title: Histologically benign, clinically aggressive: Progressive non-optic pathway pilocytic astrocytomas in adults with NF1.
abstract: Although optic pathway gliomas are the most common brain tumors associated with neurofibromatosis type 1 (NF1), extra-optic gliomas occur and may behave more aggressively with outcomes that differ by age. A retrospective case-control study was designed to describe the clinical course of adult NF1 patients with progressive extra-optic pilocytic astrocytomas (PAs) and compare to a pediatric cohort. Data for patients treated at the Johns Hopkins Comprehensive Neurofibromatosis Center from 2003 to 2013 were reviewed to identify cases (adults, age >18) and controls (pediatric, age <18) with clinically or radiographically progressive extra-optic PAs. Demographic, clinical, histologic, and radiographic data were collected. Three adult NF1 cases and four pediatric NF1 controls were identified. Mean age was 32.3 ± 9.5 years, 66% male (cases); 12.8 ± 4.2 years, 100% male (controls). Symptomatic progression occurred in two-of-three adults (67%) while the majority of pediatric patients presented with isolated radiographic progression (n = 3, 75%). Onset tended to be more rapid in adults (4 ± 1 vs. 14 ± 8.3 months, P = 0.10). Subtotal resection was the treatment for all pediatric patients. Radiotherapy (n = 2), chemotherapy (n = 2), and targeted, biologic agents (n = 2) were administered in adults. Although all pediatric patients are living, outcomes were universally poor in adults with progression to death in all (median survival 17.1 months, range 6.6-30.3). In conclusion, despite grade I histology, all three adult NF1 patients with progressive extra-optic PAs suffered an aggressive clinical course which was not seen in pediatric patients. Clinicians should be aware of this clinico-histologic discrepancy when counseling and managing adult NF1 patients with progressive extra-optic PAs. © 2016 Wiley Periodicals, Inc.
authors.affiliation: Department of Neurology, Johns Hopkins Hospital, Baltimore, Maryland.
authors.forename: Roy E
authors.initials: RE
authors.lastname: Strowd
authors.affiliation: Department of Pathology, Johns Hopkins Hospital, Baltimore, Maryland.
authors.forename: Fausto J
authors.initials: FJ
authors.lastname: Rodriguez
authors.affiliation: Division of Neurology, Department of Pathology, Duke University Medical Center, Durham, North Carolina.
authors.forename: Roger E
authors.initials: RE
authors.lastname: McLendon
authors.affiliation: Division of Neurology, Department of Internal Medicine, Duke University Medical Center, Durham, North Carolina.
authors.forename: James J
authors.initials: JJ
authors.lastname: Vredenburgh
authors.affiliation: Department of Neurology, Johns Hopkins Hospital, Baltimore, Maryland.
authors.forename: Aaron B
authors.initials: AB
authors.lastname: Chance
authors.affiliation: Department of Neurosurgery, Johns Hopkins Hospital, Baltimore, Maryland.
authors.forename: George
authors.initials: G
authors.lastname: Jallo
authors.affiliation: Department of Neurosurgery, Johns Hopkins Hospital, Baltimore, Maryland.
authors.forename: Alessandro
authors.initials: A
authors.lastname: Olivi
authors.affiliation: Department of Neurosurgery, Johns Hopkins Hospital, Baltimore, Maryland.
authors.forename: Edward S
authors.initials: ES
authors.lastname: Ahn
authors.affiliation: Department of Neurology, Johns Hopkins Hospital, Baltimore, Maryland.
authors.forename: Jaishri O
authors.initials: JO
authors.lastname: Blakeley
mesh_terms: D000328:Adult; D001254:Astrocytoma / Q000175:diagnosis* / Q000209:etiology* / Q000401:mortality / Q000628:therapy; D001706:Biopsy; D001921:Brain / Q000473:pathology; D016022:Case-Control Studies; D003131:Combined Modality Therapy; D003952:Diagnostic Imaging; D018450:Disease Progression; D005260:Female; D006801:Humans; D008297:Male; D060787:Neoplasm Grading; D009456:Neurofibromatosis 1 / Q000150:complications* / Q000175:diagnosis; D010641:Phenotype; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
keywords: adult; brain tumor; extra-optic glioma; neurofibromatosis type 1; pediatric; pilocytic astrocytoma