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ORIGINAL RESEARCH article

Front. Immunol.

Sec. Cancer Immunity and Immunotherapy

Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1604866

This article is part of the Research TopicExploring T Cell Driven Immunotherapies: From CAR T and TILs to T Cell EngagersView all 6 articles

Low-dose glucocorticoid improves progression-free survival of children with B cell acute lymphoblastic leukaemia following chimeric antigen receptor T-cell therapy

Provisionally accepted
Hui  ZhangHui ZhangYuxuan  WangYuxuan WangQi  JiQi JiQinyi  ZhangQinyi ZhangChonglian  QiuChonglian QiuSaihu  HuangSaihu HuangXingqiang  DongXingqiang DongJian  PanJian PanJun  LuJun LuZhenjiang  BaiZhenjiang BaiShaoyan  HuShaoyan HuShuiyan  WuShuiyan Wu*
  • Children's Hospital of Soochow University, Suzhou, China

The final, formatted version of the article will be published soon.

Background The prognostic impact of immunosuppressant therapies for cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), along with the outcomes and prognosis of children with relapsed/refractory B cell acute lymphoblastic leukaemia (B-ALL) undergoing chimeric antigen receptor (CAR) T-cell therapy, varies across populations. However, studies specifically focusing on these factors in the paediatric B-ALL population remain limited. Methods We investigated the effects of immunosuppressants on outcome efficacy and prognosis in a retrospective cohort of 120 patients treated with CAR T-cell infusion at a single institution from March 2017 to August 2023. The 30-day complete response rate, progression-free survival (PFS), overall survival (OS), and event-free survival (EFS) were evaluated. Results The median age of the patients was 8.0 years (range, 2.2–18.0 years). Following CAR T-cell therapy, 91.67 % of patients developed CRS and 25.83 % developed ICANS. At 1 month after CAR T-cell infusion, 70.83 % of patients received tocilizumab (TCZ), 24.17 % received ruxolitinib (RUX), and 50.83 % received glucocorticoids (GC) for CRS or ICANS management. By day 30, 92.08 % of patients achieved a complete response. The complete-response rates did not differ between the GC and non-GC, TCZ and non-TCZ, or RUX and non-RUX groups. The median follow-up time was 20.6 months (range, 4.26–38.82 months). OS, EFS, and PFS did not significantly differ between the RUX and non-RUX or TCZ and non-TCZ groups. However, patients receiving low-dose GC (≤8 mg kg⁻¹) exhibited better PFS than the non-GC group, with multivariable analysis demonstrating low-dose GC as an independent protective factor for PFS (hazard ratio, 0.45; 95 % confidence interval, 0.21–0.96). Conclusions In the context of CRS/ICANS management, low-dose GC independently confers long-term PFS benefits to pediatric B-ALL patients without compromising CAR T-cell activity when using appropriate GC, TCZ, or RUX regimens.

Keywords: cytokine release syndrome, Immune Effector Cell-Associated Neurotoxicity Syndrome, glucocorticoid, chimeric antigen receptor T cell therapy, B cell acute lymphoblastic leukaemia

Received: 02 Apr 2025; Accepted: 20 Oct 2025.

Copyright: © 2025 Zhang, Wang, Ji, Zhang, Qiu, Huang, Dong, Pan, Lu, Bai, Hu and Wu. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: Shuiyan Wu, wushuiyany@163.com

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