key papersKey paper

ANBL1221: irinotecan-temozolomide with temsirolimus or dinutuximab in relapsed or refractory neuroblastoma

In children whose neuroblastoma had come back, adding the anti-GD2 antibody dinutuximab to irinotecan and temozolomide shrank the cancer in half of them, while adding temsirolimus almost never did.

Children's Oncology Group open-label randomised phase 2 selection trial: 35 eligible children with relapsed, refractory or progressive neuroblastoma were randomised to irinotecan and temozolomide with temsirolimus (n 18) or with dinutuximab plus GM-CSF (n 17).

One patient (6 percent) responded with temsirolimus against nine (53 percent, four partial and five complete responses) with dinutuximab. Pain, hypokalaemia, neutropenia, thrombocytopenia, anaemia, fever and hypoxia were the common grade 3 or worse events with dinutuximab; no deaths were attributed to treatment.

Randomised controlled trialChanged practice35 participants233 citations

Authors

Mody R, Naranjo A, Van Ryn C, et al.

Published

The Lancet Oncology, 2017

Full text

doi:10.1016/S1470-2045(17)30355-8 PubMed 28549783

Findings

Objective response 53 percent (95% CI 29.2 to 76.7) with irinotecan-temozolomide-dinutuximab versus 6 percent (0.0 to 16.1) with irinotecan-temozolomide-temsirolimus.
Grade 3 or worse pain in 44 percent and hypokalaemia in 38 percent of dinutuximab patients; one grade 4 hypoxia met unacceptable toxicity criteria.

What it means

Chemo-immunotherapy with dinutuximab became the salvage standard for relapsed neuroblastoma and the regimen to move into first-line induction.

Caveats