Pub Date : 2022-04-01Epub Date: 2021-12-21DOI: 10.2217/nmt-2021-0031
Tjalf Ziemssen, Anna Kurzeja, Bogdan Muresan, Jennifer S Haas, Jessica Alexander, Maurice T Driessen
Aim: To evaluate adherence, healthcare resource utilization (HRU) and costs for glatiramer acetate (GA; injectable), dimethyl fumarate (oral) and teriflunomide (oral) in relapsing multiple sclerosis. Patients & methods: Retrospective analyses of a claims database. Results: Teriflunomide patients were older with more co-morbidities and fewer relapses versus GA and dimethyl fumarate. GA patients were mostly disease-modifying therapies (DMTs)-treatment naive. Treatment adherence was 61-70%. All DMTs reduced HRU versus pre-index. Costs were comparable across cohorts. High adherence reduced hospitalizations and several costs versus low adherers. Conclusion: Adherence rates were high and comparable with all DMTs. Similar (and high) reductions in HRU and costs occurred with all DMTs. High adherence improved economic outcomes versus low adherence. Thus, investing in adherence improvement is beneficial to improve outcomes in relapsing multiple sclerosis.
{"title":"Real-world patient characteristics, treatment patterns and costs in relapsing multiple sclerosis patients treated with glatiramer acetate, dimethyl fumarate or teriflunomide in Germany.","authors":"Tjalf Ziemssen, Anna Kurzeja, Bogdan Muresan, Jennifer S Haas, Jessica Alexander, Maurice T Driessen","doi":"10.2217/nmt-2021-0031","DOIUrl":"https://doi.org/10.2217/nmt-2021-0031","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate adherence, healthcare resource utilization (HRU) and costs for glatiramer acetate (GA; injectable), dimethyl fumarate (oral) and teriflunomide (oral) in relapsing multiple sclerosis. <b>Patients & methods:</b> Retrospective analyses of a claims database. <b>Results:</b> Teriflunomide patients were older with more co-morbidities and fewer relapses versus GA and dimethyl fumarate. GA patients were mostly disease-modifying therapies (DMTs)-treatment naive. Treatment adherence was 61-70%. All DMTs reduced HRU versus pre-index. Costs were comparable across cohorts. High adherence reduced hospitalizations and several costs versus low adherers. <b>Conclusion:</b> Adherence rates were high and comparable with all DMTs. Similar (and high) reductions in HRU and costs occurred with all DMTs. High adherence improved economic outcomes versus low adherence. Thus, investing in adherence improvement is beneficial to improve outcomes in relapsing multiple sclerosis.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":"12 2","pages":"93-107"},"PeriodicalIF":2.6,"publicationDate":"2022-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39743583","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-04-01Epub Date: 2022-02-18DOI: 10.2217/nmt-2021-0049
Andrew J Larner
Aim: To examine three different accuracy metrics for evaluation of cognitive screening instruments: overall correct classification accuracy (Acc), the sum of true positives and negatives divided by the total number tested; balanced accuracy (balanced Acc), half of the sum of sensitivity and specificity; and unbiased accuracy (unbiased Acc), removing biasing effects of random associations between test results and disease prevalence. Materials & methods: Data from a prospective test accuracy study of Mini-Addenbrooke's Cognitive Examination were used to calculate and plot the Acc measures. Results: Each Acc metric resulted in a similar pattern of results across the range of Mini-Addenbrooke's Cognitive Examination cut-offs for diagnosis of both dementia and mild cognitive impairment. Acc and balanced Acc gave more optimistic outcomes (closer to possible maximum value of 1) than unbiased Acc. Conclusion: Unbiased Acc may have advantages over Acc and balanced Acc by removing biasing effects of random associations between test result and disease prevalence.
