NEJM evidence最新文献

{"title":"Updates in Genetics - A New Review Series.","authors":"Sonja A Rasmussen, C Corey Hardin","doi":"10.1056/EVIDe2500232","DOIUrl":"https://doi.org/10.1056/EVIDe2500232","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 10","pages":"EVIDe2500232"},"PeriodicalIF":0.0,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145126758","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cendakimab in Adults and Adolescents with Eosinophilic Esophagitis.","authors":"Evan S Dellon, Christina M Charriez, Sandra Zhang, Gary W Falk, Salvatore Oliva, Christopher Ma, Jesse Siffledeen, Shauna Schroeder, Hamish Philpott, Tim Vanuytsel, Yasuhiko Abe, Kexuan Li, Carla L Zema, Ashwini Venkatasamy, Anusha K Yeshokumar, Young S Oh, Alain Schoepfer","doi":"10.1056/EVIDoa2500095","DOIUrl":"10.1056/EVIDoa2500095","url":null,"abstract":"<p><strong>Background: </strong>Eosinophilic esophagitis (EoE) is a chronic type 2 inflammatory esophageal disease driven by interleukin 13 (IL-13). Cendakimab, a high-affinity monoclonal antibody, binds IL-13, blocking interaction with receptors IL-13 receptor alpha 1 and IL-13 receptor alpha 2.</p><p><strong>Methods: </strong>In this phase 3 trial, we randomly assigned patients with EoE 12 to 75 years of age to cendakimab 360 mg once weekly for 48 weeks (QW/QW), cendakimab 360 mg once weekly (QW) for 24 weeks, then 360 mg every other week for weeks 24 to 48 (QW/Q2W), or placebo for 48 weeks. Coprimary end points at week 24 were change from baseline in dysphagia days, measured by a validated patient-reported modified Daily Symptom Diary, and histologic response (peak esophageal eosinophil count ≤6 per high-power field). Secondary end points included endoscopic features and safety. Cendakimab QW/QW and QW/Q2W regimens were assessed as a single treatment group in the analyses from week 0 to week 24 (cendakimab QW) and as separate treatment groups versus placebo in the analyses from weeks 24 to 48.</p><p><strong>Results: </strong>Among 430 patients randomly assigned to cendakimab (QW/QW, n=143; QW/Q2W, n=143) or placebo (n=144), reduction from baseline in dysphagia days at week 24 was significantly greater with cendakimab QW versus placebo (least-squares mean change [standard error], -6.1 [0.3] vs. -4.2 [0.4] days; <i>P</i><0.001). Histologic response at week 24 was achieved in 28.6% of patients with cendakimab QW versus 2.2% with placebo (<i>P</i><0.001). Cendakimab improved endoscopic severity from baseline to week 24, compared with placebo (least-squares mean change [standard error] -5.2 [0.24] points vs. -1.2 [0.34] points). Efficacy was maintained at week 48. Adverse events occurred in 83.8%, QW/QW, and 84.6%, QW/Q2W, of patients with cendakimab and 73.4% with placebo through week 48.</p><p><strong>Conclusions: </strong>Cendakimab demonstrated statistically significant improvements in symptoms, histologic response, and endoscopic features of EoE versus placebo; the adverse-event and side-effect profile was not dose limiting. (Funded by Bristol Myers Squibb; ClinicalTrials.gov number, NCT04753697.).</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 10","pages":"EVIDoa2500095"},"PeriodicalIF":0.0,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145126716","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Live-Attenuated Intranasal RSV Vaccine in Infants and Toddlers.","authors":"Olubukola T Idoko, Sergio L Vargas, Agustin Bueso, Doris Rivera, Henderson Edward, Michael Simon, Peyman Banooni, Shaun Berger, Stephane Janicot, Camille Vercasson, Sophie Pallardy, Rapi Nteene, Haritha Adhikarla, Eric Gerchman, Monica Gasparotto, Scott Gallichan, Enrique Rivas, Ursula J Buchholz, Peter L Collins, Sanie Sesay, Sanjay Gurunathan, Iris De Bruijn, Mandeep S Dhingra","doi":"10.1056/EVIDoa2500026","DOIUrl":"10.1056/EVIDoa2500026","url":null,"abstract":"<p><strong>Background: </strong>RSV/ΔNS2/Δ1313/I1314L (RSVt) (Sanofi) is a candidate live-attenuated intranasal respiratory syncytial virus (RSV) vaccine for infants and toddlers.