Neurology. Clinical practice最新文献

{"title":"Feasibility for a Community-Based Parkinson Disease Cohort in Hawaii.","authors":"Emma Krening, Ruby Shuman, Malika Faouzi, Kenny Thai, Fay Gao, Caroline M Tanner, G Webster Ross, Michiko K Bruno","doi":"10.1212/CPJ.0000000000200603","DOIUrl":"https://doi.org/10.1212/CPJ.0000000000200603","url":null,"abstract":"<p><strong>Background and objectives: </strong>Most of the research in Parkinson disease (PD) has been conducted in White populations. There is a gap in the understanding of PD in other racial/ethnic groups, such as Asian Americans (AAs) and Native Hawaiians or Pacific Islanders (NHPIs). Therefore, we aimed to test the feasibility of a longitudinal cohort and understand attitudes and barriers to research participation in a pilot study of AA, NHPI, and White patients with PD in Hawai'i.</p><p><strong>Methods: </strong>We conducted a prospective pilot study of AA, NHPI, and White patients with PD, without dementia, to compare characteristics between racial groups' self-reported quality of life (QoL), interest in participating in research, health care utilization, and barriers to accessing health care, at baseline and 6-month follow-up visits. This was a single-center study completed at the Parkinson's and Movement Disorder Center at The Queen's Medical Center in Honolulu, Hawai'i from March 2023 to June 2024.</p><p><strong>Results: </strong>Of 146 patients screened for eligibility, 79 completed study enrollment (mean 68.8 years, male = 59%). Demographics, PD history, and questionnaires from baseline and follow-up visits were analyzed for 78 participants (AA = 27, NHPI = 26, White = 25), with 1 participant who dropped out. Differences were found between groups' household income, comfort with technology, and awareness of advanced PD therapies. There was no significant difference between groups' self-reported QoL, barriers to PD care, and attitudes toward clinical research.</p><p><strong>Discussion: </strong>AA and NHPI patients with PD in this study were no less apt to participate in research compared with White patients with PD. Although there were no significant differences in the variables evaluated among different racial subgroups, our results warrant further research.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"16 3","pages":"e200603"},"PeriodicalIF":3.2,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13045732/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147623419","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Automated Intracranial Hemorrhage Detection: Real-World Experience in a Large Comprehensive Stroke Center.","authors":"Aakanksha Sriwastwa, Michael Oswald, Kara Weiss, Bin Zhang, Yasmin Ninette Aziz, Achala Vagal","doi":"10.1212/CPJ.0000000000200607","DOIUrl":"10.1212/CPJ.0000000000200607","url":null,"abstract":"<p><strong>Background and objectives: </strong>Automated intracranial hemorrhage (ICH) detection tools are widespread, yet data are limited regarding their performance in real-world practice.</p><p><strong>Methods: </strong>We retrospectively analyzed noncontrast CT head images of consecutive code stroke patients from January 2022 to February 2023 at a comprehensive stroke center. Patients were included if their indication was stroke, and images were assessed by the automated platform. Radiology reports were considered the gold standard. The primary outcome was the performance of the automated software tool compared with that of board-certified radiologists in ICH detection. The sensitivity, specificity, positive predictive value, negative predictive value, and accuracy were calculated using SAS software.</p><p><strong>Results: </strong>Of 1,434 code stroke CT scans, 1,402 (98%) were analyzed using the automated detection tool. Thirty-two studies were considered nondiagnostic because of severe motion degradation, and these were excluded. The mean patient age was 67 ± 16 years, with 51% of patients being women and 65% of patients being White. The software tool accurately identified 105 of 129 ICH cases (81%) and 1,255 of the 1,273 non-ICH cases (99%). The sensitivity, specificity, positive predictive value, and negative predictive value with 95% CIs were 81% (74%-88%), 99% (98%-99%), 85% (78%-91%), and 98% (97%-99%), respectively. Automated software sensitivity was highest for intra-axial hemorrhage (IAH) at 94%. Extra-axial hemorrhage (EAH) had a sensitivity of just 43%. Sensitivity of mixed IAH and EAH was 89%, representing a significant increase from isolated EAH.