Background: Post-stroke spasticity affects a significant proportion of stroke survivors and impairs quality of life. Repeated intramuscular injections of OnabotulinumtoxinA are widely used for spasticity management; however, long-term real-world outcomes remain underreported. This study aimed to evaluate the long-term safety and treatment continuation patterns, and to identify predictors of treatment discontinuation associated with repeated onabotulinumtoxinA treatment.
Methods: This retrospective, single-center study included 224 post-stroke patients treated with OnabotulinumtoxinA between 2012 and 2023. Inclusion criteria were a diagnosis of post-stroke spasticity, initiation of treatment before 2021, and at least three years of follow-up. Outcomes assessed included treatment continuation rates, reasons for discontinuation, dose trends, and predictors of treatment discontinuation. Logistic regression and repeated measures ANOVA were used for statistical analyses.
Results: Of the 224 patients, 94 (42%) continued treatment as of December 2023. Reasons for discontinuation included improvement and completion (n = 59, 45.4%), unknown reason (n = 39, 30.0%), Change of doctor or relocation (n = 13, 10.0%), insufficient efficacy (n = 13, 10.0%), switch to other treatment (n = 4, 3.1%), and adverse events (n = 2, 1.5%). Long-term treatment was associated with progressive dose escalation (p < 0.05).Logistic regression analysis showed that cerebral infarction was significantly associated with treatment completion due to improvement(p = 0.004), while a lower initial dose demonstrated a non-significant trend toward treatment completion (p = 0.051).
Conclusions: Repeated onabotulinumtoxinA injections were not associated with unexpected safety concerns over long-term follow-up. Approximately 25% of patients discontinued treatment during the observation period, including cases documented as treatment completion due to clinical improvement. Stroke type was associated with treatment discontinuation patterns, supporting the importance of individualized long-term treatment planning.
{"title":"Long-term safety and treatment discontinuation patterns of OnabotulinumtoxinA for post-stroke spasticity: a retrospective study.","authors":"Ken Azuma, Michiyuki Kawakami, Naoko Watabe, Takayuki Kamimoto, Osamu Oshima, Yuka Yamada, Ayako Wada, Aiko Ishikawa, Tetsuya Tsuji","doi":"10.1186/s12883-025-04621-7","DOIUrl":"https://doi.org/10.1186/s12883-025-04621-7","url":null,"abstract":"<p><strong>Background: </strong>Post-stroke spasticity affects a significant proportion of stroke survivors and impairs quality of life. Repeated intramuscular injections of OnabotulinumtoxinA are widely used for spasticity management; however, long-term real-world outcomes remain underreported. This study aimed to evaluate the long-term safety and treatment continuation patterns, and to identify predictors of treatment discontinuation associated with repeated onabotulinumtoxinA treatment.</p><p><strong>Methods: </strong>This retrospective, single-center study included 224 post-stroke patients treated with OnabotulinumtoxinA between 2012 and 2023. Inclusion criteria were a diagnosis of post-stroke spasticity, initiation of treatment before 2021, and at least three years of follow-up. Outcomes assessed included treatment continuation rates, reasons for discontinuation, dose trends, and predictors of treatment discontinuation. Logistic regression and repeated measures ANOVA were used for statistical analyses.</p><p><strong>Results: </strong>Of the 224 patients, 94 (42%) continued treatment as of December 2023. Reasons for discontinuation included improvement and completion (n = 59, 45.4%), unknown reason (n = 39, 30.0%), Change of doctor or relocation (n = 13, 10.0%), insufficient efficacy (n = 13, 10.0%), switch to other treatment (n = 4, 3.1%), and adverse events (n = 2, 1.5%). Long-term treatment was associated with progressive dose escalation (p < 0.05).Logistic regression analysis showed that cerebral infarction was significantly associated with treatment completion due to improvement(p = 0.004), while a lower initial dose demonstrated a non-significant trend toward treatment completion (p = 0.051).</p><p><strong>Conclusions: </strong>Repeated onabotulinumtoxinA injections were not associated with unexpected safety concerns over long-term follow-up. Approximately 25% of patients discontinued treatment during the observation period, including cases documented as treatment completion due to clinical improvement. Stroke type was associated with treatment discontinuation patterns, supporting the importance of individualized long-term treatment planning.</p>","PeriodicalId":9170,"journal":{"name":"BMC Neurology","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-01-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145958790","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-01-12DOI: 10.1186/s12883-025-04577-8
Grace K Lank, Shreya Budhiraja, Jordan I Gaelen, Shreya Mukherjee, Tracey Singer, Aditi Venkatesh, Millenia Jimenez, Janet Miller, Melissa Lopez, C J Duax, David Blahnik, Doug King, Barbara A Hanson, Aasheeta P Bawa, Shreyas Bharadwaj, Ayush Batra, Eric M Liotta, Igor J Koralnik
Background: Long COVID, or post-acute sequelae of SARS-CoV-2 infection (PASC), affects 14 million people in the US. Neurologic manifestations of PASC (Neuro-PASC) are particularly debilitating. However, the evolution of these symptoms and factors associated with recovery are poorly understood. This study aimed to characterize Neuro-PASC symptom evolution using a mobile phone application and assess user experience.
