Anand P Jillella, Sandra J Lee, Jessica K Altman, Selina M Luger, Martin S Tallman, James M Foran, Danielle Bradshaw, Lisa Y Law, Locke J Bryan, Abdallah Abou Zahr, Kebede H Begna, Alexander E Perl, Joseph J L Vadakara, Rubina Qamar, Raymond C Bergan, Michael J Fisch, Ruth C Carlos, Lynne I Wagner, Vamsi K Kota, Mark R Litzow
{"title":"急性早幼粒细胞白血病和早期死亡率的学术界合作：ECOG-ACRIN EA9131试验。","authors":"Anand P Jillella, Sandra J Lee, Jessica K Altman, Selina M Luger, Martin S Tallman, James M Foran, Danielle Bradshaw, Lisa Y Law, Locke J Bryan, Abdallah Abou Zahr, Kebede H Begna, Alexander E Perl, Joseph J L Vadakara, Rubina Qamar, Raymond C Bergan, Michael J Fisch, Ruth C Carlos, Lynne I Wagner, Vamsi K Kota, Mark R Litzow","doi":"10.1001/jamaoncol.2024.7033","DOIUrl":null,"url":null,"abstract":"<p><strong>Importance: </strong>Acute promyelocytic leukemia (APL) is an acute illness that presents with cytopenia, infections, and disseminated intravascular coagulation. Achieving remission has been shown to make a major difference in patient outcomes; however, early death rates in the first month have been as high as 30% due to acute presentation, comorbidities, the rarity of APL, and clinician inexperience.</p><p><strong>Objective: </strong>To develop treatment strategies that would decrease estimated 1-month mortality from 30% to below 15%.</p><p><strong>Design, setting, and participants: </strong>In this nonrandomized clinical trial, a treatment algorithm that focused on supportive care was used to prevent early death in patients with APL treated at academic and community health centers between August 2017 and July 2021. Because of the rarity of the disease, expert support was available 24/7 from 7 designated APL experts at 6 participating academic lead centers, and included an additional 293 community centers. When a patient presented with APL, an expert was contacted and a consensus treatment plan was developed using the algorithm and expert suggestions. There were no exclusion criteria and all patients with a confirmed diagnosis of APL regardless of age or comorbid conditions were enrolled. Expert support was provided throughout induction. Initial data analysis was conducted May 2023.</p><p><strong>Main outcomes and measures: </strong>One-month mortality; additional objectives were to compare outcomes in academic and community centers and assess 1-year and overall survival.</p><p><strong>Results: </strong>A total of 201 patients were enrolled from 43 centers; 62 at lead centers and 139 from 37 community centers. The median age was 53 years (range, 18-91 years), with 72 patients (35.8%) who were aged 60 years or older; 105 patients (52.2%) were male. Fifty-two patients (26.4%) were diagnosed with high-risk APL. The 1-month mortality rate was 6 deaths of 201 patients (3.0%; 95% CI, 1.1%-6.4%) after adjusting for 1 interim analysis. In a secondary analysis using the Kaplan-Meier method, the 1-month overall survival (OS) rate was 97.0% (95% CI, 93.5%-98.6%) and the 1-year OS rate was 94.5% (95% CI, 90.3%-96.9%).</p><p><strong>Conclusions and relevance: </strong>In this nonrandomized clinical trial, use of an algorithm combined with expert support resulted in a dramatic decrease in early death in academic and community centers. Population-wide survival improved in this highly curable disease, which suggests that implementing an accessible support system with APL experts for comanagement is the most logical next step.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov Identifier: NCT03253848.</p>","PeriodicalId":48661,"journal":{"name":"Jama Oncology","volume":" ","pages":""},"PeriodicalIF":20.1000,"publicationDate":"2025-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11869096/pdf/","citationCount":"0","resultStr":"{\"title\":\"Academic Community Partnership in Acute Promyelocytic Leukemia and Early Mortality: The ECOG-ACRIN EA9131 Trial.\",\"authors\":\"Anand P Jillella, Sandra J Lee, Jessica K Altman, Selina M Luger, Martin S Tallman, James M Foran, Danielle Bradshaw, Lisa Y Law, Locke J Bryan, Abdallah Abou Zahr, Kebede H Begna, Alexander E Perl, Joseph J L Vadakara, Rubina Qamar, Raymond C Bergan, Michael J Fisch, Ruth C Carlos, Lynne I Wagner, Vamsi K Kota, Mark R Litzow\",\"doi\":\"10.1001/jamaoncol.2024.7033\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Importance: </strong>Acute promyelocytic leukemia (APL) is an acute illness that presents with cytopenia, infections, and disseminated intravascular coagulation. Achieving remission has been shown to make a major difference in patient outcomes; however, early death rates in the first month have been as high as 30% due to acute presentation, comorbidities, the rarity of APL, and clinician inexperience.</p><p><strong>Objective: </strong>To develop treatment strategies that would decrease estimated 1-month mortality from 30% to below 15%.</p><p><strong>Design, setting, and participants: </strong>In this nonrandomized clinical trial, a treatment algorithm that focused on supportive care was used to prevent early death in patients with APL treated at academic and community health centers between August 2017 and July 2021. Because of the rarity of the disease, expert support was available 24/7 from 7 designated APL experts at 6 participating academic lead centers, and included an additional 293 community centers. When a patient presented with APL, an expert was contacted and a consensus treatment plan was developed using the algorithm and expert suggestions. There were no exclusion criteria and all patients with a confirmed diagnosis of APL regardless of age or comorbid conditions were enrolled. Expert support was provided throughout induction. Initial data analysis was conducted May 2023.