Neurology and Therapy最新文献

{"title":"Real-World Claims Analysis to Characterize the Burden of Tardive Dyskinesia in Long-Term Care Settings.","authors":"Morgan Bron, Gideon Aweh, Eric Jen, Amita Patel","doi":"10.1007/s40120-025-00820-z","DOIUrl":"10.1007/s40120-025-00820-z","url":null,"abstract":"<p><strong>Introduction: </strong>Tardive dyskinesia (TD), a persistent and often debilitating movement disorder, is associated with prolonged exposure to dopamine receptor-blocking agents. Individuals aged ≥ 60 years are at increased risk for TD and TD-related burden (e.g., impaired balance, difficulty swallowing), which can complicate management in long-term care (LTC) settings. We evaluated the prevalence of TD diagnoses and characterized the populations, treatment patterns, and healthcare resource utilization within specific LTC settings.</p><p><strong>Methods: </strong>This retrospective, longitudinal, observational study used the STATinMED Real-World Insights Database (1/2017-12/2012). Commercial, Medicaid, and Medicare enrollees with a TD diagnosis code who had ≥ 1 LTC stay, continuous claims data capture for ≥ 1 year pre-LTC facility admission, and ≥ 1 year post-LTC facility discharge were included. Demographics and clinical characteristics were captured for 12 months pre-LTC index stay. Clinical outcomes were collected for 12 months post-index LTC stay.</p><p><strong>Results: </strong>Of 20,176 patients identified, 2294 had ≥ 2 years continuous benefits and were included. Most patients were aged ≥ 65 years (64.6%), female (67.3%), and Medicare enrollees (76.8%). Mean Charlson Comorbidity Index score was 3.72 (standard deviation: 4.2) for all patients, suggesting high comorbidity burden. Two-thirds (66.1%) of the population had mood disorders, and antidepressants were the most widely used medication (56.1%). Polypharmacy was prevalent: nearly half (47.9%) of the population was prescribed ≥ 3 medications with central nervous system properties, which can increase risk of falls and cognitive impairment in older adults; 64.8% of patients had ≥ 1 emergency department visit any time post-LTC stay.</p><p><strong>Conclusions: </strong>Our findings demonstrated individuals with TD in LTC settings have a high comorbidity burden and polypharmacy, particularly for medications with anticholinergic properties. Further investigation is warranted to evaluate the impact of TD in older adults in LTC settings and explore interventional practices that can improve clinical outcomes, such as falls with injury and activities of daily living decline.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2217-2226"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450166/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144993009","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 Risk Factors for Serious Infections in Patients with Neuromyelitis Optica Spectrum Disorder: A Claims Database Study in Japan.","authors":"Noriko Isobe, Tetsuro Oda, Tomohiro Yamaguchi, Yuta Kamei, Takahiko Tsumuraya, Akinori Yuri, Ayako Nakasone, Keiko Asao, Shinichi Matsuda","doi":"10.1007/s40120-025-00794-y","DOIUrl":"10.1007/s40120-025-00794-y","url":null,"abstract":"<p><strong>Introduction: </strong>Serious infection is a leading cause of mortality in patients with neuromyelitis optica spectrum disorder (NMOSD). We assessed the incidence of and risk factors for serious infections in patients with NMOSD.</p><p><strong>Methods: </strong>This observational, retrospective cohort study included patients with a first NMOSD diagnosis (index date) between January 2016 and August 2022 in Japan. Data were extracted between April 2008 and August 2022 from the Medical Data Vision database. A serious infection was defined as an infection diagnosed during hospitalization. We described the incidence rate, cumulative incidence, and estimated hazard ratios (HRs) of potential risk factors using a Cox proportional hazard model with time-fixed and time-varying covariates.</p><p><strong>Result: </strong>In this study (n = 4231), the incidence rate of serious infections was 5.77 [95% confidence interval (CI) 5.23-6.35] per 100 person-years, and the cumulative incidence ranged from 2.87% (95% CI 2.33-3.49%) at 6-month follow-up to 12.48% (95% CI 10.62-14.50%) at 5-year follow-up. Age [≥ 75 years (ref. 18-35 years); HR 2.48, 95% CI 1.36-4.53], cancer (HR 1.91, 95% CI 1.11-3.29), diabetes mellitus (HR 1.43, 95% CI 1.04-1.96), neurogenic bladder (HR 1.97, 95% CI 1.46-2.66), urolithiasis (HR 1.97, 95% CI 1.02-3.78), the number of NMOSD relapses (HR 1.27, 95% CI 1.03-1.56) and a low daily dose of oral glucocorticoid (> 0 mg and < 5 mg [ref. 0 mg]; HR 1.80, 95% CI 1.21-2.69) were associated with an increased risk of serious infections.