Pub Date : 2024-10-01Epub Date: 2024-06-12DOI: 10.1212/CPJ.0000000000200333
Thy P Nguyen, Deepa Dongarwar, Rajesh K Gupta, Suur Biliciler, Kazim A Sheikh
Background and objectives: This study presents results from a survey of physicians performing electrodiagnostic studies to assess average volume. We also assessed how different factors (trainees, technologists, age of the physician, and case complexity) affected volume. Productivity is an important factor for physicians across practice settings. However, unlike evaluation and management services for neurologists, there are no published data for benchmarks of average volume of electrodiagnostic studies.
Methods: A 34-question survey was designed collecting information on demographics, electrodiagnostic study volume, technologists, trainees, referrals, and case complexity. The anonymous survey was disseminated through a QR code or hyperlink to multiple online neurology, physical medicine and rehabilitation, electromyography, and neuromuscular forums. The primary outcome was EMG volume including number of EMGs per half-day and EMG volume per year. We conducted bivariate association analysis between primary outcomes and respondent characteristics using the Pearson χ2 test. Multivariable regression models determined factors associated with each of our outcome variables.
Results: A total of 201 respondents initiated the survey. 71% were certified in adult neurology, 19.6% in physical medicine and rehabilitation, and 2.7% in pediatric neurology. 37.5% practiced in academic medicine. The remaining respondents were from private practice, group, solo, hospital employed, or other. 83% of respondents allotted a dedicated half-day to performing EMGs. The median number of EMGs scheduled during a half-day was within 3-4 (45%). 30% and 7% scheduled 5-6 or more than 7 patients per half-day, respectively. The median number of EMGs performed per year was within 251-500 (37%).
Discussion: This national, cross-sectional survey evaluates average metrics of EMG volume. Our survey showed that the median number of EMGs annually lies between 251 and 500 studies (37%). In addition, for those respondents who allotted a dedicated half-day to performing EMGs, the median number of EMG studies scheduled per half-day lies between 3 and 4 studies (45%). In multivariate analysis, respondent characteristics of age of the physician (older than 45), working with nerve conduction technologists, and holding the position of EMG director were associated with increased EMG volume.
{"title":"A National Cross-Sectional Survey of EMG Physician Volume.","authors":"Thy P Nguyen, Deepa Dongarwar, Rajesh K Gupta, Suur Biliciler, Kazim A Sheikh","doi":"10.1212/CPJ.0000000000200333","DOIUrl":"10.1212/CPJ.0000000000200333","url":null,"abstract":"<p><strong>Background and objectives: </strong>This study presents results from a survey of physicians performing electrodiagnostic studies to assess average volume. We also assessed how different factors (trainees, technologists, age of the physician, and case complexity) affected volume. Productivity is an important factor for physicians across practice settings. However, unlike evaluation and management services for neurologists, there are no published data for benchmarks of average volume of electrodiagnostic studies.</p><p><strong>Methods: </strong>A 34-question survey was designed collecting information on demographics, electrodiagnostic study volume, technologists, trainees, referrals, and case complexity. The anonymous survey was disseminated through a QR code or hyperlink to multiple online neurology, physical medicine and rehabilitation, electromyography, and neuromuscular forums. The primary outcome was EMG volume including number of EMGs per half-day and EMG volume per year. We conducted bivariate association analysis between primary outcomes and respondent characteristics using the Pearson χ<sup>2</sup> test. Multivariable regression models determined factors associated with each of our outcome variables.</p><p><strong>Results: </strong>A total of 201 respondents initiated the survey. 71% were certified in adult neurology, 19.6% in physical medicine and rehabilitation, and 2.7% in pediatric neurology. 37.5% practiced in academic medicine. The remaining respondents were from private practice, group, solo, hospital employed, or other. 83% of respondents allotted a dedicated half-day to performing EMGs. The median number of EMGs scheduled during a half-day was within 3-4 (45%). 30% and 7% scheduled 5-6 or more than 7 patients per half-day, respectively. The median number of EMGs performed per year was within 251-500 (37%).</p><p><strong>Discussion: </strong>This national, cross-sectional survey evaluates average metrics of EMG volume. Our survey showed that the median number of EMGs annually lies between 251 and 500 studies (37%). In addition, for those respondents who allotted a dedicated half-day to performing EMGs, the median number of EMG studies scheduled per half-day lies between 3 and 4 studies (45%). In multivariate analysis, respondent characteristics of age of the physician (older than 45), working with nerve conduction technologists, and holding the position of EMG director were associated with increased EMG volume.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11194787/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141450990","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}
Pub Date : 2024-10-01Epub Date: 2024-06-18DOI: 10.1212/CPJ.0000000000200332
Gwen Zeigler, Cole A Harrington, Nicole Rosendale, Christos Ganos, Valeria Roldan, Anna Pace, Sasha Alick-Lindstrom, Casey Orozco-Poore, Wissam Deeb, Margaret L Hansen, Z Paige L'Erario
Purpose of review: To summarize the literature on neurologic care for transgender and gender-diverse (TGD) people and provide implications for clinical practice.
Recent findings: There are limited data on the frequency and management of neurologic conditions among TGD people. TGD people have a higher prevalence of various neurologic conditions compared with cisgender or general population cohorts, including migraine, subjective cognitive decline, sleep disturbances, functional disorders, and cerebrovascular disease. Gender-affirming hormone therapy interacts with commonly prescribed neurologic medications and increases stroke risk among transfeminine people. Sex hormones and sex chromosomes may play a role in neurodegeneration and disability progression in neuroimmunologic diseases. Clitoral reduction surgeries on intersex children can cause neurologic disability and sexual dysfunction in adulthood. Socioeconomic disparities among TGD people contribute to health care barriers.
Summary: Neurologists should consider the unique experiences and health care needs of TGD people in their clinical practice and research protocols.
