reliability (Cronbach's alpha) and construct-related validity (item convergent and discriminant validity). As unsatisfactory results were demonstrated, another structure was defined using a principal component analysis (PCA) combined with content of items. Psychometric properties of this new structure were assessed. Scores were compared between low, moderate and high compliance profile groups defined using data collected with the Travalert electronic device. RESULTS: Analyses were performed with the 169 patients who completed at least half of the EDSQ items. The hypothesized structure showed a Cronbach's alpha lower than 0.70 for four dimensions out of six and an overall poor construct-related validity (range of item-scale correlations: 0.00-0.68). The new structure obtained with the PCA included six dimensions: concern about treatment (five items); concern about disease (two items); satisfaction with patient-clinician relationship (five items); positive beliefs (three items); treatment convenience (three items); and self-declared compliance (three items). A score ranging from 0 to 100 was calculated for each dimension, with higher scores indicating more of the attribute referred to in the dimension. Internal consistency reliability was good (Cronbach's alpha greater than 0.70 for five dimensions). The structure offered good construct-related validity (range of item-scale correlations: 0.36-0.82). Ceiling effects of 21% and 49%, were observed for the satisfaction with patient-clinician relationship and self-declared compliance scores. Patients in low compliance profile group reported the lowest score for the satisfaction with patient-clinician relationship, positive beliefs, treatment convenience and self-declared compliance dimensions, and the highest score for the concern about treatment dimension. CONCLUSIONS: The scoring of the EDSQ was developed and the questionnaire proved to have satisfactory psychometric properties. EDSQ scores showed a promising relationship to compliance profiles. The EDSQ could be used in future studies.

Application of the disease-specific Quality of Life Assessment of Growth Hormone Deficiency in Adults (QoL-AGHDA) questionnaire in a general population: results from a French panel study.

OBJECTIVES: To assess the psychometric properties of the disease-specific Quality of Life Assessment of Growth Hormone Deficiency in Adults (QoL-AGHDA) questionnaire in a general population, and collect French normative data. METHODS: A postal survey was conducted on 2900 adult panelists representative of the French population. The participants were asked to complete a questionnaire including the QoL-AGHDA and an evaluation of their overall health status (OHS). The QoL-AGHDA score ranges from 0 to 25, a lower score indicating better QoL. Psychometric properties of the QoL-AGHDA were assessed. The mean QoL-AGHDA scores were described by sex and age groups. RESULTS: The return rate was 75%. The quality of completion and internal consistency reliability were good: 95% of the respondents completed all 25 QoL-AGHDA items and Cronbach's alpha was 0.86. The QoL-AGHDA score was able to discriminate between the respondents according to their OHS (from 1.5 for excellent to 12.3 for poor OHS, P < 0.001). The mean QoL-AGHDA score was 4.6 for the overall population, 5.1 for females and 4.2 for males, and ranged from 4.8 for the youngest to 6.1 for the oldest respondents. CONCLUSIONS: The QoL-AGHDA questionnaire showed good psychometric properties when administered in the French population. French reference values were collected, completing the QoL-AGHDA normative database already available in several European countries.