Outcomes of patients with intermediate-risk neuroblastoma presenting with motor deficits relating to intraspinal tumor extension: A report from the Children's Oncology Group study ANBL0531

Pediatr Blood Cancer. 2025 Jan;72(1):e31407. doi: 10.1002/pbc.31407. Epub 2024 Nov 6.

Abstract

Background: 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.

Methods: 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.

Results: 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.

Conclusion: 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.

Keywords: intermediate risk; laminectomy; neuroblastoma; neurologic; paralysis; scoliosis; spinal cord compression.

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Infant
  • Male
  • Neuroblastoma* / complications
  • Neuroblastoma* / mortality
  • Neuroblastoma* / pathology
  • Neuroblastoma* / therapy
  • Prospective Studies
  • Spinal Cord Compression / etiology
  • Spinal Cord Compression / pathology
  • Spinal Cord Neoplasms / complications
  • Spinal Cord Neoplasms / mortality
  • Spinal Cord Neoplasms / pathology
  • Spinal Cord Neoplasms / therapy
  • Spinal Neoplasms / complications
  • Spinal Neoplasms / pathology