Expert Review of Clinical Pharmacology最新文献

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Messenger interference RNA therapies targeting apolipoprotein C-III and angiopoietin-like protein 3 for mixed hyperlipidemia: the future of plozasiran and zodasiran. 针对脂蛋白 C-III 和血管生成素样蛋白 3 的信使干扰 RNA疗法治疗混合型高脂血症:plozasiran 和 zodasiran 的未来。
IF 4.3 3区 医学
Expert Review of Clinical Pharmacology Pub Date : 2024-11-01 Epub Date: 2024-11-03 DOI: 10.1080/17512433.2024.2423724
Zonghao Pan, Muhammad Adnan Zaman, Sidra Kalsoom, Yani Zhang
{"title":"Messenger interference RNA therapies targeting apolipoprotein C-III and angiopoietin-like protein 3 for mixed hyperlipidemia: the future of plozasiran and zodasiran.","authors":"Zonghao Pan, Muhammad Adnan Zaman, Sidra Kalsoom, Yani Zhang","doi":"10.1080/17512433.2024.2423724","DOIUrl":"10.1080/17512433.2024.2423724","url":null,"abstract":"<p><strong>Introduction: </strong>Mixed hyperlipidemia represents a substantial public health issue and a considerable burden on healthcare systems. Although the introduction of statins and LDL-cholesterol lowering agents have significantly reduced the incidence of atherosclerotic cardiovascular diseases (ASCVD), a significant portion of the population continues to exhibit ASCVD progression due to elevated triglyceride-rich lipoprotein (TRL) levels. This persistent risk has catalyzed the development of novel pharmacological interventions targeting these lipoproteins.</p><p><strong>Areas covered: </strong>Our special report commenced with a targeted PubMed search using keywords such as 'plozasiran,' 'zodasiran,' and terms related to APOC3 and ANGPTL3. As the review progressed, emergent research questions guided further searches, allowing for the inclusion of additional relevant articles to comprehensively illustrate the linkage between TRLs and cardiovascular disease, discuss the roles of APOC3, ANGPTL3, and the pharmaceutical agents that target these proteins, and provide a comparison on the ARCHES-2 and MUIR trials.</p><p><strong>Expert opinion: </strong>The ARCHES-2 and MUIR trials demonstrated effective triglyceride reduction by these therapies, yet it is uncertain if this correlates with significant clinical benefits. Advances in antisense oligonucleotide technology, especially the GalNAc delivery platform, show promise for personalized lipid management, though challenges such as cost and safety concerns remain.</p>","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":" ","pages":"1017-1023"},"PeriodicalIF":4.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142521483","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
Renin-angiotensin system inhibition and mortality in patients undergoing dialysis with coronary artery disease: insights from a multi-center observational study. 肾素-血管紧张素系统抑制与冠心病透析患者的死亡率:一项多中心观察研究的启示。
IF 3.6 3区 医学
Expert Review of Clinical Pharmacology Pub Date : 2024-11-01 Epub Date: 2024-10-23 DOI: 10.1080/17512433.2024.2419915
Enmin Xie, Shuoyan An, Yaxin Wu, Zixiang Ye, Xuecheng Zhao, Yike Li, Nan Shen, Yanxiang Gao, Jingang Zheng
{"title":"Renin-angiotensin system inhibition and mortality in patients undergoing dialysis with coronary artery disease: insights from a multi-center observational study.","authors":"Enmin Xie, Shuoyan An, Yaxin Wu, Zixiang Ye, Xuecheng Zhao, Yike Li, Nan Shen, Yanxiang Gao, Jingang Zheng","doi":"10.1080/17512433.2024.2419915","DOIUrl":"10.1080/17512433.2024.2419915","url":null,"abstract":"<p><strong>Background: </strong>While the survival benefits of angiotensin-converting enzyme inhibitors (ACEI) and angiotensin receptor blockers (ARB) are firmly established in the general population, their efficacy within patient undergoing dialysis with coronary artery disease (CAD) remains controversial.</p><p><strong>Methods: </strong>Between January 2015 and June 2021, 1168 patients undergoing dialysis with CAD were assessed from 30 tertiary medical centers. The primary outcome was all-cause death, and the secondary outcome was cardiovascular death. Inverse probability of treatment weighting (IPTW) and propensity score matching (PSM) were performed to account for between-group differences.</p><p><strong>Results: </strong>Overall, ACEI or ARB were prescribed to 518 patients (44.3%) upon discharge. After a median follow-up of 22.2 months, 361 (30.9%) patients died, including 243 cardiovascular deaths. The use of ACEI or ARB was associated with a significantly lower risk of all-cause (25.3% vs 35.4%, hazard ratio [HR] 0.65, 95% confidence interval [CI] 0.52-0.82, <i>p</i> < 0.001) and cardiovascular death (17.0% vs 23.8%; HR 0.64, 95% CI 0.48-0.83, <i>p</i> = 0.001). These findings remained consistent across IPTW and PSM analyses. Sensitivity analyses for ACEI and ARB use separately yielded similar results.