{"title":"Cutting-edge pharmacotherapy for hepatitis C virus infection: a comprehensive review.","authors":"Chen-Hua Liu, Yu-Ping Chang, Jia-Horng Kao","doi":"10.1080/14656566.2024.2396024","DOIUrl":"10.1080/14656566.2024.2396024","url":null,"abstract":"<p><strong>Introduction: </strong>Pharmacotherapy against hepatitis C virus (HCV) infection has tremendously improved since the advent of interferon (IFN)-free direct-acting antivirals (DAAs). Additionally, fixed-dose pangenotypic DAAs, which are safe, potent, easy for use, and can cover a wide spectrum of patients, have been recommended by professional guidelines for DAA-naïve and DAA-experienced patients with HCV.</p><p><strong>Areas covered: </strong>We review the pharmacokinetics, pharmacodynamics, and potential drug-drug interactions (DDIs) of fixed-dose pangenotypic DAA regimens, including glecaprevir/pibrentasvir (GLE/PIB), sofosbuvir/velpatasvir (SOF/VEL), and sofosbuvir/velpatasvir/voxilaprevir (SOF/VEL/VOX). Additionally, we summarize the efficacy and safety of these regimens in clinical trials as well as real-world studies for treating different populations. Lastly, we discuss unmet medical needs in managing HCV in the era of fixed-dose pangenotypic DAAs.</p><p><strong>Expert opinion: </strong>Protease inhibitors (PIs), including GLE and VOX, are prone to have more frequent DDIs, compared to the non-structural (NS) 5A and 5B inhibitors. These regimens are generally well tolerated and can be applied to different populations, except for the contraindicated use of PI-containing DAA regimens in decompensated cirrhosis. Using the first-line GLE/PIB and SOF/VEL can eradicate HCV in more than 95% of DAA-naïve patients across different populations. The viral cure usually exceeds 95% when using the rescue SOF/VEL/VOX regimen for prior DAA failures.</p>","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1691-1706"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142016824","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}
Onur Baser, Katarzyna Rodchenko, Elizabeth Vivier, Isabel Baser, Yuanqing Lu, Munira Mohamed
{"title":"The impact of approved anti-obesity medications on osteoarthritis.","authors":"Onur Baser, Katarzyna Rodchenko, Elizabeth Vivier, Isabel Baser, Yuanqing Lu, Munira Mohamed","doi":"10.1080/14656566.2024.2391524","DOIUrl":"10.1080/14656566.2024.2391524","url":null,"abstract":"<p><strong>Background: </strong>Obesity has been established as a significant risk factor for osteoarthritis. Anti-obesity medications (AOMs) have demonstrated efficacy in weight management. However, potential impact on osteoarthritis risk remains uncertain.</p><p><strong>Methods: </strong>This retrospective cohort study used Kythera data from NOV2022 to JULY2024. Patients with obesity using AOMs were identified through diagnosis and prescription claims for tirzepatide, semaglutide, or liraglutide between 1NOV2023 and 31JAN2024, with a 6-month follow-up to assess OA risk. OA risk, analyzed using Cox regression and propensity score matching, controlled for comorbidities and sociodemographic factors.</p><p><strong>Results: </strong>There were 39,394 patients living with obesity using AOM (23,933 semaglutide 12,854 tirzepatide, 2,607 liraglutide) and 72,405 without AOM use. The adjusted osteoarthritis risk was 27% % lower in AOM users than in non-users (hazard ratio (HR) = 073, 95% CI (0.67-0.79), <i>p</i> < 0.01). Among AOMs, tirzepatide was associated with a significantly lower osteoarthritis (OA) risk compared to semaglutide (HR = 0.57, 95% CI: 0.50-0.65, <i>p</i> < 0.0001). Liraglutide was linked to a significantly higher OA risk vs tirzepatide (HR = 1.63, 95% CI: 1.23-2.15, <i>p</i> = 0.0007).</p><p><strong>Conclusions: </strong>AOM use was associated with a significantly lower risk of OA and may be an effective obesity management intervention.</p>","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1565-1573"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141916490","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}
