Switching from natalizumab administration at the day hospital to administration at home. A 1 year prospective study of patient experience and quality of life in 30 consecutive patients with multiple sclerosis (TYSAD-35).

BACKGROUND: In the context of the COVID-19 pandemic, French health authorities allowed the home administration of natalizumab by a healthcare-at-home service. We evaluated the patients' perception of care quality following the transition from day-hospital to home natalizumab administration. METHODS: Thirty relapsing-remitting multiple sclerosis (MS) patients treated with natalizumab were prospectively evaluated for one year after changing onto a home treatment procedure, using MusiCare, the first MS-specific questionnaire to evaluate patient experience and MusiQol. A numerical rating scale score for satisfaction and a dedicated questionnaire concerning patient experience were completed after each infusion. The primary endpoint was the mean difference in MusiCare score between baseline and 12 months. RESULTS: From June 2020 to November 2021, 306 infusions were performed at home. Three patients withdrew from the study (one lost to follow-up and two preferred to return at the day hospital). No worsening of patient experience or quality of life was observed. The mean scores of the Musicare dimensions were higher at 12 months than at baseline, significantly for the "relationship with healthcare professionals" (p = 0.0203). The MusiQol global score remained stable but the coping and friendship dimensions were significantly better at M12 than at baseline (p = 0.0491 and p = 0.0478, respectively). The satisfaction questionnaire highlighted some pain during the infusions (21.8%) and contradictions between healthcare professionals (17.2%). The mean score for satisfaction with care was 9.1/10. No safety concerns were identified. CONCLUSION: The positive experience of patients with home natalizumab administration provides an important opportunity to improve the quality of patient care.

Assessing the experience of the quality of care of patients living with multiple sclerosis and their caregivers: The MusiCare questionnaire.

BACKGROUND AND PURPOSE: Patients with a chronic illness, such as multiple sclerosis (MS), and their natural caregivers have a specific experience of healthcare and health services. These experiences need to be assessed to evaluate the quality of care. Our objective was to develop a French-language questionnaire to evaluate the quality of care as experienced by MS patients and their natural caregivers. METHODS: Eligible patients had been diagnosed with MS according to the McDonald criteria. Eligible caregivers were individuals designated by the patients. The MusiCare questionnaire was developed in two standard phases: (i) item generation, based on interviews with patients and caregivers; and (ii) validation, consisting of validity, reliability, external validity, reproducibility, and responsiveness measures. RESULTS: In total, 1088 patients (n = 660) and caregivers (n = 488) were recruited. The initial 64-item version of MusiCare was administered to a random subsample (n = 748). The validation process generated a 35-item questionnaire. Internal consistency and scalability were satisfactory. Testing of the external validity revealed expected associations between MusiCare scores and sociodemographic and clinical data. The questionnaire showed good reproducibility and responsiveness. CONCLUSIONS: The availability of a reliable and validated French-language self-report questionnaire probing the experience of the quality of care for MS will allow the feedback of patients and caregivers to be incorporated into a continuous healthcare quality-improvement strategy.

Organizational and managerial factors associated with clinical practice guideline adherence: a simulation-based study in 36 French hospital wards.

OBJECTIVES: To identify managerial and organizational characteristics of multi-specialty medicine wards and individual characteristics of health professionals that are most strongly associated with clinical practice guidelines (CPG) adherence. DESIGN: Cross-sectional stratified cluster sample design. SETTING: Data were gathered from 36 randomly selected multi-specialty medicine wards. PARTICIPANTS: The study population included all health professionals involved in patient care working in the participating wards. MAIN OUTCOME MEASURES: The degree of CPG adherence was measured using clinical vignettes on three topics: pain management, managing heart failure and managing diabetes. Responses from each professional to each clinical case were quantified using a 10-point scale. Managerial and organizational characteristics of medical department and individual characteristics of health professionals were obtained using three questionnaires. RESULTS: The study sample consisted of 859 professionals (362 orderlies, 361 nurses and 136 physicians). Factors independently and positively associated with CPG adherence were (i) individual factors: low age of professionals, expertise in diabetology and activity in cardiology; (ii) organizational and managerial factors: good understanding between physicians and other personnel; and (iii) structural factors: computer-based test results and prescriptions, presence of medical specialists, inter-department mobility of orderlies, medium-length stay (between 7 and 10 days) and large bed capacity. CONCLUSIONS: Good CPG adherence in general medicine needs institutional dynamism, availability of clinical competence and team culture based on cooperation.

