Journal of Clinical Oncology最新文献

{"title":"Reply to: Importance of Defining Recurrence in Hepatocellular Carcinoma: Local or Both Local and Other Intrahepatic Recurrences?","authors":"Mian Xi, Yizhen Fu, Yaojun Zhang","doi":"10.1200/JCO-25-00327","DOIUrl":"10.1200/JCO-25-00327","url":null,"abstract":"","PeriodicalId":15384,"journal":{"name":"Journal of Clinical Oncology","volume":" ","pages":"2028-2029"},"PeriodicalIF":42.1,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143710054","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Novel Postneoadjuvant Prognostic Breast Cancer Staging System.","authors":"David J Winchester, Lavisha Singh, Stephen B Edge, Kimberly H Allison, William E Barlow, Veerle Bossuyt, Mariana Chavez-MacGregor, Emily F Conant, James L Connolly, Jennifer F De Los Santos, Daniel F Hayes, Nola M Hylton, Elizabeth A Mittendorf, Jennifer K Plichta, Elena Provenzano, Kilian E Salerno, Priyanka Sharma, W Fraser Symmans, Donald Weaver, Gabriel N Hortobagyi","doi":"10.1200/JCO-24-01739","DOIUrl":"10.1200/JCO-24-01739","url":null,"abstract":"<p><strong>Purpose: </strong>Prognostic staging after neoadjuvant chemotherapy (NACT) is not included in American Joint Commission on Cancer (AJCC) staging. This study addressed this deficiency by including responses to therapy with standardized staging variables in a validated prognostic staging system for patients treated with NACT.</p><p><strong>Methods: </strong>The National Cancer Database was queried to identify 140,605 patients treated with NACT between 2010 and 2018. Three response categories (no response, partial response, and complete response [pCR]) were created on the basis of comparison of clinical and post-NACT pathologic staging. Univariate and multivariate analyses of clinical stage, estrogen receptor, progesterone receptor, human epidermal growth factor receptor 2 (HER2), and grade were analyzed for each category. Predictive models for each response category were validated using the bootstrap technique. Calibration plots compared predicted and observed 3-year survival probabilities in the training and validation data sets.</p><p><strong>Results: </strong>Each validated model demonstrated statistically significant survival differences in the postneoadjuvant prognostic stage assignment. Of all patients with a pCR, 94.2% were assigned to postneoadjuvant ypStage I compared with 35.5% of patients with no response. Advancing clinical stage had a progressive but small impact on overall survival (OS) with pCR (high-grade, triple-negative breast cancer [TNBC]: cStage I, 97% <i>v</i> cStage IIIB/IIIC, 91%; grade 2 luminal A: 97% <i>v</i> 91%) but was associated with a profound decrease in OS with no response for TNBC or HER2+ disease (high-grade TNBC 89% <i>v</i> 50%) and less profound for grade 2 luminal A disease with no response (97% <i>v</i> 81%).</p><p><strong>Conclusion: </strong>We present a novel, validated prognostic staging system that predicts OS according to the response to NACT. These data will provide AJCC stage assignments for a growing proportion of patients treated with NACT.</p>","PeriodicalId":15384,"journal":{"name":"Journal of Clinical Oncology","volume":" ","pages":"1948-1960"},"PeriodicalIF":42.1,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144025830","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Follow-Up of E3311, an ECOG-ACRIN Cancer Research Group Phase II Trial of Transoral Surgery and Risk-Based Adjuvant Treatment in Human Papillomavirus-Initiated Oropharynx Cancer.","authors":"Barbara Burtness, Yael Flamand, Harry Quon, Gregory S Weinstein, Ranee Mehra, Joaquin J Garcia, Seungwon Kim, Bert W O'Malley, Enver Ozer, Chukwuemeka Ikpeazu, Wayne M Koch, Neil D Gross, R Bryan Bell, Mihir Patel, Miriam N Lango, Luc G Morris, Russell Smith, Daniel Karakla, Jeremy D Richmon, Floyd C Holsinger, Robert L Ferris","doi":"10.1200/JCO-24-02550","DOIUrl":"https://doi.org/10.1200/JCO-24-02550","url":null,"abstract":"<p><p>This phase II trial of transoral surgery (TOS) with deintensified postoperative management in human papillomavirus (HPV)-associated oropharynx cancer (OPC) enrolled patients with resectable cT1-2 stage III/IV American Joint Committee on Cancer (AJCC) seventh