Therapeutic Advances in Neurological Disorders最新文献

{"title":"Measuring incremental burdens associated with increasing preventive-treatment failures among adults with migraine: a retrospective, cross-sectional study.","authors":"Jessica Ailani, Nemin Chen, Avani Patel, Nathan Spence, Nikoletta Sternbach, Katie B Tellor, Motomori Lewis, Joshua Brown","doi":"10.1177/17562864251337431","DOIUrl":"https://doi.org/10.1177/17562864251337431","url":null,"abstract":"<p><strong>Background: </strong>Migraine inflicts substantial personal, social, and economic tolls on many adults in the United States (US). Acute and preventive medicines can offer some relief, but most patients are untreated or experience treatment failures. How preventive-treatment failures affect outcomes for patients no longer on preventive treatments or for patients with migraine is insufficiently understood.</p><p><strong>Objective: </strong>To measure the patient-reported health and economic burdens associated with increasing preventive-treatment failures among US adults with diagnosed migraine.</p><p><strong>Design: </strong>Retrospective, cross-sectional study.</p><p><strong>Methods: </strong>Data analyzed were from the 2023 US National Health and Wellness Survey. Participants were adults diagnosed with migraine, having ≥4 monthly migraine or headache days, having taken acute or preventive prescription migraine medications or currently taking acute prescription migraine medications, and taking no preventive migraine medications. Participants were categorized as never treated with preventive medicines, having failed 1 preventive medicine, or having failed ≥2 preventive medicines. Health-related quality of life (HRQoL) was assessed with the Migraine Disability Assessment, RAND's 36-Item Short Form Survey Instrument, 5-Level EuroQoL instrument (EQ-5D), Work Productivity and Activity Impairment General Health version, 9-Item Patient Health Questionnaire, and 7-Item Generalized Anxiety Disorder scale. Details about medication use and health care resource use (HCRU) were collected. Data were adjusted by inverse probability of treatment weighting and compared using two-sided two-sample <i>t</i>-tests or Chi-square tests.</p><p><strong>Results: </strong>Patients who had failed preventive treatments had poorer HRQoL, greater work productivity loss, greater nonwork activity impairment, and greater HCRU than patients who had never taken preventive treatments. The number of preventive-treatment failures scaled with disease burden. Patients with ≥2 treatment failures had significantly lower EQ-5D scores (0.69 vs 0.73) than those for prevention-naïve patients; patients with ≥2 treatment failures had significantly higher overall work productivity loss (45.9% vs 34.9%), activity impairment (46.8% vs 36.7%), and higher rates of emergency room visits (37.0% vs 25.2%), hospitalization (23.5% vs 12.3%), and neurologist visits (17.6 vs 10.9%) than those of prevention-naïve patients. Medication overuse rates were similar among patients with any treatment failures and prevention-naïve patients (migraine-specific: 34.4%-39.3%; overall: 59.2%-62.3%).</p><p><strong>Conclusion: </strong>US adults with frequent migraines who failed preventive treatments have significantly greater unmet needs and different acute medication use patterns than adults who never took treatments.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251337431"},"PeriodicalIF":4.7,"publicationDate":"2025-06-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12146588/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144259001","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Decoding crosstalk between neurotransmitters and α-synuclein in Parkinson's disease: pathogenesis and therapeutic implications.","authors":"Lihua Guan, Liling Lin, Chaochao Ma, Ling Qiu","doi":"10.1177/17562864251339895","DOIUrl":"https://doi.org/10.1177/17562864251339895","url":null,"abstract":"<p><p>Parkinson's disease (PD) is the second most common neurodegenerative disease, characterized by progressive worsening of motor symptoms. The primary pathological hallmark of PD is the degeneration of dopaminergic neurons in the substantia nigra and the presence of Lewy bodies, which are primarily composed of α-synuclein (α-syn) aggregates. Both α-syn and various neurotransmitters, including catecholamines (catechols), play crucial roles in the pathogenesis of PD, although the precise pathogenic mechanisms remain incompletely understood. The crosstalk between neurotransmitters and α-syn is intricate and multifaceted. Pathological α-syn disrupted neurotransmitters' homeostasis by impairing release and reuptake of neurotransmitters, with specific modulation of catecholaminergic and glutamatergic systems. Conversely, neurotransmitters, especially catechols, covalently modify α-syn. Such modifications significantly influence α-syn aggregation dynamics and alter its neurotoxic properties. However, determining whether these interactions induce synergistic toxicity or confer neuroprotection remains controversial. Emerging evidence suggests other neurotransmitters like serotonin and γ-aminobutyric acid may also modulate α-syn aggregation and PD progression, though their roles require further investigation. Understanding these interactions is crucial for developing novel diagnostic and multi-target therapeutic strategies.