Expert Opinion on Pharmacotherapy最新文献

Pharmacological management of PCOS: trends and insights from a 10-year bibliometric analysis. 多囊卵巢综合征的药物管理：10年文献计量学分析的趋势和见解。

IF 2.5 3区医学

Expert Opinion on Pharmacotherapy Pub Date : 2025-07-15 DOI: 10.1080/14656566.2025.2535175

Rohit Gautam, Anshu Jyoti, Asmitha Bhateja, Neena Malhotra, Taruna Arora

{"title":"Pharmacological management of PCOS: trends and insights from a 10-year bibliometric analysis.","authors":"Rohit Gautam, Anshu Jyoti, Asmitha Bhateja, Neena Malhotra, Taruna Arora","doi":"10.1080/14656566.2025.2535175","DOIUrl":"https://doi.org/10.1080/14656566.2025.2535175","url":null,"abstract":"Background: PCOS is an endocrine disorder affecting women of reproductive aged group. It has multiple manifestations including metabolic, reproductive, and psychological domains. Among PCOS patient's pharmaceutical management remains a primary approach to its treatment, particularly when lifestyle changes do not yield improvements.Objective: This study aims to highlight trends in global research concerning PCOS and its pharmacological treatment within the last decade using bibliometric analysis.Methods: A bibliometric analysis was conducted for the 2015-2024 timeframe using Web of Science database. Search queries were designed on PCOS as well its pharmacological treatment options. Data analysis was performed through visualization of collaborations, research trends etcin VOS viewer.Results: 2607 publications qualified for inclusion criteria. The most frequently treated topics were combined oral contraceptives, metformin, letrozole and inositols. China is the leading contributor toward publication volume while the U.S.A. was most cited and had the strongest links. Influence appeared to stem from Monash University whose Legro RS was deemed the most influential institution and there alongside the most impactful author.Conclusion: The study reveals global trends in PCOS pharmacotherapy, with growing focus on metabolic and molecular aspects. Key contributors and emerging therapies highlight the expanding scope and future potential of PCOS treatment research.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":""},"PeriodicalIF":2.5,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144636641","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Efficacy and safety of pitolisant in children above 6 years with narcolepsy. 匹立抗治疗6岁以上儿童发作性睡病的疗效和安全性。

IF 2.5 3区医学

Expert Opinion on Pharmacotherapy Pub Date : 2025-07-06 DOI: 10.1080/14656566.2025.2523989

Beatrice Bertini, Claudio Liguori

{"title":"Efficacy and safety of pitolisant in children above 6 years with narcolepsy.","authors":"Beatrice Bertini, Claudio Liguori","doi":"10.1080/14656566.2025.2523989","DOIUrl":"10.1080/14656566.2025.2523989","url":null,"abstract":"Introduction: The newly approved use of pitolisant in pediatric narcolepsy marks a significant advancement for patients and clinicians, given the scarcity of medications for this age group that are both safe and effective in reducing narcolepsy symptoms and improving quality of life.Areas covered: This article covers the use of pitolisant for treating narcolepsy type 1 (NT1) and 2 (NT2) in pediatric patients considering drug's pharmacokinetics and pharmacodynamics. By integrating recent literature and real-world data, the safety, the tolerability and the efficacy of this drug have been analyzed, also including evidence drawn from studies involving patients with Prader-Willi Syndrome.Expert opinion: Pitolisant represents a groundbreaking treatment for pediatric narcolepsy, addressing the paucity of safe and effective options for this age group. Its unique mechanism as a histamine H3 receptor antagonist reduces excessive daytime sleepiness and cataplexy while offering cognitive benefits. With a favorable safety profile and good tolerability, pitolisant efficiently outperforms traditional therapies, which often have distressing side effects. Its use is particularly critical in childhood, a developmental stage where factors like growth, school performance, and socialization must be carefully considered, making it a transformative option for pediatric care.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1-10"},"PeriodicalIF":2.5,"publicationDate":"2025-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144483689","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Evaluation of the effect of SGLT-2 inhibitors on atrial fibrillation recurrence in diabetic patients. SGLT-2抑制剂对糖尿病患者房颤复发的影响。

IF 2.5 3区医学

Expert Opinion on Pharmacotherapy Pub Date : 2025-07-03 DOI: 10.1080/14656566.2025.2527189

