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Prognostic value of measurable residual disease at allogeneic transplantation for adults with core binding factor acute myeloid leukemia in complete remission


Pretransplant measurable residual disease (MRD) has been shown to be associated with relapse incidence following allogeneic hematopoietic cell transplantation (HCT) for acute myeloid leukemia (AML). However, it remains less clear whether pretransplant MRD status affects transplant outcomes in core binding factor AML (CBF-AML). We retrospectively evaluated the effect of pretransplant MRD, which was measured by a polymerase chain reaction of RUNX1-RUNX1T1 or CBFB-MYH11 fusion transcripts, on transplant outcomes for a cohort of 959 adult patients with t(8;21) or inv(16) AML treated by allogeneic HCT during complete remission (CR), between 2000 and 2018. Multivariate analysis showed the absence of pretransplant MRD was significantly associated with lower relapse (hazard ratio [HR], 0.46; P < 0.001), treatment failure (HR, 0.66; P = 0.004), and overall mortality (HR, 0.72; P = 0.037) among patients with t(8;21). However, pretransplant MRD negativity was not associated with relapse (HR, 0.73; P = 0.420), treatment failure (HR, 0.64; P = 0.063), or overall mortality (HR, 0.69; P = 0.149) among patients with inv(16). In subgroup analysis, pretransplant MRD status significantly affected relapse and LFS only in patients with t(8;21) undergoing allogeneic HCT during CR2. In conclusion, our data demonstrate the different prognostic values of pretransplant MRD for CBF-AML, highlighting the need to develop effective therapeutic strategies for such MRD-positive patients.

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Fig. 1: The probability of overall survival (OS) and leukemia-free survival (LFS) in patients with t(8;21) or inv(16) acute myeloid leukemia, according to pretransplant measurable residual disease (MRD) status.
Fig. 2: The cumulative incidence of relapse and non-relapse mortality (NRM) in patients with t(8;21) or inv(16) acute myeloid leukemia, according to pre-transplant measurable residual disease (MRD) status.


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The authors thank all the physicians and staff at the participating hospitals, the Japan Marrow Donor Program, and the cord blood banks for providing clinical data to the Transplant Registry Unified Management Program of the Japanese Data Center for Hematopoietic Cell Transplantation. This work was supported in part by the Practical Research Project for Allergic Diseases and Immunology (Research Technology of Medical Transplantation) from the Japan Agency for Medical Research and Development, AMED under grant 18ek0510023h0002.

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Correspondence to Takaaki Konuma.

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Konuma, T., Kondo, T., Masuko, M. et al. Prognostic value of measurable residual disease at allogeneic transplantation for adults with core binding factor acute myeloid leukemia in complete remission. Bone Marrow Transplant 56, 2779–2787 (2021).

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