Raffaele Palmieri, Fabio Guolo, Luana Fianchi, Felicetto Ferrara, Paola Minetto, Maria Paola Martelli, Carola Riva, Patrizia Chiusolo, Michela Rondoni, Saveria Capria, Clara Minotti, Federica Pilo, Salvatore Perrone, Andrea Corbingi, Francesco Grimaldi, Giulia De Luca, Carla Fili, Caterina Alati, Francesco Mannelli, Federica Lessi, Francesco Marchesi, Lorenzo Brunetti, Debora Capelli, Anna Lina Piccioni, Calogero Vetro, Fanny Erika Palumbo, Carla Mazzone, Alessandra Sperotto, Giovanni Luzi, Michela Dargenio, Sabrina Mariani, Marco Cerrano, Antonino Mulè, Ambra Di Veroli, Elisa Meddi, Federico Moretti, Cristina Papayannidis, Alessandro Isidori, Antonella Ferrari, Sara Galimberti, Daniela Cilloni, Massimo Breccia, Francesco Lanza, Michele Gottardi, Roberto Cairoli, Anna Candoni, Livio Pagano, Elisabetta Todisco, Roberto Massimo Lemoli, Adriano Venditti, Francesco Buccisano
{"title":"Impact of fitness categorization according to SIE/SIES/GITMO criteria in therapy-related and AML-MRC receiving CPX-351.","authors":"Raffaele Palmieri, Fabio Guolo, Luana Fianchi, Felicetto Ferrara, Paola Minetto, Maria Paola Martelli, Carola Riva, Patrizia Chiusolo, Michela Rondoni, Saveria Capria, Clara Minotti, Federica Pilo, Salvatore Perrone, Andrea Corbingi, Francesco Grimaldi, Giulia De Luca, Carla Fili, Caterina Alati, Francesco Mannelli, Federica Lessi, Francesco Marchesi, Lorenzo Brunetti, Debora Capelli, Anna Lina Piccioni, Calogero Vetro, Fanny Erika Palumbo, Carla Mazzone, Alessandra Sperotto, Giovanni Luzi, Michela Dargenio, Sabrina Mariani, Marco Cerrano, Antonino Mulè, Ambra Di Veroli, Elisa Meddi, Federico Moretti, Cristina Papayannidis, Alessandro Isidori, Antonella Ferrari, Sara Galimberti, Daniela Cilloni, Massimo Breccia, Francesco Lanza, Michele Gottardi, Roberto Cairoli, Anna Candoni, Livio Pagano, Elisabetta Todisco, Roberto Massimo Lemoli, Adriano Venditti, Francesco Buccisano","doi":"10.1182/bloodadvances.2025017089","DOIUrl":null,"url":null,"abstract":"<p><strong>Abstract: </strong>CPX-351, a novel liposomal formulation of cytarabine and daunorubicin, represents the standard of care in fit patients with acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) and therapy-related AML (t-AML). Considering its better safety profile than conventional intensive chemotherapy, we investigated its cost-to-benefit ratio, in terms of overall survival and of mortality, in a large multicentric series of AML-MRC and t-AML receiving CPX-351 outside clinical trials between 2019 and 2022. Patients were classified as fit or unfit for intensive chemotherapy through a comprehensive evaluation of age, comorbidities, and performance status by adopting Italian Society of Hematology/Italian Society of Experimental Hematology/Gruppo Italiano per il Trapianto di Midollo Osseo (SIE/SIES/GITMO) criteria. Disease risk was defined according to the European LeukemiaNet 2017 classification. Before treatment start, 328 of 403 (81.4%) patients were classified as fit and 75 of 403 (18.6%) as unfit. Three hundred and ninety-six had a full genetic/cytogenetic profile, with 17 (4%) being categorized as favorable risk, 162 (41%) intermediate risk, and 217 (55%) adverse risk according to European LeukemiaNet 2017. After induction, 230 of 403 (57.1%) patients achieved complete remission, with no differences between fit (57.3%) and unfit (56%) patients. However, the 2 groups significantly differed in terms of survival (median overall survival, 18 months vs 8 months for fit and unfit patients, respectively) and of 28- and 100-day mortality (4.6% vs 10.7% at 28 days and 14.3% vs 32% at 100 days for fit and unfit patients, respectively). In conclusion, the SIE/SIES/GITMO criteria distinguished patient subgroups with different short- and long-term outcomes after treatment with CPX-351. The update or design of dedicated fitness criteria could represent a future and valid strategy to optimize the use of this specific treatment.