</p><p><strong>Methods: </strong>This phase I/II randomized clinical trial, conducted at sites in the United States (N=22), Chile (N=2), and Honduras (N=2), enrolled participants 6-18 months of age in four cohorts. In cohort 4, the primary cohort reported in this article, participants were randomly assigned to receive vaccinations on days 0 and 56 of either low-dose (LD) RSVt, high-dose (HD) RSVt, or placebo. Primary safety end points included: unsolicited systemic adverse events 30 minutes post vaccination, and solicited site and systemic reactions 28 days post vaccination. The primary immunogenicity end points were the geometric mean titers of RSV A serum neutralizing antibody after vaccinations 1 (day 56) and 2 (day 84) among RSV-naive participants at baseline.</p><p><strong>Results: </strong>Among 180 participants (LD, N=61; HD, N=58; placebo, N=61), 115 were RSV-naive at baseline (LD, N=45; HD, N=32; placebo, N=38). No unsolicited systemic adverse events occurred within 30 minutes post vaccination in the LD or HD groups. Solicited site reactions were reported by 83.1%, 74.5%, and 68.9% of participants post vaccination 1, and 75.6%, 77.8%, and 55.6% post vaccination 2, in the LD, HD, and placebo groups, respectively. Solicited systemic reactions were reported by 79.7%, 73.2%, and 77.0% of participants post vaccination 1, and 66.7%, 66.7%, and 48.1% post vaccination 2, in the LD, HD, and placebo groups, respectively. Neutralizing antibody titers among RSV-naive participants were 83.7 (95% confidence interval (CI), 49.5 to 142.0), 79.4 (95% CI, 47.2 to 134.0), and 20.6 (95% CI, 16.4 to 25.9) post vaccination 1, and 142.0 (95% CI, 86.4 to 232.2), 107.0 (95% CI, 70.0 to 163.0), and 26.3 (95% CI, 18.8 to 37.0) post vaccination 2, in the LD, HD, and placebo groups, respectively.</p><p><strong>Conclusions: </strong>The RSVt vaccine demonstrated promising immunogenicity profiles at both LD and HD strengths among infants and toddlers, without identified safety concerns. (Funded by Sanofi; ClinicalTrials.gov number, NCT04491877).</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 9","pages":"EVIDoa2500026"},"PeriodicalIF":0.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144981654","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"In-Hospital Mortality in Hemorrhagic Myocardial Infarction.","authors":"Keyur P Vora, Ankur Kalra, Chirag D Shah, Kinjal Bhatt, Andreas Kumar, Tejas Pandya, Vishal Poptani, Shing Fai Chan, Dhirendra Singh, Nithya Jambunathan, Ramesh Subramanian, Khalid Youssef, Saravanan Kanakasabai, Robert Finney, Ankit Desai, Rolf P Kreutz, Richard J Kovacs, Subha V Raman, Deepak L Bhatt, Rohan Dharmakumar","doi":"10.1056/EVIDoa2400294","DOIUrl":"10.1056/EVIDoa2400294","url":null,"abstract":"<p><strong>Background: </strong>Advances in acute ST-elevation myocardial infarction (STEMI) care have substantially decreased in-hospital mortality; however, in absolute terms, in-hospital mortality still remains high. Reperfusion injury, particularly intramyocardial hemorrhage following primary percutaneous coronary intervention (PCI), is a major predictor of adverse cardiovascular outcomes in the long term, but whether it contributes to in-hospital mortality is not known.</p><p><strong>Methods: </strong>We performed a multicenter study to investigate the use of post-PCI high-sensitivity cardiac troponin I (hs-cTn-I) as a diagnostic tool to identify hemorrhagic myocardial infarction (MI) by determining hourly hs-cTn-I thresholds (every hour up to 12 hours, and at 16, 20, 24, and 48 hours post-PCI). We then investigated the relationship between patients classified as having hemorrhagic MI based on post-PCI hs-cTn-I cutoff values and in-hospital mortality using STEMI registries containing information about 6180 patients across seven hospitals in a single large health system in the United States.