Risk stratification using FLT3 and NPM1 in acute myeloid leukemia patients autografted in first complete remission.

Shouval, Roni; Labopin, Myriam; Bomze, David; Baerlocher, Gabriela M.; Capria, Saveria; Blaise, Didier; Hänel, Mathias; Forcade, Edouard; Huynh, Anne; Saccardi, Riccardo; Milone, Giuseppe; Zuckerman, Tsila; Reményi, Péter; Versluis, Jurjen; Esteve, Jordi; Gorin, Norbert Claude; Mohty, Mohamad; Nagler, Arnon (2020). Risk stratification using FLT3 and NPM1 in acute myeloid leukemia patients autografted in first complete remission. Bone marrow transplantation, 55(12), pp. 2244-2253. Springer Nature 10.1038/s41409-020-0936-z

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FLT3-ITD and NPM1 mutation refine prognostic stratification in acute myeloid leukemia (AML) with intermediate-risk cytogenetics. However, data on their role in patients undergoing autologous stem cell transplantation (Auto-SCT) as post-remission therapy (PRT) are limited. We therefore sought to retrospectively evaluate the role of FLT3-ITD and NPM1 in a cohort of AML patients (n = 405) with intermediate-risk cytogenetics, autografted in first complete remission (CR1). Patients were transplanted between 2000 and 2014 and reported to the European Society for Blood and Marrow Transplantation (EBMT) registry. Leukemia-free survival (LFS) was the primary outcome. Median follow-up was 5.5 years. FLT3-ITDneg/NPM1WT was the leading molecular subtype (50%), followed by FLT3-ITDneg/NPM1mut (30%). In the univariate analysis, molecular subtype was associated with LFS, overall survival (OS), and relapse incidence (RI) (p < 0.001); 5-year LFS: FLT3-ITDneg/NPM1mut 62%, FLT3-ITDpos/NPM1mut 38%, FLT3-ITDneg/NPM1WT 32%, and FLT3-ITDpos/NPM1WT 21%. At 5 years, OS and RI in the FLT3-ITDneg/NPM1mut subtype were 74% and 35%, respectively. The corresponding OS and RI in other subtypes were below 48% and over 57%. In a Cox multivariable model, molecular subtype was the strongest predictor of LFS, OS, and relapse. In conclusion, AML patients with intermediate-risk cytogenetics and FLT3-ITDneg/NPM1mut experience favorable outcomes when autografted in CR1, suggesting that Auto-SCT is a valid PRT option.

Item Type:

Journal Article (Original Article)


04 Faculty of Medicine > Department of Haematology, Oncology, Infectious Diseases, Laboratory Medicine and Hospital Pharmacy (DOLS) > Clinic of Haematology and Central Haematological Laboratory

UniBE Contributor:

Baerlocher, Gabriela M.


600 Technology > 610 Medicine & health




Springer Nature




Pierrette Durand Lüthi

Date Deposited:

15 Jun 2020 09:57

Last Modified:

27 Nov 2020 01:31

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