</p><p><strong>Conclusion: </strong>The incidence of serious infections in the NMOSD population, including those mainly treated with conventional therapies in real-world settings, was comparable to that reported in clinical trials or observational studies under specific treatments. Various potential risk factors for serious infections were identified. These results may assist patients and clinicians in better decision-making regarding treatment options.</p><p><strong>Trial registration: </strong>University Hospital Medical Information Network Clinical Trials Registry (UMIN-CTR: UMIN000051151).</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1993-2009"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450173/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144659709","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":"Assessing the Safety and Efficacy of Lamotrigine as Anti-myotonic Agent in Myotonic Dystrophy Type 1 (DM1): A Longitudinal, Open-Label, Pilot Study.","authors":"Barbara Risi, Nesaiba Ait Allali, Stefano Cotti Piccinelli, Filomena Caria, Simona Damioli, Beatrice Labella, Enrica Bertella, Giorgia Giovanelli, Francesca Garofali, Giuseppina Margollicci, Roberto Carugati, Lucia Ferullo, Emanuele Olivieri, Loris Poli, Alessandro Padovani, Massimiliano Filosto","doi":"10.1007/s40120-025-00804-z","DOIUrl":"10.1007/s40120-025-00804-z","url":null,"abstract":"<p><strong>Introduction: </strong>Myotonia, defined as impaired relaxation of skeletal muscles after voluntary contraction or electrical stimulation, is a core feature of myotonic dystrophy type 1 (DM1) and can be highly disabling. The most used anti-myotonic drug, mexiletine, has limited availability and is associated with several side effects. Lamotrigine (LTG), an anti-epileptic drug that reduces voltage-sensitive sodium channel activity, has shown efficacy in treating myotonia in both in vitro models and patients with non-dystrophic myotonias. We aimed to investigate in a cohort of patients with DM1 the use of LTG as an anti-myotonic treatment in a real-world setting.</p><p><strong>Methods: </strong>We enrolled 14 consecutive adult patients with genetically confirmed DM1 and clinically significant myotonia impacting daily living (Myotonia Behaviour Scale, MBS > 1). LTG was administered in escalating doses, starting from 50 mg/day up to 200 mg/day. Efficacy was assessed using a linear mixed-effects model. Two functional timed tests [the 9-Hole Peg Test (9HPT) and the preparation of a coffee pot, devised by us and called the \"Coffee Task\" test] were performed at baseline (pre-treatment) and at each dose level. Safety data was also collected.</p><p><strong>Results: </strong>The mean age at enrollment was 40 years, and the mean disease duration was 12 years. LTG dosage had a significant positive effect on 9HPT performance at the maximum dose compared to baseline. Age and disease duration significantly influenced 9HPT results. No significant changes were observed in the other functional timed test. No serious adverse events were reported.</p><p><strong>Conclusion: </strong>This pilot, open-label study provides preliminary evidence for the efficacy and safety of LTG as an anti-myotonic treatment in patients with DM1. These findings support the need for larger, placebo-controlled trials to confirm its clinical utility.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2249-2260"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450168/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144768815","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":"Patient Preferences for Self-Injectable Preventive Treatment for Migraine: A Multi-country Discrete Choice Experiment.","authors":"Jaein Seo, Caitlin Thomas, Tommi Tervonen, Nicolas Krucien, Janet H Ford, Virginia L Stauffer, Robert A Nicholson, Kevin Harrison Duffy, Antje Tockhorn-Heidenreich","doi":"10.1007/s40120-025-00801-2","DOIUrl":"10.1007/s40120-025-00801-2","url":null,"abstract":"<p><strong>Introduction: </strong>Self-injectable calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs) and oral CGRP antagonists are currently available for migraine prevention. This study elicited the preferences of participants with migraine for self-injectable CGRP mAb autoinjectors and non-CGRP oral medication and determined the relative importance of autoinjector attributes.