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Barriers to Medication Adherence in People Living With Epilepsy. 癫痫患者药物依从性障碍。
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2025-02-01 Epub Date: 2024-11-27 DOI: 10.1212/CPJ.0000000000200403
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

Background and objectives: 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.

Methods: 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.

Results: 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).

Discussion: 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.

背景和目的:癫痫影响约1.2%的美国人口,导致340万美国人患有活动性癫痫。抗癫痫药物(ASM)被认为是主要的治疗方法,对三分之二的癫痫患者(PWE)有效,而至少三分之一的患者患有耐药性癫痫。在接受抗痉挛药物治疗的PWE中,有相当大比例的人报告不坚持使用这类药物,从而导致本可预防的癫痫发作,并有可能被不恰当地归类为耐药癫痫。持续发作与发病率、死亡率和医疗费用增加以及其他后果有关。认识到PWE与ASM依从性的斗争对于制定有效的干预措施和预防策略以改善患者预后至关重要。方法:作为癫痫学习医疗保健系统注册的一部分,我们收集了2020年至2023年在美国8家癫痫诊所就诊的4,917名患者的数据。在这项横断面研究中,我们使用逻辑回归分析来检验患者报告的癫痫发作控制(或某些地区的提供者报告的癫痫发作控制)与认可的药物依从性障碍之间的关系。此外,我们还探讨了与人口统计学变量(如性别、种族和民族)的潜在关联。数据分析使用R版本2023.06.1 + 524。结果:总体而言,18.4%(893/ 4848)报告了依从性障碍,37.7%(1447 / 3834)报告了癫痫发作控制,定义为在过去12个月或更长时间内没有癫痫发作。最常见的障碍是忘记服用ASM(48.2%),经历ASM副作用(29.2%),感觉ASM对控制癫痫发作没有帮助(21.3%)。报告依从性障碍的PWE与没有报告依从性障碍的PWE相比,癫痫发作控制的几率低0.6 (95% CI 0.4-0.7),在调整种族、民族和性别后,癫痫发作控制的几率低0.6 (95% CI 0.5-0.7)。讨论:我们观察到美国8个中心的成人PWE患者在药物依从性和癫痫发作控制方面存在显著障碍。这项研究表明,PWE可能受益于对依从性障碍的标准化筛查,并采用行为策略来解决临床遇到的个性化护理中提供的这些障碍。
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引用次数: 0
Correction to Author Disclosures. 作者披露更正。
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2025-02-01 Epub Date: 2024-11-07 DOI: 10.1212/CPJ.0000000000200411

[This corrects the article DOI: 10.1212/CPJ.0000000000200225.][This corrects the article DOI: 10.1212/CPJ.0000000000200145.].

[此处更正文章 DOI:10.1212/CPJ.0000000000200225][此处更正文章 DOI:10.1212/CPJ.0000000000200145]。
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引用次数: 0
Efficacy and Tolerability of Anti-CGRP Monoclonal Antibodies in Patients Aged ≥ 65 Years With Daily or Nondaily Migraine. 抗CGRP单克隆抗体对年龄≥65岁的每日或非每日偏头痛患者的疗效和耐受性。
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2025-02-01 Epub Date: 2024-10-08 DOI: 10.1212/CPJ.0000000000200373
Amira Salim, Sudipa Biswas, Claire Sonneborn, Olivia Hogue, Elise Hennessy, Maryann Mays, Aarushi Suneja, Zubair Ahmed, Ignacio F Mata

Background and objectives: 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.

Methods: 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.

Results: 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 (p = 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 (p = 0.82) and patients with daily migraine (p = 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%, p < 0.0001; nondaily O65, 65% vs daily O65, 49%, p = 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%).

Discussion: 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.

