Allogeneic hematopoietic stem cell transplantation for adult acute myeloid leukemia patients with nucleoporin 98 (NUP98) gene rearrangements: A real-world study in China

Abstract Nucleoporin 98 (NUP98) gene rearrangements comprise a family of rare recurrent alterations in acute myeloid leukemia (AML), and conferred dismal outcomes. The efficacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) were still unclear. In this retrospective, multicenter, real-world study, we enrolled 26 de novo adult AML patients with NUP98 rearrangements who received first allo-HSCT. The 100-day cumulative incidence of grade II–IV acute graft-versus-host disease (GVHD) after allo-HSCT was 19.2% (95% CI, 3.8–34.7%) and the 2-year cumulative incidences of moderate to severe chronic GVHD after allo-HSCT was 47.5% (95% CI, 17.0–77.9%). Ten (38.5%) patient received maintenance therapies after allo-HSCT. Among the 24 patients with MRD monitoring regularly, all of them achieved MRD negative after allo-HSCT, and 21 (87.5%) achieved persistent MRD negative until the last follow-up. The 2-year cumulative incidence of relapse and non-relapse mortality after allo-HSCT was 17.2% (95% CI, 1.4–33.1%) and 4.6% (95% CI, 0–13.7%), respectively. The 2-year probabilities of leukemia-free survival and overall survival after allo-HSCT were 78.2% (95%CI, 62.8–97.2%) and 86.3% (95%CI, 73.0–100%), respectively. In summary, we firstly identify the efficacy and safety of allo-HSCT in adult AML patients with NUP98 rearrangement, which should be further confirmed in prospective cohorts with a longer follow-up.