</p><p><strong>Discussion: </strong>Automated ICH detection software demonstrates high accuracy in detecting ICH in real-world practice. Sensitivity for IAH is particularly high, with detection of EAH reaching acceptable parameters when co-presenting with IAH but lacking in isolation.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"16 3","pages":"e200607"},"PeriodicalIF":3.2,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13020556/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147574908","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Outcomes of Subthalamic Nucleus and Globus Pallidus Interna Deep Brain Stimulation for Young-Onset Parkinson Disease.","authors":"Grace E Hey, Hikaru Kamo, Gilberto Perez Rodriguez Garcia, Tejas R Mehta, Robert Stephen Eisinger, Michael S Okun, Justin D Hilliard, Kelly D Foote, Adolfo Ramirez-Zamora","doi":"10.1212/CPJ.0000000000200605","DOIUrl":"10.1212/CPJ.0000000000200605","url":null,"abstract":"<p><strong>Background and objectives: </strong>Patients with young-onset Parkinson disease (YOPD) commonly experience early motor complications necessitating deep brain stimulation (DBS), and the subthalamic nucleus (STN) and globus pallidus interna (GPi) are the primary DBS targets used for reducing medication use and addressing dyskinesias. This study leveraged both the University of Florida Fixel Institute high volume of DBS operations and the INFORM database to evaluate motor outcomes in a large cohort of patients with YOPD treated with STN or GPi DBS, and addressed the limited available evidence and paucity of data on the long-term effects of these targets applied to YOPD.</p><p><strong>Methods: </strong>A single-center retrospective chart review was conducted on a nonrandomized cohort of adult patients with a diagnosis of YOPD treated with STN or GPi DBS between January 1999 and October 2023. Multidisciplinary evaluation was used to choose the anatomical target for each patient. Disease onset, lead location, medications, and mortality were collected and compared between cohorts. Preoperative, 6-month, and 1-, 3-, and 5-year Unified Parkinson's Disease Rating Scale (UPDRS) scores were used to compare motor outcomes.</p><p><strong>Results: </strong>There were 405 patients, and 61.3% received GPi DBS and 37.7% STN DBS. Mean disease duration at surgery was 12.3 (4.2) years for GPi and 11.5 (6.3) years for STN. GPi DBS improved ON UPDRS part 3 scores at 1 year (26.5 [11.84] vs 20.94 [9.09], <i>p</i> = 0.0024), and these scores worsened slightly by 5-year follow-up (20.94 [9.09] vs 24.72 [10.28], <i>p</i> = 0.0491). Unilateral STN DBS showed greater 6-month motor improvement vs unilateral GPi (<i>p</i> < 0.001) and remained stable from 6 months to 3 years (<i>p</i> = 0.3). GPi DBS improved ON UPDRS part 4 scores from baseline to 5 years (8.34 [3.15] vs 4.21 [2.25], <i>p</i> < 0.0001), whereas STN DBS showed no significant change (6.16 [4.21] vs 5.0 [2.98], <i>p</i> = 0.328). Although statistically significant, the changes did not meet the threshold for clinical relevance.</p><p><strong>Discussion: </strong>This large cohort study assessed long-term outcomes of STN vs GPi DBS when used in the setting of YOPD. Both targets improved motor symptoms at 1 and 5 years. STN offered modest motor benefits and greater medication reduction, while GPi DBS better addressed dyskinesia and motor fluctuations. These findings inform preoperative decision-making, although selection bias based on baseline symptoms remains a limitation of the data set.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"16 3","pages":"e200605"},"PeriodicalIF":3.2,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13020557/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147574413","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative Accuracy of TCD, TTE, TEE, and Cardiac CT in Detecting Right-to-Left Shunt in Embolic Stroke of Undetermined Source.","authors":"Vikas Ravi, Yasaman Pirahanchi, Seyed Behnam Jazayeri, Emma Rice, Dawn M Meyer, Kunal Agrawal, Royya Modir, Thomas Hemmen, Brett C Meyer, Reza Bavarsad Shahripour","doi":"10.1212/CPJ.0000000000200604","DOIUrl":"10.1212/CPJ.0000000000200604","url":null,"abstract":"<p><strong>Background and objectives: </strong>Identifying a right-to-left shunt (RLS), commonly due to a patent foramen ovale (PFO), is essential in evaluating embolic stroke of undetermined source (ESUS). While several imaging modalities are used for PFO detection, direct comparisons in a single cohort are limited. This study evaluates and compares the diagnostic performance of 4 major modalities: transcranial Doppler (TCD), transthoracic echocardiography (TTE), transesophageal echocardiography (TEE), and cardiac CT in patients with ESUS.