Methods: The Neuro-COVID Recovery Care Companion (NCRCC) mobile application consists of questionnaires integrated within Northwestern Medicine's online MyChart platform which interfaces with the electronic medical record. Neuro-PASC patients completed daily surveys of twelve Neuro-PASC symptoms and their perceived percent recovery compared to their pre-COVID baseline. Patients also completed Patient-Reported Outcomes Measurement Information System (PROMIS) quality-of-life (QoL) surveys and NIH toolbox cognitive assessments at baseline and at 3-month follow up. Participants were retrospectively classified as "Improvers" or "Non-Improvers" based on the slope and range of their percent subjective recovery.
Results: Data from 63 participants presenting an average of 12.7 months after symptom onset were analyzed, including 27 (42.9%) Improvers and 36 (57.1%) Non-Improvers. Fewer women were Improvers (50% vs 75.7%; p = 0.04). Multiple correspondence analysis showed that patients presenting with a constellation of anosmia, dysgeusia, and a lack of insomnia (p = 0.023) were less likely Improvers. Improvers had more fluctuations in their subjective recovery than Non-Improvers with greater mean variance (7.01 vs 3.79; p = 0.0004) and positive recovery slope (5.84 vs 0; p < 0.0001). There were no differences in QoL and cognition at initial assessment, but Improvers showed a trend toward increased processing speed and decreased sleep disturbance after 3 months. Both groups found the NCRCC application easy-to-use, useful, and satisfactory.
Conclusions: Our findings reveal previously unrecognized fluctuations in subjective recovery of Neuro-PASC, and that women and patients presenting with anosmia and dysgeusia are less likely to improve one year from COVID-19 onset. We found broad alterations in QoL in both groups suggesting that strategies to reduce sleep disturbance and improve cognition may contribute to subjective improvement. Our results suggest similar mobile applications may benefit patients with other ill-defined chronic diseases, by equipping and empowering them on their often windy road to recovery.
背景:长冠状病毒,或SARS-CoV-2感染的急性后后遗症(PASC),影响了美国1400万人。PASC(神经性PASC)的神经系统表现尤其衰弱。然而,这些症状的演变和与康复相关的因素了解甚少。本研究旨在利用手机应用程序表征神经- pasc症状演变并评估用户体验。方法:NCRCC移动应用程序由问卷组成,集成在西北医学在线MyChart平台中,该平台与电子病历接口。神经- pasc患者完成了12种神经- pasc症状的每日调查,并与他们的前covid基线相比,他们的感知恢复百分比。患者还在基线和3个月随访时完成了患者报告结果测量信息系统(PROMIS)生活质量(QoL)调查和NIH工具箱认知评估。根据受试者主观恢复百分比的斜率和范围,回顾性地将其分为“改善者”或“非改善者”。结果:分析了63名平均出现症状后12.7个月的参与者的数据,包括27名(42.9%)改善者和36名(57.1%)非改善者。改善者较少(50% vs 75.7%; p = 0.04)。多重对应分析显示,出现嗅觉缺失、记忆障碍和缺乏失眠的患者(p = 0.023)改善的可能性较小。改善者的主观恢复波动比非改善者更大,平均方差更大(7.01 vs 3.79; p = 0.0004),恢复斜率为正(5.84 vs 0; p)。结论:我们的研究结果揭示了之前未被认识到的神经- pasc主观恢复波动,女性和出现嗅觉缺失和记忆障碍的患者在COVID-19发病一年后改善的可能性更小。我们发现两组患者的生活质量发生了广泛的变化,这表明减少睡眠障碍和改善认知的策略可能有助于主观改善。我们的研究结果表明,类似的移动应用程序可能会使患有其他不明确的慢性疾病的患者受益,通过装备和授权他们在他们经常艰难的康复之路上。
{"title":"Characterizing Neuro-PASC outcome with the mobile Neuro-COVID recovery care companion application.","authors":"Grace K Lank, Shreya Budhiraja, Jordan I Gaelen, Shreya Mukherjee, Tracey Singer, Aditi Venkatesh, Millenia Jimenez, Janet Miller, Melissa Lopez, C J Duax, David Blahnik, Doug King, Barbara A Hanson, Aasheeta P Bawa, Shreyas Bharadwaj, Ayush Batra, Eric M Liotta, Igor J Koralnik","doi":"10.1186/s12883-025-04577-8","DOIUrl":"10.1186/s12883-025-04577-8","url":null,"abstract":"<p><strong>Background: </strong>Long COVID, or post-acute sequelae of SARS-CoV-2 infection (PASC), affects 14 million people in the US. Neurologic manifestations of PASC (Neuro-PASC) are particularly debilitating. However, the evolution of these symptoms and factors associated with recovery are poorly understood. This study aimed to characterize Neuro-PASC symptom evolution using a mobile phone application and assess user experience.</p><p><strong>Methods: </strong>The Neuro-COVID Recovery Care Companion (NCRCC) mobile application consists of questionnaires integrated within Northwestern Medicine's online MyChart platform which interfaces with the electronic medical record. Neuro-PASC patients completed daily surveys of twelve Neuro-PASC symptoms and their perceived percent recovery compared to their pre-COVID baseline. Patients also completed Patient-Reported Outcomes Measurement Information System (PROMIS) quality-of-life (QoL) surveys and NIH toolbox cognitive assessments at baseline and at 3-month follow up. Participants were retrospectively classified as \"Improvers\" or \"Non-Improvers\" based on the slope and range of their percent subjective recovery.