{"title":"Accuracy of cognitive screening instruments reconsidered: overall, balanced or unbiased accuracy?","authors":"Andrew J Larner","doi":"10.2217/nmt-2021-0049","DOIUrl":"https://doi.org/10.2217/nmt-2021-0049","url":null,"abstract":"<p><p><b>Aim:</b> To examine three different accuracy metrics for evaluation of cognitive screening instruments: overall correct classification accuracy (Acc), the sum of true positives and negatives divided by the total number tested; balanced accuracy (balanced Acc), half of the sum of sensitivity and specificity; and unbiased accuracy (unbiased Acc), removing biasing effects of random associations between test results and disease prevalence. <b>Materials & methods:</b> Data from a prospective test accuracy study of Mini-Addenbrooke's Cognitive Examination were used to calculate and plot the Acc measures. <b>Results:</b> Each Acc metric resulted in a similar pattern of results across the range of Mini-Addenbrooke's Cognitive Examination cut-offs for diagnosis of both dementia and mild cognitive impairment. Acc and balanced Acc gave more optimistic outcomes (closer to possible maximum value of 1) than unbiased Acc. <b>Conclusion:</b> Unbiased Acc may have advantages over Acc and balanced Acc by removing biasing effects of random associations between test result and disease prevalence.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":"12 2","pages":"67-76"},"PeriodicalIF":2.6,"publicationDate":"2022-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39933525","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-04-01Epub Date: 2022-02-18DOI: 10.2217/nmt-2021-0033
Melanie M Tidman, Dawn White, Timothy White
Aim: To evaluate a low carbohydrate/healthy fat/ketogenic diet (LCHF/KD) on symptoms, depression, anxiety and biomarkers in adults with Parkinson's disease (PD). Patients & methods: 16 adults ages 36-80 with PD participated in the intervention for 12 weeks. The study provided pre-post-study comparisons of biomarkers, weight, waist measurement, united Parkinson's Disease Rating Scale (UPDRS), Parkinson's Anxiety Scale (PAS) and Center for Epidemiologic Studies Depression Scale Revised-20 (CESD-R-20) Depression Scale. Results: Although LCHF/KD improves blood glucose in diabetes and seizure control in epilepsy, research gaps exist in this dietary intervention in PD. Statistically, significant improvements occurred in several measurements, PAS scores and Part I of the UPDRS. Conclusion: The LCHF/KD shows positive trends with improvements in biomarkers and anxiety symptoms. Further research is needed to evaluate dietary interventions for PD.
{"title":"Effects of an low carbohydrate/healthy fat/ketogenic diet on biomarkers of health and symptoms, anxiety and depression in Parkinson's disease: a pilot study.","authors":"Melanie M Tidman, Dawn White, Timothy White","doi":"10.2217/nmt-2021-0033","DOIUrl":"https://doi.org/10.2217/nmt-2021-0033","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate a low carbohydrate/healthy fat/ketogenic diet (LCHF/KD) on symptoms, depression, anxiety and biomarkers in adults with Parkinson's disease (PD). <b>Patients & methods:</b> 16 adults ages 36-80 with PD participated in the intervention for 12 weeks. The study provided pre-post-study comparisons of biomarkers, weight, waist measurement, united Parkinson's Disease Rating Scale (UPDRS), Parkinson's Anxiety Scale (PAS) and Center for Epidemiologic Studies Depression Scale Revised-20 (CESD-R-20) Depression Scale. <b>Results:</b> Although LCHF/KD improves blood glucose in diabetes and seizure control in epilepsy, research gaps exist in this dietary intervention in PD. Statistically, significant improvements occurred in several measurements, PAS scores and Part I of the UPDRS. <b>Conclusion:</b> The LCHF/KD shows positive trends with improvements in biomarkers and anxiety symptoms. Further research is needed to evaluate dietary interventions for PD.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":"12 2","pages":"57-66"},"PeriodicalIF":2.6,"publicationDate":"2022-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39810313","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-02-04DOI: 10.1101/2022.02.02.22270139
A. Larner
An efficiency index (EI) for the evaluation of binary classifiers was recently characterised, where EI is the ratio of classifier accuracy to inaccuracy. The purpose of this study was to further develop EI by substituting balanced accuracy and unbiased accuracy in place of accuracy, and their respective complements in place of inaccuracy, to construct balanced EI and unbiased EI measures. Additional investigations, using the dataset of a prospective pragmatic test accuracy study of a cognitive screening instrument, explored use of the log method to calculate confidence intervals for the various EI formulations; the dependence of EI formulations on prevalence; and comparison of EI formulations with analogous formulations based on the Identification Index (II), a previously described metric which is also based on accuracy and inaccuracy, where II is accuracy minus inaccuracy. EI formulations are shown to have advantages over II formulations, in particular their boundary values (0 and infinity) mean that negative values never occur, unlike the case for II, and the inflection point of 1 demarcates likelihood of correct versus incorrect classification.
