title: Mortality in Children with Optic Pathway Glioma Treated with Up-Front BB-SFOP Chemotherapy.
abstract: BACKGROUND
  In terms of overall survival (OS), limited data are available for the very long-term outcomes of children treated for optic pathway glioma (OPG) with up-front chemotherapy. Therefore, we undertook this study with the aim of clarifying long-term OS and causes of death in these patients.
  
  
  METHODS
  We initiated and analyzed a historical cohort study of 180 children with OPG treated in France with BB-SFOP chemotherapy between 1990 and 2004. The survival distributions were estimated using Kaplan-Meier method. The effect of potential risk factors on the risk of death was described using Cox regression analysis.
  
  
  RESULTS
  The OS was 95% [95% CI: 90.6-97.3] 5 years after diagnosis and significantly decreased over time without ever stabilizing: 91.6% at 10 years [95% CI: 86.5-94.8], 80.7% at 15 years [95% CI: 72.7-86.8] and 75.5% [95% CI: 65.6-83] at 18 years. Tumor progression was the most common cause of death (65%). Age and intracranial hypertension at diagnosis were significantly associated with a worse prognosis. Risk of death was increased by 3.1[95% CI: 1.5-6.2] (p=0.002) for patients less than 1 year old at diagnosis and by 5.2[95% CI: 1.5-17.6] (p=0.007) for patients with initial intracranial hypertension. Boys without diencephalic syndrome had a better prognosis (HR: 0.3 [95% CI: 0.1-0.8], p=0.007).
  
  
  CONCLUSIONS
  This study shows that i) in children with OPG, OS is not as favorable as previously described and ii) patients can be classified into 2 groups depending on risk factors (age, intracranial hypertension, sex and diencephalic syndrome) with an OS rate of 50.4% at 18 years [95% CI: 31.4-66.6] in children with the worst prognosis. These findings could justify, depending on the initial risk, a different therapeutic approach to this tumor with more aggressive treatment (especially chemotherapy) in patients with high risk factors.
authors.affiliation: Department of Pediatric Oncology, University Hospital, Angers, France; INSERM CIE5 Robert Debre Hospital, Assistance Publique-Hôpitaux de Paris, University Paris Diderot, Sorbonne Paris Cité, Paris, France.
authors.forename: Josué
authors.initials: J
authors.lastname: Rakotonjanahary
authors.affiliation: Department of Pediatric Oncology, University Hospital, Angers, France.
authors.forename: Emilie
authors.initials: E
authors.lastname: De Carli
authors.affiliation: Department of Neurosurgery, University Hospital, Angers, France.
authors.forename: Matthieu
authors.initials: M
authors.lastname: Delion
authors.affiliation: Department of Pediatric and Adolescent Oncology, Gustave Roussy Institute, Villejuif, France.
authors.forename: Chantal
authors.initials: C
authors.lastname: Kalifa
authors.affiliation: Department of Pediatric and Adolescent Oncology, Gustave Roussy Institute, Villejuif, France.
authors.forename: Jacques
authors.initials: J
authors.lastname: Grill
authors.affiliation: Department of Pediatric Oncology, Curie Institute and University Paris Descartes, Sorbonne Paris Cité, Paris, France.
authors.forename: François
authors.initials: F
authors.lastname: Doz
authors.affiliation: Department of Pediatric Oncology, Oscar Lambret Center, Lille, France.
authors.forename: Pierre
authors.initials: P
authors.lastname: Leblond
authors.affiliation: Department of Pediatric Oncology, University Hospital, Toulouse, France.
authors.forename: Anne-Isabelle
authors.initials: AI
authors.lastname: Bertozzi
authors.affiliation: Department of Pediatric Oncology, University Hospital, Angers, France.
authors.forename: Xavier
authors.initials: X
authors.lastname: Rialland
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001254:Astrocytoma / Q000188:drug therapy / Q000401:mortality*; D016190:Carboplatin / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D002945:Cisplatin / Q000627:therapeutic use; D015331:Cohort Studies; D003520:Cyclophosphamide / Q000627:therapeutic use; D018572:Disease-Free Survival; D004359:Drug Therapy, Combination; D005047:Etoposide / Q000627:therapeutic use; D005260:Female; D005602:France; D006801:Humans; D007223:Infant; D008297:Male; D020339:Optic Nerve Glioma / Q000188:drug therapy* / Q000401:mortality*; D011344:Procarbazine / Q000627:therapeutic use; D015996:Survival Rate; D014750:Vincristine / Q000627:therapeutic use
chemical_list: D000970:Antineoplastic Agents; D011344:Procarbazine; D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D016190:Carboplatin; D002945:Cisplatin