背景和目的:尽管偏头痛的发病率随着年龄的增长而下降,但仍有相当一部分年龄≥65岁的人患有偏头痛。对这一人群的治疗十分困难,他们往往被排除在临床试验之外,从而限制了偏头痛治疗效果的证据。我们的目的是评估抗降钙素基因相关肽(CGRP)单克隆抗体(mAb)疗法(erenumab、fremanezumab和galcanezumab)对≥65岁(O65)患者的疗效和耐受性:这项观察性研究使用的是2018年6月至2021年11月期间接受抗CGRP mAb治疗的患者电子病历中的回顾性数据。疗效通过从基线到治疗后每月偏头痛天数(MMDs)和头痛影响测试(HIT-6)评分的减少来确定。耐受性通过每组报告的不良事件数量进行考察。曼-惠特尼检验用于比较 U65 和 O65 患者的总体疗效和耐受性,并将其分为每日偏头痛组和非每日偏头痛组:数据集包括 U65(n = 2707;中位数[四分位间差];45.4 [35.8-53.8] 岁)或 O65(n = 304;69.5 [67.3-73.3] 岁),并进一步分为每日偏头痛组(n = 1303)和非每日偏头痛组(n = 1708)。U65(10天[0.0-17.0])和O65(10天[0.0-16.5])之间的中位偏头痛缓解率没有差异(p = 0.57)。同样,非日常偏头痛患者(p = 0.82)和日常偏头痛患者(p = 0.59)之间也未发现差异。所有组别的HIT-6评分均从严重影响降至中度/严重影响。每日和非每日组在达到50%的改善阈值方面存在差异(非每日U65,67% vs 每日U65,54%,p < 0.0001;非每日O65,65% vs 每日O65,49%,p = 0.008)。副作用报告(829/3,011),其中 U65 的发生率较高(22% O65,28% U65)。两组最常见的副作用是注射部位反应/皮疹(40%)和便秘(25%):讨论:这项回顾性分析提供了真实世界的证据,表明与U65患者相比,O65患者使用erenumab、fremanezumab和galcanezumab治疗每日或非每日偏头痛的疗效和耐受性没有差异。这些数据可能有助于指导老年人群选择偏头痛治疗方法。
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引用次数: 0
Erratum: A General Neurologist's Practical Diagnostic Algorithm for Atypical Parkinsonian Disorders: A Consensus Statement. 勘误:普通神经科医生对非典型帕金森病的实用诊断算法:共识声明。
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2025-02-01 Epub Date: 2024-10-08 DOI: 10.1212/CPJ.0000000000200399

[This corrects the article DOI: 10.1212/CPJ.0000000000200345.].

[此处更正了文章 DOI:10.1212/CPJ.0000000000200345]。
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引用次数: 0
Individualized Neuroprognostication in Neonates With Hypoxic-Ischemic Encephalopathy Treated With Hypothermia. 低温治疗缺氧缺血性脑病新生儿的个性化神经诊断。
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2025-02-01 Epub Date: 2024-10-08 DOI: 10.1212/CPJ.0000000000200370
Andrea Van Steenis, Mehmet N Cizmeci, Floris Groenendaal, Marianne Thoresen, Frances M Cowan, Linda S de Vries, Sylke J Steggerda

Background and objectives: 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.

Methods: 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.

Results: 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.

Discussion: Individualized domain-specific categorical neuroprognostication mainly based on brain MRI is feasible, reliable, and highly accurate in infants with HIE.

