BACKGROUND: We assessed the effects on visual function of murine nerve growth factor (NGF) administered as eye drop in children with optic pathway glioma (OPG) and visual impairment. METHODS: A prospective randomized double-blind controlled study was conducted in 18 pts (median age 10 yrs) with OPG-induced visual impairment. All patients were off-therapy and with stable disease by MRI. NGF eye-drop was prepared by University Hospital Pharmacy according to standard required for human use. Patients were evaluated by visual acuity, visual field, optical coherence tomography of the optic nerve fiber layer, Ganzfeld electroretinograms photopic negative response (PhNR), an indirect index of retinal ganglion cell function, and visual evoked potentials (VEPs). All patients were assesed at baseline and 15, 30, 90 and 180 days post treatment. Brain MRI was performed at baseline and at 180 days after NGF treatment. Patients received a 10-day course of 1 mg murine NGF topical administration and placebo, respectively. Based on the evidence of statistically significant drug effect, a cross over treatment with NGF was given, in open label, to patients receiving placebo. RESULTS: Ten pts received placebo and eight NGF. In treated pts there was an improvement of visual field by 40% ( p = 0.02) and 50% (p = 0.05) at 30 and at 90 days post-treatment respectively, compared to a 14% and 5% increase in the placebo group. An increase of the mean PhNR amplitude till 90 days was observed in the treated eyes (25%, p < 0.06), compared to placebo group. There was a median VEP amplitude increase from baseline, compared to placebo group, at 30 days (p < 0.05). In placebo group visual field and VEP amplitudes improved from either baseline or from the values recorded after placebo treatment. CONCLUSIONS: NGF administration appears an effective and safe therapy in patients with OPG-associated visual impairment.
Effect of nerve growth factor administered as eye-drop on visual function in children with optic glioma-associated visual impairement: A controlled, cross-over clinical trial.
Manni Luigi;
2014
Abstract
BACKGROUND: We assessed the effects on visual function of murine nerve growth factor (NGF) administered as eye drop in children with optic pathway glioma (OPG) and visual impairment. METHODS: A prospective randomized double-blind controlled study was conducted in 18 pts (median age 10 yrs) with OPG-induced visual impairment. All patients were off-therapy and with stable disease by MRI. NGF eye-drop was prepared by University Hospital Pharmacy according to standard required for human use. Patients were evaluated by visual acuity, visual field, optical coherence tomography of the optic nerve fiber layer, Ganzfeld electroretinograms photopic negative response (PhNR), an indirect index of retinal ganglion cell function, and visual evoked potentials (VEPs). All patients were assesed at baseline and 15, 30, 90 and 180 days post treatment. Brain MRI was performed at baseline and at 180 days after NGF treatment. Patients received a 10-day course of 1 mg murine NGF topical administration and placebo, respectively. Based on the evidence of statistically significant drug effect, a cross over treatment with NGF was given, in open label, to patients receiving placebo. RESULTS: Ten pts received placebo and eight NGF. In treated pts there was an improvement of visual field by 40% ( p = 0.02) and 50% (p = 0.05) at 30 and at 90 days post-treatment respectively, compared to a 14% and 5% increase in the placebo group. An increase of the mean PhNR amplitude till 90 days was observed in the treated eyes (25%, p < 0.06), compared to placebo group. There was a median VEP amplitude increase from baseline, compared to placebo group, at 30 days (p < 0.05). In placebo group visual field and VEP amplitudes improved from either baseline or from the values recorded after placebo treatment. CONCLUSIONS: NGF administration appears an effective and safe therapy in patients with OPG-associated visual impairment.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