</p><p><strong>Results: </strong>Data from 63 participants presenting an average of 12.7 months after symptom onset were analyzed, including 27 (42.9%) Improvers and 36 (57.1%) Non-Improvers. Fewer women were Improvers (50% vs 75.7%; p = 0.04). Multiple correspondence analysis showed that patients presenting with a constellation of anosmia, dysgeusia, and a lack of insomnia (p = 0.023) were less likely Improvers. Improvers had more fluctuations in their subjective recovery than Non-Improvers with greater mean variance (7.01 vs 3.79; p = 0.0004) and positive recovery slope (5.84 vs 0; p < 0.0001). There were no differences in QoL and cognition at initial assessment, but Improvers showed a trend toward increased processing speed and decreased sleep disturbance after 3 months. Both groups found the NCRCC application easy-to-use, useful, and satisfactory.</p><p><strong>Conclusions: </strong>Our findings reveal previously unrecognized fluctuations in subjective recovery of Neuro-PASC, and that women and patients presenting with anosmia and dysgeusia are less likely to improve one year from COVID-19 onset. We found broad alterations in QoL in both groups suggesting that strategies to reduce sleep disturbance and improve cognition may contribute to subjective improvement. Our results suggest similar mobile applications may benefit patients with other ill-defined chronic diseases, by equipping and empowering them on their often windy road to recovery.</p>","PeriodicalId":9170,"journal":{"name":"BMC Neurology","volume":"26 1","pages":"24"},"PeriodicalIF":2.2,"publicationDate":"2026-01-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12794446/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145951438","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}
Pub Date : 2026-01-10DOI: 10.1186/s12883-025-04560-3
Matthias N Ungerer, Kilian Reichel, Manuel Feisst, Christine Tunkl, Benjamin Quenzer, Christoph Gumbinger
Background: Long-term changes in healthy behavior are more likely when small improvements are made in daily health habits. Health applications can serve as cost-effective tools to help people adopt healthier lifestyles. In response to this need, we developed the MAPS app - an easy-to-use and innovative mobile health app for the primary prevention of stroke.
Methods: We conducted a prospective, pre-post, single-arm, multicomponent interventional pilot study over 6 months to evaluate the ability of the MAPS app to enhance healthy habits and reduce stroke risk factors among a healthy adult population recruited from an urban employer in Germany. Daily health habits were assessed using questionnaires at the beginning of the study, at 3 months, and at 6 months. We also measured estimated stroke risk over 5 and 10 years, as well as systolic blood pressure, Body Mass Index (BMI), abdominal circumference, LDL cholesterol, and HbA1c levels at the beginning and after 6 months.
Results: A total of 35 participants were recruited. One participant was lost to follow-up. Our analysis showed significant improvements in the daily health habits questionnaire after 6 months (mean change of 5.6 points; 95% confidence interval (CI) 3.20 to 7.95). We observed significant reductions in the estimated 5-year stroke risk (mean change - 0.3%; 95% CI -0.52 to -0.04), BMI (mean change - 0.6 kg/m²; 95% CI -1.17 to -0.02), and abdominal circumference (mean change - 2.8 cm; 95% CI -5.28 to -0.25). Changes in LDL cholesterol and HbA1c levels were not significant. Male participants tended to have lower initial scores on daily habit questionnaires, particularly in dietary health choices, and benefited more from participation in the study.
Conclusions: Our results suggest that the MAPS app may help promote healthier daily habits and reduce stroke risk. While promising, these preliminary findings require further validation through larger randomized controlled trials. Additionally, they indicate potential gender-specific responses that need more investigation.
Trial registration: The study was registered retrospectively in the German Clinical Trials Register on 25th November 2024. (DRKSID DRKS00034491). Trial registration was submitted before enrollment started, but it was finalized after recruitment began due to technical issues.
