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Differences in Patient-Physician Satisfaction for Migraine Treatment Medications in Patients with and without Medication-Overuse Headache: A Cross-Sectional Real-World Survey. 药物滥用性头痛患者和非药物滥用性头痛患者对偏头痛治疗药物满意度的差异:一项横断面真实世界调查。
IF 4.8 3区 医学
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-08-19 DOI: 10.1007/s40120-025-00812-z
Ryotaro Ishii, Takahiro Kitano, Masahiro Iijima, Mitsuhiro Nagano, Reiko Yoshikawa, James Jackson, Sophie Barlow, Emily Green, William Whitton, Lucy Hargreaves, Kanae Togo
{"title":"Differences in Patient-Physician Satisfaction for Migraine Treatment Medications in Patients with and without Medication-Overuse Headache: A Cross-Sectional Real-World Survey.","authors":"Ryotaro Ishii, Takahiro Kitano, Masahiro Iijima, Mitsuhiro Nagano, Reiko Yoshikawa, James Jackson, Sophie Barlow, Emily Green, William Whitton, Lucy Hargreaves, Kanae Togo","doi":"10.1007/s40120-025-00812-z","DOIUrl":"10.1007/s40120-025-00812-z","url":null,"abstract":"<p><strong>Introduction: </strong>Medication overuse headache (MOH) is incurred by the excessive use of acute medications, including over-the-counter (OTC) treatments. This study aimed to characterize the burden, management, and treatment satisfaction of patients with migraine with or without MOH in Japan.</p><p><strong>Methods: </strong>Data were derived from the Adelphi Migraine Disease Specific Programme (DSP)™, a cross-sectional survey conducted in Japan from August 2023 to February 2024. Physicians provided data for consecutive patients, including demographics, clinical characteristics, current treatment, OTC treatment usage, and treatment satisfaction. Patients voluntarily reported their symptom burden, migraine pain severity, and treatment satisfaction. Alignment between physician and patient regarding treatment satisfaction was assessed using Cohen's weighted kappa statistics and multivariable regression models.</p><p><strong>Results: </strong>Overall, 122 physicians provided data for 820 patients with migraine, 7.0% (n = 57) of whom had a diagnosis of MOH; 41.5% (n = 340) of patients self-reported data, with 3.2% (n = 11) having a diagnosis of MOH. Patients with MOH were more likely to experience chronic migraine (79%, n = 45), greater migraine frequency, and more severe migraine than those without MOH. Ninety percent of both groups received acute treatment. Patients with MOH were significantly more likely to receive preventive treatment than those without MOH (86% vs 56.6%, p < 0.001). OTC medication use was reported at 3.3% by physicians and 11.2% by patients. The alignment between physician and patient treatment satisfaction was low for acute treatment. The exploratory model analysis indicated that OTC use may have contributed to this misalignment.</p><p><strong>Conclusion: </strong>Our study revealed that the alignment regarding treatment satisfaction with acute medications is low. OTC treatment usage may have inflated physician satisfaction with prescribed acute medications and caused a discrepancy regarding satisfaction of patients with MOH. To improve patient outcomes, it is essential to align drug effectiveness ratings between physicians and patients by enhancing communication and mutual understanding.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2137-2159"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450176/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144874309","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
Volumetric Assessment of Perimesencephalic Subarachnoid Hemorrhage. 脑周围蛛网膜下腔出血的容量评估。
IF 4.8 3区 医学
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-08-22 DOI: 10.1007/s40120-025-00813-y
Emily Hoffmann, Hermann Krähling, Moritz Kleinevoss, Công Duy Bùi, Ziad Maksoud, Markus Holling, Michael Köhler, Tobias D Faizy, Christian Paul Stracke, Burak Han Akkurt
