Anticancer research最新文献

{"title":"Real-world Outcomes of Enfortumab Vedotin in Elderly Patients With Metastatic Urothelial Carcinoma.","authors":"Kyosuke Nishio, Kensuke Hirosuna, Taizo Uchimoto, Heima Niigawa, Sho Kakumae, Taisuke Jo, Hirofumi Morinaka, Wataru Fukuokaya, Atsuhiko Yoshizawa, Masanobu Saruta, Mamoru Hashimoto, Takafumi Minami, Yutaka Yamamoto, Moritoshi Sakamoto, Kazuki Nishimura, Ryoichi Maenosono, Takuya Tsujino, Yuki Yoshikawa, Fumihiko Urabe, Keiichiro Mori, Takafumi Yanagisawa, Shunsuke Tsuduki, Kiyoshi Takahara, Teruo Inamoto, Kazutoshi Fujita, Takahiro Kimura, Haruhito Azuma, Kazumasa Komura","doi":"10.21873/anticanres.17803","DOIUrl":"10.21873/anticanres.17803","url":null,"abstract":"<p><strong>Background/aim: </strong>Enfortumab vedotin (EV) is an antibody-drug conjugate approved for metastatic urothelial carcinoma (mUC); however, clinical data in elderly patients are limited. Given the increasing use of EV as a first-line or subsequent treatment, assessing its safety and efficacy in older populations is crucial.</p><p><strong>Patients and methods: </strong>This retrospective, multi-institutional study evaluated the safety and efficacy of EV in patients aged ≥80 years with platinum-refractory mUC. Clinical outcomes were compared with those in patients aged <80 years. Propensity score matching (PSM) was used to minimize selection bias.</p><p><strong>Results: </strong>Among 129 patients included, 25 (19%) were aged ≥80 years. Median follow-up was 7.7 months. There were no significant differences in overall survival (OS: 14.7 <i>vs.</i> 8.9 months, <i>p</i>=0.411), progression-free survival (PFS: 10.2 <i>vs.</i> 6.5 months, <i>p</i>=0.972), or objective response rate (ORR: 56% <i>vs.</i> 46%, <i>p</i>=0.248) between older and younger patients. Rates of treatment discontinuation and dose reduction were comparable between patients aged ≥80 and those aged <80 years, with no statistically significant differences (8% <i>vs.</i> 13%, <i>p</i>=0.529; 28% <i>vs.</i> 21%, <i>p</i>=0.438).</p><p><strong>Conclusion: </strong>EV demonstrated comparable efficacy and tolerability in patients aged ≥80 years relative to younger counterparts. These findings support the safe and effective use of EV in elderly patients with mUC and highlight the need for further prospective studies in this population.</p>","PeriodicalId":8072,"journal":{"name":"Anticancer research","volume":"45 10","pages":"4575-4583"},"PeriodicalIF":1.7,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145172393","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pancancer Landscape of Long Non-coding RNA Expression and Immune Status of the Tumor Microenvironment in Japanese Cancer Patients.","authors":"Tomoatsu Ikeya, Yasuto Akiyama, Haruo Miyata, Tadashi Ashizawa, Yasufumi Kikuchi, Akira Iizuka, Chie Maeda, Akari Kanematsu, Kazue Yamashita, Kenichi Urakami, Takeshi Nagashima, Keiichi Ohshima, Ken Yamaguchi","doi":"10.21873/anticanres.17782","DOIUrl":"10.21873/anticanres.17782","url":null,"abstract":"<p><strong>Background/aim: </strong>Advances in whole-genome sequencing have revealed that many RNA genes have important functions in various cellular processes and long non-coding RNAs (lncRNAs) have been attracting increasing attention. In the present study, we characterized a large number of lncRNAs from cancer patients and classified them based on their relationships with immune response-associated genes and cancer prognosis.</p><p><strong>Materials and methods: </strong>435 lncRNAs were identified as differentially expressed genes from 5,013 cancer patients using the <i>limma</i> package and were narrowed down to 80 lncRNAs commonly expressed in more than 4 cancer types. The relationships of these lncRNAs with immune response-associated genes were examined using Pearson's correlation and the tumor immune scoring system. Through a receiver operating characteristic (ROC) analysis of highly scored lncRNAs with the tumor-infiltrating lymphocyte (TIL) status using QuanTIseq, a cancer prognostic analysis was conducted using a multivariate Cox proportional hazards model.