Neurology and Therapy最新文献

{"title":"A Patient-centric Clinical Trial Design to Comprehensively Evaluate Low-Sodium Oxybate in People with Idiopathic Hypersomnia or Narcolepsy.","authors":"Deborah A Nichols, Teresa L Steininger, Douglas S Fuller, M Todd Kirby, Emily C Barker, Marisa Whalen, Jessica K Alexander, Sarah Akerman, David T Plante","doi":"10.1007/s40120-025-00745-7","DOIUrl":"10.1007/s40120-025-00745-7","url":null,"abstract":"<p><strong>Introduction: </strong>Low-sodium oxybate (LXB; Xywav^®) is approved to treat idiopathic hypersomnia in adults and excessive daytime sleepiness or cataplexy in individuals aged ≥ 7 years with narcolepsy. The efficacy and safety of LXB have been demonstrated in randomized controlled trials. This study will comprehensively evaluate multiple daytime and nighttime symptoms in participants with idiopathic hypersomnia and participants with narcolepsy treated with LXB.</p><p><strong>Methods: </strong>Jazz DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment; NCT05875974) is a prospective, multi-cohort, multicenter, single-arm, open-label, interventional study. Establishing multiple cohorts across different diseases aligns with more generalizable research designs enabling a broader impact. Total study duration is ~ 10-21 weeks, which includes a 2- to 6-week screening period, an 8-day baseline period, a 2- to 8-week titration period, a 2-week stable-dose period, a 1- to 2-week end-of-treatment assessment period, and a 2-week safety follow-up period. To provide a robust dataset of changes with LXB treatment which will inform healthcare providers and their patients, DUET is administering a wide range of patient- and clinician-reported outcome assessments regarding symptom severity and daytime functioning and includes objective measures of sleep (ad libitum polysomnography protocol) and sleep inertia which have not been previously tested with this type of study design. Additionally, pharmacokinetics data and clinician titration feedback are collected to inform titration/dosing guidance for clinicians. DUET was designed in a patient-centric manner to reflect a real-world approach to conducting clinical trials.</p><p><strong>Planned outcomes: </strong>Using a patient-centric design aiming to address participants' burden and improve their study experience, the DUET study will fill critical idiopathic hypersomnia and narcolepsy evidence gaps pertaining to sleep architecture (e.g., disrupted nighttime sleep) and response to LXB treatment, as well as provide data on outcomes that are meaningful to patients. Graphical abstract available for this article.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1705-1727"},"PeriodicalIF":3.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255640/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Differentiation Between Early and Severe Stages of Dementia in Claims Data Based on Diagnosis, Prescription, and Utilization Patterns.","authors":"Moritz Platen, Maresa Buchholz, Anika Rädke, Eva Gläser, Audrey Iskandar, Neeltje van den Berg, Wolfgang Hoffmann, Bernhard Michalowsky","doi":"10.1007/s40120-025-00778-y","DOIUrl":"10.1007/s40120-025-00778-y","url":null,"abstract":"<p><strong>Introduction: </strong>Claims data typically lack clinical parameters such as dementia severity, limiting insights into disease progression and related healthcare utilization and costs. Although diagnoses, prescriptions, and utilization patterns may serve as proxies, their validity is unclear. This study aimed to identify and validate these parameters to distinguish early from severe dementia stages.</p><p><strong>Methods: </strong>Baseline data from 737 patients with dementia were analyzed. Dementia severity was assessed using the Mini-Mental State Examination and classified as early (≥ 27), mild (20-26), and moderate to severe (0-19). Healthcare utilization was recorded via structured interviews. Diagnoses, long-term care levels, and prescribed medications were extracted from physicians' files. Ordinal logistic regression evaluated associations between predictors and severity, with average marginal effects (AME) quantifying impact. Sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV) were computed for key predictors.