</p><p><strong>Results: </strong>We enrolled 154 patients in a discovery cohort and 53 patients in a validation cohort. Hemorrhagic MI was diagnosed by cardiac magnetic resonance imaging. Post-PCI hs-cTn-I cutoff values for the determination of hemorrhagic MI were time dependent, with a sensitivity greater than 0.91, a specificity greater than 0.86, and an area under the curve (AUC) greater than 0.92 over the first 10 hours post-PCI, decreasing to a sensitivity greater than>0.84, a specificity greater than 0.80, and an AUC greater than 0.84 thereafter. The STEMI registry analysis demonstrated that patients classified as having hemorrhagic MI based on hs-cTn-I cutoff values had a 2.81-fold greater risk for in-hospital mortality than those classified as having had nonhemorrhagic MI (adjusted odds ratio, 2.81; 95% confidence interval, 2.17 to 3.64).</p><p><strong>Conclusions: </strong>Post-PCI troponin kinetics may have the potential to diagnose hemorrhagic MI, which was associated with in-hospital mortality. (Funded by the National Institutes of Health National Heart, Lung, and Blood Institute (grant numbers HL133407, HL136578, and HL147133) and others; ClinicalTrials.gov ID, NCT05872308).</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 9","pages":"EVIDoa2400294"},"PeriodicalIF":0.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144981710","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Should Patients with Prior Coronary Artery Bypass Grafting Who Present with Non-ST-Elevation Acute Coronary Syndromes Undergo Routine Invasive Angiography?","authors":"Matthew Kelham, Anthony Mathur, Daniel A Jones","doi":"10.1056/EVIDtt2500083","DOIUrl":"https://doi.org/10.1056/EVIDtt2500083","url":null,"abstract":"<p><p>AbstractPatients who have previously undergone coronary artery bypass grafting (CABG) may present with non-ST-elevation acute coronary syndromes (NSTE-ACSs). In this setting, guidelines recommend routine invasive angiography. However, patients with CABG were often excluded from key trials that informed contemporary guidelines and invasive angiography is known to be of higher risk in these patients. In this article, the authors review the evidence and propose a trial to address the question, \"Should patients with prior CABG who present with NSTE-ACSs routinely undergo invasive angiography?\"</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 9","pages":"EVIDtt2500083"},"PeriodicalIF":0.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144981665","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Participants as Partners in Decentralized Clinical Trials.","authors":"Paul A Harris, Alex C Cheng","doi":"10.1056/EVIDctw2500188","DOIUrl":"10.1056/EVIDctw2500188","url":null,"abstract":"<p><p>AbstractDecentralized clinical trials (DCTs), meaning those trials in which activities are conducted outside of hospital or clinic settings, offer many advantages over traditional clinical trials and, when properly operationalized, provide unique benefits to researchers, participants, and society. The ability of individuals to participate remotely from their homes adds convenience, but, more importantly, democratizes access to trials that would otherwise be limited by geographic constraints. As personal interactions with participants are diminished in DCTs, trialists must be intentional when strategizing recruitment, retention, and return of value for participants. Engaging clinical trial participants as partners in DCTs requires researchers to consider participant priorities at each stage of the trial. This article highlights technical and procedural considerations needed to support DCTs and identifies areas for trial innovation. By making DCTs and technology platforms participant centric and continuing to advance opportunities for innovation, researchers may accelerate scientific discovery, help participants feel their contributions are meaningful, and foster long-term trust in the biomedical research process.See also in <i>NEJM</i>: As-Needed Albuterol-Budesonide in Mild Asthma See also in <i>NEJM</i>: Your Combination Asthma Inhaler - Don't Leave Home without It!</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":" ","pages":"EVIDctw2500188"},"PeriodicalIF":0.