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Should Metastatic Site Inform First-Line Therapy in Clear Cell Renal Cell Carcinoma? 透明细胞肾细胞癌的一线治疗是否需要转移部位?
IF 5.1 3区 医学
Targeted Oncology Pub Date : 2026-07-01 Epub Date: 2026-05-31 DOI: 10.1007/s11523-026-01228-x
Michael A Liu, Kyle Miyazaki, Karie Runcie, Hanna Cash, Lingbin Meng
{"title":"Should Metastatic Site Inform First-Line Therapy in Clear Cell Renal Cell Carcinoma?","authors":"Michael A Liu, Kyle Miyazaki, Karie Runcie, Hanna Cash, Lingbin Meng","doi":"10.1007/s11523-026-01228-x","DOIUrl":"10.1007/s11523-026-01228-x","url":null,"abstract":"<p><p>Frontline systemic therapy for metastatic clear cell renal cell carcinoma (RCC) has evolved to include doublet immunotherapy (IO/IO) and immunotherapy-tyrosine kinase inhibitor combinations (IO/TKI), with selection typically guided by histology and risk stratification. However, optimal regimen selection remains undefined owing to limited head-to-head data. Subanalyses of landmark clinical trials and emerging real-world multicenter study data suggest differential treatment benefit derived by metastatic site, particularly for bone metastases. Evidence suggests that bone metastases promote an immunosuppressive tumor microenvironment and may preferentially upregulate genes encoding angiogenic factors, compared with other sites. Such biology-driven patterns provide a rationale for the hypothesis that IO/TKI may confer greater activity in bone-predominant disease. Liver metastases may also exhibit similar site-specific patterns due to harboring an immunosuppressive and vascular environment, but RCC-specific data remain scarce. This overview summarizes the mechanistic rationale, clinical evidence, and practical implications of tailoring treatment by disease site, highlighting critical gaps as well as future research priorities.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":"541-547"},"PeriodicalIF":5.1,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13427791/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148139013","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Real-World Treatment Patterns in Advanced Cutaneous Melanoma in the USA. 美国晚期皮肤黑色素瘤的现实世界治疗模式
IF 5.1 3区 医学
Targeted Oncology Pub Date : 2026-07-01 Epub Date: 2026-07-15 DOI: 10.1007/s11523-026-01235-y
Matthew S Block, Ashley E Stenzel, Rene Marcotte, Katrine L Wallace
{"title":"Real-World Treatment Patterns in Advanced Cutaneous Melanoma in the USA.","authors":"Matthew S Block, Ashley E Stenzel, Rene Marcotte, Katrine L Wallace","doi":"10.1007/s11523-026-01235-y","DOIUrl":"10.1007/s11523-026-01235-y","url":null,"abstract":"<p><strong>Background: </strong>Melanoma has an excellent prognosis when detected at a localized stage, but when patients present with regional or distant metastases, 5-year survival decreases to approximately 76% and 35%, respectively. Although recent advances in systemic therapies have improved outcomes for patients with advanced melanoma, these treatments are often not curative, and many patients ultimately develop disease progression requiring subsequent lines of therapy.</p><p><strong>Objective: </strong>This study aimed to characterize real-world treatment patterns among patients in the USA with advanced cutaneous melanoma who transitioned from first-line (1L) to second-line (2L) systemic therapy.</p><p><strong>Patients and methods: </strong>This retrospective, observational study used administrative claims data from a large US managed care database. Adult patients with metastatic melanoma who initiated 1L systemic therapy between January 2023 and December 2025 and subsequently received 2L therapy were included. Claims-based algorithms identified treatment regimens by line of therapy. Sankey diagrams illustrate treatment transitions overall and by BRAF V600 mutation status.