</p><p><strong>Results: </strong>Among the sample (56% female patients, mean age 80), 18% were in the early stages, 43% mild, and 39% moderate to severe. Antipsychotic prescriptions (odds ratio (OR) 3.40, 95% confidence interval (CI) 1.94-5.95), antidementia drugs (OR 2.31, 95% CI 1.56-3.40), and higher long-term care levels (OR 5.59, 95% CI 2.23-13.99 for level ≥ 4) were associated with advanced severity. AME analysis revealed that antipsychotic use reduced early-stage probability by 14% and increased severe-stage probability by 21%. Similarly, antidementia drugs lowered early-stage probability by 9% and raised severe-stage probability by 13%. Increasing care levels were associated with a 2-16% decline in early-stage probability and a 3-34% rise in severe-stage probability. The combined model showed high specificity (99.6%) and PPV (84.6%) for severe dementia, but sensitivity and NPV for early stage were low.</p><p><strong>Conclusion: </strong>Antidementia drugs, antipsychotics, and long-term care level serve as robust predictors of moderate to severe dementia, whereas early-stage detection remains challenging. Future studies should validate these markers and explore additional predictors to improve early detection in claims data.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1589-1608"},"PeriodicalIF":3.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255591/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144275464","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Network Meta-analysis of Randomized Controlled Trials Assessing Neuromodulation Therapies for Painful Diabetic Neuropathy.","authors":"Li Li, Xueqin Luo, Yong Liu, Yongjie Jiang, Yankun Chen, Yangmei Chen, Jinping Wang","doi":"10.1007/s40120-025-00759-1","DOIUrl":"10.1007/s40120-025-00759-1","url":null,"abstract":"<p><strong>Introduction: </strong>Neuromodulation therapies (including non-invasive and invasive neuromodulation) are being used to treat painful diabetic neuropathy (PDN).</p><p><strong>Methods: </strong>A systematic search of the PubMed, Embase, Cochrane Library, Web of Science, and Scopus databases was conducted, from their inception until 1 October 2024, to identify randomized controlled trials (RCTs) on neuromodulation therapies for PDN. Data were collected on pain intensity of various adjunctive therapies for PDN, including transcutaneous electrical nerve stimulation (TENS), percutaneous electrical nerve stimulation, repetitive transcranial magnetic stimulation, pulsed electromagnetic field therapy, spinal cord stimulation (SCS), transcranial direct current stimulation, frequency rhythmic electrical modulation system, mesodiencephalic modulation, and sham.</p><p><strong>Results: </strong>The data from an aggregate of 12 separate studies, comprising a total sample size of 922 participants, was subject to analysis. All seven neuromodulation therapies exhibited better outcomes in pain intensity compared to the Sham intervention, with TENS achieving the highest ranking, followed by SCS. At the final follow-up time point, statistically significant reductions in pain intensity (vs. Sham) was only observed for SCS.</p><p><strong>Conclusion: </strong>The results of this network meta-analysis should facilitate the development of clinical guidance and enhance the decision-making process for both patients and healthcare professionals, thereby identifying the most appropriate PDN treatment options.</p><p><strong>Trial registration: </strong>PROSPERO: CRD42024597208.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1355-1382"},"PeriodicalIF":3.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255642/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036569","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Perampanel as Add-on in Patients Aged ≥ 12 Years with Focal Epilepsy: A Prospective Real-World Observational Study from Southern China.","authors":"Xiaoli Shi, Xiangru Lu, Lixia Li, Yanting Lu, Lang Shen, Jinou Zheng, Yuan Wu, Lu Yu","doi":"10.1007/s40120-025-00760-8","DOIUrl":"10.1007/s40120-025-00760-8","url":null,"abstract":"<p><strong>Introduction: </strong>This study aimed to evaluate the effectiveness, tolerability and safety of perampanel (PER) as an add-on therapy for southern Chinese patients aged ≥ 12 years with focal epilepsy.</p><p><strong>Methods: </strong>This prospective cohort study enrolled consecutive patients with focal epilepsy treated between January 2023 and January 2024. Patients received PER as add-on therapy, with medication adjustments, seizure frequency and adverse events (AEs) monitored at 3, 6, 9 and 12 months. Logistic regression analyzed factors influencing 6- and 12-month treatment outcomes.