Mortality in Children with Optic Pathway Glioma Treated with Up-Front BB-SFOP Chemotherapy

Abstract

BackgroundIn 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.MethodsWe 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.ResultsThe 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).ConclusionsThis 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.

Visual outcomes after chemotherapy for optic pathway glioma in children with and without neurofibromatosis type 1: results of the International Society of Paediatric Oncology (SIOP) Low-Grade Glioma 2004 trial UK cohort

“…Optic pathway gliomas (OPGs) account for 5% of paediatric intracranial tumours 1. Most children with OPGs have neurofibromatosis type 1 (NF1), the remainder being sporadic.…”

Pediatric Optic Pathway/Hypothalamic Glioma

“…In a report on patients to whom BB-SFOP (chemotherapy using the six drugs Carboplatin/Procarbazine, Etoposide/Cisplatin and Vincristine/Cyclophosphamide) was performed in France as a form of primary care, the long-term prognosis was not as favorable as was claimed in other previous reports. 39) Although the data are biased because the cases treated surgically were excluded and only radiotherapy and other types of chemotherapies had been performed, it shows that the survival rate continues to decrease every five years and tumors were the leading cause of death. In addition, onset before the age of one and the presence of intracranial hypertension upon onset are negative predictors.…”

Hypersensitivity to Carboplatin in Children with Malignancy

“…The substitution of carboplatin with another platinum compound (such as cisplatin) may be limited by cross-reactivity of platinum-specific IgE (Pagani, 2010). The cisplatin and cyclophosphamide combination showed efficacy when alternated with carboplatin in the French “BABY-SFOP” LGG study (Rakotonjanahary et al, 2015). Despite its effectiveness, however, cisplatin is more ototoxic and nephrotoxic than carboplatin (Bertolini et al, 2004).…”