Journal of the National Cancer Institute最新文献

{"title":"Ten years of robot-assisted versus laparoscopic total mesorectal excision for rectal cancer (short-term RESOLUTION)","authors":"Rauand Duhoky, Ritch T J Geitenbeek, Guglielmo Niccolò Piozzi, Thijs A Burghgraef, Christina A Fleming, Shamsul Masum, Adrian Hopgood, Timothy Rockall, Quentin Denost, Christophe Taoum, Roel Hompes, Jim Briggs, Esther C J Consten, Jim S Khan","doi":"10.1093/jnci/djaf084","DOIUrl":"https://doi.org/10.1093/jnci/djaf084","url":null,"abstract":"Background Total mesorectal excision is the gold standard for rectal cancer surgery, with laparoscopic and robot-assisted approaches commonly employed. While robot-assisted surgery may offer technical advantages, there is limited evidence comparing short-term outcomes of laparoscopic and robot-assisted techniques, particularly in Western European populations. This study aimed to assess the short-term outcomes of laparoscopic vs robot-assisted total mesorectal excision for rectal cancer. Methods This multicentre, international, retrospective cohort study included 1749 patients who underwent laparoscopic or robot-assisted total mesorectal excision from January 2014 to January 2024. Inverse probability of treatment weighting was applied to minimise confounding. Primary outcomes were length of stay, operative time, and conversion rates. Secondary outcomes included complications and pathological outcomes within 90 days, and readmissions and reinterventions within 30 days. Results The final cohort included 680.9 laparoscopic and 1057.5 robot-assisted cases after weighting. Robot-assisted surgery showed lower conversion rates (6.1% vs 3.5%, p = .025), higher rates of primary anastomosis (80.1% vs 92.1%, p < .001), and fewer stoma formations (78.4% vs 63.7%, p < .001). Pathological outcomes indicated a higher rate of complete mesorectal excision in the robot-assisted group (77.2% vs 86.0%, p < .001), though this data was not available for all centres. Operative time was longer in the robot-assisted cohort (181.0 vs 220.0 minutes, p < .001), but no significant differences were observed in postoperative complications, length of stay, anastomotic leakage or 30-day reintervention rates. Conclusion Robot-assisted surgery low anterior resection demonstrated improved short-term outcomes with lower conversion rates, higher rates of complete mesorectal excision, and higher restorative procedure rates, which may influence longer-term oncological and patient quality of life outcomes. Trial registration https://www.isrctn.com/ISRCTN75281193","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"5 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143824874","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":"Secondary malignancies after treatment of testicular germ cell tumors: a systematic review & meta-analysis","authors":"Ahmad Mousa, Ali Amiri, Sanchit Kaushal, Emma Wilson, Viranda Jayalath, David-Dan Nguyen, Isabelle Tan, Robert J Hamilton","doi":"10.1093/jnci/djaf099","DOIUrl":"https://doi.org/10.1093/jnci/djaf099","url":null,"abstract":"Background Testicular germ cell tumours (TGCTs) are the most common malignancy in men 15-35 years of age. Management options for men with TGCTs include surgery, radiation and/or chemotherapy. Given TGCTs’ excellent survival, most patients live long enough to experience delayed treatment toxicities, warranting careful consideration of therapeutic decisions. An important outcome of interest is the development of secondary malignant neoplasms (SMNs). Methods A systematic literature search was conducted through a combination of database searches (Medline, EMBASE, and Cochrane library) and manual review. Studies evaluating the incidence of SMNs in patients following treatment for TGCTs were identified. Our primary outcome was the diagnosis of any non-germ cell SMN following treatment, compared to the general population. Meta-analyses were performed using random-effects models, with outcomes reported as standardized incidence ratios (SIR). Strength of evidence was evaluated using the GRADE framework. Results Twenty-one studies including 88,863 patients with 5,180 SMNs were included. Median follow-up was 12.5 years. The incidence of non-germ cell SMNs following definitive treatment of TGCTs varied by treatment modality. Surgery alone was not associated with an increased risk (SIR: 0.99, 95% CI: 0.84–1.17); radiation (SIR: 1.66, 95% CI: 1.43–1.93), chemotherapy (SIR: 1.65, 95% CI: 1.39–1.96), and