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Maintaining Outstanding Outcomes Using Response- and Biology-Based Therapy for Intermediate-Risk Neuroblastoma: A
Clare J Twist1, Mary Lou Schmidt2, Arlene Naranjo3
1Roswell Park Cancer Institute, Buffalo, NY.
Purpose:
The primary objective of the Children's Oncology Group study ANBL0531 (ClinicalTrials.gov identifier: NCT00499616) was to reduce therapy for subsets of patients with intermediate-risk neuroblastoma using a biology- and response-based algorithm to assign treatment duration while maintaining a 3-year overall survival (OS) of 95% or more for the entire cohort.
Patients And Methods:
Children younger than age 12 years with intermediate-risk stage 2A/2B or stage 3 tumors with favorable histology; infants younger than age 365 days with stage 3, 4 or 4S disease; and toddlers from 365 to younger than 547 days with favorable histology, hyperdiploid stage 4, or unfavorable histology stage 3 tumors were eligible. Patients with MYCN-amplified tumors were excluded. Patients were assigned to initially receive two (group 2), four (group 3), or eight (group 4) cycles of chemotherapy with or without surgery on the basis of prognostic markers, including allelic status of chromosomes 1p and 11q; ultimate duration of therapy was determined by overall response.
Results:
Between 2007 and 2011, 404 evaluable patients were enrolled. Compared with legacy Children's Oncology Group studies, subsets of patients had a reduction in treatment. The 3-year event-free survival and OS rates were 83.2% (95% CI, 79.4% to 87.0%) and 94.9% (95% CI, 92.7% to 97.2%), respectively. Infants with stage 4 tumors with favorable biology (n = 61) had superior 3-year event-free survival compared with patients with one or more unfavorable biologic features (n = 47; 86.9% [95% CI, 78.3% to 95.4%] v 66.8% [95% CI, 53.1% to 80.6%]; P = .02), with a trend toward OS advantage (95.0% [95% CI, 89.5% to 100%] v 86.7% [95% CI, 76.6% to 96.7%], respectively; P = .08). OS for patients with localized disease was 100%.
Conclusion:
Excellent survival was achieved with this treatment algorithm, with reduction of therapy for subsets of patients. More-effective treatment strategies still are needed for infants with unfavorable biology stage 4 disease.
Insights
This study reduced neuroblastoma treatment for some patients, achieving excellent survival rates. Further research is needed for infants with unfavorable biology stage 4 disease.
Area of Science:
- Pediatric Oncology
- Neuroblastoma Research
- Clinical Trial Design
Background:
- Intermediate-risk neuroblastoma treatment often involves intensive therapy.
- Optimizing treatment duration based on patient biology and response is crucial for improving outcomes and reducing toxicity.
Purpose of the Study:
- To reduce therapy for intermediate-risk neuroblastoma patients using a biology- and response-based algorithm.
- To maintain a 3-year overall survival (OS) of 95% or more in the entire cohort.
Main Methods:
- Children's Oncology Group study ANBL0531 enrolled 404 patients with intermediate-risk neuroblastoma.
- Treatment duration was determined by overall response after initial chemotherapy cycles (2, 4, or 8) based on prognostic markers.
- Exclusion criteria included MYCN-amplified tumors.
Main Results:
- The 3-year event-free survival and OS rates were 83.2% and 94.9%, respectively.
- Subsets of patients received reduced treatment compared to legacy studies.
- Infants with stage 4 tumors and favorable biology showed superior outcomes.
Conclusions:
- The biology- and response-based algorithm achieved excellent survival while reducing therapy for some patients.
- More effective strategies are needed for infants with stage 4 neuroblastoma and unfavorable biology.