{"title":"Erratum: Sexual Orientation, Gender Identity, and Experiences During, Awareness of, and Attitudes Toward Research for People with Parkinson Disease.","authors":"","doi":"10.1212/CPJ.0000000000200355","DOIUrl":"10.1212/CPJ.0000000000200355","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1212/CPJ.0000000000200304.].</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 1","pages":"e200355"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11731366/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984189","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Maria Andrea Donahue, Hammad Akram, Julianne D Brooks, Avani C Modi, Jessica Veach, Alison Kukla, Shawna W Benard, Susan T Herman, Kathleen Farrell, David M Ficker, Sahar F Zafar, William H Trescher, Deepa Sirsi, Donald J Phillips, Jacob Pellinen, Jeffrey Buchhalter, Lidia Moura, Brandy E Fureman
{"title":"Barriers to Medication Adherence in People Living With Epilepsy.","authors":"Maria Andrea Donahue, Hammad Akram, Julianne D Brooks, Avani C Modi, Jessica Veach, Alison Kukla, Shawna W Benard, Susan T Herman, Kathleen Farrell, David M Ficker, Sahar F Zafar, William H Trescher, Deepa Sirsi, Donald J Phillips, Jacob Pellinen, Jeffrey Buchhalter, Lidia Moura, Brandy E Fureman","doi":"10.1212/CPJ.0000000000200403","DOIUrl":"10.1212/CPJ.0000000000200403","url":null,"abstract":"<p><strong>Background and objectives: </strong>Epilepsy affects approximately 1.2% of the US population, resulting in 3.4 million Americans with active epilepsy. Antiseizure medication (ASM) is considered the mainstay of treatment, effective for two-thirds of people with epilepsy (PWE), while at least one-third experience drug-resistant epilepsy. A significant percentage of PWE who are treated with ASMs report nonadherence to this type of medication, leading to potentially preventable seizures and the potential for being inappropriately classified as having drug-resistant epilepsy. Ongoing seizures are associated with increased morbidity, mortality, and health care costs, among other consequences. Recognizing when PWE struggle with ASM adherence is essential for creating effective interventions and prevention strategies to improve patient outcomes.</p><p><strong>Methods: </strong>As part of the Epilepsy Learning Healthcare System Registry, we collected data from 2020 through 2023 from 4,917 individuals seen at 8 epilepsy clinics in the United States. In this cross-sectional study, we used logistic regression analysis to examine the relationship between patient-reported seizure control (or provider-reported seizure control for some sites) and endorsed barriers to medication adherence. In addition, we explored potential associations with demographic variables such as sex, race, and ethnicity. The data analysis was conducted using R version 2023.06.1 + 524.</p><p><strong>Results: </strong>Overall, 18.4% (893/4,848) reported adherence barriers and 37.7% (1,447/3,834) reported seizure control, defined as no seizures for the preceding 12 months or longer. The most prevalent barriers were forgetting to take ASMs (48.2%), experiencing ASM side effects (29.2%), and feeling as if the ASMs were not helping in controlling seizures (21.3%). The PWE who reported adherence barriers had 0.6 lower odds of having seizure control compared with those who did not report barriers (95% CI 0.4-0.7) and 0.6 lower odds of having seizure control after adjusting for race, ethnicity, and sex (95% CI 0.5-0.7).</p><p><strong>Discussion: </strong>We observed significant barriers to medication adherence and inadequate seizure control among adult PWE across 8 centers in the United States. This study suggests that PWE might benefit from standardized screening for adherence barriers with behavioral strategies to address these barriers offered during clinical encounters to personalize care.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 1","pages":"e200403"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11604104/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142751312","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Amira Salim, Sudipa Biswas, Claire Sonneborn, Olivia Hogue, Elise Hennessy, Maryann Mays, Aarushi Suneja, Zubair Ahmed, Ignacio F Mata
{"title":"Efficacy and Tolerability of Anti-CGRP Monoclonal Antibodies in Patients Aged ≥ 65 Years With Daily or Nondaily Migraine.","authors":"Amira Salim, Sudipa Biswas, Claire Sonneborn, Olivia Hogue, Elise Hennessy, Maryann Mays, Aarushi Suneja, Zubair Ahmed, Ignacio F Mata","doi":"10.1212/CPJ.0000000000200373","DOIUrl":"https://doi.org/10.1212/CPJ.0000000000200373","url":null,"abstract":"<p><strong>Background and objectives: </strong>Despite decreasing prevalence of migraine with advancing age, there remains a significant proportion of individuals aged ≥65 years with migraine. Treatment of this population is difficult and they are often excluded from clinical trials, limiting evidence regarding migraine treatment outcomes. Our objective is to assess the efficacy and tolerability of anti-calcitonin gene-related peptide (CGRP) monoclonal antibody (mAb) therapies (erenumab, fremanezumab, and galcanezumab) in patients ≥65 years (O65) compared with patients <65 (U65) with daily or nondaily migraine.