Failure of stem cell therapy to improve visual acuity in children with optic nerve hypoplasia

PurposeTo evaluate the effectiveness of stem cell therapy in improving visual acuity or optic nerve function in children with optic nerve hypoplasia (ONH).MethodsSubjects with ONH aged 7-17 years who voluntarily elected to undergo stem cell therapy in China were matched with controls on age, visual acuity, and communication level. Visual acuity, optic nerve size, and sensitivity to light were assessed before stem cell therapy and twice after. Controls were evaluated on the same schedule.ResultsTwo case–control pairs were enrolled. There were no clinically significant changes in pupillary constriction or optic nerve measurements in any participant eye. Improvement in visual acuity was noted in study participants regardless of treatment status.ConclusionsOn the basis of this small sample, there is no evidence that stem cell therapy improves visual acuity in children with ONH.