</p><p><strong>Results: </strong>In a correlation analysis of 80 lncRNA genes and immune response-associated genes, lncRNA genes were scored and classified as high, intermediate, and low immune status score groups based on the Tumor Immune Status Scoring Algorithm (TIMMUSCORA). The ROC analysis of lncRNA genes with a high score (>200) revealed high sensitivity for the identification of TILs, such as CD8⁺ T cells and B cells. Furthermore, 11 of 80 lncRNA genes were identified as prognostic genes.</p><p><strong>Conclusion: </strong>Twelve lncRNA genes with a high immune status score were identified as immunogenic and CD8⁺ T cell infiltration-associated genes, with two being indicators of a positive cancer prognosis. These results indicate the potential of 12 lncRNA genes with a high immune status score as immunogenic biomarkers associated with a positive immune response and prognosis.</p>","PeriodicalId":8072,"journal":{"name":"Anticancer research","volume":"45 10","pages":"4315-4331"},"PeriodicalIF":1.7,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145172752","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic Impact of PD-1 Polymorphisms in Non-small Cell Lung Cancer Receiving Dendritic Cell Vaccination.","authors":"Masahiro Ogasawara, Mamiko Miyashita, Yuka Yamagishi, Shuichi Ota","doi":"10.21873/anticanres.17780","DOIUrl":"10.21873/anticanres.17780","url":null,"abstract":"<p><strong>Background/aim: </strong>The prognosis of patients with non-small cell lung cancer (NSCLC) remains poor. This phase II clinical trial investigated the efficacy of dendritic cell (DC) vaccination using Wilms' tumor 1 (WT1) and/or mucin 1 (MUC1) peptides - universal tumor-associated antigens - in combination with chemotherapy in patients with advanced-stage NSCLC. Additionally, we examined whether single nucleotide polymorphisms (SNPs) in immune checkpoint genes could serve as prognostic markers for survival.</p><p><strong>Patients and methods: </strong>Forty-four eligible patients with metastatic, unresectable or recurrent NSCLC were enrolled. DCs were administered intradermally every two to four weeks in combination with chemotherapy.</p><p><strong>Results: </strong>Following the seventh vaccination, four patients achieved a partial response and 15 patients had stable disease. The median progression-free survival (PFS) and overall survival (OS) from the initiation of DC vaccination were 6.7 months and 12.3 months, respectively. Log-rank analysis revealed that neutrophil-to-lymphocyte ratio, platelet-to-lymphocyte ratio, prognostic nutritional index (PNI) and the programmed cell death 1 (PD-1) rs36084323 SNP were significantly associated with both PFS and OS. Cox regression analysis identified the PD-1 rs36084323 SNP and PNI as independent prognostic factors for both PFS and OS.</p><p><strong>Conclusion: </strong>DC vaccination with WT1 and/or MUC1 peptides combined with salvage chemotherapy appears to be a promising treatment strategy for patients with advanced or relapsed NSCLC. The PD-1 rs36084323 SNP, along with PNI, was identified as an independent prognostic factor, suggesting its potential utility as a biomarker for selecting appropriate candidates for this treatment approach.</p>","PeriodicalId":8072,"journal":{"name":"Anticancer research","volume":"45 10","pages":"4295-4304"},"PeriodicalIF":1.7,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145172292","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Retraction.","authors":"","doi":"10.21873/anticanres.17816","DOIUrl":"10.21873/anticanres.17816","url":null,"abstract":"","PeriodicalId":8072,"journal":{"name":"Anticancer research","volume":"45 10","pages":"4667"},"PeriodicalIF":1.7,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145172506","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Synchronous Gallbladder Cancer and Distal Cholangiocarcinoma Without Pancreaticobiliary Maljunction: A Case Report With Literature Review.","authors":"Shota Uemura, Yuki Kitano, Tatsunori Miyata, Teruki Sako, Yuki Hisano, Rumi Itoyama, Shigeki Nakagawa, Hirohisa Okabe, Hiromitsu Hayashi, Masaaki Iwatsuki","doi":"10.21873/anticanres.17812","DOIUrl":"10.21873/anticanres.17812","url":null,"abstract":"<p><strong>Background/aim: </strong>Reports of synchronous cancers in the biliary tract without pancreaticobiliary maljunction (PBM) are exceedingly rare. We present a case of synchronous gallbladder cancer and distal cholangiocarcinoma occurring in the absence of PBM.