Neurology and Therapy最新文献

{"title":"Safety, Tolerability and Pharmacokinetic-Pharmacodynamic Relationship of NX210c Peptide in Healthy Elderly Volunteers: Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Dose Study.","authors":"Annette Janus, Daniël Dumas, Juliette Le Douce, Sébastien Marie, Giuseppe Pasculli, Pauline Bambury, Sighild Lemarchant, Philip Kremer, Yann Godfrin","doi":"10.1007/s40120-024-00691-w","DOIUrl":"10.1007/s40120-024-00691-w","url":null,"abstract":"<p><strong>Introduction: </strong>Blood-brain barrier (BBB) integrity is fundamental to brain homeostasis, enabling control of substance exchange and safeguarding neurons against harmful toxins, pathogens, and immune cells that lead to dysregulation and inflammation involved in ageing and neurodegenerative diseases (NDD). The cyclized peptide NX210c is a thrombospondin type 1 repeat analogue derived from subcommissural organ-spondin. It exerts beneficial effects in animal models of NDD owing to its effects on neurons and endothelial cells. NX210c demonstrated a good safety profile in a single ascending dose phase 1a clinical study. The present multiple ascending dose phase 1b study was performed to evaluate the tolerability and pharmacological effects of repeated doses of NX210c in healthy elderly (age: > 55 years) volunteers.</p><p><strong>Methods: </strong>This was a randomized, placebo-controlled, double-blind study (EudraCT No. 2022-002868-76), investigating safety/tolerability, pharmacokinetics, and pharmacodynamics (including blood and cerebrospinal fluid biomarkers). Participants received 5 or 10 mg/kg NX210c or placebo (10-min infusion) thrice weekly for 4 weeks in an ascending dose fashion. Follow-up was conducted 2 weeks after last dosing.</p><p><strong>Results: </strong>The investigation included 29 participants. No serious adverse events were recorded and all adverse events were mild. Dedicated central nervous system testing did not reveal neurotoxicity. Biomarker evaluation showed a statistically significant reduction in blood claudin-5 and a trend toward reduction of blood homocysteine. In silico data modelling revealed salient pharmacokinetic-pharmacodynamic relationships, including reduction of claudin-5, neurofilament light chain, and SPARC-like protein 1 release, and degradation of homocysteine.</p><p><strong>Conclusion: </strong>Multiple doses of NX210c exhibited a good safety profile, showed non-cumulative pharmacokinetics, and exerted pharmacodynamic effects on biomarkers linked to BBB integrity. The effects of NX210c on claudin-5 and biomarkers influencing BBB integrity-and the overarching brain protection it offers-provide a novel therapeutic strategy targeting an underlying driver of neurodegenerative conditions for which disease-modifying treatments are limited or not available.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"357-377"},"PeriodicalIF":3.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11762061/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142872565","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sex Differences in Parkinson's Disease: A Narrative Review.","authors":"Carlo Cattaneo, Javier Pagonabarraga","doi":"10.1007/s40120-024-00687-6","DOIUrl":"10.1007/s40120-024-00687-6","url":null,"abstract":"<p><p>Sex differences in epidemiology, clinical features, and therapeutical responses are emerging in several movement disorders, even though they are still not widely recognized. Parkinson's disease (PD) is not an exception: men and women suffering from PD have different levels of disability. Research has been performed using multiple databases and scientific journals; this review summarizes the available evidence on sex differences in PD regarding epidemiology, risk factors, genetics, clinical phenotype, social impact, and therapeutic management. The role of hormones in determining such differences is also briefly discussed. The results confirm the existence of differences between men and women in PD; women have a higher risk of developing disabling motor complications and non-motor fluctuations compared to men, while men have a higher risk of developing cognitive impairment, postural instability, and gait disorders. Improving our knowledge in these differences may result in the implementation of strategies for disease-tailored treatment and management.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"57-70"},"PeriodicalIF":3.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11762054/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142770711","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Burden of Myasthenia Gravis in the Czech Republic: Analysis of the Nationwide Patient Registry.","authors":"Stanislav Voháňka, Aleš Tichopád, Magda Horáková, Jana Junkerová, Michala Jakubíková, Jiří Piťha, Michaela Týblová, Daniela Vlažná, Katarína Breciková, Jacek Cudny, Petr Hájek","doi":"10.1007/s40120-024-00682-x","DOIUrl":"10.1007/s40120-024-00682-x","url":null,"abstract":"<p><strong>Introduction: </strong>The main goal of this study was to describe the Czech population of patients with MG in terms of demographics, disease characteristics, management approaches, and treatment trends.