F.-C. Boyer

FACTORS RELATING TO CARER BURDEN FOR FAMILIES OF PERSONS WITH MUSCULAR DYSTROPHY

OBJECTIVE To assess the burden on family carers of persons with muscular dystrophy living in their homes and to determine factors contributing to carer burden. METHODS The study included 56 dyads of people with muscular dystrophy and their family carers. The variables for carer burden were compared by logistic regression in 2 carer groups (burden + /burden-). RESULTS The mean age of the patients with muscular dystrophy was 32.7 years (median 26.7, range 15-65 years) and that of the carers 51 years (median 48, range 30-80 years). The carers reported the care burden using the Zarit Burden Inventory (median score 23, range 0-57/88). Multivariate analysis produced 3 adjusted explicative factors: carer characteristics related to risk of perceived burden are self-report of poor social functioning on the SF-36 (OR = 26.6 (2.6-278); p=0.006), self report of anxiety on the Hospital Anxiety Scale (OR = 7.1 (1.4-36); p=0.02) and being a carer under 48 years of age (OR = 7.8 (1.7-34.5); p=0.007). However, it was difficult to dissociate the different health variables of the carers from each other. CONCLUSION This approach should lead to better decision-making by medical teams, patients and their carers.

Potentially inappropriate use of psychotropic medications in hospitalized elderly patients in France: cross-sectional analysis of the prospective, multicentre SAFEs cohort.

AbstractBackground: In France, there is evidence to suggest that 50% of elderly individuals are prescribed psychotropic medications. However, it is known that use of these agents increases the risk of falls, fractures and delirium in older people. Objective: To study the consumption of ‘potentially inappropriate medication’ (PIM) among patients aged ≥75 years, paying particular attention to psychotropic drugs and the factors influencing the use of ‘potentially inappropriate psychotropics’ (PIPs). Method: This was a cross-sectional analysis of a prospective multicentre cohort of 1306 hospitalized French patients aged ≥75 years (the SAFEs [Sujet Âgé Fragile: Évaluation et suivi (Frail Elderly Subjects: Evaluation and follow-up)] cohort). The present analysis involved the 1176 patients for whom there was information on the usual treatments being taken in the 2 weeks before hospitalization. The drugs were coded according to the Anatomical Therapeutic Chemical classification; the Beers list as updated in 2003 defined which medications were considered PIPs. Standardized geriatric assessment variables were recorded on inclusion in the study. Logistic regression analysis was performed to identify factors linked to use of psychotropics and PIPs. Results: The mean number of drugs taken was 5.7 ± 2.9 per patient. Twenty-eight percent of patients took at least one PIM. The number of patients who had taken at least one psychotropic drug in the 2 weeks before hospitalization (mean 1.6 ± 0.9 psychotropics per patient) was 589 (50.1%). More than half of both the 510 patients with a depressive syndrome and the 543 patients affected by dementia were treated with psychotropics. Multivariate analysis showed that prescription of psychotropics was linked to the presence of a dementia syndrome (odds ratio [OR] = 1.4; 95% CI 1.1, 1.9; p = 0.03), the presence of a depressive syndrome (OR = 1.7; 95% CI 1.3, 2.1; p < 0.001), living in an institution (OR = 2.2; 95% CI 1.5, 3.4; p < 0.001), use of more than five drugs (OR = 3.2; 95% CI 2.5, 4.2; p < 0.001) and Charlson’s co-morbidity score >1 (OR = 0.6; 95% CI 0.5, 0.8; p = 0.001). Nineteen percent of all psychotropics prescribed were PIPs. Of these PIPs, 66.5% were anxiolytics, 28.4% were antidepressants and 5.1% were antipsychotics. Use of PIPs in the multivariate analysis was associated only with consumption of more than five drugs (OR = 1.7; 95% CI 1.1, 2.5; p = 0.01). Conclusion: PIM use is common among hospitalized older adults in France. The most important determinant of risk of receiving a psychotropic medication or a PIP was the number of drugs being taken. The elderly, who have multiple comorbidities, complex chronic conditions and are usually receiving polypharmacy, are at increased risk for adverse drug events. These adverse events are often linked to problems that could be preventable such as delirium, depression and falls. Regular review of prescriptions would help optimize prescription of psychotropics in the elderly. The Beers list is a good tool for evaluating PIMs but is too restrictive with respect to psychotropics; in the latter respect, the list could usefully be widened.