Mücahit Yarar, Hasan Ari, Selma Ari, Ahmet Tütüncü, Mehmet Melek, Tahsin Bozat

{"title":"Evaluation of the effect of SGLT-2 inhibitors on atrial fibrillation recurrence in diabetic patients.","authors":"Mücahit Yarar, Hasan Ari, Selma Ari, Ahmet Tütüncü, Mehmet Melek, Tahsin Bozat","doi":"10.1080/14656566.2025.2527189","DOIUrl":"10.1080/14656566.2025.2527189","url":null,"abstract":"Background: The aim of this study was to investigate the effect of SGLT-2 (sodium glucose cotransporter-2) inhibitors on AF (atrial fibrillation) recurrence in patients diagnosed with diabetes mellitus (DM) who underwent Direct Current Cardioversion (DCCV) in our clinic.Research design and methods: Consecutive DCCV was performed to achieve 120 patients in SR (60 DM patients who used SGLT-2 inhibitors and 60 DM patients who didn't use SGLT-2 inhibitors). The two groups (SGLT-2 inhibitor user group and control group) were monitored for AF recurrence on the first day, then at one, three, and six months after CV.Results: During the six-month follow-up period, AF recurrence developed in 24 (40%) patients in SGLT-2 inhibitor group and in 35 (58.3%) of the control group, p = 0.04. In univariable analysis, AF duration, non-use of SGLT-2 inhibitor, left atrium diameter, TAPSE value and in multivariable analysis, AF duration OR: 4.98; 95%CI (2.39-10.38), p < 0,001 and non-use of SGLT-2 inhibitors OR: 0.35; 95%CI (0.13-0.90), p = 0.03 were found to be independent predictors for AF recurrence.Conclusion: AF recurrence ratio is significantly lower in patients using SGLT-2 inhibitors, in six-month follow-up period. AF duration and SGLT-2 inhibitor non-use were independent predictors, for AF recurrence in DM patients (NCT06951737).Clinical trial registration: www.clinicaltrials.gov identifier is: NCT06951737.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1-7"},"PeriodicalIF":2.5,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144527030","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Efficacy and safety of Mitapivat in sickle cell disease: a systematic review. 米他伐治疗镰状细胞病的疗效和安全性：一项系统综述。

IF 2.5 3区医学

Expert Opinion on Pharmacotherapy Pub Date : 2025-07-01 Epub Date: 2025-07-07 DOI: 10.1080/14656566.2025.2514504

Shalu R Chaudhary, Komal Sharma, Mahalaqua Nazli Khatib, Deepak B Saxena

{"title":"Efficacy and safety of Mitapivat in sickle cell disease: a systematic review.","authors":"Shalu R Chaudhary, Komal Sharma, Mahalaqua Nazli Khatib, Deepak B Saxena","doi":"10.1080/14656566.2025.2514504","DOIUrl":"10.1080/14656566.2025.2514504","url":null,"abstract":"Background: Sickle cell disease (SCD) is a chronic hemoglobinopathy marked by hemolytic anemia, VOEs, and multiorgan complications. Mitapivat, an oral Pyruvate Kinase (PK) activator, has emerged as a potential disease-modifying therapy. This systematic review assessed the efficacy and safety of Mitapivat in individuals with SCD.Methods: A systematic literature search was conducted from different search engines from inception. The review followed PRISMA 2020 guidelines, and the protocol was registered in PROSPERO [CRD420251000061]. RCTs and NRSIs evaluating Mitapivat monotherapy were included. ROB was assessed. A narrative synthesis was undertaken due to heterogeneity.Results: One RCT and two NRSIs involving 156 participants were included. Reduced annualized Vaso-occlusive episodes (VOE) rates compared to placebo by 51.6% (50 mg) and 70.0% (100 mg). Statistically significant hemoglobin response was observed in 46.2%-56% of participants. Increased markers of hemolysis and reduced transfusion requirements in Mitapivat groups were reported. However, SAEs were low, in 8%-19% of Mitapivat recipients.Conclusions: Preliminary evidence suggests Mitapivat demonstrates potential to reduce VOEs, improve hemoglobin levels, and decrease hemolysis with an acceptable safety profile. However, small sample sizes, lack of functional outcomes, and limited follow-up warrant cautious interpretation. Further large-scale RCTs are needed to validate these findings and establish a long-term benefit-risk balance.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1229-1236"},"PeriodicalIF":2.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144575145","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Potential of cenobamate as a broad-spectrum antiseizure medication. 辛奥巴酸作为广谱抗癫痫药物的潜力。