edition p16+ OPC without matted neck nodes. Those with clear margins, 0-1 + nodes (LN), and no extranodal extension (ENE) were observed (arm A); those with clear margins, 2-4 + LN, or ENE ≤1 mm were randomly assigned to 50 Gy (arm B) or 60 Gy (arm C); and those with involved margins, >4 + LN, or >1 mm ENE received weekly cisplatin and 60-66 Gy (arm D). Among 359 evaluable patients, the 54-month progression-free (PFS) and overall survival (OS) were 90.6% (90% CI, 87.2% to 93.1%) and 95.3% (93.0% to 96.9%), respectively. The 54-month PFS by arm was A 93.2% (79.6% to 97.8%; all four recurrences among N1 patients), B 94.9% (89.7% to 97.5%), C 90.2% (82.7% to 94.6%), and D 85.5% (77.5% to 90.8%). The 54-month OS by arm was A 97.1% (85.7% to 99.4%), B 97.9% (93.5% to 99.3%), C 95.1% (90.1% to 97.6%), and D 92.5% (86.9% to 95.7%). PFS or OS did not differ by primary site or smoking history. TOS and neck dissection with deintensified postoperative management results in outstanding 54-month PFS and OS. Among patients with favorable pathologic characteristics, those with N1 disease are at risk of late recurrence without radiation.</p>","PeriodicalId":15384,"journal":{"name":"Journal of Clinical Oncology","volume":" ","pages":"JCO2402550"},"PeriodicalIF":42.1,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144266280","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Reproducibility of Clinically Meaningful Change in Physical Capacity Tested by Online 6-Minute Walk Test.","authors":"Shaza Abo, Linda Denehy","doi":"10.1200/JCO-25-00023","DOIUrl":"10.1200/JCO-25-00023","url":null,"abstract":"","PeriodicalId":15384,"journal":{"name":"Journal of Clinical Oncology","volume":" ","pages":"2030-2031"},"PeriodicalIF":42.1,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143674032","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sequential Infusion of Mesenchymal Stem Cell for Graft-Versus-Host Disease Prevention in Haploidentical Hematopoietic Stem Cell Transplantation: An Open-Label, Multicenter, Randomized Controlled Clinical Trial.","authors":"Han Yao, Ruihao Huang, Haixia Fu, Ren Lin, Yanqi Zhang, Yimei Feng, Yu Wang, Ting Chen, Xiaoqi Wang, Lidan Zhu, Jia Liu, Yuqing Liu, Lu Zhao, Lu Wang, Peiyan Kong, Qin Wen, Cheng Zhang, Li Gao, Lei Gao, Qifa Liu, Xiaohui Zhang, Xiaojun Huang, Xi Zhang","doi":"10.1200/JCO-24-02119","DOIUrl":"10.1200/JCO-24-02119","url":null,"abstract":"<p><strong>Purpose: </strong>The aim of this open-label, multicenter, randomized controlled trial was to determine the efficacy and safety of sequential umbilical cord-derived mesenchymal stem cell (UC-MSC) infusion for graft-versus-host disease (GVHD) prevention within 3 months of haploidentical hematopoietic stem cell transplantation (haplo-HSCT).</p><p><strong>Methods: </strong>This open-label study evaluated UC-MSC infusion (administer 1 × 10⁶/kg 4 hours before the commencement of day 0, once weekly for the first month after transplantation, once every 2 weeks for the second month, and once during the third month, totaling eight doses). The primary end point was the 2-year cumulative incidence of severe chronic GVHD (cGVHD).</p><p><strong>Results: </strong>In the primary analysis, 192 qualified participants between age 18 and 60 years with haplo-HSCT in three transplant centers in China were enrolled and randomly assigned to the MSC and control groups. In the primary analysis, the estimated 2-year cumulative incidence of severe cGVHD and all grades of cGVHD was lower in the MSC group than in the control group (<i>P</i> = .033 and <i>P</i> = .022). The cumulative incidence of grade 1 to 4, 2 to 4, and 3 to 4 acute GVHD (aGVHD) in patients in the MSC group significantly decreased (all <i>P</i> < .001). The 3-year GVHD-free and relapse-free survival (GRFS) rate in the MSC group was 62.4%, which was significantly higher than that in the control group (32.0%, hazard ratio [HR], 0.34, <i>P</i> < .001). MSC infusion did not influence the cumulative incidence of relapse (<i>P =</i> .34) and nonrelapse mortality (<i>P =</i> .45).</p><p><strong>Conclusion: </strong>Our findings suggest that sequential infusion of MSCs within 3 months after haplo-HSCT significantly reduced both the incidence and severity of cGVHD and aGVHD, manifesting as a better GRFS rate for patients.