Hematopoietic stem cell transplantation for adult patients with isolated NPM1 mutated acute myeloid leukemia in first remission.

Poiré, Xavier; Labopin, Myriam; Polge, Emmanuelle; Blaise, Didier; Chevallier, Patrice; Maertens, Johan; Deconinck, Eric; Forcade, Edouard; Rambaldi, Alessandro; Baerlocher, Gabriela M.; Zuckerman, Tsila; Volin, Liisa; Schouten, Harry C; Ifrah, Norbert; Mohty, Mohamad; Esteve, Jordi; Nagler, Arnon (2019). Hematopoietic stem cell transplantation for adult patients with isolated NPM1 mutated acute myeloid leukemia in first remission. American journal of hematology, 94(2), pp. 231-239. Wiley-Liss 10.1002/ajh.25355

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Acute myeloid leukemia (AML) in first remission (CR1) with isolated NPM1 mutation (iNPM1m) is considered a good prognosis genotype, although up to one-third relapse. To evaluate the best transplant strategy, we retrospectively compared autologous stem cell transplantation (auto-SCT), related (MSD), and fully matched unrelated (MUD) allogeneic stem cell transplantation (allo-SCT). We identified 256 adult patients including 125 auto-SCT, 72 MSD, and 59 MUD. The 2-year leukemia-free survival (LFS) was 62% in auto-SCT, 69% in MUD, and 81% in MSD (P = .02 for MSD vs others). The 2-year overall survival (OS) was not different among auto-SCT, MUD, and MSD, reaching 83% (P = .88). The 2-year non-relapse mortality (NRM) was 2.5% in auto-SCT and 7.5% in allo-SCT (P = .04). The 2-year cumulative incidence of relapse (RI) was higher after auto-SCT (30%) than after MUD (22%) and MSD (12%, P = .01). In multivariate analysis, MSD versus auto-SCT but not MUD versus auto-SCT was associated with lower RI (P < .01 and P = .13, respectively) and better LFS (P = .01 and P = .31, respectively). Age correlated with higher NRM (P < .01). Allo-SCT using MSD appears as a reasonable transplant option for young patients with iNPM1m AML in CR1. Auto-SCT was followed by worse RI and LFS, but similar OS to both allo-SCT modalities.

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
04 Faculty of Medicine > Pre-clinic Human Medicine > BioMedical Research (DBMR) > Unit Childrens Hospital > Forschungsgruppe Hämatologie (Erwachsene)

UniBE Contributor:

Baerlocher, Gabriela M.


600 Technology > 610 Medicine & health








Pierrette Durand Lüthi

Date Deposited:

20 Feb 2019 16:22

Last Modified:

03 Nov 2019 12:20

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