Methodological issues regarding power of classical test theory (CTT) and item response theory (IRT)-based approaches for the comparison of patient-reported outcomes in two groups of patients - a simulation study

BackgroundPatients-Reported Outcomes (PRO) are increasingly used in clinical and epidemiological research. Two main types of analytical strategies can be found for these data: classical test theory (CTT) based on the observed scores and models coming from Item Response Theory (IRT). However, whether IRT or CTT would be the most appropriate method to analyse PRO data remains unknown. The statistical properties of CTT and IRT, regarding power and corresponding effect sizes, were compared.MethodsTwo-group cross-sectional studies were simulated for the comparison of PRO data using IRT or CTT-based analysis. For IRT, different scenarios were investigated according to whether items or person parameters were assumed to be known, to a certain extent for item parameters, from good to poor precision, or unknown and therefore had to be estimated. The powers obtained with IRT or CTT were compared and parameters having the strongest impact on them were identified.ResultsWhen person parameters were assumed to be unknown and items parameters to be either known or not, the power achieved using IRT or CTT were similar and always lower than the expected power using the well-known sample size formula for normally distributed endpoints. The number of items had a substantial impact on power for both methods.ConclusionWithout any missing data, IRT and CTT seem to provide comparable power. The classical sample size formula for CTT seems to be adequate under some conditions but is not appropriate for IRT. In IRT, it seems important to take account of the number of items to obtain an accurate formula.

Post-polio syndrome and rehabilitation

Post-polio syndrome (PPS) is the commonly affected term to describe the symptoms that may develop many years after acute paralytic poliomyelitis. The etiology of PPS is still unclear. An overuse of enlarged motor units is suspected causing denervation again due to distal degeneration of axons. Metabolic and functional changes has been described in muscle fibers of partially denervated muscles. Nevertheless, submaximal aerobic training and low intensity muscular strengthening have shown positive effects on muscular strength and cardiorespiratory system in patients affected by PPS. Aquatic therapy has a positive impact on pain and muscle function. In patients with severe fatigue, it is recommended to adapt the daily exercise routine to their specific case.

Health status in patients with Alzheimer's disease: An investigation of inter-rater agreement

Objective: The purpose of the present study was to examine the level of agreement between health status ratings provided by patients with Alzheimer’s disease and by their proxies. Background: Because proxy-completed responses are often necessary in assessing health outcomes for the elderly, it is necessary to determine the feasibility and potential limitations of using proxies as a patient substitutes. Methods: To assess the potential utility of proxy responses on health status when subjects present a cognitive impairment, this study compared the responses of 70 subjects with Alzheimer‘s disease and those of their family and/or care provider proxy using the SF-36. Agreement between proxies and patients was measured by intraclass correlation coefficients (ICCs). Results: The proportion of exact agreement between patients and proxies on the 36 items ranged from 3.3 to 41.7%. Results reveal poor to moderate agreement between patient and proxy reports. Proxy reliability varied according to the relationship of the proxy to the index subject. Agreement decreased significantly with increasing severity of dementia and with increasing severity of Physical status (Katz ADL). Agreement was better for measures of functions that are directly observable and relatively poor for more subjective measures. Conclusions: Our results confirm the importance of the information source used for patient health status.

Post-polio syndrome: Pathophysiological hypotheses, diagnosis criteria, medication therapeutics

Post-polio syndrome (PPS) refers to a clinical disorder affecting polio survivors with sequelae years after the initial polio attack. These patients report new musculoskeletal symptoms, loss of muscular strength or endurance. PPS patients are tired, in pain and experience new and unusual muscular deficits, on healthy muscles as well as deficient muscles initially affected by the Poliovirus. Once a clinical diagnosis is established, the therapeutic options can be discussed. Some pathophysiological mechanisms have been validated by research studies on PPS (inflammatory process in cerebrospinal fluid [CSF] and cytokines of the immune system). Several studies have been conducted to validate medications (pyridostigmine, immunoglobulin, coenzyme Q10) or physical exercises protocols. This article focuses on the relevance and efficacy that can be expected from these therapeutics. Very few studies reported some improvements. Medications combined to individual and supervised exercise training programs are promising therapeutic strategies for PPS patients care management.

Gender as a Modifying Factor Influencing Myotonic Dystrophy Type 1 Phenotype Severity and Mortality: A Nationwide Multiple Databases Cross-Sectional Observational Study.

