Department of Pediatrics, CHRISTUS Children's, Baylor College of Medicine, San Antonio, Texas, USA.
Department of Pediatrics, Nemours Children's Hospital, Wilmington, Delaware, USA.
Pediatr Blood Cancer. 2025 Jan;72(1):e31407. doi: 10.1002/pbc.31407. Epub 2024 Nov 6.
Tumor invasion of the spinal canal is detected radiographically in approximately 15% of patients with newly diagnosed neuroblastoma (NB). The optimal clinical approach to maintain excellent survival outcomes while minimizing long-term sequelae is yet to be defined.
Patients with intermediate-risk neuroblastoma (IR-NB) and radiographically identified intraspinal tumors who were treated on the Children's Oncology Group study ANBL0531 were studied prospectively to evaluate neurologic outcomes related to cord compression. Patients were defined as being symptomatic versus asymptomatic based on reporting of neurologic motor deficits at diagnosis. Patient characteristics, tumor biology, chemotherapy treatment, surgical interventions, and neurologic and disease outcomes are reported.
Of the 92 patients with intraspinal tumors, 42 (46%) were symptomatic and most (73%) had complete resolution of symptoms. Age, degree of motor deficit, and duration of symptoms at diagnosis were not associated with complete resolution. While symptomatic patients were more likely to undergo upfront laminectomy, laminectomy was not associated with improvement of motor symptoms. Administration of additional chemotherapy beyond initial treatment assigned per protocol to achieve the treatment end point was not associated with achieving symptom resolution.
Patients presenting with motor deficits due to intraspinal tumor had excellent survival and favorable neurologic outcomes, with the majority reporting complete resolution of motor symptoms regardless of severity and duration of symptoms at diagnosis or neurosurgical intervention. Prompt diagnosis and initiation of first-line chemotherapy treatment remain priority, while neurosurgical intervention should be reserved for patients with rapid neurologic deterioration. Biology-based therapy and tumor response should continue to be used to maintain favorable outcomes.
在新诊断的神经母细胞瘤（NB）患者中，约有 15%的患者在影像学上发现肿瘤侵犯椎管。目前尚未确定保持良好生存结果的最佳临床方法，同时最小化长期后遗症。
在儿童肿瘤学组研究 ANBL0531 中接受治疗的具有中危神经母细胞瘤（IR-NB）和影像学上发现的椎管内肿瘤的患者前瞻性研究了与脊髓压迫相关的神经功能结果。根据诊断时神经运动功能缺陷的报告，患者被定义为有症状或无症状。报告了患者特征、肿瘤生物学、化疗治疗、手术干预以及神经和疾病结局。
在 92 例有椎管内肿瘤的患者中，42 例（46%）有症状，大多数（73%）的症状完全缓解。年龄、运动缺陷程度和诊断时症状的持续时间与完全缓解无关。虽然有症状的患者更可能接受 upfront 椎板切除术，但椎板切除术与运动症状的改善无关。根据方案给予初始治疗以外的额外化疗以达到治疗终点与症状缓解无关。
因椎管内肿瘤导致运动功能障碍的患者具有良好的生存和良好的神经结局，大多数患者报告无论症状的严重程度和持续时间如何，以及诊断或神经外科干预，运动症状均完全缓解。及时诊断和开始一线化疗治疗仍然是优先事项，而神经外科干预应保留给神经功能迅速恶化的患者。基于生物学的治疗和肿瘤反应应继续用于维持良好的结局。
