White blood cell count as a powerful prognostic marker and treatment guide in paediatric acute myeloid leukaemia with NUP98 rearrangement

TY - JOUR

T1 - White blood cell count as a powerful prognostic marker and treatment guide in paediatric acute myeloid leukaemia with NUP98 rearrangement

AU - Shi, Hui

AU - Shu, Wenjing

AU - Tao, Yu

AU - Jia, Xueming

AU - Shen, Yali

AU - Yang, Hua

AU - Xun, Yang

AU - Baptista-Hon, Daniel Tomas

AU - You, Hua

N1 - Publisher Copyright: © 2025 British Society for Haematology and John Wiley & Sons Ltd.

PY - 2025/10

Y1 - 2025/10

N2 - NUP98-rearranged paediatric acute myeloid leukaemia (NUP98-r pAML) has an extremely poor prognosis, and the impact of clinical parameters and therapeutic schemes on its outcomes remains unclear. We conducted a retrospective study of the largest pAML cohort (1779 patients) and found that NUP98-r pAML has the worst prognosis among all subtypes. Furthermore, we identified white blood cell (WBC) count as the sole predictor of overall survival (OS) in NUP98-r pAML patients and validated its adverse prognostic impact in both external paediatric and adult cohorts. NUP98-r pAML patients were categorized into low-risk (WBC count ≤150 × 109/L) and high-risk (WBC count >150 × 109/L) groups based on WBC levels. Haematopoietic stem cell transplantation (HSCT) significantly improved OS and reduced the cumulative incidence of relapse (CIR) in the high-risk group but not in the low-risk group. Bortezomib significantly increased OS in NUP98::NSD1 patients within the low-risk group, and the combination of bortezomib and HSCT significantly enhanced OS in the entire NUP98-r pAML cohort. CD33 antibody (Gemtuzumab ozogamicin, GO) is not recommended for the entire NUP98-r pAML patients. In summary, WBC count is a pivotal marker for risk stratification and treatment decision-making in NUP98-r pAML patients.

AB - NUP98-rearranged paediatric acute myeloid leukaemia (NUP98-r pAML) has an extremely poor prognosis, and the impact of clinical parameters and therapeutic schemes on its outcomes remains unclear. We conducted a retrospective study of the largest pAML cohort (1779 patients) and found that NUP98-r pAML has the worst prognosis among all subtypes. Furthermore, we identified white blood cell (WBC) count as the sole predictor of overall survival (OS) in NUP98-r pAML patients and validated its adverse prognostic impact in both external paediatric and adult cohorts. NUP98-r pAML patients were categorized into low-risk (WBC count ≤150 × 109/L) and high-risk (WBC count >150 × 109/L) groups based on WBC levels. Haematopoietic stem cell transplantation (HSCT) significantly improved OS and reduced the cumulative incidence of relapse (CIR) in the high-risk group but not in the low-risk group. Bortezomib significantly increased OS in NUP98::NSD1 patients within the low-risk group, and the combination of bortezomib and HSCT significantly enhanced OS in the entire NUP98-r pAML cohort. CD33 antibody (Gemtuzumab ozogamicin, GO) is not recommended for the entire NUP98-r pAML patients. In summary, WBC count is a pivotal marker for risk stratification and treatment decision-making in NUP98-r pAML patients.

KW - acute myeloid leukaemia

KW - NUP98 rearrangement

KW - prognosis

KW - treatment

KW - white blood cell count

UR - https://www.scopus.com/pages/publications/105013237907

U2 - 10.1111/bjh.70096

DO - 10.1111/bjh.70096

M3 - Article

C2 - 40814214

AN - SCOPUS:105013237907

SN - 0007-1048

VL - 207

SP - 1464

EP - 1475

JO - British Journal of Haematology

JF - British Journal of Haematology

IS - 4

ER -