</p><p><strong>Conclusions: </strong>Our findings suggested that among patients undergoing dialysis with CAD, ACEI or ARB use was associated with a lower risk of all-cause and cardiovascular death.</p>","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":" ","pages":"1053-1062"},"PeriodicalIF":3.6,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461438","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
Population pharmacokinetics of nirmatrelvir/ritonavir in critically ill Chinese COVID-19 patients and recommendations for medication use: a two-center retrospective study. 中国 COVID-19 重症患者中尼马瑞韦/利托那韦的群体药代动力学及用药建议:一项双中心回顾性研究。
IF 3.6 3区 医学
Expert Review of Clinical Pharmacology Pub Date : 2024-11-01 Epub Date: 2024-09-30 DOI: 10.1080/17512433.2024.2410385
Junjun Xu, Jinmeng Li, Meng Chen, Huifang Jiang, Xudong Fan, Yangmin Hu, Haili Shan, Mingdong Yang, Yichao Xu, Yuying Lang, Haibin Dai, Xinjun Cai
{"title":"Population pharmacokinetics of nirmatrelvir/ritonavir in critically ill Chinese COVID-19 patients and recommendations for medication use: a two-center retrospective study.","authors":"Junjun Xu, Jinmeng Li, Meng Chen, Huifang Jiang, Xudong Fan, Yangmin Hu, Haili Shan, Mingdong Yang, Yichao Xu, Yuying Lang, Haibin Dai, Xinjun Cai","doi":"10.1080/17512433.2024.2410385","DOIUrl":"10.1080/17512433.2024.2410385","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to establish population pharmacokinetics (PPK) models of nirmatrelvir/ritonavir in critically ill Chinese patients with the coronavirus disease 2019 (COVID-19) infection, explore factors affecting the pharmacokinetics (PK) of nirmatrelvir/ritonavir.</p><p><strong>Methods: </strong>A total of 285 serum samples and clinical data were collected from 152 patients. The PPK models of nirmatrelvir/ritonavir were analyzed using nonlinear mixed-effect modeling (NONMEM) approach. The optimal dosing regimen for patients with different renal function was determined using Monte Carlo simulations.</p><p><strong>Results: </strong>The population typical values of apparent clearance (CL/F) and apparent volume of distribution (V/F) of nirmatrelvir were 2.26 L/h and 15.3 L, respectively. Notably, creatinine clearance (CrCL) significantly influenced the PK variation of nirmatrelvir. Monte Carlo simulations suggested that patients with mild-to-moderate renal impairment experienced a 22.0-59.9% increase in the area under the curve (AUC) when they were administered a standard dose of nirmatrelvir compared to those with normal renal function. The AUC in patients with severe renal impairment after administration of 150 mg q12h nirmatrelvir was similar to that in patients with normal renal function after administration of 300 mg q12h nirmatrelvir.</p><p><strong>Conclusions: </strong>PPK modeling and simulation provided a reference for the rational clinical application of nirmatrelvir/ritonavir in critically ill Chinese patients.</p>","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":" ","pages":"1071-1079"},"PeriodicalIF":3.6,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142344373","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
Role of tenecteplase in ischemic stroke after 4.5 hours: an evaluation of the TRACE-III trial. 替奈替普酶在 4.5 小时后缺血性中风中的作用:TRACE-III 试验评估。
IF 3.6 3区 医学
Expert Review of Clinical Pharmacology Pub Date : 2024-11-01 Epub Date: 2024-11-11 DOI: 10.1080/17512433.2024.2427078
Muhammad Imtiaz, Muhammad Adnan Zaman, Abeel Naseer, Sidra Kalsoom
{"title":"Role of tenecteplase in ischemic stroke after 4.5 hours: an evaluation of the TRACE-III trial.","authors":"Muhammad Imtiaz, Muhammad Adnan Zaman, Abeel Naseer, Sidra Kalsoom","doi":"10.1080/17512433.2024.2427078","DOIUrl":"10.1080/17512433.2024.2427078","url":null,"abstract":"<p><p>Tenecteplase (TNK), as a thrombolytic treatment for acute ischemic stroke (AIS), has been found to be effective when used within 4.5 hours of symptom onset. However, the efficacy of TNK after 4.5 hours is not well established, especially in patients with large vessel occlusion and with no access to thrombectomy. In this article, we will discuss the results of the recently published TRACE-III trial. The study involved 516 patients with large vessel occlusion, either