背景:当日常健康习惯的微小改善时,健康行为的长期改变更有可能发生。健康应用程序可以作为具有成本效益的工具,帮助人们采用更健康的生活方式。为了满足这一需求,我们开发了MAPS应用程序,这是一款易于使用的创新移动健康应用程序,用于初级预防中风。方法:我们进行了一项为期6个月的前瞻性、前后、单臂、多组分干预性研究,以评估MAPS应用程序在从德国一家城市雇主招募的健康成年人中增强健康习惯和降低卒中危险因素的能力。在研究开始、3个月和6个月时,通过问卷调查评估日常健康习惯。我们还测量了5年和10年的中风风险,以及开始和6个月后的收缩压、体重指数(BMI)、腹围、低密度脂蛋白胆固醇和HbA1c水平。结果:共招募35名参与者。1名参与者失去随访。我们的分析显示,6个月后日常健康习惯问卷有显著改善(平均变化5.6点;95%可信区间(CI) 3.20至7.95)。我们观察到估计的5年卒中风险(平均变化- 0.3%;95% CI -0.52至-0.04)、BMI(平均变化- 0.6 kg/m²;95% CI -1.17至-0.02)和腹围(平均变化- 2.8 cm; 95% CI -5.28至-0.25)显著降低。LDL胆固醇和HbA1c水平变化不显著。男性参与者在日常习惯问卷上的初始得分往往较低,尤其是在饮食健康选择方面,他们从参与研究中获益更多。结论:我们的研究结果表明MAPS应用程序可能有助于促进更健康的日常习惯并降低中风风险。虽然这些初步发现很有希望,但需要通过更大规模的随机对照试验进一步验证。此外,它们表明了可能的针对性别的反应,需要更多的调查。试验注册:该研究于2024年11月25日在德国临床试验注册中心回顾性注册。(DRKSID DRKS00034491)。试验注册在招生开始前提交,但由于技术问题,在招生开始后才完成。
{"title":"Mobile Health Application for the Prevention of Stroke (MAPS): a pilot, single-arm study on an innovative health application designed for primary stroke prevention.","authors":"Matthias N Ungerer, Kilian Reichel, Manuel Feisst, Christine Tunkl, Benjamin Quenzer, Christoph Gumbinger","doi":"10.1186/s12883-025-04560-3","DOIUrl":"https://doi.org/10.1186/s12883-025-04560-3","url":null,"abstract":"<p><strong>Background: </strong>Long-term changes in healthy behavior are more likely when small improvements are made in daily health habits. Health applications can serve as cost-effective tools to help people adopt healthier lifestyles. In response to this need, we developed the MAPS app - an easy-to-use and innovative mobile health app for the primary prevention of stroke.</p><p><strong>Methods: </strong>We conducted a prospective, pre-post, single-arm, multicomponent interventional pilot study over 6 months to evaluate the ability of the MAPS app to enhance healthy habits and reduce stroke risk factors among a healthy adult population recruited from an urban employer in Germany. Daily health habits were assessed using questionnaires at the beginning of the study, at 3 months, and at 6 months. We also measured estimated stroke risk over 5 and 10 years, as well as systolic blood pressure, Body Mass Index (BMI), abdominal circumference, LDL cholesterol, and HbA1c levels at the beginning and after 6 months.</p><p><strong>Results: </strong>A total of 35 participants were recruited. One participant was lost to follow-up. Our analysis showed significant improvements in the daily health habits questionnaire after 6 months (mean change of 5.6 points; 95% confidence interval (CI) 3.20 to 7.95). We observed significant reductions in the estimated 5-year stroke risk (mean change - 0.3%; 95% CI -0.52 to -0.04), BMI (mean change - 0.6 kg/m²; 95% CI -1.17 to -0.02), and abdominal circumference (mean change - 2.8 cm; 95% CI -5.28 to -0.25). Changes in LDL cholesterol and HbA1c levels were not significant. Male participants tended to have lower initial scores on daily habit questionnaires, particularly in dietary health choices, and benefited more from participation in the study.</p><p><strong>Conclusions: </strong>Our results suggest that the MAPS app may help promote healthier daily habits and reduce stroke risk. While promising, these preliminary findings require further validation through larger randomized controlled trials. Additionally, they indicate potential gender-specific responses that need more investigation.</p><p><strong>Trial registration: </strong>The study was registered retrospectively in the German Clinical Trials Register on 25th November 2024. (DRKSID DRKS00034491). Trial registration was submitted before enrollment started, but it was finalized after recruitment began due to technical issues.</p>","PeriodicalId":9170,"journal":{"name":"BMC Neurology","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145948366","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-01-10DOI: 10.1186/s12883-025-04531-8
Jonathan Strober, Keiko Ishigaki, Nate Posner, Joseph C Cappelleri, Sherry Xiao, Anna Talaga, Stephen Dukacz, Halima Iqbal, Emma Chatterton, Ella Morton, Sarah Weatherby, Jonathan de Courcy
Background: The rare genetic disease Duchenne muscular dystrophy (DMD) mostly affects males, impacting mobility and pulmonary/cardiac function from childhood. This study explored non-professional caregiving in DMD, comparing burden across regions and by care recipients' ambulatory status.
Methods: Data were from the Adelphi Real World Disease Specific Programme™, a cross-sectional survey in Europe (France, Germany, Spain), the United States of America (USA), and Japan from October 2022-November 2023. Caregivers reported health status (their own and that of dependents with DMD) via the EQ-5D-5L, and their own productivity via the Work Productivity and Impairment Index (WPAI).
