The following is a summary of “Progression-Free Survival and Patterns of Response in Patients With Relapsed High-Risk Neuroblastoma Treated With Irinotecan/Temozolomide/Dinutuximab/Granulocyte-Macrophage Colony-Stimulating Factor,” published in the January 2023 issue of Oncology by Lerman, et al.

Although children with relapsed high-risk neuroblastoma (HRNB) were frequently treated with chemotherapy and immunotherapy, little was known about the timing, duration, and evolution of the response to irinotecan/temozolomide/dinutuximab/granulocyte-macrophage colony-stimulating factor (I/T/DIN/GM-CSF) therapy.

Patients who received ≥1 cycle of I/T/DIN/GM-CSF for relapsed or progressing illness and were <30 years at the time of HRNB diagnosis qualified for the retrospective analysis. Patients who progressed through induction and had the primary refractory disease were disqualified. The International Neuroblastoma Response Criteria were used to gauge responses.

A total of 136 patients were included. MYCN was amplified in 50 of 134 tumors (37%). The objective response of I/T/DIN/GM-CSF was seen in 71 patients (49%) (objective response; 29% complete response, 14% partial response [PR], 5% minor response [MR], 21% stable disease [SD], and 30% progressive disease). According to the International Neuroblastoma Response Criteria, 22% of patients with SD or better at the initial post-I/T/DIN/GM-CSF illness assessment (13% of patients with initial SD, 33% with MR, and 41% with PR) showed an improved response. Patients got a median of 4.5 cycles, with a range of 1-31. The 1-year and 2-year progression-free survival rates were 50% and 28%, respectively, and the median progression-free survival (PFS) was 13.1 months. After stopping I/T/DIN/GM-CSF, the median PFS off all anticancer treatment was 10.4 months, with a median duration of response of 15.9 months.

Patients taking I/T/DIN/GM-CSF for relapsed HRNB exhibited objective responses in almost half of the cases. Patients with initial SD were unlikely to have an objective response, whereas <1 of 3 patients with initial MR/PR had a full recovery. In responders, I/T/DIN/GM-CSF was linked to prolonged PFS both during and after therapy cessation. In patients with recurrent HRNB, the study presented a new comparator for response and survival.