Neurology and Therapy最新文献

{"title":"Treatment of Acute Ischemic Stroke with Intravenous Urokinase (TASK-UK): Protocol for a Multicenter, Prospective, Real-World Study.","authors":"Yongli Tao, Hui Fang, Lulu Pei, Yuan Gao, Kai Liu, Xin Wang, Rui Zhang, Zhentang Cao, Yifang Zhou, Ding Zhang, Yiwei Qian, Yuesong Pan, Yuming Xu, Bo Song","doi":"10.1007/s40120-026-01006-x","DOIUrl":"10.1007/s40120-026-01006-x","url":null,"abstract":"<p><strong>Background: </strong>Intravenous thrombolysis with recombinant tissue plasminogen activator (rt-PA) and urokinase (UK) are both recommended for acute ischemic stroke (AIS) in China. Compared with rt-PA, UK is less expensive and more widely available in clinical practice.</p><p><strong>Objective: </strong>The Treatment of Acute Ischemic Stroke with Intravenous Urokinase (TASK-UK) study aims to investigate clinical outcomes among patients with AIS who receive UK thrombolysis, and to compare efficacy and cost-effectiveness between UK and rt-PA.</p><p><strong>Methods: </strong>TASK-UK is a multicenter, real-world observational study, with a planned sample size of 1000 patients in the UK group and 800 in the rt-PA group. This study enrolls patients with AIS who are eligible for intravenous thrombolysis from 20 participating centers and conducts follow-up until 90 days after thrombolysis.</p><p><strong>Planned outcomes: </strong>The primary efficacy outcome is functional independence at 90 days, defined as a modified Rankin scale (mRS) score of 0-2. The primary safety outcome is symptomatic intracranial hemorrhage (sICH) occurring within 22-36 h after thrombolysis. The exploratory outcome is defined as an intergroup comparison of direct medical costs during hospitalization.</p><p><strong>Discussion: </strong>The TASK-UK study will provide high-quality real-world evidence on the effectiveness and cost-effectiveness of UK thrombolysis for AIS.</p><p><strong>Trial registry number: </strong>NCT06194968 (ClinicalTrials.gov).</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2823-2832"},"PeriodicalIF":4.7,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13615231/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148697611","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":"Current and Emerging Therapies for Pediatric Sleep Disorders: A Narrative Review.","authors":"Lilliana Estrada Chaverri, Lourdes M DelRosso, Fernando Alberto Ceballos Fuentes","doi":"10.1007/s40120-026-01002-1","DOIUrl":"10.1007/s40120-026-01002-1","url":null,"abstract":"<p><p>Sleep disorders in children represent a significant public health concern with important implications for neurodevelopment, behavior, and overall health. Among these, pediatric obstructive sleep apnea (OSA), insomnia, narcolepsy, and sleep-related movement disorders are the most clinically relevant conditions requiring targeted therapeutic strategies. Pediatric OSA, affecting approximately 2-5% of children, is characterized by recurrent upper airway obstruction during sleep and is associated with cognitive, behavioral, and cardiovascular consequences. Management is multimodal, with adenotonsillectomy as first-line therapy, which improves most patients, although residual disease remains common. Adjunctive treatments include anti-inflammatory therapies such as intranasal corticosteroids and leukotriene receptor antagonists, positive airway pressure therapy, and emerging approaches such as high-flow nasal cannula, drug-induced sleep endoscopy-guided surgery, orthodontic interventions, and hypoglossal nerve stimulation in selected populations. Insomnia in children is primarily managed with behavioral interventions, particularly cognitive behavioral therapy for insomnia (CBT-I) and parental education strategies. Melatonin has demonstrated modest efficacy in reducing sleep latency, especially in children with neurodevelopmental disorders, although long-term safety data remain limited. Narcolepsy requires a combination of behavioral and pharmacological management, with scheduled naps as first-line