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Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in Spain, Greece, and Portugal. 西班牙、希腊和葡萄牙新生儿脊髓性肌萎缩症筛查的成本效益。
IF 4.7 3区 医学
Neurology and Therapy Pub Date : 2026-09-21 DOI: 10.1007/s40120-026-01032-9
Raquel Yahyaoui, Argirios Dinopoulos, Francina Munell, José Pedro Vieira, Manuela A Santos, David Gómez-Andrés, Grace McCarthy, Matthias Bischof, Michel A Kroes, Dimitrios I Zafeiriou
{"title":"Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in Spain, Greece, and Portugal.","authors":"Raquel Yahyaoui, Argirios Dinopoulos, Francina Munell, José Pedro Vieira, Manuela A Santos, David Gómez-Andrés, Grace McCarthy, Matthias Bischof, Michel A Kroes, Dimitrios I Zafeiriou","doi":"10.1007/s40120-026-01032-9","DOIUrl":"https://doi.org/10.1007/s40120-026-01032-9","url":null,"abstract":"<p><strong>Introduction: </strong>Patients with spinal muscular atrophy (SMA) may receive early treatment, ideally before symptoms present, if diagnosed through newborn screening (NBS). We aimed to evaluate the cost-effectiveness of including SMA in NBS programs in Spain, Greece, and Portugal.</p><p><strong>Methods: </strong>A cost-utility model, incorporating both a decision tree and Markov model, compared costs and health outcomes between two scenarios: including SMA in the NBS program with subsequent treatment compared with no NBS and treatment at symptom onset. A base-case analysis was conducted based on payers' perspective over a lifetime time horizon, with an annual discount rate of 3% for Spain, 3.5% for Greece, and 4% for Portugal applied to both costs and health effects. Model inputs were based on existing literature, local data, and expert panel opinion. Deterministic, probabilistic, and scenario sensitivity analyses were conducted to assess model robustness.</p><p><strong>Results: </strong>Including SMA in NBS programs for Spain, Greece, and Portugal would allow early identification and treatment initiation for 97% (n = 33), 89% (n = 8), and 86% (n = 6) of patients per year in Spain, Greece, and Portugal, respectively. In the base-case analysis (lifetime time horizon), NBS for SMA resulted in significant cost savings for Spain (€32,327,624), Greece (€1,778,695), and Portugal (€4,333,014) compared with a scenario with no NBS. NBS for SMA resulted in a total of 837, 199, and 135 quality-adjusted life-years (QALYs) compared with 433, 131, and 89 QALYs without NBS, leading to a gain of 404, 68, and 46 QALYs in Spain, Greece, and Portugal, respectively. Sensitivity analyses were consistent with base-case outcomes, demonstrating the robustness of base-case findings.</p><p><strong>Conclusion: </strong>In Spain, Greece, and Portugal, implementation of NBS for early identification and treatment of patients with SMA is a cost-effective strategy for improving health outcomes and reducing total costs versus clinical diagnosis and later treatment of patients at symptom onset.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149017398","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Benzodiazepines in Epilepsy: A US Perspective on Clinical Use, Pharmacology, Abuse Methods, and Public Health Considerations. 苯二氮卓类药物治疗癫痫:美国的临床应用、药理学、滥用方法和公共卫生考虑。
IF 4.7 3区 医学
Neurology and Therapy Pub Date : 2026-09-17 DOI: 10.1007/s40120-026-01007-w
Enrique Carrazana, Joseph Sirven, John Stern, Barry Gidal, Andres M Kanner, Adrian L Rabinowicz
{"title":"Benzodiazepines in Epilepsy: A US Perspective on Clinical Use, Pharmacology, Abuse Methods, and Public Health Considerations.","authors":"Enrique Carrazana, Joseph Sirven, John Stern, Barry Gidal, Andres M Kanner, Adrian L Rabinowicz","doi":"10.1007/s40120-026-01007-w","DOIUrl":"https://doi.org/10.1007/s40120-026-01007-w","url":null,"abstract":"<p><p>Benzodiazepines (BZDs) are indicated for a range of therapeutic conditions and can improve patient outcomes when used appropriately. The role of BZDs in epilepsy management is essential in that they have long been first-line agents for acute seizure cessation, most notably including status epilepticus, with each BZD offering a unique pharmacokinetic profile. BZDs are commonly used to treat seizures because of their rapid