{"title":"Current evidence for janus kinase inhibitors in adult and juvenile dermatomyositis and key comparisons.","authors":"Rachel S Wallwork, Julie J Paik, Hanna Kim","doi":"10.1080/14656566.2024.2392021","DOIUrl":"10.1080/14656566.2024.2392021","url":null,"abstract":"<p><strong>Introduction: </strong>Adult dermatomyositis (DM) and juvenile dermatomyositis (JDM) are rare autoimmune diseases with characteristic skin rashes, weakness, and other systemic features. Upregulated interferon signaling has been consistently described in both adult and juvenile DM which makes janus kinase inhibitors (jakinibs) an attractive therapeutic agent that has a targeted mechanism of action.</p><p><strong>Areas covered: </strong>Herein is a review of the growing literature of jakinib use in adult and juvenile DM, including reports on specific disease features and safety of jakinibs in this population and a comparison between adult and juvenile DM. We performed a literature review using PubMed including all English-language publications before 1 February 2024 and abstracts from key recent rheumatology conferences.</p><p><strong>Expert opinion: </strong>Jakinibs are an exciting and promising treatment in both adult and juvenile DM. Current Phase 2 and 3 randomized placebo-controlled trials of jakinibs in both adult and JDM will provide significant insights into the efficacy of this class of medication as a potentially more mechanistically targeted treatment of both skin and muscle disease. In fact, these results will likely inform the treatment paradigm of dermatomyositis in that it may even be considered as first or second line. The next five years in the therapeutic landscape of both juvenile and adult DM is an exciting time for both patients and medical providers.</p>","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1625-1645"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141970928","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}
{"title":"Factor VIII stimulants and other novel therapies for the treatment of von Willebrand disease: what's new on the horizon?","authors":"Katherine Regling, Robert F Sidonio","doi":"10.1080/14656566.2024.2391526","DOIUrl":"10.1080/14656566.2024.2391526","url":null,"abstract":"<p><strong>Introduction: </strong>Von Willebrand disease (VWD) is the most common inherited bleeding disorder, affecting about 0.6% to 1.3% of the population, and is characterized primarily by mucocutaneous bleeding secondary to defective platelet adhesion and aggregation. Current therapeutic options for those with severe disease are limited and require frequent intravenous infusions.</p><p><strong>Areas covered: </strong>This review discusses the current and recently completed clinical trials involving pathways to FVIII augmentation for the treatment of VWD. Clinical trials registered on clinicaltrials.gov and published data via PubMed searches through June 2024 were included.</p><p><strong>Expert opinion: </strong>Available treatment options to those with VWD are limited in part due to limited clinical trials, the complexity of VWD types, and the pharmacokinetics of current treatment options. The development of therapeutic options that reduce treatment burden is necessary to improve quality of life and reduce bleeding complications and in recent years there has been an increased interest from industry to apply novel therapeutics for VWD. The FVIII mimetic, emicizumab, has demonstrated early success in patients with severe VWD and is a promising treatment option for those who require prophylaxis. Furthermore, products like efanesoctocog alfa (Altuviiio®) and BT200 have achieved enhanced VWF/FVIII half-life extension could expand the current treatment landscape while concurrently minimizing treatment burden.</p>","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1427-1438"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141999665","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}
{"title":"The potential of Bruton's tyrosine kinase (BTK) inhibitors in the pharmacotherapeutic management of immune and dermatological disease.","authors":"Henry Tseng, Dédée F Murrell","doi":"10.1080/14656566.2024.2393280","DOIUrl":"10.1080/14656566.2024.2393280","url":null,"abstract":"<p><strong>Introduction: </strong>The review article explores the evolving role of Bruton's tyrosine kinase (BTK) inhibitors in immune-mediated dermatological conditions, addressing significant gaps in current treatment approaches.</p><p><strong>Areas covered: </strong>The review comprehensively discusses the mechanisms of action of BTK inhibitors, including irreversible and reversible inhibitors. Clinical applications of BTK inhibitors in dermatological diseases such as pemphigus, chronic spontaneous urticaria (CSU), hidradenitis suppurativa (HS), systemic lupus erythematosus (SLE), and atopic dermatitis are explored, highlighting recent advancements and ongoing clinical trials. Potential advantages of BTK inhibitors over existing therapies and challenges in translating preclinical findings to clinical outcomes are discussed.