American Journal of Clinical Oncology-Cancer Clinical Trials最新文献

{"title":"Immunotherapy in Extensive Stage Small-Cell Lung Cancer in First-Line and Second-Line Setting: A Systematic Review and Meta-Analysis.","authors":"Nupur Krishnan, Patsy Lee, Gabriel Boldt, Suganija Lakkunarajah, Saurav Verma, Phillip Blanchette, Jacques Raphael","doi":"10.1097/COC.0000000000001265","DOIUrl":"10.1097/COC.0000000000001265","url":null,"abstract":"<p><strong>Objectives: </strong>Despite a good response to first-line chemotherapy, small-cell lung cancer (SCLC) has high relapse rates and a poor prognosis. We conducted a systematic review and meta-analysis to assess the role of immune checkpoint inhibitors (ICIs) in the treatment of extended stage SCLC (ES-SCLC), in different lines of therapy.</p><p><strong>Methods: </strong>Medline (PubMed), EMBASE, and Cochrane Library databases between January 2010 and March 2025 and conference proceedings between 2018 and 2025 were searched for RCTs assessing ICIs versus chemotherapy in patients with ES-SCLC. Primary endpoints were overall survival (OS) and progression-free survival (PFS). Secondary endpoints included objective response rate (ORR) and grade 3+ adverse events. Pooled hazard ratios (HR) for OS and PFS were meta-analyzed using the generic inverse variance method, and random-effect models were used to compute pooled estimates. Subgroup analyses compared survival by line of therapy, sex, age, and ECOG status.</p><p><strong>Results: </strong>ICIs decreased risk of death by 19% (HR: 0.81, 95% CI: 0.76-0.86). OS benefit was regardless of age, sex, or ECOG, but only in first-line treatment. ICIs decreased the risk of disease progression by 22% (HR: 0.78, 95% CI: 0.67-0.91), with PFS benefit restricted to first-line treatment with a detrimental effect in the second line. ICIs improved ORR (OR: 0.79, 95% CI: 0.66-0.95), but were associated with increased grade 3+diarrhea (OR: 3.63, 95% CI: 1.46-9.02).</p><p><strong>Conclusions: </strong>ICIs conferred efficacy benefits and an acceptable safety profile in the treatment of patients with ES-SCLC in the first-line, but should not be used in the second-line as single agents. Biomarkers predicting long-term benefit are needed to further improve outcomes.</p>","PeriodicalId":50812,"journal":{"name":"American Journal of Clinical Oncology-Cancer Clinical Trials","volume":" ","pages":"219-228"},"PeriodicalIF":1.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145716535","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 2 Trial of Protein Kinase C Iota Inhibition With the Combination of Auranofin and Sirolimus in Patients With Recurrent Ovarian Cancer.","authors":"Aminah Jatoi, Nathan R Foster, Andrea Wahner Hendrickson, Matthew S Block, S John Weroha, Erik J Asmus, Nicole R Murray, Alan P Fields","doi":"10.1097/COC.0000000000001263","DOIUrl":"10.1097/COC.0000000000001263","url":null,"abstract":"<p><strong>Objectives: </strong>This trial served as a proof-of-concept for whether inhibition of protein kinase C iota (PKCι) with auranofin and sirolimus provide antineoplastic effects in patients with recurrent high-grade serous ovarian cancer.</p><p><strong>Methods: </strong>This drug combination was administered to patients with recurrent high-grade serous ovarian cancer. Dosing was based on unpublished phase 1 data and consisted of auranofin 6 mg and sirolimus 5 mg both orally per day of a 28-day cycle. The primary endpoint was tumor response. Available tumor tissue was assessed for PKCι protein expression by immunohistochemistry (IHC) and PRKCI copy number by fluorescence in-situ hybridization (FISH) after the start of cancer therapy.</p><p><strong>Results: </strong>Twenty-two patients were enrolled, and 21 were evaluable for all clinical trial endpoints. One patient was unevaluable because she did not receive a full chemotherapy cycle. No tumor responses were seen in the first 21 patients, resulting in early trial termination per a priori trial design. The median progression-free survival was 2.1 months (95% CI: 1.8-3.7). The median overall survival was 4.4 months (95% CI: 2.6-12.5). Fourteen (67%) patients had at least one grade 3 or worse adverse event. Nineteen of 21 evaluable patients had available tumor tissue, which showed the median PKCι copy number averaged per cell of 3 (range: 2 to 7), and PKCι expression (at least 1+) in all.</p><p><strong>Conclusions: </strong>As prescribed here, auranofin and sirolimus manifested no antineoplastic activity in patients with recurrent high-grade serous ovarian cancer that expressed PKCι.</p>","PeriodicalId":50812,"journal":{"name":"American Journal of Clinical Oncology-Cancer Clinical Trials","volume":" ","pages":"238-242"},"PeriodicalIF":1.