For specific subsets of patients with intermediate-risk neuroblastoma, an algorithm based on tumor biology and response successfully helped patients reduce treatment while maintaining a 3-year overall survival (OS) rate of 95% or higher, according to data from the Children’s Oncology Group study ANBL0531 ( Identifier: NCT00499616). The findings were recently published in the Journal of Clinical Oncology.1

The study was a single-arm, phase 3 clinical trial that enrolled patients with intermediate-risk neuroblastoma, including patients who were aged younger than 12 years with intermediate-risk stage 2A/2B or stage III tumors with favorable histology; patients who were aged younger than 365 days with stage III, IV, or IVS disease; and patients who were aged between 365 days and 547 days with tumors with favorable histology, hyperdiploid stage IV, or stage III tumors with unfavorable histology.

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Treatment assignments were made on the basis of age at diagnosis, International Neuroblastoma Staging System (INSS) stage, histology, and genetic features of the tumor. Possible treatment assignments included receiving 2, 4, or 8 cycles of chemotherapy with or without surgery.

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A total of 404 evaluable patients made up the study cohort, and for the overall study cohort, the 3-year event-free survival rate was 83.2% (95% CI, 79.4% to 87.0%) and OS rate was 94.9% (95% CI, 92.7% to 97.2%), described by the study authors as an “excellent” outcome.

Using the Children’s Oncology Group legacy studies as a comparison, treatment reduction was achieved for subsets of patients, including those with tumors that had favorable biology, those whose tumors were assessed via partial response instead of very good partial response, and those aged between 365 days and 547 days with stage III or stage IV disease.

“It is important to emphasize that intermediate-risk neuroblastoma is a heterogeneous collection of neuroblastoma phenotypes,” the study authors wrote. “Using this response- and biology-based algorithm that includes the allelic status of 1p36 and 11q23, excellent survival was achieved with refined treatment strategies for specific subsets of intermediate-risk patients.”


Twist CJ, Schmidt ML, Naranjo A, et al. Maintaining outstanding outcomes using response- and biology-based therapy for intermediate-risk neuroblastoma: A report from the Children’s Oncology Group Study ANBL0531 [published online August 6, 2019]. J Clin Oncol. doi: 10.1200/JCO.19.00919