{"title":"Evaluating binary classifiers: extending the Efficiency Index","authors":"A. Larner","doi":"10.1101/2022.02.02.22270139","DOIUrl":"https://doi.org/10.1101/2022.02.02.22270139","url":null,"abstract":"An efficiency index (EI) for the evaluation of binary classifiers was recently characterised, where EI is the ratio of classifier accuracy to inaccuracy. The purpose of this study was to further develop EI by substituting balanced accuracy and unbiased accuracy in place of accuracy, and their respective complements in place of inaccuracy, to construct balanced EI and unbiased EI measures. Additional investigations, using the dataset of a prospective pragmatic test accuracy study of a cognitive screening instrument, explored use of the log method to calculate confidence intervals for the various EI formulations; the dependence of EI formulations on prevalence; and comparison of EI formulations with analogous formulations based on the Identification Index (II), a previously described metric which is also based on accuracy and inaccuracy, where II is accuracy minus inaccuracy. EI formulations are shown to have advantages over II formulations, in particular their boundary values (0 and infinity) mean that negative values never occur, unlike the case for II, and the inflection point of 1 demarcates likelihood of correct versus incorrect classification.","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2022-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47945633","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-02-01Epub Date: 2022-01-10DOI: 10.2217/nmt-2021-0015
Hendrik Stephan Goedee, Yusuf A Rajabally
Chronic inflammatory demyelinating polyneuropathy, its variants and multifocal motor neuropathy belong to a spectrum of peripheral nerve disorders with complex dysimmune disease mechanisms. Awareness of the unique clinical phenotypes but also heterogeneity between patients is vital to arrive at early suspicion and ordering appropriate tests. This includes requirements for optimal electrodiagnostic protocol, aimed to capture sufficient electrophysiologic evidence for relevant abnormalities, a case-based approach on the eventual need to further expand the diagnostic armamentarium and correct reading of their results. Considerable phenotypical variation, diverse combinations of abnormalities found on diagnostic tests and heterogeneity in disease course and treatment response, all contribute to widespread differences in success rates on timely diagnosis and optimal treatment. We aim to provide a practical overview and guidance on relevant diagnostic and management strategies, including pitfalls and present a summary of the relevant novel developments in this field.
