Neurology and Therapy最新文献

{"title":"From Headache to Heart Health: Investigating the Migraine-Cardiovascular Disease Connection.","authors":"Claudio Tana, Dilara Onan, Roberta Messina, Marta Waliszewska-Prosół, David Garcia-Azorin, Luis Leal-Vega, Maria Begoña Coco-Martin, Raffaele Ornello, Bianca Raffaelli, Marcio Nattan Portes Souza, William Wells-Gatnik, Paolo Martelletti","doi":"10.1007/s40120-025-00785-z","DOIUrl":"https://doi.org/10.1007/s40120-025-00785-z","url":null,"abstract":"<p><p>Migraine, particularly with aura, has been consistently associated with an increased risk of cardiovascular disease, including ischemic stroke and myocardial infarction. Shared pathophysiological mechanisms such as endothelial dysfunction, platelet aggregation, systemic inflammation, and autonomic imbalance suggest that migraine may act as an early clinical marker of systemic vascular vulnerability. Psychiatric comorbidities, frequently present in chronic migraine, further compound disability and may contribute to long-term cardiovascular risk. This narrative review discusses the evolving understanding of migraine as a multisystem disorder, emphasizing its vascular and neuropsychiatric dimensions. Emerging data on calcitonin gene-related peptide (CGRP)-targeting monoclonal antibodies highlight their efficacy not only in reducing headache burden but also their favorable cardiovascular safety profile. Moreover, preliminary evidence suggests these agents may have a positive effect on mood symptoms in patients with comorbid depression and anxiety. Recognizing migraine as a condition that intersects neurological, cardiovascular, and psychiatric pathways may support earlier risk stratification and guide integrated treatment approaches in complex patient populations.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144333612","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":"Cenobamate for Adjunctive Treatment in Adult and Pediatric Patients with Refractory Lennox-Gastaut Syndrome: A Retrospective Chart Review.","authors":"Karen Keough, Alec Romick","doi":"10.1007/s40120-025-00779-x","DOIUrl":"https://doi.org/10.1007/s40120-025-00779-x","url":null,"abstract":"<p><strong>Introduction: </strong>Lennox-Gastaut syndrome (LGS) is a particularly severe developmental epileptic encephalopathy (DEE) characterized by multiple types of drug-resistant, incapacitating seizures. Despite aggressive therapy including polypharmacy, surgery, implanted devices, and dietary therapy, the prognosis remains poor, with frequent ongoing seizures and risk of injury and early death. Cenobamate (CNB) is an antiseizure medication (ASM) approved for the treatment of focal seizures in adults, but real-world experience in patients with DEEs has shown promising reductions in seizure frequency.</p><p><strong>Methods: </strong>This retrospective chart review determined the effectiveness and tolerability of CNB in 36 adult and pediatric patients with LGS under the treatment of one physician.</p><p><strong>Results: </strong>Among 36 patients (69% male, median age 15.5 years) with LGS, 86% experienced a reduction in seizure frequency after the addition of CNB (median treatment duration 23 months), including ≥ 75% reduction in 22 patients (61%) and seizure freedom in 5 patients (14%). A substantial proportion of patients (75%, n = 27) successfully reduced their concomitant medications, including the lowering or discontinuation of cannabidiol in 19 patients and clobazam in 21 patients. Adverse events were reported in two-thirds of patients, reflecting the same symptoms reported in the original approval trials, with somnolence being the most common.</p><p><strong>Conclusions: </strong>This chart review provides promising evidence for the efficacy of CNB in treating LGS. Additional prospective studies will help to clarify CNB's efficacy and safety profile for patients with LGS.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144326351","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":"Impact of Early Antipsychotic Prescription Choice on Weight Gain in the First 5 Years of Psychotic Illness: a Retrospective Cohort Study.","authors":"Adrian Heald, George Tilston, John Julian Warner-Levy, Loren Wilkins, Richard Williams, Toby Pillinger, William Deakin, Damien Longson, Lamiece Hassan, Caroline Dalton, Gavin P Reynolds, Joseph Firth","doi":"10.1007/s40120-025-00780-4","DOIUrl":"https://doi.org/10.1007/s40120-025-00780-4","url":null,"abstract":"<p><strong>Introduction: </strong>We analysed the effects of antipsychotic drug prescribing in year 1 of treatment for psychosis on future weight gain over 5 years.