{"title":"Textbook Outcomes Among Patients with Aneurysmal Subarachnoid Hemorrhage Following Endovascular Treatment.","authors":"Zisheng Liu, Yuhao Tan, Yanpeng Wei, Dongwei Dai, Rui Zhao, Qiang Li, Qinghai Huang, Yi Xu, Pengfei Yang, Jun Sun, Jianmin Liu, Qiao Zuo","doi":"10.1007/s40120-024-00577-x","DOIUrl":"10.1007/s40120-024-00577-x","url":null,"abstract":"<p><strong>Introduction: </strong>The case fatality rate among patients with aneurysmal subarachnoid hemorrhage (aSAH) has decreased progressively, with numerous patients subjected to contemporary paradigms that minimize the use of agonizing therapeutic processes. The concept of the \"Textbook Outcome\" (TO), a composite outcome that highlights numerous favorable outcomes, was developed in the context of gastrointestinal tumor surgeries and expeditiously extended across diverse surgical spheres. The aim of this study was to explore the factors hindering the achievement of optimal prognoses in postinterventional aSAH patients, employ textbook outcomes, and establish predictive models.</p><p><strong>Methods: </strong>We conducted a retrospective review of data from 1270 aSAH patients who received endovascular treatment between 2012 and 2018. We delineated an exemplary TO within the aSAH domain, characterized by favorable clinical results, minimal complications, and the absence of retreatments. This TO-oriented approach is explained within the manuscript.</p><p><strong>Results: </strong>The findings revealed that preoperative intraventricular hemorrhage (IVH), preoperative Hunt and Hess grade (H&H) ≥ 3, World Federation of Neurosurgical Societies (WFNS) grade ≥ 3, the presence of blebs on the aneurysm, aneurysms situated at branching sites, and non-stent-assisted endovascular intervention were the strongest risk factors for not achieving textbook outcomes (non-\"Textbook Outcome\" [N-TO]). Decision curve analysis and calibration analyses revealed strong concordance between the predictions of the N-TO nomogram model and the actual observations.</p><p><strong>Conclusions: </strong>Treatment Outcomes hold significant practical value in clinical studies of aSAH patients receiving endovascular treatment. The likelihood of N-TOs was predicted by IVH, H&H grade ≥ 3, WFNS grade ≥ 2, presence o f bleb on the aneurysm, and aneurysms located at branching sites.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"373-387"},"PeriodicalIF":3.7,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10951154/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139542874","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}
Neurology and TherapyPub Date : 2024-04-01Epub Date: 2024-02-01DOI: 10.1007/s40120-024-00580-2
Simona Lattanzi, Nicoletta Foschi, Chiara Martellino, Daniela Audenino, Giovanni Boero, Paolo Bonanni, Edoardo Ferlazzo, Valentina Chiesa, Filippo Dainese, Marta Piccioli, Alessandra Ferrari, Angelo Labate
{"title":"Conversion to Brivaracetam Monotherapy in Clinical Practice: A Retrospective Study.","authors":"Simona Lattanzi, Nicoletta Foschi, Chiara Martellino, Daniela Audenino, Giovanni Boero, Paolo Bonanni, Edoardo Ferlazzo, Valentina Chiesa, Filippo Dainese, Marta Piccioli, Alessandra Ferrari, Angelo Labate","doi":"10.1007/s40120-024-00580-2","DOIUrl":"10.1007/s40120-024-00580-2","url":null,"abstract":"<p><strong>Introduction: </strong>The study aimed to evaluate the effectiveness and safety of brivaracetam (BRV) as conversion monotherapy in adults with focal epilepsy treated in the context of real-world clinical practice.</p><p><strong>Methods: </strong>This was a retrospective, observational, non-interventional study in adults with focal epilepsy who converted to BRV monotherapy following the withdrawal of background antiseizure medications (ASMs). Primary effectiveness outcome was the retention rate of BRV as single ASM at 6 and 12 months. Secondary outcomes included the 6- and 12-month rates of seizure freedom. Safety and tolerability outcomes included the frequency and type of adverse events (AEs) and the occurrence of treatment discontinuation due to AEs.