{"title":"Volumetric Assessment of Perimesencephalic Subarachnoid Hemorrhage.","authors":"Emily Hoffmann, Hermann Krähling, Moritz Kleinevoss, Công Duy Bùi, Ziad Maksoud, Markus Holling, Michael Köhler, Tobias D Faizy, Christian Paul Stracke, Burak Han Akkurt","doi":"10.1007/s40120-025-00813-y","DOIUrl":"10.1007/s40120-025-00813-y","url":null,"abstract":"<p><strong>Introduction: </strong>Perimesencephalic subarachnoid hemorrhage (pmSAH) is a rare, typically benign subtype of non-aneurysmal subarachnoid hemorrhage (SAH). While the majority of patients demonstrate a positive recovery trajectory, a subset of patients experiences complications, including vasospasm, hydrocephalus, or delayed cerebral ischemia (DCI). Reliable imaging markers for risk stratification are lacking. This study evaluates whether volumetric CT-based biomarkers-validated in aneurysmal SAH (aSAH)-are also predictive for pmSAH.</p><p><strong>Methods: </strong>In this retrospective single-center study, 72 patients with confirmed pmSAH between 2011 and 2024 were analyzed. The automated volumetric segmentation was performed using 3D Slicer and TotalSegmentator to quantify intracranial volume (ICV), brain volume (BV), cerebrospinal fluid (CSF), and selective sulcal volume (SSV). The associations between volumetric parameters and clinical presentation, complications, and functional outcome (Glasgow Outcome Scale, GOS) were assessed using non-parametric statistics and Spearman correlation.</p><p><strong>Results: </strong>The median intracranial volume was 1352.7 mL, brain volume 1247.3 mL, cerebrospinal fluid volume 95.9 mL, and selective sulcal volume 19.4 mL. Vomiting at presentation was associated with higher CSF and SSV values (p = 0.04 and p = 0.005, respectively), but no significant volumetric differences were found regarding other symptoms or complications (vasospasm, hydrocephalus, DCI). GOS scores were uniformly high (median = 5), and none of the volumetric markers significantly correlated with outcome or complication rate (all p > 0.05).</p><p><strong>Conclusion: </strong>In contrast to aSAH, volumetric CT biomarkers such as ICV, BV, CSF, and SSV do not offer predictive value in patients with pmSAH. Risk stratification should continue to rely on initial hemorrhage pattern and volume, clinical monitoring, and individualized assessment rather than other volumetric parameters.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2161-2170"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450190/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962930","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
The Predictive Value of Heart Rate Variability for the Prognosis of Patients with Mild to Moderate Traumatic Brain Injury. 心率变异性对轻中度颅脑损伤患者预后的预测价值。
IF 4.8 3区 医学
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-07-04 DOI: 10.1007/s40120-025-00792-0
Chenyu Wu, Lu Liu, Du Chen, Wei Zhu, Feng Xu, Peng Yang
{"title":"The Predictive Value of Heart Rate Variability for the Prognosis of Patients with Mild to Moderate Traumatic Brain Injury.","authors":"Chenyu Wu, Lu Liu, Du Chen, Wei Zhu, Feng Xu, Peng Yang","doi":"10.1007/s40120-025-00792-0","DOIUrl":"10.1007/s40120-025-00792-0","url":null,"abstract":"<p><strong>Introduction: </strong>Traumatic brain injury (TBI) is a major cause of death and disability in adults. After the injury, patients may experience autonomic dysfunction of the heart, making heart rate variability (HRV) an important factor in predicting their prognosis. This study explored the impact of HRV upon admission on the prognosis of patients with TBI.</p><p><strong>Methods: </strong>This was a retrospective study that collected clinical data from 172 patients with TBI. Unfavorable prognosis was defined as Glasgow Outcome Scale (GOS) scores < 4, the risk factors were investigated using multivariate logistic regression analysis, and a model for unfavorable prognosis was developed. The prediction value of several models was examined by calculating the area under the receiver operating characteristic (ROC) curve.</p><p><strong>Results: </strong>Among all 172 patients, those with unfavorable prognosis (31 patients) had a lower standard deviation of normal-to-normal (NN) interval (SDNN) index than those with favorable prognosis (65 vs. 86; P = 0.001). The GCS score (OR 0.295, P < 0.001), hemoglobin (OR 0.957, P = 0.002), CK-MB (OR 1.164, P = 0.022), and SDNN index (OR 0.973, P = 0.010) were identified as independent risk variables for unfavorable prognosis using multivariate logistic regression analysis, and they were utilized to build the prognosis model. The area under the ROC curve of the prognosis model was 0.939 (95% confidence interval, 0.898-0.979).</p><p><strong>Conclusion: </strong>HRV can be used as an independent predictor of unfavorable prognosis in patients with TBI. Including the SDNN index in prognostic models helps to effectively predict the prognosis of adult patients with TBI.</p><p><strong>Trial registration: </strong>ClinicalTrial.gov Identifier NCT07024381, retrospectively registered 16 June 2025.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1889-1901"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450187/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144560631","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