{"title":"Neurologic Care for Transgender and Gender-Diverse People: A Review of Current Evidence and Clinical Implications.","authors":"Gwen Zeigler, Cole A Harrington, Nicole Rosendale, Christos Ganos, Valeria Roldan, Anna Pace, Sasha Alick-Lindstrom, Casey Orozco-Poore, Wissam Deeb, Margaret L Hansen, Z Paige L'Erario","doi":"10.1212/CPJ.0000000000200332","DOIUrl":"10.1212/CPJ.0000000000200332","url":null,"abstract":"<p><strong>Purpose of review: </strong>To summarize the literature on neurologic care for transgender and gender-diverse (TGD) people and provide implications for clinical practice.</p><p><strong>Recent findings: </strong>There are limited data on the frequency and management of neurologic conditions among TGD people. TGD people have a higher prevalence of various neurologic conditions compared with cisgender or general population cohorts, including migraine, subjective cognitive decline, sleep disturbances, functional disorders, and cerebrovascular disease. Gender-affirming hormone therapy interacts with commonly prescribed neurologic medications and increases stroke risk among transfeminine people. Sex hormones and sex chromosomes may play a role in neurodegeneration and disability progression in neuroimmunologic diseases. Clitoral reduction surgeries on intersex children can cause neurologic disability and sexual dysfunction in adulthood. Socioeconomic disparities among TGD people contribute to health care barriers.</p><p><strong>Summary: </strong>Neurologists should consider the unique experiences and health care needs of TGD people in their clinical practice and research protocols.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11195436/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141450994","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}
Pub Date : 2024-10-01Epub Date: 2024-06-11DOI: 10.1212/CPJ.0000000000200314
Scott Friedenberg, Edward Stefanowicz, Timothy Frymoyer, Clemens M Schirmer, Neil R Holland, Trudi Dempsey
Background: The combination of inadequate financial training, limited benchmarks, and mindset contribute to many physicians prioritizing revenue below quality, outcomes, and safety. This creates a challenge as hospital administrators aim to motivate clinicians to improve RVU generation and increase revenue.
Recent findings: Creating physician/administrator teams that defines and explores the gap between observed and expected financial performance in parallel with appreciating the physician's practice preferences can create new opportunities for billing. The proposed 3 phase approach emphasizes nonjudgmental communication, education and partnership. The most common and effective opportunities for improvement include billing optimization, scheduling and system infrastructure modifications.
Implications for practice: As reimbursement decrease, balancing revenue generation with physician satisfaction has become paramount. Promoting data drive bidirectional communication can lead to identifying previously unrecognized billing opportunities where change is driven by providers rather than by 1-dimensional institutional goals.
{"title":"Empowering Health Care Providers: A Collaborative Approach to Enhance Financial Performance and Productivity in Clinical Practice.","authors":"Scott Friedenberg, Edward Stefanowicz, Timothy Frymoyer, Clemens M Schirmer, Neil R Holland, Trudi Dempsey","doi":"10.1212/CPJ.0000000000200314","DOIUrl":"10.1212/CPJ.0000000000200314","url":null,"abstract":"<p><strong>Background: </strong>The combination of inadequate financial training, limited benchmarks, and mindset contribute to many physicians prioritizing revenue below quality, outcomes, and safety. This creates a challenge as hospital administrators aim to motivate clinicians to improve RVU generation and increase revenue.</p><p><strong>Recent findings: </strong>Creating physician/administrator teams that defines and explores the gap between observed and expected financial performance in parallel with appreciating the physician's practice preferences can create new opportunities for billing. The proposed 3 phase approach emphasizes nonjudgmental communication, education and partnership. The most common and effective opportunities for improvement include billing optimization, scheduling and system infrastructure modifications.</p><p><strong>Implications for practice: </strong>As reimbursement decrease, balancing revenue generation with physician satisfaction has become paramount. Promoting data drive bidirectional communication can lead to identifying previously unrecognized billing opportunities where change is driven by providers rather than by 1-dimensional institutional goals.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11194789/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141446630","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}
Pub Date : 2024-10-01Epub Date: 2024-08-15DOI: 10.1212/CPJ.0000000000200324
Hector Arciniega, Leonard B Jung, Fatima Tuz-Zahra, Yorghos Tripodis, Omar John, Nicholas Kim, Holly W Carrington, Evdokiya E Knyazhanskaya, Arushi Chamaria, Katherine Breedlove, Tim L Wiegand, Daniel Daneshvar, Tashrif Billah, Ofer Pasternak, Michael J Coleman, Charles H Adler, Charles Bernick, Laura J Balcer, Michael L Alosco, Alexander P Lin, Inga K Koerte, Jeffrey L Cummings, Eric M Reiman, Robert A Stern, Sylvain Bouix, Martha E Shenton
Background and objectives: Exposure to repetitive head impacts (RHI) is linked to the development of chronic traumatic encephalopathy (CTE), which can only be diagnosed at post-mortem. The presence of a cavum septum pellucidum (CSP) is a common finding in post-mortem studies of confirmed CTE and in neuroimaging studies of individuals exposed to RHI. This study examines CSP in living former American football players, investigating its association with RHI exposure, traumatic encephalopathy syndrome (TES) diagnosis, and provisional levels of certainty for CTE pathology.
Methods: Data from the DIAGNOSE CTE Research Project were used to compare the presence and ratio of CSP in former American football players (n = 175), consisting of former college (n = 58) and former professional players (n = 117), and asymptomatic unexposed controls without RHI exposure (n = 55). We further evaluated potential associations between CSP measures and cumulative head impact index (CHII) measures (frequency, linear acceleration, and rotational force), a TES diagnosis (yes/no), and a provisional level of certainty for CTE pathology (suggestive, possible, and probable).
Results: Former American football players exhibited a higher CSP presence and ratio than unexposed asymptomatic controls. Among player subgroups, professional players showed a greater CSP ratio than former college players and unexposed asymptomatic controls. Among all football players, CHII rotational forces correlated with an increased CSP ratio. No significant associations were found between CSP measures and diagnosis of TES or provisional levels of certainty for CTE pathology.
Discussion: This study confirms previous findings, highlighting a greater prevalence of CSP and a greater CSP ratio in former American football players compared with unexposed asymptomatic controls. In addition, former professional players showed a greater CSP ratio than college players. Moreover, the relationship between estimates of CHII rotational forces and CSP measures suggests that cumulative frequency and strength of rotational forces experienced in football are associated with CSP. However, CSP does not directly correlate with TES diagnosis or provisional levels of certainty for CTE, indicating that it may be a consequence of RHI associated with rotational forces. Further research, especially longitudinal studies, is needed for confirmation and to explore changes over time.