</p><p><strong>Case report: </strong>A 60-year-old man presented with abdominal pain and jaundice. Preoperative imaging findings revealed a 23 mm tumor in the gallbladder and a 38 mm tumor in the distal bile duct, without signs of PBM. Preoperative diagnosis included gallbladder cancer (cT2N0M0, stage II) and distal cholangiocarcinoma (cT1N0M0, stage IA), and the patient underwent subtotal stomach-preserving pancreatoduodenectomy and liver segment IVb/V resection. Histopathology confirmed distinct gallbladder cancer and distal cholangiocarcinoma, without continuity between the tumors. The patient has been alive for 32 months after surgery without any signs of recurrence.</p><p><strong>Conclusion: </strong>Although synchronous cancers in the biliary tract without PBM are rare, attention should be paid to the possibility of another lesion being present in the biliary tract during the diagnostic and treatment processes.</p>","PeriodicalId":8072,"journal":{"name":"Anticancer research","volume":"45 10","pages":"4659-4664"},"PeriodicalIF":1.7,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145172636","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Impact of Adjuvant Chemotherapy in Patients With Locally Advanced Rectal Cancer After Neoadjuvant Chemotherapy: A Propensity Score Matching Cohort Study.","authors":"Koji Fukata, Atsushi Ogura, Yuki Murata, Ryutaro Kobayashi, Shoji Kawakatsu, Shizuki Sugita, Shunsuke Onoe, Kazushi Miyata, Junpei Yamaguchi, Takashi Mizuno, Tomoki Ebata","doi":"10.21873/anticanres.17791","DOIUrl":"10.21873/anticanres.17791","url":null,"abstract":"<p><strong>Background/aim: </strong>Total neoadjuvant therapy has been widespread over the past years and the multimodal treatment strategy involving administration of neoadjuvant chemotherapy (NAC) gradually increases for patients with locally advanced rectal cancer. However, the necessity of adjuvant chemotherapy (AC) following NAC remains controversial. The aim of this study was to investigate the impact of AC on survival outcomes and the influence of perioperative chemotherapy on treatment efficacy in patients with locally advanced rectal cancer (LARC) who received NAC followed by surgery.</p><p><strong>Patients and methods: </strong>We retrospectively analyzed 107 patients with LARC who received NAC using propensity score matching (PSM) with covariates of clinical N stage, anus-preserving status, presence of severe complications (≥ Clavien-Dindo 3). All patients were planned and treated for three months with oxaliplatin-based doublet chemotherapy preoperatively.</p><p><strong>Results: </strong>There were no significant differences between AC and Non-AC groups in 50 patients of the PSM cohort. At a median follow-up of 85.6 months, the 5-year overall survival was 88.0% and the 5-year relapse-free survival (RFS) was 81.3% for the entire cohort. The 5-year RFS was significantly better in the AC than in the non-AC group (87.3% <i>vs.</i> 79.8%, <i>p</i>=0.033). Multivariate analysis demonstrated that non-AC and lateral lymph node metastasis were poor prognostic factors of RFS in patients with LARC [non-AC: Hazard ratio (HR)=4.089, 95% confidence interval (CI)=1.217-13.735, <i>p</i>=0.023; lateral lymph node metastasis: HR=9.04, 95%CI=1.737-47.040, <i>p</i>=0.009].</p><p><strong>Conclusion: </strong>AC could improve RFS in patients with LARC following NAC.</p>","PeriodicalId":8072,"journal":{"name":"Anticancer research","volume":"45 10","pages":"4431-4439"},"PeriodicalIF":1.7,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145172660","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic Impact of <i>ALK</i> Rearrangements in Resected NSCLC: A Systematic Review and Meta-analysis.","authors":"Hayoung Seong, Soo Han Kim, Mi-Hyun Kim, Jung Seop Eom","doi":"10.21873/anticanres.17799","DOIUrl":"10.21873/anticanres.17799","url":null,"abstract":"<p><strong>Background/aim: </strong>ALK receptor tyrosine kinase (<i>ALK</i>) rearrangements occur in 4-8% of non-small cell lung cancer (NSCLC) and are often associated with aggressive clinical behavior. Although ALK-targeted tyrosine kinase inhibitors significantly improve overall survival in advanced-stage NSCLC, their role in early-stage disease remains unclear. This meta-analysis (PROSPERO CRD420251012874) aimed to systematically evaluate recurrence rates and disease-free survival (DFS) outcomes in surgically resected patients with <i>ALK</i>-rearranged NSCLC compared to <i>ALK</i>-wild type (WT).