</p><p><strong>Results: </strong>Among 196 patients (full analysis set), 169 (86.2%) had drug-resistant epilepsy. Of these, 73.5% (144/196) received PER ≤ 4 mg/day. The 50% response rates at 6 and 12 months were 79.7% (114/143) and 86.0% (86/100), respectively. Retention rates at 6 and 12 months were 78.3% (148/189) and 59.3% (102/172), with cumulative retention rates of 71.8% and 63.3%, respectively. AEs occurred in 42 patients (21.4%), primarily dizziness and psychiatric symptoms. Logistic regression analysis identified disease duration of < 5 years (OR = 15.893, 95% CI = 1.418-178.158, P < 0.05) and unknown etiology (OR = 14.528, 95% CI = 2.508-84.140, P < 0.05) as predictors of higher long-term response rates.</p><p><strong>Conclusion: </strong>PER demonstrated good effectiveness and safety as an add-on therapy for focal epilepsy in southern Chinese patients aged ≥ 12 years. Lower doses of PER (≤ 4 mg/day) may achieve satisfactory effectiveness in PER-sensitive populations, while shorter disease duration and unknown etiology were associated with better long-term outcomes. These findings support PER's utility in managing focal epilepsy, particularly in drug-resistant cases.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1521-1537"},"PeriodicalIF":3.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255625/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144216372","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"What Patients Don't Say and Physicians Don't Ask: A Needs Assessment in Myasthenia Gravis Integrating Patient and Healthcare Professional Perspectives.","authors":"Cornelia Reyes Acosta, Ivo Vlaev, Alexis Rodriguez, Allison Foss, Ashwin Pinto, Channa Hewamadduma, John Vissing, Nicholas J Silvestri, Sophie Lehnerer, Marc De Backer, Natasha Monin, Sophie Barry, Ina Weisshardt","doi":"10.1007/s40120-025-00751-9","DOIUrl":"10.1007/s40120-025-00751-9","url":null,"abstract":"<p><strong>Introduction: </strong>Myasthenia gravis (MG) is a rare, autoimmune neuromuscular disease characterized by unpredictable fluctuating muscle weakness. This unpredictability makes effective patient-healthcare professional (HCP) dialogue essential for optimal diagnosis and management, with communication as a key component of shared decision-making (SDM). We designed a needs assessment to understand the differences between HCP and patient communication needs and perspectives on the impact of MG.</p><p><strong>Methods: </strong>A mixed-methods approach was utilized, comprising a survey and semi-structured interviews with HCPs and patients with MG. Quantitative data from the survey were extracted and analyzed to understand trends of knowledge, skills, and attitudes toward patient-HCP dialogue and SDM in MG. Interviews were transcribed and analyzed using principles of thematic analysis to identify perspectives on the impact of MG and challenges in communication.</p><p><strong>Results: </strong>Completed survey data were collected from 47 HCPs and 122 patients. There were discrepancies and areas of alignment in the priorities each group placed on knowledge, skills, and attitudes in MG management. Patients valued HCPs' listening skills and knowledge of treatment history, whereas HCPs prioritized knowing what matters to a patient with MG, including providing support to the patient and their family/carer. Both groups agreed on the necessity of a compassionate and informed approach to care. Interviews (10 HCPs; 10 patients) revealed key themes, including the multifaceted way in which symptoms impact patients' lives and challenges patients face while communicating their experiences.</p><p><strong>Conclusion: </strong>This needs assessment indicated general alignment between patients and HCPs on MG symptoms; however, notable disparities were found in relation to the perceived impact of these symptoms on patients' lives, and communication. While some HCPs expected patients to volunteer information on symptoms and MG-related challenges, patients expected their HCPs to ask. This highlights a need for improved communication strategies, which will foster SDM approaches.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1419-1438"},"PeriodicalIF":3.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255620/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144079195","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Persistence to Ocrelizumab Compared with Other Disease-Modifying Therapies for Multiple Sclerosis: Results from the German NeuroTransData Registry.","authors":"Stefan Braune, Petra Dirks, Seya Colloud, Qing Wang, Evan Davies, Yanic Heer, Mel Zürcher, Diana Sun, Arnfin Bergmann","doi":"10.1007/s40120-025-00762-6","DOIUrl":"10.1007/s40120-025-00762-6","url":null,"abstract":"<p><strong>Introduction: </strong>Treatment persistence is critical to obtaining full therapeutic benefit and can indicate favorable outcomes. This study examined real-world persistence with ocrelizumab (OCR) versus other disease-modifying therapies (DMTs) and its association with outcomes in relapsing-remitting multiple sclerosis (RRMS) using German NeuroTransData (NTD) registry data.