Purpose: Ikaros family zinc finger 1 (IKZF1) mutation may serve as a poor prognostic factor in B-cell acute lymphoblastic leukemia (B-ALL). This study explored the impact of autologous hematopoietic stem cell transplantation (auto-HSCT) on the outcome of B-ALL patients with IKZF1 alterations.

Methods: we detected the IKZF1 mutation at diagnosis in the bone marrow of adult patients with B-ALL, and analyzed the clinical data of these patients retrospectively.

Results: A total of 44 B-ALL patients in CR1 were enrolled in the study, including 12 recipients of auto-HSCT and 32 of allo-HSCT. Auto-HSCT demonstrated comparable 2-year overall survival (OS) (60% vs. 83.3%, p = 0.88) and leukemia-free survival rates (60% vs. 81.1%, p = 0.96) compared to allo-HSCT for patients with negative MRD. Additionally, there was no significant difference in the 2-year cumulative incidence of relapse (CIR) between auto-HSCT and allo-HSCT groups (38.9% vs. 19.5%, p=0.65), and non-relapse mortality (NRM) rates were lower in the auto-HSCT group at 0% compared to 13.2% in the allo-HSCT group (p=0.22). The treatment efficacy was evaluated from a model stratified on the risk classification and minimal residue disease (MRD) status after 1 or 2-3 chemotherapy cycles. The results revealed that after 1 cycle of chemotherapy, auto-HSCT showed promising 2-year OS(100% vs. 85.7% , p=0.35)and LFS rates(100% vs. 85% , p=0.35) , with no significant difference compared to allo-HSCT. Additionally, it displayed lower CIR and NRM(0% vs. 14.3%,p=0.35), indicating potential advantages. For MRD-negative patients after 2-3 cycles of chemotherapy, allo-HSCT may be more beneficial, as shown by the favorable 2-year OS(50% vs. 100%, p=0.018) and LFS rates(50% vs. 100%, p=0.0082), despite similar CIR(33.3% vs. 14.3%,p=0.11).

Conclusion: Auto-HSCT may be an attractive treatment for B-ALL patients of IKZF1 mutation with negative MRD after three chemotherapy cycles,especially after 1 cycle of chemotherapy. These findings provide valuable insights into the treatment efficacy of auto-HSCT and allo-HSCT in different MRD-negative patient subgroups, offering important implications for therapeutic strategies in this specific patient population.

Keywords: acute lymphoblastic leukemia, IKZF1, Allo-HSCT, Auto-HSCT,MRD

Disclosures

No relevant conflicts of interest to declare.

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2024
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