</p><p><strong>Methods: </strong>We studied how weight changed over 5 years after the first diagnosis of psychosis/schizophrenia/schizoaffective disorder/delusional disorder/affective psychosis in 17,570 individuals and investigated its association with antipsychotic drug treatments prescribed in year 1 following diagnosis, over 30 years.</p><p><strong>Results: </strong>The majority (65%) were aged 20-59 years at the time of first antipsychotic prescription. Mean baseline body-mass-Index (BMI) was similar in women versus men. Substantial increases in BMI were observed, with the greatest categorical changes seen in the obese (BMI ≥ 30 kg/m²) subjects, increasing from 30 to 43% for women and from 26% to 39% for men, while 42% of people did not significantly increase their weight. Individuals prescribed perphenazine/fluphenazine/amisulpride were most likely to remain at normal-BMI, while individuals prescribed aripiprazole/quetiapine/olanzapine/risperidone in the first year were most likely to gain weight/transition to overweight (25.0-29.9 kg/m²)/obese (≥ 30.0 kg/m²) from a normal BMI. The 'typical' agents thioridazine/chlorpromazine/flupenthixol/trifluoperazine/haloperidol were associated with an intermediate likelihood of BMI category change. In multivariate linear regression, factors associated with weight-gain were younger age/female sex(both p < 0.001), number of antipsychotic agents prescribed in 1st year (p < 0.001), plus specific agents aripiprazole (including 75% co-prescription or as 2nd line/3rd line)/olanzapine/thioridazine (p < 0.001), risperidone/quetiapine (p < 0.05). In multivariate logistic regression (weight increase ≥ 7%), the specific medication factors were similar, with odds ratios(OR) for specific medications ranging from quetiapine 1.09 (CI 1.00-1.21) to thioridazine 1.45 (CI 1.20-1.74).</p><p><strong>Conclusion: </strong>Younger women were at elevated risk for weight gain as were people prescribed multiple antipsychotics in the 1st year. Some older antipsychotics associated with as much weight gain as the newer prescribed agents. More than 40% of people did not put on weight.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144326352","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 Effectiveness of Fumarates Versus Sphingosine-1-Phosphate Receptor Modulators in Black Patients with Multiple Sclerosis.","authors":"Sophia Woodson, Edward J Gettings, Chu-Yueh Guo, Sylvia Klineova, Jong-Mi Lee, Rebecca S Romero, Aljoeson Walker, Nicholas Belviso, Sai L Shankar, Jason P Mendoza, Boyang Bian, James B Lewin, Kinyee Fong","doi":"10.1007/s40120-025-00774-2","DOIUrl":"https://doi.org/10.1007/s40120-025-00774-2","url":null,"abstract":"<p><strong>Introduction: </strong>Multiple sclerosis (MS) is a heterogeneous disease that disproportionately impacts Black people with MS (PwMS), who experience more severe disease and higher relapse rates compared with non-Black populations. Despite widespread use of fumarates and sphingosine-1-phosphate (S1P) receptor modulators as oral disease-modifying therapies (DMTs) for relapsing MS, their comparative effectiveness in Black PwMS has not been studied. This study aims to help address this gap using real-world claims data.</p><p><strong>Methods: </strong>This retrospective analysis using the Komodo Health Claims Database included Black PwMS. Patients were aged 18-64 years with ≥ 1 claim for MS diagnosis (International Classification of Diseases, Tenth Revision, Clinical Modification code G35) and ≥ 1 prescription claim for fumarates (dimethyl fumarate or diroximel fumarate) or an S1P receptor modulator (fingolimod, siponimod, ozanimod, or ponesimod) between January 2017 and April 2023. Outcomes included annualized relapse rate (ARR) and time to first relapse. Propensity score matching (2:1) and inverse probability weighting were used to balance baseline characteristics. Relapse events were identified using a claims-based algorithm.</p><p><strong>Results: </strong>The analysis included 1664 Black PwMS (1231 and 433 in fumarate and S1P treatment arms, respectively). Post-index ARRs were comparable between groups (rate ratio [RR] 1.18, p = 0.423). Kaplan-Meier analyses showed similar relapse-free proportions at 24 months (72.6% and 74.7% in fumerate and S1P populations, respectively; p = 0.152). These findings were consistent in both the propensity score-matched and inverse probability weighted populations.