Neurodegenerative disease management最新文献

Aim: Patients with relapsing-remitting multiple sclerosis (RRMS) treated with natalizumab have anecdotally reported a 'feel-good experience' (FGE). The authors characterized the FGE using survey data from patients with RRMS treated with natalizumab or other disease-modifying therapies (other-DMT). Methods: Questionnaire data from RRMS patients who use MyMSTeam, an online patient social network, were analyzed. Results: The survey included 347 patients (95 natalizumab; 252 other-DMT). More natalizumab than other-DMT patients self-reported having an FGE (62.1 vs 44.8%; p = 0.001) as well as other physical, emotional and cognitive benefits. Conclusion: This study demonstrates that physical, emotional and cognitive benefits were more commonly reported by patients treated with natalizumab than those treated with other disease-modifying therapies and helps characterize patient-reported factors associated with the FGE.

Aim: This study explores the burden of caring for an individual with neurogenic orthostatic hypotension (nOH) and an underlying neurodegenerative disease (Parkinson's disease, multiple system atrophy, pure autonomic failure or dementia with Lewy bodies). Materials & methods: A survey including several validated instruments was conducted with informal caregivers of individuals with Parkinson's disease, multiple system atrophy, pure autonomic failure or dementia with Lewy bodies. Results: Caregivers of patients with nOH (n = 60) reported greater burden across all outcomes compared with those without nOH (n = 60). Receiving pharmacological treatment for nOH was the variable most consistently associated with significantly better caregiver health-related quality-of-life (p < 0.05). Conclusion: This study demonstrates the burden of nOH on informal caregivers and highlights the potential benefit of pharmacological treatment not only for patients but also indirectly, their caregivers.

What is this summary about?: This is a summary of an article originally published in the journal Advances in Therapy. Cladribine tablets are approved for treating people with relapsing multiple sclerosis (shortened to MS). People with MS take cladribine tablets for 2 periods of 4 to 5 days per year. This analysis looks at the results from 2 studies called the CLARITY and CLARITY Extension studies. These studies looked at what effect a 2-year course of treatment with cladribine tablets had on disability over 5 years in people with MS.

How was the analysis carried out?: In this analysis, researchers measured disability worsening at regular intervals during the 2-year treatment period in the CLARITY study and thereafter in the 2-year CLARITY Extension study. As many patients had a bridging interval between CLARITY and CLARITY extension, the researchers were able to assess disability over a 5-year timeframe.

What were the results?: When measurements were taken at Year 5 of the study, disability remained stable in more than half of participants. Over the 5-year period, 70% of participants did not experience persistent disease worsening that lasted more than 6 months.

What do the results mean?: Researchers concluded that a 2-year course of cladribine tablets may provide long-term benefits on disability for up to 5 years. Clinical Trial Registration: NCT00213135 (ClinicalTrials.gov) Clinical Trial Registration: NCT00641537 (ClinicalTrials.gov).

What is this summary about?: This article summarizes the findings from a previously published article in Current Medical Research and Opinion. Cladribine tablets are an oral treatment for relapsing multiple sclerosis (shortened to MS), that are given for 4 periods of 4 to 5 days over 2 years (for a total of 20 days). In this analysis, researchers looked at the effects of taking either cladribine tablets or placebo (dummy pills) in a group of people with MS who had more active MS inflammation and had participated in a clinical study (called the CLARITY study). Some of these participants had taken prior medicines for MS.

What were the results?: Researchers found that in people with more active MS, treatment with cladribine tablets led to a lower risk of relapse and there were more people who had no relapses. People also had a lower chance of their MS worsening and had fewer new lesions in the brain. These benefits were seen regardless of whether the participants had prior treatment.

What do the results mean?: Researchers concluded that in these people with more active MS, treatment with cladribine tablets led to better outcomes over 2 years compared with treatment with placebo tablets, regardless of whether the participants had taken any prior MS treatments. Clinical Trial Registration: NCT00213135 (ClinicalTrials.gov).

Aim: The present study was conducted to assess the impact of gene (vitamin D receptor [VDR] polymorphisms) - environment (serum vitamin D and calcium levels) interaction on multiple sclerosis (MS) risk. Materials & methods: FokI, BsmI, TaqI and ApaI genotyping were performed in 149 MS patients and 127 controls. We measured serum vitamin D and calcium levels. Results: No significant difference between VDR polymorphisms and MS risk was detected. In patients with FokI ff, BsmI Bb, TaqI Tt and ApaI AA genotypes, vitamin D levels were statistically higher. Serum calcium levels were significantly lower in patients with FokI FF, Ff, all BsmI and TaqI genotypes and ApaI AA and Aa genotypes. Conclusion: No significant association was found between VDR polymorphisms with MS risk.