</p><p><strong>Methods: </strong>This observational study uses retrospective data from the electronic medical records of patients who were treated with an anti-CGRP mAb between June 2018 and November 2021. Efficacy was determined through a reduction in monthly migraine days (MMDs) and Headache Impact Test (HIT-6) scores from baseline to posttreatment. Tolerability was examined through the number of adverse events reported per group. Mann-Whitney tests were used to compare the efficacy and tolerability of U65 and O65 patients overall and separated into daily and nondaily migraine groups.</p><p><strong>Results: </strong>The dataset consisted of U65 (n = 2,707; median [interquartile range]; 45.4 [35.8-53.8] years) or O65 (n = 304; 69.5 [67.3-73.3] years) and further separated into daily (n = 1,303) and nondaily (n = 1,708) migraine. There was no difference (<i>p</i> = 0.57) in the median MMD reduction between U65 (10 days [0.0-17.0]) and O65 (10 days [0.0-16.5]). Similarly, no difference was found among patients with nondaily migraine (<i>p</i> = 0.82) and patients with daily migraine (<i>p</i> = 0.59). HIT-6 scores decreased from severe to moderate/substantial impact for all groups. The daily and nondaily groups showed differences in meeting the 50% improvement threshold (nondaily U65, 67% vs daily U65, 54%, <i>p</i> < 0.0001; nondaily O65, 65% vs daily O65, 49%, <i>p</i> = 0.008). Side effects were reported (829/3,011), with a higher incidence in the U65 (22% O65, 28% U65). The most common side effects for both groups were injection site reaction/rash (40%) and constipation (25%).</p><p><strong>Discussion: </strong>This retrospective analysis provides real-world evidence that there is no difference in the efficacy and tolerability of treatment with erenumab, fremanezumab, and galcanezumab in patients O65 when compared with patients U65 both with daily or nondaily migraine. These data may help guide the choice of migraine treatment in older populations.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 1","pages":"e200373"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11464238/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142470870","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Erratum: A General Neurologist's Practical Diagnostic Algorithm for Atypical Parkinsonian Disorders: A Consensus Statement.","authors":"","doi":"10.1212/CPJ.0000000000200399","DOIUrl":"10.1212/CPJ.0000000000200399","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1212/CPJ.0000000000200345.].</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 1","pages":"e200399"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11464216/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142470871","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Andrea Van Steenis, Mehmet N Cizmeci, Floris Groenendaal, Marianne Thoresen, Frances M Cowan, Linda S de Vries, Sylke J Steggerda
{"title":"Individualized Neuroprognostication in Neonates With Hypoxic-Ischemic Encephalopathy Treated With Hypothermia.","authors":"Andrea Van Steenis, Mehmet N Cizmeci, Floris Groenendaal, Marianne Thoresen, Frances M Cowan, Linda S de Vries, Sylke J Steggerda","doi":"10.1212/CPJ.0000000000200370","DOIUrl":"https://doi.org/10.1212/CPJ.0000000000200370","url":null,"abstract":"<p><strong>Background and objectives: </strong>To determine whether post-rewarming brain MRI enables individualized domain-specific prediction of neurodevelopmental outcomes at 2 years of age in infants treated with hypothermia for hypoxic-ischemic brain injury.</p><p><strong>Methods: </strong>We conducted a retrospective multicenter study of infants with moderate-to-severe hypoxic-ischemic encephalopathy (HIE) treated with hypothermia. Brain MRI abnormalities and the prediction of domain-specific 2-year neurodevelopmental outcomes were scored independently by 2 investigators after which consensus was reached for both imaging findings and outcome prediction. Neuroimaging patterns were categorized as normal, white matter (WM)/watershed-predominant, deep gray matter (DGM)-predominant, and near-total injury. Outcomes were predicted separately for mortality, cerebral palsy (CP) type and severity, cognitive delay, epilepsy, cerebral visual impairment (CVI), and feeding difficulties; these outcomes were predicted as highly unlikely, possible, probable, or highly likely.</p><p><strong>Results: </strong>Of the 152 study infants, 27 (18%) died. The neurodevelopmental outcome at 2 years was available in all 125 survivors. CP was seen in 21 of 125 surviving infants (17%). No infants in the highly unlikely category developed CP while 90% in the highly likely category did. When CP was predicted as possible, 40% developed CP; all were mild and ambulatory. When CP was predicted as probable, 67% developed CP of whom 40% were severe and nonambulatory. Cognitive scores were available in 104 of 125 infants (83%). Cognitive delay was seen in 23 of 104 infants (22%) (15% mild and 7% severe). When cognitive delay was predicted as highly unlikely, 92% did not develop cognitive delay and the delay was mild in those who did. When cognitive delay was considered highly likely, this developed in 100%. When epilepsy, CVI, and feeding problems were predicted as highly unlikely, 98% did not develop epilepsy; for CVI and feeding problems, this was 100% and 97%, respectively. In 27 of 152 infants (18%), the investigators reached consensus that the overall injury was severe enough to consider redirection of care; 21 of 27 infants (78%) died. Of the survivors, 5 infants developed severe CP and 1 had a mild dyskinetic CP with swallowing problems and CVI.