onset, pronounced safety and tolerability profiles, and versatility of dosage forms and available routes of administration. Despite their clinical utility in a variety of therapeutic areas for a wide range of conditions, the potential for BZD misuse and abuse remains a topic of public health concern. Particularly in combination with other central nervous system depressants (e.g., opioids, gabapentin, and alcohol), misuse and abuse of BZDs are linked to serious and potentially fatal complications. As such, the US Food and Drug Administration has issued a boxed warning regarding concurrent use of BZDs and opioids, emphasizing the dangers of co-prescribing. Policies and prescribing safeguards are implemented to regulate and promote safe use of BZDs; however, such measures may unintentionally worsen patient care by limiting access for individuals that benefit from therapy for seizure control. The objective of this paper is to identify a practical balance between ensuring access to these essential medications for those who experience clinical benefit while also supporting safe prescribing of BZDs to minimize misuse and abuse. Findings from the present review support continued research, drug utilization policies, and patient education.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148990485","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evaluation of Autonomic and Cognitive Functions in Patients with Essential Tremor. 特发性震颤患者自主神经和认知功能的评价。
IF 4.7 3区 医学
Neurology and Therapy Pub Date : 2026-09-15 DOI: 10.1007/s40120-026-01035-6
Eda Özsezen, Nevin Pazarci
{"title":"Evaluation of Autonomic and Cognitive Functions in Patients with Essential Tremor.","authors":"Eda Özsezen, Nevin Pazarci","doi":"10.1007/s40120-026-01035-6","DOIUrl":"https://doi.org/10.1007/s40120-026-01035-6","url":null,"abstract":"<p><strong>Introduction: </strong>This study aimed to evaluate autonomic and cognitive functions in patients with essential tremor (ET) and investigate their correlation.</p><p><strong>Methods: </strong>A total of 38 patients with ET and 35 healthy volunteers were included in the study. Electrophysiological assessment of the autonomic nervous system was performed using the sympathetic skin response (SSR) and RR interval variation analyses. Additionally, cognitive function was assessed using the Montreal Cognitive Assessment (MoCA) for all participants.</p><p><strong>Results: </strong>In the patient group, compared with the control group, median and tibial nerve SSR latencies were greater, and amplitudes were lower in both the upper and lower extremities. SSR latencies were prolonged in several upper- and lower-extremity recordings, and the right tibial SSR amplitude was significantly reduced (p < 0.05); the remaining amplitude differences showed a similar trend without reaching statistical significance. Although RR interval variation was lower in patients than in controls during both rest and deep breathing, no statistically significant difference was detected. MoCA scores were significantly lower in patients than in healthy volunteers. Subgroup analysis revealed that this difference was particularly prominent in visual-spatial/executive functions and language function. No correlation was detected between the SSR, RR interval variation, or MoCA in either the patient or control group.</p><p><strong>Conclusion: </strong>Patients with ET demonstrated electrophysiological findings suggestive of altered sympathetic autonomic function, together with lower cognitive performance. However, parasympathetic dysfunction was not demonstrated by analysis of RR interval variation.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148976801","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Treatment Preferences of Physicians of Pediatric Patients with Attention-Deficit/Hyperactivity Disorder in the United States and Canada: A Discrete Choice Experiment. 美国和加拿大儿童注意缺陷/多动障碍医生的治疗偏好:一个离散选择实验
IF 4.7 3区 医学
Neurology and Therapy Pub Date : 2026-09-15 DOI: 10.1007/s40120-026-01033-8
Jeff Schein, Marjolaine Gauthier-Loiselle, Yan Meng, Maryaline Catillon, Beatrice Libchaber, Francesca Lee, Martin Cloutier, Ann Childress
