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Naxitamab-combination Therapy for the Treatment of Patients With Refractory and/or Relapsed High-risk Neuroblastoma

Erin Murphy Trovillion1, Meghan Michael1, Lauren Brown1

  • 1From Atrium Health, Levine Children's Hospital, Charlotte, NC.

Journal of Pediatric Hematology/Oncology
|August 12, 2025
PubMed

Insights

A novel chemoimmunotherapy combining naxitamab and GM-CSF with cyclophosphamide and topotecan shows promise for refractory/relapsed high-risk neuroblastoma. This treatment was well-tolerated in heavily pretreated patients with poor prognoses.

Area of Science:

  • Pediatric Oncology
  • Immunotherapy
  • Neuroblastoma Research

Background:

  • Refractory/relapsed (R/R) high-risk (HR) neuroblastoma has limited treatment options.
  • There is a critical need for novel therapeutic combinations for these patients.

Purpose of the Study:

  • To evaluate the safety and tolerability of a combined chemoimmunotherapy regimen.
  • To assess the potential efficacy of naxitamab, GM-CSF, cyclophosphamide, and topotecan in R/R HR neuroblastoma.

Main Methods:

  • A case series of 4 patients with R/R HR neuroblastoma.
  • Treatment involved naxitamab (anti-GD2 antibody) and granulocyte-macrophage colony-stimulating factor (GM-CSF) combined with cyclophosphamide and topotecan.
  • Treatment administered between August 2021 and December 2022.

Main Results:

  • The combined chemoimmunotherapy regimen was well-tolerated.
  • This approach was administered to heavily pretreated patients with poor prognoses.
  • No specific efficacy data (e.g., response rates, survival) were detailed in this abstract.

Conclusions:

  • The combination of naxitamab, GM-CSF, cyclophosphamide, and topotecan appears to be a well-tolerated treatment option.
  • This chemoimmunotherapy regimen warrants further investigation for R/R HR neuroblastoma.
  • Further studies are needed to establish the efficacy of this novel combination.
Keywords:
immunotherapynaxitamabneuroblastoma

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