Nicholas J Short, Agnieszka Wierzbowska, Thomas Cluzeau, Kamel Laribi, Christian Recher, Jaroslaw Czyz, Bogdan Ochrem, Lionel Ades, Maria Pilar Gallego-Hernanz, Mael Heiblig, Ernesta Audisio, Ewa Zarzycka, Shuli Li, Nicholas Ferenc, Tammie Yeh, Douglas V Faller, Farhad Sedarati, Cristina Papayannidis
{"title":"阿扎胞苷和 venetoclax 联合或不联合培伐地司他治疗新诊断的急性髓性白血病患者。","authors":"Nicholas J Short, Agnieszka Wierzbowska, Thomas Cluzeau, Kamel Laribi, Christian Recher, Jaroslaw Czyz, Bogdan Ochrem, Lionel Ades, Maria Pilar Gallego-Hernanz, Mael Heiblig, Ernesta Audisio, Ewa Zarzycka, Shuli Li, Nicholas Ferenc, Tammie Yeh, Douglas V Faller, Farhad Sedarati, Cristina Papayannidis","doi":"10.1080/10428194.2024.2431878","DOIUrl":null,"url":null,"abstract":"<p><p>This phase 2 study investigated pevonedistat + azacitidine + venetoclax (<i>n</i> = 83) versus azacitidine + venetoclax (<i>n</i> = 81) in patients with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive chemotherapy. The study was stopped early following negative results from PANTHER, which evaluated pevonedistat in higher-risk myelodysplastic syndromes/chronic myelomonocytic leukemia or low-blast AML. Outcomes were analyzed up to the datacut. For pevonedistat + azacitidine + venetoclax versus azacitidine + venetoclax, the median follow-up was 8.44 versus 7.95 months; the complete remission (CR) rate was 45% versus 49%; composite CR (CCR; CR+CR with incomplete blood count recovery) was 77% versus 72%. There were no differences in event-free survival (primary endpoint; hazard ratio [HR]: 0.99; 95% confidence interval [CI]: 0.61-1.60; <i>p</i> = 0.477) or overall survival (HR: 1.42; 95% CI: 0.82-2.49; <i>p</i> = 0.896). In exploratory analyses in <i>IDH</i>-mutated AML, CCR rates were higher with pevonedistat + azacitidine + venetoclax versus azacitidine + venetoclax. Safety was similar between treatment arms. Efficacy/safety with azacitidine + venetoclax was consistent with the phase 3 VIALE-A study.</p><p><strong>Trial registration: </strong>NCT04266795.</p>","PeriodicalId":18047,"journal":{"name":"Leukemia & Lymphoma","volume":" ","pages":"1-11"},"PeriodicalIF":2.2000,"publicationDate":"2024-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Azacitidine and venetoclax with or without pevonedistat in patients with newly diagnosed acute myeloid leukemia.\",\"authors\":\"Nicholas J Short, Agnieszka Wierzbowska, Thomas Cluzeau, Kamel Laribi, Christian Recher, Jaroslaw Czyz, Bogdan Ochrem, Lionel Ades, Maria Pilar Gallego-Hernanz, Mael Heiblig, Ernesta Audisio, Ewa Zarzycka, Shuli Li, Nicholas Ferenc, Tammie Yeh, Douglas V Faller, Farhad Sedarati, Cristina Papayannidis\",\"doi\":\"10.1080/10428194.2024.2431878\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>This phase 2 study investigated pevonedistat + azacitidine + venetoclax (<i>n</i> = 83) versus azacitidine + venetoclax (<i>n</i> = 81) in patients with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive chemotherapy. The study was stopped early following negative results from PANTHER, which evaluated pevonedistat in higher-risk myelodysplastic syndromes/chronic myelomonocytic leukemia or low-blast AML. Outcomes were analyzed up to the datacut. For pevonedistat + azacitidine + venetoclax versus azacitidine + venetoclax, the median follow-up was 8.44 versus 7.95 months; the complete remission (CR) rate was 45% versus 49%; composite CR (CCR; CR+CR with incomplete blood count recovery) was 77% versus 72%. There were no differences in event-free survival (primary endpoint; hazard ratio [HR]: 0.99; 95% confidence interval [CI]: 0.61-1.60; <i>p</i> = 0.477) or overall survival (HR: 1.42; 95% CI: 0.82-2.49; <i>p</i> = 0.896). In exploratory analyses in <i>IDH</i>-mutated AML, CCR rates were higher with pevonedistat + azacitidine + venetoclax versus azacitidine + venetoclax. Safety was similar between treatment arms. Efficacy/safety with azacitidine + venetoclax was consistent with the phase 3 VIALE-A study.</p><p><strong>Trial registration: </strong>NCT04266795.</p>\",\"PeriodicalId\":18047,\"journal\":{\"name\":\"Leukemia & Lymphoma\",\"volume\":\" \",\"pages\":\"1-11\"},\"PeriodicalIF\":2.2000,\"publicationDate\":\"2024-11-28\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Leukemia & Lymphoma\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1080/10428194.2024.2431878\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Leukemia & Lymphoma","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/10428194.2024.2431878","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Azacitidine and venetoclax with or without pevonedistat in patients with newly diagnosed acute myeloid leukemia.
This phase 2 study investigated pevonedistat + azacitidine + venetoclax (n = 83) versus azacitidine + venetoclax (n = 81) in patients with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive chemotherapy. The study was stopped early following negative results from PANTHER, which evaluated pevonedistat in higher-risk myelodysplastic syndromes/chronic myelomonocytic leukemia or low-blast AML. Outcomes were analyzed up to the datacut. For pevonedistat + azacitidine + venetoclax versus azacitidine + venetoclax, the median follow-up was 8.44 versus 7.95 months; the complete remission (CR) rate was 45% versus 49%; composite CR (CCR; CR+CR with incomplete blood count recovery) was 77% versus 72%. There were no differences in event-free survival (primary endpoint; hazard ratio [HR]: 0.99; 95% confidence interval [CI]: 0.61-1.60; p = 0.477) or overall survival (HR: 1.42; 95% CI: 0.82-2.49; p = 0.896). In exploratory analyses in IDH-mutated AML, CCR rates were higher with pevonedistat + azacitidine + venetoclax versus azacitidine + venetoclax. Safety was similar between treatment arms. Efficacy/safety with azacitidine + venetoclax was consistent with the phase 3 VIALE-A study.
期刊介绍：
Leukemia & Lymphoma in its fourth decade continues to provide an international forum for publication of high quality clinical, translational, and basic science research, and original observations relating to all aspects of hematological malignancies. The scope ranges from clinical and clinico-pathological investigations to fundamental research in disease biology, mechanisms of action of novel agents, development of combination chemotherapy, pharmacology and pharmacogenomics as well as ethics and epidemiology. Submissions of unique clinical observations or confirmatory studies are considered and published as Letters to the Editor
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