</p><p><strong>Methods: </strong>This retrospective cohort analysis included outpatients with RRMS who initiated a DMT between January 2014 and April 2022. DMT initiation date was defined as the index date. DMTs were grouped into OCR, injectable, oral, oral for highly active disease (oral HA), and other intravenous (IV) therapies. Persistence, based on having continuous records of a DMT for 2 years from index date, was evaluated within each group. Association between persistence and the risk of relapse, 3-months confirmed disability progression (3mCDP), and sick leave were assessed.</p><p><strong>Results: </strong>Overall, 3907 patients with RRMS were included. OCR users had the highest persistence at 2 years (93%), then oral HA (78%), oral (67%), natalizumab (67%), and injectable therapies (55%). Compared with OCR users, patients initiating injectable (hazard ratio [HR] 8.51, 95% confidence interval [CI] 4.03-17.90), oral (HR 5.92, 95% CI 2.81-12.50), oral HA (HR 3.49, 95% CI 1.63-7.48) therapies, and natalizumab (HR 5.47, 95% CI 2.47-12.10) were more likely to discontinue. Adverse events (32.47%), lack of efficacy (21.17%), and patient-driven factors (19.73%) were the main reasons for discontinuation. Compared with persisters, non-persisters were associated with higher risks of relapse activity (rate ratio: 2.18, 95% CI 1.98-2.39), 3mCDP (rate ratio 1.52, 95% CI 1.28-1.77), and sick leave (rate ratio 1.71, 95% CI 1.49-1.98).</p><p><strong>Conclusion: </strong>In a German real-world setting, patients initiating OCR achieved higher rates of persistence over 2 years compared with those on other DMTs. High persistence was associated with lower risk of clinical disease activity, disease progression, and sick leave.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1695-1704"},"PeriodicalIF":3.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255630/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144199728","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Disease Course in Patients Switched from Natalizumab to Alemtuzumab: An Italian Multicenter, Prospective, Observational Study.","authors":"Simona Malucchi, Paola Perini, Francesca Rinaldi, Marta Radaelli, Maria Malentacchi, Antonio Bertolotto, Alessia Di Sapio","doi":"10.1007/s40120-025-00754-6","DOIUrl":"10.1007/s40120-025-00754-6","url":null,"abstract":"<p><strong>Introduction: </strong>Natalizumab is a highly efficacious therapy (HET) for patients with relapsing remitting multiple sclerosis (RRMS). Its prolonged use is limited by the risk of progressive multifocal leukoencephalopathy (PML) in patients positive for anti-JCV antibodies. Aims of this work were to evaluate clinical and radiological efficacy at 6 and 12 months after alemtuzumab infusion in patients switching from natalizumab and the safety of this exit strategy.</p><p><strong>Methods: </strong>This real-world, prospective, multicentric, observational study was conducted in three Italian MS centers and included a total of 35 patients with RRMS. Natalizumab treatment occurred from October 2010 to April 2021, whereas switch to alemtuzumab occurred from February 2018 to January 2023. Median washout period between the two drugs was 2 months. Patients underwent brain MRI before alemtuzumab start and then 6 and 12 months after the first alemtuzumab cycle.</p><p><strong>Results: </strong>No clinical relapse occurred during the washout period, nor between the first and second alemtuzumab infusion. Radiological activity was present in 4/35 (11%) and 2/35 (6%) patients, respectively, at 6 and 12 months after the first alemtuzumab administration. Expanded Disability Status Scale (EDSS) increase developed in 4/35 (11%) and 5/35 patients (14%), respectively, at 6 and 12 months. No PML occurred, nor any serious adverse event. For patients in center 1 (17 patients), follow-up continued for a median of 3.5 years; NEDA-3 (No Evidence of Disease Activity) was present in 14/17 patients (82%) at the end of follow-up. Autoimmunity occurred in 23% of patients.