combined chemotherapy and radiation (SIR: 2.73, 95% CI: 2.23–3.33) were associated with a moderate to large increase in risk. There was low to moderate certainty in quality of evidence by GRADE framework. Conclusions Chemotherapy, radiation, and their combination are associated with an increased risk of non-germ cell SMNs after the treatment of TGCTs.","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"38 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143824873","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":"Neoadjuvant versus perioperative chemo-immunotherapy according to pathological response in resectable NSCLC: a reconstructed individual patient data meta-analysis","authors":"Antonio Nuccio, Fabio Salomone, Alberto Servetto, Biagio Ricciuti, Daniele Marinelli, Alessandra Bulotta, Giulia Veronesi, Marina Chiara Garassino, Valter Torri, Benjamin Besse, Giuseppe Viscardi, Roberto Ferrara","doi":"10.1093/jnci/djaf090","DOIUrl":"https://doi.org/10.1093/jnci/djaf090","url":null,"abstract":"Neoadjuvant chemo-immunotherapy transformed early-stage non-small cell lung cancer (NSCLC) treatment. However, the prognostic value of different pathological responses and the impact of adjuvant immunotherapy within a chemo-immunotherapy perioperative strategy remains unclear. We estimated time-to-event outcomes by graphical reconstruction of event-free survival (EFS) curves by pathological response (pCR, MPR, no-MPR) reported in early-stage NSCLC neoadjuvant/perioperative chemo-immunotherapy trials. MPR 1-10% subgroup, previously unreported, was retrieved by removing patients achieving pCR from the MPR group. Survival analysis by pathological response and comparison between neoadjuvant/perioperative strategies within subgroups were assessed. A statistically significant EFS difference according to pathological response was found, showing a prognostic gradient shifting from pCR (good), MPR 1-10% (intermediate) and no-MPR (poor). There was no difference between neoadjuvant/perioperative strategies within subgroups, however a trend for EFS benefit with perioperative and neoadjuvant chemo-immunotherapy was observed in MPR 1-10% and no-MPR patients, respectively. In conclusio, a pathological response-based algorithm could better tailor early-stage NSCLC treatment.","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"24 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143818955","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":"A working group report from the 2024 NCI/GCSC endometrial cancer clinical trials planning meeting: refining the approach to endometrial cancer in the immunotherapy era","authors":"Casey M Cosgrove, Dmitriy Zamarin, Jose R Conejo-Garcia, Kari E Hacker, Roberto Vargas, Panagiotis A Konstantinopoulos, Haider S Mahdi, Stephanie Gaillard, Stephanie Markovina, Elise C Kohn, Sarah F Adams","doi":"10.1093/jnci/djaf089","DOIUrl":"https://doi.org/10.1093/jnci/djaf089","url":null,"abstract":"Endometrial cancer (EC) is now the leading cause of gynecologic cancer death in the United States. Recognizing the urgent need to improve outcomes for patients diagnosed with EC, The National Cancer Institute (NCI) Gynecologic Cancer Steering Committee (GCSC) convened a Clinical Trials Planning Meeting (CTPM) on January 8th and 9th 2024, “Refining the Approach to Endometrial Cancer in the Immunotherapy Era.” Multi-disciplinary experts were charged with addressing critical challenges, to optimize treatment of EC in the new immunotherapy landscape. As part of the CTPM working groups were assembled to address several important aspects of clinical trial design. Working Group 1 (WG1) focused on translational science and was tasked with reviewing the scientific literature for data on validated discriminants of response to immunotherapy to inform trial concept development by the therapy-focused groups. The WG established that molecular subtyping of EC is now the standard approach for classifying endometrial tumors. Molecular subtyping for both prognostic and predictive applications should be considered when assessing biomarkers as well as therapeutic targets. Additionally, strategies to improve immune response like incorporation of radiation as well as therapy sequencing considerations should continue to be explored. A major key observation from WG1 was lack of validated discriminants for immunotherapy response beyond mismatch repair status and tumor mutational burden and exploration of additional discriminants of