</p><p><strong>Results: </strong>A total of 44 participants with a median age of 63.5 (interquartile range 44-73.5) years were included; 17 subjects were seizure free at baseline, and 9 of them switched from levetiracetam because of lack of tolerability. The retention rate of BRV monotherapy was 88.6% (39/44) at 6 months and 83.9% (26/31) at 12 months. The rates of seizure freedom were 72.7% (32/44) in subjects with 6-month follow-up and 58.1% (18/31) in subjects with 12-month follow-up. The median maintenance dosage of BRV monotherapy was 150 (100-200) mg/day at 6 months and 125 (100-200) mg/day in subjects with 12-month follow-up. Adverse events were recorded in 6/44 (13.6%) participants and led to BRV discontinuation in 2/44 (4.5%) cases. The reported AEs were somnolence (n = 3), fatigue (n = 2), and irritability (n = 1); no serious AEs were experienced. In 21/44 (47.7%) participants, BRV monotherapy resulted from the direct switch from levetiracetam. The rates of treatment retention and seizure freedom at 6 and 12 months were higher among people who switched from levetiracetam to BRV monotherapy.</p><p><strong>Conclusion: </strong>Brivaracetam may be a valuable treatment of focal seizures in people who converted to monotherapy in a real-life setting.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"389-398"},"PeriodicalIF":3.7,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10951135/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139651250","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}
Neurology and TherapyPub Date : 2024-02-01Epub Date: 2023-12-02DOI: 10.1007/s40120-023-00567-5
Luis Genis-Najera, Maria Elena Sañudo-Maury
{"title":"Bioequivalence Study of Two Tablet Formulations of Clonazepam 2 mg: A Randomized, Open-Label, Crossover Study in Healthy Mexican Volunteers Under Fasting Conditions.","authors":"Luis Genis-Najera, Maria Elena Sañudo-Maury","doi":"10.1007/s40120-023-00567-5","DOIUrl":"10.1007/s40120-023-00567-5","url":null,"abstract":"<p><strong>Introduction: </strong>The prevalence of neurological disorders is high among the Mexican population. Clonazepam is primarily indicated to treat panic disorders, certain kinds of epilepsy such as status epilepticus, childhood motor seizures (petit mal absence, Lennox-Gastaut syndrome, and infantile spasms), anxiety, and muscle spasm. This study was performed to compare bioequivalence between two oral tablet formulations of clonazepam 2 mg in healthy Mexican volunteers under fasting conditions.</p><p><strong>Methods: </strong>This phase I, randomized, open-label, two-treatment, crossover study included 30 healthy volunteers. Subjects were randomly assigned to either test or reference formulation of clonazepam 2 mg. Each study period was separated by 21-day washout period. Blood samples were collected at pre-dose and up to 72 h after drug administration. Clonazepam concentrations were determined using a validated ultra-flow liquid chromatography-tandem mass spectrometric method. Pharmacokinetic parameters were determined using a non-compartmental method. Two formulations were considered bioequivalent if geometric mean ratios (test/reference) were between 80% and 125%. Safety was evaluated by recording adverse events.</p><p><strong>Results: </strong>Pharmacokinetic parameters were comparable between test and reference formulations. The mean maximum plasma concentration (C<sub>max</sub>) was ≈ 13 ng/mL, area under the plasma concentration-time curve from time 0 to last measurable concentration (AUC<sub>0-t</sub>) was ≈ 360 ng h/mL, time to reach maximum plasma concentration (T<sub>max</sub>) was ≈ 3 h, and elimination half-life (t<sub>1/2</sub>) was ≈ 43 h. Geometric mean ratios (90% confidence interval) of C<sub>max</sub> (99.2-115.3%), AUC<sub>0-t</sub> (100.6-110.6%), and AUC<sub>0-∞</sub> (98.5-111.6%) were within the bioequivalence range. Seven non-serious adverse events (mostly asymptomatic hypotension) were recorded.</p><p><strong>Conclusion: </strong>The test and reference formulations of clonazepam 2 mg were bioequivalent and well tolerated in healthy Mexican volunteers under fasting conditions.</p><p><strong>Protocol authorization number: </strong>213301410B0051 (Approved on April 13, 2021).</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"141-152"},"PeriodicalIF":3.7,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10787705/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138470644","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}