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145330931","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":"Heart Disease Mortality After Primary Cancer Surgery in the United States.","authors":"Jessica J Bai, Kyle A Mani, Daxuan Deng, Luke Rothermel, Jonathan Shoag, Daniel E Spratt, Ming Wang, Nicholas G Zaorsky","doi":"10.1097/COC.0000000000001261","DOIUrl":"10.1097/COC.0000000000001261","url":null,"abstract":"<p><strong>Objectives: </strong>Patients with cancer may be at higher risk of heart disease from anticancer therapies. However, there is limited data on the risks of heart disease mortality after primary cancer site surgery. This study sought to evaluate the incidence, timing, and risk factors of heart disease mortality after primary cancer site surgery.</p><p><strong>Methods: </strong>The Surveillance, Epidemiology, and End Results (SEER) database was used to perform a retrospective population-based study of cancer patients who underwent primary surgical resection from 2000 to 2020. The incidence of heart disease mortality after primary cancer site surgery was described by standardized mortality ratios (SMRs) and the timing of heart disease mortality after surgery was characterized. Risk factors were identified using Fine and Gray competing risk analysis.</p><p><strong>Results: </strong>Among the 1,390,585 cancer patients who underwent primary surgical resection from 2000 to 2020, 178,303 (12.8%) died of heart disease. The SMR of heart disease death after surgery was 6.85 (95% CI: 6.82-6.88, P <0.001). SMRs were highest in cancers of the brain and other nervous system, esophagus, liver and intrahepatic bile duct, pancreas, and lung and bronchus. Approximately 50% of all heart disease deaths occurred within the first 5 years after surgery for all cancers. Risk factors included older age, male sex, Black race, unmarried status, and rurality.</p><p><strong>Conclusions: </strong>The incidence of death from heart disease was significantly elevated in patients who underwent primary cancer site surgery compared with the general US population. These findings can be used to guide surgical planning and follow-up strategies.</p>","PeriodicalId":50812,"journal":{"name":"American Journal of Clinical Oncology-Cancer Clinical Trials","volume":" ","pages":"229-237"},"PeriodicalIF":1.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145535041","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 Cost-effectiveness Analysis of Adjuvant Alectinib in Patients With Resectable ALK -positive Non-small Cell Lung Cancer in the United States.","authors":"Amy L Cummings, Jesse Sussell, Katherine L Rosettie, Fadoua El Moustaid, Sarika Ogale, Celina Ngiam, Nick Jovanoski, Melina Arnold, Jay M Lee","doi":"10.1097/COC.0000000000001248","DOIUrl":"10.1097/COC.0000000000001248","url":null,"abstract":"<p><strong>Objectives: </strong>We evaluated the cost-effectiveness of adjuvant alectinib versus adjuvant platinum-based chemotherapy for patients with resectable (stage IB to IIIA) ALK + non-small cell lung cancer using a US societal perspective.</p><p><strong>Methods: </strong>We developed a cohort-level Markov model to compare adjuvant alectinib versus platinum-based chemotherapy using a lifetime time horizon (40 y) with a monthly cycle length and 3% discounting of health state utilities and costs. Patients started in a disease-free health state; downstream health states included treated (first- or second-line) or untreated metastatic or nonmetastatic recurrence, or death. Patient characteristics, adjuvant treatment patterns, and health utilities were based on the ALINA trial.</p><p><strong>Results: </strong>When measured across a lifetime time horizon, adjuvant alectinib was estimated to lead to 3.1 additional quality-adjusted life-years (QALYs) with $429,925 lower total costs per patient, demonstrating a dominant cost-effectiveness ratio compared with adjuvant platinum-based chemotherapy (more effective and less costly). The net monetary benefit in favor of adjuvant alectinib was $895,766 at a willingness-to-pay (WTP) threshold of $150,000/QALY gained. In probabilistic sensitivity analysis, adjuvant alectinib has a 99.6% probability of being more effective and less costly than adjuvant chemotherapy. The incremental cost-effectiveness ratio for adjuvant alectinib remained dominant when all model inputs were varied in the one-way sensitivity analysis.</p><p><strong>Conclusions: </strong>In comparison with platinum-based chemotherapy, adjuvant alectinib offers substantial additional clinical and economic value to society.</p>","PeriodicalId":50812,"journal":{"name":"American Journal of Clinical Oncology-Cancer Clinical Trials","volume":" ","pages":"205-211"},"PeriodicalIF":1.