{"title":"Cavum Septum Pellucidum in Former American Football Players: Findings From the DIAGNOSE CTE Research Project.","authors":"Hector Arciniega, Leonard B Jung, Fatima Tuz-Zahra, Yorghos Tripodis, Omar John, Nicholas Kim, Holly W Carrington, Evdokiya E Knyazhanskaya, Arushi Chamaria, Katherine Breedlove, Tim L Wiegand, Daniel Daneshvar, Tashrif Billah, Ofer Pasternak, Michael J Coleman, Charles H Adler, Charles Bernick, Laura J Balcer, Michael L Alosco, Alexander P Lin, Inga K Koerte, Jeffrey L Cummings, Eric M Reiman, Robert A Stern, Sylvain Bouix, Martha E Shenton","doi":"10.1212/CPJ.0000000000200324","DOIUrl":"10.1212/CPJ.0000000000200324","url":null,"abstract":"<p><strong>Background and objectives: </strong>Exposure to repetitive head impacts (RHI) is linked to the development of chronic traumatic encephalopathy (CTE), which can only be diagnosed at post-mortem. The presence of a cavum septum pellucidum (CSP) is a common finding in post-mortem studies of confirmed CTE and in neuroimaging studies of individuals exposed to RHI. This study examines CSP in living former American football players, investigating its association with RHI exposure, traumatic encephalopathy syndrome (TES) diagnosis, and provisional levels of certainty for CTE pathology.</p><p><strong>Methods: </strong>Data from the DIAGNOSE CTE Research Project were used to compare the presence and ratio of CSP in former American football players (n = 175), consisting of former college (n = 58) and former professional players (n = 117), and asymptomatic unexposed controls without RHI exposure (n = 55). We further evaluated potential associations between CSP measures and cumulative head impact index (CHII) measures (frequency, linear acceleration, and rotational force), a TES diagnosis (yes/no), and a provisional level of certainty for CTE pathology (suggestive, possible, and probable).</p><p><strong>Results: </strong>Former American football players exhibited a higher CSP presence and ratio than unexposed asymptomatic controls. Among player subgroups, professional players showed a greater CSP ratio than former college players and unexposed asymptomatic controls. Among all football players, CHII rotational forces correlated with an increased CSP ratio. No significant associations were found between CSP measures and diagnosis of TES or provisional levels of certainty for CTE pathology.</p><p><strong>Discussion: </strong>This study confirms previous findings, highlighting a greater prevalence of CSP and a greater CSP ratio in former American football players compared with unexposed asymptomatic controls. In addition, former professional players showed a greater CSP ratio than college players. Moreover, the relationship between estimates of CHII rotational forces and CSP measures suggests that cumulative frequency and strength of rotational forces experienced in football are associated with CSP. However, CSP does not directly correlate with TES diagnosis or provisional levels of certainty for CTE, indicating that it may be a consequence of RHI associated with rotational forces. Further research, especially longitudinal studies, is needed for confirmation and to explore changes over time.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11332980/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142004872","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}
Pub Date : 2024-10-01Epub Date: 2024-06-10DOI: 10.1212/CPJ.0000000000200323
Anelyssa D'Abreu, Azziza Bankole, Jaideep Kapur, Carol A Manning, Pavel Chernyavskiy
Background and objectives: The Area Deprivation Index (ADI) provides a validated and multidimensional metric of areal disadvantage. Our goals were to determine if the ADI influences the likelihood of receiving workup based on published guidelines and an etiologic diagnosis of dementia in Central and Western Virginia.
Methods: We collected deidentified data from the electronic health record of individuals aged 50-105 years diagnosed with dementia at the University of Virginia (UVA) Medical Center (2016-2021) and at Carillion Clinic (2018-2021). Visit-specific ICD-10 codes were used to classify each dementia diagnosis as "disease-specific" (e.g., Alzheimer disease) or "general" (e.g., unspecified dementia). Following the American Academy of Neurology guidelines, we considered the evaluation performed as "adequate" if patients had vitamin B12, thyroid-stimulating hormone, and brain CT or magnetic resonance imaging within 6 months of the initial diagnosis. Census tract ADI was linked to study participants using the unique census tract identifier derived from the participants' home addresses at the time of diagnosis. Statistical modeling occurred under a Bayesian paradigm implemented using a standard code in R.
Results: The study included 13,431 individuals diagnosed with dementia at UVA (n = 7,152) and Carillion Clinic (n = 6,279). Of those, 32.5% and 20.4% received "disease-specific" diagnoses at UVA and Carillion Clinic and 8.2% and 20.4% underwent "adequate" workup, respectively. The adjusted relationship between census tract ADI and the likelihood of a disease-specific diagnosis was U-shaped: Residence in moderately disadvantaged areas was associated with the lowest likelihood of disease-specific diagnosis.
Discussion: Most patients diagnosed with dementia did not receive an adequate evaluation or an etiologic diagnosis. Those living in locations just above the national median ADI levels had the lowest likelihood of receiving an etiologic diagnosis, lower than those in the least and most deprived areas. Renewed awareness efforts among providers are needed to increase compliance with diagnostic guidelines.
