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J Clin Oncol. 2019 Dec 1;37(34):3243-3255. doi: 10.1200/JCO.19.00919. Epub 2019 Aug 6.

Maintaining Outstanding Outcomes Using Response- and Biology-Based Therapy for Intermediate-Risk Neuroblastoma: A Report From the Children's Oncology Group Study ANBL0531.

Author information

1
Roswell Park Cancer Institute, Buffalo, NY.
2
University of Illinois at Chicago, Chicago, IL.
3
University of Florida, Gainesville, FL.
4
Harvard Medical School, Boston, MA.
5
Children's Hospital Los Angeles, Los Angeles, CA.
6
Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
7
Emory University, Atlanta, GA.
8
Palmetto Health-USC Medical Group, Columbia, SC.
9
Children's Hospital of Philadelphia, Philadelphia, PA.
10
Children's Hospital Colorado, Aurora, CO.
11
Nemours Children's Health Center, Jacksonville, FL.
12
Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
13
Nationwide Children's Hospital, Columbus, OH.
14
The Ohio State University College of Medicine, Columbus, OH.
15
University of California, San Francisco, School of Medicine, and UCSF Benioff Children's Hospital, San Francisco, CA.
16
Seattle Children's Hospital, Seattle, WA.
17
The University of Chicago, Chicago, IL.

Abstract

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.

PMID:
31386611
PMCID:
PMC6881103
[Available on 2020-12-01]
DOI:
10.1200/JCO.19.00919

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