</p><p><strong>Methods: </strong>Adults from the USA, the UK, and Germany with episodic or chronic migraine who had taken migraine preventive treatments within the past 5 years completed a discrete choice experiment (DCE) online. Participants completed 12 experimental choice tasks, choosing their preferred treatment from three options (two hypothetical self-injectable CGRP mAbs autoinjectors, a non-CGRP oral medication), described by seven autoinjector attributes varied by levels. DCE data were analyzed using an error-component logit model to obtain relative attribute importance (RAI) and to estimate predicted choice probabilities (PCP) for autoinjector profiles.</p><p><strong>Results: </strong>In total 1067 participants (51.3% with episodic migraine; 52.6% female; median age 40 years) completed the DCE. Common preventive treatments used were anti-epileptics (47.3%), beta blockers (41.4%), and antidepressants (36.7%). Throughout the DCE, autoinjectors were chosen in 86.3% of cases over non-CGRP oral medication. The most important attribute in participants' treatment choices was injection duration, with a preference for shorter injection duration (RAI 37.0%), followed by auto-retractable needle removal over manual pull-out (RAI 30.8%), longer storage at room temperature (RAI 15.2%), and no pinching over pinching (RAI 12.5%). Participants were less concerned by dose confirmation (RAI 3.4%), injection steps (RAI 0.6%), and dosing schedule (RAI 0.5%). Elicited preferences suggest that an autoinjector profile comparable to galcanezumab (PCP 44.6%) had a higher likelihood (p < 0.001) of being chosen over profiles comparable to erenumab (PCP 28.8%) or fremanezumab three injections quarterly (PCP 26.6%).</p><p><strong>Conclusion: </strong>Participants tended to prefer self-injectable CGRP mAb autoinjectors over non-CGRP oral preventive medications for migraine. Preferences among autoinjectors were driven by injection duration, auto-retractability of needle removal, storage requirements, and autoinjector base and pinching requirements.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2033-2051"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450188/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144768816","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":"Sequential use of OnabotulinumtoxinA and Erenumab in Chronic Migraine: Retrospective Real-World Report on Bidirectional Switching.","authors":"Carolin Luisa Hoehne, Aysenur Sahin, Lucas Hendrik Overeem, Kristin Sophie Lange, Mira Pauline Fitzek, Cornelius Angerhöfer, Uwe Reuter, Bianca Raffaelli","doi":"10.1007/s40120-025-00803-0","DOIUrl":"10.1007/s40120-025-00803-0","url":null,"abstract":"<p><strong>Introduction: </strong>In clinical practice, switching between preventive treatments is common in patients with chronic migraine when efficacy is insufficient or tolerability is poor. With the advent of more targeted therapies, such as onabotulinumtoxinA and calcitonin gene-related peptide (CGRP) monoclonal antibodies, treatment options have expanded, yet evidence to guide sequencing decisions remains limited. The aim of this study was to investigate the real-world effectiveness of switching between onabotulinumtoxinA and erenumab and vice versa in patients with chronic migraine who showed inadequate response to their initial preventive treatment.</p><p><strong>Methods: </strong>This retrospective real-world study included patients with chronic migraine treated at the Headache Center, Charité-Universitätsmedizin Berlin between October 2022 and December 2024. Eligible patients had received both onabotulinumtoxinA and erenumab in sequence, switching as a result of insufficient efficacy or tolerability. A very good response was defined as a ≥ 50% reduction in monthly headache days in the third month after the switch.