Results: Of 216 individuals with DMD, 45.8%, 41.7% and 12.5% were from Europe, USA and Japan, respectively; 54.4% were ambulatory, 68.4%, 52.2% and 11.1% in Europe, USA and Japan, respectively. Individuals with DMDs' mean (standard deviation; SD) EQ-5D-5L index value was 0.19 (0.27), with no significant difference across regions, but was higher in ambulatory than non-ambulatory individuals (p < 0.0001). Individuals with DMDs' mean (SD) EQ-VAS score was 24.3 (27.9), differed across regions (p < 0.0001), but did not differ by ambulatory status. In the USA, Europe and Japan, 38.9%, 23.5% and 7.4% of caregivers, respectively, worked full-time (p = 0.0020). Caregiver employment did not differ significantly between ambulatory/non-ambulatory dependents. Mean caregiver absenteeism, presenteeism and overall work impairment were 9.7%, 37.8% and 42.4%, respectively, with no significant differences between regions or ambulatory/non-ambulatory dependents. Mean activity impairment was 49.0%, (Japan: 67.6%, Europe: 49.4%, USA: 43.4%; p = 0.0002), and higher in caregivers of non-ambulatory than ambulatory dependents (p < 0.0001). Most caregivers reported no problems with their own mobility, self-care, usual activities, or pain/discomfort. The severity of anxiety/depression varied across regions (p < 0.0001), with no significant difference by dependents' ambulatory status. Mean (SD) caregivers' EQ-VAS score and index value were 26.9% (30.3%) and 0.83 (0.23), respectively. EQ-VAS score differed significantly across regions (USA: 52.1% [33.3%], Europe: 8.9% [1.7%], Japan: 8.9% [1.7%]; p < 0.0001), but not by dependents' ambulatory status. EQ-5D-5L index value did not differ significantly across regions or with dependents' ambulatory status.
Conclusions: Non-professional caregiving in DMD presented a substantial burden, varying across regions, and greater for those caring for non-ambulatory than ambulatory dependents.
背景:杜氏肌营养不良症(DMD)是一种罕见的遗传性疾病,主要发生在男性身上,从童年开始就影响运动能力和肺/心脏功能。本研究探讨了DMD的非专业护理,比较了不同地区的负担和护理对象的流动状况。方法:数据来自Adelphi Real World Disease Specific program™,这是一项横断面调查,于2022年10月至2023年11月在欧洲(法国、德国、西班牙)、美国和日本开展。护理人员通过EQ-5D-5L报告健康状况(他们自己和患有DMD的家属),并通过工作效率和损害指数(WPAI)报告他们自己的工作效率。结果:216例DMD患者中,来自欧洲、美国和日本的分别占45.8%、41.7%和12.5%;欧洲、美国和日本分别为54.4%、68.4%、52.2%和11.1%。DMD患者的EQ-5D-5L平均(标准差;SD)指数为0.19(0.27),各地区差异无统计学意义,但门诊患者的EQ-5D-5L指数高于非门诊患者(p)。结论:DMD患者的非专业护理负担较大,且各地区差异较大,非门诊患者的护理负担大于门诊患者。
{"title":"Caregiver burden in Duchenne muscular dystrophy in Europe, Japan, and the United States - a real-world study.","authors":"Jonathan Strober, Keiko Ishigaki, Nate Posner, Joseph C Cappelleri, Sherry Xiao, Anna Talaga, Stephen Dukacz, Halima Iqbal, Emma Chatterton, Ella Morton, Sarah Weatherby, Jonathan de Courcy","doi":"10.1186/s12883-025-04531-8","DOIUrl":"https://doi.org/10.1186/s12883-025-04531-8","url":null,"abstract":"<p><strong>Background: </strong>The rare genetic disease Duchenne muscular dystrophy (DMD) mostly affects males, impacting mobility and pulmonary/cardiac function from childhood. This study explored non-professional caregiving in DMD, comparing burden across regions and by care recipients' ambulatory status.</p><p><strong>Methods: </strong>Data were from the Adelphi Real World Disease Specific Programme™, a cross-sectional survey in Europe (France, Germany, Spain), the United States of America (USA), and Japan from October 2022-November 2023. Caregivers reported health status (their own and that of dependents with DMD) via the EQ-5D-5L, and their own productivity via the Work Productivity and Impairment Index (WPAI).</p><p><strong>Results: </strong>Of 216 individuals with DMD, 45.8%, 41.7% and 12.5% were from Europe, USA and Japan, respectively; 54.4% were ambulatory, 68.4%, 52.2% and 11.1% in Europe, USA and Japan, respectively. Individuals with DMDs' mean (standard deviation; SD) EQ-5D-5L index value was 0.19 (0.27), with no significant difference across regions, but was higher in ambulatory than non-ambulatory individuals (p < 0.0001). Individuals with DMDs' mean (SD) EQ-VAS score was 24.3 (27.9), differed across regions (p < 0.0001), but did not differ by ambulatory status. In the USA, Europe and Japan, 38.9%, 23.5% and 7.4% of caregivers, respectively, worked full-time (p = 0.0020). Caregiver employment did not differ significantly between ambulatory/non-ambulatory dependents. Mean caregiver absenteeism, presenteeism and overall work impairment were 9.7%, 37.8% and 42.4%, respectively, with no significant differences between regions or ambulatory/non-ambulatory dependents. Mean activity impairment was 49.0%, (Japan: 67.6%, Europe: 49.4%, USA: 43.4%; p = 0.0002), and higher in caregivers of non-ambulatory than ambulatory dependents (p < 0.0001). Most caregivers reported no problems with their own mobility, self-care, usual activities, or pain/discomfort. The severity of anxiety/depression varied across regions (p < 0.0001), with no significant difference by dependents' ambulatory status. Mean (SD) caregivers' EQ-VAS score and index value were 26.9% (30.3%) and 0.83 (0.23), respectively. EQ-VAS score differed significantly across regions (USA: 52.1% [33.3%], Europe: 8.9% [1.7%], Japan: 8.9% [1.7%]; p < 0.0001), but not by dependents' ambulatory status. EQ-5D-5L index value did not differ significantly across regions or with dependents' ambulatory status.</p><p><strong>Conclusions: </strong>Non-professional caregiving in DMD presented a substantial burden, varying across regions, and greater for those caring for non-ambulatory than ambulatory dependents.</p>","PeriodicalId":9170,"journal":{"name":"BMC Neurology","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145948323","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-01-09DOI: 10.1186/s12883-025-04603-9
Xiaoyue Hu, Xin Liu, Yiyun Fan, Hao Lei, Tianxin Zhang, Hong Zhou, Deicheng Zhao, Zhaoxiang Zhang, Ziling Huang, Bang Luo, Qing Ye, Yulan Dong
Background: Futile recanalisation (FR) defined as functional dependence despite successful reperfusion, is common after endovascular therapy for acute stroke. We aimed to evaluate whether the Vessel Distance Index (VDI), a novel imaging parameter, has the potential to predict the risk of FR.