</p><p><strong>Methods: </strong>We retrospectively identified patients with ESUS referred to our neurosonology laboratory from July 2023 to May 2025, who underwent at least 3 of the 4 diagnostic modalities. A confirmed PFO was defined by concordant positive findings on at least 2 tests. Diagnostic performance metrics (sensitivity, specificity, positive predictive value, negative predictive value [NPV], accuracy, and area under the curve [AUC]) were calculated, with pairwise comparisons assessed using the DeLong test.</p><p><strong>Results: </strong>Of 469 screened patients, 130 met inclusion criteria. TCD showed the highest sensitivity (100%), NPV (100%), and an accuracy of 87.6%, although specificity was moderate at 75.0%. After excluding patients with mild PFO or extracardiac shunts, specificity and accuracy improved to 81% and 90.8%, respectively. TTE achieved a sensitivity of 83.3% and specificity of 93.3%, with an overall accuracy of 88.1%. TEE had a sensitivity of 95.1%, specificity of 87.5%, and the highest overall accuracy at 91.3%. Cardiac CT had perfect specificity (100%) but the lowest sensitivity (45.8%), limiting its standalone diagnostic utility. AUC comparisons revealed superior diagnostic performance for TCD, TTE, and TEE over cardiac CT (<i>p</i> values <0.05), but no significant differences among TCD, TTE, and TEE.</p><p><strong>Discussion: </strong>TCD is a highly sensitive, noninvasive first-line tool for detecting RLS in ESUS, particularly in acute or resource-limited settings. Confirmatory TTE, TEE, or CT can be tailored to context, with a tiered approach optimizing PFO detection and prevention strategies. Results should be interpreted cautiously, given the referral and selection bias. Larger prospective multicenter studies are needed to validate this approach and improve outcomes.</p><p><strong>Classification of evidence: </strong>This study provides Class IV evidence that (1) TCD and TEE were highly sensitive in detecting PFO; (2) TTE and cardiac CT were highly specific in detecting PFO; (3) TCD, TTE, and TEE had superior diagnostic performance over cardiac CT (as defined by AUC comparison analysis); and (4) there were no significant differences in overall diagnostic performance among TCD, TTE, and TEE.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"16 3","pages":"e200604"},"PeriodicalIF":3.2,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13020560/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147574964","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinically Suspected Neurodegeneration in Histiocytic Neoplasms as Causes of Neurologic Decline: A Retrospective Study.","authors":"Amir Barmada, Ronald S Go, John C Benson, Gaurav Goyal, W Oliver Tobin","doi":"10.1212/CPJ.0000000000200579","DOIUrl":"10.1212/CPJ.0000000000200579","url":null,"abstract":"<p><strong>Objectives: </strong>The aim of this study was to evaluate the causes of suspected neurodegeneration in adults with histiocytic neoplasms.</p><p><strong>Methods: </strong>Patients with histiocytic neoplasms were identified. Inclusion criteria were (1) diagnosis of histiocytic neoplasm; (2) the treating hematologist suspected neurodegeneration; and (3) patients age 18 years or older. Active CNS histiocytic neoplasm was defined as new or enlarging T2 lesions, or gadolinium enhancing lesions on MRI.</p><p><strong>Results: </strong>Neurodegeneration was suspected in 23 of 478 patients. A progressive neurologic disorder was confirmed in 15 of 23 patients and was associated with an underlying active (12/15) or inactive (3/15) histiocytic neoplasm. Of the 8 patients without a progressive neurologic disorder, diagnoses included fixed deficit from histiocytic neoplasm (3/8), depression (2/8), fatigue (1/8), pain (1/8), and stroke (1/8). Of the 3 of 15 patients with progressive neurologic dysfunction and inactive CNS histiocytic neoplasm, all had progressive pontocerebellar dysfunction. Progressive pontocerebellar atrophy on MRI was present in 10 of 13 patients with progressive symptoms, and 2 of 6 patients with no progressive symptoms.</p><p><strong>Discussion: </strong>Progressive neurologic dysfunction in adult patients with histiocytic neoplasms is most frequently because of an active histiocytic neoplasm or nonhistiocytic etiology. A minority of patients have presumed nonneoplastic progressive neurologic dysfunction, primarily associated with pontocerebellar atrophy.