</p><p><strong>Results: </strong>Out of 632 screened patients, 78 met the inclusion criteria (84.6% female; mean age 43 ± 14 years). Of these, 54 switched from onabotulinumtoxinA to erenumab, and 24 from erenumab to onabotulinumtoxinA. A very good response was observed in 14 patients (17.9%): 10/54 (18.5%) after switching to erenumab and 4/24 (16.7%) after switching to onabotulinumtoxinA.</p><p><strong>Conclusion: </strong>Sequential preventive treatment with onabotulinumtoxinA and erenumab resulted in a very good response in about one-fifth of patients. Although both treatments target the CGRP pathway, their distinct mechanisms of action may still provide benefit when switching therapies after initial failure.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2053-2061"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450149/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144768817","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":"Advances in Ischemic Stroke Treatment: Current and Future Therapies.","authors":"Benjamin W Y Lo, Hitoshi Fukuda","doi":"10.1007/s40120-025-00810-1","DOIUrl":"10.1007/s40120-025-00810-1","url":null,"abstract":"<p><p>This review summarizes current concepts in our understanding of stroke anatomy, pathophysiology of cerebral hypoperfusion, and collateral circulation. It also provides an evidence-based update in stroke trials and treatments assessed using PRISMA guidelines. Intravenous thrombolysis, endovascular thrombectomy for anterior circulation strokes, blood pressure control after endovascular thrombectomy, and medical management principles are discussed. Endovascular thrombectomy and medical therapy improves functional independence at 90 days in anterior circulation strokes even in late windows up to 24 h post symptom onset regardless of infarct core size. Intensive systolic blood pressure control acutely post thrombectomy is associated with harm and worse outcomes. This review also provides an evidence-based update on neurorehabilitation strategies with emerging interventions such as brain-computer interface and robotics having the potential to maximize neuroplasticity for potential improvement and recovery post stroke.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1783-1796"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450147/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144835865","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":"Licensed Subcutaneous Infusion Therapies in Advanced Parkinson's Disease: An Indirect Treatment Comparison and Cost-Minimisation Analysis.","authors":"Marc Vérin, Ángel Sesar, Bharat Amlani, Niall Smith, Konstantinos Kipentzoglou, Stephen Montgomery, Alice Reading, Andrei Karlsson, Anna Willis, Andrew J Lees","doi":"10.1007/s40120-025-00789-9","DOIUrl":"10.1007/s40120-025-00789-9","url":null,"abstract":"<p><strong>Introduction: </strong>Parkinson's disease is the second most common neurodegenerative disorder. In advanced Parkinson's disease, subcutaneous (SC) infusion therapies represent minimally invasive and reversible treatment options. In the United Kingdom (UK), licensed SC infusion therapies include apomorphine and foslevodopa-foscarbidopa; both represent effective and generally well-tolerated therapies, although uncertainties regarding their relative efficacy, safety and costs remain.</p><p><strong>Methods: </strong>The relative efficacy and safety of apomorphine and foslevodopa-foscarbidopa was assessed via Bucher indirect treatment comparison (ITC) of TOLEDO (NCT02006121) and M15-736 (NCT04380142) data, with findings used to support a cost-minimisation analysis (CMM). Thirteen outcomes were evaluated. Efficacy and safety outcomes were measured as mean differences and risk differences, respectively. The CMM, conducted from a UK healthcare payer perspective, considered treatment acquisition and concomitant therapy costs over a 6.34-year horizon (obtained from a published observational study).</p><p><strong>Results: </strong>Bucher ITC results provided evidence for a comparable efficacy for apomorphine and foslevodopa-foscarbidopa in advanced Parkinson's disease. ITCs also indicated comparable safety, although a trend in favour of apomorphine was identified for hallucinations and most infusion site reactions assessed. The CMM demonstrated a clear per-patient cost benefit for apomorphine versus foslevodopa-foscarbidopa (£120,173.70), primarily driven by lower drug acquisition costs.