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Patient and Physician Perspectives of Treatment Burden in Multiple Sclerosis. 多发性硬化症患者和医生对治疗负担的看法。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-04 DOI: 10.1007/s40120-024-00654-1
Barry A Singer, Dawn Morgan, Julie A Stamm, Anita A Williams
{"title":"Patient and Physician Perspectives of Treatment Burden in Multiple Sclerosis.","authors":"Barry A Singer, Dawn Morgan, Julie A Stamm, Anita A Williams","doi":"10.1007/s40120-024-00654-1","DOIUrl":"10.1007/s40120-024-00654-1","url":null,"abstract":"<p><p>The number of disease-modifying therapies (DMTs) approved for the treatment of multiple sclerosis (MS) has greatly increased in recent decades, leading to higher treatment complexity. DMTs can differ in mode and frequency of administration, benefit-risk profile, and associated costs. Patients with MS contend not only with the burden of their chronic disease but also with the treatment burden of their MS therapy. Adhering to dosing schedules and infusion appointments can be difficult for busy, working-age patients or those with limited access to transportation. Patients and healthcare professionals (HCPs) may have differing priorities, concerns, and preferences when selecting treatment, potentially affecting treatment satisfaction and, importantly, adherence. Additionally, patients face direct and indirect costs related to treatment. These factors can all contribute to a high treatment burden on patients, impacting their quality of life and potentially leading to worse patient outcomes. HCPs, patients, and caregivers must work together to alleviate treatment burden through effective communication, shared decision-making, appreciating each other's perspectives, and additional HCP support. Consideration of treatment burden into clinical guidelines is also warranted. In this review, we examine key factors impacting treatment burden for patients with MS, with a focus on the patient perspective as provided by our patient authors, and provide strategies to minimize treatment burden.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1507-1525"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541994/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142126276","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Discrete-Choice Experiment Assessing the Patient Preferences and Real-World Experiences of Patients with Migraine in Japan. 一项离散选择实验,评估日本偏头痛患者的偏好和真实世界的经历。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-30 DOI: 10.1007/s40120-024-00663-0
Takao Takeshima, Akira Yuasa, Yukie Michelle Lloyd, Louis Patrick Watanabe, Kazumasa Kamei, Akiho Terasawa, Josh Coulter, Lucy Abraham, Brett Hauber, Masahiro Iijima
{"title":"A Discrete-Choice Experiment Assessing the Patient Preferences and Real-World Experiences of Patients with Migraine in Japan.","authors":"Takao Takeshima, Akira Yuasa, Yukie Michelle Lloyd, Louis Patrick Watanabe, Kazumasa Kamei, Akiho Terasawa, Josh Coulter, Lucy Abraham, Brett Hauber, Masahiro Iijima","doi":"10.1007/s40120-024-00663-0","DOIUrl":"10.1007/s40120-024-00663-0","url":null,"abstract":"<p><strong>Introduction: </strong>Migraine is a debilitating headache disorder with a high prevalence in Japan that imposes significant societal burden. Although the Japanese Clinical Practice Guideline for Headache Disorders 2021 recommends both acute and preventive migraine treatments, the usage of preventive treatments is still limited. Therefore, it is crucial to understand the treatment preferences of patients with migraine pertaining to both acute and preventive treatments.</p><p><strong>Methods: </strong>A mixed-methods study including a discrete choice experiment (DCE) was conducted with Japanese patients with migraine (10 for the qualitative interviews, and 400 for the DCE) who were recruited from the Rakuten Insight panel. The DCE presented hypothetical treatment options including oral acute, oral preventive, and injectable preventive medications. Six attributes (method of delivery, reduction of pain, impact of headaches on daily routines, dosage adjustability, and temporary and persistent side effects) each with three levels were included in the survey. A hierarchical Bayesian model was used to estimate relative attribute importance scores (RAI) for all attributes.</p><p><strong>Results: </strong>For the 400 participants in the DCE, the most common age bracket was 40-49 years old, and the majority were female (66.75%). RAI estimates indicated that \"method of delivery\" was the most important attribute for patients (RAI 51.92, SD = 10.20), followed by \"reduction of pain when experiencing a headache\" (RAI 17.00, SD = 7.74). Oral preventive treatments were preferred over injectable preventive treatments. The qualitative interviews showed that patients prefer oral medications to injectable treatments, and a lack of awareness regarding preventive treatments.