response and resistance will be critical with the increasing use of immunotherapy in EC.","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"26 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143818958","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":"Effect size estimation in cooperative group and industry sponsored phase 3 oncology trials","authors":"Sonal S Noticewala, Adam Grippin, Ramez Kouzy, Joseph Abi Jaoude, Avital Miller, Alexander Sherry, Ethan B Ludmir","doi":"10.1093/jnci/djaf097","DOIUrl":"https://doi.org/10.1093/jnci/djaf097","url":null,"abstract":"Overly optimistic estimations of effect sizes may lead to underpowered studies and risk of erroneously dismissing effective treatments. To understand the prevalence and factors contributing to estimation of effect sizes, we evaluated 385 superiority-design phase 3 oncology randomized controlled trials (RCTs) with pre-specified and observed hazard ratios (HR) in published manuscripts. Of these, 88% were sponsored by industry and 22.6% were sponsored by cooperative groups. Few studies (10%) provided justification of their chosen sample size in the manuscript or available protocol. Overly optimistic estimations of effect sizes were common in cooperative group studies and, by contrast, uncommon in industry-sponsored studies. Moreover, industry-sponsored trials meeting the primary endpoint frequently achieved significantly lower HRs than expected. Together, these data suggest a correlation between study sponsorship, clinical trial power calculations, and subsequent trial outcomes.","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"20 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143824875","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":"Antihypertensive drugs and survival outcomes in oropharyngeal squamous cell carcinoma patients","authors":"Che-Min Lee, Brennan J Wadsworth, Ryan Urban, Meredith A Clark, Rocky Shi, Daegan Sit, Sarah N Hamilton, Kevin L Bennewith","doi":"10.1093/jnci/djaf056","DOIUrl":"https://doi.org/10.1093/jnci/djaf056","url":null,"abstract":"Background Radiation therapy is commonly used to treat head and neck cancer patients, and response may be improved by combining radiation with preexisting medications. Based on recent preclinical and retrospective patient data, we hypothesized that antihypertensive drugs may improve radiotherapy outcomes. Methods Retrospective analyses were conducted on 1077 oropharyngeal squamous cell carcinoma and 608 nasopharyngeal carcinoma patients, all of whom received radiation therapy. Univariate and multivariate analyses were conducted to assess overall survival, disease-specific survival, and locoregional control for cancer patients taking angiotensin receptor blockers (ARBs), angiotensin-converting enzyme inhibitors, calcium channel blockers, or beta blockers compared with propensity score–matched groups of patients not taking these medications. Results Oropharyngeal squamous cell carcinoma patients taking antihypertensive medications were statistically older and had higher Charlson Comorbidity Indices at diagnosis. However, these patients had statistically significant improved overall survival, disease-specific survival, and locoregional control compared with propensity score–matched oropharyngeal squamous cell carcinoma patients who were not taking antihypertensive medications, with ARB users showing the greatest improvements. Antihypertensive drugs did not affect outcomes in the nasopharyngeal carcinoma patient cohort. Conclusion The use of antihypertensive medications, and particularly ARBs, was associated with improved outcomes in oropharyngeal squamous cell carcinoma patients who had more advanced age and higher Charlson Comorbidity Indices at diagnosis. This study supports future prospective testing of ARBs in conjunction with radiation therapy in this group of higher risk oropharyngeal squamous cell carcinoma patients. Additionally, this study illustrates the need to use propensity score matching to identify patient subgroups that may benefit from a given treatment in retrospective analyses.","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"33 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143775503","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":"Extended endocrine therapy use and decision making after breast cancer diagnosis","authors":"Allison W Kurian, Allison K C Furgal, Archana Radhakrishnan, Christine M Veenstra, Paul Abrahamse, Kevin C Ward, Ann S