What is this summary about?: This is a summary of an article originally published in the Multiple Sclerosis Journal. The article presents the results from the CLARITY and CLARITY Extension studies, which looked at how well cladribine tablets work in treating people with multiple sclerosis (shortened to MS). Cladribine tablets are a medication approved for treating relapsing forms of MS. This study looked at how treatment with cladribine tablets affects the frequency and severity of relapses in people with MS. A relapse is when new symptoms develop or old symptoms return or get worse after a period of stability or improvement.

What happened in the studies?: In the CLARITY study, 870 people received either cladribine tablets (3.5 mg/kg, the approved dose) or placebo (a dummy pill). After the CLARITY study ended, some participants who received cladribine tablets chose to take part in a second study called the CLARITY Extension study. Of these participants, 98 were given placebo for 2 more years following an interval period of up to 10 months between participating in each study.

What were the results?: People with MS who were treated with cladribine tablets for 2 years in the CLARITY study had lower risks of any relapse compared with those given placebo. Participants taking placebo (after cladribine tablets) in the CLARITY Extension study experienced these same benefits, which continued for up to 3 more years after having received cladribine tablets in the CLARITY study.

What do the results mean?: Researchers concluded the recommended 2-year dosing of cladribine tablets may reduce the number and severity of relapses in people with MS for up to 5 years. Clinical Trial Registration: NCT00213135 (ClinicalTrials.gov) Clinical Trial Registration: NCT00641537 (ClinicalTrials.gov).

Aim: To evaluate glatiramer acetate (GA) or IFN-β effects on quality of life (QoL) in people with relapsing/remitting multiple sclerosis (PwRRMS) in Greece. Methods: A prospective, practice-based study. QoL/function/symptoms were assessed by seven questionnaires/scales. Results: Significant increases in Short Form-36 (SF-36) health survey scores occurred with GA in four of the eight domains and three of the eight domains at 6 and 12 months, respectively, versus baseline. Similar and significant SF-36 score improvements occurred with GA in treatment-naive PwRRMS. SF-36 scores were unaffected in GA-treated, IFN-β treatment-experienced PwRRMS, or with IFN-β versus baseline. Slight improvements in fatigue and sexual satisfaction were evident (6 months). No deteriorations were seen in the other four instruments. Conclusion: The findings show that 12-month treatment with GA, but not IFN-β, improved certain QoL parameters in treatment-naive PwRRMS.

What is this summary about?: This is a plain language summary of an article published in Alzheimer's & Dementia. It looks at a type of test called a lumbar puncture (also known as spinal tap) used in people suspected of having Alzheimer's disease or some other form of dementia. This summary focuses on how to do a lumbar puncture safely.

Why is this important?: Alzheimer's disease is a progressive condition, which means it gets worse over time. This leads to difficulties with thinking and memory. People with Alzheimer's disease show a build up of proteins called amyloid-β and tau in the brain. This is followed by a gradual loss of brain cells and brain function. The changes in the brain are thought to occur years before symptoms appear. Lumbar puncture is a medical procedure during which samples of cerebrospinal fluid are collected. In Alzheimer's disease, it is used to examine cerebrospinal fluid biomarkers that can help diagnose disease. Lumbar puncture is traditionally considered as a painful and invasive procedure with frequent side effects. However, multiple studies indicate that a lumbar puncture can be performed safely. Side effects are typically mild and do not require specialist intervention.

What are the key takeaways?: Despite the low risk of serious complications associated with a lumbar puncture, physicians and their patients may be reluctant to recommend or undergo this procedure. Patient education, specialist training, as well as new methods concerning patient safety are important factors to support the widespread use of lumbar puncture in Alzheimer's disease.

Friedreich's ataxia (FRDA), a neurodegenerative disease characterized by ataxia and other neurological features, affects 1 in 50,000-100,000 individuals in the USA. However, FRDA also includes cardiac, orthopedic and endocrine dysfunction, giving rise to many secondary disease characteristics. The multifaceted approach for clinical care has necessitated the development of disease-specific clinical care guidelines. New developments in FRDA include the advancement of clinical drug trials targeting the NRF2 pathway and frataxin restoration. Additionally, a novel understanding of gene silencing in FRDA, reflecting a variegated silencing pattern, will have applications to current and future therapeutic interventions. Finally, new perspectives on the neuroanatomy of FRDA and its developmental features will refine the time course and anatomical targeting of novel approaches.