The Prognostic Impact of Additional Molecular and Cytogenetic Abnormalities on AML Patients with NPM1- and/or FLT3-ITD Mutations Receiving Intensive Chemotherapy: Real-World Data from the Greek Registry
Year: 2025
Type: article
Study design: Observational Study
Abstract: Background/Objective: The prognostic impact of additional cytogenetic aberrations and molecular abnormalities (such as MDS-related mutations, mutations in myeloid genes and the KRAS/NRAS mutations) in patients with NPM1- and/or FLT3-ITD-mutated AML remains elusive. Methods: This retrospective, multicentre study of real-world data aimed to investigate the impact of these mutations and cytogenetic abnormalities on the prognosis of patients with NPM1- and/or FLT3-ITD-mutated AML, treated with intensive chemotherapy. Results: In a cohort of 161 patients, the only parameters identified to affect the outcomes (EFS and OS) were the age of the patient, primary refractory disease, the presence of a NPM1 mutation and the use of allogenic stem cell transplantation (allo-SCT) within the first complete remission. More specifically, ages below the median conferred significantly improved outcomes, whereas primary refractory disease exhibited a negative correlation with the EFS and OS. Subsequent subgroup analysis, stratifying patients into three groups (Group 1: NPM1mutated/FLT3wt; Group 2: NPM1mutated/FLT3mutated; Group 3: NPM1wt/FLT3mutated). revealed that allo-SCT in CR1 improved the outcomes (EFS and OS) in Groups 2 and 3, but had no additional impact in Group 1. Conclusions: Age, primary refractory disease and allogenic stem cell transplantation in the first complete response were found to have a prognostic impact on outcomes, Interestingly, no significant association was detected between the poor prognostic cytogenetic abnormalities or the presence of additional mutations in myeloid genes, MDS-related genes or KRAS/NRAS genes and the outcomes in any group of patients.
Source: Cancers
Authors: Ioanna Lazana, Maria Papathanassiou, Ioannis Konstantellos, Tatiana Tzenou, Anastasia Kopsaftopoulou, Maria Liga, Vasiliki Violaki, Lydia Kyriazopoulou, Konstantinos Gkirkas, Apostolia Papalexandri, Eleni Plata, Evrydiki Michalis, Theoni Leonidopoulou, Maria Garofalaki, Anastasia Sioni, Irene Tziotziou, Chrysavgi Lalayanni, Dimitrios Kiousiafes, Theodoros P. Vassilakopoulos, Eleni Kapsali, Alexandros Spyridonidis, Ioannis Baltadakis, Maria Angelopoulou, Ioanna Sakellari, Panagiotis Tsirigotis
Institutions: National and Kapodistrian University of Athens, University General Hospital Attikon, G. Papanikolaou General Hospital, Evangelismos Hospital, Laiko General Hospital of Athens, General University Hospital of Patras, Chania General Hospital ”St. George”, University Hospital of Ioannina, General Hospital of Athens G. Genimatas, Sismanoglio General Hospital, St Savas Hospital
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $3,499
Funder: Pfizer
OpenAlex ID: W4407640749