proximal middle cerebral artery or internal carotid artery, with salvageable brain tissue and no endovascular thrombectomy access. Key safety outcomes included symptomatic intracranial hemorrhage and death. TNK treatment resulted in a higher percentage of patients with a modified Rankin scale score of 0 or 1 at 90 days than standard medical treatment. Mortality at 90 days was 13.3% with TNK and 13.1% with standard medical treatment. The trial found that TNK treatment for Chinese patients with ischemic stroke resulted in less disability and similar survival compared to standard medical treatment. However, there was a higher incidence of symptomatic intracranial hemorrhage within 36 hours.</p>","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":" ","pages":"1013-1016"},"PeriodicalIF":3.6,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142581196","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
Prophylaxis and treatment of acute intraocular pressure rise after cataract surgery: considerations to aid in decision-making. 白内障手术后急性眼压升高的预防和治疗:辅助决策的考虑因素。
IF 4.3 3区 医学
Expert Review of Clinical Pharmacology Pub Date : 2024-11-01 Epub Date: 2024-11-11 DOI: 10.1080/17512433.2024.2427104
Eitan A Katz, Shivani Majmudar, Ahmad A Aref
{"title":"Prophylaxis and treatment of acute intraocular pressure rise after cataract surgery: considerations to aid in decision-making.","authors":"Eitan A Katz, Shivani Majmudar, Ahmad A Aref","doi":"10.1080/17512433.2024.2427104","DOIUrl":"10.1080/17512433.2024.2427104","url":null,"abstract":"","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":" ","pages":"995-997"},"PeriodicalIF":4.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142580974","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
Methotrexate polyglutamates. 甲氨蝶呤多聚谷氨酸盐。
IF 3.6 3区 医学
Expert Review of Clinical Pharmacology Pub Date : 2024-11-01 Epub Date: 2024-10-15 DOI: 10.1080/17512433.2024.2416674
Zhilin Yang, Jiayi Mo, Wenshu Li, Zhigang Zhao, Shenghui Mei
{"title":"Methotrexate polyglutamates.","authors":"Zhilin Yang, Jiayi Mo, Wenshu Li, Zhigang Zhao, Shenghui Mei","doi":"10.1080/17512433.2024.2416674","DOIUrl":"10.1080/17512433.2024.2416674","url":null,"abstract":"<p><strong>Introduction: </strong>Methotrexate polyglutamates (MTXPGs) are intracellular metabolites of methotrexate (MTX) that play a critical role in the drug's activity, influencing both its efficacy and toxicity. As the exact implications of MTXPGs in these processes remain a subject of debate, a comprehensive review of MTXPGs could provide valuable insights for clinicians and pharmacists, potentially minimizing adverse reactions and enhancing therapeutic outcomes.</p><p><strong>Areas covered: </strong>A comprehensive search of relevant literature was conducted in PubMed and Web of Science databases, including studies from their inception to April 2024. Eligible studies included reviews, clinical trials, and real-world analyses. Additional manual searches and citation reviews were also performed. This review aims to explore MTXPGs with a primary focus on their pharmacokinetics, analytical methods, determinants of drug exposure, and their correlation with MTX efficacy and toxicity.</p><p><strong>Expert opinion: </strong>MTXPGs have not yet garnered significant attention in clinical practice. However, multiple studies have demonstrated a relationship between MTXPGs and the efficacy and toxicity of MTX, suggesting a potential avenue for personalized treatment strategies. Future research should aim to further validate and refine this correlation. Additionally, attention should also be directed toward other metabolites of MTX, which may hold clinical significance.</p>","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":" ","pages":"1025-1037"},"PeriodicalIF":3.6,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142399864","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
Plasma protein binding of arsenic species in acute promyelocytic leukemia patients and their relationships with hepatic and renal function. 急性早幼粒细胞白血病患者血浆蛋白中砷的结合力及其与肝肾功能的关系。
IF 4.3 3区 医学
Expert Review of Clinical Pharmacology Pub Date : 2024-11-01 Epub Date: 2024-10-18 DOI: 10.1080/17512433.2024.2417666
Chunlu Gao, Rui Duan, Shuo Tian, Chunrong Pang, Hong Zhang, Haixia Yang, Xin Hai