Methods: Patients who underwent mechanical thrombectomy for acute anterior circulation large vessel occlusion between March 2022 and January 2025 were retrospectively reviewed. FR was defined as a modified Rankin Scale (mRS) score ≥ 3 at 90 days post-procedure. Univariate and multivariate logistic regression analyses were performed to identify independent predictors of FR. The discriminatory ability of significant variables was evaluated using receiver operating characteristic (ROC) curve analysis. The area under the receiver operating characteristic curve (AUC) and decision curve analysis (DCA) were used to compare FR risk models constructed from significant variables. Youden's index was used to determine the optimal cut-off threshold of VDI. Additionally, the Jonckheere-Terpstra trend test was used to evaluate whether VDI levels showed a monotonic trend across cohorts stratified by occlusion site and infarct progression rate (IPR) associated with FR risk.
Results: A total of 26 patients, accounting for 41.3% of the cohort (n = 63), were identified with FR. Advanced age, higher NIHSS score, lower ASPECTS score, and higher VDI value were independently associated with FR (p < 0.05). The ROC curve demonstrated that the combined model achieved the best predictive performance (AUC = 0.93, p < 0.001), outperforming any single variable. VDI showed a significant upward trend with FR risk in high IPR, and MCA groups (p < 0.05).
Conclusion: These findings suggest that VDI and traditional indicators can accurately predict long-term clinical outcomes before surgery and provide guidance for clinical practice.
{"title":"Progress on using CTA-based vessel distance index to predict futile recanalization: a retrospective observational study.","authors":"Xiaoyue Hu, Xin Liu, Yiyun Fan, Hao Lei, Tianxin Zhang, Hong Zhou, Deicheng Zhao, Zhaoxiang Zhang, Ziling Huang, Bang Luo, Qing Ye, Yulan Dong","doi":"10.1186/s12883-025-04603-9","DOIUrl":"https://doi.org/10.1186/s12883-025-04603-9","url":null,"abstract":"<p><strong>Background: </strong>Futile recanalisation (FR) defined as functional dependence despite successful reperfusion, is common after endovascular therapy for acute stroke. We aimed to evaluate whether the Vessel Distance Index (VDI), a novel imaging parameter, has the potential to predict the risk of FR.</p><p><strong>Methods: </strong>Patients who underwent mechanical thrombectomy for acute anterior circulation large vessel occlusion between March 2022 and January 2025 were retrospectively reviewed. FR was defined as a modified Rankin Scale (mRS) score ≥ 3 at 90 days post-procedure. Univariate and multivariate logistic regression analyses were performed to identify independent predictors of FR. The discriminatory ability of significant variables was evaluated using receiver operating characteristic (ROC) curve analysis. The area under the receiver operating characteristic curve (AUC) and decision curve analysis (DCA) were used to compare FR risk models constructed from significant variables. Youden's index was used to determine the optimal cut-off threshold of VDI. Additionally, the Jonckheere-Terpstra trend test was used to evaluate whether VDI levels showed a monotonic trend across cohorts stratified by occlusion site and infarct progression rate (IPR) associated with FR risk.</p><p><strong>Results: </strong>A total of 26 patients, accounting for 41.3% of the cohort (n = 63), were identified with FR. Advanced age, higher NIHSS score, lower ASPECTS score, and higher VDI value were independently associated with FR (p < 0.05). The ROC curve demonstrated that the combined model achieved the best predictive performance (AUC = 0.93, p < 0.001), outperforming any single variable. VDI showed a significant upward trend with FR risk in high IPR, and MCA groups (p < 0.05).</p><p><strong>Conclusion: </strong>These findings suggest that VDI and traditional indicators can accurately predict long-term clinical outcomes before surgery and provide guidance for clinical practice.</p>","PeriodicalId":9170,"journal":{"name":"BMC Neurology","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145942455","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-01-09DOI: 10.1186/s12883-025-04624-4
Anna Sobanska, Anna Sulek, Iwona Stepniak, Ewelina Elert-Dobkowska, Aleksandra Wierzbicka, Wanda Lojkowska, Maria Rakowicz
{"title":"Subclinical involvement of central nervous system structures other than motor or sensory tracts in SPG3A and SPG4 patients.","authors":"Anna Sobanska, Anna Sulek, Iwona Stepniak, Ewelina Elert-Dobkowska, Aleksandra Wierzbicka, Wanda Lojkowska, Maria Rakowicz","doi":"10.1186/s12883-025-04624-4","DOIUrl":"https://doi.org/10.1186/s12883-025-04624-4","url":null,"abstract":"","PeriodicalId":9170,"journal":{"name":"BMC Neurology","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145932073","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-01-09DOI: 10.1186/s12883-025-04617-3
Jiangying Cai, Lingzhi Zhao, Li Wang, Wanxia Yang, Lina Gao, Chongge You
{"title":"Construction of a prediction model for large-artery atherosclerosis ischemic stroke based on blood biomarkers and ultrasonic imaging.","authors":"Jiangying Cai, Lingzhi Zhao, Li Wang, Wanxia Yang, Lina Gao, Chongge You","doi":"10.1186/s12883-025-04617-3","DOIUrl":"https://doi.org/10.1186/s12883-025-04617-3","url":null,"abstract":"","PeriodicalId":9170,"journal":{"name":"BMC Neurology","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145942428","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Background: Diphenhydramine (DPH) is a common over-the-counter medication. DPH overdoses occur across all age groups, and abuse among adolescents has been increasing in recent years. We report two adolescent cases in which a false-positive tricyclic antidepressant (TCA) reaction on a urinary drug screening kit (UDS) led to the diagnosis of DPH poisoning, with a discussion of the mechanism of false positives based on an experiment using UDS and a literature review of the false positive cases by DPH.
