Longitudinal cohort study of long-term outcomes of pediatric patients treated for optic pathway gliomas

A longitudinal cohort study at Gustave Roussy (Villejuif, France) evaluated long term outcomes of pediatric patients treated for optic pathway gliomas (OPG). The study included 182 5-year survivors treated before age 18 years and alive at 5 years from diagnosis. With a median follow-up of 17.2 years (range 5-41), there were 82 relapses, 9 second malignancies, and 15 deaths as first events after 5 years, resulting in 20-years conditional survival of 79.9% (95% CI 71-86) and late events-free survival of 43.5% (36-51). Neurofibromatosis 1 (NF1; HR 3.0; 95% CI 1.4-6.8), hypothalamic involvement (3.2; 1.4-7.3), and radiotherapy (2.8; 1.1-6.7) were significantly associated with conditional overall survival in multivariable analyses. Ninety-five percent of 5-year OPG survivors suffered from any health condition, especially lack of visual acuity, pituitary deficiency, and neurocognitive impairment. NF1 (HR 2.1) was associated with precocious puberty. With a median time post diagnosis of 4.2 years, 33 cerebrovascular events were observed in 21 patients. The authors conclude that among survivors of pediatric OPG, morbidity and mortality were high.