</p><p><strong>Conclusion: </strong>The main difference between UK licensed SC infusion therapies for advanced Parkinson's disease relates to cost as opposed to clinical outcome, with some evidence for improved tolerability of apomorphine. This supports the continued use of apomorphine as first-line SC infusion treatment for advanced Parkinson's disease in UK clinical practice.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1919-1933"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450198/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144584401","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":"Safety of Risdiplam in Japanese Patients with Spinal Muscular Atrophy: A 12‑Month Interim Analysis of a Postmarketing Surveillance Study.","authors":"Kayoko Saito, Toshio Saito, Reiko Arakawa, Yasuhiro Takeshima, Hisahide Nishio, Yuka Ishikawa, Masahisa Katsuno, Takahiko Tsumuraya, Hiromitsu Kawata, Yuki Miyano, Hirofumi Komaki","doi":"10.1007/s40120-025-00795-x","DOIUrl":"10.1007/s40120-025-00795-x","url":null,"abstract":"<p><strong>Introduction: </strong>Risdiplam, an oral splicing modifier for the survival motor neuron-2 gene (SMN2), is approved for treating spinal muscular atrophy (SMA). While its safety and efficacy have been demonstrated in global trials, there are limited real-world data on its safety in Japanese patients with SMA. This all-case postmarketing surveillance (PMS) study aimed to assess the safety and usage patterns of risdiplam in Japan.</p><p><strong>Methods: </strong>This 12-month interim analysis is part of an ongoing PMS study that includes Japanese patients with SMA who have received risdiplam. The full observation period for this PMS is 24 months from the initiation of risdiplam treatment. Safety data, including adverse drug reactions (ADRs), were collected from case report forms (CRFs) submitted by participating healthcare facilities. ADRs were coded using the MedDRA/J classification.</p><p><strong>Results: </strong>This study included 538 patients with SMA from 259 institutions in Japan between August 2021 and August 2022. The median age (minimum-maximum) at enrolment was 22.5 (0-83) years, and 51.5% of patients were male. SMA type II (47.2%) and III (27.9%) were the most common phenotypes. The median treatment duration was 366.0 days, and 86.1% of patients continued risdiplam treatment. ADRs were reported in 112 patients (20.8%), while serious ADRs were reported in eight patients (1.5%). The most common ADRs (classified by MedDRA System Organ Class) were gastrointestinal disorders in 86 (16.0%) patients (diarrhoea in 43 [8.0%], faeces soft in 23 [4.3%] and stomatitis in 10 [1.9%] patients). Exploratory analysis suggested that advanced age, comorbidities and concomitant medication use might be associated with an increased incidence of gastrointestinal ADRs.</p><p><strong>Conclusions: </strong>This 12-month interim analysis of PMS data indicated that risdiplam was well tolerated among Japanese patients with SMA, consistent with previous clinical trial findings. A comprehensive evaluation of the safety and efficacy of risdiplam will be provided in the final 24-month analysis.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2083-2094"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450135/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144804450","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":"Beyond Efficacy: Persistence, NEDA, and Therapeutic Decision-Making in First-Line Multiple Sclerosis Treatment.","authors":"Clara Helena López-Caneda, Sergio Antón-Fuente, Maria José Pérez-Haro, Cesar Manuel Sánchez-Franco, Elena Alvarez-Rodríguez, Marta Aguado-Valcarcel, Maria Marcos-Bobillo, Marta Torrente-Carballido, Ines González-Suárez","doi":"10.1007/s40120-025-00811-0","DOIUrl":"10.1007/s40120-025-00811-0","url":null,"abstract":"<p><strong>Introduction: </strong>Injectable drugs, including interferon-beta and glatiramer acetate (collectively referred to as BRACE), dimethyl fumarate (DMF), and teriflunomide (TER) are commonly used as initial disease-modifying therapies (DMTs) for multiple sclerosis (MS), especially in patients with favorable prognostic profiles. Despite their continued use, real-world comparative data on long-term treatment persistence and comprehensive disease control remain limited.