</p><p><strong>Conclusions: </strong>This real-world, claims-based analysis demonstrates that fumarates and S1P receptor modulators have similar effectiveness in reducing relapses among Black PwMS, with > 72% of patients in both treatment groups remaining relapse-free at 24 months. Given the underrepresentation of Black patients in MS clinical trials, these results provide valuable real-world evidence to guide treatment decisions for this population.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144302589","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":"Development and Validation of PARCOMS Composite Scales for Assessing Disease Progression and Treatment Effects in Parkinson's Disease.","authors":"Gil L'Italien, Basia Rogula, Lauren Powell, Michele Potashman, Samuel P Dickson, Nick Kozauer, Patrick O'Keefe, Ellen Korol, Madeleine Crabtree, Fernanda Nagase, Vlad Coric, Jordan Dubow, Liana S Rosenthal, Suzanne Hendrix","doi":"10.1007/s40120-025-00771-5","DOIUrl":"https://doi.org/10.1007/s40120-025-00771-5","url":null,"abstract":"<p><strong>Introduction: </strong>Measures designed to comprehensively assess Parkinson's disease (PD) irrespective of disease stage and treatment status may be unable to capture nuances in disease progression, particularly in early-stage PD. The objective of this paper is to develop PARkinson's COMposite Scales (PARCOMS) with increased responsiveness to clinical decline using items of the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) for three discrete cohorts of patients.</p><p><strong>Methods: </strong>Patients with confirmed PD from the Parkinson's Progression Markers Initiative (PPMI) data were assigned to three cohorts based on use of dopaminergic treatment, stage of disease, and presence of motor complication. For each cohort, items from MDS-UPDRS Part I (PARCOMS-Non-Motor) and Parts II and III (PARCOMS-Motor) were selected based on responsiveness using partial least squares (PLS) regression. The responsiveness of the scales was estimated using mean-to-standard deviation ratios (MSDRs) of their change values.</p><p><strong>Results: </strong>Compared to the original MDS-UPDRS, MSDRs for PARCOMS-Motor increased 13.1% (untreated cohort, n = 430), 78.2% (treated-without-motor-complications cohort, n = 426), and 100.6% (treated-with-motor-complications cohort, n = 538). The MSDR increases observed for PARCOMS-Non-Motor were 13.9%, 6.8%, and 20.7%, respectively. Across cohorts, turning in bed and speech items were large contributors to the PARCOMS-Motor scales. Items for cognitive impairment and urinary problems were substantial contributors to PARCOMS-Non-Motor across cohorts. There was variability in the weighting of items representing different clinical concepts across cohorts for each composite, confirming heterogeneity in disease progression across disease stages.</p><p><strong>Conclusions: </strong>PD stage-specific composite measures were developed and demonstrated greater sensitivity to change than the original MDS-UPDRS, supporting the value of weighted composites tailored for disease stage.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144302590","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":"Real-World Treatment Outcomes in Black, Hispanic, Asian, and White People with Multiple Sclerosis Treated with Fumarates in the USA.","authors":"Sophia Woodson, Edward J Gettings, Chu-Yueh Guo, Sylvia Klineova, Jong-Mi Lee, Rebecca S Romero, Aljoeson Walker, Kinyee Fong, Jason P Mendoza, Nicholas Belviso, Boyang Bian, James B Lewin, Sai L Shankar","doi":"10.1007/s40120-025-00773-3","DOIUrl":"https://doi.org/10.1007/s40120-025-00773-3","url":null,"abstract":"<p><strong>Introduction: </strong>Multiple sclerosis (MS) is a heterogeneous disease affecting a diverse population. Compared with white people with MS (PwMS), Black PwMS have more severe disease and higher incidence of MS, whereas Hispanic PwMS experience earlier onset disease; however, MS is not adequately studied in these groups. We compared the effectiveness of fumarates across Black, Hispanic, Asian, and white PwMS.</p><p><strong>Methods: </strong>This retrospective analysis using the Komodo Health database included PwMS. Patients with a claim for diroximel fumarate or dimethyl fumarate were followed from disease-modifying therapy (DMT) initiation to loss of follow-up or discontinuation. Outcomes included annualized relapse rate (ARR), time to post-index first relapse, healthcare resource use (HRU), healthcare costs (HCCs), and change in absolute lymphocyte counts (ALCs). Race/ethnicity was self-reported.