</p><p><strong>Methods: </strong>We selected all patients, both incident and prevalent, who were enrolled in the Czech MyReg registry between August 24, 2015 and November 19, 2021. For the descriptive analysis, all patients enrolled in the registry, regardless of their date of diagnosis or date of enrolment, were included. We analyzed the following disease-related endpoints: myasthenia gravis composite (MGC) score, forced vital capacity (FVC), and Myasthenia Gravis Foundation of America (MGFA) clinical classification.</p><p><strong>Results: </strong>The incidence showed a consistent increasing trend from 0.62 to 3.13. The mean MGC score was 5.0 (median 4.0, 95% CI 4.7, 5.3) representing mild form of MG. The difference in FVC from the predicted value in patients during and without myasthenic crisis was 58.93% (95% CI 37.27, 80.59) and 75.93% (95% CI 74.87, 77.00), respectively. We identified 70 patients (5.0%) with refractory MG, of whom 58.6% were female. The MGFA classifications in those with refractory vs. non-refractory disease was as follows: IIa 21.8% vs 23.2%, IIb 45.3% vs 33.6%, and IIIb 14.1% vs 4.6%, respectively.</p><p><strong>Conclusion: </strong>Our analysis shows that the incidence of MG is increasing in the Czech Republic and that patients with refractory disease, of whom up to 58% are female, have a higher burden of disease than non-refractory patients.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"227-242"},"PeriodicalIF":3.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11762035/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142770710","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Incidence and Prevalence of Alzheimer's Disease in Medicare Beneficiaries.","authors":"Haixin Zhang, Amir Abbas Tahami Monfared, Quanwu Zhang, Lawrence S Honig","doi":"10.1007/s40120-024-00695-6","DOIUrl":"10.1007/s40120-024-00695-6","url":null,"abstract":"<p><strong>Introduction: </strong>The availability of anti-amyloid therapy for mild cognitive impairment (MCI) due to Alzheimer's disease and mild Alzheimer's dementia (AD) has underscored the need for realistic estimates of the population with AD/MCI within the healthcare system to assure adequate preparedness. We hypothesize that administrative databases can provide real-world epidemiologic estimates reflecting the population with diagnosed (known) MCI and AD. This study was conducted to estimate diagnostic incidence and prevalence of AD and all-cause MCI among the Medicare fee-for-service (FFS) and Medicare Advantage (MA) beneficiaries in the United States.</p><p><strong>Methods: </strong>This was a retrospective analysis of Medicare beneficiaries (aged 65 and older) with identified diagnoses of AD/MCI based on ≥ 2 diagnostic codes ≥ 30 days apart. Incidence/prevalence estimates were reported per 10,000 person-years.</p><p><strong>Results: </strong>In FFS, AD incidence (2008-2018) decreased (138 to 104); MCI incidence increased (8 to 47), but the sum (MCI + AD) was relatively stable (146 to 151). Prevalence (2008-2017) increased for AD (318 to 354), and MCI (13 to 99). In MA (2016) epidemiological estimates were consistent with FFS. In 2017, older age, female sex and the Northeastern region were consistently associated with higher AD/MCI prevalence among FFS beneficiaries.</p><p><strong>Conclusion: </strong>In FFS, AD/MCI diagnostic prevalence increased over 10 years, especially for MCI; prevalence estimates in MA (2016) were comparable. Diagnostic prevalence in 2016 (FFS + MA) was 3.4% for AD and 0.85% for MCI. Our findings address the reality of Alzheimer's disease in clinical practice in the United States that is confronted by healthcare professionals, payors, healthcare decision-makers, patients, and caregivers, and may offer a realistic gauge for patient triage for treatment, healthcare resource allocation, and health-systems' operational prioritization. With the availability of anti-amyloid treatments, we anticipate that the population with diagnosed MCI/AD within the Medicare database may rise over time; therefore, periodic updates of incidence/prevalence estimates may provide support for timely healthcare decision-making.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"319-333"},"PeriodicalIF":3.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11762046/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854850","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Study of Limosilactobacillus reuteri for the Treatment of Children with Chronic Tic Disorders/Tourette Syndrome: A Mid-Term Efficacy Evaluation.","authors":"Yan Liang, Lin Wan, Guanglei Wang, Huimin Yan, Jing Zhang, Xinting Liu, Ziyan Zhang, Gang Zhu, Guang Yang","doi":"10.1007/s40120-024-00693-8","DOIUrl":"10.1007/s40120-024-00693-8","url":null,"abstract":"<p><strong>Introduction: </strong>Gut microbiota plays an important role in tic disorders (TDs); however, clinical research on probiotics for chronic TDs treatment is lacking. We aimed to investigate the effectiveness of probiotics, hypothesizing that their clinical efficacy is comparable to that of clonidine in treating chronic TDs.