Characteristics of Postural Instability in Idiopathic Normal Pressure Hydrocephalus. 特发性正常压力脑积水的体位不稳定特征。
IF 4.8 3区 医学
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-07-13 DOI: 10.1007/s40120-025-00786-y
Eunhee Park, Kyunghun Kang, Janghoon Lee, Jong Taek Lee, Ki-Su Park, Chul-Hyun Kim, Yu-Sun Min
{"title":"Characteristics of Postural Instability in Idiopathic Normal Pressure Hydrocephalus.","authors":"Eunhee Park, Kyunghun Kang, Janghoon Lee, Jong Taek Lee, Ki-Su Park, Chul-Hyun Kim, Yu-Sun Min","doi":"10.1007/s40120-025-00786-y","DOIUrl":"10.1007/s40120-025-00786-y","url":null,"abstract":"<p><strong>Introduction: </strong>In patients with idiopathic normal pressure hydrocephalus (iNPH), the characteristics of static steady-state balance disturbance are not as well understood as those related to gait. This study aimed to investigate differences in center of pressure (COP) parameters between patients with iNPH and age-matched healthy controls.</p><p><strong>Methods: </strong>A total of 56 patients diagnosed with probable iNPH, who responded positively to cerebrospinal fluid tap test (CSFTT), along with 25 age-matched healthy controls, were enrolled. Prior to the CSFTT, COP measurements were taken using a force plate while participants stood quietly with eyes open (EO) and eyes closed (EC). We calculated the values of mediolateral (ML) and anteroposterior (AP) directions and ML/AP ratio in terms of the velocity of COP (vCOP), the standardized COP path length (Std_vCOP), and the peak power spectral density (PSD) at 0-0.5 Hz and 0.5-1.0 Hz. Additionally, we calculated the average distance from the COP center (Dist_COP) and the base of support (BOS). A Mann-Whitney U test with Bonferroni correction was used to compare differences between healthy controls and patients with iNPH under each visual condition (EO vs. EC).</p><p><strong>Results: </strong>There were no significant differences in gender, age, height, and weight between the healthy controls and the patients with iNPH. Patients with iNPH demonstrated greater spontaneous sway in the ML direction of vCOP (p < 0.001), increased variability in both ML and AP directions of Std_vCOP (p < 0.001 for both), a larger Dist_COP (p < 0.001) and BOS (p < 0.001) in the EC condition. Under the EO condition, patients with iNPH exhibited a significantly higher ML/AP ratio (p < 0.001) and larger BOS (p < 0.001). Furthermore, peak PSD at 0-0.5 Hz, reflecting low-frequency oscillations, was more pronounced in the ML direction and in the ML/AP ratio (p < 0.001 for both under EO), as well as in the AP direction (p < 0.001 under EC) in patients with iNPH. However, no significant differences were observed in the high-frequency oscillations (0.5-1.0 Hz).</p><p><strong>Conclusions: </strong>These findings suggest that patients with iNPH exhibit distinct COP patterns, particularly greater variability in the ML direction during quiet standing. In contrast, the AP direction appears to be more influenced by visual conditions when compared to healthy controls. This result highlights the potential need for tailored balance training in patients with iNPH.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1961-1974"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450156/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144626790","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
Use of Corticosteroids in Myasthenia Gravis: Expert Opinion for Daily Management. 使用皮质类固醇治疗重症肌无力:专家意见的日常管理。
IF 4.8 3区 医学
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-08-05 DOI: 10.1007/s40120-025-00796-w
Renato Mantegazza, Giovanni Antonini, Matteo Gastaldi, Rocco Liguori, Michelangelo Maestri, Elena Pegoraro, Barbara Polistena, Carmelo Rodolico, Francesco Habetswallner