</p><p><strong>Results: </strong>This study included 6800 PwMS (Black, n = 1241; Hispanic, n = 777; Asian, n = 132; white, n = 4650). The average exposure duration of fumarates was 449-559 days. Black PwMS had higher baseline disease burden versus white PwMS, were less likely to have commercial insurance plans, and were more likely to reside in a state with a higher poverty rate. ARRs (12-month pre-index to post-index) were significantly reduced across groups. The Kaplan-Meier estimated proportion of relapse-free patients at 2 years was similar across groups (Black, 77.0%; Hispanic, 75.4%; Asian, 81.7%; white, 80.5%). There was a smaller decline in ALC from month 0 to month 12 in Black PwMS versus other racial/ethnic groups.</p><p><strong>Conclusion: </strong>Consistent with prior studies, these results demonstrate the effectiveness of fumarates across racial and ethnic MS subgroups. This is the largest analysis to date of the treatment effects of any individual class of DMT in Black and Hispanic PwMS. Infographic available for this article.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144302591","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":"Patient Experience of Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease: Qualitative Patient and Clinician Interviews Informing the Development of a Conceptual Model.","authors":"Jeffrey L Bennett, Asha Paireddy, Charlotte Cox, Megan Mayhew, Julia Stein, Teresa Gasalla, Béatrice Tugaut","doi":"10.1007/s40120-025-00770-6","DOIUrl":"https://doi.org/10.1007/s40120-025-00770-6","url":null,"abstract":"<p><strong>Introduction: </strong>Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is a rare, autoimmune demyelinating central nervous system disease. Currently, little is known about the signs, symptoms, and health-related quality of life (HRQoL) impact of MOGAD from the patient perspective. This study explored the patient experience of MOGAD through concept elicitation interviews with patients with MOGAD and clinicians to develop a conceptual model of MOGAD.</p><p><strong>Methods: </strong>A preliminary conceptual model of MOGAD signs, symptoms, treatments, and HRQoL impacts was developed based on a review of the published literature. Thematic analysis of semi-structured interviews with patients and clinicians was used to develop the final conceptual model.</p><p><strong>Results: </strong>Twelve patients with MOGAD and two clinicians were interviewed. The most common patient-reported symptoms were eye pain, fatigue, body aches/pain, headaches, and blurred vision. Eye pain and body aches/pain were reported as the most bothersome symptoms and most important to improve with treatment. The HRQoL impacts most commonly reported by patients were difficulty with carrying out household chores, inability to work, depression, and difficulty walking. Impacts on work/school were considered by patients as the most bothersome to their HRQoL and the most important to resolve. Following the interviews, a final conceptual model was produced that reported 32 symptoms across seven domains (constitutional; visual; general neurological; sensory-motor neurological; genitourinary; gastrointestinal; and chest-related, respiratory, and throat) and 50 HRQoL impacts across eight domains (emotional wellbeing, activities of daily living, physical functioning, social functioning, work/school, cognitive functioning, sleep, and financial).</p><p><strong>Conclusions: </strong>This study provides important insights into the patient experience of MOGAD from patient and clinician perspectives, highlighting the underappreciated burden of the disease. The final conceptual model demonstrates the heterogeneity of MOGAD symptoms and their impact on patients' HRQoL. Future treatment trials should consider including appropriate measures to evaluate the key symptoms and HRQoL impacts identified in this study.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144285809","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":"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":"https://doi.org/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":""},"PeriodicalIF":3.9,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Safety and Efficacy of Repeated Cycles of RimabotulinumtoxinB in the Treatment of Chronic Sialorrhea: Results of the OPTIMYST Trial.","authors":"Rajesh Pahwa, Eric Molho, Mark Lew, Khashayar Dashtipour, Ramon A Gil, Fredy J Revilla, Thomas Clinch, Peibing Qin, Stuart H Isaacson","doi":"10.1007/s40120-025-00777-z","DOIUrl":"https://doi.org/10.1007/s40120-025-00777-z","url":null,"abstract":"<p><strong>Introduction: </strong>Botulinum toxin injections into the salivary glands inhibit saliva production by reducing the release of acetylcholine at the parasympathetic nerve terminals within the salivary gland. The phase 3 study reported here assessed the safety, tolerability, and effectiveness of repeated cycles of rimabotulinumtoxinB (RIMA) injections in adults with troublesome sialorrhea.