therapy and medications such as stimulants, sodium oxybate, and pitolisant targeting excessive daytime sleepiness and cataplexy. Sleep-related movement disorders, including restless legs syndrome and periodic limb movement disorder, are frequently associated with iron deficiency and are primarily treated with iron supplementation alongside behavioral strategies. Overall, the treatment of pediatric sleep disorders requires a multidisciplinary, individualized approach that integrates behavioral, pharmacologic, and interventional therapies. Advances in precision medicine, device-based treatments, and targeted interventions are expanding the therapeutic landscape, emphasizing the importance of early recognition and tailored management to improve long-term outcomes in children.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2321-2339"},"PeriodicalIF":4.7,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13615275/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148621014","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":"Impact of Oral Cladribine on TSPO-PET-Measurable Immune Cell Activation in Multiple Sclerosis.","authors":"Olli Rajalehto, Markus Matilainen, Jussi Lehto, Eveliina Honkonen, Mikko Koivumäki, Saara Wahlroos, Johan Rajander, Olavi Misin, Amelie Luoma, Sini Laaksonen, Anna Vuorimaa, Marcus Sucksdorff, Maija Saraste, Laura Airas","doi":"10.1007/s40120-026-01044-5","DOIUrl":"https://doi.org/10.1007/s40120-026-01044-5","url":null,"abstract":"<p><strong>Introduction: </strong>Oral cladribine reduces focal inflammatory activity in relapsing-remitting multiple sclerosis (RRMS). Preclinical studies suggest it may also modulate microglial activation. In this prospective, single-arm pilot study, we investigated the effect of oral cladribine on glial activation using 18 kDa translocator protein (TSPO) positron emission tomography (PET) in a real-world, longitudinal RRMS cohort.</p><p><strong>Methods: </strong>People with RRMS initiating oral cladribine underwent TSPO-PET with [¹¹C]PK11195, 3-T brain magnetic resonance imaging (MRI), serum neurofilament light chain (sNfL) and glial fibrillary acidic protein (sGFAP) measurements, and clinical assessments at baseline and 18 months. Age- and sex-matched healthy controls underwent a single PET and MRI. The primary outcome was change in TSPO availability quantified as a percentage of TSPO-active voxels and distribution volume ratio. Secondary outcomes included brain volumetrics, paramagnetic rim lesions, serum biomarkers, clinical measures, and patient-reported outcomes.</p><p><strong>Results: </strong>Fourteen people with RRMS and 14 healthy controls were enrolled. Baseline TSPO availability did not differ between groups. Over 18 months, TSPO availability quantified as percentage of TSPO-active voxels increased 21% in normal-appearing white matter (NAWM) (mean [95% CI] change 1.2 [0.2-2.3]; p = 0.026) and 16% in the whole brain (mean [95% CI] change 0.9 [0.01-1.8]; p = 0.047). Both the median (1st to 3rd quartile) annualized relapse rate (from 0.67 [0.17-0.67] to 0.00 [0.00-0.00]; p = 0.033) and the age- and BMI-adjusted sNfL Z-score (from 0.69 [0.16-1.22] to - 0.03 [- 0.39 to 0.21]; p = 0.038) decreased significantly.</p><p><strong>Conclusion: </strong>TSPO-binding in the NAWM increased during oral cladribine treatment despite suppressed relapses and decreased sNfL Z-score. These exploratory findings raise the possibility that cladribine may have limited ability to control smoldering pathology. This possibility warrants further investigation in larger prospective studies.</p><p><strong>Trial registration: </strong>This study was registered on ClinicalTrials.gov (NCT04239820) on January 21, 2020.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149112101","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}