背景和目标:地区贫困指数(ADI)是一种经过验证的多维度地区贫困指标。我们的目标是确定 ADI 是否会影响弗吉尼亚州中部和西部地区根据已发布的指南和痴呆病因诊断接受检查的可能性:我们从弗吉尼亚大学(UVA)医疗中心(2016-2021年)和Carillion诊所(2018-2021年)诊断出患有痴呆症的50-105岁患者的电子健康记录中收集了去身份化数据。使用特定就诊的 ICD-10 编码将每项痴呆诊断分为 "特定疾病"(如阿尔茨海默病)或 "一般疾病"(如未指定的痴呆)。根据美国神经病学学会的指导方针,如果患者在初次诊断后 6 个月内进行了维生素 B12、促甲状腺激素和脑 CT 或磁共振成像检查,我们则认为所进行的评估是 "充分的"。人口普查区 ADI 与研究参与者相关联,使用的是根据参与者诊断时家庭住址得出的唯一人口普查区标识符。统计建模采用贝叶斯范式,使用 R 语言的标准代码实现:研究包括 13,431 名在弗吉尼亚大学(n = 7,152 人)和 Carillion 诊所(n = 6,279 人)确诊为痴呆症的患者。其中,32.5%和20.4%的患者在UVA和Carillion诊所接受了 "特定疾病 "诊断,8.2%和20.4%的患者接受了 "充分 "检查。人口普查区 ADI 与疾病特异性诊断可能性之间的调整关系呈 U 型:居住在中度贫困地区的患者获得疾病特异性诊断的可能性最低:讨论:大多数被诊断为痴呆症的患者没有得到充分的评估或病因诊断。那些居住在略高于全国平均每日生活费中位数水平的地区的患者得到病因诊断的可能性最低,低于那些居住在最贫困和最贫困地区的患者。需要提高医疗服务提供者的认识,以加强对诊断指南的遵守。
{"title":"Association of the Area Deprivation Index With Dementia Basic Workup and Diagnosis in Central and Western Virginia: A Cross-Sectional Study.","authors":"Anelyssa D'Abreu, Azziza Bankole, Jaideep Kapur, Carol A Manning, Pavel Chernyavskiy","doi":"10.1212/CPJ.0000000000200323","DOIUrl":"10.1212/CPJ.0000000000200323","url":null,"abstract":"<p><strong>Background and objectives: </strong>The Area Deprivation Index (ADI) provides a validated and multidimensional metric of areal disadvantage. Our goals were to determine if the ADI influences the likelihood of receiving workup based on published guidelines and an etiologic diagnosis of dementia in Central and Western Virginia.</p><p><strong>Methods: </strong>We collected deidentified data from the electronic health record of individuals aged 50-105 years diagnosed with dementia at the University of Virginia (UVA) Medical Center (2016-2021) and at Carillion Clinic (2018-2021). Visit-specific ICD-10 codes were used to classify each dementia diagnosis as \"disease-specific\" (e.g., Alzheimer disease) or \"general\" (e.g., unspecified dementia). Following the American Academy of Neurology guidelines, we considered the evaluation performed as \"adequate\" if patients had vitamin B12, thyroid-stimulating hormone, and brain CT or magnetic resonance imaging within 6 months of the initial diagnosis. Census tract ADI was linked to study participants using the unique census tract identifier derived from the participants' home addresses at the time of diagnosis. Statistical modeling occurred under a Bayesian paradigm implemented using a standard code in R.</p><p><strong>Results: </strong>The study included 13,431 individuals diagnosed with dementia at UVA (n = 7,152) and Carillion Clinic (n = 6,279). Of those, 32.5% and 20.4% received \"disease-specific\" diagnoses at UVA and Carillion Clinic and 8.2% and 20.4% underwent \"adequate\" workup, respectively. The adjusted relationship between census tract ADI and the likelihood of a disease-specific diagnosis was U-shaped: Residence in moderately disadvantaged areas was associated with the lowest likelihood of disease-specific diagnosis.</p><p><strong>Discussion: </strong>Most patients diagnosed with dementia did not receive an adequate evaluation or an etiologic diagnosis. Those living in locations just above the national median ADI levels had the lowest likelihood of receiving an etiologic diagnosis, lower than those in the least and most deprived areas. Renewed awareness efforts among providers are needed to increase compliance with diagnostic guidelines.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11195434/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141450991","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}
Pub Date : 2024-10-01Epub Date: 2024-06-12DOI: 10.1212/CPJ.0000000000200335
Jennifer Morganroth, Leah Zuroff, Amanda C Guidon, Grant T Liu, Shawn J Bird, Sunil Singhal, Gil I Wolfe, Ali G Hamedani
Background and objectives: In 2016, a randomized controlled trial demonstrated the clinical efficacy of trans-sternal thymectomy for patients with non-thymomatous myasthenia gravis (MG). Whether large-scale changes occurred in clinical practice after this trial is unknown.
Methods: We performed a retrospective longitudinal cross-sectional analysis using National Inpatient Sample (NIS) data from 2012 to 2019. Our study included hospitalized adults at least 18 years of age diagnosed with MG without an associated thymoma. We used joinpoint regression to analyze annual trends in thymectomy volume and surgical approach (minimally invasive vs trans-sternal) from 2012 to 2019. Using logistic regression models, we examined patient and hospital-level factors that may have influenced whether thymectomy was performed, such as age, sex, race, insurance payor, hospital size and teaching status, and Elixhauser Comorbidity Index. Sampling weights were applied to account for the complex survey design of NIS.
Results: The total number of thymectomy procedures increased by 69.8% per year (95% CI 40.1-105.8) between 2012 and 2019. Trans-sternal thymectomies increased by 62.8% per year (95% CI 35.8-95.2) and minimally invasive thymectomies by 83.7% per year (95% CI 38.1-144.3). Thymectomies were significantly more likely to occur in 2017-2019 compared with 2012-2016 (OR 1.93, 95% CI 1.62-2.31). In a multivariable regression model, several factors decreased the odds of patients with MG having a thymectomy: older age, Black race (OR 0.62, 95% CI 0.49-0.77), female (OR 0.73, 95% CI 0.63-0.86), and higher Elixhauser Comorbidity Index. Patients in medium (OR 1.82, 95% CI 1.30-2.55) or large (OR 2.81, 95% CI 2.07-3.82) size and urban teaching hospitals (OR 6.09, 95% CI 2.65-13.97) were more likely to undergo thymectomy.
Discussion: Thymectomy is being performed more frequently for non-thymomatous MG, especially after 2016 after publication of a positive phase III clinical trial. There are several disparities in thymectomy utilization that warrant further attention.
