Retrospective study of irinotecan/temozolomide/dinutuximab/GM-CSF for pediatric relapsed high-risk neuroblastoma

Although chemoimmunotherapy is widely used for treatment of children with relapsed high-risk neuroblastoma (HRNB), little is known about timing, duration, and evolution of response after irinotecan/temozolomide/dinutuximab/granulocyte-macrophage colony-stimulating factor (I/T/DIN/GM-CSF) therapy. A multicenter retrospective study included patients who were age ≤ 30 years at diagnosis of HRNB. Patients received at least one cycle of I/T/DIN/GM-CSF for relapsed or progressive disease. Approximately half of patients had objective responses. More than one of three patients with minor or partial response on first evaluation ultimately had complete response. The median duration of response was 13.1 months, and the 2-year progression-free survival rate was 28%. I/T/DIN/GM-CSF was associated with extended PFS in responders both during and after discontinuation of treatment.