Expert Opinion on Pharmacotherapy最新文献

Introduction: Tourette syndrome is a neurodevelopmental disorder characterized by chronic motor and vocal tics. Both tic severity and co-morbid psychiatric disorders can affect patients' health-related quality of life to an extent that warrants active treatment interventions. Different decisional approaches can be implemented to determine the need for pharmacotherapy.

Areas covered: A critical appraisal of the existing literature on the clinical scenarios where pharmacotherapy for Tourette syndrome is deemed necessary has highlighted three indications: (1) physical discomfort - e.g. pain or injury; (2) emotional or social problems - e.g. depression or isolation; or (3) functional interference - e.g. impairment of academic achievements.

Expert opinion: Pharmacotherapy for tic disorders aims to reduce tic severity enough to improve daily functioning and health-related quality of life rather than to eliminate symptoms. Expert consensus emphasizes patient-centered decision-making that integrates disease-specific quality of life measures with objective tic severity scales. Medications may be prioritized when tics cause significant impairment, pose medical risks, require rapid control, or coexist with treatable co-morbidities. Current guidelines rely largely on limited trials and expert opinion, with dopamine-modulating agents and alpha-2 agonists being most commonly used. Further research should focus on open questions about real-world effectiveness and generalizability of individual pharmacological agents.

Introduction: Several studies have identified Hypoxia-Inducible Factor 2-alpha (HIF-2α) as a key oncogenic driver in clear cell renal cell carcinoma (ccRCC). In this context, belzutifan emerges as an oral inhibitor of HIF-2α, that acts by blocking the heterodimerization of HIF-2α with HIF-1β, thereby preventing the transcription of hypoxia-target genes and reducing VEGF-mediated tumor growth. The LITESPARK series of studies recently defined the emerging role of belzutifan in the therapeutic landscape of RCC, and further studies are ongoing.

Areas covered: In the current review, we discuss the role of belzutifan as an innovative and promising therapeutic strategy for targeting a key biological mechanism in ccRCC.

Expert opinion: According to available evidence and data, belzutifan has generally been well tolerated, though anemia is a frequent on-target side effect and, along with hypoxia, necessitates monitoring throughout treatment. As reported, ongoing phase III clinical trials are set to yield potentially practice-changing results, investigating innovative combinatorial strategies including belzutifan. Ongoing advancement of predictive biomarkers and understanding of resistance mechanisms could improve patient selection and identify new drug targets, thereby increasing the clinical efficacy of belzutifan.

Introduction: Antimalarial agents, particularly hydroxychloroquine and chloroquine, are widely used in systemic autoimmune diseases. While ocular toxicity is well established, the extent and clinical relevance of potential auditory toxicity remain unclear. We conducted a systematic review to evaluate the available evidence on auditory adverse effects associated with antimalarial use in systemic autoimmune diseases.

Methods: A systematic search was performed in PubMed, Scopus, and the Cochrane Library from inception to January 2025. Observational and interventional studies reporting audiological outcomes in patients exposed to antimalarials were included, excluding malaria-related treatments. Risk of bias was assessed according to study design. Due to substantial clinical and methodological heterogeneity, results were synthesized descriptively without meta-analysis.

Results: Sixteen studies encompassing heterogeneous autoimmune populations and audiological methods were included. The reported prevalence of hearing abnormalities varied widely across studies and diagnostic techniques. Associations with antimalarial exposure, dose, or duration were inconsistent. Some studies described subclinical cochlear or brainstem alterations, while others found no significant differences compared with non-exposed patients.

Conclusions: Available evidence is limited and heterogeneous, precluding causal inferences regarding antimalarial-related auditory toxicity. Increased clinical awareness of auditory symptoms may be reasonable, but standardized monitoring is not supported. Prospective controlled studies with standardized audiological assessments are needed.

Introduction: Tenosynovial giant cell tumors (TGCTs) are locally aggressive mesenchymal neoplasms that can cause chronic disability. Surgery is a mainstay of treatment but can carry a high risk of morbidity or can confer limited benefit. As such, effective alternative treatment options are needed, including systemic agents. Improved understanding of the pathophysiology of TGCTs has led to such drug development.

Areas covered: Vimseltinib is a switch-control tyrosine kinase inhibitor designed to selectively inhibit CSF1R, which, along with CSF1, is responsible for the synovial inflammation that characterizes TGCTs. In this drug evaluation, we review the mechanism of action, pharmacologic properties, clinical efficacy and current role within the TGCT treatment landscape of vimseltinib.

Expert opinion: Vimseltinib demonstrated a significant response rate as well as meaningful symptomatic improvement in patients with TGCTs. Furthermore, there is an absence of severe toxicities that have arisen with other agents in the TGCT treatment space, specifically liver failure. Vimseltinib is a favorable option for patients with TGCTs and further efforts to determine its place in the sequence of overall management are needed.

