{"title":"Why is the treatment and management of amyotrophic lateral sclerosis so difficult?","authors":"Philippe Corcia, Rachele Piras, Christian Lunetta","doi":"10.1080/14737175.2024.2415002","DOIUrl":"10.1080/14737175.2024.2415002","url":null,"abstract":"","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142399861","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"An overview of ganaxolone as a treatment for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder.","authors":"Alfie Gould, Sam Amin","doi":"10.1080/14737175.2024.2385937","DOIUrl":"10.1080/14737175.2024.2385937","url":null,"abstract":"<p><strong>Introduction: </strong>Cyclin-dependent kinase-Like 5 (CDKL5) deficiency disorder (CDD) is a rare neurodevelopmental condition commonly characterized by drug-resistant, refractory epilepsy, and seizures beginning in infancy. Most patients use multiple drugs, yet seizures remain difficult to control. So far, no conventional anti-seizure medications have been proven to be effective in individuals with CDD, in well-conducted studies.</p><p><strong>Areas covered: </strong>In this review, the authors assess the pharmacokinetics, early studies and appraise a recent study investigating the efficacy and safety of the oral suspension of ganaxolone (3α-hydroxy-3β-methyl-5α-pregnan-20-one) as an adjunctive therapy to treat seizures in CDD. The authors also discuss the impact of this drug on non-seizure outcomes.</p><p><strong>Expert opinion: </strong>Ganaxolone is a neuroactive 3β-methylated synthetic analogue of the potent agonist of gamma-aminobutyric acid type A receptors, allopregnanolone. Ganaxolone is the only drug that has been studied in a robust randomized controlled trial and been proven to be effective in this population.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141855339","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
William Blanks, Marcus Hanshaw, Daniela A Perez-Chadid, Brandon Lucke-Wold
{"title":"Emerging frontiers in Chronic Traumatic Encephalopathy: early diagnosis and implications for neurotherapeutic interventions.","authors":"William Blanks, Marcus Hanshaw, Daniela A Perez-Chadid, Brandon Lucke-Wold","doi":"10.1080/14737175.2024.2385952","DOIUrl":"10.1080/14737175.2024.2385952","url":null,"abstract":"<p><strong>Introduction: </strong>Chronic Traumatic Encephalopathy (CTE) is a neurodegenerative disorder associated with repetitive head trauma. Historically, the diagnosis has been primarily clinical, which has hindered definitive early diagnosis and proactive intervention.</p><p><strong>Areas covered: </strong>The authors analyze the recent advancements in early diagnosis of CTE by examining biomarkers, imaging, and clinical decision tools. They discuss the identification of neuropathologies - such as tau aggregates - through novel techniques ranging from blood sampling and to brain density scanning. The reader will walk away with a better understanding of current advancements in early detection and be better equipped to deal with encephalopathies secondary to trauma in clinical practice.</p><p><strong>Expert opinion: </strong>Tremendous progress has been made in understanding the pathophysiology of CTE. Despite these advancements, CTE treatment is still primarily symptomatic rather than underlying disease. Future research should focus on integrating current understanding of CTE pathophysiology with treatment modalities.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141906368","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Alessandra Burini, Gaia Pellitteri, Giovanni Merlino, Annacarmen Nilo, Yan Tereshko, Pierluigi Dolso, Gian Luigi Gigli, Mariarosaria Valente
{"title":"Current and emerging pharmaceutical strategies for the treatment and management of restless legs syndrome.","authors":"Alessandra Burini, Gaia Pellitteri, Giovanni Merlino, Annacarmen Nilo, Yan Tereshko, Pierluigi Dolso, Gian Luigi Gigli, Mariarosaria Valente","doi":"10.1080/14737175.2024.2385947","DOIUrl":"10.1080/14737175.2024.2385947","url":null,"abstract":"<p><strong>Introduction: </strong>Restless legs syndrome (RLS) is a sensory-motor sleep disorder that affects up to 13% of adults in the Western world and 2-4% of children. It impairs night sleep with an impact on daily performances and life quality. Thus, moderate-to-severe RLS requires pharmacological treatment.</p><p><strong>Areas covered: </strong>In the present review, which is based on PubMed searches with no time limits, the authors discuss the recommended pharmacotherapy for RLS in addition to other emerging treatment options. The authors provide coverage to the current recommendations for both adults and pediatric patients with RLS.