The Utility of Tissue-Agnostic Drugs in Lung Cancer Treatment.

IF 4.5 Q2 PHARMACOLOGY & PHARMACY

Pharmaceutical Medicine Pub Date : 2025-10-01 DOI:10.1007/s40290-025-00582-x

Leping Kong, Ben Grobman, Arian Mansur, Adele Lee, Connor Bondarchuk, Hannah Erdogan, Christine Y Lu

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Abstract

Tissue-agnostic therapies, which target molecular alterations across cancer types, have been approved by the US Food and Drug Administration (FDA) since 2017. These approvals were based on clinical trials enrolling patients across diverse tumor types, regardless of the tumor's site of origin. This analysis evaluated the efficacy and safety of FDA-approved tissue-agnostic therapies in patients with lung cancer, the leading cause of cancer-related deaths worldwide, harboring targetable genetic alterations. Current evidence suggests that these therapies show promise in treating NTRK gene fusions (larotrectinib, entrectinib, repotrectinib), BRAF V600E mutation (dabrafenib and trametinib), and RET fusions (selpercatinib) in lung cancer. Reported rates of grade 3 or higher treatment-related or treatment-emergent adverse events ranged from 10 to 59.7%. However, available data are limited by small sample sizes (ranging from 4 to 247 participants per trial-cohort) and the absence of control groups. Larger, controlled studies and real-world evaluations are needed to confirm these findings and inform broader clinical use.

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组织不可知药物在肺癌治疗中的应用。

自2017年以来，针对不同癌症类型的分子改变的组织不可知疗法已获得美国食品和药物管理局（FDA）的批准。这些批准是基于临床试验，招募了不同肿瘤类型的患者，而不管肿瘤的起源部位。该分析评估了fda批准的组织不可知疗法在肺癌患者中的疗效和安全性，肺癌是全球癌症相关死亡的主要原因，含有可靶向的基因改变。目前的证据表明，这些疗法在治疗肺癌的NTRK基因融合（larorectinib、entrectinib、repotrectinib）、BRAF V600E突变（dabrafenib和trametinib）和RET融合（selpercatinib）方面显示出希望。报告的3级或以上治疗相关不良事件或治疗引起的不良事件发生率从10%到59.7%不等。然而，可获得的数据受到样本量小（每个试验队列4至247名参与者）和缺乏对照组的限制。需要更大规模的对照研究和真实世界的评估来证实这些发现，并为更广泛的临床应用提供信息。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Pharmaceutical Medicine PHARMACOLOGY & PHARMACY-

CiteScore

5.10

自引率

4.00%

发文量

期刊介绍： Pharmaceutical Medicine is a specialist discipline concerned with medical aspects of the discovery, development, evaluation, registration, regulation, monitoring, marketing, distribution and pricing of medicines, drug-device and drug-diagnostic combinations. The Journal disseminates information to support the community of professionals working in these highly inter-related functions. Key areas include translational medicine, clinical trial design, pharmacovigilance, clinical toxicology, drug regulation, clinical pharmacology, biostatistics and pharmacoeconomics. The Journal includes:Overviews of contentious or emerging issues.Comprehensive narrative reviews that provide an authoritative source of information on topical issues.Systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by PRISMA statement.Original research articles reporting the results of well-designed studies with a strong link to wider areas of clinical research.Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pharmaceutical Medicine may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.