Immune responses against the mutated region of cytoplasmatic NPM1 might contribute to the favorable clinical outcome of AML patients with NPM1 mutations (NPM1mut).

摘要

Immune responses directed against epitopes derived from the mutated region of nucleophosmin 1 (NPM1) by NPM1 mu-specific CD8(+) cytotoxic T cells (CTLs) might be involved in the rejection of NPMlmu myeloid leukemic blasts. NPM1 mutations are one of the most frequent molecular alterations in acute myeloid leukemia (AML) and are an important prognostic marker. The mutations cause an abnormal shift of the NPM1 protein from the nucleus to the cytoplasm, described by Falini et al. AML patients with NPM1mu, but without FLT3 internal tandem duplication (ITD) mutation, show improved overall survival. TVPMimu'/FLT3-ITD-negative patients do not seem to benefit from allogeneic stem cell transplantation in first-line treatment; however, this issue is still under evaluation, further clinical trials are ongoing, and also minimal residual disease (MRD) has to be considered in treatment decision.