</p><p><strong>Discussion: </strong>Individualized domain-specific categorical neuroprognostication mainly based on brain MRI is feasible, reliable, and highly accurate in infants with HIE.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 1","pages":"e200370"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11464227/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142470875","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Mary K Schroth, Jennifer Deans, Diana X Bharucha Goebel, W Bryan Burnette, Basil T Darras, Bakri H Elsheikh, Marcia V Felker, Andrea Klein, Jena Krueger, Crystal M Proud, Aravindhan Veerapandiyan, Robert J Graham
{"title":"Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations.","authors":"Mary K Schroth, Jennifer Deans, Diana X Bharucha Goebel, W Bryan Burnette, Basil T Darras, Bakri H Elsheikh, Marcia V Felker, Andrea Klein, Jena Krueger, Crystal M Proud, Aravindhan Veerapandiyan, Robert J Graham","doi":"10.1212/CPJ.0000000000200374","DOIUrl":"https://doi.org/10.1212/CPJ.0000000000200374","url":null,"abstract":"<p><strong>Background and objectives: </strong>Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by biallelic variants of the <i>Survival Motor Neuron 1</i> gene (<i>SMN1</i>) that affects approximately 1 in 15,000 live births. Availability of 3 SMN-enhancing treatments for SMA has led to urgency to review how clinicians and patients use these treatments for SMA, while additional research and real-world data and experience are being collected. This work describes important factors to assist with decision-making for SMN-enhancing treatments.</p><p><strong>Methods: </strong>A systematic literature review was conducted on SMN-enhancing treatments for SMA and related studies. A working group of American and European health care providers with expertise in SMA care identified barriers and developed recommendations through a modified Delphi technique with serial surveys and feedback through virtual meetings to fill gaps for information where evidence is limited. A community working group of an individual living with SMA and caregivers provided insight and perspective on SMA treatments and support through a virtual meeting to guide recommendations.</p><p><strong>Results: </strong>The health care provider working group and the community working group agreed that when determining whether to start, change, add, or discontinue a treatment, essential considerations include patient and family/caregiver perspective, and treatment safety and side effects. When initiating treatment for patients newly diagnosed with SMA, important patient characteristics are age and <i>Survival Motor Neuron 2</i> gene <i>(SMN2)</i> copy number. Furthermore, when initiating, changing, or adding treatment, current clinical status and comorbidities drive decision-making. When considering a medication or treatment plan change, unless there is an urgent indication, a treatment and associated patient outcomes should be monitored for a minimum of 6-12 months. When determining a treatment plan with an adolescent or adult with SMA, consider factors such as quality of life, burden vs benefit of treatment, and reproductive issues. Access to care coordination and interdisciplinary/multidisciplinary care are essential to treatment success.</p><p><strong>Discussion: </strong>Sharing information about current knowledge of treatments and shared decision-making between health care providers and patients living with SMA and caregivers are essential to overcoming barriers to providing SMN-enhancing treatments.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 1","pages":"e200374"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11464225/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142470882","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jamison Seabury, Jennifer Weinstein, Anika Varma, Spencer James Rosero, Charlotte Engebrecht, Abigail Arky, Christine Zizzi, Nuran Dilek, Abigail Mathewson, Susan Salem-Spencer, Elizabeth J Santos, Chad Rydel Heatwole
{"title":"Patient- and Caregiver-Reported Impact of Symptoms in Alzheimer Disease, Mild Cognitive Impairment, and Dementia.","authors":"Jamison Seabury, Jennifer Weinstein, Anika Varma, Spencer James Rosero, Charlotte Engebrecht, Abigail Arky, Christine Zizzi, Nuran Dilek, Abigail Mathewson, Susan Salem-Spencer, Elizabeth J Santos, Chad Rydel Heatwole","doi":"10.1212/CPJ.0000000000200418","DOIUrl":"10.1212/CPJ.0000000000200418","url":null,"abstract":"<p><strong>Background and objectives: </strong>In preparation for future clinical trials involving individuals with Alzheimer disease (AD), mild cognitive impairment (MCI), and dementia, it is important to ascertain the widespread impact of symptoms from the direct perspectives of patients and caregivers. In this study, we performed cross-sectional surveys using large-scale patient and caregiver data to identify the prevalence and average impact of symptoms and symptomatic themes experienced by adults with AD, MCI, and dementia. Subsequent analyses were used to determine which demographic and disease-specific factors are associated with more severe disease.