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"16 2","pages":"e200579"},"PeriodicalIF":3.2,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12802971/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145990209","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The SIH Impact Inventory: A Pilot Study of a Novel Instrument Assessing Quality of Life in Spontaneous Intracranial Hypotension.","authors":"Victor Liaw, Deborah I Friedman","doi":"10.1212/CPJ.0000000000200585","DOIUrl":"10.1212/CPJ.0000000000200585","url":null,"abstract":"<p><strong>Background and objectives: </strong>Spontaneous intracranial hypotension (SIH) profoundly affects quality of life. We aimed to identify and quantify various aspects of patients' experiences with SIH.</p><p><strong>Methods: </strong>We piloted an \"SIH Impact Inventory,\" a cross-sectional survey developed in collaboration with patients, family members, and caregivers. Potential participants were identified from a single center using diagnosis and procedure codes. Participants completed the inventory online using REDCap between December 2021 and April 2022.</p><p><strong>Results: </strong>Ninety-eight adult patients completed the inventory. The mean age was 50.6 years, and 69.4% were female. Sixty-three percent had a confirmed diagnosis of SIH, and 36.7% had clinically suspected but unconfirmed SIH. The mean time to diagnosis was 2.0 (interquartile range: 0.5-4.8) years; 25.5% went undiagnosed for 5 or more years, and 75% were initially misdiagnosed. The 3 most common symptoms were head pain, neck pain, and \"brain fog.\" Of those undergoing epidural blood patch procedures, 22% experienced relief of symptoms for a median time of 1.3 months; those with a confirmed diagnosis had more prolonged relief. 58.2% reported experiencing rebound intracranial hypertension after a therapeutic procedure. Surgical repair of the leak was most likely to result in a symptom-free status (<i>p</i> = 0.003) than nonsurgical treatments. Of those working for compensation when they developed SIH, 95.2% indicated that the condition affected their ability to work and 65.1% stopped working. The financial burden was substantial for 65.3% of our cohort, with medical expenses (98.4%) and travel for health care (65.6%) being the most prevalent expenses. SIH negatively affected personal and family relationships for most patients.</p><p><strong>Discussion: </strong>Individuals with confirmed and suspected SIH experience difficulties related to the disorder itself and the lengthy process of diagnosis and treatment. Our findings demonstrate the marked impact of SIH on employment, education, interpersonal relationships, and finances. Compared with previous studies, our cohort reported considerable cognitive difficulties, with rates approaching those of head pain. Heightened awareness of SIH, referral to a center with expertise in SIH, increasing the number and geographic distribution of SIH centers, and advances in diagnostic and treatment modalities can help alleviate some of the challenges that patients face.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"16 2","pages":"e200585"},"PeriodicalIF":3.2,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12854680/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146106470","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy of IV Ketamine in Refractory/Super-Refractory Status Epilepticus: A Systematic Review and Meta-Analysis.","authors":"Abhijit Vijay Lele, Alex Raquer, Jorge Mejia-Mantilla, Samuel Ern Hung Tsan, Gentle Sunder Shrestha, Victor Lin, Samuel Neal Blacker, Sean Marinelli, Peter Chee Seong Tan, Sarah Wahlster, Andres Gempeler","doi":"10.1212/CPJ.0000000000200584","DOIUrl":"10.1212/CPJ.0000000000200584","url":null,"abstract":"<p><strong>Background and objectives: </strong>Intravenous ketamine is increasingly used for refractory and super-refractory status epilepticus (RSE/SRSE), yet its efficacy and optimal use remain uncertain. We therefore aimed to synthesize the available evidence to quantify the effectiveness of ketamine in achieving seizure cessation and to explore differences in treatment characteristics between patients who respond and those who do not.</p><p><strong>Methods: </strong>We conducted a systematic review and meta-analysis to estimate the pooled seizure cessation rate associated with intravenous ketamine. Secondary analyses compared ketamine initiation timing, dosing, and infusion duration between patients who achieved seizure cessation (responders) and those who did not (nonresponders).