IF 2.5 3区医学

Expert Opinion on Pharmacotherapy Pub Date : 2025-07-01 Epub Date: 2025-06-11 DOI: 10.1080/14656566.2025.2517352

Susan M Melnick, Sunita N Misra, Marc Kamin, Louis Ferrari, Kelli J Glenn

{"title":"Potential of cenobamate as a broad-spectrum antiseizure medication.","authors":"Susan M Melnick, Sunita N Misra, Marc Kamin, Louis Ferrari, Kelli J Glenn","doi":"10.1080/14656566.2025.2517352","DOIUrl":"10.1080/14656566.2025.2517352","url":null,"abstract":"Introduction: The process of diagnosing patients with epilepsy and selecting the optimal drug remains a significant challenge. Broad-spectrum antiseizure medications (ASMs) treat focal and generalized seizures without worsening other seizure types, making them the drug of choice when the exact seizure classification for patients is unknown. Cenobamate, an ASM approved for the treatment of adult focal seizures, has demonstrated strong efficacy, including high rates of seizure freedom. Evidence suggests that cenobamate has potential to be a broad-spectrum ASM.Areas covered: Considerations for the selection of broad- versus narrow-spectrum ASMs are discussed and an overview of preclinical, clinical, and real-world evidence relating to cenobamate as a potential broad-spectrum ASM is presented.Expert opinion: Combined evidence from preclinical data, clinical study data, and real-world evidence may indicate that cenobamate may be a broad-spectrum ASM. Evidence is accumulating regarding the utility of cenobamate in treating primary generalized tonic-clonic (PGTC) and seizures associated with developmental epileptic encephalopathies such as Lennox-Gastaut syndrome and Dravet syndrome. Results from the ongoing cenobamate trial in patients with idiopathic generalized epilepsy will determine the efficacy of cenobamate for the treatment of PGTC seizures.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1177-1189"},"PeriodicalIF":2.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144233652","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Tackling multidrug-resistant Gram-negative infections in children globally: current therapeutic options and perspectives. 应对全球儿童多重耐药革兰氏阴性感染：目前的治疗选择和前景。

IF 2.5 3区医学

Expert Opinion on Pharmacotherapy Pub Date : 2025-07-01 Epub Date: 2025-06-23 DOI: 10.1080/14656566.2025.2519690

Lorenzo Chiusaroli, Costanza Tripiciano, Cecilia Liberati, Marica De Pieri, Giulia Brigadoi, Daniele Donà

{"title":"Tackling multidrug-resistant Gram-negative infections in children globally: current therapeutic options and perspectives.","authors":"Lorenzo Chiusaroli, Costanza Tripiciano, Cecilia Liberati, Marica De Pieri, Giulia Brigadoi, Daniele Donà","doi":"10.1080/14656566.2025.2519690","DOIUrl":"10.1080/14656566.2025.2519690","url":null,"abstract":"Introduction: Over the past two decades, the global burden of multidrug-resistant organisms has grown steadily, representing a major concern in pediatric healthcare. Among these, hospital-acquired infections caused by multidrug-resistant Gram-negative bacteria (MDR-GNB) are particularly challenging to manage in children, due to limited therapeutic options and the scarcity of pharmacokinetic data in the pediatric population. Although several new antibiotics - especially β-lactams combined with β-lactamase inhibitors - have become available, uncertainties remain regarding their optimal use in pediatric populations.Areas covered: This review explores potential treatment strategies for MDR-GNB infections in children, with a focus on pathogens listed in the WHO priority list. It examines the pharmacological properties of both traditional and newly approved antibiotics, assessing their role and applicability in pediatric clinical practice.Expert opinion: New β-lactam antibiotics, alone or in combination with β-lactamase inhibitors - such as ceftazidime-avibactam, ceftolozane-tazobactam, meropenem-vaborbactam, imipenem-cilastatin-relebactam, and cefiderocol - have emerged as preferred options for treating carbapenem-resistant and difficult-to-treat Gram-negative infections. Therapy should be guided by pathogen identification and resistance mechanisms, as susceptibility profiles vary widely based on the resistance-mechanism. Older agents like colistin, fosfomycin, nitrofurantoin, and aminoglycosides remain important, particularly in resource-limited settings, despite concerns over toxicity and safety.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1205-1220"},"PeriodicalIF":2.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144301457","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Low-grade IDH-mutant gliomas: from standard post-surgical treatments to novel IDH inhibitors. 低级别IDH突变胶质瘤：从标准的术后治疗到新型IDH抑制剂。

