# ANBL1221 expansion: irinotecan, temozolomide and dinutuximab with GM-CSF in refractory or relapsed neuroblastoma

Source: https://onco.cc/key-papers/paper-anbl1221-expansion-dinutuximab-gm-csf-mody-jco-2020/  
OnCo record `paper-anbl1221-expansion-dinutuximab-gm-csf-mody-jco-2020` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Across 53 children treated with irinotecan, temozolomide and dinutuximab for relapsed neuroblastoma, four in ten had their tumours shrink and 85 percent were alive a year later, confirming the combination as the standard salvage treatment.

## Summary

Report of the randomised and expansion cohorts of ANBL1221: 17 randomised and 36 non-randomly assigned patients received irinotecan, temozolomide, dinutuximab and GM-CSF.

Objective responses occurred in 52.9 percent of randomised and 36.1 percent of expansion patients, 41.5 percent overall, with stable disease in a further 22 of 53. One-year progression-free and overall survival were 67.9 and 84.9 percent. Fever and infection, neutropenia, pain and diarrhoea were the common grade 3 or higher toxicities; higher dinutuximab trough levels were associated with response.

## Fields

- Kind: Key paper
- Last checked: 2026-09-22
- Journal: Journal of Clinical Oncology
- Year: 2020
- DOI: 10.1200/JCO.20.00203
- Authors: Mody R, Yu AL, Naranjo A, et al.
- Findings: Objective response 41.5 percent (95% CI 28.2 to 54.8) in 53 patients; 52.9 percent in the randomised cohort and 36.1 percent in the expansion cohort.; One-year progression-free survival 67.9 percent and overall survival 84.9 percent.; Higher dinutuximab trough concentrations associated with response.
- What it means: Confirms irinotecan-temozolomide-dinutuximab as the reference salvage regimen and motivated frontline chemo-immunotherapy trials (ANBL17P1, ANBL1531).
- Caveats: Single-arm expansion; response rate was lower in the expansion cohort than in the randomised cohort.

## Sources

- J Clin Oncol 2020: https://doi.org/10.1200/JCO.20.00203
- PubMed: https://pubmed.ncbi.nlm.nih.gov/32343642/

## Connected records

- cancers: [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/)
- drugs: [Dinutuximab (ch14.18) / dinutuximab beta](https://onco.cc/drugs/dinutuximab/), [Irinotecan (and liposomal irinotecan)](https://onco.cc/drugs/irinotecan/), [Sargramostim](https://onco.cc/drugs/sargramostim/), [Temozolomide](https://onco.cc/drugs/temozolomide/)
- trials: [ANBL1221](https://onco.cc/trials/anbl1221/)
- journals: [Journal of Clinical Oncology](https://onco.cc/journals/jco/)

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