Department of Childhood and Adolescent Cancer, Gustave Roussy Cancer Campus, Villejuif, France.
Department of Imaging, Gustave Roussy Cancer Campus, Villejuif, France.
Pediatr Blood Cancer. 2025 Jan;72(1):e31350. doi: 10.1002/pbc.31350. Epub 2024 Oct 14.
Persistent metaiodobenzylguanidine (mIBG)-positive skeletal metastases post induction in high-risk neuroblastoma correlate with a poor outcome. The aim of this study was to investigate the potential rationale for a prospective randomized study evaluating the impact on event-free survival of the irradiation of residual oligo-skeletal metastases.
Patients over 1 year with a stage M neuroblastoma treated between 2000 and 2020 at Gustave Roussy were identified. Patients with a positive mIBG scan at diagnosis and persistent skeletal metastases after high-dose chemotherapy (HDC) were included. Data were retrospectively collected and mIBG scans reviewed by two nuclear medicine physicians.
Persistent skeletal uptake after HDC was observed in 30/201 patients (15%). Four patients reached a complete response at the end of maintenance treatment and did not relapse (median follow-up [FU] 8 years [1.8-11.8]), while two patients had progressive disease during maintenance. Among the 24 patients with persistent skeletal uptakes at the end of treatment, seven had a persistent response (median FU 8.2 years [4-15.6]). Median SIOPEN (International Society of Paediatric Oncology European Neuroblastoma) scores post consolidation and at the end of treatment were, respectively, 2 [1-6] and 2 [0-4] for patients with persistent responses compared to 4 [1-28] and 2 [1-17] for patients with progressive diseases. Median SIOPEN score at progression was 34 [2-56].
Our study underlines that only a minority of patients had persistent skeletal mIBG-positive scans after HDC. Recurrence mainly occurred in disease sites present at diagnosis that cleared with chemotherapy. On-therapy control of the disease is the main challenge. These results highlight the complexity of conducting a randomized study exploring this strategy.
高危神经母细胞瘤诱导后持续性碘代苄胍（mIBG）阳性骨骼转移与不良预后相关。本研究旨在探讨对残留寡发性骨骼转移进行照射以评估对无事件生存影响的前瞻性随机研究的潜在依据。
回顾性分析 2000 年至 2020 年在古斯塔夫·鲁西（Gustave Roussy）接受治疗的 1 年以上、M 期神经母细胞瘤患者。纳入初诊时 mIBG 扫描阳性且大剂量化疗（HDC）后仍有骨骼转移的患者。收集数据并由两名核医学医生回顾 mIBG 扫描。
30/201 例患者（15%）在 HDC 后出现持续性骨骼摄取。4 例患者在维持治疗结束时达到完全缓解且未复发（中位随访 8 年[1.8-11.8]），而 2 例患者在维持治疗期间疾病进展。24 例治疗结束时骨骼持续摄取的患者中，7 例持续缓解（中位随访 8.2 年[4-15.6]）。与疾病进展患者相比，持续缓解患者巩固治疗后和治疗结束时 SIOPEN（国际小儿肿瘤协会欧洲神经母细胞瘤）评分分别为 2[1-6]和 2[0-4]，疾病进展患者分别为 4[1-28]和 2[1-17]。进展时 SIOPEN 评分中位数为 34[2-56]。
本研究强调，只有少数患者在 HDC 后出现持续性骨骼 mIBG 阳性扫描。复发主要发生在化疗清除的初诊时存在的疾病部位。疾病的治疗期控制是主要挑战。这些结果突出了开展探索该策略的随机研究的复杂性。