</p><p><strong>Expert opinion: </strong>Current evidence suggests removing all causes of secondary RLS, including iron deficiency, chronic renal failure, drugs, and treating other sleep disorders that may worsen symptoms. Also, intermittent RLS should be addressed with behavioral measures and on-demand therapy. For chronic persistent RLS, α<sub>2</sub>δ calcium channel ligands are a first-line pharmacological approach, whereas dopamine agonists are associated with increased risk and should be spared. When RLS is refractory to first-line treatment, polytherapy, or opioid monotherapy should be considered. Nonetheless, some patients may not reach sustained symptom relief. Further research is needed to better understand the pathophysiology of RLS and to develop newer more effective drugs.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141855340","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"An update on the pharmacological management of Tourette syndrome and emerging treatment paradigms.","authors":"Abhishek Lenka, Joseph Jankovic","doi":"10.1080/14737175.2024.2382463","DOIUrl":"10.1080/14737175.2024.2382463","url":null,"abstract":"<p><strong>Introduction: </strong>Tourette syndrome (TS) is a childhood-onset neurobehavioral disorder characterized by tics. Pharmacotherapy is advised for patients whose symptoms affect their quality of life.</p><p><strong>Areas covered: </strong>The authors review the tic phenomenology and TS diagnostic criteria. The bulk of this article focuses on pharmacotherapeutic options for treating tics. They also highlight pharmacotherapies in the research pipeline.</p><p><strong>Expert opinion: </strong>Tic treatment must be tailored to individual needs. Behavioral therapy is the first line of treatment. Most with bothersome tics need pharmacotherapy and rarely, for medication-refractory cases, surgical therapy is indicated. Alpha-2 agonists are considered in patients with mild tics, especially in those with attention deficit with or without hyperactivity. Second-generation antipsychotics like aripiprazole and tiapride may be considered for severe tics. However, prescribers should be mindful of potential side effects, especially drug-induced movement disorders. Botulinum toxin injections may be considered for focal motor tics. Topiramate can be considered when other treatments are ineffective, and its benefits outweigh the risks. The same holds true for vesicular monoamine transporter-2 inhibitors, as they are deemed to be safe and effective in real-world use and open-label trials despite not meeting primary endpoints in placebo-controlled trials. Cannabinoids may be considered in adults if the approaches above do not control tics.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141733886","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Giuseppe Schirò, Salvatore Iacono, Giuseppe Salemi, Paolo Ragonese
{"title":"The pharmacological management of myelin oligodendrocyte glycoprotein-immunoglobulin G associated disease (MOGAD): an update of the literature.","authors":"Giuseppe Schirò, Salvatore Iacono, Giuseppe Salemi, Paolo Ragonese","doi":"10.1080/14737175.2024.2385941","DOIUrl":"10.1080/14737175.2024.2385941","url":null,"abstract":"<p><strong>Introduction: </strong>Myelin oligodendrocyte glycoprotein-immunoglobulin G associated disease (MOGAD) is a clinical entity distinct from multiple sclerosis and aquaporin-4 (AQP4+)-IgG-positive neuromyelitis optica spectrum disorder. There is a lack of evidence regarding the efficacy and safety of current treatments used for MOGAD.</p><p><strong>Areas covered: </strong>In this article, the authors review the currently available literature on the pharmacological management of MOGAD. This article is based on an extensive search for articles including meta-analyses, clinical trials, systematic reviews, observational studies, case series and case reports.</p><p><strong>Expert opinion: </strong>Intravenous high-dose methylprednisolone is the most common therapy for acute attack with patients having a good treatment response. In cases with poor recovery, intravenous immunoglobulins (IVIG) or plasma-exchange proved to be effective. Maintenance therapies include mycophenolate mofetil, azathioprine, IVIG, oral corticosteroids, rituximab, and interleukin-6 receptor (IL6-R) antagonists. Rituximab is the most used drug while IL6-R antagonists emerged as an effective option for people not responding to current treatments. Larger prospective studies with longer follow-ups are needed to confirm whether the blockage of the IL6-R is an effective and safe option. Since there is no evidence of major safety issues related to the new available therapies, the authors believe that waiting for disease activity to consider a possible treatment change, is an unwise approach.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897179","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Guidance on antipsychotic selection for agitation and aggressive behavior in persons with Huntington's disease.","authors":"James E Eaton, Daniel O Claassen","doi":"10.1080/14737175.2024.2376836","DOIUrl":"10.1080/14737175.2024.2376836","url":null,"abstract":"","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141563081","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