Neurology and TherapyPub Date : 2024-02-01Epub Date: 2024-01-05DOI: 10.1007/s40120-023-00573-7
Sharon Stoll, Kathleen Costello, Scott D Newsome, Hollie Schmidt, Amy B Sullivan, Barry Hendin
{"title":"Insights for Healthcare Providers on Shared Decision-Making in Multiple Sclerosis: A Narrative Review.","authors":"Sharon Stoll, Kathleen Costello, Scott D Newsome, Hollie Schmidt, Amy B Sullivan, Barry Hendin","doi":"10.1007/s40120-023-00573-7","DOIUrl":"10.1007/s40120-023-00573-7","url":null,"abstract":"<p><p>Shared decision-making (SDM) between the patient and their healthcare provider (HCP) in developing treatment plans is increasingly recognized as central to improving treatment adherence and, ultimately, patient outcomes. In multiple sclerosis (MS), SDM is particularly crucial for optimizing treatment in a landscape that has grown more complex with the availability of newer, high-efficacy MS therapies. However, little direct evidence on the effectiveness of SDM is available to guide practice. Multiple factors, including patient age, ethnic background, perceptions, invisible MS symptoms, and psychological comorbidities can influence a patient's willingness and ability to participate in SDM. HCPs need to appreciate these factors and ask the right questions to break down obstacles to SDM. The HCP has a responsibility to help patients feel adequately informed and comfortable in having an active role in their care. This review identifies potential barriers to SDM and provides a strategy for HCPs to overcome these obstacles through patient (and caregiver) discussions to ensure optimal patient satisfaction with treatment and thus the best possible outcomes for their patients.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"21-37"},"PeriodicalIF":3.9,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10787702/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139098426","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}
Neurology and TherapyPub Date : 2024-02-01Epub Date: 2023-11-30DOI: 10.1007/s40120-023-00563-9
Xiaoling Pan, Yingjian Pei, Meixia Zhang, Wansi Zhong, Jin Hu, Zhimin Wang, Dongjuan Xu, Min Lou, Hongfang Chen, Zhicai Chen
{"title":"Association of Atrial Fibrillation with Remote Intracerebral Hemorrhage After Intravenous Thrombolysis: Results from a Multicenter Study in China.","authors":"Xiaoling Pan, Yingjian Pei, Meixia Zhang, Wansi Zhong, Jin Hu, Zhimin Wang, Dongjuan Xu, Min Lou, Hongfang Chen, Zhicai Chen","doi":"10.1007/s40120-023-00563-9","DOIUrl":"10.1007/s40120-023-00563-9","url":null,"abstract":"<p><strong>Introduction: </strong>This study aimed to investigate the association between atrial fibrillation (AF), particularly newly diagnosed AF, and remote intracerebral hemorrhage (rICH) in patients with ischemic stroke who were treated with intravenous thrombolysis (IVT).</p><p><strong>Methods: </strong>This observational study was conducted on patients with ischemic stroke who received IVT with recombinant tissue-type plasminogen activator. The data were taken from a multicenter prospective registry of a Chinese population. rICH was defined as any extraischemic hemorrhage detected on computerized tomography (CT) 24 h after intravenous thrombolysis. We collected and compared the demographic data and clinical characteristics of all the patients with rICH to those of patients without any type of hemorrhagic transformation. The association between AF and rICH was analyzed using univariate analysis and binary logistic regression.</p><p><strong>Results: </strong>A total of 20,697 patients were included in the study, with 1566 (7.6%) experiencing intracerebral hemorrhage (ICH), 586 (2.8%) experiencing rICH, and 19,131 (92.4%) not experiencing any form of hemorrhagic transformation. Univariate analysis revealed significant differences in age, pre-thrombolysis systolic blood pressure, baseline National Institute of Health Stroke Scale score, previously known AF, newly diagnosed AF, coronary heart disease, congestive heart failure, hyperhomocysteinemia, and history of thrombolysis between the rICH and control groups (P < 0.05). Further multivariate logistic regression analysis indicated that total AF (OR 1.821, 95% CI 1.082-3.065, P < 0.05), previously known AF (OR 1.470, 95% CI 1.170-1.847), and newly diagnosed AF (OR 1.920, 95% CI 1.304-2.825) were independently associated with rICH.