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251339895"},"PeriodicalIF":4.7,"publicationDate":"2025-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12141805/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144249711","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Complement inhibitor therapy as a steroid-sparing strategy in generalized myasthenia gravis.","authors":"Sofia Marini, Silvia Falso, Federico Habetswallner, Martina Marini, Raffaele Iorio","doi":"10.1177/17562864251332037","DOIUrl":"10.1177/17562864251332037","url":null,"abstract":"<p><strong>Background: </strong>Standard management of generalized myasthenia gravis associated with anti-acetylcholine receptor autoantibodies (AChR-gMG) includes corticosteroids and nonsteroidal immunosuppressants. Complement inhibitors (CI) represent a more tailored therapeutic strategy. Real-world data on the steroid-sparing efficacy of CI remain limited.</p><p><strong>Objective: </strong>To investigate the steroid-sparing efficacy of CI in AChR-gMG.</p><p><strong>Design: </strong>We identified 69 AChR-gMG patients on corticosteroids treated with azathioprine (AZA), mycophenolate mofetil (MMF), or CI.</p><p><strong>Methods: </strong>Steroid tapering was assessed by comparing corticosteroid dosage at several time-points to baseline.</p><p><strong>Results: </strong>Steroids reductions were statistically significant for all therapies at every time point compared to baseline (all <i>p</i> < 0.001). Pairwise comparisons using the Mann-Whitney <i>U</i> test revealed significant differences between CI and MMF at 3 months (<i>p</i> = 0.0372), 6 months (<i>p</i> = 0.0193), and 9 months (<i>p</i> = 0.0321) and between CI and AZA at 6 months (<i>p</i> = 0.0415).</p><p><strong>Conclusion: </strong>CI rapidly and effectively reduced corticosteroid dosage in most AChR-gMG patients, suggesting their potential role as steroid-sparing therapeutic options.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251332037"},"PeriodicalIF":4.7,"publicationDate":"2025-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12126683/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144200077","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Approaches for treating neuropsychiatric symptoms in Parkinson's disease: a narrative review.","authors":"Jon Rodríguez-Antigüedad, Gonzalo Olmedo-Saura, Javier Pagonabarraga, Saül Martínez-Horta, Jaime Kulisevsky","doi":"10.1177/17562864251336903","DOIUrl":"10.1177/17562864251336903","url":null,"abstract":"<p><p>Neuropsychiatric symptoms in Parkinson's disease (PD) are highly prevalent and profoundly disabling, often emerging even before the onset of motor symptoms. As the disease progresses, these symptoms usually become increasingly impairing and are now recognized as having the greatest impact on quality of life not only for patients but also for caregivers. In recent years, there have been significant advances in the diagnosis and management of neuropsychiatric symptoms. However, there are still substantial gaps in therapeutic approaches and algorithms, with limited pharmacological and nonpharmacological treatment options currently available. One of the main reasons for this is the complex molecular and neural bases of these symptoms, which involve both dopaminergic and nondopaminergic neurotransmission systems and extend far beyond the nigrostriatal pathway. As a result, the drugs currently recommended for treating neuropsychiatric symptoms in PD are few and supported by limited evidence. In this context, the experience of the treating neurologist remains critical in selecting the most appropriate individualized therapy. The aim of this paper is to review the available therapeutic options and provide an overview of current research efforts, particularly those focusing on pharmacological treatments.