{"title":"Perspectives of Adult Patients and Caregivers on the Management and Challenges Associated with Neurofibromatosis Type 1 and Plexiform Neurofibromas: A Multinational Qualitative Survey.","authors":"Ayo Adeyemi, Susanna Burckhardt, Riccardo Buttarelli, Michelle Erdmann, Rosalie Ferner, Brendon Pommier, Geting Su, Hui Zhang, Abby Crites","doi":"10.1007/s40120-026-01018-7","DOIUrl":"https://doi.org/10.1007/s40120-026-01018-7","url":null,"abstract":"<p><strong>Introduction: </strong>Adults with plexiform neurofibromas arising from neurofibromatosis type 1 (NF1-PN) experience a multifaceted range of clinical symptoms and manifestations, which can impact multiple organ systems and reduce quality of life (QoL). This qualitative study aimed to better understand the experiences of adults and caregivers of adults with NF1-PN and their perspectives on treatment, challenges, and unmet needs across several geographic regions.</p><p><strong>Methods: </strong>Web-assisted telephone interviews were conducted between January 31, 2024, and April 15, 2024, with adults who had a diagnosis of NF1-PN or with caregivers, focusing on diagnosis, interactions with healthcare professionals (HCPs), transition of care, pain management, and unmet needs. Participants were recruited through HCP referrals, patient organizations, or patient databases.</p><p><strong>Results: </strong>The study included 65 patients with NF1-PN and 14 caregivers in Canada, China, France, Germany, Italy, Japan, Spain, and the UK. Patients reported living with multiple NF1-PN-associated morbidities, including pain, disfigurement, psychiatric disorders, and chronic migraines. NF1-PN impacted daily living from childhood to adulthood and affected relationships and mental and emotional health. Surgery (~50%) and pain medications (~40%) were the most common treatments; nearly all participants (~90%) mentioned the need for disease-modifying treatments. Most patients (~85%) diagnosed as children transitioned to adult care, although there was variability in the transition experience. Some patients (~10%) dropped off during transition; others (~10%) experienced difficulty adjusting to adult specialists.</p><p><strong>Conclusions: </strong>The real-life perspectives of adult patients and caregivers highlighted that NF1-PN has a profoundly negative impact on QoL and mental health. At the time of the study, the only available treatments for this patient population were surgery or medications for symptom management. Participants identified unmet needs, such as a desire for improved treatment options that halt disease progression and reduce pain while being noninvasive, convenient, and accessible. Improved processes for the transition to adult care are needed.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149112173","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}

{"title":"From Biomarker Anchors to Disease-State Transitions: A Biologically Anchored Probabilistic Principal Component Analysis Framework for Alzheimer's Disease Progression Modeling.","authors":"Babak Haji, Amir Abbas Tahami Monfared","doi":"10.1007/s40120-026-01036-5","DOIUrl":"https://doi.org/10.1007/s40120-026-01036-5","url":null,"abstract":"<p><strong>Introduction: </strong>To develop and validate a biomarker-anchored probabilistic principal component analysis (PPCA) framework for identifying biologically interpretable latent dimensions of Alzheimer's disease (AD) and evaluating their utility for disease progression modeling.</p><p><strong>Methods: </strong>Data from 1058 participants that are amyloid-positive in the Alzheimer's Disease Neuroimaging Initiative (ADNI) were analyzed. Anchored PPCA used biomarker data only; cognitive, functional, diagnostic, and prognostic outcomes were withheld from latent-space construction and reserved for validation. Amyloid (A) and tau (T) factors were biologically anchored, neurodegeneration (N) was softly constrained, and a ventricular-vascular/residual (V/R) factor was empirically estimated. Robustness, reproducibility, held-out validity, clinical associations, prognostic performance, and clinical-state transitions were evaluated.