{"title":"Treatment Preferences of Physicians of Pediatric Patients with Attention-Deficit/Hyperactivity Disorder in the United States and Canada: A Discrete Choice Experiment.","authors":"Jeff Schein, Marjolaine Gauthier-Loiselle, Yan Meng, Maryaline Catillon, Beatrice Libchaber, Francesca Lee, Martin Cloutier, Ann Childress","doi":"10.1007/s40120-026-01033-8","DOIUrl":"https://doi.org/10.1007/s40120-026-01033-8","url":null,"abstract":"<p><strong>Introduction: </strong>Evidence on how treatment attributes, especially adverse events, influence preferences of physicians treating pediatric patients with attention-deficit/hyperactivity disorder (ADHD) is scant. This discrete choice experiment aimed to address this evidence gap.</p><p><strong>Methods: </strong>A web-based survey was completed by physicians treating pediatric patients with ADHD in the United States (US) and Canada recruited from a market research firm's panel (12/2024-01/2025). Treatment attributes evaluated included efficacy (improvement in ADHD symptoms) and safety (irritability, upper abdominal pain, insomnia, somnolence, decreased appetite, vomiting), identified from clinical inputs and published data. Physicians' preferences and willingness to trade-off were estimated using conditional logistic regression. Attributes' relative importance was assessed. Subgroups were stratified by physician subspecialty and by patients' age group.</p><p><strong>Results: </strong>The sample included 404 physicians in the US and 402 in Canada (50.2% and 49.8% psychiatrists, respectively). US physicians were on average willing to trade 0.42, 0.36, 0.34, 0.32, and 0.22 percentage points of improvement in ADHD symptoms to achieve a 1-percentage-point reduction in the risk of irritability, upper abdominal pain, insomnia, somnolence, and decreased appetite, respectively, with similar ranges for Canadian physicians (0.22-0.38). In the US, the relative importance of efficacy and that of all safety-related attributes assessed was both 50.0%, whereas, in Canada, this was 53.7% and 46.3%, respectively. In both countries, the most important safety-related attributes were the risks of insomnia (US: 14.5%; Canada: 12.5%), somnolence (US: 13.7%; Canada: 11.0%), and decreased appetite (US: 10.6%; Canada: 11.4%). Psychiatrists placed greater relative importance on efficacy compared to primary care physicians/pediatricians. Findings were consistent when physicians considered treating children and adolescents.</p><p><strong>Conclusion: </strong>US and Canadian physicians valued efficacy and safety similarly when considering pediatric ADHD treatments, with some differences by subspecialty. Understanding drivers of physician preferences and how they may diverge from that of parents/caregivers may facilitate informed discussions and effective shared decision-making.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148977420","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Update on Parkinson's Disease Psychosis: Epidemiology, Mechanisms and Management. 帕金森氏病精神病最新进展:流行病学、机制和管理。
IF 4.7 3区 医学
Neurology and Therapy Pub Date : 2026-09-09 DOI: 10.1007/s40120-026-01014-x
Caroline Kruse, Daniel Weintraub, John Duda, James F Morley
{"title":"Update on Parkinson's Disease Psychosis: Epidemiology, Mechanisms and Management.","authors":"Caroline Kruse, Daniel Weintraub, John Duda, James F Morley","doi":"10.1007/s40120-026-01014-x","DOIUrl":"https://doi.org/10.1007/s40120-026-01014-x","url":null,"abstract":"<p><p>Psychosis in Parkinson's disease (PD) is a common and debilitating non-motor symptom that can occur at any stage of the disease. PD psychosis (PDP) manifests as a diverse clinical spectrum that can include misperceptions, passage and presence phenomena, hallucinations, and delusions, alone or in combination. This review explores the epidemiology and underlying neurobiological mechanisms of psychosis in PD, describes the symptom spectrum experienced by patients, and reviews the evidence supporting current and future therapies for psychosis in PD. Pimavanserin and clozapine are the only pharmacologic therapies for PDP that have been found to be efficacious in randomized controlled trials, though quetiapine is most frequently used despite lacking positive trial results. Future research that continues to clarify the exact pathophysiology of psychosis in PD will improve the current therapeutic approaches, and ongoing randomized clinical trials of these therapies are necessary to identify the safest and most efficacious treatments for PD patients with psychosis.