title: Visual outcome of a cohort of children with neurofibromatosis type 1 and optic pathway glioma followed by a pediatric neuro-oncology program.
abstract: We evaluated the visual outcome of a cohort of children with neurofibromatosis type 1 (NF1) and optic pathway glioma (OPG) treated according to standardized therapeutic guidelines. The study population consisted of all consecutive patients with NF1 and OPG referred to a specialized pediatric neuro-oncology program between 1994 and 2004. Treatment was instituted only in cases of progressive disease or clinical deterioration. Treatment modalities were chemotherapy (based on vincristine/carboplatin) for children younger than 5 years and radiotherapy for all others. Ten boys and 10 girls (seven with a positive family history) entered the trial (median age at diagnosis of OPG, 29 months). At a median follow-up time of 78 months, seven patients had been treated with chemotherapy only, four with radiotherapy, and four with chemotherapy plus radiotherapy. Five patients were observed only. Currently, 18 are alive and two have died. Eight patients were treated for progressive visual loss in the face of stable disease, five for tumor volume increase without visual deterioration, and two for symptomatic tumor volume increase. At referral, six children had a visual acuity (VA) of < 30% in both eyes; eight children had 100% VA bilaterally. At referral, the visual field (VF) could be assessed in three children: One had VF loss in both eyes, one had VF loss in one eye, and one had normal VF. At last follow-up, eight children had VA < 20% in both eyes; only two children had 100% VA in both eyes. Among 11 children who had some visual function, three had VF loss in one eye and three in both eyes, and five had an intact VF. Contrast and color sensitivity were abnormal in seven and six patients, respectively. Thirteen children fell into the WHO hypovision category. In summary, among the 15 children treated, one had a definitive and two a mild improvement in VA. In conclusion, the visual outcome of this selected cohort of NF1 patients with OPG is unsatisfactory. A critical reappraisal of the therapeutic strategy adopted is needed.
authors: Department of Pediatrics, University Hospital of Padua, Via Giustiniani 3, 35128 Padua, Italy.
authors: Paola
authors: P
authors: Dalla Via
authors: Enrico
authors: E
authors: Opocher
authors: Maria Luisa
authors: ML
authors: Pinello
authors: Milena
authors: M
authors: Calderone
authors: Elisabetta
authors: E
authors: Viscardi
authors: Maurizio
authors: M
authors: Clementi
authors: Pier Antonio
authors: PA
authors: Battistella
authors: Anna Maria
authors: AM
authors: Laverda
authors: Liviana
authors: L
authors: Da Dalt
authors: Giorgio
authors: G
authors: Perilongo
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects*; D016190:Carboplatin / Q000008:administration & dosage / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D015350:Contrast Sensitivity / Q000187:drug effects / Q000528:radiation effects; D005074:Evoked Potentials, Visual; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D009456:Neurofibromatosis 1 / Q000150:complications / Q000628:therapy*; D020339:Optic Nerve Glioma / Q000150:complications / Q000628:therapy*; D011878:Radiotherapy / Q000009:adverse effects*; D014750:Vincristine / Q000008:administration & dosage / Q000009:adverse effects; D014786:Vision Disorders / Q000209:etiology*; D014794:Visual Fields / Q000187:drug effects / Q000528:radiation effects
chemical_list: D014750:Vincristine; D016190:Carboplatin
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