</p><p><strong>Methods: </strong>Fifteen adults with AD (6), MCI (8), and dementia (1) and 15 caregivers of adults with AD (7), MCI (6), and dementia (2) participated in qualitative interviews providing 1,166 and 1,097 unique quotes pertaining to symptom burden. Using open-ended questions from a comprehensive interview guide, participants were asked to identify the symptoms of AD that have the greatest effect on their lives or the lives of the individual for whom they provide care. A cross-sectional survey was then implemented inquiring about the potential symptoms of importance identified during preliminary qualitative interviews. Four-hundred thirty-three individuals (patients and caregivers) participated in the cross-sectional survey, providing more than 35,000 symptom rating responses. Subsequent analyses were conducted to determine how demographic and disease-specific characteristics correlate with symptomatic theme prevalence.</p><p><strong>Results: </strong>The most frequent symptomatic themes reported by individuals with AD, MCI, and dementia in the cross-sectional survey were memory problems (99.0%), problems thinking (90.3%), and communication difficulties (80.4%). Patients identified decreased satisfaction in social situations (1.45), fatigue (1.45), and memory problems (1.41) as the most impactful symptomatic themes (range 0-4). Patient-reported symptomatic theme prevalence was strongly associated with the Modified Rankin Scale (mRS) for neurologic disability.</p><p><strong>Discussion: </strong>Individuals with AD, MCI, and dementia experience a variety of symptoms that significantly affect their daily lives. These symptoms, many underrecognized, are of variable importance to individuals with these diseases and may inform potential targets for future therapeutic intervention as well as facilitate the development and validation of disease-specific outcome measures.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 1","pages":"e200418"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11668520/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142885981","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kathryn A Wyman-Chick, Tanis J Ferman, Daniel Weintraub, Melissa J Armstrong, Bradley F Boeve, Ece Bayram, Ella Chrenka, Matthew J Barrett
{"title":"Distinguishing Prodromal Dementia With Lewy Bodies From Prodromal Alzheimer Disease: A Longitudinal Study.","authors":"Kathryn A Wyman-Chick, Tanis J Ferman, Daniel Weintraub, Melissa J Armstrong, Bradley F Boeve, Ece Bayram, Ella Chrenka, Matthew J Barrett","doi":"10.1212/CPJ.0000000000200380","DOIUrl":"10.1212/CPJ.0000000000200380","url":null,"abstract":"<p><strong>Background and objectives: </strong>It can be clinically challenging to differentiate dementia with Lewy bodies (DLB) and Alzheimer disease (AD). As potential therapies emerge with the goal of slowing or halting misfolded protein aggregation, it is imperative to be able to identify individuals before the disease becomes disabling. Differentiating between DLB and AD in the preclinical or prodromal phase of DLB and AD becomes more important. Studies are needed to validate the proposed criteria for prodromal DLB.</p><p><strong>Methods: </strong>Longitudinal data were obtained from the Uniform Data Set of the National Alzheimer's Coordinating Center. Included participants had a baseline diagnosis of normal or mild cognitive impairment and a consecutive 2-year follow-up diagnosis of DLB or AD. We examined whether core DLB clinical features, supportive neuropsychiatric features, and neuropsychological data in the 2 years preceding the dementia diagnosis distinguished DLB from AD.</p><p><strong>Results: </strong>We identified 143 participants with DLB and 429 age-matched/sex-matched participants with AD. The presence of 2 or more core DLB features in the year before dementia diagnosis yielded the greatest AUC (0.793; 95% CI 0.748-0.839) in distinguishing prodromal DLB from prodromal AD. Sleep disturbances, hallucinations, and a cognitive profile of worse processing speed, attention, and visuoconstruction performance were evident at least 2 years before the dementia diagnosis in DLB compared with AD.</p><p><strong>Discussion: </strong>Data from this multisite, longitudinal, well-characterized research North American cohort support the validity of the recently published criteria for prodromal DLB. In the prodromal stage, patients who subsequently develop DLB are more likely to have core DLB clinical features and worse attention, processing speed, and visuospatial performance than those who go on to develop AD. Differentiation of DLB and AD before dementia emerges provides an opportunity for early, disease-specific intervention and overall management.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 1","pages":"e200380"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11464229/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142470868","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}