</p><p><strong>Expert opinion/commentary: </strong>BTK inhibitors represent a promising therapeutic avenue for immune-mediated dermatological conditions, offering oral administration, targeted pathway inhibition, and a favorable safety profile compared to biologic therapies. Ongoing research and clinical trials hold the potential to address unmet needs and reshape the therapeutic landscape in dermatology.</p>","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1657-1665"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141999666","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}
{"title":"Pharmacotherapy for CD55 deficiency with CHAPLE disease: how close are we to a cure?","authors":"Salim Can, Melek Yorgun Altunbas, Ahmet Ozen","doi":"10.1080/14656566.2024.2388267","DOIUrl":"10.1080/14656566.2024.2388267","url":null,"abstract":"","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1421-1426"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141874498","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}
{"title":"Evaluating the safety and efficacy of seladelpar for adults with primary biliary cholangitis.","authors":"Allyce Caines, Sheri Trudeau, Stuart C Gordon","doi":"10.1080/14656566.2024.2390120","DOIUrl":"10.1080/14656566.2024.2390120","url":null,"abstract":"<p><strong>Introduction: </strong>Seladelpar (MBX-8025) is a once-daily administered highly specific PPAR-δ agonist in Phase 3 and extension trials for use in patients with primary biliary cholangitis (PBC).</p><p><strong>Areas covered: </strong>This review provides background on current treatment options for PBC, and summarizes clinical trial data regarding the safety and effectiveness of seladelpar within the context of these treatments.</p><p><strong>Expert opinion: </strong>Clinical trials results demonstrate the safety and tolerability of seladelpar use for PBC, including in patients with cirrhosis. The primary composite endpoint (ALP <1.67 times ULN, decrease ≥ 15% from baseline, and TB ≤ULN) was met in 61.7% of the patients treated with seladelpar and in 20% receiving placebo (<i>p</i> < 0.001). Moreover, pruritus - a cardinal and often intractable symptom of PBC - was improved with seladelpar treatment, as were overall quality of life measurements. Improvements in markers of inflammation were likewise observed. These biochemical and clinical findings therefore represent landmark developments in PBC treatment and offer a therapeutic option for PBC.</p>","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1517-1523"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897176","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}
{"title":"Ospemifene and vulvovaginal atrophy: an update of the clinical profile for post-menopausal women.","authors":"Costantino Di Carlo, Angelo Cagnacci, Filippo Murina, Silvia Maffei, Angelamaria Becorpi, Stefano Lello","doi":"10.1080/14656566.2024.2391009","DOIUrl":"10.1080/14656566.2024.2391009","url":null,"abstract":"<p><strong>Introduction: </strong>The demand for effective and safe treatments of genitourinary syndrome (GSM) in post-menopausal women (PMW) is growing. Published data on the efficacy and safety of ospemifene (OSP) prompt an updated literature review to enlighten possible improvements in the GSM treatment.</p><p><strong>Area covered: </strong>We searched articles published in English from 2010 to 2023 through Medline (PubMed) and Embase databases with Boolean terms: OSP, PMW, GSM, endometrium, breast cancer, cardiometabolic syndrome, bone metabolism, adherence to treatment, and patient satisfaction. We selected randomized controlled trials (RCTs) and observational and cross-sectional studies and completed the search manually.</p><p><strong>Expert opinion: </strong>Of the 157 retrieved records, 25 primary studies met the inclusion criteria (15 regarding efficacy and safety, two for additional effects, and four for adherence and satisfaction with the OSP treatment). Seven RCTs involved nearly 5,000 patients, 10 out of 18 prospective observational studies 563, and six retrospective analyses 356,439. Evidence of OSP treatment in PMW with GSM relies on RCTs and remarkable real-world data. The 25 primary studies showcased the high clinical response to symptoms, the favorable safety profile of OSP with very few adverse events, a neutral impact on the endometrium, breast, bone, and thrombosis, and the possible improvement of cardiovascular risk factors.</p>","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1541-1554"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141916489","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}