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1126-1133"},"PeriodicalIF":7.7000,"publicationDate":"2026-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12915183/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood advances","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1182/bloodadvances.2025017089","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Abstract: CPX-351, a novel liposomal formulation of cytarabine and daunorubicin, represents the standard of care in fit patients with acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) and therapy-related AML (t-AML). Considering its better safety profile than conventional intensive chemotherapy, we investigated its cost-to-benefit ratio, in terms of overall survival and of mortality, in a large multicentric series of AML-MRC and t-AML receiving CPX-351 outside clinical trials between 2019 and 2022. Patients were classified as fit or unfit for intensive chemotherapy through a comprehensive evaluation of age, comorbidities, and performance status by adopting Italian Society of Hematology/Italian Society of Experimental Hematology/Gruppo Italiano per il Trapianto di Midollo Osseo (SIE/SIES/GITMO) criteria. Disease risk was defined according to the European LeukemiaNet 2017 classification. Before treatment start, 328 of 403 (81.4%) patients were classified as fit and 75 of 403 (18.6%) as unfit. Three hundred and ninety-six had a full genetic/cytogenetic profile, with 17 (4%) being categorized as favorable risk, 162 (41%) intermediate risk, and 217 (55%) adverse risk according to European LeukemiaNet 2017. After induction, 230 of 403 (57.1%) patients achieved complete remission, with no differences between fit (57.3%) and unfit (56%) patients. However, the 2 groups significantly differed in terms of survival (median overall survival, 18 months vs 8 months for fit and unfit patients, respectively) and of 28- and 100-day mortality (4.6% vs 10.7% at 28 days and 14.3% vs 32% at 100 days for fit and unfit patients, respectively). In conclusion, the SIE/SIES/GITMO criteria distinguished patient subgroups with different short- and long-term outcomes after treatment with CPX-351. The update or design of dedicated fitness criteria could represent a future and valid strategy to optimize the use of this specific treatment.
CPX-351是一种新型阿糖胞苷和柔柔比星的脂质体制剂，代表了骨髓增生异常相关改变（AML-MRC）和治疗相关急性髓系白血病（tAML）患者的标准护理。考虑到CPX-351的安全性优于常规强化化疗，我们在2019年至2022年期间接受CPX-351的AML-MRC和tAML大型多中心系列临床试验中调查了其总生存期和死亡率的成本/收益比。采用SIE/SIES/GITMO标准，综合评价年龄、合并症及表现状况，对患者进行适合或不适合强化化疗的分类。根据ELN2017分类定义疾病风险。治疗开始前，328/403（81.4%）为适合，75/403（18.6%）为不适合。根据ELN2017, 396人具有完整的遗传/细胞遗传学特征，其中17人（4%）被归类为有利风险，162人（41%）被归类为中等风险，217人（55%）被归类为不利风险。诱导后，230/403例患者（57.1%）达到完全缓解，适合（57.3%）和不适合（56%）之间无差异。然而，两组在生存（健康和不健康患者的中位总生存期为18个月vs 8个月）和28天和100天死亡率方面存在显著差异（健康和不健康患者的28天死亡率分别为4.6% vs 10.7%， 100天死亡率分别为14.3% vs 32%）。综上所述，SIE/SIES/GITMO标准区分了CPX-351治疗后短期和长期结果不同的患者亚组。更新或设计专门的健康标准可以代表未来和有效的策略，以优化这种特殊治疗的使用。
期刊介绍：
Blood Advances, a semimonthly medical journal published by the American Society of Hematology, marks the first addition to the Blood family in 70 years. This peer-reviewed, online-only, open-access journal was launched under the leadership of founding editor-in-chief Robert Negrin, MD, from Stanford University Medical Center in Stanford, CA, with its inaugural issue released on November 29, 2016.
Blood Advances serves as an international platform for original articles detailing basic laboratory, translational, and clinical investigations in hematology. The journal comprehensively covers all aspects of hematology, including disorders of leukocytes (both benign and malignant), erythrocytes, platelets, hemostatic mechanisms, vascular biology, immunology, and hematologic oncology. Each article undergoes a rigorous peer-review process, with selection based on the originality of the findings, the high quality of the work presented, and the clarity of the presentation.
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