</p><p><strong>Results: </strong>Of 1772 patients who met study criteria, 680 (38.4%) were classified as presumed BRAF V600-mutated (BRAFmut) on the basis of treatment exposure, and 1092 (61.6%) were classified as presumed BRAF wild-type (BRAFwt). Immune checkpoint inhibitor (ICI)-based regimens were the most common first-line (1L) therapies (78.5%), while BRAF + MEK inhibitors accounted for 14.4%. In second-line (2L) therapy, sequencing patterns were diverse, with no dominant 2L pathway observed. BRAF + MEK inhibitors were the most frequently used 2L therapy (27.5%). In patients with BRAFmut, targeted therapy predominated in 2L (71.8%), whereas ICI-based regimens remained dominant in patients with BRAFwt (84.9%). 1L therapy was significantly associated with 2L treatment selection (p < 0.001). Treatment distributions differed across index years for both 1L (p = 0.001) and 2L (p = 0.005) therapies. Over time, use of PD-1 monotherapy decreased, while use of nivolumab plus ipilimumab increased; use of BRAF + MEK therapy remained stable.</p><p><strong>Conclusions: </strong>Although treatment practices for advanced melanoma continue to evolve in response to emerging clinical evidence, substantial uncertainty remains regarding optimal treatment sequencing after 1L progression. These findings highlight the need for prospective sequencing studies and more effective therapeutic options following disease progression.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":"569-580"},"PeriodicalIF":5.1,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13427969/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148456764","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Real-World Treatment Patterns and Clinical Outcomes Among Chinese Patients with Stage III NSCLC: The MOOREA Study. 中国III期非小细胞肺癌患者的实际治疗模式和临床结果:MOOREA研究
IF 5.1 3区 医学
Targeted Oncology Pub Date : 2026-05-01 Epub Date: 2026-05-03 DOI: 10.1007/s11523-026-01217-0
Ligang Xing, Jinming Yu, Xiangjiao Meng, Ren Zhao, Weihua Yang, Yubiao Guo, Juan Li, Min Fan, Chuangying Xiao, Yanping Ren, Lihua Dong, Dongqing Lv, Lujun Zhao, Yingcheng Lin, Xiaotao Zhang, Long Chen, Ye Wang, Yuhui Zhang, Aimin Zang, Gregory Cheng, Anwen Liu, Naixin Liang, Baisong Lou, Jun Wang, Xiaolong Fu, Anhui Shi, Ning Xu, Junfeng Liu, Zuo-Lin Xiang
{"title":"Real-World Treatment Patterns and Clinical Outcomes Among Chinese Patients with Stage III NSCLC: The MOOREA Study.","authors":"Ligang Xing, Jinming Yu, Xiangjiao Meng, Ren Zhao, Weihua Yang, Yubiao Guo, Juan Li, Min Fan, Chuangying Xiao, Yanping Ren, Lihua Dong, Dongqing Lv, Lujun Zhao, Yingcheng Lin, Xiaotao Zhang, Long Chen, Ye Wang, Yuhui Zhang, Aimin Zang, Gregory Cheng, Anwen Liu, Naixin Liang, Baisong Lou, Jun Wang, Xiaolong Fu, Anhui Shi, Ning Xu, Junfeng Liu, Zuo-Lin Xiang","doi":"10.1007/s11523-026-01217-0","DOIUrl":"10.1007/s11523-026-01217-0","url":null,"abstract":"<p><strong>Background: </strong>Stage III non-small cell lung cancer (NSCLC) presents marked heterogeneity under evolving therapeutic paradigms. Real-world evidence on current treatment practices and outcomes remains limited.</p><p><strong>Objective: </strong>The MOOREA study aimed to evaluate real-world molecular testing, treatment patterns, and clinical outcomes of treatment-naïve Chinese patients with stage III NSCLC.</p><p><strong>Patients and methods: </strong>MOOREA is a prospective, multicenter Chinese study enrolling patients with untreated stage III NSCLC (16 July 2019 to 28 February 2022) from 28 hospitals. Patients were consecutively enrolled. The primary endpoint was treatment pattern of cohort 1 (C1; unresectable stage III NSCLC), and the secondary endpoints included molecular testing pattern, progression-free survival (PFS), overall survival (OS) of C1, and treatment pattern of cohort 2 (C2; resectable stage III NSCLC).