</p>","PeriodicalId":15384,"journal":{"name":"Journal of Clinical Oncology","volume":" ","pages":"1997-2006"},"PeriodicalIF":42.1,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143982021","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Writing a Medical Memoir: Lessons From a Long, Steep Road.","authors":"David I Marks","doi":"10.1200/JCO-24-02472","DOIUrl":"10.1200/JCO-24-02472","url":null,"abstract":"<p><p>The purpose of this essay is to take readers of the Journal on my challenging journey of writing a memoir describing my patients and career.</p>","PeriodicalId":15384,"journal":{"name":"Journal of Clinical Oncology","volume":" ","pages":"2024-2026"},"PeriodicalIF":42.1,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144020903","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"International Myeloma Society/International Myeloma Working Group Consensus Recommendations on the Definition of High-Risk Multiple Myeloma.","authors":"Hervé Avet-Loiseau, Faith E Davies, Mehmet K Samur, Jill Corre, Mattia D'Agostino, Martin F Kaiser, Marc S Raab, Niels Weinhold, Norma C Gutierrez, S Bruno Paiva, Paola Neri, Katja Weisel, Francesco Maura, Brian A Walker, Mark Bustoros, A Keith Stewart, Saad Z Usmani, Jens Hillengass, Wee Joo Chng, Jonathan J Keats, Joaquin Martinez-Lopez, Adam S Sperling, Cyrille Touzeau, Fenghuang Zhan, Noopur S Raje, Michele Cavo, Niccolò Bolli, Irene M Ghobrial, Madhav V Dhodapkar, Sundar Jagannath, Andrew Spencer, Samir Parekh, Nizar J Bahlis, Sagar Lonial, Pieter Sonneveld, Leif Bergsagel, Robert Z Orlowski, Gareth Morgan, María Victoria Mateos, S Vincent Rajkumar, Jesus F San Miguel, Kenneth C Anderson, Philippe Moreau, Shaji Kumar, Felipe Prósper, Nikhil C Munshi","doi":"10.1200/JCO-24-01893","DOIUrl":"https://doi.org/10.1200/JCO-24-01893","url":null,"abstract":"<p><p>Despite significant improvements in survival of patients with multiple myeloma (MM), outcomes remain heterogeneous, and a significant proportion of patients experience suboptimal outcomes. Importantly, traditional prognostic factors based on data from patients treated with older therapies no longer capture prognosis accurately in the contemporary era of novel triplet or quadruplet therapies. Therefore, risk stratification requires refinement in the context of available and investigational treatment options in routine practice and clinical trials, respectively. The current identification of high-risk MM (HRMM) in routine practice is based on the Revised International Staging System, which stratifies patients using a combination of widely available serum biomarkers and chromosomal abnormalities assessed via fluorescence in situ hybridization. In recent years, a substantial body of evidence concerning additional clinical, biological, and molecular/genomic prognostic factors has accumulated, along with new MM risk stratification tools and consensus reports. The International Myeloma Society, along with the International Myeloma Working Group, convened an Expert Panel with the primary aim of revisiting the definition of HRMM and formulating a practical and data-driven consensus definition, based on new evidence from molecular/genomic assays, updated clinical data, and contemporary risk stratification concepts. The Panel proposes the following Consensus Genomic Staging (CGS) of HRMM which relies upon the presence of at least one of these abnormalities: (1) del(17p), with a cutoff of >20% clonal fraction, and/or <i>TP53</i> mutation; (2) an IgH translocation including t(4;14), t(14;16), or t(14;20) along with 1q+ and/or del(1p32); (3) monoallelic del(1p32) along with 1q+ or biallelic del(1p32); or (4) β2 microglobulin ≥5.5 mg/L with normal creatinine (<1.2 mg/dL).