Validation and optimization of the French Generic Adherence for Chronic Diseases Profile (GACID-P) using classical test and item response theory.

BACKGROUND: The Generic Adherence for Chronic Diseases Profile is a French generic scale (GACID-P) developed to measure adherence in several disease areas such as cardiology, rheumatology, diabetes, cancer and infectiology. METHOD: We aimed to study the measurement invariance of the Generic Adherence for Chronic Diseases Profile by an item response model, optimize the new instrument version from item response model and qualitative content analyses results, and validate the instrument. The metric properties of the optimized version were studied according to classical test theory and item response model analysis. RESULTS: A sample of 397 patients consulting at two French hospitals (in diabetes, cardiology, rheumatology, cancerology and infectiology) and in four private practices was recruited; 314 (79%) patients also completed the questionnaire 15 days later. Factor analyses revealed four dimensions: "Forgetting to take medication", "Intention to comply with treatment", "Limitation of risk-related consumer habits" and "Healthy lifestyle". The item response model and content analyses optimized these four dimensions, regrouping 32 items in four dimensions of 25 items, including one item conditioned on tobacco use. The psychometric properties and scale calibration were satisfactory. One score per dimension was calculated as the sum of the items for the dimensions "Forgetting to take medication" and "Intention to comply with treatment" and as a weighted score according to the item response model analysis for the two other dimensions because of differential item functioning found for two items. CONCLUSION: Four adherence profile scores were obtained. The instrument validity was documented by a theoretical approach and content analysis. The Generic Adherence for Chronic Diseases Profile is now available for research targeting adherence in a broad perspective.

An algorithm to determine the date when the McDonald criteria are met for the diagnosis of relapsing-remitting multiple sclerosis.

BACKGROUND: A patient is diagnosed with multiple sclerosis once they meet the McDonald criteria of dissemination in space and time. Studies of cohorts of patients with multiple sclerosis need a reproducible way to determine an accurate date of diagnosis. We developed an automatic data-driven algorithm to determine the date when the MacDonald criteria are met, which we validated with the Registre Lorrain des Scleroses en Plaques (ReLSEP), a regional French registry of patients with multiple sclerosis. METHODS: We developed an algorithm to determine the date of diagnosis based on clinical and paraclinical data adapted from the four versions of the McDonald criteria. For validation, the dates of diagnosis generated by the algorithm were compared with those determined by an expert physician using the patients' files as the gold standard. We calculated the sensitivity and specificity of the algorithm to provide a date, then we tested the equivalence between the dates of the gold standard and the algorithm (two-one-sided-t-test). RESULTS: The algorithm used every possibility of determining dissemination in space and time according to the four sets. The sensitivity of the algorithm was 100% for the four sets, and specificity ranged between 95 and 100%. The difference between the dates of diagnosis found by the physician and the algorithm was usually less than 2 weeks (equivalence test P<0.0001). CONCLUSION: The algorithm appears to be an efficient surrogate to accurately determine dates of diagnosis of multiple sclerosis in datasets of patients.

Predictive factors of functional independence after optimal reperfusion in anterior circulation ischaemic stroke with indication for intravenous thrombolysis plus mechanical thrombectomy.

BACKGROUND AND PURPOSE: Intravenous thrombolysis plus mechanical thrombectomy (IVT + MT) is the best current management of acute stroke due to large-vessel occlusion and results in optimal reperfusion for most patients. Nevertheless, some of these patients do not subsequently achieve functional independence. The aim was to identify baseline factors associated with 3-month independence after optimal reperfusion and to validate a prediction model. METHODS: All consecutive patients with intracranial anterior large-vessel occlusion, with indication for IVT + MT and achieving optimal reperfusion (defined as modified Treatment in Cerebral Ischaemia score 2b-3), from the THRACE trial and the ETIS registry, were included in order to identify a prediction model. The primary outcome was 3-month independence [modified Rankin Scale (mRS) score ≤ 2]. Multivariate inferences invoked forward logistic regression, multiple imputation and bootstrap resampling. Predictive performance was assessed by c-statistic. Model validation was conducted on patients from the ASTER trial. RESULTS: Amongst 139 patients (mean age 65.5 years; 54.3% female), predictors of 3-month mRS ≤ 2 (n = 82) were younger age [odds ratio 0.62 per 10-year increase; 95% confidence interval (CI) 0.53-0.72] and higher Alberta Stroke Program Early Computed Tomography Score (ASPECTS) (odds ratio 1.65 per 1-point increase; 95% CI 1.47-1.86) with c-statistic 0.77. Model validation (n = 104/181 patients with 3-month mRS ≤ 2) demonstrated a moderate discrimination (c-statistic 0.74; 95% CI 0.66-0.81) combining age and ASPECTS. The validation model was improved by the adjunction of three candidate variables that were found to be predictors. Addition of baseline National Institutes of Health Stroke Scale (NIHSS) score, history of vascular risk factor and onset-to-reperfusion time significantly improved discrimination (c-statistic 0.85; 95% CI 0.83-0.87). CONCLUSIONS: After optimal reperfusion, younger age, higher ASPECTS, lower NIHSS score, shorter onset-to-reperfusion time and absence of vascular risk factor were predictive of independence and could help to guide patient management.