</p><p><strong>Patients and methods: </strong>We conducted a systematic literature review according to PRISMA 2020 guidelines. Eligible studies included patients with surgically resected NSCLC with reported recurrence rate and DFS. Pooled recurrence rates, hazard ratios (HRs) for DFS, and recurrence patterns were analyzed using random effects models.</p><p><strong>Results: </strong>Fifteen studies encompassing 4,776 patients (630 <i>ALK</i>-rearranged and 4,146 <i>ALK</i>-WT) were included. The pooled recurrence rates were 40.2% and 30.2% for ALK-rearranged and <i>ALK</i>-WT patients, respectively. <i>ALK</i>-rearranged patients demonstrated a trend toward increased recurrence risk (risk ratio=1.20; 95% confidence interval=0.98-1.49; <i>p</i>=0.084) and significantly reduced DFS (HR=1.42; 95% confidence interval=1.08-1.88; <i>p</i>=0.013), particularly within 3 years follow-up and stage I subgroups. Furthermore, patients with <i>ALK</i>-rearranged NSCLC experienced significantly higher rates of systemic (RR, 1.32; <i>p</i>=0.027) and brain metastases (RR=1.71; <i>p</i>=0.046).</p><p><strong>Conclusion: </strong>Surgically resected <i>ALK</i>-rearranged NSCLC involves an increased risk of recurrence and reduced DFS, notably within the first 3 years, accompanied by higher rates of systemic and brain metastases. These findings underscore the importance of tailored postoperative surveillance strategies and suggest the potential role of perioperative ALK-tyrosine kinase inhibitors.</p>","PeriodicalId":8072,"journal":{"name":"Anticancer research","volume":"45 10","pages":"4519-4534"},"PeriodicalIF":1.7,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145172745","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development and Validation of a Nomogram of Predict Eligibility for Second-line Chemotherapy in Pancreatic Cancer.","authors":"Kentaro Sato, Rei Suzuki, Naoki Konno, Hiroki Irie, K O Watanabe, Hiromichi Imaizumi, Yuichi Waragai, Mitsuru Sugimoto, Hiroyuki Asama, Hiroshi Shimizu, Rei Ohira, Hiromasa Ohira","doi":"10.21873/anticanres.17798","DOIUrl":"10.21873/anticanres.17798","url":null,"abstract":"<p><strong>Background/aim: </strong>For pancreatic cancer, combination therapies, such as gemcitabine plus nab-paclitaxel (GnP) or FOLFIRINOX (FFX), are recommended as first-line (FL) chemotherapy. Second-line (SL) chemotherapy has been shown to improve survival, but factors predicting eligibility for SL chemotherapy remain unclear. This study aimed to identify factors predicting eligibility for SL chemotherapy and to develop a predictive nomogram.</p><p><strong>Patients and methods: </strong>This retrospective multicenter study included 278 patients with unresectable PC who received FL chemotherapy between 2017 and 2023. Patients were divided into the development (n=140) and external validation (n=138) cohorts. Univariate and multivariate logistic regression analyses were performed to identify predictors of eligibility for SL chemotherapy. A nomogram was developed using these independent predictors and was validated <i>via</i> calibration curves and c-statistics.</p><p><strong>Results: </strong>The independent predictors of eligibility for SL chemotherapy included age (<70 years old), C-reactive protein (CRP) levels, and the FL regimen (combination <i>vs.</i> monotherapy). The nomogram demonstrated good predictive accuracy (c-statistic: 0.78) in the development cohort and acceptable accuracy (c-statistic: 0.68) in the validation cohort. The calibration curves showed good agreement between predicted and observed probabilities.</p><p><strong>Conclusion: </strong>The nomogram developed herein is a reliable tool for predicting eligibility for SL chemotherapy among PC patients, thereby facilitating informed treatment decisions.</p>","PeriodicalId":8072,"journal":{"name":"Anticancer research","volume":"45 10","pages":"4509-4518"},"PeriodicalIF":1.7,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145172623","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychological Stress <i>Versus</i> Outcome in Patients With Breast Disease and Breast Cancer: A Prospective 25-year Follow-up Study in Patients With Breast Cancer Symptoms.","authors":"Matti Eskelinen, Riika Koskela, Tuomas Selander, Kai Kaarniranta, Paula Ollonen, Maaret Eskelinen","doi":"10.21873/anticanres.17741","DOIUrl":"https://doi.org/10.21873/anticanres.17741","url":null,"abstract":"<p><strong>Background/aim: </strong>This study evaluated whether a psychological inventory (Forsen inventory, FI) for measuring the severity of psychological stress (PS) could predict long-term outcomes in patients having breast carcinoma (BC) symptoms.