背景和目的:确定回温后脑部核磁共振成像能否对因缺氧缺血性脑损伤而接受低体温治疗的婴儿2岁时的神经发育结局进行个体化领域特异性预测:我们对接受低体温治疗的中重度缺氧缺血性脑病(HIE)婴儿进行了一项回顾性多中心研究。脑部核磁共振成像异常和对特定领域两年神经发育结果的预测由两名研究人员独立评分,然后就成像结果和结果预测达成共识。神经影像学模式分为正常、白质(WM)/分水岭为主、深灰质(DGM)为主和接近完全损伤。对死亡率、脑瘫(CP)类型和严重程度、认知发育迟缓、癫痫、脑性视力障碍(CVI)和喂养困难分别进行了预测;这些结果被预测为极不可能、可能、可能或极有可能:结果:在研究的 152 名婴儿中,27 名(18%)死亡。所有 125 名存活者均有 2 岁时的神经发育结果。125名存活婴儿中有21名(17%)患有CP。极不可能发生 CP 的婴儿中没有人发生 CP,而极有可能发生 CP 的婴儿中有 90% 的人发生了 CP。在预测可能患上 CP 的情况下,40% 的婴儿患上了 CP;所有患儿的病情都很轻微,可以行走。当 CP 被预测为可能时,67% 的婴儿患上了 CP,其中 40% 的婴儿病情严重且无法行走。125 名婴儿中有 104 名(83%)可获得认知评分。104 例婴儿中有 23 例(22%)出现认知发育迟缓(15% 为轻度,7% 为重度)。当认知迟缓被预测为极不可能发生时,92% 的婴儿未出现认知迟缓,出现认知迟缓的婴儿认知迟缓程度较轻。当认知迟缓被认为极有可能发生时,100%的婴儿都出现了认知迟缓。当癫痫、CVI 和喂养问题被预测为极不可能发生时,98% 的婴儿没有发生癫痫;CVI 和喂养问题的发生率分别为 100% 和 97%。在 152 名婴儿中,有 27 名婴儿(占 18%)的整体损伤严重到需要考虑调整护理方向,调查人员对此达成了共识;27 名婴儿中有 21 名(占 78%)死亡。在幸存者中,5 名婴儿发展为重度脊髓灰质炎,1 名婴儿患有轻度运动障碍脊髓灰质炎,并伴有吞咽困难和 CVI:讨论:主要基于脑磁共振成像的个体化特定领域分类神经诊断对 HIE 婴儿是可行、可靠和高度准确的。
{"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}
引用次数: 0
Outcomes and Recurrence Rates Among Patients With Provoked and Cryptogenic Cerebral Venous Thrombosis: Analysis of the ACTION CVT. 诱发性和隐源性脑静脉血栓患者的预后和复发率:ACTION CVT分析。
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2025-02-01 Epub Date: 2024-10-08 DOI: 10.1212/CPJ.0000000000200381
Sami Al Kasab, Eyad Almallouhi, Liqi Shu, Kimberly P Kicielinski, Setareh Salehi Omran, David S Liebeskind, Adeel S Zubair, Maria C Vedovati, Maurizio Paciaroni, Kateryna Antonenko, Mirjam R Heldner, Adam de Havenon, Nils Henninger, Shadi Yaghi

Background and objectives: Cerebral venous thrombosis (CVT) is a rare cause of stroke. While the standard treatment is anticoagulation, the type and duration of anticoagulation depends on the underlying etiology. This study aims to identify prevalence, risk factors, and recurrent venous thromboembolism (VTE) rates among patients with idiopathic (cryptogenic) CVT and CVT provoked by transient (peripartum, hormonal treatment, infection, trauma) and persistent (cancer, thrombophilia) factors.

Methods: We used the ACTION-CVT retrospective database which included consecutive patients who were treated for CVT in 27 stroke centers in the United States, Europe, and New Zealand from January 2015 to December 2020. We compared baseline characteristics and outcomes of patients with cryptogenic, transient provoked (TP) and those with persistent provoked (PP) CVT. Baseline characteristics was compared between the groups using χ2 test, t test, or Mann-Whitney U test as appropriate, followed by multivariable regression. We used Kaplan-Meier survival analysis to assess outcome occurrence. We used interaction analysis and Cox regression to assess the risks of recurrent VTE in patients with CVT.

