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The Patient Information Sheet (PIS) is an important aspect of the consent process in a clinical trial that provides potential participants the necessary information for deciding whether to take part in a specific study and for understanding their rights pertaining to participation. User Testing was originally developed to assess how written information about medicinal products performs with its intended users. User testing has been proposed in a small number of clinical trials and has been able to identify subjects' ability or inability to find and understand important information related to providing a valid consent to participate. A 21-item ad hoc user testing questionnaire was applied to 2 PIS used in clinical trials in MS. Sixty subjects were allocated to a group testing an observational study PIS (O-PIS) or to a group testing an intervention study PIS (I-PIS). In the O-PIS group, 19.4% of subjects located all relevant information within the text (21/21) and 3.4% of subjects in the I-PIS group. Overall, 82.1% of subjects testing the O-PIS understood the text and 53.5% of subjects testing the I-PIS understood the text. In the category 'nature and purpose of the trial', one-third of subjects did not understand the text, including the aim of the study. User testing should be considered as a valid tool in evaluating the comprehensibility of PIS in the context of clinical trials MS to assure that subjects provide a valid consent to participate.

Background Everyday-life activities often require performing dual tasks (DT), with consequent possible occurrence of motor-cognitive or motor-motor interference. This could reduce quality of life, in particular in people with neurological diseases. However, there is lack of validated tools to assess the patients’ perspective on DT difficulties in this population. Therefore, we developed the Dual-task Impact on Daily-living Activities-Questionnaire (DIDA-Q) and tested its psychometric properties in people with multiple sclerosis (PwMS). Methods Items were generated based on existing scales, DT paradigms used in previous studies and the opinion of a multi-stakeholder group, including both experts and PwMS. Twenty DT constituted the preliminary version of the DIDA-Q which was administered to 230 PwMS. The psychometric properties of the scale were evaluated including internal consistency, validity and reliability. Results Nineteen items survived after exploratory factor analysis, showing a three-factor solution which identifies the components mostly contributing to DT perceived difficulty (i.e., balance and mobility, cognition and upper-limb ability). The DIDA-Q appropriately fits the graded response model, with first evaluations supporting internal consistency (Cronbach's alpha=0.95), validity (70% of the hypotheses for convergent and discriminant constructs confirmed) and reliability (intraclass correlation coefficients=0.95) of this tool. Conclusion The DIDA-Q could be used in research and clinical settings to discriminate individuals with low vs. high cognitive-motor or motor-motor interference, and to develop and evaluate the efficacy of personalized DT rehabilitative treatments in PwMS.

The purpose of this study is to analyze the role of genes known to influence muscle performances on the outcome after enzyme replacement treatment (ERT) in type II Glycogenosis (GSDII). We analyzed 16 patients receiving ERT for ≥ two years. We assessed the changes in muscle strength by hand-held dynamometry, muscle mass by quantitative MRI, and resistance to exercise by the 6-minute walking test. Exercise gene assessment included angiotensin converting enzyme insertion/deletion polymorphism (ACE), alpha-actinin3 R577X polymorphism (ACTN3), and peroxisome proliferator activated receptor alpha G/C polymorphism (PPARα). Independent of disease severity, one third of patients had a poor response to ERT, which was found to be associated with ACE DD genotype. The ACTN3 null polymorphism appeared to exert a positive effect on treatment efficacy, while PPARα did not seem to exert any influence at all. We conclude that poor treatment outcome in ACE DD genotypes is in line with previous observation of a worse disease course in this subpopulation, and suggests the need for a more careful follow-up and individualized treatment approaches for these patients. Exercise genes may provide a new opportunity for studying the outcome after treatment and the muscle regeneration abilities in other models of genetic myopathies.

Background Exposure to allergen may induce a modified T H 2 response characterized by high IgG 4 levels, absence of IgE sensitization, and a decreased risk of allergic respiratory symptoms. Objective To assess the association of IgG 4 level with allergic respiratory symptoms in a community-based sample of adults. Methods Information on exposure to cats, respiratory symptoms, and mattress allergen levels was obtained from 2780 adults. Levels of cat and house dust mite (HDM) specific IgE, IgG, and IgG 4 were measured. The association of exposure to allergen with IgG 4 and of IgG 4 with symptoms was assessed. Results Geometric mean (GM) cat specific IgG and IgG 4 was higher in subjects who had a cat that was allowed in the bedroom than in subjects without a cat (adjusted ratio of GM IgG 4 , 1.41; 95% CI, 1.25-1.57). Levels of HDM specific IgG and IgG 4 were similar in subjects with undetectable and high (>20.22 μg/g) mattress Der 1 levels (adjusted ratio of GM IgG 4 , 1.02; 95% CI, 0.89-1.17). There was no evidence that high cat or HDM specific IgG 4 levels were associated with less IgE sensitization or with fewer symptoms. Conclusion In this community-based sample of adults, high IgG 4 levels to cat or HDM were not associated with a lower risk of allergic respiratory symptoms. Clinical implications In adults, high cat allergen exposure does not protect against respiratory symptoms.

Background: Several studies in European homes have described allergen levels from the house dust mite species Dermatophagoides pteronyssinus and to a lesser extent Dermatophagoides farinae, but geographic comparisons of exposure levels and risk factors have been hampered by a lack of standardized methods. Objective: To study the distribution and determinants of the major house dust mite allergens Der p 1 and Der f 1 in 10 European countries using a common protocol. Methods: During home visits with 3580 participants of the European Community Respiratory Health Survey II from 22 study centers, mattress dust was sampled and analyzed for Der p 1, Der f 1, and Der 2 allergen. Information on housing characteristics was obtained by both observations and interview. Results: Der 1 and Der 2 allergens were detectable (≥0.1 μg/g) in 68% and 53% of the samples, respectively. Large differences in allergen levels between study centers were observed, and geographic patterns for Der p 1 and Der f 1 were different. Low winter temperatures reduced Der p 1 rather than Der f 1. Important risk factors for high allergen levels included an older mattress, a lower floor level of the bedroom, limited ventilation of the bedroom, and dampness for Der p 1 but not for Der f 1. Conclusion: There are large qualitative and quantitative differences of house dust mite allergen levels in Europe, which can partly be explained by geographic and housing characteristics. Clinical implications: Mite allergen exposure may be reduced by replacing the mattress regularly and increasing ventilation of the bedroom, particularly in winter. © 2006 American Academy of Allergy, Asthma and Immunology.