</p><p><strong>Conclusions: </strong>Alemtuzumab is a valid exit strategy after natalizumab interruption.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1451-1459"},"PeriodicalIF":3.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255628/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144111154","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pharmacokinetics of Atogepant in Healthy Lactating Female Participants: Results from a Phase 1 Lactation Study.","authors":"Ramesh R Boinpally, Jonathan H Smith, Rosa L De Abreu Ferreira, Joel M Trugman","doi":"10.1007/s40120-025-00772-4","DOIUrl":"10.1007/s40120-025-00772-4","url":null,"abstract":"<p><strong>Introduction: </strong>Atogepant is approved for the preventive treatment of migraine and is taken orally once daily. This work aimed to characterize the plasma and milk pharmacokinetics of atogepant in lactating females.</p><p><strong>Methods: </strong>An open-label, phase 1 study (NCT05892757) was conducted in 12 healthy, lactating adult women 1-6 months postpartum from July 11, 2023, to February 22, 2024. A single 60-mg dose of atogepant was administered to participants to determine atogepant's plasma and milk pharmacokinetics, the excretion of atogepant in breast milk, and the relative infant dose (RID). Atogepant was analyzed using validated LC-MS/MS assays in plasma and breast milk samples collected up to 24 h after dosing and during specified intervals through 24 h, respectively. Plasma and milk pharmacokinetic parameters were estimated using non-compartmental methods and compared using linear mixed-effects models. Safety was assessed via adverse event reporting, clinical labs, vital signs, and ECGs throughout the study.</p><p><strong>Results: </strong>The mean (range) milk-to-plasma ratio for atogepant was 0.076 (0.023-0.104). With nearly undetectable levels of atogepant in breast milk 16-24 h after dosing, the cumulative mean (range) amount of atogepant excreted in breast milk over 24 h was 0.009 mg (0.005-0.016 mg; 0.015% of 60-mg dose), and the mean (range) RID was 0.19% (0.06-0.33%). The mean plasma and milk peak concentrations of atogepant were 779 ng/mL and 57.0 ng/mL, respectively, and the corresponding AUC values were 3270 ng·h/mL and 238 ng·h/mL, respectively. Atogepant exposures in breast milk were 93% lower compared to plasma. Two participants (16.7%) experienced AEs. These included abdominal pain (n = 1) and dyspepsia (n = 1), both of which were non-serious and mild in severity. No new safety signals were identified in this small group of healthy lactating women.</p><p><strong>Conclusion: </strong>The cumulative mean amount of atogepant recovered in breast milk over 24 h following a 60-mg dose was 0.009 mg, with a RID of 0.19%. CLINICAL TRIAL REGISTRATION: NCT05892757; https://clinicaltrials.gov/study/NCT05892757 .</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1461-1473"},"PeriodicalIF":3.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255638/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144199737","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Spectrum of Acquired Demyelinating Syndromes in Children: A Tertiary Hospital Experience.","authors":"Mohammed Almuqbil, Lama Aljomah, Nora Almahmoud, Waleed Altuwaijri, Ahmad Alrumayyan, Muhammad T Alrifai","doi":"10.1007/s40120-025-00768-0","DOIUrl":"10.1007/s40120-025-00768-0","url":null,"abstract":"<p><strong>Introduction: </strong>Although acquired demyelinating syndromes (ADS) are rare in children, the incidence and prevalence of ADS vary internationally. As data on pediatric ADS in Saudi Arabia is limited, the aim of this study was to describe the clinical spectrum of pediatric ADS, its clinical characteristics, and management options at a tertiary hospital in Saudi Arabia.</p><p><strong>Methods: </strong>A retrospective observational study was conducted at King Abdulaziz Medical City (KAMC) and King Abdullah Specialized Children Hospital (KASCH) in Riyadh, Saudi Arabia between January 2016 and December 2022. All patients with ADS fulfilling criteria of each subtype (multiple sclerosis (MS), clinically isolated syndrome (CIS), acute disseminated encephalomyelitis (ADEM), and myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD)) were included in this study.