</p>","PeriodicalId":15384,"journal":{"name":"Journal of Clinical Oncology","volume":" ","pages":"JCO2401893"},"PeriodicalIF":42.1,"publicationDate":"2025-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144258178","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Phase III Trial of Pirtobrutinib Versus Idelalisib/Rituximab or Bendamustine/Rituximab in Covalent Bruton Tyrosine Kinase Inhibitor-Pretreated Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (BRUIN CLL-321).","authors":"Jeff P Sharman, Talha Munir, Sebastian Grosicki, Lindsey E Roeker, John M Burke, Christine I Chen, Norbert Grzasko, George Follows, Zoltán Mátrai, Alessandro Sanna, Lugui Qiu, Ru Feng, Vu Minh Hua, Wojciech Jurczak, Matthias Ritgen, Shuhua Yi, Francesc Bosch, Catherine C Coombs, Katherine Bao, Vishalkumar Patel, Bin Liu, Livia Compte, Ananya Guntur, Denise Y Wang, Marisa Hill, Ching Ching Leow, Paolo Ghia, Paul M Barr","doi":"10.1200/JCO-25-00166","DOIUrl":"https://doi.org/10.1200/JCO-25-00166","url":null,"abstract":"<p><strong>Purpose: </strong>Pirtobrutinib, a noncovalent, Bruton tyrosine kinase inhibitor (BTKi), has shown clinical efficacy and a favorable safety profile. BRUIN CLL-321 was an open-label, randomized phase III study conducted exclusively in patients with R/R chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) previously treated with cBTKi, and compared pirtobrutinib with investigator's choice (IC) of idelalisib/rituximab (IdelaR) or bendamustine/rituximab (BR).</p><p><strong>Methods: </strong>Patients were randomly assigned 1:1 to receive pirtobrutinib (200 mg once daily) or IC of IdelaR or BR, and were stratified by previous use of venetoclax and del(17p). The primary end point was independent review committee-assessed progression-free survival (PFS). Secondary end points included time to next treatment or death (TTNT), overall survival (OS), and safety. The primary PFS end point was met at the time of the primary analysis (August 29, 2023), and updated results are reported from the final OS analysis (August 29, 2024).</p><p><strong>Results: </strong>A total of 238 patients were randomly assigned to receive pirtobrutinib (n = 119) or IC (n = 119; IdelaR [n = 82], BR [n = 37]). The PFS hazard ratio (HR) was 0.54 ([95% CI, 0.39 to 0.75]; <i>P</i> = .0002), with a median PFS of 14 months (95% CI, 11.2 to 16.6) in the pirtobrutinib group and 8.7 months (95% CI, 8.1 to 10.4) with IC. The unadjusted OS HR was 1.09 ([95% CI, 0.68 to 1.75]; <i>P</i> = .7202), and 18-month OS rate was 73.4% (95% CI, 63.9 to 80.7) in the pirtobrutinib group and 70.8% (95% CI, 60.9 to 78.7) with IC. Median TTNT was 24 months (95% CI, 17.8 to 29.7) with pirtobrutinib versus 10.9 months (95% CI, 8.7 to 12.5) with IC (HR, 0.37 [95% CI, 0.25 to 0.52]). At a median follow-up of 17.2 months, grade ≥3 treatment-emergent adverse events (AEs) were lower with pirtobrutinib (57.7%) than IC (73.4%). Treatment discontinuation due to AE occurred in 20 (17.2%) patients receiving pirtobrutinib and 38 (34.9%) patients receiving IC.</p><p><strong>Conclusion: </strong>Pirtobrutinib improved PFS and TTNT, and demonstrated favorable tolerability, versus IdelaR/BR in exclusively cBTKi pretreated patients with CLL/SLL.</p>","PeriodicalId":15384,"journal":{"name":"Journal of Clinical Oncology","volume":" ","pages":"JCO2500166"},"PeriodicalIF":42.1,"publicationDate":"2025-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248139","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Novel Systemic Anticancer Treatments and Health Services Use at the End of Life Among Adults With Cancer.","authors":"Javaid Iqbal, Rahim Moineddin, Kieran L Quinn, Christopher M Booth, Craig C Earle, Stephanie Lheureux, Robert Grant, Jenny Lau, Lisa W Le, Peter Tanuseputro, James Downar, Gary Rodin, Hsien Seow, Jillian Tsai, Robert A Fowler, Breffni Hannon, Monika K Krzyzanowska, Camilla Zimmermann","doi":"10.1200/JCO-24-02816","DOIUrl":"https://doi.org/10.1200/JCO-24-02816","url":null,"abstract":"<p><strong>Purpose: </strong>Use of chemotherapy at the end of life (EOL) is discouraged, but evidence to guide decisions on the use of novel systemic anticancer treatment (SACT) agents is lacking. We examined trends of use among SACT types and association with health services use at the EOL.</p><p><strong>Materials and methods: </strong>We analyzed Canadian Ontario Cancer Registry data for adults diagnosed with solid tumors or hematologic malignancies within 5 years of death who received SACT between March 2015 and March 2021. Receipt of SACT in the last 30 days of life was categorized as chemotherapy alone, chemotherapy and immunotherapy, immunotherapy alone, and targeted therapy alone. Outcomes included high health services use, including multiple (≥2) emergency department (ED) visits, multiple (≥2) hospitalizations, or any (≥1) intensive care unit admission, and hospital deaths. Segmented linear regression estimated monthly trends; multivariable logistic regression estimated adjusted odds ratios (aORs) of outcomes for various SACT types.