</p><p><strong>Results: </strong>In total, 486 patients were analyzed (C1: 379; C2: 107). Molecular testing rates were: EGFR (20.0%), ALK (15.0%), and PD-L1 (13.0%). Of the 45.6% (173/379) of individuals in C1 who received chemoradiotherapy (CRT), 53.8% (93/173) underwent consolidation therapy, including 37.6% (35/93) who received immunotherapy (IO). In C2, lobectomy was the main surgical approach (85.8%, 91/106), whereas pneumonectomy was performed on 14.2% of patients (15/106). Adjuvant treatment was planned for 71.4% (75/105) of the patients in C2. For C1, the median follow-up was 27.5 months, with PFS and OS of 12.6 (95% CI: 11.0-14.0) and 33.3 months (95% CI: 29.6-not estimable), respectively. Subgroup analysis showed better OS and PFS for patients receiving CRT with IO consolidation versus CRT only, especially for those who underwent more than six IO consolidation cycles (24-month OS: 79.3% versus 66.4%; PFS: 49.6% versus 24.2%).</p><p><strong>Conclusions: </strong>MOOREA reveals the real-world management of stage III NSCLC in 20 provinces/cities in mainland China and Hong Kong SAR. Patients with unresectable tumors derived significant benefit from radiotherapy and consolidation after CRT. Substantial disparity persists between actual practice and guideline recommendations, necessitating efforts to enhance adherence to guideline-based care.</p><p><strong>Clinical trial registration: </strong>NCT04023812.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":"385-398"},"PeriodicalIF":5.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13222273/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147821161","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Indirect Comparison of the Efficacy of Pembrolizumab Versus Nivolumab as Adjuvant Options for Stage IIB/IIC Melanoma. Pembrolizumab与Nivolumab作为辅助治疗IIB/IIC期黑色素瘤疗效的间接比较
IF 5.1 3区 医学
Targeted Oncology Pub Date : 2026-05-01 Epub Date: 2026-05-08 DOI: 10.1007/s11523-026-01221-4
Dimitrios A Andreikos, Iro Argyropoulou, Charalampos Theocharopoulos, Amalia Anastasopoulou, Dimitra Stefanou, Giorgos Lyrarakis, Dirk Schadendorf, Helen Gogas, Dimitrios C Ziogas
{"title":"Indirect Comparison of the Efficacy of Pembrolizumab Versus Nivolumab as Adjuvant Options for Stage IIB/IIC Melanoma.","authors":"Dimitrios A Andreikos, Iro Argyropoulou, Charalampos Theocharopoulos, Amalia Anastasopoulou, Dimitra Stefanou, Giorgos Lyrarakis, Dirk Schadendorf, Helen Gogas, Dimitrios C Ziogas","doi":"10.1007/s11523-026-01221-4","DOIUrl":"10.1007/s11523-026-01221-4","url":null,"abstract":"<p><strong>Background: </strong>Adjuvant anti-PD-1 therapy improves outcomes in resected stage IIB/IIC melanoma at high risk of recurrence; however, no randomized head-to-head trial has directly compared pembrolizumab with nivolumab.</p><p><strong>Objective: </strong>The objective was to compare the efficacy of pembrolizumab and nivolumab as adjuvant therapies in resected stage IIB/IIC melanoma using an indirect treatment comparison (ITC).</p><p><strong>Patients and methods: </strong>An anchored Bucher ITC was performed linking the results of KEYNOTE-716 and the CheckMate-76K trials. Primary analyses used the most recent follow-up hazard ratios (HRs), with secondary analyses based on the original trial publications.</p><p><strong>Results: </strong>The ITC showed no statistically significant differences between pembrolizumab and nivolumab for recurrence-free survival (RFS; HR 0.97, 95% confidence intervals (CI) 0.69-1.35) or distant metastasis-free survival (DMFS; HR 0.81, 95% CI 0.54-1.21). Subgroup analyses for RFS were also nonsignificant: stage IIB HR 0.97, 95% CI 0.62-1.50; stage IIC HR 1.00, 95% CI 0.61-1.65; age < 65 years HR 1.20, 95% CI 0.76-1.89; and age ≥ 65 years HR 0.77, 95% CI 0.47-1.25. Secondary analyses based on the original trial publications yielded nonsignificant estimates that numerically favored nivolumab, with RFS HR 1.48 (95% CI 0.98-2.23) and DMFS HR 1.26 (95% CI 0.74-2.13).</p><p><strong>Conclusions: </strong>This ITC demonstrated no statistically significant differences between pembrolizumab and nivolumab for RFS or DMFS in resected stage IIB/IIC melanoma, with consistently null findings across subgroup analyses. Longer follow-up attenuated earlier numerical trends, with effect estimates converging toward unity.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":"421-432"},"PeriodicalIF":5.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13222263/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147843230","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
