Clinical Effects of RUNX1 Mutations on the Outcomes of Patients with Acute Myeloid Leukemia Treated with Allogeneic Hematopoietic Stem-Cell Transplantation

Curr Oncol. 2025 May 22;32(6):294. doi: 10.3390/curroncol32060294.

Abstract

It is reported that AML with RUNX1 mutations is associated with poorer response to conventional chemotherapy, lower rates of complete remission (CR), leukemia-free survival (LFS), and overall survival (OS). We aimed to evaluate the prognostic impact of RUNX1 mutations following allogeneic hematopoietic stem cell transplantation (allo-HSCT) by comparing clinical outcomes in AML patients with and without RUNX1 mutations. We retrospectively analyzed 91 AML patients (33 RUNX1+ and 58 RUNX1-) who received their first HSCT at Peking University People's Hospital. The median age of the cohort was 38 years (range: 6-64), with 73 patients (80%) receiving Haploidentical HSCT and 18 patients (20%) receiving sibling-matched allo-HSCT. In univariate analyses, no significant differences in survival outcomes were observed. For the RUNX1-mutation group and RUNX1-wild-type group, the 2-year cumulative incidence of relapse (CIR) was (12.6% vs. 7.6%, p = 0.472), the 2-year non-relapse mortality (NRM) rate was (9.6% vs. 7.2%, p = 0.747), the 2-year LFS was (77.8% vs. 85.2%, p = 0.426), and the 2-year OS rate was (85.9% vs. 92.7%, p = 0.397). We did not observe any negative impact of RUNX1 mutations on clinical outcomes, suggesting that allo-HSCT (especially Haplo-HSCT) may mitigate the adverse prognostic influence of RUNX1 mutations in AML.

Keywords: RUNX1 gene; acute myeloid leukemia; haploidentical hematopoietic stem cell transplantation; hematological malignancies.

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Core Binding Factor Alpha 2 Subunit* / genetics
  • Female
  • Hematopoietic Stem Cell Transplantation* / methods
  • Humans
  • Leukemia, Myeloid, Acute* / genetics
  • Leukemia, Myeloid, Acute* / mortality
  • Leukemia, Myeloid, Acute* / therapy
  • Male
  • Middle Aged
  • Mutation*
  • Prognosis
  • Retrospective Studies
  • Transplantation, Homologous
  • Treatment Outcome
  • Young Adult

Substances

  • Core Binding Factor Alpha 2 Subunit
  • RUNX1 protein, human