{"title":"Evidence base for investigative and therapeutic modalities in chronic inflammatory demyelinating polyneuropathy and multifocal motor neuropathy.","authors":"Hendrik Stephan Goedee, Yusuf A Rajabally","doi":"10.2217/nmt-2021-0015","DOIUrl":"https://doi.org/10.2217/nmt-2021-0015","url":null,"abstract":"<p><p>Chronic inflammatory demyelinating polyneuropathy, its variants and multifocal motor neuropathy belong to a spectrum of peripheral nerve disorders with complex dysimmune disease mechanisms. Awareness of the unique clinical phenotypes but also heterogeneity between patients is vital to arrive at early suspicion and ordering appropriate tests. This includes requirements for optimal electrodiagnostic protocol, aimed to capture sufficient electrophysiologic evidence for relevant abnormalities, a case-based approach on the eventual need to further expand the diagnostic armamentarium and correct reading of their results. Considerable phenotypical variation, diverse combinations of abnormalities found on diagnostic tests and heterogeneity in disease course and treatment response, all contribute to widespread differences in success rates on timely diagnosis and optimal treatment. We aim to provide a practical overview and guidance on relevant diagnostic and management strategies, including pitfalls and present a summary of the relevant novel developments in this field.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":"12 1","pages":"35-47"},"PeriodicalIF":2.6,"publicationDate":"2022-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39665374","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-02-01Epub Date: 2022-02-03DOI: 10.2217/nmt-2021-0045
Simona Bonavita, Luigi Lavorgna, Hilary Worton, Dominic Jack
What is this summary about?: This is a plain language summary of an article originally published in the journal Frontiers in Neurology. People with multiple sclerosis (often shortened to MS) may have concerns about pregnancy and fertility. To understand more about these concerns, 332 people with MS in the USA, UK, France, Germany, Italy, and Spain took a survey with questions about how they made family planning decisions.
What were the results?: Most of the survey participants (around 82%) were women. The survey found that people with MS were less likely to have children than people without MS. Over half (56%) of people with MS said the disease impacted their family planning decisions in some way, almost one quarter (22%) significantly changed their plans for the timing of their pregnancy or number of children, and 14% decided against having children. For almost 4 out of 5 (81%) people with MS the main source of family planning information was healthcare professionals.
What do the results of the study mean?: Overall, MS significantly impacted patients' decisions about family planning.
{"title":"Family planning in people with multiple sclerosis: a plain language summary.","authors":"Simona Bonavita, Luigi Lavorgna, Hilary Worton, Dominic Jack","doi":"10.2217/nmt-2021-0045","DOIUrl":"10.2217/nmt-2021-0045","url":null,"abstract":"<p><strong>What is this summary about?: </strong>This is a plain language summary of an article originally published in the journal Frontiers in Neurology. People with multiple sclerosis (often shortened to MS) may have concerns about pregnancy and fertility. To understand more about these concerns, 332 people with MS in the USA, UK, France, Germany, Italy, and Spain took a survey with questions about how they made family planning decisions.</p><p><strong>What were the results?: </strong>Most of the survey participants (around 82%) were women. The survey found that people with MS were less likely to have children than people without MS. Over half (56%) of people with MS said the disease impacted their family planning decisions in some way, almost one quarter (22%) significantly changed their plans for the timing of their pregnancy or number of children, and 14% decided against having children. For almost 4 out of 5 (81%) people with MS the main source of family planning information was healthcare professionals.</p><p><strong>What do the results of the study mean?: </strong>Overall, MS significantly impacted patients' decisions about family planning.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":"12 1","pages":"9-14"},"PeriodicalIF":2.3,"publicationDate":"2022-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39883518","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-02-01Epub Date: 2021-12-17DOI: 10.2217/nmt-2021-0028
Thomas Müller
Amantadine is an old, antiviral compound, which moderately improves motor behavior in Parkinson's disease. Its current resurgence results from an innovative, delayed uptake and extended release amantadine hydrochloride capsule, given at bedtime once daily. It is the only approved compound for reduction of involuntary movements, so called dyskinesia, in fluctuating orally levodopa treated patients. It additionally ameliorates 'OFF' intervals characterized by impaired motor behavior. These beneficial effects result from higher and more continuous brain delivery of amantadine. Future clinical research is warranted on preventive effects of this amantadine capsule combined with enzyme blockers of central monoamine oxidase B and peripheral catechol-O-methyltransferase on motor complications in orally levodopa treated patients, as all these pharmacological principles support the concept of continuous dopamine substitution.