{"title":"Plasma protein binding of arsenic species in acute promyelocytic leukemia patients and their relationships with hepatic and renal function.","authors":"Chunlu Gao, Rui Duan, Shuo Tian, Chunrong Pang, Hong Zhang, Haixia Yang, Xin Hai","doi":"10.1080/17512433.2024.2417666","DOIUrl":"10.1080/17512433.2024.2417666","url":null,"abstract":"<p><strong>Objectives: </strong>Percentage protein binding (%PB) of arsenic species in acute promyelocytic leukemia (APL) patients treated with arsenic trioxide remains unclear. It can be different depending on the status of hepatic or renal function.</p><p><strong>Methods: </strong>This study obtained steady-state blood samples from normal APL patients and those with hepatic or renal impairment. %PB of inorganic arsenic (iAs), monomethylarsonic acid (MMA<sup>V</sup>), and dimethylarsinic acid (DMA<sup>V</sup>) was determined by analyzing free and total plasma concentrations using ultrafiltration method by HPLC-HG-AFS.</p><p><strong>Results: </strong>There is a linear relationship between free and total plasma concentrations for iAs (r<sup>2</sup> = 0.952), MMA<sup>V</sup> (r<sup>2</sup> = 0.603), and DMA<sup>V</sup> (r<sup>2</sup> = 0.945). For patients with normal hepatic and renal function, mean %PB was as follows: iAs at 26.7 ± 14.3%, MMA<sup>V</sup> at 53.3 ± 11.9%, and DMA<sup>V</sup> at 24.7 ± 7.8%. %PB decreased in patients with renal impairment, with MMA<sup>V</sup> and DMA<sup>V</sup> showing statistically significant differences (<i>p</i> < 0.05 for MMA<sup>V</sup>, <i>p</i> < 0.01 for DMA<sup>V</sup>). No significant differences in %PB between normal and hepatic impairment group were observed.</p><p><strong>Conclusion: </strong>Free arsenic species fraction can be estimated by total concentration. DMA<sup>V</sup> and iAs present low %PB, while MMA<sup>V</sup> exhibits a relatively high %PB in plasma. Level of %PB is more likely to be affected by renal function and age.</p>","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":" ","pages":"1063-1069"},"PeriodicalIF":4.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461437","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
SGLT2i and GLP1RA effects in patients followed in a hospital diabetology consultation. SGLT2i 和 GLP1RA 对医院糖尿病咨询随访患者的影响。
IF 4.3 3区 医学
Expert Review of Clinical Pharmacology Pub Date : 2024-11-01 Epub Date: 2024-10-27 DOI: 10.1080/17512433.2024.2421872
António Cabral Lopes, Olga Lourenço, Manuel Morgado
{"title":"SGLT2i and GLP1RA effects in patients followed in a hospital diabetology consultation.","authors":"António Cabral Lopes, Olga Lourenço, Manuel Morgado","doi":"10.1080/17512433.2024.2421872","DOIUrl":"10.1080/17512433.2024.2421872","url":null,"abstract":"<p><strong>Background: </strong>We aimed to investigate the effects of sodium-glucose cotransporter 2 inhibitors (SGLT2i) and glucagon-like peptide-1 receptor agonists (GLP1RA) in patients with type 2 diabetes mellitus (T2DM) in clinical practice.</p><p><strong>Research design and methods: </strong>A total of 340 patients were included. Data on age, gender, antidiabetic medications, and bioanalytical parameters were collected at baseline and one year later. Were analyzed estimated glomerular filtration rate (eGFR), blood sodium and potassium levels, blood pressure, weight, cardiovascular risk, and glycated hemoglobin (HbA1c).</p><p><strong>Results: </strong>Patients treated with SGLT2i exhibited a significant improvement in eGFR at the endpoint compared to baseline (<i>p</i> = 0.006). Both treatment groups experienced reductions in systolic blood pressure at the endpoint; especially patients treated with SGLT2i (<i>p</i> = 0.0002). GLP1RA treatment resulted in a statistically significant weight reduction from baseline to endpoint (<i>p</i> < 0.0001), with a higher percentage of patients achieving ≥ 5% weight loss compared to the non-GLP1RA group (33.6% vs. 19.8%). Both SGLT2i and GLP1RA treatments significantly reduced cardiovascular risk scores (<i>p</i> = 0.004 and <i>p</i> = 0.002, respectively). Additionally, both treatments were associated with a significant reduction in HbA1c levels at the endpoint (<i>p</i> = 0.010 and <i>p</i> = 0.002, respectively).</p><p><strong>Conclusions: </strong>Our findings suggest that SGLT2i and GLP1RA offer beneficial effects in patients with T2DM.</p>","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":" ","pages":"1081-1088"},"PeriodicalIF":4.3,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142497733","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
Type II RAF inhibitor tovorafenib for the treatment of pediatric low-grade glioma. 治疗小儿低级别胶质瘤的 II 型 RAF 抑制剂托伐非尼。
IF 3.6 3区 医学