Results: Among 1,025 included participants with CVT, 510 (49.8%) had no identified risk factor (cryptogenic), 363 (35.4%) had at least one transient provoking factor, and 152 (14.8%) had a persistent provoking factor. Patients with TP CVT were younger (p = 0.003) and more likely to be female patients (p < 0.001). When compared with patients with TP CVT, the risk of recurrent VTE was greater in patients with PP CVT (HR 2.59, 95% CI 1.29-5.22, p = 0.008) and nonsignificantly elevated in patients with cryptogenic CVT (HR 1.85. 95% CI 0.98-3.59, p = 0.059). In the interaction analysis, there was a trend toward higher rate of recurrent VTE in female patients with cryptogenic CVT and male patients with PP CVT.

Discussion: In this multicenter study, we found that outcomes of CVT differed depending on the underlying etiology. The risk of recurrent VTE in the PP and cryptogenic CVTs may be influenced by sex.

背景和目的:脑静脉血栓(CVT)是中风的罕见病因。虽然标准治疗是抗凝,但抗凝的类型和持续时间取决于潜在的病因。本研究旨在确定特发性(隐源性)CVT 患者以及一过性(围产期、激素治疗、感染、创伤)和持续性(癌症、血栓性疾病)因素引起的 CVT 患者的患病率、风险因素和复发性静脉血栓栓塞(VTE)率:我们使用了 ACTION-CVT 回顾性数据库,其中包括 2015 年 1 月至 2020 年 12 月期间在美国、欧洲和新西兰 27 个卒中中心接受 CVT 治疗的连续患者。我们比较了隐源性、一过性诱发(TP)和持续性诱发(PP)CVT 患者的基线特征和预后。我们采用χ2检验、t检验或Mann-Whitney U检验对两组患者的基线特征进行比较,然后进行多变量回归。我们使用 Kaplan-Meier 生存分析来评估结果发生率。我们采用交互分析和 Cox 回归评估 CVT 患者复发 VTE 的风险:在 1025 名 CVT 患者中,510 人(49.8%)没有确定的风险因素(隐源性),363 人(35.4%)至少有一个短暂的诱发因素,152 人(14.8%)有一个持续的诱发因素。TP CVT 患者更年轻(p = 0.003),更可能是女性患者(p < 0.001)。与 TP CVT 患者相比,PP CVT 患者复发 VTE 的风险更高(HR 2.59,95% CI 1.29-5.22,p = 0.008),而隐源性 CVT 患者复发 VTE 的风险无显著升高(HR 1.85,95% CI 0.98-3.59,p = 0.059)。在交互分析中,女性隐源性 CVT 患者和男性 PP CVT 患者的复发性 VTE 发生率呈上升趋势:在这项多中心研究中,我们发现CVT的预后因病因而异。在 PP 型和隐源性 CVT 中,复发性 VTE 的风险可能受性别影响。
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引用次数: 0
Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations. 最佳实践中的脊髓肌肉萎缩症更新:治疗注意事项建议》。
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2025-02-01 Epub Date: 2024-10-08 DOI: 10.1212/CPJ.0000000000200374
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

Background and objectives: Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by biallelic variants of the Survival Motor Neuron 1 gene (SMN1) 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.

Methods: 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.

Results: 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 Survival Motor Neuron 2 gene (SMN2) 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.

Discussion: 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.