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251336903"},"PeriodicalIF":4.7,"publicationDate":"2025-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12106999/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144161122","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Risk of stroke under disease modifying therapies for multiple sclerosis: a systematic review.","authors":"Maria-Ioanna Stefanou, Aikaterini Theodorou, Annerose Mengel, Konstantinos Melanis, Christos Bakirtzis, Vasileios Giannopapas, Dimitrios K Kitsos, Markus Kowarik, Katharina Feil, Dimitrios Tzanetakos, Vasiliki Zouvelou, Elizabeth Andreadou, John S Tzartos, Sotirios Giannopoulos, Georgios Tsivgoulis","doi":"10.1177/17562864251321669","DOIUrl":"10.1177/17562864251321669","url":null,"abstract":"<p><strong>Background: </strong>Epidemiological research indicates a heightened incidence of cerebrovascular disorders among patients with multiple sclerosis (MS).</p><p><strong>Objectives: </strong>The aim of the present systematic review was to investigate the potential association between disease modifying therapies (DMTs) and the risk of stroke in MS patient populations.</p><p><strong>Data sources and methods: </strong>A systematic literature search was performed in MEDLINE and SCOPUS databases up to April 6, 2024, to identify randomized-controlled clinical trials (RCT), registry-based and cohort-studies, case-series, and case-reports reporting on acute ischemic stroke (AIS) or intracerebral hemorrhage (ICH) in MS patients under different DMTs.</p><p><strong>Results: </strong>Twenty-one studies were included: 1 RCT, 6 registry-based or cohort studies, 2 case-series and 11 case-reports. Overall, DMTs appear to reduce the risk of stroke in MS patients, with DMT exposure linked to a 50% reduction of the risk of stroke compared to no DMT exposure. Although glatiramer acetate and dimethyl fumarate appear to lower the risk of stroke, concerns about fingolimod exists due to an observed elevated risk for ischemic heart disease and hypertension. Despite the absence of detected safety concerns with alemtuzumab at the MS population level, alemtuzumab-related complications, although rare, signal the need for heightened clinical vigilance. Similarly, β-interferons have been linked to life-threatening adverse events, comprising thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS). No associations between the risk of stroke and other DMTs, comprising natalizumab and teriflunomide, were detected; yet, newly approved DMTs were underrepresented.</p><p><strong>Conclusion: </strong>These findings highlight the importance of personalizing DMT selection and monitoring cardiovascular risk factors to reduce stroke risk in patients with MS.</p><p><strong>Trial registration: </strong>PROSPERO CRD42024534470.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251321669"},"PeriodicalIF":4.7,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12099087/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144143578","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Consulting, diagnosis and treatment patterns in migraine: results from the Migraine in Poland cross-sectional survey.","authors":"Marta Waliszewska-Prosół, Marcin Straburzyński, Sławomir Budrewicz, Karol Marschollek, Magdalena Nowaczewska, Paweł Gać, Richard B Lipton","doi":"10.1177/17562864251338675","DOIUrl":"https://doi.org/10.1177/17562864251338675","url":null,"abstract":"<p><strong>Background: </strong>The Migraine in Poland study is a cross-sectional survey that assesses symptomatology, consulting, diagnosis, treatment and impact of migraine in Poland.</p><p><strong>Objectives: </strong>The purpose of this article is to define patterns of care for migraine in Polish patients.</p><p><strong>Methods: </strong>The survey was conducted from August 2021 to June 2022. Participants were recruited through various channels, targeting mostly persons suffering from headaches. The web survey included questions allowing for diagnosis according to the International Classification of Headache Disorders. A detailed questionnaire evaluated healthcare system utilization, history of diagnosis, as well as the use of acute or preventive treatment, including non-pharmacological methods.</p><p><strong>Results: </strong>In total, 3225 individuals aged 13-80 (mean age 38.9) responded to the questionnaire (87.1% were women). Migraine without aura (MwoA) diagnosis was confirmed in 1679 (52.7%) of subjects, and 1571 (93.6%) of them consulted a medical professional for their headaches in the past. Among those who consulted for headache, 91% reported having received a medical diagnosis of migraine. 