</p><p><strong>Conclusion: </strong>This study found that the \"method of delivery\" was the most important driver of treatment preferences of patients with migraine in Japan, with oral acute medications being preferred. Oral preventive treatments were found to be preferred over injectable treatments. These results may indicate the need for increased education regarding preventive treatments, as well as the need for further development of these treatments.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1661-1683"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541993/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142350926","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Real-World Persistence with Ocrelizumab in Multiple Sclerosis: a Systematic Review. 多发性硬化症患者使用奥克立珠单抗的真实世界持久性:系统综述。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-10-19 DOI: 10.1007/s40120-024-00667-w
John L Petrie, Charlie A Smith, Donna Fountain, Gerardo Machnicki
{"title":"Real-World Persistence with Ocrelizumab in Multiple Sclerosis: a Systematic Review.","authors":"John L Petrie, Charlie A Smith, Donna Fountain, Gerardo Machnicki","doi":"10.1007/s40120-024-00667-w","DOIUrl":"10.1007/s40120-024-00667-w","url":null,"abstract":"<p><p>In clinical trials, the percentage of patients discontinuing treatment with ocrelizumab due to adverse events was low. However, real-world populations are often more diverse than randomized controlled trials (RCTs), therefore it is important to assess discontinuation rates in real-world studies. This systematic literature review (SLR) was conducted to identify real-world discontinuation and persistence data for ocrelizumab in studies of patients with relapsing remitting multiple sclerosis (RRMS) and primary progressive multiple sclerosis (PPMS). Searches were conducted in MEDLINE and Embase to identify relevant real-world studies that met pre-determined Population, Intervention, Comparison, Outcomes, and Study (PICOS) criteria. Only articles published in English were included, but the study country was not restricted. A total of 30 studies were included, with the majority reporting real-world persistence data that appear to be similar to or better than in the pivotal clinical trials, with only 1 study reporting higher discontinuation rates due to adverse events compared with the clinical trials. Other studies identified reported that the risk of discontinuation was higher for other disease-modifying therapies (DMTs) compared with ocrelizumab, and adherence was also higher for ocrelizumab versus other DMTs. These findings have clinical relevance, as other studies have reported improved clinical outcomes and lower care costs for patients that are persistent or adherent to other DMTs.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1597-1605"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541982/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142470987","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Association Between Constipation and Positron Emission Tomography and Blood-Based Biomarkers in Older Cognitively Unimpaired Adults with Higher Amyloid-β Burden. 淀粉样蛋白-β负荷较高的认知功能未受损的老年人便秘与正电子发射断层扫描和血液生物标志物之间的关系
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-10-22 DOI: 10.1007/s40120-024-00666-x
Heling Chu, Chuyi Huang, Fang Xie, Qihao Guo
{"title":"The Association Between Constipation and Positron Emission Tomography and Blood-Based Biomarkers in Older Cognitively Unimpaired Adults with Higher Amyloid-β Burden.","authors":"Heling Chu, Chuyi Huang, Fang Xie, Qihao Guo","doi":"10.1007/s40120-024-00666-x","DOIUrl":"10.1007/s40120-024-00666-x","url":null,"abstract":"<p><strong>Introduction: </strong>Constipation may be linked to cognitive decline and a higher risk of Alzheimer's disease (AD). We aimed to investigate the association between constipation and positron emission tomography (PET) and blood-based AD biomarkers in older cognitively unimpaired (CU) adults with higher Aβ burden.</p><p><strong>Methods: </strong>Constipation was diagnosed according to Rome IV criteria and the severity of constipation was evaluated by using a validated self-reported questionnaire. The participants underwent the examination of plasma AD biomarkers and <sup>18</sup>F-florbetapir PET and <sup>18</sup>F-MK6240 PET scans; the latter was only performed in the validation cohort. Correlation and multiple linear regression analyses were used to investigate the association between constipation and AD biomarkers.