title: Topical nerve growth factor as a visual rescue strategy in pediatric optic gliomas: a pilot study including electrophysiology.
abstract: BACKGROUND
  To date, no specific therapy is available for optic glioma (OG)-induced visual loss.
  
  
  OBJECTIVE
  To evaluate the effects on visual function of murine nerve growth factor (NGF) eye drop administration in children with severe visual impairment due to low-grade OGs.
  
  
  METHODS
  Five patients with OGs and advanced optic nerve atrophy were assessed before and after a single 10-day course of 1 mg murine NGF topical administration by clinical evaluation, visual evoked potentials (VEPs), and brain magnetic resonance imaging (MRI). VEPs, the main functional outcome measure, were recorded at baseline and 1, 30, 45, 90, and 180 days posttreatment. MRI examinations were performed at baseline and at 180 days after NGF treatment. Six untreated control patients with OGs also underwent serial VEPs, clinical testing, and MRI assessments.
  
  
  RESULTS
  After NGF treatment, median VEPs amplitude showed a progressive increase from the baseline values (P < .01). VEPs reached a maximum amplitude at 90 days (170% increase) and declined at 180 days, still remaining above the baseline level. Perception of spontaneous visual phosphenes was noted in all patients after NGF administration. MRI showed stable tumor size. In controls, clinical findings and VEPs did not show any significant change over the observation period.
  
  
  CONCLUSIONS
  The findings from the study show that NGF administration may be an effective and safe adjunct therapy in children with optic atrophy due to OGs. The beneficial effect on optic nerve function suggests a visual rescuing mechanism exerted by murine NGF on the residual viable optic pathways.
authors: Catholic University of Rome, Rome, Italy. bfalsini@rm.unicatt.it
authors: Benedetto
authors: B
authors: Falsini
authors: Antonio
authors: A
authors: Chiaretti
authors: Giuseppe
authors: G
authors: Barone
authors: Marco
authors: M
authors: Piccardi
authors: Filomena
authors: F
authors: Pierri
authors: Cesare
authors: C
authors: Colosimo
authors: Ilaria
authors: I
authors: Lazzareschi
authors: Antonio
authors: A
authors: Ruggiero
authors: Vincenzo
authors: V
authors: Parisi
authors: Antonello
authors: A
authors: Fadda
authors: Emilio
authors: E
authors: Balestrazzi
authors: Riccardo
authors: R
authors: Riccardi
mesh_terms: D000287:Administration, Topical; D000293:Adolescent; D000818:Animals; D001766:Blindness / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D002648:Child; D002675:Child, Preschool; D004568:Electrodiagnosis / Q000379:methods; D005260:Female; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D051379:Mice; D020932:Nerve Growth Factor / Q000008:administration & dosage* / Q000009:adverse effects; D009416:Nerve Regeneration / Q000187:drug effects* / Q000502:physiology; D009900:Optic Nerve / Q000187:drug effects* / Q000473:pathology / Q000503:physiopathology; D020339:Optic Nerve Glioma / Q000150:complications / Q000175:diagnosis / Q000503:physiopathology*; D010865:Pilot Projects; D014795:Visual Pathways / Q000187:drug effects / Q000473:pathology / Q000503:physiopathology
chemical_list: D020932:Nerve Growth Factor