</p><p><strong>Results: </strong>Forty-five pediatric patients with ADS were analyzed, with the majority diagnosed with MS. The median age of onset was higher in the MS group compared to monophasic CIS and MOGAD, with statistically significant differences in age at onset between the MS group and both the CIS and MOGAD groups (p = 0.0002). Significant differences were also observed in the type of initial central nervous system (CNS) attack, with optic neuritis being more common in MS and transverse myelitis in CIS (p < 0.0001). Laboratory results revealed a higher incidence of cerebrospinal fluid (CSF) oligoclonal bands in patients with MS, which was statistically significant (p =  0.04), and MOG antibodies were found in all patients with MOGAD. Intravenous pulse steroids were administered in most patients, while disease-modifying drugs (DMTs) were employed most frequently in patients with MS. The Expanded Disability Status Scale scores indicated little disability in most patients with MS and CIS, with more disability noted in a subgroup of ADEM. Overall, the study underscores the clinical heterogeneity of pediatric ADS and points out the statistically significant difference in age at onset, presenting features, and laboratory findings among ADS subtypes.</p><p><strong>Conclusions: </strong>This study provides a thorough overview of pediatric ADS, including important distinctions between MS, ADEM, CIS, and MOGAD. Marked differences in age at onset, presentation, and imaging among these subtypes are informative for maximizing diagnosis and treatment. The key findings are the subsequent development of MS from CIS and MOGAD, varying patterns of first attack, and imaging characteristics like callososeptal interface lesions in MS and posterior fossa hyperintensities in MOGAD. All these indicate the need for individualized diagnostic and therapeutic approaches for improved outcomes.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1495-1506"},"PeriodicalIF":3.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255633/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144216370","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Consequences of Cladribine Tablets for the Treatment of Highly Active Relapsing-Remitting Multiple Sclerosis in Italy.","authors":"Barbara Polistena, Anna Maria Provenzano, Caterina Rizzi, Elena Colombo, Roberto Bergamaschi","doi":"10.1007/s40120-025-00761-7","DOIUrl":"10.1007/s40120-025-00761-7","url":null,"abstract":"<p><strong>Introduction: </strong>Multiple sclerosis (MS) is a chronic autoimmune disease impacting the central nervous system. Individuals with highly active relapsing-remitting MS (HA-RRMS) frequently experience symptomatic episodes lasting more than 24 h, followed by a remission period completely or partially symptom free. This study aimed to assess the economic impact of cladribine tablets for the treatment of patients with HA-RRMS in the Italian context.</p><p><strong>Methods: </strong>A Markov model developed by PRECISIONheor for simulating treatment outcomes in patients with HA-RRMS aged over 18 years over three treatment lines was adapted to the Italian National Health System organization. The model considers direct costs of treatment and relapse management, as well as costs related to adverse events and indirect costs. The model adopts the societal perspective and a 4-year time horizon. Uncertainty was addressed through deterministic sensitivity analysis.</p><p><strong>Results: </strong>The analysis considered 16,691 out of 55,635 Italian patients with HA-RRMS (30%). Treatment with cladribine tablets extended the duration of patients' responsive stage (relapse free) from 1.3 months (worst scenario) to 1.4 months (best scenario) when compared to oral disease-modifying therapies (DMTs) and from 2.4 to 5.7 months when compared to monoclonal antibody DMTs. cladribine tablets were associated with cost savings ranging from 69.9% to 10.9% compared to oral DMTs and from 74.3% to 33.0% compared to monoclonal antibody DMTs. The cost savings per additional month in responsive stage ranged from € 27,663.2 to € 4154.3 vs oral DMTs and from € 16,224.5 to € 3035.3 vs monoclonal antibody DMTs.</p><p><strong>Conclusion: </strong>Cladribine tablets are associated with a benefit in terms of prolonging the non-relapsing phase in patients with HA-RRMS, as well as substantial direct cost savings in comparison with all other DMTs. Our analysis suggests that adopting cladribine tablets for HA-RRMS enables clinical benefits over a 4-year time horizon and savings for Italian society.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1507-1520"},"PeriodicalIF":3.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255608/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144216371","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}