</p><p><strong>Results: </strong>Among 68,963 patients, 18,337 (26.6%) received SACT at the EOL. From March 2015 to March 2020, use of SACT at the EOL increased (0.072% per month; <i>P</i> < .001), mainly driven by increased use of immunotherapy alone (0.064% per month; <i>P</i> < .001). Adjusted odds of high health services use and hospital death were more than two-fold greater among patients receiving SACT at the EOL (vs. none); individual aORs of high health services use and hospital death were 2.20 and 2.72 for chemotherapy alone, 2.36 and 3.10 for chemotherapy and immunotherapy, 1.92 and 2.27 for immunotherapy alone, and 1.75 and 2.37 for targeted therapy alone, respectively.</p><p><strong>Conclusion: </strong>Use of SACT at the EOL increased significantly over time, driven by increased use of immunotherapy. SACT use at the EOL, regardless of its type, was associated with high health services use and hospital death. Guidelines on the use of SACT at the EOL should include novel cancer treatments.</p>","PeriodicalId":15384,"journal":{"name":"Journal of Clinical Oncology","volume":" ","pages":"JCO2402816"},"PeriodicalIF":42.1,"publicationDate":"2025-06-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144225601","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term (≥5-Year) Remission and Survival After Treatment With Ciltacabtagene Autoleucel in CARTITUDE-1 Patients With Relapsed/Refractory Multiple Myeloma.","authors":"Sundar Jagannath, Thomas G Martin, Yi Lin, Adam D Cohen, Noopur Raje, Myo Htut, Abhinav Deol, Mounzer Agha, Jesus G Berdeja, Alexander M Lesokhin, Jessica J Liegel, Adriana Rossi, Alex Lieberman-Cribbin, Saad Z Usmani, Binod Dhakal, Samir Parekh, Hui Li, Feng Wang, Rocio Montes de Oca, Vicki Plaks, Huabin Sun, Arnob Banerjee, Jordan M Schecter, Nikoletta Lendvai, Deepu Madduri, Tamar Lengil, Jieqing Zhu, Mythili Koneru, Muhammad Akram, Nitin Patel, Octavio Costa Filho, Andrzej J Jakubowiak, Peter M Voorhees","doi":"10.1200/JCO-25-00760","DOIUrl":"https://doi.org/10.1200/JCO-25-00760","url":null,"abstract":"<p><p>CARTITUDE-1 evaluated ciltacabtagene autoleucel (cilta-cel) in patients with heavily pretreated relapsed/refractory multiple myeloma (RRMM). We describe overall survival (OS), ≥5-year progression-free outcomes, associated biomarkers, and safety, with a median study follow-up of 61.3 months. For the 97 treated patients, median OS was 60.7 months (95% CI, 41.9 to not estimable). One third (32/97) of patients remain alive and progression-free for ≥5 years after a single cilta-cel infusion, without maintenance treatment. Twelve of these patients treated at a single center underwent serial minimal residual disease (MRD) and positron emission tomography-computed tomography assessments, and all (100%) were MRD-negative (at least 10^-5 threshold) and imaging-negative at year 5 or later after cilta-cel. Baseline characteristics, including the presence of high-risk cytogenetics and extramedullary disease, were generally comparable for the 32 patients who were progression-free for ≥5 years versus patients who had progressive disease by year 5. A trend of lower baseline tumor burden, higher fraction of naïve T-cells in the cilta-cel drug product, higher T cell-to-neutrophil ratio, higher hemoglobin and platelets at baseline, and higher effector-to-target ratio were associated with ≥5-year progression-free status. The safety profile of cilta-cel remained consistent with previous reports. To our knowledge, our data provide the first evidence that cilta-cel is potentially curative in patients with RRMM.</p>","PeriodicalId":15384,"journal":{"name":"Journal of Clinical Oncology","volume":" ","pages":"JCO2500760"},"PeriodicalIF":42.1,"publicationDate":"2025-06-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144208663","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}