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144602415","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Search for a Respiratory Syncytial Virus Vaccine.","authors":"Peter F Wright","doi":"10.1056/EVIDe2500194","DOIUrl":"https://doi.org/10.1056/EVIDe2500194","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 9","pages":"EVIDe2500194"},"PeriodicalIF":0.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144981723","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An Individualized Prediction Model for Early-Stage Classic Hodgkin's Lymphoma.","authors":"Angie Mae Rodday, Andrew M Evens, Matthew J Maurer, Jenica N Upshaw, Nicholas Counsell, Sara Rossetti, Cheryl Chang, Zhu Cui, Qingyan Xiang, Raphael Mwangi, Ranjana Advani, Marc Andre, Andrea Gallamini, Annette E Hay, David C Hodgson, Richard T Hoppe, Martin Hutchings, Peter Johnson, Eric Mou, Stephen Opat, John Raemaekers, Kerry J Savage, Susan K Parsons, John Radford","doi":"10.1056/EVIDoa2500115","DOIUrl":"10.1056/EVIDoa2500115","url":null,"abstract":"<p><strong>Background: </strong>A predictive model for early-stage classic Hodgkin's lymphoma (cHL) does not exist. Leveraging patient-level data from large clinical trials and registries, we developed and validated a model that we term the Early-Stage cHL International Prognostication Index (E-HIPI) to predict 2-year progression-free survival (PFS).</p><p><strong>Methods: </strong>We developed the model using the Transparent Reporting of a Multivariable Prediction Model for Individual Prognosis or Diagnosis (TRIPOD) guidelines in 3000 adults with newly diagnosed early-stage cHL from four international phase III clinical trials conducted from 1994 to 2011. External validation was performed in two cohorts, totaling 2360 treated patients from five international cHL registries (1996 to 2019). Two-year PFS was estimated using a Cox model with pretreatment variables selected using backward elimination. Internal validation corrected for overfitting. External validation assessed discrimination and calibration. The final model was also compared against European Organisation for Research and Treatment of Cancer (EORTC) favorable or unfavorable status.</p><p><strong>Results: </strong>The median age in the development cohort was 31.2 years; 77.4% had stage II disease. The estimated 2-year PFS was 93.7%. Final variables retained in the model were sex and continuous values of maximum tumor diameter (MTD), and levels of hemoglobin and albumin. The optimism-corrected C statistic in the development cohort was 0.63 (95% confidence interval, 0.60 to 0.69). Two-year PFS was lower in the validation cohorts 1 (90.3%) and 2 (91.6%). In validation cohort 1, the C statistic was 0.63 and the calibration slope was near 1, but overall calibration indicated underprediction, which improved on updating the intercept. The performance was similar in validation cohort 2. In addition, higher-risk E-HIPI scores were associated with worse outcomes in both the EORTC unfavorable and favorable subgroups. When included altogether in one Cox model, the E-HIPI was associated with PFS, whereas EORTC favorable or unfavorable status was not. Online risk calculators were developed (https://rtools.mayo.edu/holistic_ehipi/).</p><p><strong>Conclusions: </strong>Utilizing objective, continuous, and readily available variables, we developed and validated a new prediction model for early-stage cHL. Male sex, lower hemoglobin or albumin levels, and higher MTDs were associated with worse PFS. (Funded by the National Cancer Institute; grant number, NCI R01 CA 262265-04.).</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":" ","pages":"EVIDoa2500115"},"PeriodicalIF":0.