Targeted Oncology最新文献

{"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":"https://doi.org/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":""},"PeriodicalIF":4.0,"publicationDate":"2026-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"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":"https://doi.org/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":""},"PeriodicalIF":4.0,"publicationDate":"2026-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Epidemiology and Real-World Outcomes in Patients with Human Epidermal Growth Factor Receptor 2 (HER2)-Mutant Non-small Cell Lung Cancer by Region: A Targeted Literature Review.","authors":"Koichi Goto, Hidetoshi Hayashi, Sonia S Maruti, Heiko Zettl, Lalith Mittal, Smit Pathak, Xiuning Le","doi":"10.1007/s11523-026-01213-4","DOIUrl":"https://doi.org/10.1007/s11523-026-01213-4","url":null,"abstract":"<p><p>Human epidermal growth factor receptor 2 (HER2) mutations in non-small cell lung cancer (NSCLC) are associated with aggressive disease and poor prognosis. Improved understanding of patient characteristics is vital to advance personalized treatment and improve outcomes. In this structured protocol-based targeted literature review, we assessed the epidemiology and 'real-world' outcomes in patients with HER2-mutant NSCLC by region, and by mutation category (tyrosine kinase domain [TKD] and non-TKD). Across 64 studies, the frequency of HER2 mutations ranged from 1% to 5%. Patients with HER2-mutant NSCLC were more frequently female, never smokers, and exhibited a high burden of central nervous system involvement. The HER2 mutational landscape differed between TKD and non-TKD groups, with non-TKD mutations associated with higher frequencies of concomitant mutations and higher tumor mutational burden. Few studies systematically assessed HER2 mutation oncogenicity; however, TKD mutations appear to be more commonly oncogenic than non-TKD mutations. Further information regarding the oncogenicity of uncommon HER2 mutations is required. Most patients received first-line platinum-doublet chemotherapy or chemoimmunotherapy, with no clear second-line standard of care. Clinical outcomes were modest. There is a clear unmet need for HER2-directed agents. Three such agents have gained accelerated US Food and Drug Administration (FDA) approval for use in previously treated HER2-mutant NSCLC: the antibody-drug conjugate, trastuzumab deruxtecan; the HER2-specific tyrosine kinase inhibitor (TKI), zongertinib; and the HER2/EGFR TKI, sevabertinib. Zongertinib has also been granted accelerated FDA approval in a first-line setting. The emergence of multiple treatment options highlights the importance of early HER2 mutation testing to guide treatment sequencing and maximize patient benefit.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2026-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147843194","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":"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":"https://doi.org/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":""},"PeriodicalIF":4.0,"publicationDate":"2026-05-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Correction: Real-World Outcomes of Sequential Afatinib and Osimertinib Versus Afatinib and Chemotherapy in EGFR-Mutant NSCLC: Taiwan Multicenter GIANT Study.","authors":"How-Wen Ko, Ying-Ting Liao, Sheng-Kai Liang, Yen-Hsiang Huang, Yu-Ching Lin, Ping-Chi Liu, Chin-Chou Wang, Jeng-Shiuan Tsai, Yuh-Min Chen, Jin-Yuan Shih, Tsung-Ying Yang, Cheng-Ta Yang","doi":"10.1007/s11523-026-01216-1","DOIUrl":"https://doi.org/10.1007/s11523-026-01216-1","url":null,"abstract":"","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2026-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147781796","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":"Consideration of Enrollment Rates of Asian, Asian Pacific Islander, and Hispanic Patients in Non-small Cell Lung Cancer Trials.","authors":"Vinay Nittur, Cathleen J Park, Misako Nagasaka","doi":"10.1007/s11523-026-01218-z","DOIUrl":"https://doi.org/10.1007/s11523-026-01218-z","url":null,"abstract":"<p><p>Despite efforts over the last decade by clinical investigators and organizations to enhance diversity in clinical trials, significant underrepresentation of Asian, Asian Pacific Islander, and Hispanic patients remains an issue. Our editorial highlights key barriers to achieving equity in clinical trial enrollment among patients with non-small cell lung cancer (NSCLC) along with strategies to improve diversity to ensure more generalized clinical outcomes.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2026-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147781842","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":"Therapeutic Strategies Targeting the Tumor-Stromal Microenvironment in Colorectal Cancer: A Narrative Review.","authors":"Floor Heilijgers, Marie-Christine Bakker, Bente van Hest, Ingrid Franken, Frank M Speetjens, Koen C M J Peeters, Jeanine Roodhart, Wilma E Mesker","doi":"10.1007/s11523-026-01220-5","DOIUrl":"https://doi.org/10.1007/s11523-026-01220-5","url":null,"abstract":"<p><p>Despite advances in colorectal cancer (CRC) treatment, outcomes in advanced disease remain poor. Current therapies mainly target the epithelial compartment of the tumor, yet increasing evidence highlights the tumor microenvironment (TME), particularly the tumor stroma, as a prognostic and potentially predictive factor. This narrative review summarizes current evidence on stromal features as biomarkers and therapeutic targets in CRC. These stromal markers, such as fibroblast-associated protein (FAP), are associated with adverse outcomes, underscoring their clinical relevance. However, therapies directly targeting these stromal components have largely failed to improve survival as single agents. Combination strategies, stromal modulation integrated with established treatments, appear more promising. However, to date, stromal targeting remains challenging and has not led to standard-of-care treatment options. This highlights the need for more effective agents, the rational design of combination strategies, and improved patient selection. Combining stroma modulators with standard therapy, guided by stromal biomarkers, may enhance efficacy and help overcome the TME-induced resistance.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2026-04-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147781833","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":"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":"https://doi.org/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":""},"PeriodicalIF":4.0,"publicationDate":"2026-04-22","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}

{"title":"Isatuximab: A Review in Transplant-Ineligible Newly Diagnosed Multiple Myeloma.","authors":"Sheridan M Hoy","doi":"10.1007/s11523-026-01210-7","DOIUrl":"https://doi.org/10.1007/s11523-026-01210-7","url":null,"abstract":"<p><p>Isatuximab (isatuximab-irfc; SARCLISA^®) is an anti-CD38 monoclonal antibody approved in the EU and the USA for use in combination with bortezomib, lenalidomide and dexamethasone for the treatment of adults with newly diagnosed multiple myeloma (NDMM) who are ineligible for autologous stem cell transplantation (ASCT). In a phase III study in this patient population, isatuximab-bortezomib-lenalidomide-dexamethasone significantly prolonged progression-free survival (PFS) and generally improved the depth of tumour response versus bortezomib-lenalidomide-dexamethasone. Overall survival (OS) data were immature at the time of this analysis. Health-related quality of life was not affected by adding isatuximab to bortezomib-lenalidomide-dexamethasone. Only a slight increase in toxicity resulted from the addition of isatuximab to bortezomib-lenalidomide-dexamethasone, with the safety findings from the study consistent with the known safety profile of isatuximab-bortezomib-lenalidomide-dexamethasone. Although further interim and final PFS and OS data are awaited, current evidence indicates that isatuximab-bortezomib-lenalidomide-dexamethasone is a useful addition to the treatment options available for adults with NDMM who are ineligible for ASCT.</p>","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2026-04-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147781847","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":"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":"https://doi.org/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":""},"PeriodicalIF":4.0,"publicationDate":"2026-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}