A pilot study of genomic-guided induction therapy followed by immunotherapy with difluoromethylornithine maintenance for high-risk neuroblastoma.

ABSTRACT:

Background

Survival for patients with high-risk neuroblastoma (HRNB) remains poor despite aggressive multimodal therapies.

Aims

To study the feasibility and safety of incorporating a genomic-based targeted agent to induction therapy for HRNB as well as the feasibility and safety of adding difluoromethylornithine (DFMO) to anti-GD2 immunotherapy.

Methods

Twenty newly diagnosed HRNB patients were treated on this multicenter pilot trial. Molecular tumor boards selected one of six targeted agents based on tumor-normal whole exome sequencing and tumor RNA-sequencing results. Treatment followed standard upfront HRNB chemotherapy with the addition of the selected targeted agent to cycles 3-6 of induction. Following consolidation, DFMO (750 mg/m² twice daily) was a

SUBMITTER: Kraveka JM 

PROVIDER: S-EPMC9675391 | biostudies-literature | 2022 Nov

REPOSITORIES: biostudies-literature