{"entity":{"id":"paper-anbl1221-expansion-dinutuximab-gm-csf-mody-jco-2020","kind":"paper","name":"ANBL1221 expansion: irinotecan, temozolomide and dinutuximab with GM-CSF in refractory or relapsed neuroblastoma","aka":[],"tldr":"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.\n\nObjective 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.","asOf":"2026-09-22","links":[{"label":"J Clin Oncol 2020","url":"https://doi.org/10.1200/JCO.20.00203"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/32343642/"}],"tags":[],"related":[],"cancers":["neuroblastoma-high-risk","neuroblastoma"],"sections":[],"technologies":[],"targets":[],"drugs":["dinutuximab","irinotecan","temozolomide","sargramostim"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["anbl1221"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["jco"],"dependsOn":[],"notes":[],"journal":"Journal of Clinical Oncology","year":2020,"doi":"10.1200/JCO.20.00203","pmid":"32343642","authors":"Mody R, Yu AL, Naranjo A, et al.","paperType":"observational","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."],"whatItMeans":"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."],"changedPractice":true,"participants":53},"route":"/key-papers/paper-anbl1221-expansion-dinutuximab-gm-csf-mody-jco-2020/","neighbours":{"cancer":[{"id":"neuroblastoma-high-risk","kind":"cancer","name":"High-risk neuroblastoma","route":"/cancers/neuroblastoma-high-risk/"},{"id":"neuroblastoma","kind":"cancer","name":"Neuroblastoma (paediatric)","route":"/cancers/neuroblastoma/"}],"drug":[{"id":"dinutuximab","kind":"drug","name":"Dinutuximab (ch14.18) / dinutuximab beta","route":"/drugs/dinutuximab/"},{"id":"irinotecan","kind":"drug","name":"Irinotecan (and liposomal irinotecan)","route":"/drugs/irinotecan/"},{"id":"sargramostim","kind":"drug","name":"Sargramostim","route":"/drugs/sargramostim/"},{"id":"temozolomide","kind":"drug","name":"Temozolomide","route":"/drugs/temozolomide/"}],"trial":[{"id":"anbl1221","kind":"trial","name":"ANBL1221","route":"/trials/anbl1221/"}],"journal":[{"id":"jco","kind":"journal","name":"Journal of Clinical Oncology","route":"/journals/jco/"}]}}