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145253645","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":"Real World Radiographic Response Rates and Preoperative Attrition in Patients With Non-Small Cell Lung Cancer (NSCLC) Treated With Neoadjuvant Chemoimmunotherapy.","authors":"Cole Friedes, Michelle Iocolano, Nikhil Yegya-Raman, John C Kucharczuk, Taine T V Pechet, Roger B Cohen, Charu Aggarwal, Melina E Marmarelis, William P Levin, Keith A Cengel, Christine A Ciunci, Christopher D'Avella, Aditi P Singh, Christiana W Davis, Corey J Langer, Jeffrey Bradley, Steven J Feigenberg","doi":"10.1097/COC.0000000000001260","DOIUrl":"10.1097/COC.0000000000001260","url":null,"abstract":"<p><strong>Objectives: </strong>To evaluate real-world rates of radiographic response and surgery in patients treated with neoadjuvant chemoimmunotherapy for stage II to III NSCLC.</p><p><strong>Methods: </strong>We evaluated a prospectively maintained single-institution database of patients of stage II to III NSCLC treated with neoadjuvant chemoimmunotherapy from January 2022 to July 2024. Rates of radiographic response, surgery, and reasons for abandoning surgery were recorded. Toxicity was graded according to the Common Terminology Criteria for Adverse Events for systemic treatment and the Clavien-Dindo Scale for surgery.</p><p><strong>Results: </strong>Overall, 1243 patients were screened: 323 had stage II-III NSCLC that could be treated with curative intent, of whom 111 were considered eligible for surgery and 36 were treated with neoadjuvant chemoimmunotherapy, which will reflect the remainder of this report. The overall response rate (ORR) was 53% and median radiographic change in the sum of tumor diameter was -34% (IQR -44 to -11). In patients with PD-L1 ≥50%, the ORR was 76%. Most patients had evidence of radiographic downstaging after neoadjuvant treatment. The rate of surgical intervention was 58% (n=21). No patient thought to require a pneumonectomy before neoadjuvant therapy underwent surgery. Pathologic complete response rate was seen in 5 of the 21 patients (24%). There were 42 adverse events from chemoimmunotherapy and 19 from surgery, of which 12% and 11% were grade 3 or higher, respectively.</p><p><strong>Conclusions: </strong>At a tertiary care center, the rate of surgical intervention after neoadjuvant chemoimmunotherapy was 58%. These results require further validation in additional external cohorts and highlight the need for optimal patient selection to ensure the use of curative surgery.</p>","PeriodicalId":50812,"journal":{"name":"American Journal of Clinical Oncology-Cancer Clinical Trials","volume":" ","pages":"212-218"},"PeriodicalIF":1.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145460220","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":"Optimizing Natural Killer Cellular Therapy in Acute Myeloid Leukemia.","authors":"Binsah George, Manu Pandey, Jacob Herstein, Abhishek Maiti","doi":"10.1097/COC.0000000000001262","DOIUrl":"10.1097/COC.0000000000001262","url":null,"abstract":"<p><p>Acute myeloid leukemia (AML) continues to pose a major hurdle in hematologic oncology, driven by its genetic complexity and tendency to resist standard therapies. Even with progress in treatment-such as high-dose chemotherapy and hematopoietic stem cell transplantation (HSCT)-outcomes remain unsatisfactory for many patients. In recent years, immunotherapy has emerged as an appealing strategy to improve survival by strengthening the body's own anti-leukemia defenses. Natural killer (NK) cells, a critical component of innate immunity, have shown strong potential for directly eliminating AML cells without prior antigen exposure. This review outlines the role of NK cells in AML immune surveillance, mechanisms by which their function becomes impaired in the disease, and the current therapeutic approaches harnessing NK cells in AML management. We also discuss key obstacles and opportunities, including strategies to boost NK cell activity, counter immune escape, and improve treatment durability. Continued investigation is essential to refine NK cell-based therapies and bring patient-tailored immunotherapeutic options into broader clinical use.</p>","PeriodicalId":50812,"journal":{"name":"American Journal of Clinical Oncology-Cancer Clinical Trials","volume":" ","pages":"247-256"},"PeriodicalIF":1.