Natural history and outcome of optic pathway gliomas in children.
BACKGROUND
The optimal management of optic pathway gliomas (OPGs) is complicated by their variable natural history, the association with neurofibromatosis type 1 (NF1) and difficulties in defining progression and response to treatment.


METHODS
This study is a retrospective review of all children presenting to a single institution with an OPG between 1990 and 2004.


RESULTS
Of the 133 children included, 78 (59%) had NF1; 87 (71 NF1) were observed initially, of whom 23 (11 NF1) subsequently required treatment. Forty-six patients received immediate treatment. Initial treatment, without or with an observation period, comprised chemotherapy alone (32, 11 NF1); debulking + chemotherapy (15, 4 NF1); gross total resection (6); radiotherapy (2); debulking + radiotherapy (3); and debulking only (12, 3 NF1). Overall, 16 patients were irradiated during the study period. Four children died (overall survival at 5 and 10 years was 97.6% and 94.6% for those who required treatment). Progression-free survival (PFS) for the 69 patients who needed treatment was 48%. There was no difference in PFS between chemotherapy versus chemotherapy + debulking or debulking alone. PFS for the NF1 patients who required treatment was similar to that of non-NF1 patients. Mean follow-up time was 9.0 (range 0.6-18.0, median 8.6) years.


CONCLUSIONS
The study confirms the complexity of OPGs and that NF1 is a major determinant of the resultant behavior of the tumor.
Nicolin
Gary
G
Pediatric Brain Tumor Program, Division of Hematology/Oncology, Hospital for Sick Children, Toronto, Ontario, Canada.
Parkin
Patricia
P
Mabbott
Donald
D
Hargrave
Darren
D
Bartels
Ute
U
Tabori
Uri
U
Rutka
James
J
Buncic
J Raymond
JR
Bouffet
Eric
E
D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use; D001925:Brain Damage, Chronic / Q000209:etiology; D016190:Carboplatin / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D016371:Cranial Irradiation / Q000009:adverse effects; D018450:Disease Progression; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D008607:Intellectual Disability / Q000209:etiology; D008130:Lomustine / Q000008:administration & dosage; D008297:Male; D009456:Neurofibromatosis 1 / Q000453:epidemiology; D020339:Optic Nerve Glioma / Q000150:complications / Q000188:drug therapy / Q000453:epidemiology* / Q000473:pathology / Q000532:radiotherapy / Q000601:surgery; D011344:Procarbazine / Q000008:administration & dosage; D012189:Retrospective Studies; D016019:Survival Analysis; D013866:Thioguanine / Q000008:administration & dosage; D016896:Treatment Outcome; D014747:Vinblastine / Q000008:administration & dosage; D014750:Vincristine / Q000008:administration & dosage; D014786:Vision Disorders / Q000209:etiology
D011344:Procarbazine; D014750:Vincristine; D014747:Vinblastine; D008130:Lomustine; D016190:Carboplatin; D013866:Thioguanine