Oral versus intravenous high-dose methylprednisolone for treatment of relapses in patients with multiple sclerosis (COPOUSEP): a randomised, controlled, double-blind, non-inferiority trial.

BACKGROUND: High doses of intravenous methylprednisolone are recommended to treat relapses in patients with multiple sclerosis, but can be inconvenient and expensive. We aimed to assess whether oral administration of high-dose methylprednisolone was non-inferior to intravenous administration. METHODS: We did this multicentre, double-blind, randomised, controlled, non-inferiority trial at 13 centres for multiple sclerosis in France. We enrolled patients aged 18-55 years with relapsing-remitting multiple sclerosis who reported a relapse within the previous 15 days that caused an increase of at least one point in one or more scores on the Kurtzke Functional System Scale. With use of a computer-generated randomisation list and in blocks of four, we randomly assigned (1:1) patients to either oral or intravenous methylprednisolone, 1000 mg, once a day for 3 days. Patients, treating physicians and nurses, and data and outcome assessors were all masked to treatment allocation, which was achieved with the use of saline solution and placebo capsules. The primary endpoint was the proportion of patients who had improved by day 28 (decrease of at least one point in most affected score on Kurtzke Functional System Scale), without need for retreatment with corticosteroids, in the per-protocol population. The trial was powered to assess non-inferiority of oral compared with intravenous methylprednisolone with a predetermined non-inferiority margin of 15%. This trial is registered with ClinicalTrials.gov, number NCT00984984. FINDINGS: Between Jan 29, 2008, and June 14, 2013, we screened 200 patients and enrolled 199. We randomly assigned 100 patients to oral methylprednisolone and 99 patients to intravenous methylprednisolone with a mean time from relapse onset to treatment of 7·0 days (SD 3·6) and 7·4 days (3·9), respectively. In the per-protocol population, 66 (81%) of 82 patients in the oral group and 72 (80%) of 90 patients in the intravenous group achieved the primary endpoint (absolute treatment difference 0·5%, 90% CI -9·5 to 10·4). Rates of adverse events were similar, but insomnia was more frequently reported in the oral group (77 [77%]) than in the intravenous group (63 [64%]). INTERPRETATION: Oral administration of high-dose methylprednisolone for 3 days was not inferior to intravenous administration for improvement of disability scores 1 month after treatment and had a similar safety profile. This finding could have implications for access to treatment, patient comfort, and cost, but indication should always be properly considered by clinicians. FUNDING: French Health Ministry, Ligue Française contre la SEP, Teva.

Primary gliomatosis cerebri involving gray matter in pediatrics: a distinct entity? A multicenter study of 14 cases.

BACKGROUND AND PURPOSE: Gliomatosis cerebri (GC) is a rare neoplasm including a variety of tumors, with extremely variable evolution and heterogeneity of prognosis. It may appear either de novo or after a focal glioma, involve predominantly the white or the gray matter, and concern either pediatric or adult patients. We focused on primary GC involving exclusively gray matter in a pediatric population in order better to define the presentation and outcome of this disease. PATIENTS AND METHODS: We reviewed the databases of seven Departments of Pediatric Oncology to identify pediatric cases of GC between 1990 and 2007. Patients were included if they demonstrated a diffuse infiltrative process involving gray matter in magnetic resonance imaging (MRI) and histological tissue analyses, confirming a proliferative glial disorder. RESULTS: Fourteen patients with a median age of 8 years were identified. Epilepsy was the main presenting symptom. Brain MRI showed a lesion of the temporal and insular cerebral cortex associated with tumoral infiltration of the thalami and the basal ganglia. Histological examination confirmed the diagnosis of high-grade glioma. Prognosis was always very gloomy in the short term, with a median survival of less than a year. CONCLUSION: This rare entity, whose prognosis is appalling whatever the treatment proposed, should be clearly identified within the heterogeneous group of GC in the same way as diffuse intrinsic pontine gliomas have been identified among brain stem tumors. Systematic biopsies appear essential to permit the molecular studies which will assist in guiding the choice of future targeted treatments.