</p><p><strong>Results: </strong>Two cohorts were included in our study. A total of 404 older participants with 126 of whom Aβ-PET positive were enrolled in the development cohort. Multiple linear regression analysis showed constipation was associated with plasma t-Tau, p-Tau-181, and neurofilament light chain (NfL) in participants with Aβ-PET (+). Meanwhile, no/mild constipation was associated with lower Aβ-PET standard uptake value ratio. The association between constipation and plasma biomarkers was different in the subgroups stratified by age, sex and APOE ε4 genotype. The above associations were further validated in the validation cohort containing 36 Aβ-PET (+) participants. Importantly, no/mild constipation was associated with less Tau burden evaluated by <sup>18</sup>F-MK6240 PET Braak stages.</p><p><strong>Conclusion: </strong>Our data indicate that no/mild constipation may be associated with lower plasma t-Tau, p-Tau-181, and NfL as well as less Aβ and Tau burden in older CU adults with Aβ deposition. Improving constipation and being away from defecation disorders may help reduce the risk of AD development.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1701-1715"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541973/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142470988","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
COVID-19 Vaccination Response in Patients with Multiple Sclerosis Treated with Ofatumumab in the United States: A Medical Record Review. 美国接受奥法妥木单抗治疗的多发性硬化症患者对 COVID-19 疫苗接种的反应:病历回顾。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-10-23 DOI: 10.1007/s40120-024-00671-0
Rahul H Dave, Heidi Crayton, Augusto Miravalle, Ming-Hui Tai, Kerri Wyse, Katherine Houghton, Abby Hitchens, Regina Berkovich
{"title":"COVID-19 Vaccination Response in Patients with Multiple Sclerosis Treated with Ofatumumab in the United States: A Medical Record Review.","authors":"Rahul H Dave, Heidi Crayton, Augusto Miravalle, Ming-Hui Tai, Kerri Wyse, Katherine Houghton, Abby Hitchens, Regina Berkovich","doi":"10.1007/s40120-024-00671-0","DOIUrl":"10.1007/s40120-024-00671-0","url":null,"abstract":"<p><strong>Introduction: </strong>Real-world data are required to provide a greater understanding of the impact of ofatumumab on the ability to mount an effective immune response following the receipt of approved COVID-19 vaccinations. This retrospective real-world analysis aimed to describe the humoral immune response to COVID-19 vaccination during ofatumumab treatment in patients with multiple sclerosis (MS).</p><p><strong>Methods: </strong>Data from patients with MS treated with ofatumumab who were fully vaccinated against COVID-19 infection were abstracted from medical charts at four clinical sites in the USA. Patient characteristics and humoral response were summarized descriptively. Differences in humoral response were documented on the basis of vaccination status during ofatumumab treatment (i.e., after full vaccination and after booster vaccination) and prior disease-modifying treatment (DMT) exposure (i.e., DMT naïve, prior anti-CD20/sphingosine 1-phosphate [S1P] therapy, prior non-anti-CD20/S1P therapy). The sample size precluded formal statistical analysis.</p><p><strong>Results: </strong>Thirty-eight patients were included. The mean (standard deviation) duration of ofatumumab treatment upon data collection was 20.4 (4.6) months (treatment ongoing for 35 [92%] patients). Definitive humoral response after full vaccination was documented for 34 patients, of whom 20 (60%) were seropositive. Definitive humoral response after booster vaccination was documented among five patients, of whom three (60%) were seropositive. Among patients who were DMT naïve prior to ofatumumab (n = 15), 73% were seropositive; among patients exposed to prior anti-CD20/S1P therapy (n = 14), 33% were seropositive; and among patients exposed to prior non-anti-CD20/S1P therapy (n = 9), 56% were seropositive. Patients naïve to DMT had been living with an MS diagnosis for a shorter duration than those experienced with DMTs.</p><p><strong>Conclusion: </strong>Patients with MS receiving ongoing treatment with ofatumumab can mount a positive humoral response to a COVID-19 vaccination. Prior treatment with anti-CD20 or S1P DMTs may be a risk factor for lower humoral response.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1737-1745"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541983/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142504886","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Symptoms of Hereditary Transthyretin Amyloidosis: The Patient and Physician Perspective. 遗传性转甲状腺素淀粉样变性的症状:患者和医生的视角。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-06 DOI: 10.1007/s40120-024-00657-y
Michael Lane, Michael Polydefkis