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145662698","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":"H-Index and Promotion in Academic Radiation Oncology.","authors":"Benjamin K Talom, Christina C Huang, Monique N Westley, Yvonne C Cotterell, Yilun Sun, Reshma Jagsi, Shearwood McClelland","doi":"10.1097/COC.0000000000001264","DOIUrl":"10.1097/COC.0000000000001264","url":null,"abstract":"<p><strong>Objectives: </strong>Academic promotion in radiation oncology is influenced by multiple factors, including scholarly productivity and demographic characteristics. While citation-based metrics such as the h-index are increasingly used as objective measures of academic output, the impact of demographic factors such as sex and underrepresented minority (URM) status remains inadequately defined. This study represents the first evaluation of the predictive value of h-index, sex, and URM status on academic promotion.</p><p><strong>Methods: </strong>A retrospective cohort of 554 radiation oncologists from 51 NCI-designated Comprehensive Cancer Centers, initially identified in 2019 (T1) and re-evaluated in 2023 (T2), was assessed. Academic promotion status, h-index (2019), sex, URM status, and institutional affiliation were recorded. A generalized linear mixed model assessed associations between these variables and promotion status, with significance defined as P <0.05.</p><p><strong>Results: </strong>The cohort included 203 women (36.7%) and 21 URMs (3.8%); overall, 338 (61%) received promotions between T1 and T2. The mean h-index was 12.3 (median=9), with promoted individuals averaging 15.3 versus 10 for those not promoted. A statistically significant association was found between a higher h-index and promotion ( P <0.0001). Further analysis revealed that neither female sex (odds ratio: 1.02, 95% CI: 0.68-1.52; P =0.94) nor URM status (odds ratio: 0.57, 95% CI: 0.19-1.71; P =0.32) was significantly associated with promotion.</p><p><strong>Conclusions: </strong>In the first examination of the impact of h-index on radiation oncology promotion, a higher h-index is a statistically significant predictor of academic promotion among radiation oncologists. Given limited statistical power to detect differences by demographic characteristics and ongoing underrepresentation of certain groups compared with the population, ongoing work to ensure fair access to opportunities for all remains important.</p>","PeriodicalId":50812,"journal":{"name":"American Journal of Clinical Oncology-Cancer Clinical Trials","volume":" ","pages":"243-246"},"PeriodicalIF":1.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145349775","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":"Combination of Ponatinib and Blinatumomab in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia: A Systematic Review and Meta-Analysis.","authors":"Faiza Fatima, Anurag Jha, Ahmed Raza, Fatima Aslam, Aeliya Mirza, Umaima Cheema, Hammad Javaid, Shamikha Cheema, Zaheer Qureshi","doi":"10.1097/COC.0000000000001303","DOIUrl":"https://doi.org/10.1097/COC.0000000000001303","url":null,"abstract":"<p><strong>Objectives: </strong>Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) is traditionally treated with tyrosine kinase inhibitors (TKIs) alongside chemotherapy. Recent studies suggest that combining ponatinib and blinatumomab may offer a highly effective, chemotherapy-free alternative. We performed this study to assess the efficacy and safety of ponatinib combined with blinatumomab in adult patients with Ph+ ALL, across both newly diagnosed and relapsed/refractory settings.</p><p><strong>Methods: </strong>This systematic review and meta-analysis included 8 studies evaluating ponatinib plus blinatumomab in Ph+ ALL. Primary outcomes were overall survival (OS) and event-free survival (EFS). Secondary outcomes included complete molecular response (CMR), relapse rates, hematologic response, and adverse events (AEs). Risk of bias was assessed using the Newcastle-Ottawa Scale. Analyses were conducted using a random-effects model.</p><p><strong>Results: </strong>The pooled OS was 84%, with higher survival in newly diagnosed patients (93%) versus refractory cases (50%). EFS was 64% overall, with 86% in the newly diagnosed subgroup. The pooled CMR rate was 89%, and hematologic response reached 93%. Relapse rates were low, particularly in frontline settings. The pooled AE rate was 17%, decreasing to 11% after sensitivity analysis, indicating a manageable safety profile.</p><p><strong>Conclusions: </strong>Ponatinib combined with blinatumomab demonstrates high efficacy and tolerability as a frontline, chemotherapy-free regimen in Ph+ ALL. These findings support further investigation in larger, randomized trials to confirm long-term outcomes and solidify its place in standard treatment protocols.