Neurodegenerative disease management最新文献

What is this summary about?: Patient registries contain anonymous data from people who share the same medical condition. The MSBase registry contains information from over 80,000 people living with multiple sclerosis (MS) across 41 countries. Using information from the MSBase registry, the GLIMPSE (Generating Learnings In MultiPle SclErosis) study looked at real-life outcomes in 3475 people living with MS who were treated with cladribine tablets (Mavenclad^®) compared with other oral treatments.

What were the results?: Results showed that people treated with cladribine tablets stayed on treatment for longer than other treatments given by mouth. They also had fewer relapses (also called flare ups of symptoms) than people who received a different oral treatment for their MS.

What do the results mean?: The results provide evidence that, compared with other oral treatments for MS, cladribine tablets are an effective medicine for people living with MS.

Aim: Early and ongoing access to rehabilitation and exercise may preserve functional mobility and quality of life for persons with Parkinson disease (PwP). The aim of the current study was to describe the experience of PwP who participated in a 7-day retreat. Materials & methods: A phenomenological approach was used to describe the lived experience of PwP. Results: Three themes emerged from interviews: a community of shared information where participants discussed exercising and learning with other PwP; improved control of Parkinson's disease symptoms, including performing physical tasks more easily and renewed motivation for their long-term plans for exercise because of the retreat. Conclusion: A 7-day retreat for PwP positively impacted perceived control of disease-related symptoms and intentions to continue exercise.

What is this summary about?: This summary explains the findings from a recent investigation that combined the results of over 1000 people from three clinical studies to understand the safety of evobrutinib. Evobrutinib is an oral medication (taken by mouth), being researched as a potential treatment for multiple sclerosis (MS). This medication was also investigated in rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE). Over 1000 people have taken evobrutinib as part of three separate phase 2 clinical studies. These studies looked at how much of the drug should be taken, how safe the drug is, and how well it might work for treating a certain medical condition.

What were the results?: Evobrutinib was well-tolerated by participants in all three studies. The number of side effects reported by participants taking the medication was very similar to those reported by participants taking the placebo (a 'dummy' treatment without a real drug). The most common side effects in clinical studies were urinary tract infections, headache, swelling of the nose and throat, diarrhoea and blood markers of potential liver damage (these returned to normal once the treatment was stopped).

What do the results mean?: The safety data from all three clinical studies are encouraging and can be used to inform further research into using evobrutinib in MS. Clinical Trial Registration: NCT02975349 (multiple sclerosis), NCT03233230 (rheumatoid arthritis), NCT02975336 (systemic lupus erythematosus) (ClinicalTrials.gov).

Aim: This study aimed to explore the impact of caring for an individual with neurogenic orthostatic hypotension (nOH). Methods: Informal caregivers of individuals with nOH and either Parkinson's disease, multiple system atrophy, pure autonomic failure or dementia with Lewy bodies completed semi-structured interviews. Data were analyzed using thematic analysis; the identified concepts were used to develop a conceptual model. Results: Twenty informal caregivers were interviewed. Analysis identified several areas of caregiver impact due to nOH including impact on their time, particularly a need to supervise the patient to prevent falls as well as a lack of freedom and negative physical, work and social impacts. Many reported negative emotional impacts, including worry, stress or fear about the patient falling, depression and frustration. The conceptual model shows the relationships between concepts. Conclusion: The results highlight the wide-ranging impact of nOH, and the specific impact of the fear of falls on informal caregivers' lives.

Introduction: Living with a neuromuscular disease often leads to a need for specialized rehabilitation due to the complexity and progression of the diseases. Aim: To investigate cross-sectoral collaboration on rehabilitation for patients with neuromuscular diseases among hospital professionals to inform future targeted rehabilitation services. Patients & methods: The design was qualitative using the interpretive description methodology and the theoretical lens of symbolic interactionism. Ethnographic fieldwork was conducted, and 50 hospital professionals included, 19 of whom were interviewed. Results & conclusion: The results emphasize the importance of relations when collaborating across sectors. The professionals acted and made choices in relation to dilemmas and influences of diagnosis and progression, professional demarcations in multiprofessional teams, and cross-sectoral collaboration toward a mutual goal.