背景和目的:2016年,一项随机对照试验证明了经胸骨胸腺切除术对非胸腺瘤性肌无力(MG)患者的临床疗效。该试验后,临床实践是否发生了大规模的变化尚不得而知:我们利用 2012 年至 2019 年的全国住院患者样本(NIS)数据进行了一项回顾性纵向横断面分析。我们的研究纳入了至少 18 岁、被诊断为 MG 且未伴有胸腺瘤的住院成人。我们使用连接点回归分析了 2012 年至 2019 年胸腺切除术量和手术方式(微创与经胸腔)的年度趋势。利用逻辑回归模型,我们研究了可能影响是否进行胸腺切除术的患者和医院层面的因素,如年龄、性别、种族、保险支付方、医院规模和教学状况以及埃利克豪斯综合指数。考虑到 NIS 复杂的调查设计,采用了抽样权重:2012年至2019年期间,胸腺切除术的总数每年增加69.8%(95% CI 40.1-105.8)。经胸骨胸腺切除术每年增加 62.8%(95% CI 35.8-95.2),微创胸腺切除术每年增加 83.7%(95% CI 38.1-144.3)。与 2012-2016 年相比,2017-2019 年胸腺切除术的发生率明显更高(OR 1.93,95% CI 1.62-2.31)。在多变量回归模型中,有几个因素降低了MG患者进行胸腺切除术的几率:年龄较大、黑人种族(OR 0.62,95% CI 0.49-0.77)、女性(OR 0.73,95% CI 0.63-0.86)和Elixhauser综合指数较高。中型(OR 1.82,95% CI 1.30-2.55)或大型(OR 2.81,95% CI 2.07-3.82)医院和城市教学医院(OR 6.09,95% CI 2.65-13.97)的患者更有可能接受胸腺切除术:胸腺切除术越来越多地用于非胸腺肿瘤性MG,尤其是在2016年一项积极的III期临床试验公布后。在胸腺切除术的使用方面存在一些差异,值得进一步关注。
{"title":"Trends and Disparities in the Utilization of Thymectomy for Myasthenia Gravis in the United States.","authors":"Jennifer Morganroth, Leah Zuroff, Amanda C Guidon, Grant T Liu, Shawn J Bird, Sunil Singhal, Gil I Wolfe, Ali G Hamedani","doi":"10.1212/CPJ.0000000000200335","DOIUrl":"10.1212/CPJ.0000000000200335","url":null,"abstract":"<p><strong>Background and objectives: </strong>In 2016, a randomized controlled trial demonstrated the clinical efficacy of trans-sternal thymectomy for patients with non-thymomatous myasthenia gravis (MG). Whether large-scale changes occurred in clinical practice after this trial is unknown.</p><p><strong>Methods: </strong>We performed a retrospective longitudinal cross-sectional analysis using National Inpatient Sample (NIS) data from 2012 to 2019. Our study included hospitalized adults at least 18 years of age diagnosed with MG without an associated thymoma. We used joinpoint regression to analyze annual trends in thymectomy volume and surgical approach (minimally invasive vs trans-sternal) from 2012 to 2019. Using logistic regression models, we examined patient and hospital-level factors that may have influenced whether thymectomy was performed, such as age, sex, race, insurance payor, hospital size and teaching status, and Elixhauser Comorbidity Index. Sampling weights were applied to account for the complex survey design of NIS.</p><p><strong>Results: </strong>The total number of thymectomy procedures increased by 69.8% per year (95% CI 40.1-105.8) between 2012 and 2019. Trans-sternal thymectomies increased by 62.8% per year (95% CI 35.8-95.2) and minimally invasive thymectomies by 83.7% per year (95% CI 38.1-144.3). Thymectomies were significantly more likely to occur in 2017-2019 compared with 2012-2016 (OR 1.93, 95% CI 1.62-2.31). In a multivariable regression model, several factors decreased the odds of patients with MG having a thymectomy: older age, Black race (OR 0.62, 95% CI 0.49-0.77), female (OR 0.73, 95% CI 0.63-0.86), and higher Elixhauser Comorbidity Index. Patients in medium (OR 1.82, 95% CI 1.30-2.55) or large (OR 2.81, 95% CI 2.07-3.82) size and urban teaching hospitals (OR 6.09, 95% CI 2.65-13.97) were more likely to undergo thymectomy.</p><p><strong>Discussion: </strong>Thymectomy is being performed more frequently for non-thymomatous MG, especially after 2016 after publication of a positive phase III clinical trial. There are several disparities in thymectomy utilization that warrant further attention.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11194790/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141450995","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}
Pub Date : 2024-10-01Epub Date: 2024-06-10DOI: 10.1212/CPJ.0000000000200330
Andrew B Wolf, John R Corboy
Objectives: Disease-modifying therapy (DMT) for multiple sclerosis (MS) after natalizumab-associated progressive multifocal leukoencephalopathy (PML) is controversial due to concern for recurrent PML. We describe DMT utilization for over a decade in a patient with MS who survived PML.
Methods: Case report.
Results: A 36-year-old woman was diagnosed with MS in 2002 and treated with interferon beta-1a until 2006, when she transitioned to natalizumab due to relapses. She presented in 2012 with 2 months of progressive cognitive and gait concerns and was diagnosed with PML by positive CSF JC virus testing with concordant clinical and MRI findings. She was treated with plasma exchange and then corticosteroids for PML immune reconstitution inflammatory syndrome before starting glatiramer acetate for DMT. She transitioned to dimethyl fumarate in 2013 after MS activity on MRI with negative CSF JC virus testing. Owing to worsening footdrop consistent with progression, she transitioned to ocrelizumab in 2017 and then to ofatumumab in 2020 due to logistics of medication administration. There has been no clinicoradiographic or CSF evidence of recurrent PML.
Discussion: DMT selection is challenging for patients with MS who survive PML. We used an escalation approach extending to ocrelizumab and ofatumumab due to MS progression. Anti-CD20 DMTs are a high-efficacy option post-PML.
Classification of evidence: This provides Class IV evidence. It is a single observational study without controls.
{"title":"Escalation to Anti-CD20 Treatment for Multiple Sclerosis Following Natalizumab-Associated Progressive Multifocal Leukoencephalopathy.","authors":"Andrew B Wolf, John R Corboy","doi":"10.1212/CPJ.0000000000200330","DOIUrl":"10.1212/CPJ.0000000000200330","url":null,"abstract":"<p><strong>Objectives: </strong>Disease-modifying therapy (DMT) for multiple sclerosis (MS) after natalizumab-associated progressive multifocal leukoencephalopathy (PML) is controversial due to concern for recurrent PML. We describe DMT utilization for over a decade in a patient with MS who survived PML.</p><p><strong>Methods: </strong>Case report.</p><p><strong>Results: </strong>A 36-year-old woman was diagnosed with MS in 2002 and treated with interferon beta-1a until 2006, when she transitioned to natalizumab due to relapses. She presented in 2012 with 2 months of progressive cognitive and gait concerns and was diagnosed with PML by positive CSF JC virus testing with concordant clinical and MRI findings. She was treated with plasma exchange and then corticosteroids for PML immune reconstitution inflammatory syndrome before starting glatiramer acetate for DMT. She transitioned to dimethyl fumarate in 2013 after MS activity on MRI with negative CSF JC virus testing. Owing to worsening footdrop consistent with progression, she transitioned to ocrelizumab in 2017 and then to ofatumumab in 2020 due to logistics of medication administration. There has been no clinicoradiographic or CSF evidence of recurrent PML.</p><p><strong>Discussion: </strong>DMT selection is challenging for patients with MS who survive PML. We used an escalation approach extending to ocrelizumab and ofatumumab due to MS progression. Anti-CD20 DMTs are a high-efficacy option post-PML.</p><p><strong>Classification of evidence: </strong>This provides Class IV evidence. It is a single observational study without controls.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11194788/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141450992","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}
Pub Date : 2024-10-01Epub Date: 2024-08-15DOI: 10.1212/CPJ.0000000000200322
Miranda M Wan, Nora D Cristall, Lara J Cooke
Background and objectives: Despite significant advances in the treatment of neurologic disorders, many conditions require complex care planning and advanced care planning. Neurologists are in a unique position because they are integral in providing patient centered care, understanding neurologic disease and illness trajectory, and how disease can affect patients' sense of self and values. Currently, little is known about neurologists' perceptions and challenges in care planning and palliative care for their patients.
