Nerve growth factor improves visual loss in childhood optic gliomas: a randomized, double-blind, phase II clinical trial

Abstract

Paediatric optic pathway gliomas are low-grade brain tumours characterized by slow progression and invalidating visual loss. Presently there is no strategy to prevent visual loss in this kind of tumour. This study evaluated the effects of nerve growth factor administration in protecting visual function in patients with optic pathway glioma-related visual impairment. A prospective randomized double-blind phase II clinical trial was conducted in 18 optic pathway glioma patients, aged from 2 to 23 years, with stable disease and severe visual loss. Ten patients were randomly assigned to receive a single 10-day course of 0.5 mg murine nerve growth factor as eye drops, while eight patients received placebo. All patients were evaluated before and after treatment, testing visual acuity, visual field, visual-evoked potentials, optic coherence tomography, electroretinographic photopic negative response, and magnetic resonance imaging. Post-treatment evaluations were repeated at 15, 30, 90, and 180 days Brain magnetic resonance imaging was performed at baseline and at 180 days. Treatment with nerve growth factor led to statistically significant improvements in objective electrophysiological parameters (electroretinographic photopic negative response amplitude at 180 days and visual-evoked potentials at 30 days), which were not observed in placebo-treated patients. Furthermore, in patients in whom visual fields could still be measured, visual field worsening was only observed in placebo-treated cases, while three of four nerve growth factor-treated subjects showed significant visual field enlargement. This corresponded to improved visually guided behaviour, as reported by the patients and/or the caregivers. There was no evidence of side effects related to nerve growth factor treatment. Nerve growth factor eye drop administration appears a safe, easy and effective strategy for the treatment of visual loss associated with optic pathway gliomas.

Retracted: MicroRNA‐21 promotes glioma cell proliferation and inhibits senescence and apoptosis by targeting SPRY1 via the PTEN/PI3K/AKT signaling pathway

“…After counter‐staining, all sections were sealed with neutral gum. Immunohistochemical results were determined as follows: by double‐blind method, 4 high‐power visual fields (200 cells in each field) were randomly selected and observed under a light microscope. Positive cell number in each field was counted, and the average positive rate was calculated.…”

Combined Intravitreal Dexamethasone Implant and Cataract Surgery in Patients with Diabetic Retinopathy: Effect on Retinal Morphology and Function

“…For each patient, Ganzfeld electroretinograms were recorded with a specific, published protocol employed to isolate and analyze the PhNR from the single flash cone-mediated responses [18][19][20]. The amplitudes of the PhNR and of the cone b wave were measured in each recording session using a Retimax instrument (CSO Company Florence, Italy).…”

Optic Nerve Glioma in Two Sisters with Family History of Neurofibromatosis Type 1

“…The exact causes of the disease are not yet elucidated, and there is no consensus regarding the therapeutic approach and the prevention of visual impairment. 8,9 The treatment options for ONG vary from observation to the use of either surgical removal, radiotherapy, and/or chemotherapy and multimodality protocols.…”