HER2-Mutant Non-Small Cell Lung Cancer Podcast: Knowledge Is Power. her2突变型非小细胞肺癌播客:知识就是力量。
IF 5.1 3区 医学
Targeted Oncology Pub Date : 2026-05-01 Epub Date: 2026-05-26 DOI: 10.1007/s11523-026-01214-3
Vivek Subbiah, Martin Dietrich, Xiuning Le
{"title":"HER2-Mutant Non-Small Cell Lung Cancer Podcast: Knowledge Is Power.","authors":"Vivek Subbiah, Martin Dietrich, Xiuning Le","doi":"10.1007/s11523-026-01214-3","DOIUrl":"10.1007/s11523-026-01214-3","url":null,"abstract":"<p><p>In the first episode of our podcast series, we introduce the emerging landscape of HER2-mutant non-small cell lung cancer (NSCLC), highlighting its place within the broader context of lung cancer. We explain what makes HER2-mutant NSCLC unique, from its epidemiology and biology to the molecular mechanisms driving cancer growth. We discuss the evolution of precision medicine in NSCLC, the role of HER2 as a therapeutic target, and how advances in molecular profiling have enabled the development of targeted therapies. The episode also covers the latest treatment strategies, including antibody-drug conjugates and tyrosine kinase inhibitors, and reviews the challenges and opportunities that define this aggressive cancer subtype. This podcast aims to enhance understanding of HER2-mutant NSCLC and the evolving therapeutic landscape that is reshaping management strategies for this aggressive disease.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":"315-319"},"PeriodicalIF":5.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13222286/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148035350","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
First-Line Therapy for Advanced Renal Cell Carcinoma: Score-Matched Analysis of Dual Combination Immunotherapy versus Combination of Tyrosine Kinase Inhibitors and Immunotherapy in the TOURS Multicenter Study. 晚期肾细胞癌的一线治疗:双重联合免疫治疗与酪氨酸激酶抑制剂联合免疫治疗在TOURS多中心研究中的评分匹配分析
IF 5.1 3区 医学
Targeted Oncology Pub Date : 2026-05-01 Epub Date: 2026-04-22 DOI: 10.1007/s11523-026-01219-y
Hiromitsu Watanabe, Keita Nakane, Takuhisa Nukaya, Taku Naiki, Takahiro Yasui, Kiyoshi Takahara, Takuya Koie, Teruo Inamoto, Hideaki Miyake
{"title":"First-Line Therapy for Advanced Renal Cell Carcinoma: Score-Matched Analysis of Dual Combination Immunotherapy versus Combination of Tyrosine Kinase Inhibitors and Immunotherapy in the TOURS Multicenter Study.","authors":"Hiromitsu Watanabe, Keita Nakane, Takuhisa Nukaya, Taku Naiki, Takahiro Yasui, Kiyoshi Takahara, Takuya Koie, Teruo Inamoto, Hideaki Miyake","doi":"10.1007/s11523-026-01219-y","DOIUrl":"10.1007/s11523-026-01219-y","url":null,"abstract":"<p><strong>Background: </strong>Standard first-line treatment for patients with advanced renal cell carcinoma (aRCC) has commonly included combined immune-oncology (IO) agents (IO-IO) or combinations of a tyrosine kinase inhibitor (TKI) and an IO agent (IO-TKI); however, there are few head-to-head comparative real-world studies, especially involving Japanese cohorts.</p><p><strong>Objective: </strong>To compare prognoses of patients treated with IO-IO or IO-TKI therapy in routine Japanese clinical practice.</p><p><strong>Methods: </strong>This retrospective study included 416 International Metastatic RCC Database Consortium (IMDC) intermediate- and poor-risk patients with aRCC treated with either IO-IO or IO-TKI at four institutions from September 2018 to February 2026. Effectiveness and safety outcomes were comprehensively compared. Overall (OS) and progression-free survival (PFS) rates were estimated with the Kaplan-Meier method.