Daniel J Zheng, Gary Hettinger, Catherine Aftandilian, Kira Bona, Emi H Caywood, Anderson B Collier, Caitlin W Elgarten, Cody Gathers, Taumoha Ghosh, M Monica Gramatges, Meret Henry, Yuan-Shung V Huang, Yimei Li, Craig Lotterman, Kelly Maloney, Amir Mian, Tamara P Miller, Arunkumar Modi, Rajen Mody, Elaine Morgan, Regina Myers, Haley Newman, Jose Ortiz, Alix E Seif, Caroline Smith, Jamie Stokke, Xin Wang, Naomi Winick, Jennifer J Wilkes, Victor Wong, Richard Aplenc, Kelly D Getz
{"title":"儿童急性髓性白血病临床试验细胞分子风险的真实世界普遍性：REAL-AML队列报告","authors":"Daniel J Zheng, Gary Hettinger, Catherine Aftandilian, Kira Bona, Emi H Caywood, Anderson B Collier, Caitlin W Elgarten, Cody Gathers, Taumoha Ghosh, M Monica Gramatges, Meret Henry, Yuan-Shung V Huang, Yimei Li, Craig Lotterman, Kelly Maloney, Amir Mian, Tamara P Miller, Arunkumar Modi, Rajen Mody, Elaine Morgan, Regina Myers, Haley Newman, Jose Ortiz, Alix E Seif, Caroline Smith, Jamie Stokke, Xin Wang, Naomi Winick, Jennifer J Wilkes, Victor Wong, Richard Aplenc, Kelly D Getz","doi":"10.1093/jnci/djaf234","DOIUrl":null,"url":null,"abstract":"Cytomolecular features critical for risk-stratified treatment determination in pediatric acute myeloid leukemia (AML) were expanded in Children’s Oncology Group (COG) Phase III trial AAML1831 based on previous trials. It remains unknown whether the cytomolecular risk profiles are generalizable to the real-world. We addressed this knowledge gap using a nationally representative real-world cohort of 913 pediatric AML patients. Distributions of cytomolecular risk profiles and individual markers were comparable for trial-enrolled and non-enrolled patients, as well as across social drivers of trial enrollment (race/ethnicity, language, insurance, acuity). Compared to patients with only favorable cytomolecular markers (4-year OS 89.48%; 95% CI: 84.46%-92.95%), patients with both favorable and unfavorable (hazards ratio [HR] = 2.49, 95% CI : 1.18-5.23), neutral (HR = 4.33, 95% CI : 2.75-6.82), and only unfavorable (HR = 5.80, 95% CI: 3.70-9.11) markers all had increased hazards of death. Cytomolecular risk informed by trial data appears to be generalizable to the real-world setting in pediatric AML.","PeriodicalId":501635,"journal":{"name":"Journal of the National Cancer Institute","volume":"195 1","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Real-world generalizability of clinical trial cytomolecular risk in pediatric acute myeloid leukemia: a report from the REAL-AML cohort\",\"authors\":\"Daniel J Zheng, Gary Hettinger, Catherine Aftandilian, Kira Bona, Emi H Caywood, Anderson B Collier, Caitlin W Elgarten, Cody Gathers, Taumoha Ghosh, M Monica Gramatges, Meret Henry, Yuan-Shung V Huang, Yimei Li, Craig Lotterman, Kelly Maloney, Amir Mian, Tamara P Miller, Arunkumar Modi, Rajen Mody, Elaine Morgan, Regina Myers, Haley Newman, Jose Ortiz, Alix E Seif, Caroline Smith, Jamie Stokke, Xin Wang, Naomi Winick, Jennifer J Wilkes, Victor Wong, Richard Aplenc, Kelly D Getz\",\"doi\":\"10.1093/jnci/djaf234\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Cytomolecular features critical for risk-stratified treatment determination in pediatric acute myeloid leukemia (AML) were expanded in Children’s Oncology Group (COG) Phase III trial AAML1831 based on previous trials. It remains unknown whether the cytomolecular risk profiles are generalizable to the real-world. We addressed this knowledge gap using a nationally representative real-world cohort of 913 pediatric AML patients. Distributions of cytomolecular risk profiles and individual markers were comparable for trial-enrolled and non-enrolled patients, as well as across social drivers of trial enrollment (race/ethnicity, language, insurance, acuity). Compared to patients with only favorable cytomolecular markers (4-year OS 89.48%; 95% CI: 84.46%-92.95%), patients with both favorable and unfavorable (hazards ratio [HR] = 2.49, 95% CI : 1.18-5.23), neutral (HR = 4.33, 95% CI : 2.75-6.82), and only unfavorable (HR = 5.80, 95% CI: 3.70-9.11) markers all had increased hazards of death. Cytomolecular risk informed by trial data appears to be generalizable to the real-world setting in pediatric AML.\",\"PeriodicalId\":501635,\"journal\":{\"name\":\"Journal of the National Cancer Institute\",\"volume\":\"195 1\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-08-20\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of the National Cancer Institute\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1093/jnci/djaf234\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of the National Cancer Institute","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1093/jnci/djaf234","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Real-world generalizability of clinical trial cytomolecular risk in pediatric acute myeloid leukemia: a report from the REAL-AML cohort
Cytomolecular features critical for risk-stratified treatment determination in pediatric acute myeloid leukemia (AML) were expanded in Children’s Oncology Group (COG) Phase III trial AAML1831 based on previous trials. It remains unknown whether the cytomolecular risk profiles are generalizable to the real-world. We addressed this knowledge gap using a nationally representative real-world cohort of 913 pediatric AML patients. Distributions of cytomolecular risk profiles and individual markers were comparable for trial-enrolled and non-enrolled patients, as well as across social drivers of trial enrollment (race/ethnicity, language, insurance, acuity). Compared to patients with only favorable cytomolecular markers (4-year OS 89.48%; 95% CI: 84.46%-92.95%), patients with both favorable and unfavorable (hazards ratio [HR] = 2.49, 95% CI : 1.18-5.23), neutral (HR = 4.33, 95% CI : 2.75-6.82), and only unfavorable (HR = 5.80, 95% CI: 3.70-9.11) markers all had increased hazards of death. Cytomolecular risk informed by trial data appears to be generalizable to the real-world setting in pediatric AML.
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