</p><p><strong>Results: </strong>Fourteen studies comprising 388 adult patients (249 responders, 139 nonresponders) were included. The pooled seizure cessation rate with ketamine was 64% (95% CI 49%-76%) with moderate heterogeneity (<i>I</i> ² = 54.1%). Sensitivity analysis showed no single study substantially influenced results, supporting robustness. Responders received ketamine earlier (3.2 ± 2.6 days) than non-responders (4.3 ± 2.6 days), mean difference of -0.90 days (95% CI: -1.31 to -0.49; <i>p</i> < 0.0001). The mean maintenance dose was 2.5 ± 1.4 mg/kg/hr (responders: 2.5 ± 1.3; nonresponders: 2.6 ± 1.4), with no significant difference between groups (mean difference -0.14 mg/kg/hr; 95% CI -0.45 to 0.18; <i>p</i> = 0.39). Infusion duration averaged 5.0 ± 4.2 days in both groups, with no significant difference (mean difference -0.07 days; 95% CI -1.02 to 0.88; <i>p</i> = 0.88). Ketamine discontinuation due to adverse events was rare (0.7%, 3/55 patients).</p><p><strong>Discussion: </strong>Intravenous ketamine demonstrates consistent effectiveness and safety as an adjunctive therapy in RSE/SRSE. However, the timing of initiation cannot be reliably linked to improved clinical outcomes given current methodological limitations and heterogeneity across studies. Future prospective research using standardized definitions and rigorous temporal data collection is needed to clarify whether the timing of ketamine initiation independently influences therapeutic success and to define its optimal integration within established status epilepticus (SE) treatment algorithms.</p><p><strong>Registration: </strong>The systematic review was registered (June 7, 2024) with the International Prospective Register of Systematic Reviews (PROSPERO, CRD42024549523).</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"16 2","pages":"e200584"},"PeriodicalIF":3.2,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12807489/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145998634","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effect of Erenumab on Patient-Reported Outcomes in Episodic Migraine in Asia, the Middle East, and Latin America: Results From the EMPOwER Study.","authors":"Shuu-Jiun Wang, Artemio A Roxas, Bibiana Saravia, Byung Kun Kim, Debashish Chowdhury, Naji J Riachi, Mei-Ling Sharon Tai, Surat Tanprawate, Tai Ngoc Tran, Yi Jing Zhao, Wendy Su, Shihua Wen, Subhayan Mondal, Laurent Ecochard, Michal Arkuszewski","doi":"10.1212/CPJ.0000000000200565","DOIUrl":"10.1212/CPJ.0000000000200565","url":null,"abstract":"<p><strong>Background and objectives: </strong>Migraine is a significant disabling neurologic headache disorder globally. Evaluating patient-related outcomes (PROs) is necessary to assess the impact of therapeutic interventions in preventive therapy. An exploratory analysis of data from the EMPOwER study examined the effect of erenumab on PROs in patients with episodic migraine (EM) in regions underrepresented in the pivotal Phase 3 trials of erenumab, specifically Asia, the Middle East, and Latin America.</p><p><strong>Methods: </strong>Patients (N = 900) were randomized (2:3:3) to receive monthly subcutaneous injections of erenumab 140 mg, erenumab 70 mg, or placebo. Adjusted mean changes from baseline in the Headache Impact Test (HIT-6), Migraine Physical Function Impact Diary (MPFID), modified Migraine Disability Assessment (mMIDAS), and EuroQoL 5-dimension 5-level scale (EQ-5D-5L) scores were assessed during the double-blind treatment phase of 3 months.</p><p><strong>Results: </strong>A statistically significant reduction from baseline in the HIT-6 total score was observed for erenumab 140 mg (-9.34, <i>p</i> < 0.001) and 70 mg (-8.39, <i>p</i> = 0.004) vs placebo (-6.62) at Month 3. Improvement in MPFID scores was also greater in the erenumab groups vs the placebo group (Everyday Activity: 140 mg, -5.61 [<i>p</i> = 0.002]; 70 mg, -4.94 [<i>p</i> = 0.011]; placebo, -3.19; Physical Impairment: 140 mg, -4.27 [<i>p</i> = 0.014]; 70 mg, -3.95 [<i>p</i> = 0.021]; placebo, -2.31) at Month 3. Similar findings were observed for mMIDAS scores (140 mg -8.99 [<i>p</i> < 0.001], 70 mg -8.11 [<i>p</i> = 0.011] vs placebo [-6.59]) and the EQ-5D-5L quality-of-life visual analog scale scores (140 mg 8.13 [<i>p</i> = 0.017], 70 mg 7.08 [<i>p</i> = 0.088] vs placebo [5.22]), although no meaningful between-group difference was noted for index values.