</p><p><strong>Results: </strong>We used propensity-score matching to compare 324 patients (IO-IO: 162; IO-TKI: 162). The IO-TKI cohort showed significantly higher objective response rates than the IO-IO cohort (66 versus 44%, respectively, p < 0.01), longer PFS (17.1 versus 8.4 months, respectively, HR = 0.56, p < 0.01), and longer OS (51.7 versus 31.5 months, respectively, HR = 0.70, p = 0.04), although OS did not reach significance in the IMDC risk-stratified subgroups. While all-grade adverse events (AEs) were more common in the IO-TKI group (94 versus 83%, respectively, p < 0.01), rates of immune-related AEs and corticosteroid administration were significantly higher in the IO-IO cohort. The study limitations include the retrospective design and treatment strategy solely decided by each physician.</p><p><strong>Conclusions: </strong>IO-TKI combinations were associated with higher rates of adverse events but survival benefits in IMDC intermediate- and poor-risk patients with aRCC.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":"399-410"},"PeriodicalIF":5.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147781801","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
Posterior Reversible Encephalopathy Syndrome with Angiogenesis Inhibitors for Solid Tumours: Clues from a Disproportionality Analysis of the FDA Adverse Event Reporting System and Pharmacodynamics. 血管生成抑制剂治疗实体肿瘤的后路可逆性脑病综合征:来自FDA不良事件报告系统和药效学的歧化分析的线索。
IF 5.1 3区 医学
Targeted Oncology Pub Date : 2026-05-01 Epub Date: 2026-05-06 DOI: 10.1007/s11523-026-01222-3
Monia Donati, Chiara Cancellerini, Valentina Giunchi, Simone Rossi, Francesco Massari, Francesco Gelsomino, Elisabetta Poluzzi, Emanuel Raschi
{"title":"Posterior Reversible Encephalopathy Syndrome with Angiogenesis Inhibitors for Solid Tumours: Clues from a Disproportionality Analysis of the FDA Adverse Event Reporting System and Pharmacodynamics.","authors":"Monia Donati, Chiara Cancellerini, Valentina Giunchi, Simone Rossi, Francesco Massari, Francesco Gelsomino, Elisabetta Poluzzi, Emanuel Raschi","doi":"10.1007/s11523-026-01222-3","DOIUrl":"10.1007/s11523-026-01222-3","url":null,"abstract":"<p><strong>Background: </strong>Posterior reversible encephalopathy syndrome (PRES) is a rare neurologic condition that may occur as an adverse drug reaction (ADR).</p><p><strong>Objective: </strong>We aimed to characterize post-marketing reporting of PRES related to angiogenesis inhibitors targeting the vascular endothelial growth factor and its receptor (VEGF/VEGFR) and to explore the underlying pharmacodynamic mechanisms.</p><p><strong>Methods: </strong>A disproportionality analysis was performed within the FDA Adverse Event Reporting System (FAERS) database. The Bayesian information component (IC) was calculated to detect signals of disproportionate reporting (SDRs), with subgroup analyses by treatment regimen, and coreported drugs as proxies for risk factors of PRES. A network analysis identified clusters of coreported events, and univariate regression models that interpolated disproportionality and receptor affinities (extracted from ChEMBL and IUPHAR/BPS databases) were performed.</p><p><strong>Results: </strong>We found 856 reports of PRES associated with anti-VEGF therapies (88.5% by healthcare professionals; 49% by tyrosine kinase inhibitors; 48% by monoclonal antibodies; 72% with immunotherapy in the 2021-2024 period). Patients were mainly females (68%), with a median age of 62 years (IQR 52-69). SDRs emerged for 12 drugs, notably bevacizumab [N = 393; IC = 3.17; 95% confidence interval (CI) 3.00-3.29] and lenvatinib (132; 3.27; 2.98-3.48). Hypertension, headache, confusional state, and proteinuria emerged in a coreported syndromic cluster. Only fibroblast growth factor receptor (FGFR) 4 emerged with a significant inverse association (β = - 0.107, p = 0.001).</p><p><strong>Conclusions: </strong>Although causality cannot be established, these findings raise the hypothesis that PRES could be a class effect of angiogenesis inhibitors and call for ongoing epidemiological surveillance and timely multiprofessional management of hypertension as a risk-minimization strategy. The potential modulatory role of FGFR4 deserves further mechanistic translational studies.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":"433-445"},"PeriodicalIF":5.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13222291/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147843151","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Real-World Outcomes of Ivosidenib in IDH1-Mutant Biliary Tract Cancer: A Multicenter Retrospective Analysis and Comparison with the TrinetX Database. Ivosidenib治疗idh1突变型胆道癌的实际结果:多中心回顾性分析和与TrinetX数据库的比较
