Neurodegenerative disease management最新文献

Aims: This retrospective cohort study describes real-world safety and tolerability outcomes in United States-based edaravone-treated patients with ALS.

Patients & methods: Amerita Specialty Infusion Services provided IV edaravone to patients with ALS treated with their first dose between 25 September 2017-30 September 2022. Mean ALS Functional Rating Scale-Revised (ALSFRS-R) scores and forced vital capacity (FVC) %-predicted measures were recorded within ± 100 days from care initiation to approximate baseline values.

Results: Included patients (n = 243) received/were still receiving IV edaravone/edaravone oral suspension as of 30 September 2022. At initiation, 66.7% were male, mean age ± SD was 61.2 ± 11.2 years, and 61.3% were covered by government insurance. In patients with provider-recorded ALSFRS-R (n = 115) and FVC (n = 84) %-predicted measures within ± 100 days from care initiation, mean ± SD values were 35.1 ± 8.9 and 72.3% ± 21.7%, respectively. Mean ± SD therapy duration was 13.5 ± 11.4 months. Discontinuation reasons included death/hospice (n = 82), patient's choice (n = 38), doctor's choice (n = 31), insurance (n = 18), and other (n = 12). Reasons for IV edaravone discontinuation and IV edaravone administration access device were not associated.

Conclusions: Treatment discontinuation was primarily related to ALS disease progression/death, rather than safety or tolerability. This study representative of real-world patients with ALS suggests that edaravone showed consistent safety and tolerability profiles with previous studies.

Multiple sclerosis [MS] is an immune-mediated disease driven by peripherally mediated and central nervous system compartmentalized pathophysiologic processes. Major advances have led to a plethora of disease modifying therapies [DMTs] available for the treatment of MS. Among the wide range of DMTs, the immune reconstitution therapies [IRTs] are a unique subset of therapy that have the potential for durable benefit without the need for chronic administration. Cladribine tablets are a higher-efficacy oral IRT that selectively targets lymphocytes, causing a transient lymphopenia, followed by immune reconstitution of a different repertoire of lymphocytes that may result in durable benefit. The intermittent dosing schedule and efficacy in reducing measures of disease activity in both the short and long-term make cladribine an attractive DMT choice for a broad range of individuals with relapsing forms of MS. This narrative review summarizes the available efficacy and safety data from clinical trials and real-world studies on cladribine tablets.

Parkinson's Disease (PD) is the second most common neurodegenerative disorder. Hypokinetic dysarthria, a motor speech disorder affects approximately 90% of PD patients. In Morocco, linguistic features of Moroccan Arabic may influence how voice impairment appear in PD. This study investigates the acoustic characteristics of Moroccan PD patients' voices. This was a case-control study involving 30 Moroccan PD patients, both medicated and unmedicated) and 30 healthy control participants. Voice recordings were collected from participants before and after dopaminergic treatment. Four tasks were performed: sustained vowel production, spontaneous speech, diadochokinesis, and reading tasks in both Arabic and French. The Unified Parkinson's Disease Rating Scale was computed for each state and data were analyzed using PRAAT software (v6.2), for phonatory and articulatory parameters. PD patients exhibited significant alterations in shimmer, jitter, and Harmonics-to-Noise and Noise-to-Harmonic Ratios, reflecting pitch and vocal intensity instability. A "hoarse" and "breathy" vocal quality was noted. Rhythm disturbances were marked by an increased number of long pauses (≥500 ms), indicating difficulty in sustaining speech flow. No adverse events were reported. Moroccan PD patients exhibit distinctive voice and rhythm abnormalities, particularly in vowel articulation, phonation, and timing. These markers may serve as reliable indicators for PD diagnosis and progression.

Paratonia is a type of hypertonia with involuntary resistance to passive movement depending on the pace and force being applied. People with paratonia may find it challenging to modify their movements and posture. Limited awareness among healthcare professionals can lead to a delay in diagnosis and inadequate treatment. Here, we present two cases diagnosed with dementia due to Alzheimer's disease. The two patients developed stiffness in their bodies as the dementia progressed. Paratonia was diagnosed using the Paratonia Assessment Inventory. Severity of paratonia was assessed using the Modified Ashworth scale for paratonia (MAS-P). The caregiver's primary concern was the stiffness of the body, which created difficulties in routine care. Amantadine was initiated in both patients. Both patients demonstrated improvement in paratonia following amantadine treatment, with reduced stiffness and greater ease in caregiving tasks. The present case series highlights the role of amantadine in the management of paratonia and can contribute to the development of more treatment options.

Introduction: Parkinson's disease (PD) is a progressive neurodegenerative disorder with no known cure, affecting both motor and non-motor functions. This review examines the therapeutic potential of dance as a holistic intervention to complement conventional PD treatments.

Methods: This systematic review followed PRISMA guidelines. Studies included were randomized controlled trials, longitudinal studies, and meta-analyses evaluating dance interventions in PD. Databases searched included PubMed, Scopus, and Web of Science. Exclusion criteria were case studies and non-peer-reviewed sources.

Results: Sixty-six studies involving over 1,200 participants were identified. Evidence shows that dance improves motor functions such as gait, balance, and coordination, while also enhancing non-motor outcomes including mood, anxiety, depression, and cognition. Dance's rhythmic movements, cognitive demands, and social interaction stimulate neurochemical pathways linked to motor and emotional regulation. Furthermore, group dance fosters social connectedness and reduces isolation. Online and community-based programs demonstrate feasibility and accessibility across diverse populations.

