INTRAVITREAL INJECTION OF AUTOLOGOUS BONE MARROW-DERIVED MONONUCLEAR CELLS FOR HEREDITARY RETINAL DYSTROPHY A Phase I Trial

Autor(es)

Siqueira, Rubens C. ^{[1, 2]} ; Messias, Andre ^[1] ; Voltarelli, Julio C. ^[3] ; Scott, Ingrid U. ^{[4, 5]} ; Jorge, Rodrigo ^[1]

Afiliação do(s) autor(es)

^[1] Univ Sao Paulo, Sch Med Ribeirao Preto, Dept Ophthalmol, BR-14049 Ribeirao Preto - Brazil

^[2] Sch Med Catanduva, Dept Ophthalmol, Catanduva - Brazil

^[3] Univ Sao Paulo, Sch Med Ribeirao Preto, Dept Internal Med, Ribeirao Preto - Brazil

^[4] Penn State Coll Med, Dept Ophthalmol, Hershey, PA - USA

^[5] Penn State Coll Med, Dept Publ Hlth Sci, Hershey, PA - USA

• Tipo de documento: Artigo Científico
• Fonte: RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES; v. 31, n. 6, p. 1207-1214, JUN 2011.
• Citações Web of Science: 59

Resumo

Purpose: To evaluate the short-term (10 months) safety of a single intravitreal injection of autologous bone marrow-derived mononuclear cells in patients with retinitis pigmentosa or cone-rod dystrophy. Methods: A prospective, Phase I, nonrandomized, open-label study including 3 patients with retinitis pigmentosa and 2 patients with cone-rod dystrophy and an Early Treatment Diabetic Retinopathy Study best-corrected visual acuity of 20/200 or worse. Evaluations including best-corrected visual acuity, full-field electroretinography, kinetic visual field (Goldman), fluorescein and indocyanine green angiography, and optical coherence tomography were performed at baseline and 1, 7, 13, 18, 22, and 40 weeks after intravitreal injection of 10 X 10(6) autologous bone marrow-derived mononuclear cells (0.1 mL) into 1 study eye of each patient. Results: No adverse event associated with the injection was observed. A 1-line improvement in best-corrected visual acuity was measured in 4 patients 1 week after injection and was maintained throughout follow-up. Three patients showed undetectable electroretinography responses at all study visits, while 1 patient demonstrated residual responses for dark-adapted standard flash stimulus (a wave amplitude approximately 35 mu V), which remained recordable throughout follow-up, and 1 patient showed a small response (a wave amplitude approximately 20 mu V) recordable only at Weeks 7, 13, 22, and 40. Visual fields showed no reduction (with a Goldman Standard V5e stimulus) for any patient at any visit. No other changes were observed on optical coherence tomography or fluorescein and indocyanine green angiograms. Conclusion: Intravitreal injection of autologous bone marrow-derived mononuclear cells in eyes with advanced retinitis pigmentosa or cone-rod dystrophy was associated with no detectable structural or functional toxicity over a period of 10 months. Further studies are required to investigate the role, if any, of autologous bone marrow-derived mononuclear cell therapy in the management of retinal dystrophies. RETINA 31: 1207-1214, 2011 (AU)

• Processo FAPESP: 07/59078-8 - Emprego do eletroretinograma multifocal e da adaptação ao escuro medida com eletroretinograma de campo total na detecção e segmento clínico da doença macular relacionada à idade
• Beneficiário: André Marcio Vieira Messias
• Modalidade de apoio: Auxílio à Pesquisa - Jovens Pesquisadores