Methods: Neurologists from one Canadian academic institution participated in a 30-minute semistructured interview from November 2022 to April 2023. Interviews were conducted until saturation was reached and confirmed. Interviews occurred online through a secure platform or in-person and were recorded. Data were analyzed using a constant comparative method using constructivist grounded theory. Member checking was conducted post interview.
Results: Ten neurologists participated across a broad spectrum of neurology experience and subspecialties. We developed a detailed theory of understanding neurologists' attitudes and perceptions of palliative care. When neurologists delay or fail to initiate care planning discussions or palliative care, it results from a complex interplay between patient, physician, and resource accessibility factors. Certain contextual factors, such as a first visit or follow-up, inpatient vs outpatient setting, clinic culture, and the type of clinic practice, are factors that can influence these conversations. As a result, physicians may fail to use available resources, or they may involve other care providers or refer to subspecialty neurologic clinics. However, this delay can still lead to patient and provider harm. Opportunities to improve care exist with continuing education opportunities for trainees and staff, collaboration with palliative care specialists, and health systems support, such as increasing public awareness to address misconceptions about palliative care and resource availability.
Discussion: Our findings identify that failure or delay to initiate care planning and palliative care by neurologists results from a complex interplay between local culture, experience, context, practice type, and patient factors. Opportunities to improve care include increasing educational opportunities, building integrated and collaborative practices, and dedicated health systems support.
{"title":"Neurologists' Attitudes and Perceptions on Palliative Care: A Qualitative Study.","authors":"Miranda M Wan, Nora D Cristall, Lara J Cooke","doi":"10.1212/CPJ.0000000000200322","DOIUrl":"10.1212/CPJ.0000000000200322","url":null,"abstract":"<p><strong>Background and objectives: </strong>Despite significant advances in the treatment of neurologic disorders, many conditions require complex care planning and advanced care planning. Neurologists are in a unique position because they are integral in providing patient centered care, understanding neurologic disease and illness trajectory, and how disease can affect patients' sense of self and values. Currently, little is known about neurologists' perceptions and challenges in care planning and palliative care for their patients.</p><p><strong>Methods: </strong>Neurologists from one Canadian academic institution participated in a 30-minute semistructured interview from November 2022 to April 2023. Interviews were conducted until saturation was reached and confirmed. Interviews occurred online through a secure platform or in-person and were recorded. Data were analyzed using a constant comparative method using constructivist grounded theory. Member checking was conducted post interview.</p><p><strong>Results: </strong>Ten neurologists participated across a broad spectrum of neurology experience and subspecialties. We developed a detailed theory of understanding neurologists' attitudes and perceptions of palliative care. When neurologists delay or fail to initiate care planning discussions or palliative care, it results from a complex interplay between patient, physician, and resource accessibility factors. Certain contextual factors, such as a first visit or follow-up, inpatient vs outpatient setting, clinic culture, and the type of clinic practice, are factors that can influence these conversations. As a result, physicians may fail to use available resources, or they may involve other care providers or refer to subspecialty neurologic clinics. However, this delay can still lead to patient and provider harm. Opportunities to improve care exist with continuing education opportunities for trainees and staff, collaboration with palliative care specialists, and health systems support, such as increasing public awareness to address misconceptions about palliative care and resource availability.</p><p><strong>Discussion: </strong>Our findings identify that failure or delay to initiate care planning and palliative care by neurologists results from a complex interplay between local culture, experience, context, practice type, and patient factors. Opportunities to improve care include increasing educational opportunities, building integrated and collaborative practices, and dedicated health systems support.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11332981/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142009129","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}
Pub Date : 2024-10-01Epub Date: 2024-07-17DOI: 10.1212/CPJ.0000000000200329
Shumei Man, David Bruckman, Ken Uchino, Jesse D Schold, Jarrod Dalton
Background and objectives: Post-acute care is critical for patient functional recovery and successful community transition. This study aimed to understand the current racial, ethnic, and regional disparities in post-acute service utilization after stroke.
Methods: This retrospective cross-sectional study included patients hospitalized for ischemic stroke and intracerebral hemorrhage in 2017-2018 using the National Inpatient Sample. Discharge destinations were classified as follows: (1) facility including inpatient rehabilitation, skilled nursing facility, and facility hospice; (2) home health care (HHC), including home health and home hospice; and (3) home without HHC. Multinomial logistic regression was used to study the odds of discharge to a facility over home and HHC over home without HHC by race, ethnicity, insurance, and census division, adjusting for clinical factors and survey design.
Results: Among the 1,000,980 weighted ischemic stroke admissions, 66.9% were White, 17.6% Black, 9.5% Hispanic, 3.1% Asian American/Pacific Islander, and 0.4% Native American. Relative to private insurance, uninsured patients had the lowest adjusted odds of facility over home discharge (0.44; 95% CI 0.40-0.48) and HHC discharge over home without HHC (0.79; 95% CI 0.71-0.88). Compared with White patients, only Hispanic patients with Medicare/Medicaid insurance or self-pay had lower odds of facility over home discharge (adjusted OR 0.80 and 0.75, respectively; 95% CI 0.76-0.84 and 0.63-0.93). Uninsured Hispanic patients also had lower odds of HHC discharge over home without HHC than White patients (0.74; 95% CI 0.57-0.97). Facility discharge rate was the highest in East North Central (39.2%) and lowest in Pacific (31.2%). HHC discharge rate was the highest in New England (20.2%) and lowest in West North Central (10.3%), which had the highest home without HHC discharge (46.1%). Compared with New England, other census divisions had lower odds of facility over any home discharge with Pacific being the lowest (adjusted OR, 0.66; 95% CI 0.60-0.71) and HHC over home without HHC discharge with West North Central being the lowest (adjusted OR, 0.33; 95% CI 0.29-0.38). Similar patterns were observed in intracerebral hemorrhage.