Hamilton, Timothy P Hofer, Steven J Katz, Lauren P Wallner, Sarah T Hawley","doi":"10.1093/jnci/djaf076","DOIUrl":"https://doi.org/10.1093/jnci/djaf076","url":null,"abstract":"Purpose Adjuvant endocrine therapy is recommended to extend beyond five years for stage II breast cancer, with less consensus for extension in stage I. We aimed to understand use of and decision-making about extended endocrine therapy. Patients and Methods Women aged 20-79 diagnosed with stage I-II breast cancer in 2014-15 and reported to Georgia and Los Angeles County SEER registries were surveyed at seven months and again at six years post-diagnosis (N = 2,361; response rate = 60%). Women with estrogen receptor (ER) and/or progesterone receptor (PR)-positive disease were asked about their decision whether to continue therapy. Results Of 831 women, 591 had completed or were completing five years of endocrine therapy. Among those who had decided (n = 557), 46.9% decided to continue (39.4% stage I, 62.4% stage II). On multivariable analysis, factors associated with continuation for stage I were worry about recurrence (adjusted odds ratio (aOR) 3.35, 95% confidence interval (CI) 1.78-6.32); desire for most extensive treatment (aOR 2.15; CI 1.11-4.14); and primary care physician participation (aOR 4.30; CI 2.22-8.32). Side effects were inversely associated with continuation (stage I aOR 0.21: CI 0.11-0.41). Associations were similar for stage II; for stage I only, bilateral mastectomy and chemotherapy were associated with continuation. Decision to continue did not vary by race, ethnicity or demographic factors. Conclusions Nearly 40% of women with stage I breast cancer, and two-thirds with stage II, decided to extend endocrine therapy. Decision-making was influenced by patient values and varied little by stage. These results can inform physician-patient discussion about extending endocrine therapy.","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"16 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143744987","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":"Development of a Multi-Institutional Dataset to Validate a Novel Inflammatory Breast Cancer Diagnostic Score","authors":"Filipa Lynce, Samuel M Niman, Megumi Kai, Sean Ryan Ma, Elizabeth Troll, Li Li, Kathy D Miller, Reshma Jagsi, Ginny Mason, Beth A Overmoyer, H T Carisa Le-Petross, Faina Nakhlis, Savitri Krishnamurthy, Beth T Harrison, Susie X Sun, Eren D Yeh, Jennifer R Bellon, Laura E Warren, Michael C Stauder, Meredith M Regan, Wendy A Woodward","doi":"10.1093/jnci/djaf088","DOIUrl":"https://doi.org/10.1093/jnci/djaf088","url":null,"abstract":"Purpose Susan G. Komen, the Inflammatory Breast Cancer (IBC) Research Foundation, and the Milburn Foundation convened patient advocates, clinicians, and researchers to propose novel quantitative scoring rubrics for IBC diagnosis. In this study, we developed a multi-institutional clinical dataset to test and validate the proposed scoring system. Methods IBC (N = 988) and non-IBC (N = 332) cases were identified at two institutions with dedicated multidisciplinary IBC programs. The non-IBC cohort included consecutive cT4b and cT4c patients. Standard operating procedures (SOPs) were developed for all ambiguous findings and languages. Three different methods were used for the imputation of missing data, resulting in three separate datasets. The sensitivity, specificity, and area under the receiver operator characteristic curve (AUC-ROC) were used to assess the discrimination of the proposed scoring rubric. Results The distribution of “true IBC” cases was 19.7% very likely IBC, 49.1% strong possibility of IBC, 0.4% weak possibility of IBC, 0.1% very unlikely IBC, and 30.7% unknown; corresponding groupings for true non-IBC cases were 0.6% very likely IBC, 51.8% strong possibility of IBC, 9.9% weak possibility of IBC, 2.1% very unlikely IBC, and 35.5% unknown. AUC-ROC values for missing data imputation methods were similar (0.83–0.84); exploratory score refinement improved the AUC-ROC to 0.88–0.89. Conclusion Using the largest multi-institutional IBC clinical database to date, the score has been validated and is available for clinical use at https://www.komen.org/ibc-calc to assist healthcare providers and their patients in IBC diagnosis. Exploratory score refinement demonstrates the potential to increase specificity; however, any change requires separate validation.","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"26 