Conclusions: Dance provides multidimensional benefits for individuals with PD, spanning physical, cognitive, emotional, and social domains. Despite these promising findings, limitations such as small sample sizes, methodological heterogeneity, and lack of direct comparisons with other exercise modalities remain. Larger standardized trials are needed to confirm efficacy and support integration of dance into PD treatment programs.

Background: To identify if a combination of blood-based biomarkers related to inflammation and oxidative stress predict treatment response to nabilone for Alzheimer's disease (AD)-associated agitation.

Research design and methods: Agitation was assessed using the Cohen-Mansfield Agitation Inventory (CMAI). Serum concentrations of 13 markers were quantified. Univariable and multivariable regression were used to determine differences in CMAI change given nabilone and placebo. A model combining biomarkers with clinical predictors was also evaluated.

Results: Overall, 38 participants enrolled in the original trial (76% male, mean ± SD age 87 ± 10). Nabilone was more efficacious in participants with higher IL-6, higher 8-ISO, higher 24S-OHC, and lower clusterin. Participants in the first tertile (T1) of index scores demonstrated better response to nabilone compared to placebo with a mean difference in CMAI change of -20.6 (95%CI: -30.3, -10.4). During the nabilone phase, 83% of participants in T1 were responders versus 38% in T2 + 3 (Fisher's p = .01). In the combined model, T1 showed better response to nabilone with a mean difference in CMAI change of -26.4 (95%CI: -34.0, -19.6). The proportion of responders was significantly higher in T1 (91%, n = 11) compared to T2 + 3 (32%, n = 19) (Fisher's p = .002).

Conclusion: A combination of biomarkers could help characterize responders and non-responders to nabilone.

Background: We designed this systematic review and meta-analysis to estimate a pooled odds ratio of developing MS by coffee consumption.

Methods: PubMed, Scopus, EMBASE, CINAHL, Web of Science, Ovid, and google scholar were searched by an expert researcher for articles published before January 2024. The gray literature including references from included studies, and conference abstracts was searched.

Results: The literature search revealed 521 articles; after deleting duplicates, 323 articles remained. Eight studies were included for meta-analysis. A total of 2193 MS cases and 2344 controls were evaluated. In cases, 1072 individuals were coffee users, while 1295 individuals in the control group were coffee consumers. The pooled odds ratio (OR) of coffee consumption and risk of MS was 0.78 (95%CI:0.69-0.88) (I² = 92.9%, p < 0.001).

Conclusion: Coffee consumption was associated with a lower risk of MS in pooled analysis, but the substantial heterogeneity limits the strength and generalizability of this conclusion. Larger, multi-centric studies are recomended.

Background: This study aimed to characterize lingual pressure (LP) and clarify its association with dysphagia in patients with amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA).

Methods: We examined 52 patients with ALS (Spinal-onset, 39; bulbar-onset, 13) and 36 patients with MSA (MSA-C, 26; MSA-P, 10). LP was measured using a balloon-type instrument during isometric contractions. Maximum LP (MLP) and 80% endurance of LP (ELP; duration above 80% of MLP during 7 seconds) were analyzed against videofluoroscopic findings of dysphagia.

Results: Both parameters significantly correlated with food intake scales, especially in ALS. Liquid aspiration was predicted in ALS with 80.8% accuracy (AUC = 0.794, p < 0.001) using MLP cutoff of 20.5 kPa, and in MSA with 75.0% accuracy (AUC = 0.786, p < 0.01) using an ELP cutoff of 1.68 second. Predictive accuracy improved in spinal-onset ALS and MSA-C.

Conclusion: LP thus may represent a physiological indicator for dysphagia detection and management.

Introduction: Motor Neurone Disease (MND) is a debilitating neurodegenerative condition affecting individuals, families, and carers. Evidence-informed care can improve outcomes, and guidelines play a key role in supporting this. A preliminary search showed limited guidelines exist, with none developed for the Australian context. This review aimed to identify existing health and social care guidelines for MND.

Methods: A scoping review was conducted using Joanna Briggs Institute (JBI) methodology. Guidelines addressing health and social care for people with MND, gene carriers, family members, or carers were included regardless of publication status. Five databases and additional sources were searched. Two reviewers independently screened citations, and data were extracted and analysed descriptively, with qualitative content analysis grouping guideline questions. Reporting followed PRISMA-ScR.

Results: Forty-two guidelines covering 133 questions were included. Most focused on symptom management for people with MND and were produced by professional bodies. Few used GRADE methodology or systematic reviews, and only seven assessed risk of bias. No guideline was developed for Australia.

Conclusions: A high-quality, evidence-based MND guideline following best practice development methods is needed.

This case report describes a 54-year-old Chinese woman with progressive gait instability, speech slowing, and cerebellar ataxia. Initial symptoms included mild bradykinesia and frequent backward falls. Neurological evaluation revealed supranuclear gaze palsy, hyporeflexia, and cerebellar dysfunction (SARA 26/40). Differential diagnoses included MSA, SCA, and PD. Genetic testing excluded SCA, while advanced imaging ruled out other disorders. The patient met criteria for probable PSP-C, a rare PSP subtype marked by cerebellar ataxia. [¹⁸F]PM-PBB3 tau PET imaging demonstrated abnormal tau accumulation in the striatum, thalamus, midbrain, and pons, consistent with PSP-C neuropathology. This case underscores the diagnostic utility of tau PET in distinguishing PSP-C from overlapping conditions and provides novel imaging evidence in an Asian cohort. Findings highlight the potential of noninvasive tau-targeted imaging for early detection and management of PSP-C.