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148920389","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Effectiveness of Rimegepant for the Acute Treatment of Migraine Among Participants Using Different Types of Preventive Therapy: Analyses from CONFIDENCE. 利美吉坦对不同类型预防治疗参与者急性偏头痛的有效性:来自CONFIDENCE的分析。
IF 4.7 3区 医学
Neurology and Therapy Pub Date : 2026-09-09 DOI: 10.1007/s40120-026-01024-9
Richard B Lipton, Peter J Goadsby, Patricia Pozo-Rosich, Alexandre Urani, Giorgio Lambru, Todd J Schwedt, Kristina M Fanning, Ryan C Bostic, Feng Dai, Lucy Abraham, Karin Hygge Blakeman
{"title":"Effectiveness of Rimegepant for the Acute Treatment of Migraine Among Participants Using Different Types of Preventive Therapy: Analyses from CONFIDENCE.","authors":"Richard B Lipton, Peter J Goadsby, Patricia Pozo-Rosich, Alexandre Urani, Giorgio Lambru, Todd J Schwedt, Kristina M Fanning, Ryan C Bostic, Feng Dai, Lucy Abraham, Karin Hygge Blakeman","doi":"10.1007/s40120-026-01024-9","DOIUrl":"https://doi.org/10.1007/s40120-026-01024-9","url":null,"abstract":"<p><strong>Introduction: </strong>This analysis assessed the real-world effectiveness of rimegepant for the acute treatment of migraine in patients taking different types of preventive medication.</p><p><strong>Methods: </strong>The prospective, observational, CONFIDENCE study recruited participants in the United States with 3-14 headache days/30 days and a rimegepant prescription for the acute treatment of migraine via the Migraine Buddy<sup>®</sup> app. Over a 28-day period, participants reported daily on the occurrence, treatment, and outcomes of migraine attacks. The proportion of rimegepant-treated attacks that achieved meaningful pain relief, meaningful improvement in function, or treatment satisfaction were calculated by preventive medication type [none; oral preventive medication (OPM) only; onabotulinumtoxinA (onaBoNT-A) with/without OPM; or calcitonin gene-related peptide monoclonal antibody (CGRP mAb) with/without OPM]. Pain and functional outcomes were compared between attacks treated with rimegepant versus other acute medication combinations using adjusted generalized linear mixed models with logit link and random intercept for each participant.</p><p><strong>Results: </strong>Among 2169 rimegepant-treated attacks, meaningful pain relief was achieved at 2 h and 4 h in 61.3% and 81.8%, respectively; corresponding values for meaningful improvement in function were 57.6% and 78.5%. Odds for all outcomes were higher with rimegepant versus other acute medication treatment combinations. Satisfaction with rimegepant treatment of the attack was > 60% across domains related to speed and effectiveness. Rates and adjusted odds ratios [aORs (95% CI)] for meaningful pain relief within 2 h with rimegepant versus other acute medication treatment combinations were: all attacks 61.3% versus 49.6%, aOR = 1.93 (1.52, 2.45), P < 0.001; no preventive medication 70.0% versus 41.8%, aOR = 4.56 (2.09, 9.92), P < 0.001; OPM 64.7% versus 56.5%, aOR = 1.76 (1.06, 2.90), P < 0.05; onaBoNT-A 62.0% versus 49.5%, aOR = 1.89 (1.24, 2.87), P < 0.01; and CGRP mAb 54.4% versus 46.8%, aOR = 1.66 (1.09, 2.53), P < 0.05.</p><p><strong>Conclusion: </strong>High rates of meaningful pain relief, meaningful improvement in function, and treatment satisfaction were observed with rimegepant for the acute treatment of migraine when used alongside different types of preventive medication in the real world.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov, NCT06467370. Graphical abstract available for this article.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148920380","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cenobamate Improves Emotional Dysregulation, Impulsivity, and Apathy in Drug-Resistant Focal Epilepsy: A Longitudinal Neuropsychological Study. Cenobamate改善耐药局灶性癫痫患者的情绪失调、冲动和冷漠:一项纵向神经心理学研究。