Background Myotonic Dystrophy type 1 (DM1) is one of the most heterogeneous hereditary disease in terms of age of onset, clinical manifestations, and severity, challenging both medical management and clinical trials. The CTG expansion size is the main factor determining the age of onset although no factor can finely predict phenotype and prognosis. Differences between males and females have not been specifically reported. Our aim is to study gender impact on DM1 phenotype and severity. Methods We first performed cross-sectional analysis of main multiorgan clinical parameters in 1409 adult DM1 patients (>18y) from the DM-Scope nationwide registry and observed different patterns in males and females. Then, we assessed gender impact on social and economic domains using the AFM-Téléthon DM1 survey (n = 970), and morbidity and mortality using the French National Health Service Database (n = 3301). Results Men more frequently had (1) severe muscular disability with marked myotonia, muscle weakness, cardiac, and respiratory involvement; (2) developmental abnormalities with facial dysmorphism and cognitive impairment inferred from low educational levels and work in specialized environments; and (3) lonely life. Alternatively, women more frequently had cataracts, dysphagia, digestive tract dysfunction, incontinence, thyroid disorder and obesity. Most differences were out of proportion to those observed in the general population. Compared to women, males were more affected in their social and economic life. In addition, they were more frequently hospitalized for cardiac problems, and had a higher mortality rate. Conclusion Gender is a previously unrecognized factor influencing DM1 clinical profile and severity of the disease, with worse socio-economic consequences of the disease and higher morbidity and mortality in males. Gender should be considered in the design of both stratified medical management and clinical trials.

Predictors of rapid cognitive decline among demented subjects aged 75 or more: ('Sujet Agé Fragile--Evaluation et Suivi' Cohort-SAFES).

To identify factors predictive of rapid cognitive decline (RCD) among elderly subjects aged 75 or over suffering from dementia.

Aerobic capacities and exercise tolerance in neuromuscular diseases: A descriptive study

INTRODUCTION Up until now, there has been little description of the test exercises carried out in patients with neuromuscular diseases. However, prescription of physical therapy by exercise requires rigorous individualized assessment of the patients physical endurance parameters. OBJECTIVE To describe exercise tolerance and external limitation factors in a sample group of adult patients suffering from neuromuscular diseases. MATERIALS AND METHODS A descriptive retrospective study of exercise tests realized at the Reims university hospital for patients divided into three groups according to their pathologies: CMT hereditary neuropathies, muscular dystrophies, metabolic myopathies. RESULTS Forty-four (44) tests were analyzed. Cessation was occasioned on 63.6% of the cases by muscular symptomatology, with no possibility of maintaining the cycling frequency in 29.5% of the overall population. Insufficient lung volume recruitment was involved in 61.4% of the patients, final oxygen pulse (VO2/heart rate) was 80% lower than the theoretical pulse in 50% of the patients, and there existed an early first ventilatory threshold in 54.5% of the cases. Peripheral deconditioning was highly severe in 18% of the population and significantly greater in the subjects suffering from dystrophies (VT1 at 31% of the maximum theoretical O2, P<0.001). DISCUSSION The main limitation factor in exercise tests is peripheral. Ventilatory and cardiovascular limitations can nonetheless be added on; while they are absent when the patient is at rest, they are unmasked in effort situations. Exercise tests could be of great interest in screening and managing the relevant pathologies. Multicenter studies on homogeneous populations could facilitate definition of the procedures specific to exercise tests for patients suffuring from neuromuscular diseases.

Effect of Whole Body Cryotherapy interventions on health-related quality of life in fibromyalgia patients: a randomized controlled trial

INTRODUCTION Although fibromyalgia syndrome (SFM) affects 2-4 percent of adults, research has not identified a preferred therapeutic option for patients worldwide yet. Based on recent findings, it can be expected that whole body cryotherapy can improve health-reported quality of life by alleviating the symptoms of musculoskeletal pain and fatigue. OBJECTIVE Our aim was to determine whether whole body cryotherapy only can result in improved perceived health and quality of life in fibromyalgia patients. METHODS 24 patients with fibromyalgia diagnosis were randomized into 2 groups (n=11 in the whole body cryotherapy group, n=13 in the control group). In the whole body cryotherapy group, 10 sessions of whole body cryotherapy were performed (in addition to usual care) in a standard cryotherapy room over a duration of 8days. Subjects in the control group did not change anything in their everyday activities. Quality of life was assessed just before and one month after treatment. RESULTS Compared with the control group, patients in the whole body cryotherapy group reported significantly improved for health-reported quality of life. These effects lasted for at least one month following intervention. CONCLUSION Based on these findings, whole body cryotherapy can be recommended as an effective clinically adjuvant approach in the improvement of health-related quality of life in fibromyalgia patients.