Predictive value of global longitudinal strain in a surgical population of organic mitral regurgitation.

BACKGROUND: Because of the lack of reliable echocardiographic parameters to predict recovery after surgery, the optimal timing of surgery for severe mitral regurgitation remains controversial. The aim of this study was to determine whether global longitudinal strain (GLS) recorded preoperatively could help in predicting left ventricular (LV) ejection fraction (LVEF) postoperatively. METHODS: A total of 88 patients (mean age, 63 ± 13 years; 59 men) with severe degenerative mitral regurgitation were included prospectively in this study. Rest echocardiography was performed before and 6 ± 1 months after mitral valve surgery. Patients were divided into two groups: group A (postoperative LVEF ≥ 50%) and group B (postoperative LVEF < 50%). RESULTS: In group B, patients had larger preoperative LV end-systolic diameters (21.6 ± 2.6 vs 19.2 ± 3.7 mm/m(2), P = .02) and impaired preoperative GLS (-17 ± 2.8% vs -19.6 ± 3.6%, P = .01), whereas there was no difference in preoperative LVEF. Preoperative LV end-systolic diameter ≥ 22 mm/m(2) and GLS < -18% were independent predictors of postoperative LV dysfunction. CONCLUSIONS: LV end-systolic diameter is a well-recognized prognostic marker. In addition, this study demonstrates the additive and independent predictive value of preoperative GLS for predicting postoperative LV dysfunction.

Impact of aortic stenosis on longitudinal myocardial deformation during exercise.

UNLABELLED: In aortic stenosis (AS), left ventricular (LV) adaptation to exercise has poorly been examined. Changes in LV ejection fraction may lack accuracy in identifying the presence of intrinsic myocardial impairment. AIMS: We sought to determine the impact of aortic stenosis (AS) on left ventricular (LV) longitudinal function at exercise in a series of asymptomatic patients with AS and preserved LV ejection fraction. METHODS AND RESULTS: Long-axis function was assessed at rest and at exercise by using 2D speckle tracking of myocardial deformation in 207 AS patients (aortic valve area 0.87 ± 0.19 cm²) and 43 aged-matched control subjects. When compared with control subjects, patients with AS have reduced longitudinal myocardial function at rest (-20.2 ± 2.7 vs. -15.4 ± 4.0%) and at peak exercise (-25.0 ± 3.7 vs. -16.5 ± 4.9%) (P < 0.0001 for both). Exercise changes in global longitudinal strain were correlated with changes in LV ejection in controls but not in patients with AS. Changes in LV global longitudinal strain during test were lower in AS patients with an abnormal response to exercise (-0.5 ± 2.7 vs. -1.5 ± 2.8%, P = 0.001). In multivariate analysis, a lower global longitudinal strain at rest (P = 0.04), a higher increase in mean trans-valvular pressure gradient (P < 0.001) at exercise, and smaller exercise-induced changes in global longitudinal strain (P < 0.001) were associated with an abnormal exercise test. CONCLUSION: In AS, subnormal LV function can be reliably identified by 2D strain imaging at rest and during a sub-maximal exercise. That sensitive measure of LV systolic function is depressed in AS and even more in patients having the most severe AS.