Case reports: Case 1: A 13-year-old female patient was admitted for confusion and seizures. A UDS, SIGNIFY™ ER (Abbott Diagnostics Medical, USA) (SIG), showed a positive reaction to TCA. However, empty bottles of DPH-containing drugs, not TCA, were found at her home. She improved with supportive care alone and was discharged without sequelae. Case 2: A 13-year-old female patient presented with a generalized tonic-clonic seizure and impaired consciousness. On arrival, SIG showed a positive reaction to TCA. After admission, she confessed to having taken an overdose of multiple drugs, including DPH. In both cases, the blood DPH concentrations were in the toxic range, and urine DPH concentrations were below 100 micrograms per milliliter (µg/mL).
Experiment: DPH standard samples were added to urine from a healthy adult to create urine samples with multiple concentrations of DPH, which were subsequently used in SIG. Although the result was TCA-negative at 100 µg/mL, it turned out to be false positive at 500 µg/mL, suggesting that the kit-specific threshold concentration for TCA false positivity exists between 100 and 500 µg/mL.
Discussion and conclusions: Our cases and literature review indicate that SIG is prone to yield false positive TCA results in DPH overdose. Considering the increasing incidence of over-the-counter medication overdoses among adolescents in Japan, it is anticipated that there will be more pediatric cases of DPH poisoning in emergency departments. When a TCA-positive result is obtained on SIG, clinicians should consider not only TCA intoxication but also the possibility of DPH overdose. The mechanism of false positives cannot be explained by DPH alone, and the involvement of metabolites is suggested.
背景:苯海拉明(DPH)是一种常见的非处方药。DPH过量发生在所有年龄组,青少年滥用近年来一直在增加。我们报告了两例青少年病例,其中三环抗抑郁药(TCA)在尿药筛查试剂盒(UDS)上的假阳性反应导致了DPH中毒的诊断,并根据UDS的实验讨论了假阳性的机制,并对DPH假阳性病例的文献进行了综述。病例报告:病例1:一名13岁女患者因精神错乱和癫痫发作入院。一个UDS, SIGNIFY™ER (Abbott Diagnostics Medical, USA) (SIG)对TCA表现出阳性反应。然而,在她家中发现的是含有dph的空瓶药物,而不是TCA。患者仅接受支持性治疗后病情好转,出院时无后遗症。病例2:一名13岁女性患者表现为全身性强直阵挛发作和意识受损。到达后,SIG对TCA表现出积极的反应。入院后,她承认服用了过量的多种药物,包括DPH。在这两种情况下,血液DPH浓度均在毒性范围内,尿液DPH浓度均低于100微克/毫升(µg/mL)。实验:将DPH标准样品加入健康成人的尿液中,制成多种DPH浓度的尿液样本,随后用于SIG。虽然在100µg/mL时结果为TCA阴性,但在500µg/mL时结果为假阳性,这表明试剂盒特异性的TCA假阳性阈值浓度存在于100 - 500µg/mL之间。讨论和结论:我们的病例和文献综述表明,SIG在DPH过量时容易产生假阳性的TCA结果。考虑到日本青少年中非处方药物过量的发生率越来越高,预计急诊部将有更多的儿童DPH中毒病例。当SIG检测结果为TCA阳性时,临床医生不仅要考虑TCA中毒,还要考虑DPH过量的可能性。假阳性的机制不能仅由DPH来解释,可能与代谢物有关。
{"title":"False-positive tricyclic antidepressant results in diphenhydramine overdose: a report of two cases and a literature review.","authors":"Ryuichi Ito, Satoko Kobayashi, Hiroshi Terashima, Takaya Hanawa, Akiko Kinumaki, Shoichiro Kanda, Satoko Otsuka, Asuka Kaizaki-Mitsumoto, Satoshi Numazawa, Yutaka Harita","doi":"10.1186/s12883-025-04620-8","DOIUrl":"https://doi.org/10.1186/s12883-025-04620-8","url":null,"abstract":"<p><strong>Background: </strong>Diphenhydramine (DPH) is a common over-the-counter medication. DPH overdoses occur across all age groups, and abuse among adolescents has been increasing in recent years. We report two adolescent cases in which a false-positive tricyclic antidepressant (TCA) reaction on a urinary drug screening kit (UDS) led to the diagnosis of DPH poisoning, with a discussion of the mechanism of false positives based on an experiment using UDS and a literature review of the false positive cases by DPH.</p><p><strong>Case reports: </strong>Case 1: A 13-year-old female patient was admitted for confusion and seizures. A UDS, SIGNIFY<sup>™</sup> ER (Abbott Diagnostics Medical, USA) (SIG), showed a positive reaction to TCA. However, empty bottles of DPH-containing drugs, not TCA, were found at her home. She improved with supportive care alone and was discharged without sequelae. Case 2: A 13-year-old female patient presented with a generalized tonic-clonic seizure and impaired consciousness. On arrival, SIG showed a positive reaction to TCA. After admission, she confessed to having taken an overdose of multiple drugs, including DPH. In both cases, the blood DPH concentrations were in the toxic range, and urine DPH concentrations were below 100 micrograms per milliliter (µg/mL).