92.5% of MwoA participants declared the current use of some form of treatment. Non-steroidal anti-inflammatory drugs and acetaminophen were the most frequently used acute medications (<i>n</i> = 1318, 78.5%) followed by combination analgesics, especially those containing codeine (<i>n</i> = 991, 59%). Triptans/ergots were used by 57.1%. A total of 22.8% of subjects used acute treatment with a frequency indicating medication-overuse. Prophylactic treatment was at some point used by 35.68%, while 11.49% were currently on preventive medications. The most frequently prescribed preventives were iprazochrome (8.99%), followed by flunarizine (8.10%) and topiramate (5.90%). A total of 23.28% subjects used nutraceuticals for migraine prevention (most frequently magnesium).</p><p><strong>Conclusion: </strong>Despite high consultation and diagnosis rates among Polish patients with migraine, there is a need for improving standards of care, especially in regard to choice of treatment. There is also a need to raise public awareness of the dangers of codeine-based medications (available over-the-counter in Poland).</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251338675"},"PeriodicalIF":4.7,"publicationDate":"2025-05-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12066855/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144011627","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Admission systolic blood pressure and short-term outcomes after dual antiplatelet therapy in patients with minor ischemic stroke or transient ischemic attack.","authors":"Federico De Santis, Eleonora De Matteis, Lucio D'Anna, Michele Romoli, Tiziana Tassinari, Valentina Saia, Silvia Cenciarelli, Chiara Bedetti, Chiara Padiglioni, Bruno Censori, Valentina Puglisi, Luisa Vinciguerra, Maria Guarino, Valentina Barone, Marialuisa Zedde, Ilaria Grisendi, Marina Diomedi, Maria Rosaria Bagnato, Marco Petruzzellis, Domenico Maria Mezzapesa, Vincenzo Inchingolo, Manuel Cappellari, Cecilia Zivelonghi, Paolo Candelaresi, Vincenzo Andreone, Giuseppe Rinaldi, Alessandra Bavaro, Anna Cavallini, Stefan Moraru, Maria Grazia Piscaglia, Valeria Terruso, Marina Mannino, Alessandro Pezzini, Giovanni Frisullo, Francesco Muscia, Maurizio Paciaroni, Maria Giulia Mosconi, Andrea Zini, Ruggiero Leone, Carmela Palmieri, Letizia Maria Cupini, Michela Marcon, Rossana Tassi, Enzo Sanzaro, Giulio Papiri, Giovanna Viticchi, Daniele Orsucci, Anne Falcou, Simone Beretta, Roberto Tarletti, Patrizia Nencini, Eugenia Rota, Federica Nicoletta Sepe, Delfina Ferrandi, Luigi Caputi, Gino Volpi, Salvatore La Spada, Mario Beccia, Claudia Rinaldi, Vincenzo Mastrangelo, Francesco Di Blasio, Paolo Invernizzi, Giuseppe Pelliccioni, Maria Vittoria De Angelis, Laura Bonanni, Giampietro Ruzza, Emanuele Alessandro Caggia, Monia Russo, Agnese Tonon, Maria Cristina Acciarri, Chiara Di Fino, Cinzia Roberti, Giovanni Manobianca, Gaspare Scaglione, Francesca Pistoia, Alberto Fortini, Antonella De Boni, Alessandra Sanna, Alberto Chiti, Marcella Caggiula, Maela Masato, Massimo Del Sette, Francesco Passarelli, Maria Roberta Bongioanni, Manuela De Michele, Stefano Ricci, Raffaele Ornello, Simona Sacco, Matteo Foschi","doi":"10.1177/17562864251332720","DOIUrl":"https://doi.org/10.1177/17562864251332720","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Elevated baseline systolic blood pressure (SBP) was associated with poor outcomes following dual antiplatelet therapy (DAPT) in patients with non-cardioembolic minor ischemic stroke (MIS) or high-risk transient ischemic attack (TIA) in clinical trials.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;We aimed to assess the impact of admission SBP on the short-term outcomes after DAPT in patients with non-cardioembolic MIS or high-risk TIA.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We performed an inverse probability weighted (IPW) analysis from a prospective multicentric real-world study (READAPT) including patients with non-cardioembolic MIS (National Institute of Health Stroke Scale of 0-5) or high-risk TIA (ABCD2 ⩾4) who initiated DAPT within 48 h of symptom onset. The primary effectiveness outcome was the 90-day risk of new ischemic stroke or other vascular events. The secondary effectiveness outcomes were the 90-day modified Rankin Scale score ordinal shift, vascular and all-cause mortality, 24-h early neurological improvement or deterioration. The safety outcomes included the 90-day risk of moderate-to-severe and any bleedings, symptomatic intracranial hemorrhage, and 24-h hemorrhagic transformation. We used Cox proportional hazards regression with restricted cubic splines to model the continuous relationship between SBP and the hazard ratio (HR) of new vascular events. We selected SBP = 124 mm Hg as cut-off point for the IPW weighting. Outcomes were compared using Cox and generalized logistic regression analyses, adjusted for residual confounders.