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Use of algorithm safely reduces therapy for certain children with neuroblastoma
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A biology- and response-based treatment algorithm helped reduce therapy for subsets of children with intermediate-risk neuroblastoma while maintaining high rates of 3-year OS, according to results of a prospective phase 3 study published in Journal of Clinical Oncology.
More effective strategies still are needed for infants who have stage IV disease with unfavorable biology, researchers noted.
“Neuroblastoma is characterized by a broad array of clinical behavior, and biomarkers are used to classify risk and stratify treatment,” Clare J. Twist, MD, pediatric oncologist at Roswell Park Comprehensive Cancer Center, and colleagues wrote. “Previous multi-institutional and cooperative group risk-based clinical trials have led to substantial improvement in outcomes for children with neuroblastoma, with increasingly intensive multimodality approaches for high-risk patients and successive reductions in therapy for patients with low- and intermediate-risk disease.”
The 404 patients in the single-arm study comprised children aged younger than 12 years with intermediate-risk stage IIA/IIB or stage III neuroblastoma with favorable histology; infants younger than 365 days with stage III, IV or IVS disease; and toddlers aged 365 days to less than 547 days with favorable histology, hyperdiploid stage IV or unfavorable histology stage III tumors.
Researchers assigned the patients to receive an initial two (group 2; n = 175), four (group 3; n = 141) or eight (group 4; n = 88) cycles of chemotherapy with or without surgery based on prognostic markers, such as age at diagnosis, genetic features of the tumor and allelic status of chromosomes 1p and 11q.
Overall response determined the ultimate duration of therapy, with the goal of reducing therapy without sacrificing OS.
Results showed 3-year EFS of 83.2% (95% CI, 79.4-87) and 3-year OS of 94.9% (95% CI, 92.7-97.2) among all patients, with subsets having received less treatment compared with legacy Children’s Oncology Group studies.
Infants with stage IV tumors with favorable biology (n = 61) had better 3-year EFS than infants (n = 47) with one or more unfavorable biologic features (86.9% vs. 66.8%; P = .02). They also showed an OS advantage (95% vs. 86.7%), which was not statistically significant.
Patients with localized disease had an OS rate of 100%.
Reversible myelosuppression was the most common grade 3 or higher chemotherapy-related toxicity. Nonhematologic toxicity was rare, and no patients experienced hearing loss.
The refined treatment strategies based on the algorithm will serve as that new standard of care for children with intermediate-risk neuroblastoma, according to researchers.
“Pediatric oncologists have been at the forefront of working collaboratively, and over the past 50 years, we have conducted a series of sequential clinical trials testing treatments for pediatric cancer that have resulted in new standards of care and remarkable improvement in outcome,” Susan Cohn, MD, chief of the section of pediatric hematology, oncology and stem cell transplant and professor of pediatrics at University of Chicago, said in a press release. “Collating the clinical trial results conducted around the world and linking these clinical data with genomic, imaging and other data sets in the Pediatric Cancer Data Commons will enable additional research advances that are likely to ultimately change our treatment strategies and further improve the outcome of children with cancer.”– by John DeRosier
Disclosures: Cohn reports stock ownership in and/or research funding from AbbVie, Amgen, Eli Lilly, Jazz Pharmaceuticals, Merck, Pfizer, Resmed, Sanofi, United Therapeutics, Varex Imaging, Varian Medical Systems and Vermillion. Twist reports no relevant financial disclosures. Please see the study for all other authors’ relevant financial disclosures.
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