IF 2.5 3区医学

Expert Opinion on Pharmacotherapy Pub Date : 2025-07-01 Epub Date: 2025-06-06 DOI: 10.1080/14656566.2025.2516617

Roberta Rudà, Francesco Bruno, Alessia Pellerino, Edoardo Pronello, Riccardo Soffietti

{"title":"Low-grade IDH-mutant gliomas: from standard post-surgical treatments to novel IDH inhibitors.","authors":"Roberta Rudà, Francesco Bruno, Alessia Pellerino, Edoardo Pronello, Riccardo Soffietti","doi":"10.1080/14656566.2025.2516617","DOIUrl":"10.1080/14656566.2025.2516617","url":null,"abstract":"Introduction: Adult-type IDH-mutant diffuse gliomas grade 2 are rare tumors mainly affecting young patients, classified by WHO 2021 into IDH-mutant astrocytomas and IDH-mutant 1p/19q codeleted oligodendrogliomas. IDH-mutant grade 2 gliomas are slowly growing tumors; however, they grow continuously, and almost all patients will ultimately recur. Surgical resection is the first option, followed by observation with MRI in low-risk patients and radio-chemotherapy in high-risk patients. Early clinical trials and phase 3 INDIGO trial have demonstrated the efficacy of vorasidenib, a dual IDH1/2 inhibitor, in prolonging imaging-based progression-free survival and time-to-next-intervention.Areas covered: This review covers the following areas: importance of surgical resection, traditional treatments after surgery, mechanisms of IDH mutations and IDH inhibitors in preclinical models, early clinical studies on ivosidenib and vorasidenib, INDIGO trial, the future role of vorasidenib, open issues beyond INDIGO trial, and novel IDH targeting strategies.Expert opinion: IDH1/2 mutations are ideal targets of therapy and early clinical studies and INDIGO phase 3 trial confirmed the clinical efficacy of vorasidenib. Long-term follow-up is needed to better define the efficacy across different subgroups of patients. Overall, vorasidenib will replace observation with MRI for low-risk patients and allow to delay radiotherapy and chemotherapy and their adverse effects.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1163-1175"},"PeriodicalIF":2.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144247197","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

External control arm with real world data to assess the effect of semaglutide on chronic kidney disease risk among patients with type 2 diabetes. 用真实世界数据评估西马鲁肽对2型糖尿病患者慢性肾脏疾病风险的影响的外部对照。

IF 2.5 3区医学

Expert Opinion on Pharmacotherapy Pub Date : 2025-07-01 Epub Date: 2025-06-13 DOI: 10.1080/14656566.2025.2518329

Onur Baser, Yuanqing Lu

{"title":"External control arm with real world data to assess the effect of semaglutide on chronic kidney disease risk among patients with type 2 diabetes.","authors":"Onur Baser, Yuanqing Lu","doi":"10.1080/14656566.2025.2518329","DOIUrl":"10.1080/14656566.2025.2518329","url":null,"abstract":"Background: Clinical trials have shown semaglutide effective in mitigating risks associated with type 2 diabetes and chronic kidney disease. However, semaglutide's real-world effectiveness and long-term outcomes are not fully established.Research design & methods: Using 2019-2024 Kythera Labs data, an external control arm was created using criteria identified in the FLOW clinical trial. Primary outcomes were major kidney disease events (kidney failure onset and ≥ 50% reduction in estimated glomerular filtration rate from baseline). Propensity score matching and Cox regression were used to determine risk-adjusted outcomes.Results: The control arm (n = 896,257) was compared with the clinical trial cohort (n = 1,766). After propensity score matching on age, sex, socioeconomic status, and comorbidities, semaglutide treatment was associated with a 26% reduction in primary event risk compared with the comparator group (702 vs 1,068 events; HR: 0.74; 95% CI, 0.67-0.81), consistent with the 24% risk reduction observed in the clinical trial.Conclusion: Semaglutide treatment was linked to a significantly lower risk of clinically relevant renal outcomes. Our findings provide robust real-world evidence that supports the FLOW trial results regarding the renoprotective effects of semaglutide, highlighting its promise as an effective therapeutic option for managing renal complications.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1237-1243"},"PeriodicalIF":2.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144257685","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Hereditary angioedema treatment beyond biologics: current state of preventive and on-demand approaches and new perspectives. 超越生物制剂的遗传性血管性水肿治疗：预防和按需方法的现状和新观点。