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;From 2278 patients in the READAPT cohort, we included 1291 MIS or high-risk TIAs (mean age 70.6 ± 11.4 years; 65.8% males). After IPW, patients with admission SBP ⩾124 mm Hg versus &lt;124 mm Hg had a significantly higher risk of 90-day ischemic stroke or other vascular events (adjusted HR: 2.14 (95% CI 1.07%-4.98%); &lt;i&gt;p&lt;/i&gt; = 0.033) and of 24-h early neurological deterioration (adjusted risk difference: 1.91% (95% CI 0.60%-3.41%); &lt;i&gt;p&lt;/i&gt; = 0.006). The overall risk of safety outcomes was low, although patients with SBP ⩾124 mm Hg on admission showed higher rates of 90-day moderate-to-severe and any bleeding events (adjusted risk difference: 1.24% (95% CI 0.38%-2.14%); &lt;i&gt;p&lt;/i&gt; = 0.004 and 6.18% (95% CI 4.19%-8.16%); &lt;i&gt;p&lt;/i&gt; &lt; 0.001; respectively), as well as of 24-h hemorrhagic transformation (adjusted risk difference: 1.57% (95% CI 0.60%-2.55%); &lt;i&gt;p&lt;/i&gt; = 0.001). Subgroup analysis showed a significant interaction between admission SBP, sex, and time to DAPT start in predicting 90-day new vascular events (&lt;i&gt;p&lt;/i&gt; for interaction &lt;0.001 and 0.007, respectively).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;In patients with non-cardioembolic MIS or high-risk TIA, higher levels of admission SBP may be associated with an increased risk of new vascular events, early neurological deterioration, and bleeding after DAPT use. Future studies should further investigate if op","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251332720"},"PeriodicalIF":4.7,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12046176/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143998842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-effectiveness of ofatumumab for the treatment of relapsing forms of multiple sclerosis in the United Arab Emirates.","authors":"Jihad Inshasi, Yomna Nowier, Evelina A Zimovetz, Ahmed Moussa, Ahmed Shatila, Ali Hassan, Mohamed Farghaly, Mona Thakre, Mustafa Shakra, Sara Al Dallal, Suzan I A Noori, Tayseer Zain Alabdin, Sherouq Khattab, Bassem Yamout","doi":"10.1177/17562864251330260","DOIUrl":"https://doi.org/10.1177/17562864251330260","url":null,"abstract":"<p><strong>Background: </strong>Multiple sclerosis (MS) is an autoimmune disease of the central nervous system, associated with demyelination and inflammation. Relapsing forms of MS (RMS) encompass all patients with MS experiencing relapses. While there is currently no cure for MS, the introduction of several disease-modifying therapies (DMTs) has significantly reduced the risk of relapse and disability in patients with MS. There is a high unmet need for an easy-to-administer DMT that can be used early in the treatment pathway.</p><p><strong>Objectives: </strong>This study aimed to evaluate the cost-effectiveness of ofatumumab compared with other commonly used DMTs for the treatment of RMS from a health payer perspective in the United Arab Emirates (UAE).</p><p><strong>Design: </strong>A cost-effectiveness analysis.</p><p><strong>Methods: </strong>A Markov model, based on expanded disability status scale (EDSS) health states, was developed using the UAE payer's perspective and a 65-year time horizon. The baseline patient distribution across the EDSS states was aligned to the population of ASCLEPIOS I and II trials. The British Columbia database informed natural history transition probabilities. Treatment effects were applied by delaying disability progression and reducing the number of relapses and were sourced from a network meta-analysis. The health care resource utilization was derived from interviews with local experts, who also validated the model structure and comparators, and utility inputs from published studies. In the absence of an officially defined willingness-to-pay threshold in the UAE, it was assumed to be United Arab Emirates Dirham (AED) 369,854 per quality-adjusted life-year (QALY), which is twice the UAE gross domestic product per capita.</p><p><strong>Results: </strong>Base-case results indicated that ofatumumab was dominant over ocrelizumab, dimethyl fumarate, fingolimod, and natalizumab intravenous; was more cost-effective compared with teriflunomide, fingolimod (generic), glatiramer acetate, interferons, and best supportive care; and resulted in an incremental cost-effectiveness ratio of AED 713,068 per QALY versus cladribine. Sensitivity analyses were in line with the results of the base-case analysis.</p><p><strong>Conclusion: </strong>From the UAE payer's perspective, ofatumumab could be a cost-effective treatment alternative for patients with RMS.