</p><p><strong>Methods: </strong>Patients were randomly assigned to receive either Limosilactobacillus reuteri or clonidine transdermal patch treatment for 8 weeks while maintaining their existing treatment. The Yale Global Tic Severity Scale (YGTSS); Swanson, Nolan, and Pelham-IV Scale (SNAP-IV); and Child Behavior Check List (CBCL) scores were assessed before and after treatment.</p><p><strong>Results: </strong>We matched the patients in both groups for age, sex, age at onset, and tic type. A significant improvement in YGTSS scores was observed in both groups (p = 0.024). The improvement in attention deficits on the SNAP-IV scale was similar between the two groups, with no significant difference (p = 0.465). For hyperactivity disorder, after matching patients in both groups for age, sex, age at onset, tic type, and Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) scores, a significant difference in improvement was observed between the groups (p = 0.010), with the probiotics group showing greater improvement (0.3 ± 0.58 vs. 0.1 ± 0.50). At 9 weeks, social ability on the CBCL scale increased by 3.2 ± 6.26 from baseline in the probiotics group and by 0.6 ± 4.07 in the clonidine group, with a significant difference between the two (p = 0.049). Although there was no significant difference in behavioral problems between the two groups (p = 0.347), the trend of improvement was more pronounced in the probiotics group than in the clonidine group (12.7 ± 25.86 vs. 8.4 ± 13.15).</p><p><strong>Conclusion: </strong>The mid-term efficacy evaluation demonstrated that L. reuteri, when added to the treatment of children with chronic TDs, was more effective in improving tic symptoms than clonidine transdermal patch treatment. Additionally, it provided moderate improvement in hyperactivity symptoms.</p><p><strong>Trial registration: </strong>chictr.org.cn (registration numbers ChiCTR2200056708, ChiCTR2200056578).</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"279-290"},"PeriodicalIF":3.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11762037/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854844","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Characteristics and Outcomes in Patients with Ruptured Middle Cerebral Artery Aneurysms: A Multicenter Study in Northern China.","authors":"Xiuhu An, Jingliang Su, Bingxin Duan, Long Zhao, Bangyue Wang, Yan Zhao, Tianxing Li, Shuai Zhou, Xinyu Yang, Zhenbo Liu","doi":"10.1007/s40120-024-00673-y","DOIUrl":"10.1007/s40120-024-00673-y","url":null,"abstract":"<p><strong>Introduction: </strong>The long-term prognosis of ruptured middle cerebral artery aneurysms (MCAAs) in northern China remains unclear. The aim of this study is to analyze the epidemiological characteristics and long-term outcomes of ruptured MCAAs in northern China.</p><p><strong>Methods: </strong>We included patients who were consecutively admitted for ruptured MCAAs to 12 tertiary care centers in northern China from January 2017 to December 2020. Kaplan‒Meier curves were used to compare survival in hazard strata. The Cox proportional hazards model was used to analyze risk factors and mortality risk, whereas logistic regression was used to identify factors influencing 2-year survival. Subgroup analyses were performed to verify the robustness of the results.</p><p><strong>Results: </strong>Data on 959 patients with ruptured MCAAs were analyzed; 16.4% of these patients had ruptured intracranial aneurysms (RIAs) and were registered in the Chinese cerebral aneurysm database. The mean follow-up duration was 3.0 years (range 0-6.2 years). The 3-month and 2-year mortality rates were 15.5% and 18.2%, respectively. The risk factors for mortality were identified via Cox regression and were as follows: age > 70 years, previous stroke, combined intracerebral hemorrhage (ICH)/intraventricular hemorrhage (IVH), poor Hunt and Hess grade, multiple aneurysms, and conservative treatment (CT). The positive association between the risk of death and CT was consistent across subgroups. According to logistic regression, hypertension, previous stroke, combined ICH/IVH, Hunt and Hess grade, and WFNS (World Federation of Neurological Surgeons) score were identified as factors negatively influencing 2-year survival.</p><p><strong>Conclusion: </strong>We detail the epidemiologic characteristics and long-term outcomes of MCAAs. The risk factors for mortality included age > 70 years, previous stroke, combined ICH/IVH, poor Hunt and Hess grade, and multiple aneurysms. Compared with microsurgical treatment (MST), CT is associated with an increased risk of mortality, while the risk of mortality associated with endovascular treatment (EVT) is not significantly different. Two-year survival was associated with hypertension, previous stroke, ICH/IVH, and poor grades at admission.