Discussion: Significant insurance-dependent racial and ethnic disparities and regional variations were evident in post-acute service utilization after stroke. Targeted efforts are needed to improve post-acute service access for uninsured patients especially Hispanic patients and people in certain regions.
背景和目标:急性期后护理对患者的功能恢复和成功重返社区至关重要。本研究旨在了解目前中风后急性期服务利用的种族、民族和地区差异:这项回顾性横断面研究纳入了 2017-2018 年因缺血性中风和脑内出血住院的患者,使用的是全国住院患者样本。出院目的地分类如下:(1)设施,包括住院康复、专业护理设施和设施临终关怀;(2)家庭保健(HHC),包括家庭保健和家庭临终关怀;(3)无 HHC 的家庭。在对临床因素和调查设计进行调整后,我们使用多项式逻辑回归法研究了出院后去医疗机构而不是去家庭以及去 HHC 而不是去没有 HHC 的家庭的几率,并对种族、民族、保险和人口普查分区进行了分析:在 1,000,980 例加权缺血性卒中住院患者中,66.9% 为白人,17.6% 为黑人,9.5% 为西班牙裔,3.1% 为亚裔美国人/太平洋岛民,0.4% 为美洲原住民。相对于私人保险,未参保患者出院后入住医疗机构的调整后几率最低(0.44;95% CI 0.40-0.48),出院后入住 HHC 的调整后几率最低(0.79;95% CI 0.71-0.88)。与白人患者相比,只有拥有医疗保险/医疗补助保险或自费的西语裔患者出院时选择医疗机构而非居家的几率较低(调整后 OR 分别为 0.80 和 0.75;95% CI 分别为 0.76-0.84 和 0.63-0.93)。与白人患者相比,未参保的西班牙裔患者出院后入住 HHC 的几率也低于不入住 HHC 的患者(0.74;95% CI 0.57-0.97)。设施出院率最高的是东北部(39.2%),最低的是太平洋地区(31.2%)。HHC 出院率最高的是新英格兰地区(20.2%),最低的是中北部西部地区(10.3%),该地区没有 HHC 出院的家庭比例最高(46.1%)。与新英格兰地区相比,其他人口普查分区的设施出院率低于任何家庭出院率,其中太平洋地区最低(调整后 OR,0.66;95% CI 0.60-0.71),HHC 出院率低于无 HHC 的家庭出院率,中北部西部最低(调整后 OR,0.33;95% CI 0.29-0.38)。在脑出血中也观察到类似的模式:讨论:在中风后的急性期服务利用方面,与保险相关的种族和民族差异以及地区差异非常明显。需要有针对性地改善无保险患者,尤其是西班牙裔患者和某些地区的患者使用急性期后服务的情况。
{"title":"Racial, Ethnic, and Regional Disparities of Post-Acute Service Utilization After Stroke in the United States.","authors":"Shumei Man, David Bruckman, Ken Uchino, Jesse D Schold, Jarrod Dalton","doi":"10.1212/CPJ.0000000000200329","DOIUrl":"10.1212/CPJ.0000000000200329","url":null,"abstract":"<p><strong>Background and objectives: </strong>Post-acute care is critical for patient functional recovery and successful community transition. This study aimed to understand the current racial, ethnic, and regional disparities in post-acute service utilization after stroke.</p><p><strong>Methods: </strong>This retrospective cross-sectional study included patients hospitalized for ischemic stroke and intracerebral hemorrhage in 2017-2018 using the National Inpatient Sample. Discharge destinations were classified as follows: (1) facility including inpatient rehabilitation, skilled nursing facility, and facility hospice; (2) home health care (HHC), including home health and home hospice; and (3) home without HHC. Multinomial logistic regression was used to study the odds of discharge to a facility over home and HHC over home without HHC by race, ethnicity, insurance, and census division, adjusting for clinical factors and survey design.</p><p><strong>Results: </strong>Among the 1,000,980 weighted ischemic stroke admissions, 66.9% were White, 17.6% Black, 9.5% Hispanic, 3.1% Asian American/Pacific Islander, and 0.4% Native American. Relative to private insurance, uninsured patients had the lowest adjusted odds of facility over home discharge (0.44; 95% CI 0.40-0.48) and HHC discharge over home without HHC (0.79; 95% CI 0.71-0.88). Compared with White patients, only Hispanic patients with Medicare/Medicaid insurance or self-pay had lower odds of facility over home discharge (adjusted OR 0.80 and 0.75, respectively; 95% CI 0.76-0.84 and 0.63-0.93). Uninsured Hispanic patients also had lower odds of HHC discharge over home without HHC than White patients (0.74; 95% CI 0.57-0.97). Facility discharge rate was the highest in East North Central (39.2%) and lowest in Pacific (31.2%). HHC discharge rate was the highest in New England (20.2%) and lowest in West North Central (10.3%), which had the highest home without HHC discharge (46.1%). Compared with New England, other census divisions had lower odds of facility over any home discharge with Pacific being the lowest (adjusted OR, 0.66; 95% CI 0.60-0.71) and HHC over home without HHC discharge with West North Central being the lowest (adjusted OR, 0.33; 95% CI 0.29-0.38). Similar patterns were observed in intracerebral hemorrhage.</p><p><strong>Discussion: </strong>Significant insurance-dependent racial and ethnic disparities and regional variations were evident in post-acute service utilization after stroke. Targeted efforts are needed to improve post-acute service access for uninsured patients especially Hispanic patients and people in certain regions.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11259533/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141734661","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}
Pub Date : 2024-10-01Epub Date: 2024-08-15DOI: 10.1212/CPJ.0000000000200340
Gregory F Guzauskas, Sarah J Tabrizi, Jeffrey D Long, Astri Arnesen, Jamie L Hamilton, Daniel O Claassen, Lorraine R Munetsi, Shahid Malik, Idaira Rodríguez-Santana, Talaha M Ali, Frank Zhang
Background and objectives: Disease-modifying treatments (DMTs) such as gene therapy are currently under investigation as a potential treatment for Huntington disease (HD). Our objective was to estimate the long-term natural history of HD progression and explore the potential efficacy impacts and value of a hypothetical DMT using a decision-analytic modeling framework.