David W Goodman, Samuele Cortese, Stephen V Faraone
{"title":"Why is ADHD so difficult to diagnose in older adults?","authors":"David W Goodman, Samuele Cortese, Stephen V Faraone","doi":"10.1080/14737175.2024.2385932","DOIUrl":"10.1080/14737175.2024.2385932","url":null,"abstract":"","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141888897","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Andrea Amerio, Gabriele Arduino, Fabio Fesce, Alessandra Costanza, Andrea Aguglia, Mario Amore, Gianluca Serafini
{"title":"Advances in the management of bipolar disorder in children and adolescents: an update on the literature.","authors":"Andrea Amerio, Gabriele Arduino, Fabio Fesce, Alessandra Costanza, Andrea Aguglia, Mario Amore, Gianluca Serafini","doi":"10.1080/14737175.2024.2386429","DOIUrl":"10.1080/14737175.2024.2386429","url":null,"abstract":"<p><strong>Introduction: </strong>Early diagnosis and treatment concerning bipolar disorder (BD) are related to a better functioning over the long-term period. Although pharmacotherapy is indicated for approximately all youths with BD, nearly one-third of patients do not receive adequate medications for their condition.</p><p><strong>Areas covered: </strong>The authors discuss the available scientific evidence from the current literature about the management of BD in both children and adolescents, giving particular focus to the efficacy and tolerability of the available pharmacological agents. Studies were identified searching MEDLINE and retrieved from reference listings of relevant articles and through consultation with experts in the field.</p><p><strong>Expert opinion: </strong>Many D2-blockers, approved by the Food and Drug Administration (FDA) based on their antimanic properties in youths, are related to both short- and long-term side effects. Lurasidone was found to be effective for the treatment of acute juvenile bipolar depression, while lithium for the treatment and recurrence prevention of manic/mixed episodes. The most common anticonvulsants were found to be most useful as adjunctive antimanic agents in non-responders to first-line monotherapies. No data was found to support the use of antidepressants in juvenile BD.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141888895","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Mathilde Chevignard, Hugo Câmara-Costa, Georges Dellatolas
{"title":"Predicting and improving outcome in severe pediatric traumatic brain injury.","authors":"Mathilde Chevignard, Hugo Câmara-Costa, Georges Dellatolas","doi":"10.1080/14737175.2024.2389921","DOIUrl":"10.1080/14737175.2024.2389921","url":null,"abstract":"<p><strong>Introduction: </strong>Severe pediatric traumatic brain injury (spTBI), including abusive head trauma (AHT) in young children, is a major public health problem. Long-term consequences of spTBI include a large variety of physical, neurological, biological, cognitive, behavioral and social deficits and impairments.</p><p><strong>Areas covered: </strong>The present narrative review summarizes studies and reviews published from January 2019 to February 2024 on spTBI. Significant papers published before 2019 were also included. The article gives coverage to the causes of spTBI, its epidemiology and fatality rates; disparities, inequalities, and socioeconomic factors; critical care; outcomes; and interventions.</p><p><strong>Expert opinion: </strong>There are disparities between countries and according to socio-economic factors regarding causes, treatments and outcomes of spTBI. AHT has an overall poor outcome. Adherence to critical care guidelines is imperfect and the evidence-base of guidelines needs further investigations. Neuroimaging and biomarker predictors of outcomes is a rapidly evolving domain. Long-term cognitive, behavioral and psychosocial difficulties are the most prevalent and disabling. Their investigation should make a clear distinction between objective (clinical examination, cognitive tests, facts) and subjective measures (estimations using patient- and proxy-reported questionnaires), considering possible common source bias in reported difficulties. Family/caregiver-focused interventions, ecological approaches, and use of technology in delivery of interventions are recommended to improve long-term difficulties after spTBI.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141975454","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}