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251330260"},"PeriodicalIF":4.7,"publicationDate":"2025-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038200/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144016043","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Association of CT perfusion parameters with outcomes in patients with medium vessel occlusion undergoing endovascular thrombectomy.","authors":"Yoel Schwartzmann, Hamza Joubran, Tamer Jubeh, Issa Metanis, Aviva Alpernas, Tali Jonas-Kimchi, Udi Sadeh, John M Gomori, Jose E Cohen, Hen Hallevi, Jeremy Molad, Ronen R Leker","doi":"10.1177/17562864251333515","DOIUrl":"https://doi.org/10.1177/17562864251333515","url":null,"abstract":"<p><strong>Background: </strong>Imaging biomarkers obtained on computed tomography perfusion (CTP) have proven effective in predicting outcomes after endovascular thrombectomy (EVT) in patients with large vessel occlusion stroke. However, the association of CTP imaging biomarkers with outcomes in medium-size vessel occlusion (MeVO) stroke patients remains unknown.</p><p><strong>Objectives: </strong>Therefore, we aimed to explore whether CTP parameters can be used for selection of a subset of MeVO patients that are more likely to benefit from EVT.</p><p><strong>Methods: </strong>Consecutively enrolled acute MeVO stroke patients treated with EVT were included. All patients underwent CTP on admission and follow-up noncontrast CT 24 h post-EVT. CTP parameters including core and penumbra volumes were obtained. Excellent outcome, defined as a modified Rankin score of 0-1 at 90 days poststroke was the primary outcome, and survival at 90 days was the safety outcome. Regression analyses were performed to examine the associations between different CTP parameters and outcomes.</p><p><strong>Results: </strong>Overall, 70 patients with MeVO were included (47% male, median age 75), and 66 (94%) had long-term follow-up data. Of those included, 26 patients (39%) had excellent outcomes and 2 (3%) had symptomatic intracerebral hemorrhage. On regression analysis, hypoperfused volumes on CTP were associated with excellent outcomes (adjusted odds ratio (aOR) 1.02, 95% confidence intervals (CI) 1.001-1.037), whereas core volume was not. Other factors associated with excellent outcome included admission National Institutes of Health Stroke Scale score (aOR 0.85, 95% CI 0.73-0.98) and lack of hypertension (aOR 0.07, 95% CI 0.07-0.62). Core or penumbral volumes were not associated with survival.</p><p><strong>Conclusion: </strong>Higher volumes of hypoperfused tissue on CTP are associated with a higher likelihood of excellent outcome. Core and penumbral volumes are not associated with increased mortality in patients with MeVO that undergo EVT.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251333515"},"PeriodicalIF":4.7,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12035288/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144040324","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Eculizumab as fast-acting rescue therapy for pembrolizumab-induced impending crisis state of myasthenia gravis: a case report.","authors":"Ying Huang, Futian Wu, Yihan Xiong, Kangling Huang, Xin Chang, Xiaofeng Chen, Xiali Chen, Qiwen Zhang, Liangyu Zou, Xuejun Fu, Huadong Zhang, Qianhui Xu","doi":"10.1177/17562864251333518","DOIUrl":"https://doi.org/10.1177/17562864251333518","url":null,"abstract":"<p><p>Immune checkpoint inhibitors, such as pembrolizumab, have demonstrated substantial therapeutic efficacy in the treatment of cancer. However, immune-related adverse events, including myasthenia gravis (MG), present significant clinical challenges. This study presents the case of a 75-year-old male patient who developed generalized acetylcholine receptor antibody-positive MG following the first infusion of pembrolizumab for cholangiocarcinoma. The patient's symptoms rapidly progressed to an impending myasthenic crisis (MGFA Class IVB) within 20 days of pembrolizumab administration. Eculizumab was used as a rescue therapy due to the unavailability of conventional treatments, resulting in rapid and significant symptom relief, with sustained improvement during maintenance therapy. The use of eculizumab as rescue therapy presents a viable treatment option for pembrolizumab-induced MG, owing to its rapid therapeutic effects.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251333518"},"PeriodicalIF":4.7,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12035124/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144033231","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}