[Prognostic analysis of NUP98 rearrangement in patients with pediatric acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation].
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Objective: To analyze the prognosis and impact of NUP98 rearrangements (NUP98-r) pre- and post-allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with pediatric acute myeloid leukemia (pAML) with NUP98-r who underwent allo-HSCT. Methods: This was a retrospective, single-arm, observational cohort study. This study enrolled patients with pAML with NUP98-r who underwent allo-HSCT at Peking University People's Hospital from April 2018 to October 2024. The clinical characteristics, NUP98-r status pre- and post-allo-HSCT, relapse, and survival outcomes of the patients were collected. The impact of NUP98-r status pre- and post-allo-HSCT on prognosis was analyzed. Results: Among 31 patients with pAML with NUP98-r who underwent allo-HSCT, 16 (51.6%) were males and 15 (48.4%) were females, with a median age of 8 years (range: 2-18 years). The median follow-up time was 35 months (range: 6-66 months). The most common NUP98-r subtype was NUP98::NSD1 (22 patients, 71.0%), followed by NUP98::HOXA9 (5 patients, 16.1%). Concurrent FLT3-ITD mutation was present in 18 patients (58.1%) and WT1 mutation in 14 patients (45.2%). Seven patients relapsed post-allo-HSCT, with a median time to relapse of 6 months (range: 3-48 months). The 100-day cumulative incidence of grade Ⅱ-Ⅳ acute graft-versus-host disease (GVHD) was (35.4±8.6) %, and the 3-year cumulative incidence of chronic GVHD was (64.8±9.2) %. The 3-year overall survival, event-free survival (EFS), and cumulative incidence of relapse (CIR) rates were (74.0±9.6) %, (63.9±10.1) %, and (21.3±8.7) %, respectively. The 3-year EFS was higher in patients who were negative for NUP98-r pre-allo-HSCT [ (92.3±7.4) %] than in those positive for NUP98-r [ (42.3± 15.3) %] (P=0.044). The 3-year CIR was higher in patients positive for NUP98-r pre-allo-HSCT [ (34.8±14.6) %] than in those negative for NUP98-r (0) (P=0.036). All patients who were positive for NUP98-r within the first 3 months post-allo-HSCT subsequently relapsed. Conclusion: Allo-HSCT is an effective method for treating NUP98-r pAML, and NUP98-r positivity pre-allo-HSCT is associated with higher CIR and lower EFS. Therefore, efforts should be made to achieve a negative NUP98-r status through treatment before undergoing allo-HSCT.