Long-term follow-up of patients with intermediate-risk neuroblastoma treated with response- and biology-based therapy: A report from the Children's Oncology Group study ANBL0531.

Autor: Barr EK; Department of Pediatrics, Texas Tech University Health Sciences, Lubbock, Texas, USA., Naranjo A; Department of Biostatistics, University of Florida Children's Oncology Group Statistics and Data Center, Gainesville, Florida, USA., Twist CJ; Roswell Park Comprehensive Cancer Center, Buffalo, New York, USA., Tenney SC; Department of Biostatistics, University of Florida Children's Oncology Group Statistics and Data Center, Gainesville, Florida, USA., Schmidt ML; Department of Pediatrics, University of Illinois at Chicago, Chicago, Illinois, USA., London WB; Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA., Gastier-Foster J; Department of Pediatrics and Pathology/Immunology, Baylor College of Medicine, Houston, Texas, USA., Adkins ES; Department of Pediatrics, Palmetto Health-USC Medical Group, Columbia, South Carolina, USA., Mattei P; Department of Surgery, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA., Handler MH; Department of Neurosurgery, Children's Hospital Colorado, Aurora, Colorado, USA., Matthay KK; Department of Pediatrics, University of California San Francisco School of Medicine, San Francisco, California, USA., Park JR; Department of Oncology, St.Jude Children's Research Hospital, Memphis, Tennessee, USA., Maris JM; Department of Pediatrics, Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Desai AV; Department of Pediatrics, University of Chicago, Chicago, Illinois, USA., Cohn SL; Department of Pediatrics, University of Chicago, Chicago, Illinois, USA.
Jazyk: angličtina
Zdroj: Pediatric blood & cancer [Pediatr Blood Cancer] 2024 Aug; Vol. 71 (8), pp. e31089. Date of Electronic Publication: 2024 May 31.
DOI: 10.1002/pbc.31089
Abstrakt: Background: We previously reported excellent three-year overall survival (OS) for patients with newly diagnosed intermediate-risk neuroblastoma treated with a biology- and response-based algorithm on the Children's Oncology Group study ANBL0531. We now present the long-term follow-up results.
Methods: All patients who met the age, stage, and tumor biology criteria for intermediate-risk neuroblastoma were eligible. Treatment was based on prognostic biomarkers and overall response. Event-free survival (EFS) and OS were estimated by the Kaplan-Meier method.
Results: The 10-year EFS and OS for the entire study cohort (n = 404) were 82.0% (95% confidence interval (CI), 77.2%-86.9%) and 94.7% (95% CI, 91.8%-97.5%), respectively. International Neuroblastoma Staging System stage 4 patients (n = 133) had inferior OS compared with non-stage 4 patients (n = 271; 10-year OS: 90.8% [95% CI, 84.5%-97.0%] vs 96.6% [95% CI, 93.9%-99.4%], p = .02). Infants with stage 4 tumors with ≥1 unfavorable biological feature (n = 47) had inferior EFS compared with those with favorable biology (n = 61; 10-year EFS: 66.8% [95% CI, 50.4%-83.3%] vs 86.9% [95% CI, 76.0%-97.8%], p = .02); OS did not differ (10-year OS: 84.4% [95% CI, 71.8%-97.0%] vs 95.0% [95% CI, 87.7%-100.0%], p = .08). Inferior EFS but not OS was observed among patients with tumors with (n = 26) versus without (n = 314) 11q loss of heterozygosity (10-year EFS: 68.4% [95% CI, 44.5%-92.2%] vs 83.9% [95% CI, 78.7%-89.2%], p = .03; 10-year OS: 88.0% [95% CI, 72.0%-100.0%] vs 95.7% [95% CI, 92.8%-98.6%], p = .09).
Conclusions: The ANBL0531 trial treatment algorithm resulted in excellent long-term survival. More effective treatments are needed for subsets of patients with unfavorable biology tumors.
(© 2024 Wiley Periodicals LLC.)
Databáze: MEDLINE
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