</p><p><strong>Results: </strong>Anchored PPCA identified four biologically coherent dimensions: A, T, N, and V/R. Solutions were reproducible across repeated initializations and split-half analyses, and generalized to held-out participants, particularly for A, T, and N. The model explained approximately 60% of model-implied standardized biomarker variance, with highest explained variance for A and T and lowest for V/R. Latent factors explained variance in outcomes not used for model construction: 49.7% for ADAS-Cog13, 35.1% for CDR-SB, and 27.8% for FAQ; they also aligned with diagnostic classifications. Cox-model C-indices were 0.843 for latent factors alone and 0.933 for clinical-plus-latent factors, with better fit than the clinical benchmark. T showed the strongest prognostic association, followed by A, N, and V/R. After adjustment for baseline clinical severity, A, T, and N remained independently prognostic; V/R did not. Clinical-state transitions were predominantly monotonic and demonstrated marked sojourn-time dependence.</p><p><strong>Conclusion: </strong>Biomarker-only anchored PPCA provides a biologically grounded representation of AD that validates against cognition, function, diagnosis, and clinical progression. A, T, and N may capture disease processes not fully reflected in cross-sectional clinical severity, whereas V/R appears more closely related to contemporaneous clinical status. Duration-dependent transitions support semi-Markov disease-progression and Shared Latent Disease Process models. External validation is warranted.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149075334","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}

{"title":"Correction: Patient Experience with 10 mg Intranasally Administered Zavegepant as an Acute Treatment of Migraine Attacks: Qualitative Research Findings.","authors":"Noah Rosen, Heather Moran, Josh Coulter, Alexandra Kissling, William You, Lewis Kopenhafer, Kathleen Beusterien, Jason Isaacs, Karina Nakajima, Jessica Cirillo, Samantha Sweeney","doi":"10.1007/s40120-026-01034-7","DOIUrl":"https://doi.org/10.1007/s40120-026-01034-7","url":null,"abstract":"","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149063216","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}

{"title":"Maintenance of Minimal Manifestation Status with Individualized-Interval Efgartigimod in Generalized Myasthenia Gravis: Study Protocol for a Single-Arm, Open-Label Clinical Trial.","authors":"Tetsu Suzuki, Motoki Fujimaki, Kota Igari, Shogo Ohuchi, Izumi Mihashi, Takashi Hosaka, Ayako Shioya, Makoto Terada, Shinji Saiki","doi":"10.1007/s40120-026-01031-w","DOIUrl":"https://doi.org/10.1007/s40120-026-01031-w","url":null,"abstract":"<p><strong>Introduction: </strong>Efgartigimod, a human immunoglobulin G1 Fc fragment targeting the neonatal Fc receptor, improves symptoms in generalized myasthenia gravis (gMG). It remains unknown whether minimal manifestation (MM) status achieved with efgartigimod can be durably maintained over the long term, and whether subcutaneous (SC) self-administration reduces treatment burden and supports quality of life. We designed a prospective study to evaluate maintenance of MM with low-dose oral prednisolone (PSL) ≤ 5 mg/day (MM-5 mg) under interval-adjusted efgartigimod administration.</p><p><strong>Methods: </strong>This single-arm, open-label, single-center exploratory study will enroll 24 adults of either sex (aged 18 to < 80 years) with gMG who have achieved MM, defined as a Myasthenia Gravis Activities of Daily Living (MG-ADL) score ≤ 2 or MG Composite score ≤ 4, on efgartigimod with oral PSL ≤ 5 mg/day (commonly with a concomitant calcineurin inhibitor), in a preceding trial or routine care. Efgartigimod (intravenous efgartigimod alfa or SC efgartigimod alfa/vorhyaluronidase alfa) is given once weekly for 4 weeks per cycle. The intercycle interval is individualized from the worst weekly MG-ADL score: shortened by 1 week upon worsening, extended by 1 week after two stable cycles. PSL and permitted background oral immunosuppressants are held constant. Treatment lasts up to 55 weeks, with a 4-week post-observation period.