背景和目的:脊髓性肌萎缩症(SMA)是一种常染色体隐性遗传疾病,由存活运动神经元 1 基因(SMN1)的双偶变异引起,每 15,000 名活产婴儿中约有 1 人患病。目前已有 3 种 SMN 增强疗法可用于治疗 SMA,这使得临床医生和患者迫切需要重新审视如何使用这些疗法治疗 SMA,同时正在收集更多的研究和实际数据与经验。本研究描述了有助于SMN增强疗法决策的重要因素:方法:对SMA的SMN增强疗法及相关研究进行了系统的文献综述。一个由具有 SMA 护理专业知识的美国和欧洲医疗保健提供者组成的工作组通过改良的德尔菲技术(Delphi technique)确定了障碍并提出了建议,同时通过虚拟会议进行连续调查和反馈,以填补证据有限的信息空白。由一名 SMA 患者和护理人员组成的社区工作组通过虚拟会议提供了有关 SMA 治疗和支持的见解和观点,以指导建议的制定:结果:医疗服务提供者工作组和社区工作组一致认为,在决定是否开始、改变、增加或中止一种治疗方法时,基本考虑因素包括患者和家属/护理者的观点以及治疗的安全性和副作用。在对新确诊的 SMA 患者开始治疗时,重要的患者特征是年龄和生存运动神经元 2 基因 (SMN2) 拷贝数。此外,在开始、改变或增加治疗时,当前的临床状态和并发症也会影响决策。在考虑更换药物或治疗方案时,除非有紧急适应症,否则应至少监测 6-12 个月的治疗效果和相关的患者预后。在为患有 SMA 的青少年或成人确定治疗方案时,应考虑生活质量、治疗负担与收益以及生育问题等因素。获得护理协调和跨学科/多学科护理对治疗成功至关重要:讨论:在医疗服务提供者和 SMA 患者及护理者之间分享有关当前治疗知识的信息并共同决策,对于克服提供 SMN 增强治疗的障碍至关重要。
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引用次数: 0
Distinguishing Prodromal Dementia With Lewy Bodies From Prodromal Alzheimer Disease: A Longitudinal Study. 区分前驱型路易体痴呆与前驱型阿尔茨海默病:一项纵向研究
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2025-02-01 Epub Date: 2024-10-08 DOI: 10.1212/CPJ.0000000000200380
Kathryn A Wyman-Chick, Tanis J Ferman, Daniel Weintraub, Melissa J Armstrong, Bradley F Boeve, Ece Bayram, Ella Chrenka, Matthew J Barrett

Background and objectives: 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.

Methods: 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.

Results: 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.

Discussion: 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.

背景和目的:区分路易体痴呆(DLB)和阿尔茨海默病(AD)在临床上具有挑战性。随着以减缓或阻止错误折叠蛋白聚集为目标的潜在疗法的出现,必须能够在疾病致残之前识别出患者。在 DLB 和 AD 的临床前或前驱阶段区分 DLB 和 AD 变得更加重要。需要进行研究来验证所提出的DLB前驱期标准:纵向数据来自国家阿尔茨海默氏症协调中心的统一数据集。纳入的参与者基线诊断为正常或轻度认知障碍,并连续两年随访诊断为DLB或AD。我们研究了核心 DLB 临床特征、支持性神经精神特征以及痴呆诊断前 2 年的神经心理学数据是否将 DLB 与 AD 区分开来:我们发现了 143 名 DLB 患者和 429 名年龄/性别匹配的 AD 患者。在痴呆诊断前一年出现 2 个或 2 个以上 DLB 核心特征的 AUC(0.793;95% CI 0.748-0.839)最高,可将前驱 DLB 与前驱 AD 区分开来。与AD相比,DLB患者在确诊痴呆症至少2年前就出现了明显的睡眠障碍、幻觉以及处理速度、注意力和视觉建构能力下降等认知特征:这个多地点、纵向、特征明确的北美研究队列的数据支持了最近公布的前驱DLB标准的有效性。在前驱阶段,后来发展为DLB的患者更有可能具有DLB的核心临床特征,其注意力、处理速度和视觉空间表现也比后来发展为AD的患者差。在痴呆症出现之前区分 DLB 和 AD,为早期进行疾病特异性干预和整体管理提供了机会。
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引用次数: 0
Patient- and Caregiver-Reported Impact of Symptoms in Alzheimer Disease, Mild Cognitive Impairment, and Dementia. 患者和护理人员报告的阿尔茨海默病、轻度认知障碍和痴呆症状的影响
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2025-02-01 Epub Date: 2024-12-06 DOI: 10.1212/CPJ.0000000000200418
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

Background and objectives: 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.

Methods: 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.

Results: 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.

