Division of Hematology/Oncology/BMT, Department of Pediatrics, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio, USA.
Department of Radiology, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, Washington, USA.
Pediatr Blood Cancer. 2023 Aug;70(8):e30418. doi: 10.1002/pbc.30418. Epub 2023 May 18.
Diagnostic mIBG (meta-iodobenzylguanidine) scans are an integral component of response assessment in children with high-risk neuroblastoma. The role of end-of-induction (EOI) Curie scores (CS) was previously described in patients undergoing a single course of high-dose chemotherapy (HDC) and autologous hematopoietic cell transplant (AHCT) as consolidation therapy.
We now examine the prognostic significance of CS in patients randomized to tandem HDC and AHCT on the Children's Oncology Group (COG) trial ANBL0532.
A retrospective analysis of mIBG scans obtained from patients enrolled in COG ANBL0532 was performed. Evaluable patients had mIBG-avid, International Neuroblastoma Staging System (INSS) stage 4 disease, did not progress during induction therapy, consented to consolidation randomization, and received either single or tandem HDC (n = 80). Optimal CS cut points maximized the outcome difference (≤CS vs. >CS cut-off) according to the Youden index.
For recipients of tandem HDC, the optimal cut point at diagnosis was CS = 12, with superior event-free survival (EFS) from study enrollment for patients with CS ≤ 12 (3-year EFS 74.2% ± 7.9%) versus CS > 12 (59.2% ± 7.1%) (p = .002). At EOI, the optimal cut point was CS = 0, with superior EOI EFS for patients with CS = 0 (72.9% ± 6.4%) versus CS > 0 (46.5% ± 9.1%) (p = .002).
In the setting of tandem transplantation for children with high-risk neuroblastoma, CS at diagnosis and EOI may identify a more favorable patient group. Patients treated with tandem HDC who exhibited a CS ≤ 12 at diagnosis or CS = 0 at EOI had superior EFS compared to those with CS above these cut points.
诊断用 mIBG（间碘苄胍）扫描是高危神经母细胞瘤患者反应评估的一个组成部分。此前，在接受单一疗程高剂量化疗（HDC）和自体造血细胞移植（AHCT）作为巩固治疗的患者中，已经描述了诱导结束（EOI）居里分数（CS）的作用。
我们现在检查了在儿童肿瘤学组（COG）试验 ANBL0532 中随机接受双 HDC 和 AHCT 的患者 CS 的预后意义。
对 COG ANBL0532 入组患者的 mIBG 扫描进行了回顾性分析。可评估的患者具有 mIBG 阳性、国际神经母细胞瘤分期系统（INSS）分期 4 期疾病，在诱导治疗期间没有进展，同意进行巩固随机化，并且接受了单 HDC 或双 HDC（n=80）。根据 Youden 指数，最优 CS 切点最大化了结果差异（CS ≤ vs. >CS 截止值）。
对于接受双 HDC 的患者，诊断时的最佳切点为 CS=12，CS ≤ 12 的患者从研究入组开始的无事件生存（EFS）更优（3 年 EFS 为 74.2%±7.9%），而 CS > 12 的患者为 59.2%±7.1%（p=0.002）。在 EOI 时，最佳切点为 CS=0，CS=0 的患者的 EOI EFS 更高（72.9%±6.4%），而 CS > 0 的患者为 46.5%±9.1%（p=0.002）。
在高危神经母细胞瘤儿童接受双移植的情况下，诊断时的 CS 和 EOI 可能确定更有利的患者群体。在诊断时 CS ≤ 12 或 EOI 时 CS = 0 的接受双 HDC 治疗的患者与 CS 高于这些切点的患者相比，EFS 更高。