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"119-133"},"PeriodicalIF":3.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11762049/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142562484","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment Patterns and Healthcare Costs Among Patients with Stroke and Spasticity: A 2-Year Longitudinal Study.","authors":"Michael Hull, Vamshi Ruthwik Anupindi, Jing He, Mitchell DeKoven, Jumaah Goldberg, Jonathan Bouchard","doi":"10.1007/s40120-024-00692-9","DOIUrl":"10.1007/s40120-024-00692-9","url":null,"abstract":"<p><strong>Introduction: </strong>Post-stroke spasticity (PSS) occurs in ~25-43% of patients between 2 weeks and 3 months following a stroke. This retrospective claims study examined the occurrence of spasticity, treatment patterns, healthcare resource utilization, and healthcare costs among patients who experienced a stroke over a 2-year period.</p><p><strong>Methods: </strong>Analyses were conducted using healthcare claims from the IQVIA PharMetrics Plus database of commercially/self-insured members from 2015 to 2021. Patients were selected based on two International Classification of Diseases, 10th revision diagnosis codes for stroke requiring an inpatient stay (index date) with continuous enrollment with medical/pharmacy benefits 12 months before (pre-index) and 24 months starting on the index date (post-index). PSS was defined by a diagnosis code for spastic hemiplegia or muscle contracture starting ≥ 7 days post-index, or claims indicating PSS treatment [botulinum toxin A (BoNT-A) or muscle relaxants] any time in the post-index period. A generalized linear model was developed to estimate cost ratios between patients with and without PSS.</p><p><strong>Results: </strong>Overall, 7851 patients fulfilled study criteria; 47.7% were treated with physical or occupational therapy, 11.3% with muscle relaxants, and 0.8% with BoNT-A; 12.4% met the post-index definition of PSS; 84.2% were identified using muscle relaxant or BoNT-A codes, 6.6% using diagnosis codes, and 9.2% using both. Median time to codes identifying PSS was 213 days. Patients treated with BoNT-A received an average of three treatments, starting 253 days (median) post-stroke. Mean all-cause healthcare costs were US$62,875 among patients with PSS versus $44,472 among patients without (P < 0.001), representing 39.6% higher adjusted all-cause healthcare costs among patients with PSS versus patients without PSS.</p><p><strong>Conclusion: </strong>Patients with PSS utilized numerous treatment modalities and experienced higher mean all-cause healthcare costs than did those without PSS. Earlier identification to optimize treatment of PSS may represent an opportunity for cost savings within managed healthcare systems.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"261-278"},"PeriodicalIF":3.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11762044/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142838492","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain.","authors":"Juan José García-Peñas, Rocío Calvo-Medina, Adrián García-Ron, Antonio Gil-Nagel, Vicente Villanueva, Rocío Sánchez-Carpintero","doi":"10.1007/s40120-024-00677-8","DOIUrl":"10.1007/s40120-024-00677-8","url":null,"abstract":"<p><strong>Background: </strong>Despite considerable evidence for the efficacy and safety of stiripentol in Dravet syndrome (DS), some aspects of stiripentol use remain challenging in clinical practice, such as dose titration and the adjustment of concomitant antiseizure medications (ASMs) to prevent potential adverse effects.</p><p><strong>Aim: </strong>To (1) provide practical recommendations on the initiation of stiripentol treatment in patients with DS, (2) evaluate its effectiveness in the patient, and (3) guide the management of drug interactions and other aspects of treatment monitoring.</p><p><strong>Methods: </strong>Six Spanish neurologists (the authors) with expertise in the management of pediatric and adult patients with DS held a meeting in early 2024 to develop expert recommendations regarding the use of stiripentol in DS, based on a review of the literature and their common clinical experience.</p><p><strong>Results: </strong>According to these recommendations, stiripentol can be administered to patients with DS of any age, although its initiation and titration vary according to age group. Individualized adjustment of concomitant ASMs, such as valproic acid and clobazam or drugs specifically for DS (i.e., fenfluramine), at initiation and during stiripentol treatment, can mitigate drug interactions, thereby increasing the long-term tolerability of stiripentol treatment. In specific cases, stiripentol doses of > 50 mg/kg/day may be contemplated, and acute stiripentol administration may be considered to control refractory status epilepticus. Blood tests should be performed before starting stiripentol, at 3, 6, and 12 months after starting treatment, and then annually, except in the event of adverse effects, when additional testing may be necessary. Most adverse effects can be adequately managed by adjusting concomitant medications.