</p><p><strong>Main outcomes and measures: </strong>One-month mortality; additional objectives were to compare outcomes in academic and community centers and assess 1-year and overall survival.</p><p><strong>Results: </strong>A total of 201 patients were enrolled from 43 centers; 62 at lead centers and 139 from 37 community centers. The median age was 53 years (range, 18-91 years), with 72 patients (35.8%) who were aged 60 years or older; 105 patients (52.2%) were male. Fifty-two patients (26.4%) were diagnosed with high-risk APL. The 1-month mortality rate was 6 deaths of 201 patients (3.0%; 95% CI, 1.1%-6.4%) after adjusting for 1 interim analysis. In a secondary analysis using the Kaplan-Meier method, the 1-month overall survival (OS) rate was 97.0% (95% CI, 93.5%-98.6%) and the 1-year OS rate was 94.5% (95% CI, 90.3%-96.9%).</p><p><strong>Conclusions and relevance: </strong>In this nonrandomized clinical trial, use of an algorithm combined with expert support resulted in a dramatic decrease in early death in academic and community centers. Population-wide survival improved in this highly curable disease, which suggests that implementing an accessible support system with APL experts for comanagement is the most logical next step.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov Identifier: NCT03253848.</p>\",\"PeriodicalId\":48661,\"journal\":{\"name\":\"Jama Oncology\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":20.1000,\"publicationDate\":\"2025-02-27\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11869096/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Jama Oncology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1001/jamaoncol.2024.7033\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"Biochemistry, Genetics and Molecular Biology\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Jama Oncology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1001/jamaoncol.2024.7033","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"Biochemistry, Genetics and Molecular Biology","Score":null,"Total":0}
Academic Community Partnership in Acute Promyelocytic Leukemia and Early Mortality: The ECOG-ACRIN EA9131 Trial.
Importance: Acute promyelocytic leukemia (APL) is an acute illness that presents with cytopenia, infections, and disseminated intravascular coagulation. Achieving remission has been shown to make a major difference in patient outcomes; however, early death rates in the first month have been as high as 30% due to acute presentation, comorbidities, the rarity of APL, and clinician inexperience.
Objective: To develop treatment strategies that would decrease estimated 1-month mortality from 30% to below 15%.
Design, setting, and participants: In this nonrandomized clinical trial, a treatment algorithm that focused on supportive care was used to prevent early death in patients with APL treated at academic and community health centers between August 2017 and July 2021. Because of the rarity of the disease, expert support was available 24/7 from 7 designated APL experts at 6 participating academic lead centers, and included an additional 293 community centers. When a patient presented with APL, an expert was contacted and a consensus treatment plan was developed using the algorithm and expert suggestions. There were no exclusion criteria and all patients with a confirmed diagnosis of APL regardless of age or comorbid conditions were enrolled. Expert support was provided throughout induction. Initial data analysis was conducted May 2023.
Main outcomes and measures: One-month mortality; additional objectives were to compare outcomes in academic and community centers and assess 1-year and overall survival.
Results: A total of 201 patients were enrolled from 43 centers; 62 at lead centers and 139 from 37 community centers. The median age was 53 years (range, 18-91 years), with 72 patients (35.8%) who were aged 60 years or older; 105 patients (52.2%) were male. Fifty-two patients (26.4%) were diagnosed with high-risk APL. The 1-month mortality rate was 6 deaths of 201 patients (3.0%; 95% CI, 1.1%-6.4%) after adjusting for 1 interim analysis. In a secondary analysis using the Kaplan-Meier method, the 1-month overall survival (OS) rate was 97.0% (95% CI, 93.5%-98.6%) and the 1-year OS rate was 94.5% (95% CI, 90.3%-96.9%).
Conclusions and relevance: In this nonrandomized clinical trial, use of an algorithm combined with expert support resulted in a dramatic decrease in early death in academic and community centers. Population-wide survival improved in this highly curable disease, which suggests that implementing an accessible support system with APL experts for comanagement is the most logical next step.
期刊介绍：
At JAMA Oncology, our primary goal is to contribute to the advancement of oncology research and enhance patient care. As a leading journal in the field, we strive to publish influential original research, opinions, and reviews that push the boundaries of oncology science.
Our mission is to serve as the definitive resource for scientists, clinicians, and trainees in oncology globally. Through our innovative and timely scientific and educational content, we aim to provide a comprehensive understanding of cancer pathogenesis and the latest treatment advancements to our readers.
We are dedicated to effectively disseminating the findings of significant clinical research, major scientific breakthroughs, actionable discoveries, and state-of-the-art treatment pathways to the oncology community. Our ultimate objective is to facilitate the translation of new knowledge into tangible clinical benefits for individuals living with and surviving cancer.
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