</p><p><strong>Conclusions: </strong>This study suggests that AF, regardless of whether it is newly diagnosed or previously known, may be associated with the occurrence of rICH following intravenous thrombolysis. Interestingly, our findings suggest that newly diagnosed AF may have a stronger impact on rICH than previously known AF, although confirmation from more studies is needed.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"127-139"},"PeriodicalIF":3.7,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10787712/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138461198","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}
Neurology and TherapyPub Date : 2024-02-01Epub Date: 2023-11-10DOI: 10.1007/s40120-023-00561-x
Sui-Yi Xu, Qi Zhang, Chang-Xin Li
{"title":"Paroxysmal Sympathetic Hyperactivity After Acquired Brain Injury: An Integrative Review of Diagnostic and Management Challenges.","authors":"Sui-Yi Xu, Qi Zhang, Chang-Xin Li","doi":"10.1007/s40120-023-00561-x","DOIUrl":"10.1007/s40120-023-00561-x","url":null,"abstract":"<p><p>Paroxysmal sympathetic hyperactivity (PSH) mainly occurs after acquired brain injury (ABI) and often presents with high fever, hypertension, tachycardia, tachypnea, sweating, and dystonia (increased muscle tone or spasticity). The pathophysiological mechanisms of PSH are not fully understood. Currently, there are several views: (1) disconnection theory, (2) excitatory/inhibitory ratio, (3) neuroendocrine function, and (4) neutrophil extracellular traps. Early diagnosis of PSH remains difficult, given the low specificity of its diagnostic tools and unclear pathogenesis. According to updated case analyses in recent years, PSH is now more commonly observed in patients with stroke, with tachycardia and hypertension as the main clinical manifestations, which is not fully consistent with previous data. To date, the PSH Assessment Measure tool is optimal for the early identification of PSH and stratification of symptom severity. Clinical strategies for the management of PSH are divided into three main points: (1) reduction of stimulation, (2) reduction of sympathetic excitatory afferents, and (3) inhibition of the effects of sympathetic hyperactivity on target organs. However, use of drugs and standards have not yet been harmonized. Further investigation on the relationship between PSH severity and long-term neurological prognosis in patients with ABI is required. This review aimed to determine the diagnostic and management challenges encountered in PSH after ABI.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"11-20"},"PeriodicalIF":3.7,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10787720/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"72014929","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}
Neurology and TherapyPub Date : 2024-02-01Epub Date: 2023-11-04DOI: 10.1007/s40120-023-00560-y
Jessica Braid
{"title":"Letter to the Editor Regarding \"Validation of a Set of Instruments to Assess Patient- and Caregiver-Oriented Measurements in Spinal Muscular Atrophy: Results of the SMA-TOOL Study\".","authors":"Jessica Braid","doi":"10.1007/s40120-023-00560-y","DOIUrl":"10.1007/s40120-023-00560-y","url":null,"abstract":"","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"251-254"},"PeriodicalIF":3.7,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10787713/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71484277","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}
Neurology and TherapyPub Date : 2024-02-01Epub Date: 2024-01-04DOI: 10.1007/s40120-023-00568-4
Sunita N Misra, Randa Jarrar, John M Stern, Danielle A Becker, Enrique Carrazana, Adrian L Rabinowicz