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143744991","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":"Prevalence by therapy line and incidence of breast cancer brain metastases in 18,075 patients","authors":"Sarah L Sammons, Thibaut Sanglier, Jose Pablo Leone, Timothy K Erick, Peter Lambert, Filippo Montemurro, Raf Poppe, Eleonora Restuccia, Sara M Tolaney, Nancy U Lin","doi":"10.1093/jnci/djaf048","DOIUrl":"https://doi.org/10.1093/jnci/djaf048","url":null,"abstract":"Importance Brain metastases portend poor prognosis in patients with metastatic breast cancer (MBC). Designing treatment and prevention clinical trials requires knowledge of brain metastases incidence with each line of therapy. Objectives We assessed the prevalence and cumulative incidence of brain metastases in a large MBC patient cohort by subtype and line of therapy, and the impact of HER2-low expression on prevalence. Design, Setting and Outcomes We analyzed brain metastases prevalence in patients with MBC in a nationwide electronic health record-derived de-identified database. The primary outcome was first diagnosis of brain metastases. We estimated prevalence and incidence of brain metastases by MBC subtype, including HER2-low and therapy line. We used the cumulative incidence function to estimate brain metastases risk in patients without brain metastases at initiation of systemic therapy. All p-values are two-sided, and a p-value ≤ 0.05 indicates statistical significance. Results Among 18,075 patients with MBC, 1,102 (6.1%) had at least one brain metastasis at first-line therapy initiation. For the remaining 16,973 patients, cumulative incidence of brain metastases at 60 months was 10% in patients with hormone receptor-positive (HR+)/HER2- disease, 23% for HR+/HER2+ disease, 34% for HR-/HER2+ disease, and 22% for triple-negative breast cancer (TNBC). HER2-low expression within HR+/HER2- and TNBC subtypes had no impact on brain metastases incidence. Brain metastases prevalence increased per line of therapy for patients with all breast cancer subtypes. Conclusions Brain metastases incidence increases per line of therapy for every MBC subtype. The HER2-low biomarker does not impact brain metastases incidence within historical subtypes.","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"11 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143745004","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":"Incorporation of patient-reported outcomes in pediatric cancer clinical trials: design, implementation, and dissemination","authors":"Katie A Greenzang, Kathleen E Montgomery, Adam DuVall, Michael E Roth, Mark Krailo, Michelle M Nuño, Lindsay Renfro, Natalie DelRocco, John Doski, Kara Kelly, Sharon M Castellino, Jennifer McNeer, Maureen M O’Brien, Damon Reed, Katherine Janeway, Pamela S Hinds, Sue Zupanec, Susan K Parsons","doi":"10.1093/jnci/djaf083","DOIUrl":"https://doi.org/10.1093/jnci/djaf083","url":null,"abstract":"Understanding the patient experience of treatment toxicities and their impact on health-related quality of life (HRQoL) of cancer treatments requires asking patients themselves using patient-reported outcomes (PROs). Over the past twenty years, the National Institutes of Health (NIH) sponsored several tools, namely Patient-Reported Outcome Measurement Information System (PROMIS) measures and the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) for precisely this purpose: to ensure valid, reliable tools to collect and detect patient-reported toxicities or adverse events and their impact on HRQoL. These PRO measures have been widely incorporated in clinical trials for adults with cancer. Yet, despite considerable work developing and validating developmentally appropriate versions of these measures for pediatric and adolescent self-report, PRO inclusion in pediatric and adolescent and young adult (AYA) clinical trials has lagged. Here we discuss optimal strategies to integrate validated PRO tools and sound analytic methodologies in clinical trials for children and AYAs with cancer, highlighting lessons learned from recent successes and ongoing experiences developing and opening cross-network trials for children and AYAs through the Children’s Oncology Group (COG) for patients with classic Hodgkin lymphoma (cHL), osteosarcoma (OS), and acute lymphoblastic leukemia (ALL).","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"72 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143745006","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}