{"title":"Symptoms of Hereditary Transthyretin Amyloidosis: The Patient and Physician Perspective.","authors":"Michael Lane, Michael Polydefkis","doi":"10.1007/s40120-024-00657-y","DOIUrl":"10.1007/s40120-024-00657-y","url":null,"abstract":"<p><p>This article has been co-authored by a patient living with hereditary transthyretin (ATTRv) amyloidosis and a neurologist. This rare, progressive disease is associated with impairment of multiple organ systems, including the nerves, heart, and the gastrointestinal tract, forcing patients to live with and adapt to a range of debilitating symptoms. Here, the patient and physician discuss how the symptoms of ATTRv amyloidosis profoundly impact day to day life, the difficulties with identifying the disease, and how this effects the diagnosis experience. In recent years, significant advancements have been made in the treatment and management of ATTRv amyloidosis. However, the authors highlight the urgency of increasing awareness of the disease among the wider medical community, as well as in patients who notice the symptoms, to ensure that earlier diagnosis and appropriate treatment are achieved.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1527-1533"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541984/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142140655","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Passive Anti-amyloid Beta Monoclonal Antibodies: Lessons Learned over Past 20 Years. 被动抗淀粉样蛋白 Beta 单克隆抗体:过去 20 年的经验教训。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-10-08 DOI: 10.1007/s40120-024-00664-z
Alexandra Wicker, Jahnavi Shriram, Boris Decourt, Marwan Noel Sabbagh
{"title":"Passive Anti-amyloid Beta Monoclonal Antibodies: Lessons Learned over Past 20 Years.","authors":"Alexandra Wicker, Jahnavi Shriram, Boris Decourt, Marwan Noel Sabbagh","doi":"10.1007/s40120-024-00664-z","DOIUrl":"10.1007/s40120-024-00664-z","url":null,"abstract":"<p><p>Alzheimer's disease (AD) is a neurodegenerative disorder that significantly impairs cognitive and functional abilities, placing a substantial burden on both patients and caregivers. Current symptomatic treatments fail to halt the progression of AD, highlighting the urgent need for more effective disease-modifying therapies (DMTs). DMTs under development are classified as either passive or active on the basis of their mechanisms of eliciting an immune response. While this review will touch on active immunotherapies, we primarily focus on anti-amyloid beta monoclonal antibodies (mAbs), a form of passive immunotherapy, discussing their multifaceted role in AD treatment and the critical factors influencing their therapeutic efficacy. With two mAbs now approved and prescribed in the clinical setting, it is crucial to reflect on the lessons learned from trials of earlier mAbs that have shaped their development and contributed to their current success. These insights can then guide the creation of even more effective mAbs, ultimately enhancing therapeutic outcomes for patients with AD while minimizing adverse events.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1571-1595"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541067/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142392096","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Effects of Manual Acupuncture Versus Sham Acupuncture in patients with Post-Stroke Depression: A Randomized Clinical Trial. 手针与假针对脑卒中后抑郁症患者的影响:随机临床试验。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-10-25 DOI: 10.1007/s40120-024-00672-z
Conghui Wei, Jinling Chen, Qu Yang, Jingjing Xu, Qingsong Li, Fulin Li, Yu Liu, Jun Luo
{"title":"Effects of Manual Acupuncture Versus Sham Acupuncture in patients with Post-Stroke Depression: A Randomized Clinical Trial.","authors":"Conghui Wei, Jinling Chen, Qu Yang, Jingjing Xu, Qingsong Li, Fulin Li, Yu Liu, Jun Luo","doi":"10.1007/s40120-024-00672-z","DOIUrl":"10.1007/s40120-024-00672-z","url":null,"abstract":"<p><strong>Background: </strong>Post-stroke depression (PSD) is a prevalent psychiatric complication in stroke patients, severely reducing quality of life and delaying social recovery in stroke survivors. Clinical studies have shown that acupuncture can be used as an alternative approach for PSD. The aim of this study was to examine the safety, efficacy, and electroencephalogram (EEG) mechanism of acupuncture in treating PSD patients.</p><p><strong>Methods: </strong>From October 28, 2022 to May 16, 2023, this single-center, single-blind, randomized clinical trial was conducted at the Second Affiliated Hospital of Nanchang University. A total of 56 eligible subjects were assigned in a random manner, with an equal distribution between two groups: the manual acupuncture (MA) group and the sham acupuncture (SA) group. The primary outcome was the Hamilton Depression Scale-24 (HAMD-24); the secondary outcomes included the Pittsburgh Sleep Quality Index (PSQI), the National Institutes of Health Stroke Scale (NIHSS), the Barthel index, EEG power spectrum, and EEG imaginary coherent (iCOH).