Magalie Tardif, Nida Usmani, Maja Krajinovic, Henrique Bittencourt
{"title":"Cytokine release syndrome after CAR T-cell therapy for B-cell acute lymphoblastic leukemia in children and young adolescents: storms make trees take deeper roots.","authors":"Magalie Tardif, Nida Usmani, Maja Krajinovic, Henrique Bittencourt","doi":"10.1080/14656566.2024.2387673","DOIUrl":"10.1080/14656566.2024.2387673","url":null,"abstract":"<p><strong>Introduction: </strong>Chimeric antigen receptor (CAR) T-cells have revolutionized cancer treatment, showing significant success, including treatment of pediatric B-cell acute lymphoblastic leukemia (B-ALL). Despite their efficacy, cytokine release syndrome (CRS) emerges as a common early adverse effect that can be life threatening in severe cases, resulting from the immune system's targeted activation against tumors.</p><p><strong>Areas covered: </strong>This review concentrates on CRS in children and young adults undergoing CAR T-cell therapy for B-ALL. It explores CRS pathophysiology, clinical presentation, and incidence, emphasizing the importance of a consensus definition and grading to homogenize the treatment according to the severity of symptoms. We will discuss the standard of care treatment of CRS but also novel approaches. We will highlight the importance of managing CRS without compromising the efficacy of immune cell activation against tumors.</p><p><strong>Expert opinion: </strong>As CAR T-cell therapy in pediatric B-ALL become increasingly available and used, optimal management of CRS becomes increasingly important. Early recognition and timely management has improved. Further information will aid us to identify optimal timing of tocilizumab and corticosteroids. Continued bench research coupled with clinical studies and biomarker discovery will allow for valuable insights into CRS pathophysiology and patient and/or cell-targeted treatments.</p>","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1497-1506"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141859465","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}
{"title":"An update on the use of 'biotics' in pediatric infectious gastroenteritis.","authors":"Alicia Wampers, Koen Huysentruyt, Yvan Vandenplas","doi":"10.1080/14656566.2024.2387672","DOIUrl":"10.1080/14656566.2024.2387672","url":null,"abstract":"<p><strong>Introduction: </strong>Acute gastroenteritis (AGE) is the consequence of a disturbed gastro-intestinal microbiome. Certain probiotic strains (<i>Lacticaseibacillus rhamnosus</i>, <i>Saccharomyces boulardii</i> CNCM I-745, <i>Limosilactobacillus reuteri (L. reuteri</i>) DSM 17,938, the combination of <i>L. rhamnosus</i> 19070-2 and <i>L. reuter</i>i DSM 12,246) reduce the duration and severity of diarrhea.</p><p><strong>Areas covered: </strong>Relevant literature was sourced from PubMed and CINAHL. Important reviews until 2021 were summarized in tables. New evidence for pro-, pre-, syn- and postbiotics in AGE was searched for. Postbiotics offer advantages regarding product stability and show accumulating evidence. Heterogeneity in studies regarding the in- and exclusion criteria, primary and secondary endpoints, type, dose, timing and duration of biotic administration limits the evidence.</p><p><strong>Expert opinion: </strong>Development of a core outcome set for children with AGE would be beneficial, as its application would increase the homogeneity of the available evidence. The vast majority of the 'biotics' is registered as food supplement. Regulations for food supplements prioritize safety over efficacy, making them considerably more tolerant compared to the regulation for registration as medication. We recommend that at least one randomized controlled trial is published with the commercialized product before marketing the product, despite the fact that legislation regarding food supplements requires only safety data.</p>","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":"1483-1496"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141874497","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}