{Reference Type}: Journal Article
{Title}: Very long-term outcomes of pediatric patients treated for optic pathway gliomas: A longitudinal cohort study.
{Author}: Morin A;Allodji R;Kariyawasam D;Touraine P;Puget S;Beccaria K;De Carli E;Kieffer V;Rivollet S;Abbou S;Fayech C;Souchard V;Dufour C;De Vathaire F;Bolle S;Grill J;Fresneau B;
{Journal}: Neuro Oncol
{Volume}: 26
{Issue}: 7
{Year}: 2024 Jul 5
{Factor}: 13.029
{DOI}: 10.1093/neuonc/noae045
{Abstract}: <B>BACKGROUND: </B>Optic pathway gliomas (OPGs) represent 5% of childhood brain tumors. Successive relapses lead to multiple treatments exposing to late complications.<BR><B>METHODS: </B>We included patients treated at Gustave Roussy (GR) between January 1980 and December 2015 for OPG, before 18 years old and alive at 5 years from diagnosis. Mortality and physical health conditions data were extracted from medical data files and updated, thanks to the GR long-term follow-up program and French national mortality registry for patients included in the French Childhood Cancer Survivor Study.<BR><B>RESULTS: </B>We included 182 5-year OPG-childhood survivors in the analysis (sex ratio M/F 0.8, 35% with neurofibromatosis type 1 [NF1]). With a median follow-up of 17.2 years (range = 5-41), we registered 82 relapses, 9 second malignancies, and 15 deaths as first events after 5 years, resulting in 20-year conditional overall survival (C-OS) and late events-free survival of 79.9% (95% confidence interval [CI] = 71-86) and 43.5% (95% CI = 36-51), respectively. Radiotherapy exposure in NF1 patients (hazard ratio [HR] = 6, 95% CI = 1.7-21.2) and hypothalamic involvement (HR = 3.2, 95% CI = 1.4-7.3) were significantly associated with C-OS in multivariable analyses. Ninety-five percent of 5-year OPG survivors suffered from any health condition, especially visual acuity "<1/10" (n = 109), pituitary deficiency (n = 106), and neurocognitive impairment (n = 89). 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.<BR><B>CONCLUSIONS: </B>Late relapses, second malignancies, and cerebrovascular diseases are severe late events resulting in premature mortality. Morbidity is high and needs after-cancer care to improve quality of life. Risk factors could be considered to better stratify long-term follow-up.