Methods: We developed a health state transition model that separately analyzed 40-year-old individuals with prefunctional decline (PFD, HD Integrated Staging System [HD-ISS] stage <3, total functional score [TFC] 13), active functional decline Shoulson and Fahn category 1 (SF1, HD-ISS stage 3, TFC 13-11), and SF2 (HD-ISS stage 3, TFC 10-7). Three-year outcomes from the TRACK-HD longitudinal study were linearly extrapolated to estimate the long-term health outcomes and costs of each population. For PFD individuals, we used the HD-ISS to predict the onset of functional decline. HD costs and quality-adjusted life years (QALYs) were estimated over a lifetime horizon by applying health state-specific costs and utilities derived from a related HD burden-of-illness study. We then estimated the long-term health impacts of hypothetical DMTs that slowed or delayed onset of functional decline. We conducted sensitivity analyses to assess model uncertainties.
Results: The expected life years for 40-year-old PFD, SF1, and SF2 populations were 20.46 (95% credible range [CR]: 19.05-22.30), 13.93 (10.82-19.08), and 10.99 (8.28-22.07), respectively. The expected QALYs for PFD, SF1, and SF2 populations were 15.93 (14.91-17.44), 8.29 (6.36-11.79), and 5.79 (4.14-12.91), respectively. The lifetime costs of HD were $508,200 ($310,300 to $803,700) for the PFD population, $1.15 million ($684,500 to $1.89 million) for SF1 individuals, and $1.07 million ($571,700 to $2.26 million) for SF2 individuals. Although hypothetical DMTs led to cost savings in the PFD population by delaying the cost burdens of functional decline, they increased costs in SF1 and SF2 populations by prolonging time spent in expensive progressive HD states.
Discussion: Our novel HD-modeling framework estimates HD progression over a lifetime and the associated costs and QALYs. Our approach can be used for future cost-effectiveness models as positive DMT clinical trial evidence becomes available.
背景和目的:目前正在研究基因治疗等疾病改变疗法(DMT)作为亨廷顿病(HD)的潜在治疗方法。我们的目标是估算 HD 进展的长期自然史,并使用决策分析建模框架探讨假设的 DMT 的潜在疗效影响和价值:方法:我们建立了一个健康状态转换模型,对 40 岁的功能衰退前期(PFD,HD 综合分期系统 [HD-ISS] 阶段)患者进行单独分析:40岁PFD、SF1和SF2人群的预期寿命年数分别为20.46(95%可信范围[CR]:19.05-22.30)、13.93(10.82-19.08)和10.99(8.28-22.07)。PFD、SF1和SF2人群的预期QALY分别为15.93(14.91-17.44)、8.29(6.36-11.79)和5.79(4.14-12.91)。PFD人群终生的HD成本为50.82万美元(31.03-80.37万美元),SF1人群为115万美元(68.45-189万美元),SF2人群为107万美元(57.17-226万美元)。尽管假定的 DMTs 通过延迟功能衰退的成本负担而为 PFD 群体节省了成本,但它们通过延长昂贵的渐进性 HD 状态的时间而增加了 SF1 和 SF2 群体的成本:我们的新型 HD 模型框架可估算出 HD 在患者一生中的进展情况以及相关成本和 QALY。在获得积极的 DMT 临床试验证据后,我们的方法可用于未来的成本效益模型。
{"title":"Long-Term Health Outcomes of Huntington Disease and the Impact of Future Disease-Modifying Treatments: A Decision-Modeling Analysis.","authors":"Gregory F Guzauskas, Sarah J Tabrizi, Jeffrey D Long, Astri Arnesen, Jamie L Hamilton, Daniel O Claassen, Lorraine R Munetsi, Shahid Malik, Idaira Rodríguez-Santana, Talaha M Ali, Frank Zhang","doi":"10.1212/CPJ.0000000000200340","DOIUrl":"10.1212/CPJ.0000000000200340","url":null,"abstract":"<p><strong>Background and objectives: </strong>Disease-modifying treatments (DMTs) such as gene therapy are currently under investigation as a potential treatment for Huntington disease (HD). Our objective was to estimate the long-term natural history of HD progression and explore the potential efficacy impacts and value of a hypothetical DMT using a decision-analytic modeling framework.</p><p><strong>Methods: </strong>We developed a health state transition model that separately analyzed 40-year-old individuals with prefunctional decline (PFD, HD Integrated Staging System [HD-ISS] stage <3, total functional score [TFC] 13), active functional decline Shoulson and Fahn category 1 (SF1, HD-ISS stage 3, TFC 13-11), and SF2 (HD-ISS stage 3, TFC 10-7). Three-year outcomes from the TRACK-HD longitudinal study were linearly extrapolated to estimate the long-term health outcomes and costs of each population. For PFD individuals, we used the HD-ISS to predict the onset of functional decline. HD costs and quality-adjusted life years (QALYs) were estimated over a lifetime horizon by applying health state-specific costs and utilities derived from a related HD burden-of-illness study. We then estimated the long-term health impacts of hypothetical DMTs that slowed or delayed onset of functional decline. We conducted sensitivity analyses to assess model uncertainties.</p><p><strong>Results: </strong>The expected life years for 40-year-old PFD, SF1, and SF2 populations were 20.46 (95% credible range [CR]: 19.05-22.30), 13.93 (10.82-19.08), and 10.99 (8.28-22.07), respectively. The expected QALYs for PFD, SF1, and SF2 populations were 15.93 (14.91-17.44), 8.29 (6.36-11.79), and 5.79 (4.14-12.91), respectively. The lifetime costs of HD were $508,200 ($310,300 to $803,700) for the PFD population, $1.15 million ($684,500 to $1.89 million) for SF1 individuals, and $1.07 million ($571,700 to $2.26 million) for SF2 individuals. Although hypothetical DMTs led to cost savings in the PFD population by delaying the cost burdens of functional decline, they increased costs in SF1 and SF2 populations by prolonging time spent in expensive progressive HD states.</p><p><strong>Discussion: </strong>Our novel HD-modeling framework estimates HD progression over a lifetime and the associated costs and QALYs. Our approach can be used for future cost-effectiveness models as positive DMT clinical trial evidence becomes available.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11332983/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142004873","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}