{"title":"Rapid Rescue Treatment with Diazepam Nasal Spray Leads to Faster Seizure Cluster Termination in Epilepsy: An Exploratory Post Hoc Cohort Analysis.","authors":"Sunita N Misra, Randa Jarrar, John M Stern, Danielle A Becker, Enrique Carrazana, Adrian L Rabinowicz","doi":"10.1007/s40120-023-00568-4","DOIUrl":"10.1007/s40120-023-00568-4","url":null,"abstract":"<p><strong>Introduction: </strong>Although prompt treatment of status epilepticus is standard of care, the effect of timing of rescue therapy administration for seizure clusters in epilepsy remains unknown. Seizure clusters are a rare but clinically important condition, and benzodiazepines are the cornerstone rescue therapy for seizure clusters in epilepsy. We characterized temporal patterns from a large dataset of treated seizure clusters in the safety study of diazepam nasal spray.</p><p><strong>Methods: </strong>This post hoc analysis used timing data of treated seizure clusters recorded by care partners and patients in seizure diaries during a 1-year safety study. Data analysis used time from seizure start to administration of diazepam.</p><p><strong>Results: </strong>From 4466 observations, 3225 had data meeting criteria for analysis. Overall, median times from seizure start to dose administration, dose administration to seizure termination, and total seizure duration were 2, 3, and 7 min, respectively. In seizure clusters treated in < 5 min (median 1.0 min), median time from dose to seizure termination was 2.0 min, and median total seizure duration was 4.0 min. Among seizure clusters treated in ≥ 5 min (median 10.0 min), median time to seizure termination was 10.0 min, and median total seizure duration was 23.0 min. Previously published safety results reported that over a mean participation of 1.5 years, 82.2% of patients had ≥ 1 treatment-emergent adverse events (TEAEs) irrespective of relationship to treatment, including 30.7% with serious TEAEs; 18.4% had TEAEs deemed at least possibly related to the study drug, none of which were serious. There were no events of cardiorespiratory depression.</p><p><strong>Conclusion: </strong>Echoing the importance of early use of benzodiazepines in status epilepticus, the findings from this exploratory analysis of patients with refractory epilepsy and frequent seizure clusters identify a potential benefit of early diazepam nasal spray treatment leading to faster seizure resolution within the seizure cluster. Trial Registration Information: ClinicalTrials.gov identifier NCT02721069 ( https://clinicaltrials.gov/ct2/show/NCT02721069 ).</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"221-231"},"PeriodicalIF":3.7,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10787722/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139087834","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}
Neurology and TherapyPub Date : 2024-02-01Epub Date: 2024-01-05DOI: 10.1007/s40120-023-00571-9
Pablo Rebollo, Sofía García-López, Mónica Povedano, María G Cattinari, Mercedes Martínez-Moreno, Ángeles Terrancle, Rosana Cabello-Moruno, Juan F Vázquez-Costa
{"title":"Design and Validation of a Clinical Outcome Measure for Adolescents and Adult Patients with Spinal Muscular Atrophy: SMA Life Study Protocol.","authors":"Pablo Rebollo, Sofía García-López, Mónica Povedano, María G Cattinari, Mercedes Martínez-Moreno, Ángeles Terrancle, Rosana Cabello-Moruno, Juan F Vázquez-Costa","doi":"10.1007/s40120-023-00571-9","DOIUrl":"10.1007/s40120-023-00571-9","url":null,"abstract":"<p><strong>Introduction: </strong>The objective of this study is to develop a clinical tool for the evaluation and follow-up of adolescent and adult patients with 5q spinal muscular atrophy (SMA) and to design its validation.</p><p><strong>Methods: </strong>This prospective, non-interventional study will be carried out at five centres in Spain and will include patients aged 16 years or older with a confirmed diagnosis of 5q SMA (biallelic mutation of the survival motor neuron 1 [SMN1] gene). A panel of experts made up of neurologists, physiatrists and Spanish patients' association (FundAME), participated in the design of the clinical tool. Physicians will administer the tool at three time points (baseline, 12 months and 24 months). Additionally, data from other questionnaires and scales will be collected. Once recruitment is achieved, an interim statistical analysis will be performed to assess its psychometric properties by applying Rasch analysis and classical statistical tests.