</p>","PeriodicalId":50812,"journal":{"name":"American Journal of Clinical Oncology-Cancer Clinical Trials","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147788306","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":"Early Detection of Colorectal Cancer Following Changes to Screening Guidelines: A Population-Based Analysis of Adults Aged 45 to 49.","authors":"Boniface Mensah, Albert E Orhin, Natalie A Y Akoto, Seunghee Han, Ramya Vasireddy, Rachel A Boateng, Aida Metri, Kevin Eid, Simardeep Singh, Priyanka Kanth","doi":"10.1097/COC.0000000000001332","DOIUrl":"https://doi.org/10.1097/COC.0000000000001332","url":null,"abstract":"<p><strong>Objectives: </strong>Colorectal cancer (CRC) is the third most common cancer, with millions of new cases and deaths annually. There is an increasing incidence of CRC among patients <50 years, leading to recommendations for CRC screening initiation at age 45 by the American Cancer Society (ACS) in May 2018. However, it is unclear if CRC screening at ages 45 to 49 has increased the detection of early-stage CRC (ES-CRC).</p><p><strong>Methods: </strong>We conducted a retrospective cohort study using the Surveillance, Epidemiology, and End Results (SEER) database from 2015 to 2022, including individuals aged 45 to 49 diagnosed with CRC. The primary outcome was the diagnosis of ES-CRC (stages 1 and 2). The exposure was the time of diagnosis, categorized as preguideline (2015 to 2018) and postguideline (2019 to 2022). Multivariable logistic regression assessed the association between time period and ES-CRC diagnosis, adjusting for demographic and socioeconomic factors.</p><p><strong>Results: </strong>Among 28,532 individuals, 47.0% were diagnosed preguideline and 53.0% postguideline. The proportion of ES-CRC increased significantly postguideline (31.1%) compared with preguideline update (29.8%, P=0.012). Notably, a decline in ES-CRC diagnoses was observed in 2020, likely due to disruptions in cancer screening during the COVID-19 pandemic. The adjusted odds of ES-CRC diagnosis was higher postguideline compared with preguideline update (AOR: 1.068; 95% CI: 1.004-1.137). Subgroup analyses revealed higher odds of ES-CRC diagnosis among females, Asians and Black individuals postguideline update.</p><p><strong>Conclusions: </strong>Following the 2018 ACS guideline change, ES-CRC diagnoses increased among adults aged 45 to 49. These findings suggest the updated screening recommendations may have facilitated earlier detection in this age group.</p>","PeriodicalId":50812,"journal":{"name":"American Journal of Clinical Oncology-Cancer Clinical Trials","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147787627","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":"Efficacy of Fibroblast Growth Factor Receptor Inhibitors (FGFRi) in Locally Advanced or Metastatic Cholangiocarcinoma With FGFR Fusions or Rearrangements: A Meta-Analysis of Clinical Trials.","authors":"Abdul Rehman, Jacopo Lin, Sakshi Arora, Jonathan D Mohnkern, Dhvanit Rajdeep, Rubia A S Lopes Seixas","doi":"10.1097/COC.0000000000001331","DOIUrl":"https://doi.org/10.1097/COC.0000000000001331","url":null,"abstract":"<p><strong>Objectives: </strong>The efficacy of various FGFR inhibitors (FGFRi) has been reported for advanced cholangiocarcinoma patients harboring FGFR fusion genes or rearrangements. However, the pooled efficacy of FGFRi is still lacking.</p><p><strong>Methods: </strong>PubMed, Cochrane, and Embase databases were systematically searched from inception to Jan 2026 for studies evaluating the efficacy of FGFRi in advanced cholangiocarcinoma patients previously treated with chemotherapy. The primary outcomes were the objective response rate, disease control rate, progression-free survival, and overall survival. Secondary outcomes included safety parameters. A proportional meta-analysis was performed using a random-effects model.Heterogeniety was assessed using I2 statistics.</p><p><strong>Results: </strong>Eight studies with 463 patients were included. In the pooled analysis, the objective response rate was 40.4% (n=171/463; 95% CI: 31.64%-49.81%), and the disease control rate was 92.48% (n=368/435; 95% CI: 81.68%-98.72%). Progression-free survival was 64.3% (n=164/255; 95% CI: 58.24%-69.96%) and 37.33% (n=84/255; 95% CI: 31.26%-43.84%) at 6 and 12 months, respectively. Overall survival rates were 91.45% (n=139/152; 95% CI: 85.83%-94.97%) and 70.67% (n=159/225; 95% CI: 64.39%-76.25%) at 6 and 12 months, respectively. The common adverse events were hyperphosphatemia (75.4%), alopecia (44.53%), stomatitis (33.20%), diarrhea (32.15%), fatigue (33.64%), dry eyes (25.20%), nail discolouration (17.38%), ALT elevation (14.53%), and AST elevation (16.93%).</p><p><strong>Conclusions: </strong>Our analysis suggests that FGFR alteration testing and the use of FGFRi as an effective alternative option in advanced cholangiocarcinoma patients previously treated with chemotherapy.</p>","PeriodicalId":50812,"journal":{"name":"American Journal of Clinical Oncology-Cancer Clinical Trials","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147730573","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}