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Background: Increasingly, patients, clinicians, and regulators call for more evidence on the impact of innovative medicines on quality of life (QoL). We assessed the effects of disease-modifying therapies (DMTs) on QoL in people with multiple sclerosis (PwMS). Methods: Randomized trials assessing approved DMTs in PwMS with results for at least one outcome referred to as "quality of life" were searched in PubMed and ClinicalTrials.gov. Results: We identified 38 trials published between 1999 and 2023 with a median of 531 participants (interquartile range (IQR) 202 to 941; total 23,225). The evaluated DMTs were mostly interferon-beta (n = 10; 26%), fingolimod (n = 7; 18%), natalizumab (n = 5; 13%), and glatiramer acetate (n = 4; 11%). The 38 trials used 18 different QoL instruments, with up to 11 QoL subscale measures per trial (median 2; IQR 1–3). QoL was never the single primary outcome. We identified quantitative QoL results in 24 trials (63%), and narrative statements in 15 trials (39%). In 16 trials (42%), at least one of the multiple QoL results was statistically significant. The effect sizes of the significant quantitative QoL results were large (median Cohen's d 1.02; IQR 0.3–1.7; median Hedges' g 1.01; IQR 0.3–1.69) and ranged between d 0.14 and 2.91. Conclusions: Certain DMTs have the potential to positively impact QoL of PwMS, and the assessment and reporting of QoL is suboptimal with a multitude of diverse instruments being used. There is an urgent need that design and reporting of clinical trials reflect the critical importance of QoL for PwMS. [ABSTRACT FROM AUTHOR]

Background: Pragmatic trials are increasingly recognized for providing real-world evidence on treatment choices. Objective: The objective of this study is to investigate the use and characteristics of pragmatic trials in multiple sclerosis (MS). Methods: Systematic literature search and analysis of pragmatic trials on any intervention published up to 2022. The assessment of pragmatism with PRECIS-2 (PRagmatic Explanatory Continuum Indicator Summary-2) is performed. Results: We identified 48 pragmatic trials published 1967–2022 that included a median of 82 participants (interquartile range (IQR) = 42–160) to assess typically supportive care interventions (n = 41; 85%). Only seven trials assessed drugs (15%). Only three trials (6%) included >500 participants. Trials were mostly from the United Kingdom (n = 18; 38%), Italy (n = 6; 13%), the United States and Denmark (each n = 5; 10%). Primary outcomes were diverse, for example, quality-of-life, physical functioning, or disease activity. Only 1 trial (2%) used routinely collected data for outcome ascertainment. No trial was very pragmatic in all design aspects, but 14 trials (29%) were widely pragmatic (i.e. PRECIS-2 score ⩾ 4/5 in all domains). Conclusion: Only few and mostly small pragmatic trials exist in MS which rarely assess drugs. Despite the widely available routine data infrastructures, very few trials utilize them. There is an urgent need to leverage the potential of this pioneering study design to provide useful randomized real-world evidence. [ABSTRACT FROM AUTHOR]

Physical activities are important to maintain and promote physical functioning and activities of daily living in people with dementia. Nurses are in a key position to deliver physical activity interventions in nursing homes. However, synthesized strategies for sustainable implementation of physical activity interventions in nursing homes for people with dementia are lacking. We aimed at synthesizing the evidence on nurse-led physical activity interventions and at identifying facilitators and barriers to sustainable implementation of physical activity interventions for people with dementia. Systematic review. We performed a comprehensive literature search combining database searches (MEDLINE, CINAHL, CENTRAL, Web of Science Core Collection; last search: September 27, 2023) and supplementary search methods (citation tracking, web searching, clinical guideline database searching). We considered studies on nurse-led physical activity interventions for people with dementia living in nursing homes for eligibility; published as journal articles and related material in English, French, German language, without restrictions on primary study design and publication year. Independently and in duplicate, we assessed the references' eligibility and the quality of the included studies. We used the Mixed Methods Appraisal Tool to appraise quality of included studies. We piloted and double-checked data extractions, and summarized the results narratively and graphically (harvest plot). We prospectively registered our review (PROSPERO CRD42021271833). We identified 24 studies (of which eleven were randomized trials) assessing various interventions that we categorized into physical exercise (n = 8), care-integrated physical activity (n = 6), outdoor or walking activity (n = 5), technology-supported physical exercise (n = 3), and dancing (n = 2). Types of outcomes and length of follow-up varied widely. Effects were inconclusive, except for outdoor or walking activities showing an overall positive impact on physical outcomes. For eight interventions, we identified evidence on implementation barriers (b) and facilitators (f), including staff shortage (b), staff time resources (b), or ease of implementation for staff (f) and adaptability of interventions (f) or refusal to participate of people with dementia (b). The results of our review provide a comprehensive overview on types, characteristics and effects of nurse-led physical activity interventions for people with dementia in nursing homes. Based on evidence from a range of study designs and sources, we came to the conclusion that all stakeholders involved considered physical activity interventions for people with dementia as useful and relevant. Outcome measures varied widely and a clear conclusion on effectiveness remains open. [ABSTRACT FROM AUTHOR]