</p><p><strong>Results: </strong>The tool will consist of up to 53 items to assess functional status from a clinical perspective in seven key dimensions (bulbar, respiratory, axial, lower, upper, fatigability and other symptoms), which will be collected together with objective clinical measures (body mass index, forced vital capacity, pinch strength and 6-minute walk test).</p><p><strong>Conclusions: </strong>The validation of this tool will facilitate the clinical evaluation of adult and adolescent patients with SMA and the quantification of their response to new treatments in both clinical practice and research.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"233-249"},"PeriodicalIF":3.7,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10787721/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139098425","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}
Neurology and TherapyPub Date : 2024-02-01Epub Date: 2023-11-01DOI: 10.1007/s40120-023-00556-8
Richard B Lipton, Janette Contreras-De Lama, Daniel Serrano, Ella Engstrom, Nicolai D Ayasse, Weijie Poh, François Cadiou, Aubrey Manack Adams
{"title":"Real-World Use of Ubrogepant as Acute Treatment for Migraine with an Anti-Calcitonin Gene-Related Peptide Monoclonal Antibody: Results from COURAGE.","authors":"Richard B Lipton, Janette Contreras-De Lama, Daniel Serrano, Ella Engstrom, Nicolai D Ayasse, Weijie Poh, François Cadiou, Aubrey Manack Adams","doi":"10.1007/s40120-023-00556-8","DOIUrl":"10.1007/s40120-023-00556-8","url":null,"abstract":"<p><strong>Introduction: </strong>Although acute and preventive treatments for migraine are commonly given in combination, data on the real-world effectiveness of ubrogepant as an acute treatment when used with an anti-calcitonin gene-related peptide (CGRP) monoclonal antibody (with or without onabotulinumtoxinA) are limited. This analysis sought to evaluate the real-world effectiveness, treatment satisfaction, and optimization of ubrogepant for the acute treatment of migraine when used in combination with an anti-CGRP monoclonal antibody, with or without concomitant onabotulinumtoxinA.</p><p><strong>Methods: </strong>This prospective, multiple-attack, open-label, observational study (COURAGE) assessed meaningful pain relief (MPR), return to normal function (RNF), treatment satisfaction, and acute treatment optimization of ubrogepant (50 or 100 mg) when combined with an anti-CGRP monoclonal antibody, onabotulinumtoxinA, or both in adult users of Migraine Buddy, a migraine tracking application.</p><p><strong>Results: </strong>In the ubrogepant and anti-CGRP monoclonal antibody arm (n = 245), following the first ubrogepant-treated attack, 61.6% (151/245) and 80.4% (197/245) of ubrogepant-treated participants achieved MPR at 2 and 4 h post-dose, respectively, and 34.7% (85/245) and 55.5% (136/245) achieved RNF at 2 and 4 h post-dose, respectively. Across up to 10 ubrogepant-treated attacks (N = 1153), MPR was achieved in 51.3% (592/1153) and 73.5% (847/1153) at 2 and 4 h post-dose, respectively. RNF was achieved by 32.2% (371/1153) and 53.2% (613/1153) at 2 and 4 h post-dose. After 30 days, 72.7% (168/231) of participants reported satisfaction (using a 7-point scale) with ubrogepant when used in combination with an anti-CGRP monoclonal antibody, and 79.7% (184/231) of participants achieved acute treatment optimization (defined as moderate-maximum treatment efficacy using the Migraine Treatment Optimization Questionnaire-4).</p><p><strong>Conclusion: </strong>Real-world ubrogepant use with an anti-CGRP monoclonal antibody was associated with MPR, RNF, satisfaction, and acute treatment optimization.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"69-83"},"PeriodicalIF":3.7,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10787718/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71425480","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}