Impact of fitness categorization according to SIE/SIES/GITMO criteria in therapy-related and AML-MRC receiving CPX-351
Open Access
- 12 February 2026
- journal article
- research article
- Published by American Society of Hematology in Blood Advances
- Vol. 10 (4) , 1126-1133
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.Keywords
This publication has 22 references indexed in Scilit:
- Moving toward individualized target-based therapies in acute myeloid leukemiaAnnals of Oncology, 2022
- Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELNBlood, 2022
- Multisite 11-year experience of less-intensive vs intensive therapies in acute myeloid leukemiaBlood, 2021
- CPX-351 treatment in secondary acute myeloblastic leukemia is effective and improves the feasibility of allogeneic stem cell transplantation: results of the Italian compassionate use programBlood Cancer Journal, 2020
- Final safety and efficacy results from the CPX-351 early access program for older patients with high-risk or secondary acute myeloid leukemiaLeukemia & Lymphoma, 2020
- Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panelBlood, 2017
- The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemiaBlood, 2016
- Geriatric assessment predicts survival for older adults receiving induction chemotherapy for acute myelogenous leukemiaBlood, 2013
- Consensus-based definition of unfitness to intensive and non-intensive chemotherapy in acute myeloid leukemia: a project of SIE, SIES and GITMO group on a new tool for therapy decision makingLeukemia, 2013
- Allogeneic transplantation for therapy-related myelodysplastic syndrome and acute myeloid leukemiaBlood, 2010