</p><p><strong>Experiment: </strong>DPH standard samples were added to urine from a healthy adult to create urine samples with multiple concentrations of DPH, which were subsequently used in SIG. Although the result was TCA-negative at 100 µg/mL, it turned out to be false positive at 500 µg/mL, suggesting that the kit-specific threshold concentration for TCA false positivity exists between 100 and 500 µg/mL.</p><p><strong>Discussion and conclusions: </strong>Our cases and literature review indicate that SIG is prone to yield false positive TCA results in DPH overdose. Considering the increasing incidence of over-the-counter medication overdoses among adolescents in Japan, it is anticipated that there will be more pediatric cases of DPH poisoning in emergency departments. When a TCA-positive result is obtained on SIG, clinicians should consider not only TCA intoxication but also the possibility of DPH overdose. The mechanism of false positives cannot be explained by DPH alone, and the involvement of metabolites is suggested.</p>","PeriodicalId":9170,"journal":{"name":"BMC Neurology","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145942391","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-01-09DOI: 10.1186/s12883-025-04581-y
Qi Tian, Mengqi Liu, Fuxin Zhong, Xintong Liu, Yang Lü
Background: Lecanemab has been approved for the treatment of mild cognitive impairment due to Alzheimer's disease (AD) and mild AD dementia based on the efficacy in slowing cognitive decline and preliminary safety data from the phase Ⅲ Clarity AD trial. However, this trial excluded patients with high risk of cerebral hemorrhage, such as individuals with intracranial aneurysms or > 4 microhemorrhages.
Case presentation: A 70-year-old male with mild AD, intracranial aneurysm, microhemorrhages, and APOE ε3/ε4 genotype received lecanemab after multidisciplinary evaluation and informed consent. Over six months of intensive monitoring, cognitive function stabilized with no deterioration, daily activities were preserved, microhemorrhages remained stable (with one new small lesion noted at 3 months), and no aneurysm rupture or severe adverse events (including amyloid-related imaging abnormalities) occurred.
Conclusions: This case suggests that, despite hemorrhage risks, lecanemab may have a manageable risk-benefit profile in selected real-world AD patients under intensive monitoring and multidisciplinary care, with its application beyond clinical trial criteria requiring more nuanced and individualized consideration.
{"title":"Lecanemab treatment for mild alzheimer's disease with high risk of cerebral hemorrhage: a case report.","authors":"Qi Tian, Mengqi Liu, Fuxin Zhong, Xintong Liu, Yang Lü","doi":"10.1186/s12883-025-04581-y","DOIUrl":"https://doi.org/10.1186/s12883-025-04581-y","url":null,"abstract":"<p><strong>Background: </strong>Lecanemab has been approved for the treatment of mild cognitive impairment due to Alzheimer's disease (AD) and mild AD dementia based on the efficacy in slowing cognitive decline and preliminary safety data from the phase Ⅲ Clarity AD trial. However, this trial excluded patients with high risk of cerebral hemorrhage, such as individuals with intracranial aneurysms or > 4 microhemorrhages.</p><p><strong>Case presentation: </strong>A 70-year-old male with mild AD, intracranial aneurysm, microhemorrhages, and APOE ε3/ε4 genotype received lecanemab after multidisciplinary evaluation and informed consent. Over six months of intensive monitoring, cognitive function stabilized with no deterioration, daily activities were preserved, microhemorrhages remained stable (with one new small lesion noted at 3 months), and no aneurysm rupture or severe adverse events (including amyloid-related imaging abnormalities) occurred.</p><p><strong>Conclusions: </strong>This case suggests that, despite hemorrhage risks, lecanemab may have a manageable risk-benefit profile in selected real-world AD patients under intensive monitoring and multidisciplinary care, with its application beyond clinical trial criteria requiring more nuanced and individualized consideration.</p>","PeriodicalId":9170,"journal":{"name":"BMC Neurology","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145942471","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}