</p><p><strong>Methods: </strong>This retrospective study analyzed 400 patients initiating BRACE (n = 132), DMF (n = 130), or TER (n = 138) between 2014 and 2024 in routine clinical practice. Persistence was defined as treatment continuation without interruptions for ≥ 6 months. Effectiveness was evaluated using cumulative no evidence of disease activity (NEDA)-2 and NEDA-3 status. NEDA-2 included the absence of clinical relapses and confirmed disability progression; NEDA-3 additionally required the lack of MRI activity. Loss of NEDA status was marked from the first occurrence of any criterion failure.</p><p><strong>Results: </strong>Injectables showed significantly higher discontinuation rates (70.5%) compared to patients with TER (42.0%) and DMF (48.5%) (p < 0.001), with divergence evident after year 3. Median time to discontinuation was 3.55 years for BRACE, 4.88 years for TER, and 5.78 years for DMF. No significant differences were observed in NEDA-2 or NEDA-3 survival. Patients with TER showed higher NEDA-2 rates at 1 year (87%) than DMF (74%) and BRACE (77%) (p < 0.05), but this difference was not sustained over time.</p><p><strong>Conclusions: </strong>In real-world practice, oral therapies tend to be associated with better long-term persistence than injectables, while effectiveness measured by cumulative NEDA-3 remains comparable. These findings highlight the role of patient-centered factors such as tolerability and administration route in treatment adherence, supporting cumulative NEDA as a meaningful outcome in clinical decision-making.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2095-2106"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450137/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144822133","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":"Global Societal Burden of Alzheimer's Disease by Severity: a Targeted Literature Review.","authors":"Maria A Cavaco, Se Ryeong Jang, Christopher Olsen, Carolyn Bodnar, Nicole Ferko","doi":"10.1007/s40120-025-00815-w","DOIUrl":"10.1007/s40120-025-00815-w","url":null,"abstract":"<p><strong>Introduction: </strong>Alzheimer's disease (AD) is among the costliest of illnesses for the elderly, placing a significant burden on healthcare systems and caregivers. Despite the depth of evidence, reviews lack a holistic assessment of such costs, falling short of illustrating unmet medical needs. The objective of this review was to therefore characterize the total societal economic burden of AD, broken down by care setting and disease severity.</p><p><strong>Methods: </strong>A targeted literature search of systematic reviews, cost-of-illness, and observational studies published between 2013 and 2024 was conducted on MEDLINE and Embase to identify articles reporting the economic burden of AD. Grey literature was hand-searched. Both direct and indirect costs were assessed, including societal burdens not often reported by AD-specific cost-of-illness studies such as financial delinquencies.</p><p><strong>Results: </strong>In total, 81 articles were reviewed in depth, including 20 systematic reviews and 61 studies or reports. Findings consistently demonstrated that societal costs of AD or dementia typically increased by at least 50% between consecutive severity levels, increasing with disease progression. Informal caregiving often comprised close to half of societal costs, regardless of care setting, disease severity, or region. While studies reporting costs of mild cognitive impairment (MCI) were limited, the economic burden reported for this stage was appreciable compared to mild AD. Evidence for the impact of AD, as early as MCI, on quality of life (e.g., emotional and mental strain) and personal financial management capabilities was also identified.</p><p><strong>Conclusion: </strong>This review provides a comprehensive overview, from studies spanning over more than a decade, of the substantial societal economic burden associated with AD, across cost categories, care settings, disease stages, and regions. This review may be used to inform health economic evaluations of novel interventions with potential to reduce the enormous and growing global economic burden of AD and dementia.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1797-1826"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450196/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962857","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}