</p><p><strong>Patients and methods: </strong>A total of 115 patients with BC symptoms completed the FI scale and associations between FI scale and long-term outcomes were analysed using survival models.</p><p><strong>Results: </strong>The FI scores correlated with Beck Depression Inventory (BDI) (<i>r</i>=0.678, <i>p</i><0.001), Montgomery-Asberg Depression Rating Scale (MADRS) (<i>r</i>=0.820, <i>p</i><0.001), and State-Trait Anxiety inventory (STAI) (<i>r</i>=0.556, <i>p</i><0.001) levels. In Cox regression models, higher FI scores predicted poorer 25-year relapse-free survival (RFS) in BC patients (HR=2.60, <i>p</i>=0.05) and poorer 25-year overall survival (OS) in patients with benign breast disease (BBD) (HR=7.79, <i>p</i>=0.05). Kaplan-Meier analysis showed significantly better 25-year RFS among BC patients with low FI scores (<14) compared to those with high scores (≥14) (58.3% <i>vs.</i> 30.0%, <i>p</i>=0.046), and higher 25-year OS in BBD patients with low FI scores (95.8% <i>vs.</i> 71.4%, <i>p</i>=0.022).</p><p><strong>Conclusion: </strong>The FI scale, though underutilized, effectively captures PS and significantly predicts long-term outcomes in patients with breast disease. These findings suggest that incorporating the FI into diagnostic evaluations could enhance risk stratification and management for women with BC symptoms.</p>","PeriodicalId":8072,"journal":{"name":"Anticancer research","volume":"45 9","pages":"3817-3826"},"PeriodicalIF":1.7,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144940000","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Phase II Trial of Pemetrexed Plus Oxaliplatin in Patients With Advanced Biliary Tract Cancer After Failure of Gemcitabine Plus Cisplatin-based Chemotherapy.","authors":"Jwa Hoon Kim, Changhoon Yoo, Hyehyun Jeong, Jaekyung Cheon, Kyu-Pyo Kim, Baek-Yeol Ryoo, Heung-Moon Chang, Chung Ryul Oh, Hye Won Ryu, Inkeun Park","doi":"10.21873/anticanres.17756","DOIUrl":"https://doi.org/10.21873/anticanres.17756","url":null,"abstract":"<p><strong>Background/aim: </strong>This study aimed to evaluate the efficacy and safety of pemetrexed/oxaliplatin (PemOx) in patients with advanced or metastatic biliary tract cancer (aBTC) after failure of gemcitabine/cisplatin (GP)-based chemotherapy.</p><p><strong>Patients and methods: </strong>This investigator-initiated, multicenter trial was conducted at four tertiary referral centers in South Korea. PemOx was administered as follows: intravenous Pem 500 mg/m² and Ox 120 mg/m² on day 1, every three weeks. The primary endpoint was the objective response rate (ORR). Secondary endpoints included progression-free survival (PFS), overall survival (OS), and treatment-related adverse events (TRAEs). Based on the Minimax two-stage design, a total of 30 patients were planned to be enrolled to detect an increase in the ORR from 5% to 20%, with two-sided α=0.05, β=0.2, and a 10% dropout rate.</p><p><strong>Results: </strong>Between November 2023 and November 2024, 30 patients were enrolled. The median age of the patients was 68 years, and 70% were male. Five patients achieved a partial response, resulting in an ORR of 16.7% and a disease control rate of 76.7%. With a median follow-up duration of 11.0 months (range=6.9-15.1 months), the median PFS and OS were 3.6 months [95% confidence interval (CI)=1.9-5.3] and 9.9 months (95%CI=6.8-13.0), respectively. Dose delays or reductions were required in 13 (43.3%) and four (13.3 %) patients, respectively. Grade 3 TRAEs were reported in three patients (10%). No grade ≥4 TRAEs were reported.</p><p><strong>Conclusion: </strong>PemOx showed modest efficacy and manageable TRAEs in patients with aBTC after the failure of first line GP-based chemotherapy, although the primary endpoint was not met.</p>","PeriodicalId":8072,"journal":{"name":"Anticancer research","volume":"45 9","pages":"3983-3991"},"PeriodicalIF":1.7,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144940005","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}