Left atrial function assessed by trans-thoracic echocardiography in patients treated by ablation for a lone paroxysmal atrial fibrillation.

AIMS: Atrial fibrillation (AF) may induce three kinds of atrial remodelling: morphological, contractile, and electrical. Maintain of sinus rhythm is usually associated with left atrial (LA) volume decrease, but little is known about the evolution of its mechanical properties. We sought to explore LA mechanical and morphological remodelling in patients with lone paroxysmal-AF treated by catheter ablation (CA). METHODS AND RESULTS: We prospectively included 31 patients (56.4 ± 10 years). We also followed 15 age- and gender-matched controls to get normal values. Each patient had a complete echocardiography at baseline and at 3-month and 1-year follow-up. LA-anatomical reverse remodelling was documented: indexed LA volume decreased from 39 mL/m(2) at baseline to 31 mL/m(2) at 1 year (P < 0.001). However, it remained larger than controls (31 vs. 23 mL/m(2), P = 0.001). LA compliance improved (LA lateral systolic peaks of strain = 50 vs. 31%, P < 0.05) without reaching controls values as estimated by 50 vs. 81%, P < 0.05). LA contractility increased as highlighted by A'-peak velocity (10 cm/s at 1Y-F/up vs. 7.5 at baseline, P = 0.01) and LA late diastolic peaks of strain rate (septal: -3 vs. -1 s(-1), lateral: -3 vs. -1.4 s(-1), P < 0.05). We show a functional remodelling at 1 year, with most contractile parameters being comparable to controls, whereas LA compliance remains significantly altered. CONCLUSION: AF-CA could reverse LA anatomical and functional remodelling. Despite improvement, LA compliance remains altered after 1 year, probably reflecting irreversible fibrosis.

Psychological effects of false-positive results in cystic fibrosis newborn screening: a two-year follow-up.

OBJECTIVE: To evaluate parental stress after a false-positive result at the time of the cystic fibrosis (CF) newborn screening (NBS), attributable to heterozygotism or persistent hypertrypsinemia. STUDY DESIGN: A prospective study was conducted in 86 French families at 3, 12, and 24 months after NBS. A psychologist conducted interviews with a questionnaire, the Perceived Stress Scale, and the Vulnerable Child Scale. RESULTS: Overall, 96.5% of parents said they had been anxious at the time of the sweat test. However, 86% felt entirely reassured 3 months after the test. The mean Perceived Stress Scale score did not differ from that observed in the French population. Mean Vulnerable Child Scale scores were high, associated with a low Parental Perception of Child Vulnerability. These results did not differ significantly at 1 and 2 years. In total, 86% to 100% of families no longer worried about CF. All parents stated that they would have the test performed again for another child. CONCLUSIONS: CF NBS can lead to false-positive results, causing parental anxiety, which quickly decreases after a sweat test performed soon after the phone call.

Clinical contributions of 64-slice computed tomography in the evaluation of cardiomyopathy of unknown origin.

BACKGROUND: Meta-analyses have confirmed the high performance of multislice computed tomography (MSCT) in coronary stenosis detection. Recent reports have described the study of left ventricular anatomy and function and coronary venous anatomy with MSCT. AIMS: We sought to compare, in patients with cardiomyopathy of unknown origin, the performance of MSCT versus angiography for significant coronary artery disease detection and versus transthoracic echocardiography (TTE) for left ventricular anatomy and function evaluation, and to assess its ability to characterize coronary venous anatomy. METHODS: Fifty-nine patients with cardiomyopathy (left ventricular ejection fraction [LVEF] less than or equal to 40%) of unknown origin, in sinus rhythm, underwent MSCT, TTE and coronary angiography. RESULTS: Twenty-four (3%) of 724 analysable coronary segments (97%) and 12 (20%) patients had significant coronary artery disease. MSCT sensitivity, specificity, and positive and negative predictive values for coronary artery disease detection were 87.5%, 98.5%, 67.7% and 99.6% in the per-segment assessment and 100%, 91%, 75% and 100% in the per-patient evaluation, respectively. Statistical analyses showed good agreement between MSCT and TTE in LVEF measurement (33+/-10% vs 32+/-11%, p=0.4, mean difference=0.7%, limits of agreement+/-13.6%) and a small LVED diameter overestimation (65.0+/-9.3mm vs 63.6+/-9.4mm, p=0.03). MSCT allowed detection of the posterolateral vein in 86% of cases. CONCLUSIONS: In selected patients presenting with idiopathic cardiomyopathy, MSCT is accurate for coronary artery disease detection and is a useful coronary venous imaging tool. MSCT studies of left ventricular function and morphology were mostly concordant with TTE measurements.

