Treatment of High-Risk Neuroblastoma

Patients most at risk for disease progression and mortality are older than 18 months, have metastatic disease or localized disease with unfavorable biology such as MYCN amplification, or have unfavorable histology. For more information about the Children's Oncology Group (COG) risk categories, see Table 3.

Approximately 8% to 10% of infants with stage MS disease have MYCN-amplified tumors and are usually treated using high-risk protocols. The 5-year event-free survival (EFS) and overall survival (OS) rates were 60% and 64%, respectively, for the infants with stage MS disease and MYCN amplification (n = 23), among the 5,000 patients enrolled in the COG ANBL00B1 (NCT00904241) trial.[1]

For children with high-risk neuroblastoma who received current treatments, the 5-year OS rate was about 60% for patients diagnosed between 2007 and 2017.[1] Children with aggressively treated, high-risk neuroblastoma may develop late recurrences, some more than 5 years after completion of therapy.[2,3]

A study from the International Neuroblastoma Risk Group (INRG) database found 146 patients with distant metastases limited to lymph nodes, termed stage 4N, who tended to have favorable-biology disease and a good outcome (5-year OS rate, 85%). This finding suggests that for this very rare, special subgroup of high-risk, stage 4 patients, less-intensive therapy might be considered.[4] These more favorable outcomes were confirmed in a single-institution study of 51 patients.[5]