IF 5.1 3区 医学
Targeted Oncology Pub Date : 2026-05-01 Epub Date: 2026-04-20 DOI: 10.1007/s11523-026-01211-6
Lena Dreikhausen, Alexander Olkus, Michael Dill, Christoph Springfeld, Philipp Heumann, Nadine Schulte, Konstantina Atanasova, Matthias P Ebert, Anne Letsch, Anna M Wandmacher, Tianzuo Zhan
{"title":"Real-World Outcomes of Ivosidenib in IDH1-Mutant Biliary Tract Cancer: A Multicenter Retrospective Analysis and Comparison with the TrinetX Database.","authors":"Lena Dreikhausen, Alexander Olkus, Michael Dill, Christoph Springfeld, Philipp Heumann, Nadine Schulte, Konstantina Atanasova, Matthias P Ebert, Anne Letsch, Anna M Wandmacher, Tianzuo Zhan","doi":"10.1007/s11523-026-01211-6","DOIUrl":"10.1007/s11523-026-01211-6","url":null,"abstract":"<p><strong>Background: </strong>Ivosidenib is established as targeted therapy in patients with isocitrate dehydrogenase 1 (IDH1)-mutant advanced biliary tract cancer based on results of the phase III ClarIDHy trial. However, data on the efficacy of ivosidenib in real-world cohorts are limited.</p><p><strong>Objective: </strong>This retrospective analysis aims to provide data on the efficacy and safety of ivosidenib in biliary tract cancer in a real-world cohort.</p><p><strong>Methods: </strong>Patients with IDH1 mutant biliary tract cancer treated with ivosidenib at four German university medical centers from 2020 to 2024 were retrospectively identified. Clinicopathological parameters and treatment courses were collected. Furthermore, clinical outcomes of patients with biliary tract cancer who received ivosidenib were retrospectively retrieved from the global health network database TriNetX.</p><p><strong>Results: </strong>In total, 14 patients who received ivosidenib were identified. Mean age at diagnosis was 61 years and 50% of patients were female. Nine patients had an IDH1 R132C, four an IHD1 R132L, and one an IDH1 R132S mutation. Ten patients received ivosidenib as second-line therapy and four in later lines. Partial response was observed in two patients, stable disease in three, and progressive disease in nine patients. Median progression-free survival was 5.6 months (95% confidence interval 4.3, not estimated) and median overall survival was 12.7 months (95% confidence interval 8.2, not estimated). No discontinuation of ivosidenib because of toxicity was observed. A total of 228 patients were identified in the TriNetX database. Mean age was 64.4 years and 64% of patients were female. Median overall survival in this cohort was 9.6 months (95% confidence interval 8.07, 11.19).</p><p><strong>Conclusions: </strong>Real-world data from our cohort and the TriNetX database support the oncological outcomes reported in the ClarIDHy trial and other real-world cohorts.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":"411-420"},"PeriodicalIF":5.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13222294/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147730006","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Isatuximab: A Review in Transplant-Ineligible Newly Diagnosed Multiple Myeloma. 依沙妥昔单抗:对不适合移植的新诊断多发性骨髓瘤的研究综述。
IF 5.1 3区 医学
Targeted Oncology Pub Date : 2026-05-01 Epub Date: 2026-04-21 DOI: 10.1007/s11523-026-01210-7
Sheridan M Hoy
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引用次数: 0
Consideration of Enrollment Rates of Asian, Asian Pacific Islander, and Hispanic Patients in Non-small Cell Lung Cancer Trials. 非小细胞肺癌试验中亚洲、亚太岛民和西班牙裔患者入组率的考虑
IF 5.1 3区 医学
Targeted Oncology Pub Date : 2026-05-01 DOI: 10.1007/s11523-026-01218-z
Vinay Nittur, Cathleen J Park, Misako Nagasaka
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引用次数: 0
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