IF 2.5 3区医学

Expert Opinion on Pharmacotherapy Pub Date : 2025-07-01 Epub Date: 2025-06-24 DOI: 10.1080/14656566.2025.2509782

Andrea Zanichelli, Giada De Angeli, Irene Baroni, Marta Mansi, Giuseppe Caravella, Rosario Caruso

{"title":"Hereditary angioedema treatment beyond biologics: current state of preventive and on-demand approaches and new perspectives.","authors":"Andrea Zanichelli, Giada De Angeli, Irene Baroni, Marta Mansi, Giuseppe Caravella, Rosario Caruso","doi":"10.1080/14656566.2025.2509782","DOIUrl":"10.1080/14656566.2025.2509782","url":null,"abstract":"Introduction: Hereditary angioedema (HAE) is a genetic rare condition characterized by recurrent attacks of swelling that might be potentially life-threatening. Recurrence and severity of attacks may impact psychological life, expectations and productivity. We aim to review the state-of-the-art of HAE preventive and on-demand treatment of non-biologic drugs, providing a perspective of their personalized use and development.Areas covered: This literature analysis integrates international guidelines and clinical trial data on on-demand therapies and short-/long-term prophylaxis. Modern medications should be considered and personalized for HAE patients to provide benefits compatible with patients' lifestyles, preferences, and experiences. Accordingly, a new era toward oral formulations has begun starting from berotralstat, with a consistent number of drugs under development.Expert opinion: All HAE patients should have an effective on-demand treatment available in case of attacks. Long-term prophylaxis (LTP) should be considered and individualized for all patients at every visit, following a shared decision-making approach to optimize disease control while limiting side effects. Parenteral administration of LTP is associated with treatment complexities and barriers. Oral treatment could address practical needs for HAE patients both in preventive and on-demand setting, avoiding injection-related side effects, reducing treatment burden, and improving quality of life. In the next future, significant advances in HAE therapeutics could result from gene therapy.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1221-1228"},"PeriodicalIF":2.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144474394","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Current and emerging pharmacotherapies in Lennox-Gastaut syndrome. lenox - gastaut综合征的当前和新兴药物治疗。

IF 2.5 3区医学

Expert Opinion on Pharmacotherapy Pub Date : 2025-07-01 Epub Date: 2025-06-29 DOI: 10.1080/14656566.2025.2516630

Debopam Samanta, Manan Nath

{"title":"Current and emerging pharmacotherapies in Lennox-Gastaut syndrome.","authors":"Debopam Samanta, Manan Nath","doi":"10.1080/14656566.2025.2516630","DOIUrl":"10.1080/14656566.2025.2516630","url":null,"abstract":"Introduction: Lennox-Gastaut syndrome (LGS) is a severe childhood-onset developmental and epileptic encephalopathy characterized by multiple drug-resistant seizures, cognitive impairments, and distinctive EEG patterns. Given its profound impact on patients' quality of life, developing effective pharmacotherapies remains a critical clinical challenge.Areas covered: This review examines FDA-approved medications for LGS (clonazepam, felbamate, lamotrigine, topiramate, rufinamide, clobazam, cannabidiol, and fenfluramine), commonly used off-label antiseizure medications, emerging treatments in clinical trials, and precision therapeutics targeting etiology-specific mechanisms. The literature encompasses randomized controlled trials, observational studies, and expert consensus statements on treatment approaches and challenges.Expert opinion: Despite therapeutic advances, most patients with LGS lack individualized treatment plans with regular adjustments. Current management requires a multimodal approach integrating pharmacotherapy with other interventions. Future progress depends on improved natural history studies, standardized data collection, advanced preclinical models, innovative trial designs, and addressing healthcare inequities. While emerging precision therapies targeting genetic causes show promise, the field urgently needs better strategies to optimize existing treatments while developing disease-modifying approaches that address both seizures and non-seizure outcomes.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1133-1147"},"PeriodicalIF":2.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144224874","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0