What is this summary about?: This plain language summary of an article published in Molecular Psychiatry, reviews the evidence supporting the role of the amyloid-β (Aβ) pathway and its dysregulation in Alzheimer's disease (AD), and highlights the rationale for drugs targeting the Aβ pathway in the early stages of the disease.

Why is this important?: Aβ is a protein fragment (or peptide) that exists in several forms distinguished by their size, shape/structure, degree of solubility and disease relevance. The accumulation of Aβ plaques is a hallmark of AD. However, smaller, soluble aggregates of Aβ - including Aβ protofibrils - also play a role in the disease. Because Aβ-related disease mechanisms are complex, the diagnosis, treatment and management of AD should be reflective of and guided by up-to-date scientific knowledge and research findings in this area. This article describes the Aβ protein and its role in AD, summarizing the evidence showing that altered Aβ clearance from the brain may lead to the imbalance, toxic buildup and misfolding of the protein - triggering a cascade of cellular, molecular and systematic events that ultimately lead to AD.

What are the key takeaways?: The physiological balance of brain Aβ levels in the context of AD is complex. Despite many unanswered questions, mounting evidence indicates that Aβ has a central role in driving AD progression. A better understanding of the Aβ pathway biology will help identify the best therapeutic targets for AD and inform treatment approaches.

Aim: To evaluate the feasibility of using activity monitors in a physical activity (PA) intervention in people with Parkinson's (PD) and Huntington's disease (HD). Materials & methods: People with early-stage PD (n = 13) and HD (n = 14) enrolled in a 4-month coaching program, wore a Fitbit, and were guided through a behavioral intervention to facilitate PA uptake. Wear time, wear habits and activity metrics (e.g., steps) were analyzed. Results: Retention rate was 85% and participants had an average 92.3% (±9.2) valid wear days. Daily wear time was 18.4 (±4.5) h. Day & night Fitbit wearers showed improvements in steps (d = 1.02) and MET×min/week (d = 0.69) compared with day-only wearers. Conclusion: Implementing wearables in a coaching intervention was feasible and provided insights into PA behavior.

Introduction: Patient-reported outcomes (PROs) are valuable measures for routine clinical care of people with multiple sclerosis (pwMS). Materials: 646 pwMS treated with interferon-β-1a (IFN-β-1a) were retrospectively included from the New York State Multiple Sclerosis Consortium. Clinical and PRO data at enrollment and 3 year follow-up were collected. PwMS with stable disease and disability worsening were matched (1:1) based on age, Expanded Disability Status Scale (EDSS) scores and disease duration. Disability worsening was determined based on trial criteria. Results: PwMS with future EDSS worsening had higher baseline and follow-up timed-25-foot walk (6.6 vs 5.5 s; 9.1 vs 5.5 s; p < 0.001) when compared with stable pwMS. Worsening pwMS reported higher baseline difficulties in getting up (odds ratio [OR] = 2.4; p = 0.009), climbing stairs (OR = 1.6; p = 0.024) and standing (OR = 2.2; p < 0.001). Worsening pwMS reported greater lower limb limitations (OR = 2.3; p = 0.004) and fatigue (OR = 1.8; p = 0.002). Conclusion: Higher fatigue and lower limb functional limitations are significant predictors of future disability worsening in pwMS.

Levodopa is the most effective agent for treating the symptoms of Parkinson's disease (PD). However, levodopa-induced dyskinesia remains a significant complication that manifests after few years of treatment, for which therapeutic options remain limited. Several agonists of the serotonin type 1A (5-HT_1A) receptor with varying levels of efficacy and interaction at other sites, have been tested in the clinic. Clinical trials testing 5-HT_1A agonists have yielded inconsistent results in alleviating dyskinesia, especially that the antidyskinetic benefit observed was often accompanied by an adverse effect on motor function. In this article, we summarize and analyze the various clinical trials performed with 5-HT_1A agonists in PD patients with dyskinesia and offer perspectives on the future of this class of agents in PD.