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Rubens C. Siqueira, Rodrigo Jorge, Andre Messias, Ingrid Scott, Julio Voltarelli; Transplantation of Autologous Bone Marrow Derived Stem Cells for Retinitis Pigmentosa. Invest. Ophthalmol. Vis. Sci. 2011;52(14):2217.
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To evaluate the short-term (10 months) safety of a single intravitreal injection of autologous bone marrow derived stem cells (ABMSC) in patients with retinitis pigmentosa (RP).
This prospective, phase I, nonrandomized, open-label study was approved by the local and National Institutional Review Boards (ClinicalTrials.gov Identifier: NCT01068561).Five patients with RP were included, age-gender: 31-F; 35-M; 23-F; 33-M; and 35-M. Evaluations were performed at baseline, and 1, 7, 13, 18, 22, and 40 weeks after intravitreal injection of 10 x 106 autologous bone marrow derived stem cells(0.1 ml). All patients presented with a best-corrected visual acuity (ETDRS BCVA) of 20/200 or worse at baseline. In addition to comprehensive ophthalmological examination, patients underwent standard full field electroretinography (ERG - ISCEV Standard); kinetic visual field (Goldman); fluorescein and indocyanine-green angiography; and optical coherence tomography (OCT).
No adverse effects due to the injection were observed. A 1-line improvement in ETDRS BCVA was measured in 4 patients 1 week after injection and was maintained throughout follow-up. Three patients showed undetectable ERG responses at all study visits, while one patient showed residual responses for dark-adapted standard flash stimulus (a-wave amplitude around 35 µV) which remained recordable throughout follow-up. One patient showed a small response (a-wave amplitude around 20 µV) recordable only at weeks 7, 13, 22 and 40. Visual fields showed no reduction (Goldman Standard V5e) for any patient at any visit. No other changes were observed on OCT or fluorescein and indocyanine green angiograms.
Intravitreal injection of ABMSC in eyes with advanced RP was followed by absence of detectable structural or functional retinal worsening over a period of 10 months. Further studies are required to investigate the role, if any, of bone marrow derived stem cell therapy for the management of RP.
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