</p><p><strong>Results: </strong>Compared to the SA group, the MA group exhibited significant improvements in HAMD-24, NIHSS, PSIQ, and Barthel index at week 6. The total improvement rate was 85.71% in the MA group and 28.57% in the SA group. After 6 weeks of treatment, the alpha and beta bands power spectrum increased significantly, while the delta and theta bands power spectrum decreased significantly in the MA group compared to the SA group. The iCOH analysis showed that the MA group had significantly higher functional connectivity in the four bands than the SA group.</p><p><strong>Conclusions: </strong>Acupuncture might be regarded as an adjunctive treatment for PSD patients with improvements in their neurological deficits, sleep quality, and depression. Meanwhile, the mechanism of acupuncture in treating PSD patients may be through decreasing the slow wave power spectrum and increasing the fast wave power spectrum, and enhancing brain functional connectivity.</p><p><strong>Trial registration: </strong>Chinese Clinical Trial Registry (ChiCTR2200065112/2022-10-28).</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1717-1735"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541975/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142504891","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Estimating the Economically Justifiable Price of Limited-Duration Treatment with Donanemab for Early Symptomatic Alzheimer's Disease in the United States. 估算美国使用多奈单抗对早期症状性阿尔茨海默病进行限期治疗的经济合理价格。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-18 DOI: 10.1007/s40120-024-00649-y
Malaz Boustani, Erin G Doty, Louis P Garrison, Lee J Smolen, Timothy M Klein, Daniel R Murphy, Andrew W Spargo, Mark Belger, Joseph A Johnston
{"title":"Estimating the Economically Justifiable Price of Limited-Duration Treatment with Donanemab for Early Symptomatic Alzheimer's Disease in the United States.","authors":"Malaz Boustani, Erin G Doty, Louis P Garrison, Lee J Smolen, Timothy M Klein, Daniel R Murphy, Andrew W Spargo, Mark Belger, Joseph A Johnston","doi":"10.1007/s40120-024-00649-y","DOIUrl":"10.1007/s40120-024-00649-y","url":null,"abstract":"<p><strong>Introduction: </strong>The goal of this economic model is to estimate an economically justifiable price (EJP) for using donanemab for the treatment of early symptomatic Alzheimer's disease (AD) in the United States based on clinical data from the phase 3 TRAILBLAZER-ALZ 2 trial (NCT04437511).</p><p><strong>Methods: </strong>We adapted an AD Markov state-transition model developed by the Institute for Clinical and Economic Review to estimate the EJP for donanemab at different willingness-to-pay (WTP) thresholds from the health care system perspective and the societal perspective as co-base cases.</p><p><strong>Results: </strong>Assuming a WTP threshold of $150,000 per quality-adjusted life-year (QALY) gained, the model estimates a 1-year (13-dose) EJP for donanemab of $80,538 from the health care system perspective and $91,126 from the societal perspective; at a WTP threshold of $100,000 per QALY gained, the model estimates a 1-year (13-dose) EJP for donanemab of $44,691 from the health care system perspective and $55,419 from the societal perspective. Mean total treatment costs per patient at the $150,000 per QALY gained EJP derived from the health care system perspective were estimated at $77,812 based on the average number of doses of donanemab patients received in the co-base case analysis. One-way sensitivity analysis (OWSA) indicated that treatment efficacy, disease severity at the time of treatment initiation, and duration of treatment effect were the main drivers of the potential EJP.</p><p><strong>Conclusions: </strong>Results from this modeling simulation informed by the TRAILBLAZER-ALZ 2 study support an EJP for limited-duration treatment with donanemab that exceeds per-dose list prices for currently available amyloid-targeting therapies, implying potentially lower lifetime costs and better value for money.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1641-1659"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541978/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142292222","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Correction: Safety of Deutetrabenazine for the Treatment of Tardive Dyskinesia and Chorea Associated with Huntington Disease. 更正:治疗与亨廷顿病相关的迟发性运动障碍和舞蹈症的杜特拉嗪的安全性。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 DOI: 10.1007/s40120-024-00660-3
Samuel Frank, Karen E Anderson, Hubert H Fernandez, Robert A Hauser, Daniel O Claassen, David Stamler, Stewart A Factor, Joohi Jimenez-Shahed, Hadas Barkay, Amanda Wilhelm, Jessica K Alexander, Nayla Chaijale, Steve Barash, Juha-Matti Savola, Mark Forrest Gordon, Maria Chen
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