IF 4.7 3区 医学
Neurology and Therapy Pub Date : 2026-09-08 DOI: 10.1007/s40120-026-01022-x
Ilaria Sammarra, Adriana Saraceno, Iolanda Martino, Alessia Vono, Francesca Felicia Operto, Francesco Fortunato, Antonio Gambardella
{"title":"Cenobamate Improves Emotional Dysregulation, Impulsivity, and Apathy in Drug-Resistant Focal Epilepsy: A Longitudinal Neuropsychological Study.","authors":"Ilaria Sammarra, Adriana Saraceno, Iolanda Martino, Alessia Vono, Francesca Felicia Operto, Francesco Fortunato, Antonio Gambardella","doi":"10.1007/s40120-026-01022-x","DOIUrl":"https://doi.org/10.1007/s40120-026-01022-x","url":null,"abstract":"<p><strong>Introduction: </strong>Although effects on cognitive function have been reported during cenobamate (CNB) treatment, far less is known about how it may affect neuropsychological domains such as apathy, emotion dysregulation, and impulsivity. This prospective longitudinal study aims to assess psychopathological and cognitive changes after CNB introduction in adults with drug-resistant focal epilepsy, accounting for seizure outcome and burden of concomitant anti-seizure medication (ASM).</p><p><strong>Methods: </strong>We consecutively recruited adults with drug-resistant focal epilepsy who had undergone a comprehensive neuropsychological evaluation at baseline, before starting CNB, and at follow-up, 12 months after treatment introduction. The psychopathological battery included Beck Depression Inventory-II (BDI-II), State-Trait Anxiety Inventory, Difficulties in Emotion Regulation Scale (DERS-36), Barratt Impulsiveness Scale (BIS-11), Apathy Evaluation Scale (AES), and Quality of Life in Epilepsy Inventory (QoLIE-31). The cognitive assessment comprised Rey Auditory Verbal Learning Test (RAVLT), Digit-Span, Rey-Osterrieth Complex Figure Test, WEIGL, FAS, Stroop Color and Word Test, and Symbol Digit Modalities Test (SDMT). We applied linear mixed-effects models adjusted for responder status and total concomitant ASM defined daily dose (DDD) ratio.</p><p><strong>Results: </strong>Our cohort included 27 individuals (16 female; mean age 42.4 ± 16.7 years) showing a 74% responder rate with 30% achieved seizure freedom at follow-up. CNB treatment was associated with favorable changes in BDI-II (q = 0.009), DERS-36 (q = 2.36 × 10<sup>-5</sup>), BIS-11 (q = 6.30 × 10<sup>-8</sup>), AES (q = 4.87 × 10<sup>-5</sup>), and QoLIE-31 (q = 0.009). RAVLT-Immediate performance likewise improved (q = 0.004), whereas SDMT worsened over time (q = 0.002); the remaining domains were unchanged. These effects remained significant after adjustment for responder status and total concomitant ASM DDD ratio: BDI-II [β (95% CI) = - 0.582 (- 0.943, - 0.221)], DERS-36 [β (95% CI) = - 0.550 (- 0.739, - 0.361)], BIS-11 [β (95% CI) = - 0.932 (- 1.144, - 0.719)], AES [β (95% CI) = - 1.001 (- 1.330, - 0.673)], QoLIE-31 [β (95% CI) = 0.488 (0.185, 0.791)], RAVLT-Immediate performance [β (95% CI) = 0.446 (0.255, 0.636)], and SDMT [β (95% CI) = - 0.294 (- 0.425, - 0.163)].</p><p><strong>Conclusion: </strong>Adjunctive CNB was correlated with psychopathological improvements alongside domain-specific cognitive changes, independent of seizure outcome and concomitant ASM burden. These findings further expand current knowledge on CNB impact and tolerability profile.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148912485","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Qualitative Study of Lived Treatment Experiences and Preferences of Long-Term DMT Users with Relapsing-Remitting Multiple Sclerosis. 复发-缓解型多发性硬化症长期DMT使用者生活治疗经历和偏好的定性研究。
IF 4.7 3区 医学
Neurology and Therapy Pub Date : 2026-08-01 Epub Date: 2026-06-05 DOI: 10.1007/s40120-026-00959-3
Clair-Antoine Veyrier, Patrick Vermersch, Olivier Casez, Olivier Heinzlef, Marianne Payet, Laura Luciani, Nicolas Collongues, Martin Duracinsky