</p><p><strong>Planned outcomes: </strong>The primary endpoint is the proportion of patients maintaining MM according to prespecified cycle-level criteria throughout the observation period while receiving PSL ≤ 5 mg/day. Secondary endpoints include changes in MG-ADL, MG Composite, the revised 15-item Myasthenia Gravis Quality of Life questionnaire, Japanese version (MG-QOL15r-J), and Quantitative MG (QMG) scores; patient-reported treatment convenience; change in serum immunoglobulin G (IgG); the intercycle-interval distribution; autoantibody titers; and the time from enrollment to protocol discontinuation due to difficulty maintaining MM-5 mg. Safety is also assessed. Graphical Abstract available for this article.</p><p><strong>Clinical trial registration: </strong>Japan Registry of Clinical Trials (jRCT): jRCTs031260127 (prospectively registered on 14 May 2026).</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149039993","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}

{"title":"Comparative Efficacy of Rozanolixizumab with Efgartigimod or Intravenous Immunoglobulin in Generalized Myasthenia Gravis Using Matching Adjusted Indirect Comparisons.","authors":"Vikalp Maheshwari, Carolina Barnett Tapia, Abhiroop Chakravarty, Jitender Takyar, Arju Dhawan, Saiju Jacob, April Betts","doi":"10.1007/s40120-026-01020-z","DOIUrl":"https://doi.org/10.1007/s40120-026-01020-z","url":null,"abstract":"<p><strong>Introduction: </strong>Generalized myasthenia gravis (gMG) is a rare autoimmune disorder affecting neuromuscular transmission. New targeted treatments including FcRn antagonists have emerged, but direct comparative evidence is lacking. This study aimed to compare the efficacy of rozanolixizumab with efgartigimod or intravenous immunoglobulin (IVIg) using matching-adjusted indirect comparisons (MAICs).</p><p><strong>Methods: </strong>Phase 3 studies of rozanolixizumab and efgartigimod were identified through a literature review. MAICs compared rozanolixizumab (7 mg/kg and 10 mg/kg) with efgartigimod 10 mg/kg IV and IVIg. Anchored comparisons (rozanolixizumab vs. efgartigimod) and unanchored comparisons (rozanolixizumab vs. IVIg) were used. Propensity score weighting balanced covariates between trial populations. Continuous outcomes were analysed using linear regression, and logistic regression for dichotomous outcomes. Treatment effects were presented as mean differences or odds ratios with 95% confidence intervals (CI). Scenario analyses were performed.</p><p><strong>Results: </strong>Rozanolixizumab efficacy was comparable to efgartigimod except for Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Composite (MGC) scores at 6 weeks which were significantly in favour of rozanolixizumab 10 mg/kg (QMG mean difference - 2.80; 95% CI - 5.00, - 0.60; MGC mean difference - 3.89; - 6.72, - 1.05). Both rozanolixizumab doses significantly improved QMG scores from baseline at 2 weeks vs. IVIg (7 mg/kg: - 1.95; - 3.79, - 0.11; 10 mg/kg: - 1.99; - 3.71, - 0.26) and 4 weeks (7 mg/kg: - 3.40; - 5.23, - 1.56; 10 mg/kg: - 3.99; - 5.81, - 2.17). QMG responders at 2 weeks were not significantly different for rozanolixizumab vs. IVIg.</p><p><strong>Conclusions: </strong>Without direct comparative evidence, these analyses provide additional context to inform treatment decisions for the management of gMG. These analyses suggest that FcRn inhibitors represent important targeted treatment options for gMG.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149039996","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}

