No abstract

Neuromuscular disorders are rare diseases with a chronic and debilitating course. Unfortunately, data on the health-related quality of life (HRQoL) in neuromuscular diseases are limited. The objective of this multicentre cross-sectional study was to compare the HRQoL in patients with amyotrophic lateral sclerosis (ALS), facioscapulohumeral muscular dystrophy (FSHD) and myasthenia gravis (MG) and to identify the determinants of the HRQoL in these diseases. We recruited 91 consecutive outpatients with ALS (n = 37), FSHD (n = 17) or MG (n = 37) in seven specialized German health centres. The HRQoL was determined using the 36-Item Short Form Health Survey (SF-36) and the EuroQol (EQ-5D). Independent predictors of the HRQoL were identified using multiple regression analysis. The HRQoL in all domains of the SF-36, except for bodily pain, was significantly reduced. The domains related to physical health (physical functioning, physical role) were most affected. The EQ-5D-index score was most reduced in ALS (0.54) and least reduced in MG (0.89). Independent predictors of a reduced HRQoL were disease severity and depression in ALS, and disease severity, depression, older age and increased body-mass index in MG. The patterns of HRQoL-impairment in neuromuscular disorders share some common features, such as a more pronounced reduction in the HRQoL related to physical health, but there are a number of disease-specific features that should be considered in outcomes of clinical trials and treatment guidelines. In addition to the treatment of motor symptoms, greater attention should be paid to the treatment of depression, which was found to be among the independent predictors of the HRQoL in ALS and MG.

Neuromuscular disorders (NMD) are chronic devastating diseases. The aim of this multicenter cross-sectional study was to evaluate the socioeconomic impact of three NMDs in Germany. Patients (n = 107) with amyotrophic lateral sclerosis (ALS), myasthenia gravis (MG) or facioscapulohumeral muscular dystrophy (FSHD) were recruited consecutively in seven centers in Germany. The health-economic data were collected using a "bottom-up" approach consisting of comprehensive questionnaires and patient diaries. Costs were evaluated from the societal perspective in 2009 Euros (EUR). Total annual costs from the societal perspective were EUR 36,380 (95% CI 27,090-47,970) per patient in ALS, EUR 26,240 (95% CI 17,770-37,940) in FSHD and EUR 14,950 (95% CI 10,470-21,730) in MG. The main components of costs were the expenditures of health insurance and the loss of productivity of patients and their caregivers. The following independent cost-driving factors were identified: disease severity, assistance in activities of daily living (ADL), dementia and younger age in ALS, disease severity in FSHD and assistance in ADL, disease severity and assistance in ADL in MG. The socioeconomic burden of NMDs in Germany is considerable. Further studies evaluating both the health-economic and clinical effects of NMD treatment as well as disease management programs and benchmarking activities are necessary.

Measurement of NAA concentrations and NAA/Cho ratios appear to be most suitable for detection of motor cortex degeneration by single-volume proton magnetic resonance spectroscopy. Reduced NAA/Cho ratios correspond to aspects of the clinical presentation and reflect disease progression in follow-up measurements.

BackgroundInfection is the leading cause of morbidity and the second leading cause of mortality in patients on renal replacement therapy. The rates of bloodstream infection in hemodialysis patients vary according to the type of venous access used. Gram-positive bacteria are most frequently isolated in blood cultures of hemodialysis patients. This study evaluated risk factors for the development of bloodstream infections in patients undergoing hemodialysis.MethodsRisk factors associated with bloodstream infections in patients on hemodialysis were investigated using a case–control study conducted between January 2010 and June 2013. Chronic renal disease patients on hemodialysis who presented with positive blood cultures during the study were considered as cases. Controls were hemodialysis patients from the same institution who did not present with positive blood cultures during the study period. Data were collected from medical records. Logistic regression was used for statistical analysis.ResultsThere were 162 patients included in the study (81 cases and 81 controls). Gram-positive bacteria were isolated with the highest frequency (72%). In initial logistic regression analysis, variables were hypertension, peritoneal dialysis with previous treatment, type and time of current venous access, type of previous venous access, previous use of antimicrobials, and previous hospitalization related to bloodstream infections. Multiple regression analysis showed that the patients who had a central venous catheter had an 11.2-fold (CI 95%: 5.17–24.29) increased chance of developing bloodstream infections compared with patients who had an arteriovenous fistula for vascular access. Previous hospitalization increased the chance of developing bloodstream infections 6.6-fold (CI 95%: 1.9–23.09).ConclusionsInfection prevention measures for bloodstream infections related to central venous catheter use should be intensified, as well as judicious use of this route for vascular access for hemodialysis. Reducing exposure to the hospital environment through admission could contribute to a reduction in bloodstream infections in this population.