Erythropoietin Therapy in a Patient Suffering From Retinitis Pigmentosa and Sideroblastic Anemia

Abstract

Purpose: : To report a patient with severe RP leading to blindness who was treated with rEpo for an associated congenital sideroblastic anemia.

Methods: : A 19-year-old male with Bardet-Biedl syndrome that included a classical, bilateral pigmentary retinopathy with secondary blindness, was treated with recombinant erythropoietin for an associated congenital sideroblastic anemia.

Results: : During the 12-month follow-up, there was a dramatic improvement of the visual acuity (20/2000 to 20/50), visual fields and retinal electrophysiological activity as well as an increase of the retinal thickness, while hematological parameters remained unchanged.

Conclusions: : rEpo treatment had no effect on the Hb level but was followed by a dramatic improvement of visual acuity and objective ophtalmologic parameters suggesting a direct effect of Epo on the retina.This provocative case report supports the concept that erythropoietin may act as a retinal growth factor and may be of benefit to a subset of patients with retinitis pigmentosa.

Keywords: retina • retinitis • regeneration