Discussion: 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.

背景和目的:在准备未来涉及阿尔茨海默病(AD)、轻度认知障碍(MCI)和痴呆患者的临床试验时,从患者和护理者的直接角度确定症状的广泛影响是很重要的。在这项研究中,我们使用大规模的患者和护理人员数据进行横断面调查,以确定患有AD、MCI和痴呆的成年人所经历的症状和症状主题的患病率和平均影响。随后的分析用于确定哪些人口统计学和疾病特异性因素与更严重的疾病相关。方法:15名患有AD (6), MCI(8)和痴呆(1)的成年人和15名患有AD (7), MCI(6)和痴呆(2)的成年人的护理人员参与了定性访谈,提供了1166和1097个与症状负担有关的独特报价。使用综合访谈指南中的开放式问题,参与者被要求确定对他们的生活或他们所提供护理的个人的生活影响最大的AD症状。然后进行横断面调查,询问在初步定性访谈中确定的重要潜在症状。433个人(患者和护理人员)参与了横断面调查,提供了超过35,000个症状评级反应。随后进行分析,以确定人口统计学和疾病特异性特征如何与症状主题患病率相关。结果:在横断面调查中,AD、MCI和痴呆患者报告的最常见症状主题是记忆问题(99.0%)、思维问题(90.3%)和沟通困难(80.4%)。患者认为在社交场合满意度下降(1.45)、疲劳(1.45)和记忆问题(1.41)是影响最大的症状主题(范围0-4)。患者报告的症状主题患病率与神经功能障碍的改良兰金量表(mRS)密切相关。讨论:患有AD、MCI和痴呆的个体会经历各种显著影响其日常生活的症状。这些症状,许多未被认识到,对患有这些疾病的个体具有不同的重要性,可能为未来治疗干预的潜在目标提供信息,并促进疾病特异性结果测量的发展和验证。
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引用次数: 0
Do I Have ADHD? Diagnosis of ADHD in Adulthood and Its Mimics in the Neurology Clinic. 我有多动症吗?成人ADHD的诊断及其在神经病学临床的模拟。
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2025-02-01 Epub Date: 2024-12-17 DOI: 10.1212/CPJ.0000000000200433
Susanna B Mierau

Attention-deficit/hyperactivity disorder (ADHD) is a lifelong neurodevelopmental disorder that causes difficulties with sustained attention, executive functioning, impulsivity, hyperactivity, and/or emotional regulation. Although many people are diagnosed with ADHD in childhood, others seek diagnosis in adulthood. Many adults have already reviewed available clinical scales or screening tools for ADHD and are referred for evaluation of attention problems by their primary care providers. Key features of the history and examination in a clinic visit can differentiate ADHD from other causes of attention problems in adults. Treatment with stimulant or nonstimulant medications for ADHD can be life-changing for adults with ADHD, increasing productivity at home and work, reducing anxiety and impulsive behaviors, and improving interpersonal and community relationships. This article aids neurologists in differentiating ADHD from other causes of attention and executive functioning problems in adults and in initiating treatment.

注意力缺陷/多动障碍(ADHD)是一种终生的神经发育障碍,它会导致持续注意力、执行功能、冲动、多动和/或情绪调节方面的困难。虽然许多人在童年时被诊断患有多动症,但其他人在成年后才寻求诊断。许多成年人已经回顾了现有的ADHD临床量表或筛查工具,并由他们的初级保健提供者推荐对注意力问题进行评估。病史和门诊检查的关键特征可以将ADHD与其他引起成人注意力问题的原因区分开来。使用兴奋剂或非兴奋剂药物治疗ADHD可以改变ADHD成年人的生活,提高家庭和工作效率,减少焦虑和冲动行为,改善人际关系和社区关系。这篇文章帮助神经科医生区分ADHD与其他引起成人注意力和执行功能问题的原因,并开始治疗。
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引用次数: 0
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Neurology. Clinical practice
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