{"title":"A Qualitative Study of Lived Treatment Experiences and Preferences of Long-Term DMT Users with Relapsing-Remitting Multiple Sclerosis.","authors":"Clair-Antoine Veyrier, Patrick Vermersch, Olivier Casez, Olivier Heinzlef, Marianne Payet, Laura Luciani, Nicolas Collongues, Martin Duracinsky","doi":"10.1007/s40120-026-00959-3","DOIUrl":"10.1007/s40120-026-00959-3","url":null,"abstract":"<p><strong>Introduction: </strong>Relapsing-remitting multiple sclerosis (RRMS) requires prolonged use of disease-modifying therapies (DMTs), often perceived as burdensome. This study explored the lived treatment experiences of long-term DMT users with RRMS.</p><p><strong>Methods: </strong>This qualitative, non-interventional study used semi-structured interviews with 20 French-speaking adults with RRMS treated with DMTs for ≥ 10 years. Participants were recruited in France via patient associations and neurology clinics. Exclusion criteria included progressive MS or cognitive impairment. Interviews were conducted remotely and transcribed for thematic analysis using NVivo software, guided by a general inductive and grounded theory framework. Data saturation was monitored, and coding was performed by multiple researchers to ensure reliability.</p><p><strong>Results: </strong>Participants (mean age 52 years; 60% female; mean disease duration 20 years) reported strong preference for oral or infrequent infusion DMTs due to lower perceived treatment burden. Frequent home injections were linked with anxiety and reduced autonomy. Voluntary treatment pauses were common, motivated by fatigue or social needs, but constrained by the fear of relapse. Treatment experiences influenced identity and required substantial daily organization.</p><p><strong>Conclusion: </strong>Long-term patients with RRMS incorporate DMTs into their routines and identities, favoring less frequent, lower-burden regimens. Findings support the need for personalized care strategies that consider treatment fatigue and promote shared decision-making.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1917-1929"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396093/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148164090","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}
引用次数: 0
Reduced Childhood Outdoor Exposure Raises Pediatric Multiple Sclerosis (PedMS) Risk. 减少儿童户外暴露增加儿童多发性硬化症(PedMS)的风险。
IF 4.7 3区 医学
Neurology and Therapy Pub Date : 2026-08-01 Epub Date: 2026-05-24 DOI: 10.1007/s40120-026-00956-6
Silvy Pilotto, Angelo Ghezzi, Stefania Maria Bova, Marzia Fronza, Pietro Annovazzi, Marta Simone, Antonio Gallo, Agnese Suppiej, Roberta Lanzillo, Sarah Rasia, Angela Berardinelli, Valentina Torri Clerici, Lucia Moiola, Maurizio Viri, Stefano Sotgiu, Simona Malucchi, Alessandra Protti, Carlotta Canavese, Giacomo Lus, Luigi M E Grimaldi, Marta Zaffira Conti, Giovanna Borriello, Giovanna De Luca, Valentina Tomassini, Alberto Priori, Martina Tosi, Nicola Pomella, Andrea Corona, Alen Zollo, Maria Pia Amato, Eleonora Cocco, Maria Trojano, Filippo Martinelli-Boneschi, Sandra D'Alfonso, Roberto Bergamaschi, Maura Pugliatti
{"title":"Reduced Childhood Outdoor Exposure Raises Pediatric Multiple Sclerosis (PedMS) Risk.","authors":"Silvy Pilotto, Angelo Ghezzi, Stefania Maria Bova, Marzia Fronza, Pietro Annovazzi, Marta Simone, Antonio Gallo, Agnese Suppiej, Roberta Lanzillo, Sarah Rasia, Angela Berardinelli, Valentina Torri Clerici, Lucia Moiola, Maurizio Viri, Stefano Sotgiu, Simona Malucchi, Alessandra Protti, Carlotta Canavese, Giacomo Lus, Luigi M E Grimaldi, Marta Zaffira Conti, Giovanna Borriello, Giovanna De Luca, Valentina Tomassini, Alberto Priori, Martina Tosi, Nicola Pomella, Andrea Corona, Alen Zollo, Maria Pia Amato, Eleonora Cocco, Maria Trojano, Filippo Martinelli-Boneschi, Sandra D'Alfonso, Roberto Bergamaschi, Maura Pugliatti","doi":"10.1007/s40120-026-00956-6","DOIUrl":"10.1007/s40120-026-00956-6","url":null,"abstract":"<p><strong>Introduction: </strong>Sun exposure may influence MS susceptibility, but evidence in pediatric-onset MS (PedMS) is limited. We examined whether reduced early-childhood outdoor time (a proxy for lower sun exposure) is associated with PedMS risk.</p><p><strong>Methods: </strong>In the Italian multicenter PEDIGREE Study, environmental data were collected using the PEQ-IT questionnaire. We enrolled individuals < 18 years with PedMS and disease duration ≤ 5 years and controls without CNS inflammatory disorders. Outdoor time was reported by season and age (0-1, 1-2, 3-5 years); reduced activity was defined as < 60 min/week.