title: Nonrandomized comparison of neurofibromatosis type 1 and non-neurofibromatosis type 1 children who received carboplatin and vincristine for progressive low-grade glioma: A report from the Children's Oncology Group.
abstract: To evaluate tumor responses, event-free survival (EFS), overall survival (OS), and toxicity of chemotherapy, children with neurofibromatosis type 1 (NF1) and progressive low-grade glioma were enrolled into the Children's Oncology Group (COG) A9952 protocol and treated with carboplatin and vincristine (CV).
  
  
  
  Non-NF1 patients were randomized to CV or thioguanine, procarbazine, 1-(2-chloroethyl)-3-cyclohexyl-1-nitrosourea, and vincristine in COG A9952. NF1 patients were assigned to CV only. NF1 patients and non-NF1 patients who were treated with CV were compared with respect to baseline characteristics, toxicity, tumor responses, EFS, and OS.
  
  
  
  A total of 127 eligible patients with NF1 were nonrandomly assigned to CV: 42 NF1 patients (33%) had events, and 6 (4.7%) died. The 5-year EFS rate was 69% ± 4% for the CV-NF1 group and 39% ± 4% for the CV-non-NF1 group (P < .001). In a univariate analysis, NF1 children had a significantly higher tumor response rate and superior EFS and OS in comparison with CV-treated children without NF1. NF1 patients and non-NF1 patients differed significantly in amount of residual tumor, extent of resection, tumor location, and pathology. According to a multivariate analysis, NF1 was independently associated with better EFS (P < .001) but not with OS. NF1 patients also had a decreased risk of grade 3 or 4 toxicities in comparison with non-NF1 patients. Three second malignant neoplasms occurred in NF1 patients receiving CV (CV-NF1 group) at a median of 7.8 years (range, 7.3-9.4 years) after enrollment, but there were none in the non-NF1 group.
  
  
  
  Children with NF1 tolerated CV well and had tumor response rates and EFS that were superior to those for children without NF1. Cancer 2016;122:1928-36. © 2016 American Cancer Society.
authors.affiliation: Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Joann L
authors.initials: JL
authors.lastname: Ater
authors.affiliation: Department of Preventive Medicine, Keck School of Medicine, University of Southern California, Los Angeles, California.
authors.forename: Caihong
authors.initials: C
authors.lastname: Xia
authors.affiliation: Children's Health Care Atlanta, Hematology-Oncology, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Claire M
authors.initials: CM
authors.lastname: Mazewski
authors.affiliation: Department of Radiology, University of Texas Southwestern Medical Center, Dallas, Texas.
authors.forename: Timothy N
authors.initials: TN
authors.lastname: Booth
authors.affiliation: Children's Hospital Los Angeles, Children's Center for Cancer and Blood Diseases, Los Angeles, California.
authors.forename: David R
authors.initials: DR
authors.lastname: Freyer
authors.affiliation: Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Packer
authors.affiliation: Department of Preventive Medicine, Keck School of Medicine, University of Southern California, Los Angeles, California.
authors.forename: Richard
authors.initials: R
authors.lastname: Sposto
authors.affiliation: Department of Neurology, Children's National Medical Center, Washington, DC.
authors.forename: Gilbert
authors.initials: G
authors.lastname: Vezina
authors.affiliation: Children's Hospital of Pittsburgh, Pediatric Neurosurgery, Pittsburgh, Pennsylvania.
authors.forename: Ian F
authors.initials: IF
authors.lastname: Pollack
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000150:complications / Q000188:drug therapy*; D016190:Carboplatin / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D005260:Female; D005910:Glioma / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D008297:Male; D009456:Neurofibromatosis 1 / Q000150:complications / Q000188:drug therapy*; D011344:Procarbazine / Q000008:administration & dosage; D013866:Thioguanine / Q000008:administration & dosage; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D011344:Procarbazine; D014750:Vincristine; D016190:Carboplatin; D013866:Thioguanine
keywords: carboplatin; childhood low-grade glioma; neurofibromatosis 1; pilocytic astrocytoma; vincristine