</p><p><strong>Discussion: </strong>Erenumab showed favorable effects on PROs when compared with placebo in patients with EM. These results enhance the evidence for erenumab as an effective preventive therapy for patients with EM.</p><p><strong>Trial registration information: </strong>Clinicaltrials.gov/study/NCT03333109.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"16 2","pages":"e200565"},"PeriodicalIF":3.2,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12867334/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146119215","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Biosimilar Therapeutics Substitution: American Academy of Neurology Position Statement.","authors":"Jonathan D Santoro, Jonathan Crowe, Cale H Coppage, Emily Klatte, Benjamin Tolchin, Claire Riley, David A Evans, A Gordon Smith","doi":"10.1212/CPJ.0000000000200574","DOIUrl":"10.1212/CPJ.0000000000200574","url":null,"abstract":"<p><p>Biologic therapeutics have revolutionized treatment of disorders for which there were previously limited options. Biologic products are typically very expensive. However, the emergence of biosimilars (a biologic product that is nearly identical to a Food and Drug Administration [FDA]-approved \"branded\" version) offers an opportunity to reduce costs after the branded product's period of patent protection ends. Despite the promise of biosimilars, some physicians have expressed concern regarding interchangeability, especially in specific patient populations. The American Academy of Neurology (AAN) supports the cost-saving potential of biosimilar therapeutics while emphasizing the importance of a balance between reducing costs, maintaining clinical efficacy, and preserving the integrity of the physician-patient relationship. This position statement from the AAN offers a framework to aid neurologists in deciding whether to switch a patient from a branded biologic product to a biosimilar therapeutic. This framework aligns with broader AAN policies on drug pricing and medical decision-making autonomy.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"16 2","pages":"e200574"},"PeriodicalIF":3.2,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12802970/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145990190","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Unravelling Narcolepsy: A Series of Complex Pediatric Cases.","authors":"Martina Gnazzo, Giulia Pisanò, Valentina Baldini, Francesca Citeroni, Francesco Canulli, Diana De Ronchi, Fabio Pizza, Giuseppe Plazzi","doi":"10.1212/CPJ.0000000000200583","DOIUrl":"10.1212/CPJ.0000000000200583","url":null,"abstract":"<p><strong>Objectives: </strong>Narcolepsy type 1 (NT1) and narcolepsy type 2 (NT2) are rare, chronic neurologic disorders of hypersomnolence. Narcolepsy type 1 results from the selective loss of orexin-producing neurons, leading to markedly reduced levels of orexin neuropeptides in the brain and CSF. NT2 shares some symptoms with the former but has no orexin deficiency. Both disorders manifest as a spectrum of debilitating symptoms, including excessive daytime sleepiness (EDS), cataplexy (NT1 only), fragmented nocturnal sleep, sleep paralysis, and hallucinations. Diagnosis is particularly challenging, especially in pediatric patients.</p><p><strong>Methods: </strong>We describe 7 pediatric patients presenting with complex narcolepsy phenotype of EDS or cataplexy with a diverse array of comorbid genetic, neurologic, and neuropsychiatric conditions.</p><p><strong>Results: </strong>These cases illustrate the diagnostic challenges in differentiating \"primary narcolepsy\" from \"narcolepsy because of a medical disorder\" (e.g., Prader-Willi Syndrome) or \"narcolepsy associated with autism spectrum disorder or very early-onset schizophrenia.\" The patients underwent a comprehensive diagnostic workup, including polysomnography, multiple sleep latency testing (performed after wash-out of concomitant medications), brain magnetic resonance imaging, CSF hypocretin-1 assay, and, in case of consistent clues, autoimmune, and genetic testing.</p><p><strong>Discussion: </strong>Ensuring accurate and prompt narcolepsy diagnosis allows effective and patient-centered management.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"16 2","pages":"e200583"},"PeriodicalIF":3.2,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12885170/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146156144","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}