</p><p><strong>Results: </strong>We included 114 PedMS cases and 121 controls. Cases were 77.2% female; mean (SD) age 16.8 (2.7) years; mean (SD) age at onset 14.2 (2.6) years; median EDSS 1.0 (range 0-4.0). Associations were strongest in winter: reduced outdoor time was associated with higher odds of PedMS in the first year (adjOR 3.02, 95%CI 1.50-6.08), ages 1-2 (adjOR 2.72, 95%CI 1.45-5.09), and ages 3-5 (adjOR 2.66, 95%CI 1.40-5.07). Across ages 0-5, low winter outdoor activity remained strongly associated (adjOR 4.30, 95%CI 1.82-10.17). Other seasons showed weaker but overall significant trends.</p><p><strong>Conclusion: </strong>Limited early-childhood outdoor activity may be associated with increased PedMS risk; larger longitudinal studies are needed.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1829-1844"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396100/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148005968","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}
引用次数: 0
Understanding the Unmet Needs of People with MS at Diagnosis and Throughout Their Care Journey: Insights from a Survey-Based Study. 了解MS患者在诊断和整个护理过程中未满足的需求:来自基于调查的研究的见解。
IF 4.7 3区 医学
Neurology and Therapy Pub Date : 2026-08-01 Epub Date: 2026-05-01 DOI: 10.1007/s40120-026-00942-y
Klaus Schmierer, Pieter van Galen, Helen Gray, Alice Laroni, Amanda Montague, Stanca Potra, Heidi Thompson
{"title":"Understanding the Unmet Needs of People with MS at Diagnosis and Throughout Their Care Journey: Insights from a Survey-Based Study.","authors":"Klaus Schmierer, Pieter van Galen, Helen Gray, Alice Laroni, Amanda Montague, Stanca Potra, Heidi Thompson","doi":"10.1007/s40120-026-00942-y","DOIUrl":"10.1007/s40120-026-00942-y","url":null,"abstract":"<p><strong>Introduction: </strong>Multiple sclerosis (MS) has a broad range of symptoms and heterogenous trajectory that requires individualised care. To optimise shared decision-making between healthcare professionals (HCPs) and people with MS (PwMS), it is important to understand communication needs from the patient perspective at diagnosis and throughout their care journey.</p><p><strong>Methods: </strong>Two multinational online surveys were conducted to explore (1) communication needs around the time of diagnosis, and (2) PwMS empowerment in communicating their specific needs and symptoms. Questionnaires included ten close-ended questions and were shared among 100 PwMS aged 18-50 years in Australia, Spain, the UK and the USA. Anonymised data were analysed by a core panel of HCPs, PwMS and patient advocacy group representatives, and key recommendations were agreed.</p><p><strong>Results: </strong>The majority of respondents were female (65-80%) and from the UK (80-87%). PwMS and caregivers are often overwhelmed and feel 'lost' at the time of diagnosis. Early regular contact is critical for effective delivery of key information and building a trusting relationship. PwMS value a clear explanation of the healthcare team and next steps, but only around a quarter (26%) had HCP roles clearly explained. PwMS are often uncertain if health changes are related to MS and 42% reported not feeling comfortable discussing 'invisible' symptoms such as cognitive, mood and emotional changes. Most respondents (54%) reported that their MS nurse was the person they were most likely to consult. Support services were not routinely offered; only 26% were informed about patient support groups. The most reported benefit of an MS-specific patient group was 'feeling less alone'.</p><p><strong>Conclusion: </strong>Regular HCP contact after diagnosis, peer group support for PwMS and their caregivers, signposting of reliable and accurate online resources and the timely offer of support services, including psychological support, should be routine elements of care from the point of diagnosis.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1663-1674"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396049/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147818262","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}
引用次数: 0
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