key papersKey paper

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

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.

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.

Observational studyChanged practice53 participants175 citations

Authors

Mody R, Yu AL, Naranjo A, et al.

Published

Journal of Clinical Oncology, 2020

Full text

doi:10.1200/JCO.20.00203 PubMed 32343642

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