Our study describes an AML patient whose leukemia cells carried the NPM1c+ mutation, and who was the recipient of allogeneic HSCT from a haploidentical donor. The patient raised a robust allorestricted CD8+ T cell response directed against the NPM1wt protein.
Favourably, the response against NPM1wt was not accompanied by side effects such as GvHD. Moreover, the induction of a high NPM1wt specific response coincided with the decrease in NPM1c+ transcripts detected, implying a beneficial graft versus leukemia effect.
On the basis of these results, we suppose that TCRs from allorestricted NPM1wt-specific T cells are worth studying in other recipients of grafts from haploidentical donors as a possible tool for TCR gene therapy.