</p><p><strong>Conclusion: </strong>These practical recommendations may be easily adapted for use in different countries, and should increase physicians' confidence in the initiation and monitoring of stiripentol treatment, thus facilitating effective management of patients with DS and improving clinical outcomes.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"27-43"},"PeriodicalIF":3.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11762041/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142568401","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Barriers in Healthcare to the Use of Optical Coherence Tomography Angiography in Multiple Sclerosis.","authors":"Lukas G Reeß, Hadi Salih, Murat Delikaya, Friedemann Paul, Frederike Cosima Oertel","doi":"10.1007/s40120-024-00670-1","DOIUrl":"10.1007/s40120-024-00670-1","url":null,"abstract":"<p><p>Optical coherence tomography angiography (OCT-A) is a state-of-the-art imaging technique for the retinal vasculature to accurately segment the capillary network and assign it to retinal layers. OCT-A is a promising technique to better understand neurological diseases with visual system manifestations, such as multiple sclerosis (MS), and to identify and characterize vascular biomarkers. Initial studies suggested vascular changes in MS and its differential diagnoses such as myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) and neuromyelitis optica spectrum disorder (NMOSD). Here we review clinical and technical aspects of OCT-A imaging and discuss the potential for the MS field as well as barriers that need to be overcome before OCT-A can be established in clinical application.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"45-56"},"PeriodicalIF":3.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11762043/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142582037","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Health-Related Quality of Life in Patients with Friedreich Ataxia Using Mobility Assistive Technologies: Limited Fit of the EQ-5D-3L Mobility Dimension.","authors":"Maresa Buchholz, Michelle Pfaff, Audrey Iskandar, Kathrin Reetz, Jörg B Schulz, Marcus Grobe-Einsler, Thomas Klockgether, Bernhard Michalowsky","doi":"10.1007/s40120-024-00694-7","DOIUrl":"10.1007/s40120-024-00694-7","url":null,"abstract":"<p><strong>Introduction: </strong>Friedreich Ataxia (FA) is a multisystem neurodegenerative disease. Affected individuals rely on mobility assistive technologies (MAT) (e.g. wheelchairs) and require long-term treatments and care. To analyse the patients' health-related quality of life (HRQoL), the EuroQol 5 Dimension 3 Level survey (EQ-5D-3L)-a widely used and recommended generic measure-is used in clinical and in health economic studies. Concerns about using the instrument in mobility-impaired individuals who might have difficulties finding appropriate response options for mobility-related items led us to investigate how the 3L dimensions perform in patients with FA using or not using MAT.</p><p><strong>Methods: </strong>Using longitudinal data from 607 patients with FA of the EFACTS study (from baseline to the 3-year follow-up), we analysed the acceptability, distribution properties, validity, and responsiveness of the EQ-5D-3L, focusing on the mobility item. Analyses were stratified for patients without and with different MAT-usage.</p><p><strong>Results: </strong>We identified that n = 177 patients used no MAT, n = 299 a wheelchair and n = 131 walking aids. The mobility item non-response was highest in wheelchair users (6.8%) and lowest in patients without MAT. Walking aid users showed the least variability, all selecting the mid-response option \"some problems\" for mobility. The mobility item correlated moderately with disease severity (r_sp = 0.35) and the activities of daily living scale (r_sp = 0.36) in wheelchair users. No correlation exists for walking aid users. The strongest health changes occurred for wheelchair users, the weakest for walking aid users. The mobility dimensions showed the highest amount of no changes.</p><p><strong>Conclusion: </strong>The EQ-5D-3L's mobility item has limitations in MAT users, particularly in walking aid users, due to a tendency towards mid-responses. These limitations may affect the efficacy and (cost)effectiveness conclusions drawn from interventions and clinical trials with mobility-impaired individuals. Further research is needed to explore the understanding and interpretation of responses of the EQ-5D in patients with FA with mobility support.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier NCT02069509.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"379-398"},"PeriodicalIF":3.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11762039/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142909885","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}