{"title":"Comparative Efficacy of Zilucoplan Versus Intravenous Immunoglobulin, Eculizumab, and Ravulizumab Using Matching-Adjusted Indirect Comparisons.","authors":"April Betts, Saiju Jacob, Abhiroop Chakravarty, Jitender Takyar, Arju Dhawan, Carolina Barnett Tapia, Vikalp Maheshwari","doi":"10.1007/s40120-026-01021-y","DOIUrl":"https://doi.org/10.1007/s40120-026-01021-y","url":null,"abstract":"<p><strong>Introduction: </strong>Generalized myasthenia gravis (gMG) is a rare, chronic autoimmune disorder which impacts signaling at the neuromuscular junction. While new targeted therapies have emerged, there is no direct comparative evidence. This study aimed to compare the efficacy of zilucoplan with eculizumab, intravenous immunoglobulin (IVIg), and ravulizumab using matching-adjusted indirect comparisons (MAICs).</p><p><strong>Methods: </strong>Phase 3 studies of complement component 5 (C5) inhibitors and IVIg were identified through a literature review. MAICs were conducted for zilucoplan versus IVIg, zilucoplan versus ravulizumab (both comparisons in patients with gMG), and zilucoplan versus eculizumab (in patients with refractory gMG). Propensity score-based weighting methods were used to balance covariates between trial populations. Continuous outcomes were analyzed through linear regression, and dichotomous outcomes were analyzed through logistic regression. Treatment effects were presented as mean differences or odds ratios (ORs) with 95% confidence intervals (CIs). Scenario analyses were performed.</p><p><strong>Results: </strong>Compared with IVIg, zilucoplan was associated with statistically significant improvements in the Quantitative Myasthenia Gravis score (QMG) at 2 weeks (OR: - 1.95; 95% CI: - 3.50, - 0.39) and 4 weeks (OR: - 3.30; 95% CI: - 4.89, - 1.71). Compared with eculizumab at 24/26 weeks, the MAIC analysis showed statistically significant differences favoring zilucoplan for the MG Activities of Daily Living (MG-ADL) score (OR: - 1.99; 95% CI: - 3.30, - 0.69), the Quantitative Myasthenia Gravis (QMG) score (OR: - 3.23; 95% CI: - 4.63, - 1.82), and the Myasthenia Gravis Composite questionnaire (MGC) (OR: - 4.27; 95% CI: - 6.79, - 1.76) in patients with refractory gMG. Compared with ravulizumab at 24/26 weeks, the MAIC analysis showed statistically significant differences favoring zilucoplan for the MG-ADL (OR: - 2.70; 95% CI: - 3.74, - 1.67), QMG (OR: - 5.72; 95% CI: - 7.02, - 4.43), and the Myasthenia Gravis Quality of Life 15-item revised scale (MG-QoL-15r) (OR: - 5.51; 95% CI - 7.63, - 3.38) scores.</p><p><strong>Conclusions: </strong>In this analysis, zilucoplan was associated with a significantly higher magnitude of clinically meaningful improvement in QMG and MG-ADL scores compared to IVIg and two C5 inhibitors, eculizumab and ravulizumab. In the absence of head-to-head clinical data, these findings provide additional evidence to assist with treatment decisions in this setting.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149040039","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}

{"title":"The Role of Neurologists in the Management of Uncontrolled Epilepsy in Spain: Opportunities for Optimization.","authors":"David García-Azorín, Luis Lobato, Javier Riancho, Juan J Poza, Ángel Aledo-Serrano","doi":"10.1007/s40120-026-01039-2","DOIUrl":"https://doi.org/10.1007/s40120-026-01039-2","url":null,"abstract":"<p><p>Epilepsy is a major societal burden with a significant impact on patients' health and quality of life. The primary goal in epilepsy management is to achieve freedom from all types of seizures. Early treatment with antiseizure medications plays a crucial role in this regard, preventing seizure recurrence, limiting disease progression and comorbidities, and improving both prognosis and quality of life. However, initial treatment is typically managed by non-epilepsy specialists, and there are considerable gaps in care that need to be addressed. In order to support non-epilepsy experts in the initial management of uncontrolled epilepsy, we report here on the most significant challenges and obstacles encountered by non-epilepsy specialists in their daily clinical routine, based on literature evidence and outcomes from our recent survey of Spanish neurologists, and provide recommendations to improve patient care.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149025626","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}