Yu Xuedi, Kang Suyi, Ge Junjie, Wang Jingfu
Department of Pediatric Oncology, Shandong Cancer Hospital and Institute, Shandong First Medical University and Shandong Academy of Medical Sciences, Jinan, 250000, China.
BMC Pediatr. 2025 Mar 17;25(1):203. doi: 10.1186/s12887-025-05568-x.
High-risk neuroblastoma (HR-NB) is associated with high metastatic and relapse rates that require intensive multimodal treatment. We evaluated the efficacy and safety of dinutuximab beta as first-line maintenance immunotherapy in pediatric patients with HR-NB in real-world clinical settings in China.
We retrospectively reviewed the clinical records of pediatric patients with newly diagnosed HR-NB in the hospital from October 2021 to November 2023. Patients treated with dinutuximab beta in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) and isotretinoin as the first-line maintenance therapy were included in this study. Among patients with residual disease after completing induction and consolidation treatment, those with partial response (PR) or very good partial response (VGPR) except for bone marrow (BM) residue were also administrated vincristine/irinotecan/temozolomide (VIT) chemotherapy.
Fifty-one patients with newly diagnosed HR-NB who achieved at least PR before immunotherapy were evaluated. At the end of immunotherapy, the objective response rate (ORR) in 33 patients with evidence of disease was 60.6% (95% confidence interval (CI), 42.1-77.1%) and the complete response rate (CRR; n = 18) was 54.5% (95% CI, 36.4-71.9%). The 2-year event-free survival (EFS) rate and overall survival (OS) rate were 80.1% (95% CI, 66.2-88.8%) and 97.6% (95% CI, 84.3-99.7%), respectively. The 2-year EFS rate was higher in patients with CR (94.4%; 95% CI, 66.6-99.2%) than in non-CR patients (72.6%; 95% CI, 53.9-84.7%). Dinutuximab beta was well tolerated in patients and had fewer side effects, which decreased over time. Co-treatment of dinutuximab beta with VIT chemotherapy did not require discontinuation in patients undergoing immunochemotherapy.
The study showed promising efficacy and safety of dinutuximab beta as the first-line maintenance immunotherapy for pediatric patients with HR-NB. Notably, the combination of dinutuximab beta with GM-CSF and VIT chemotherapy could be used for treating patients who did not achieve CR after previous multimodal therapy.
高危神经母细胞瘤（HR-NB）具有高转移率和高复发率，需要强化多模式治疗。我们评估了在中国真实世界临床环境中，地努图希单抗β作为一线维持免疫疗法在HR-NB儿科患者中的疗效和安全性。
我们回顾性分析了2021年10月至2023年11月在我院新诊断为HR-NB的儿科患者的临床记录。本研究纳入接受地努图希单抗β联合粒细胞-巨噬细胞集落刺激因子（GM-CSF）和异维A酸作为一线维持治疗的患者。在完成诱导和巩固治疗后仍有残留疾病的患者中，除骨髓（BM）残留外达到部分缓解（PR）或非常好的部分缓解（VGPR）的患者也接受长春新碱/伊立替康/替莫唑胺（VIT）化疗。
评估了51例新诊断的HR-NB患者，这些患者在免疫治疗前至少达到PR。免疫治疗结束时，33例有疾病证据的患者的客观缓解率（ORR）为60.6%（95%置信区间（CI），42.1-77.1%），完全缓解率（CRR；n = 18）为54.5%（95% CI，36.4-71.9%）。2年无事件生存率（EFS）和总生存率（OS）分别为80.1%（95% CI，66.2-88.8%）和97.6%（95% CI，84.3-99.7%）。CR患者的2年EFS率（94.4%；95% CI，66.6-99.2%）高于非CR患者（72.6%；95% CI，53.9-84.7%）。患者对地努图希单抗β耐受性良好，副作用较少，且随时间减少。地努图希单抗β与VIT化疗联合治疗在接受免疫化疗的患者中无需停药。
该研究显示地努图希单抗β作为HR-NB儿科患者的一线维持免疫疗法具有良好的疗效和安全性。值得注意的是，地努图希单抗β与GM-CSF和VIT化疗联合可用于治疗先前多模式治疗后未达到CR的患者。