Frequency and predictors of renal artery stenosis in patients with coronary artery disease.

BACKGROUND: Renal artery stenosis (RAS) remains underdiagnosed because of nonspecific clinical manifestations, including in patients with coronary artery disease (CAD). AIMS: To estimate the prevalence and identify predictors of RAS in patients with CAD undergoing coronary angiography. SETTING: University-based medical centre. METHODS: We enrolled 650 consecutive patients (mean age=67+/-10 years, 80% men) with confirmed CAD. All patients underwent selective renal arteriography in the same procedure. We estimated the prevalence of RAS, defined as a >50% lesion. Multiple variable analysis of factors associated with presence of RAS was carried out using a logistic regression model. Variables that emerged as predictors by single-variable analysis were included in the model, along with variables that were tentatively associated with RAS, based on a literature review. RESULTS: RAS was detected in 94 patients (14.5%, 95% CI: 11.8-17.2%), including 20 (3.1%) with bilateral lesions. By single-variable analysis and presence and number of coronary artery stenoses (P<.001), hypertension (P=.001), and creatinine clearance <90 ml/min (P<.001) were associated with an increased risk of RAS. By multiple variable analysis, male sex (P<.05), presence and number of coronary artery lesions (P<.01), hypertension (P=.001), and renal insufficiency (P<.001) predicted the presence of RAS. CONCLUSIONS: The main clinical predictors of RAS in patients with CAD were hypertension, renal insufficiency, and multivessel CAD. These observations might help defining a high-risk subgroup of patients in need of meticulous investigations of both CAD and RAS.

Relationship between age at diagnosis and clinicopathologic features of renal cell carcinoma.

OBJECTIVES: To analyse the influence of age at diagnosis on tumour characteristics and cancer-specific survival in renal cell carcinoma (RCC). METHODS: Data on age, tumour characteristics, and survival for 4774 patients from 12 European RCC databases were recorded. Patients were divided into four groups according to age at diagnosis: < or =40, >40 and <60, > or =60 and <80, and > or =80 yr. The following variables were analysed: TNM stage, Fuhrman grade, tumour size, symptoms at diagnosis, ECOG performance status (PS), and cancer-specific survival. The groups were compared for usual clinical and pathologic variables, and cancer-specific survival. RESULTS: The four groups accounted for 288 (6%), 1839 (38.5%), 2499 (52.3%), and 148 cases (3.2%), respectively. Differences were found among groups for tumour stage, symptoms at diagnosis, ECOG PS, Fuhrman grade (p<0.001), tumour size, M stage, and histologic subtype (p: 0.02). Patients < or =40 yr were more likely to have papillary or chromophobe RCCs and less likely to have clear-cell RCCs. No significant difference was found among groups for N stage (p: 0.15). The 5-yr cancer-specific survival rates for the four age categories were 85%, 74%, 70%, and 69%, respectively. In multivariate analysis age category remained an independent prognostic parameter (p<0.001). CONCLUSIONS: Renal tumours diagnosed in younger age are characterized by lower tumour stages and grades as well as favourable histologic patterns compared with tumours in older patients. Basic research is required for explaining such a relationship between age, tumour aggressiveness, and therefore tumour biology.