Introduction: Despite the fact that IgA nephropathy (IgAN) is the most common chronic glomerulonephritis across the age spectrum worldwide, there is a lack of evidence-based treatment recommendations for pediatric patients. Moreover, current treatment options in children are very limited. This review aims to provide a comprehensive overview of current therapeutic strategies for managing IgAN, with a particular focus on new drugs recently evaluated in adult cohorts, as well as novel treatments reported in pediatric case studies.

Areas covered: A literature search was conducted using Pubmed and Scopus, covering the period from January 2013 to June 2025. Studies relevant to topic were included. Currently, there are numerous adult trials studying medications that target fundamental IgAN pathogenetic pathways, with very promising results in terms of proteinuria reduction and stabilization of kidney function.

Expert opinion: The higher disease activity and longer life expectancy in pediatric patients create a critical need for the evaluation of modern therapies in children with IgAN. Thus, it is crucial to prioritize pediatric clinical trials and to focus on removing the barriers that limit their implementation.

Introduction: Functional dyspepsia (FD) is a highly prevalent disorder of gut - brain interaction, characterized by heterogeneous symptoms, overlapping pathophysiology, and limited treatment efficacy. Iberogast®, a multi-herbal phytomedicine available for more than five decades, has emerged as a promising therapeutic option by addressing multiple mechanisms simultaneously.

Areas covered: This narrative review summarizes the pharmacological rationale, mechanisms of action, and clinical evidence supporting Iberogast's role in FD and related conditions. Literature was identified through searches of PubMed, Scopus, and Web of Science, complemented by reference screening, with the last update on 19 August 2025. Particular attention is given to the original nine-component formulation (STW 5) and the refined six-component version (STW 5-II). Evidence from randomized controlled trials, meta-analyses, mechanistic studies, and real-world data is synthesized to provide an integrated appraisal of efficacy, safety, and therapeutic positioning.

Expert opinion: Iberogast remains an evidence-based phytomedicine with a favorable safety profile, filling therapeutic gaps in FD by targeting motility, hypersensitivity, inflammation, and barrier function. Future research should include trials comparing Iberogast with established drugs, evaluating cyclic therapy in real-world settings, and exploring new indications such as irritable bowel syndrome, inflammatory bowel disease, use in pediatrics, and overlapping dyspepsia - reflux phenotypes.

Introduction: This review provides an updated overview of the evolving pharmacologic landscape of human epidermal growth factor receptor 2 (HER2)-targeted therapies in breast cancer, highlighting key clinical trial data, recent indication expansions, resistance mechanisms, and emerging therapeutic agents in development.

Areas covered: This review draws on a comprehensive literature search of published studies and major oncology conference proceedings, focusing primarily on data from key phase III clinical trials of HER2-targeted therapies. Studies published within the past decade that have influenced current standards of care were prioritized.

Expert opinion: The treatment paradigm for HER2-expressing breast cancer is evolving rapidly. Trastuzumab deruxtecan has redefined management for both HER2-positive and the newly recognized HER2-low subgroups, while tucatinib offers a critical systemic option for central nervous system metastases. Optimal sequencing and resistance management remain critical challenges in this dynamic field. Nonetheless, HER2-targeted pharmacotherapy continues to advance rapidly, driven by innovative drug designs and strategic clinical developments.

Introduction: Endometriosis is a chronic inflammatory condition affecting ~10% of reproductive-age individuals and contributing significantly to infertility, pain, and reduced quality of life. Since our 2020 review, new pharmacologic strategies, updated guidelines, and advances in clinical trial evidence have reshaped the therapeutic landscape. Effective, patient-centered management is essential to lessen the burden of disease.

Areas covered: This review synthesizes current evidence-based pharmacotherapy for endometriosis, integrating 2022 European Society of Human Reproduction and Embryology recommendations and including a literature review of PubMed, with an emphasis on articles published after 2020. First-line therapies, including NSAIDs, combined oral contraceptives, and progestins such as dienogest, remain central, while GnRH agonists/antagonists and aromatase inhibitors are considered in refractory cases. Recent data highlight add-back therapy to reduce hypoestrogenic side effects. We also review postoperative regimens, fertility-preserving strategies, management in post-hysterectomy and postmenopausal populations, and therapies under investigation - including anti-inflammatory, antifibrotic, angiogenesis-modulating, and microbiome-targeting approaches.

Expert opinion: Hormonal suppression remains the cornerstone of treatment, but novel nonhormonal strategies and advances in precision medicine hold promise for more durable and individualized care. Ongoing clinical trials, artificial intelligence - assisted diagnostics, and fertility-focused pharmacotherapies represent exciting frontiers. Multimodal, patient-tailored approaches will be key to optimizing long-term outcomes in endometriosis management.