[Predictive factors for skeletal-related events in lung cancer]. / Facteurs prédictifs de survenue d'évènements osseux dans le cancer bronchique.

INTRODUCTION: Skeletal-related events (SRE) are common in patients with bone metastatic lung cancer and have a negative impact on quality of life and survival. The objective of this study is to identify predictive factors for SRE occurrence among this population. METHODS: We conducted a 3-year retrospective study including 100 lung cancer patients with bone metastasis. RESULTS: Eighty-two patients presented at least one SRE (69.5% at baseline). The median occurrence for SRE was 4.5 months and severe bone pain was the most common SRE (56%). The alkaline phosphatase serum level>120IU/L (hazard ratio [sHR]=2.8; 95% confidence interval (CI) [1.5-5.4]; P=0.002) and calcemia>2.6mmol/L ([sHR]=9.7; 95% CI [5.1-18.4]; P<0.001) were identified as risk factors for SRE occurrence while the presence of an initial SRE was associated with a decrease of this risk ([sHR]=0.2; 95% CI [0.1-0.4]; P<0.001). CONCLUSION: The elevated alkaline phosphatase serum level and hypercalcemia are risk factors for SRE occurrence in bone metastatic lung cancer patients and should be used as biomarkers to adapt current medical practice for these patients.

Optimizing DyNaChron instrument for assessing chronic nasal dysfunction symptoms by Rasch analysis.

BACKGROUND: The DyNaChron (Dysfonctionnement Nasal Chronique) questionnaire is a self-reporting 78-item instrument assessing six symptoms and their consequences of chronic nasal dysfunction. Patients complete items of a symptom domain only when it is present but in case the patient presents several or all symptoms, its length can limit its use. Here, we aimed to optimize, or shorten, the DyNaChron for clinical use. METHODS: A total of 640 patients in 14 rhinology outpatient clinics all over France completed the original DyNaChron questionnaire before the first rhinologic clinic and 15 days later. The optimization process involved Rasch analysis and then qualitative content analyses. Rasch analysis flagged items with a floor/ceiling effect or with important differential item functioning and an expert committee decided whether to retain the flagged items on the basis of clinical importance and statistical characteristics. The psychometric properties of the optimized version were studied according to classical test theory and Rasch analysis. RESULTS: Rasch analysis revealed 4 items with underfit, 6 with an extreme score, 2 that were highly locally dependent and 16 with differential item functioning which 5 of these 16 items were retained after content analysis. In total, 19 flagged items were removed. Factorial analysis confirmed the preservation of the initial instrument structure in the optimized scale; psychometrics properties and scale calibration were the same as or better than the original version. CONCLUSION: The shortened DyNaChron optimizes the quality of assessment by deleting redundant items and reduces the burden on respondents; the structure is preserved and the psychometrics properties are improved.

Clinical follow-up of 411 patients with relapsing and progressive multiple sclerosis 10 years after discontinuing mitoxantrone treatment: a real-life cohort study.

BACKGROUND AND PURPOSE: Mitoxantrone (MITOX) has been used to treat patients with aggressive multiple sclerosis (MS) for decades. We aimed to describe the effectiveness and adverse events over 10 years post-MITOX in patients with relapsing and progressive MS from an exhaustive real-life database. METHODS: Data from patients who received MITOX before 1 January 2006 were collected from the MS Lorraine registry. Expanded Disability Status Scale (EDSS) scores and annual relapse rates (ARRs) year by year during follow-up and the year prior to MITOX were compared. Time to the first relapse and a 1-point increase in EDSS score were used in Cox multivariate models to find associations with potential predictive factors. RESULTS: A total of 411 patients were included. The ARR for the 155 relapsing patients had decreased from 2.0 (SD 1.20) the year before treatment to 0.3 (SD 0.31) by year 10 (P < 0.0001). The EDSS score increased from 2.8 (SD 1.44) to 4.8 (SD 1.90) by year 10 (P < 0.0001). A high ARR at MITOX initiation was associated with a longer time to a 1-point increase in EDSS score (hazard ratio, 0.81; 95% confidence interval, 0.67-0.99; P = 0.04). The EDSS score in 256 progressive patients increased from 5.0 (SD 1.33) to 6.5 (SD 1.26) by year 10 (P < 0.0001). We identified four cases of acute myeloid leukemias. CONCLUSIONS: Patients with the most active forms of MS are the most likely to benefit from MITOX in the long term.