{"title":"Use of Corticosteroids in Myasthenia Gravis: Expert Opinion for Daily Management.","authors":"Renato Mantegazza, Giovanni Antonini, Matteo Gastaldi, Rocco Liguori, Michelangelo Maestri, Elena Pegoraro, Barbara Polistena, Carmelo Rodolico, Francesco Habetswallner","doi":"10.1007/s40120-025-00796-w","DOIUrl":"10.1007/s40120-025-00796-w","url":null,"abstract":"<p><strong>Introduction: </strong>Myasthenia gravis, a rare autoimmune disorder characterized by muscle weakness and fatigue, it is a mainly B-cell mediated condition with antibodies directed against the acetylcholine receptor or functionally related molecules at the neuromuscular junction. Corticosteroids are still the most used treatment, as they are cheap and characterized by a rapid response. However, their long-term administration is associated with frequent and often severe side effects.</p><p><strong>Methods: </strong>We used the Expert Opinion methodology: a panel of eight neurologists, known to be experts in the management of MG patients, and one specialist in pharmacoeconomics, were brought together to discuss clinical relevant issues about the use of corticosteroids in MG.</p><p><strong>Results: </strong>Increasing doses of corticosteroids may temporarily exacerbate the symptoms of MG and clinical exacerbations can lead to severe consequences. In addition, prolonged chronic corticosteroid therapy carries a burden in terms of indirect costs due to side effects, which has prompted strategies to obtain the maximum benefits with minimal side effects.</p><p><strong>Conclusion: </strong>The panel concludes that, in the near future, therapeutic strategies based on the use drugs with better tolerability and potentially lower direct and indirect costs, will be necessary.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2063-2081"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450191/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144784884","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
Real-World Claims Analysis to Characterize the Burden of Tardive Dyskinesia in Long-Term Care Settings. 长期护理环境中迟发性运动障碍负担特征的真实世界索赔分析。
IF 4.8 3区 医学
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-09-04 DOI: 10.1007/s40120-025-00820-z
Morgan Bron, Gideon Aweh, Eric Jen, Amita Patel
{"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}
引用次数: 0
Incidence and Risk Factors for Serious Infections in Patients with Neuromyelitis Optica Spectrum Disorder: A Claims Database Study in Japan. 视谱神经脊髓炎患者严重感染的发生率和危险因素:日本索赔数据库研究
IF 4.8 3区 医学
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-07-17 DOI: 10.1007/s40120-025-00794-y
Noriko Isobe, Tetsuro Oda, Tomohiro Yamaguchi, Yuta Kamei, Takahiko Tsumuraya, Akinori Yuri, Ayako Nakasone, Keiko Asao, Shinichi Matsuda
{"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}
引用次数: 0
Assessing the Safety and Efficacy of Lamotrigine as Anti-myotonic Agent in Myotonic Dystrophy Type 1 (DM1): A Longitudinal, Open-Label, Pilot Study. 评估拉莫三嗪作为抗肌强直剂治疗1型肌强直营养不良(DM1)的安全性和有效性:一项纵向、开放标签、试点研究。
IF 4.8 3区 医学
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-08-02 DOI: 10.1007/s40120-025-00804-z
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
{"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}
引用次数: 0
Patient Preferences for Self-Injectable Preventive Treatment for Migraine: A Multi-country Discrete Choice Experiment. 患者对自我注射预防治疗偏头痛的偏好:一个多国离散选择实验。
IF 4.8 3区 医学
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-08-02 DOI: 10.1007/s40120-025-00801-2
Jaein Seo, Caitlin Thomas, Tommi Tervonen, Nicolas Krucien, Janet H Ford, Virginia L Stauffer, Robert A Nicholson, Kevin Harrison Duffy, Antje Tockhorn-Heidenreich
{"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}
引用次数: 0
Sequential use of OnabotulinumtoxinA and Erenumab in Chronic Migraine: Retrospective Real-World Report on Bidirectional Switching. 顺序使用OnabotulinumtoxinA和Erenumab治疗慢性偏头痛:双向切换的回顾性真实世界报告。
IF 4.8 3区 医学
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-08-02 DOI: 10.1007/s40120-025-00803-0
Carolin Luisa Hoehne, Aysenur Sahin, Lucas Hendrik Overeem, Kristin Sophie Lange, Mira Pauline Fitzek, Cornelius Angerhöfer, Uwe Reuter, Bianca Raffaelli
{"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}
引用次数: 0
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