金刚烷胺是一种古老的抗病毒化合物,可以适度改善帕金森病患者的运动行为。它目前的复苏源于一种创新的、延迟吸收和缓释的盐酸金刚烷胺胶囊,每天一次在睡前服用。它是唯一被批准的化合物,用于减少波动口服左旋多巴治疗患者的不自主运动,即所谓的运动障碍。它还能改善以运动行为受损为特征的“OFF”间隔。这些有益的效果是由于金刚烷胺的大脑输送量更高、更持续。金刚烷胺胶囊联合中枢单胺氧化酶B和外周儿茶酚- o -甲基转移酶阻断剂对口服左旋多巴患者运动并发症的预防作用有待进一步的临床研究,因为所有这些药理学原理都支持持续多巴胺替代的概念。
{"title":"GOCOVRI<sup>®</sup> (amantadine) extended-release capsules in Parkinson's disease.","authors":"Thomas Müller","doi":"10.2217/nmt-2021-0028","DOIUrl":"https://doi.org/10.2217/nmt-2021-0028","url":null,"abstract":"<p><p>Amantadine is an old, antiviral compound, which moderately improves motor behavior in Parkinson's disease. Its current resurgence results from an innovative, delayed uptake and extended release amantadine hydrochloride capsule, given at bedtime once daily. It is the only approved compound for reduction of involuntary movements, so called dyskinesia, in fluctuating orally levodopa treated patients. It additionally ameliorates 'OFF' intervals characterized by impaired motor behavior. These beneficial effects result from higher and more continuous brain delivery of amantadine. Future clinical research is warranted on preventive effects of this amantadine capsule combined with enzyme blockers of central monoamine oxidase B and peripheral catechol-O-methyltransferase on motor complications in orally levodopa treated patients, as all these pharmacological principles support the concept of continuous dopamine substitution.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":"12 1","pages":"15-28"},"PeriodicalIF":2.6,"publicationDate":"2022-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39734057","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Background: Accurate diagnosis and management of patients with rapidly progressive dementia may be challenging during the COVID-19 pandemic, which has negatively influenced the diagnostic performances, medical resource allocation and routine care for all non-COVID-19 diseases. Case presentation: We herein present a case of a 57-year-old male with rapidly progressive cognitive decline, headache, diplopia, myalgia, unsteady gait, aggression, depression, insomnia, hallucinations and delusions of persecution. COVID-19-associated encephalitis was briefly considered as a differential diagnosis. However, this hypothesis was rejected upon further investigation. A final diagnosis of sporadic Creutzfeldt-Jakob disease was made. Conclusion: A timely and accurate diagnosis of Creutzfeldt-Jakob disease gives patients and their families the chance to receive a good standard of healthcare and avoid extensive evaluations for other conditions.
{"title":"COVID-19-associated encephalitis or Creutzfeldt-Jakob disease: a case report.","authors":"Gooya Tayyebi, Seyed Kazem Malakouti, Behnam Shariati, Leila Kamalzadeh","doi":"10.2217/nmt-2021-0025","DOIUrl":"https://doi.org/10.2217/nmt-2021-0025","url":null,"abstract":"<p><p><b>Background:</b> Accurate diagnosis and management of patients with rapidly progressive dementia may be challenging during the COVID-19 pandemic, which has negatively influenced the diagnostic performances, medical resource allocation and routine care for all non-COVID-19 diseases. <b>Case presentation:</b> We herein present a case of a 57-year-old male with rapidly progressive cognitive decline, headache, diplopia, myalgia, unsteady gait, aggression, depression, insomnia, hallucinations and delusions of persecution. COVID-19-associated encephalitis was briefly considered as a differential diagnosis. However, this hypothesis was rejected upon further investigation. A final diagnosis of sporadic Creutzfeldt-Jakob disease was made. <b>Conclusion:</b> A timely and accurate diagnosis of Creutzfeldt-Jakob disease gives patients and their families the chance to receive a good standard of healthcare and avoid extensive evaluations for other conditions.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":"12 1","pages":"29-34"},"PeriodicalIF":2.6,"publicationDate":"2022-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8765092/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39951919","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}