</p><p><strong>Methods: </strong>In this phase 3, open-label multicenter study, 187 adult participants with troublesome sialorrhea due to Parkinson disease (65.8%), amyotrophic lateral sclerosis (13.9%), and other etiologies (20.3%) received up to 4 cycles of RIMA treatment (3500 U every 11-15 weeks).</p><p><strong>Results: </strong>Participants (69% male, 31% female; mean age 64.1 years) had sialorrhea for a mean of 3.2 years at baseline with a mean Unstimulated Salivary Flow Rate (USFR) of 0.63 ± 0.49 g/min. During the first treatment cycle, RIMA significantly reduced the mean±standard deviation (SD) USFR from baseline to week 4 by - 0.34 ± 0.37 g/min (p < 0.0001), and efficacy was maintained through week 13 (- 0.14 ± 0.29 g/min; p < 0.0001). Reductions were maintained at subsequent injection cycles 2-4, with mean absolute USFRs at weeks 4 and 13 of each cycle similar to those of cycle 1. Most adverse events (AEs) were mild, and the most commonly reported AEs in each cycle that were considered to be treatment-related were dry mouth (≤ 15.5% participants/cycle) and dental caries (≤ 6.0% participants/cycle).</p><p><strong>Conclusion: </strong>This study demonstrates that RIMA 3500 U safely reduces saliva production over repeated treatment cycles through 1 year, thereby supporting its utility in the management of troublesome sialorrhea in adults.</p><p><strong>Clinicaltrials: </strong></p><p><strong>Gov identifier: </strong>NCT02610868.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144266848","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":"Levodopa Intestinal Gel Infusion Therapies in Advanced Parkinson's Disease: A Swedish Study on Real-World Use and Costs.","authors":"Per Odin, Christoffer Tærud, Jenny Samuelsson, Emma Sabelström, Jeanette Lagerlund, Jonatan Freilich, M Natalia Stelmaszuk","doi":"10.1007/s40120-025-00766-2","DOIUrl":"https://doi.org/10.1007/s40120-025-00766-2","url":null,"abstract":"<p><strong>Introduction: </strong>This study evaluated real-world cassette use and cost of levodopa-carbidopa intestinal gel (LCIG; 2000 mg levodopa equivalent dose [LED]) and levodopa-entacapone-carbidopa intestinal gel (LECIG; 1250 mg LED) pump treatments among patients with advanced Parkinson's disease (PD) in Sweden.</p><p><strong>Methods: </strong>This was a non-interventional, longitudinal, retrospective, comparative study of patients with PD using data from Swedish national registries (National Patient Register [NPR] and Prescribed Drug Register [PDR]). Patients were enrolled in the study from January 1, 2017, to May 31, 2022 (NPR) or June 30, 2022 (PDR). Patients had two or more dispensed prescription records for either LCIG (January 1, 2017, onward) or LECIG (January 1, 2019, onward) and at least one diagnosis of PD on or before their first dispensed prescription. Average daily cassette use, costs (Swedish krona [SEK]), treatment persistence, and treatment patterns were assessed.</p><p><strong>Results: </strong>Overall, 419 patients (LCIG, n = 276; LECIG, n = 180; both LCIG and LECIG, n = 37 [cohorts not mutually exclusive]) were included. Mean (SD) daily cassette use was significantly higher More patients discontinued LECIG 1.28 (0.40) versus 1.09 (0.28) at 1-365 days (p < 0.0001). LECIG use was higher than LCIG at 365 days regardless of prior levodopa pump experience. The overall cost of LECIG was higher than that of LCIG; the highest cost difference between treatments was at 365 days (daily cost mean [SD], LCIG 991.23 [259.43] SEK vs. LECIG 1100.23 [341.04] SEK; p = 0.0036). Annual per-patient costs were approximately 40,000 SEK higher for LECIG versus LCIG. More patients discontinued LECIG than LCIG treatment, with 68.9% and 75.2% of those receiving LECIG and LCIG, respectively, still on treatment at 365 days.</p><p><strong>Conclusion: </strong>Real-world data show that the number of dispensed cassettes and overall treatment costs are higher for LECIG than LCIG treatment among patients with advanced PD in Sweden.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144258546","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}