Expert Review of Clinical Pharmacology Pub Date : 2024-11-01 Epub Date: 2024-10-19 DOI: 10.1080/17512433.2024.2418405
Tianqiao Zhang, Bo Xu, Fan Tang, Zunbo He, Jiecan Zhou
{"title":"Type II RAF inhibitor tovorafenib for the treatment of pediatric low-grade glioma.","authors":"Tianqiao Zhang, Bo Xu, Fan Tang, Zunbo He, Jiecan Zhou","doi":"10.1080/17512433.2024.2418405","DOIUrl":"10.1080/17512433.2024.2418405","url":null,"abstract":"<p><strong>Introduction: </strong>Pediatric low-grade glioma (pLGG) is the most prevalent childhood brain tumor group, currently regarded as a chronic disease. On 23 April 2024, the U.S. FDA approved a new type II RAF inhibitor, tovorafenib (OJEMDA<sup>TM</sup>), previously known as DAY101, for the treatment of patients aged 6 months and older with relapsed or refractory (R/R) pLGG harboring a BRAF fusion or rearrangement, or BRAF V600E mutation.</p><p><strong>Areas covered: </strong>This article aims to review the pharmacological properties of tovorafenib and evaluate its efficacy and safety in the treatment of R/R pLGG. We conducted a systematic search of PubMed and Web of Science databases for English-language publications related to tovorafenib, including journal articles and conference abstracts, up through 20 August 2024.</p><p><strong>Expert opinion: </strong>As the first and only FDA-approved medicine for children with BRAF fusions or rearrangements, which are the most common molecular alteration in pLGG, tovorafenib shows superior central nervous system penetration without the paradoxical activation of the MAPK pathway reported for type I BRAF inhibitors. Phase 1 and the pivotal phase 2 trials have demonstrated that tovorafenib monotherapy is generally well-tolerated and exhibits encouraging signs of meaningful, rapid and sustained clinical activity in children and young adults with BRAF-altered pLGG.</p>","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":" ","pages":"999-1008"},"PeriodicalIF":3.6,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461440","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
An overview on disease modifying and symptomatic drug treatments for multiple sclerosis. 多发性硬化症的改病和对症药物治疗概述。
IF 3.6 3区 医学
Expert Review of Clinical Pharmacology Pub Date : 2024-10-08 DOI: 10.1080/17512433.2024.2410393
Gloria Dalla Costa, Letizia Leocani, Mariaemma Rodegher, Luca Chiveri, Alessandro Gradassi, Giancarlo Comi
{"title":"An overview on disease modifying and symptomatic drug treatments for multiple sclerosis.","authors":"Gloria Dalla Costa, Letizia Leocani, Mariaemma Rodegher, Luca Chiveri, Alessandro Gradassi, Giancarlo Comi","doi":"10.1080/17512433.2024.2410393","DOIUrl":"https://doi.org/10.1080/17512433.2024.2410393","url":null,"abstract":"<p><strong>Introduction: </strong>Multiple sclerosis (MS) is an inflammatory and degenerative autoimmune condition, resulting frequently in a disabling condition. Significant improvements of long-term prognosis have been recently achieved with an early and more aggressive use of disease modifying therapies (DMTs). Addressing the complexity of managing its progressive forms remains a significant challenge.</p><p><strong>Areas covered: </strong>This review provides an update on DMTs for relapsing-remitting MS (RRMS) and progressive MS and their efficacy, safety, and mechanism of action, emphasizing the critical role of biomarkers in optimizing treatment decisions. Moreover, some key information on drugs used to manage symptoms such as pain, fatigue, spasticity and urinary problems will be provided. The literature search was conducted using PubMed, Embase, and Cochrane Library databases covering the period from January 2000 to January 2024.</p><p><strong>Expert opinion: </strong>Major advances have been achieved in the treatment of RRMS. Treatment should start immediately as soon as the neurologist is confident with the diagnosis and its choice should be based on the prognostic profile and on the patient's propensity to accept drug-related risks. The therapeutic landscape for progressive MS is quite disappointing and necessitates further innovation. Personalized medicine, leveraging biomarker insights, holds promise for refining treatment efficacy and patient outcomes.</p>","PeriodicalId":12207,"journal":{"name":"Expert Review of Clinical Pharmacology","volume":" ","pages":"1-21"},"PeriodicalIF":3.6,"publicationDate":"2024-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142389219","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
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