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12498259/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144328016","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A 74-Year-Old Woman with Leg Weakness.","authors":"Florence Morriello, Justin Chiu","doi":"10.1056/EVIDmr2400472","DOIUrl":"https://doi.org/10.1056/EVIDmr2400472","url":null,"abstract":"<p><p>AbstractMorning Report is a time-honored tradition where physicians-in-training present cases to their colleagues and clinical experts to collaboratively examine an interesting patient's presentation. The Morning Report section seeks to carry on this tradition by presenting a patient's chief concern and story, inviting the reader to develop a differential diagnosis and discover the diagnosis alongside the authors of the case. This report examines the story of a 74-year-old woman who presents to the emergency department with a 3-month history of bilateral leg weakness and paresthesia. Using questions, physical examination, and testing, an illness script for the presentation emerges. As the clinical course progresses, the differential is refined until a final diagnosis is made.</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 9","pages":"EVIDmr2400472"},"PeriodicalIF":0.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144981651","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pemigatinib for Myeloid/Lymphoid Neoplasms with <i>FGFR1</i> Rearrangement.","authors":"Srdan Verstovsek, Jean-Jacques Kiladjian, Alessandro M Vannucchi, Jay L Patel, Alessandro Rambaldi, William E Shomali, Stephen T Oh, Kensuke Usuki, Claire N Harrison, Ellen K Ritchie, Luke P Akard, Juan Carlos Hernández-Boluda, Françoise Huguet, Philomena Colucci, Huiling Zhen, Natalia Oliveira, Aidan Gilmartin, Cheryl Langford, Tracy I George, Andreas Reiter, Jason Gotlib","doi":"10.1056/EVIDoa2500017","DOIUrl":"10.1056/EVIDoa2500017","url":null,"abstract":"<p><strong>Background: </strong>Myeloid/lymphoid neoplasms with fibroblast growth factor receptor 1 rearrangements (MLN-<i>FGFR1</i>) are associated with poor prognosis. They are caused by chromosome 8p11 rearrangements that result in <i>FGFR1</i> fusion genes and constitutive FGFR1 activation. We report on a phase 2 study, in which there were no concurrent control patients, termed FIGHT-203, in which we evaluated the FGFR1-3 inhibitor, pemigatinib, for the treatment of MLN-<i>FGFR1</i>.</p><p><strong>Methods: </strong>We assigned eligible patients to receive oral pemigatinib 13.5 mg once daily (2 weeks on followed by 1 week off or continuously). End points included complete response rate (primary) and complete cytogenetic response rate. Responses were assessed locally by investigators per protocol-defined criteria and were retrospectively adjudicated by a central review committee using criteria defined by the committee.</p><p><strong>Results: </strong>Of 47 treated patients (safety population), 45 had confirmed <i>FGFR1</i> rearrangement and were analyzed for efficacy; of these patients, 24 (53%) were in the chronic phase of illness; 18 (40%) were in blast phase; and three previously treated patients (7%) exhibited the rearrangement without morphologic bone marrow or extramedullary involvement. The overall complete response rate, as determined by central review, was 74% (31 out of 42); this occurred in 96% (23 out of 24) of patients in chronic phase and 44% (8 out of 18) of patients in blast phase. A complete cytogenetic response was observed in 73% (33 out of 45) of patients overall, consisting of 88% (21 out of 24) of patients in chronic phase, 50% (9 out of 18) of patients in blast phase, and all three patients who had a rearrangement only. The median duration of complete response was not reached (95% confidence interval, 27.9 months to not reached). The most common any-grade treatment-emergent adverse event was hyperphosphatemia (76%); the most common grade-3-and-over event was stomatitis (19%). Pemigatinib discontinuation, interruption, and dose reduction occurred in 5 (11%), 30 (64%), and 28 (60%) patients, respectively.</p><p><strong>Conclusions: </strong>In our study, pemigatinib manifested near complete efficacy in chronic-phase patients with MLN-<i>FGFR1</i>, while the complete response rate was close to 50% in blast-phase patients. Toxicities were manageable with dose modifications. (Funded by Incyte Corporation; FIGHT-203 ClinicalTrials.gov number, NCT03011372.).</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 9","pages":"EVIDoa2500017"},"PeriodicalIF":0.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144981720","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}