Department of Ophthalmology, Otorhinolaryngology and Head and Neck Surgery, School of Medicine of Ribeirão Preto, University of São Paulo, Ribeirao Preto, Brazil.
Retina. 2011 Jun;31(6):1207-14. doi: 10.1097/IAE.0b013e3181f9c242.
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.
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 × 10(6) autologous bone marrow-derived mononuclear cells (0.1 mL) into 1 study eye of each patient.
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 μV), which remained recordable throughout follow-up, and 1 patient showed a small response (a wave amplitude approximately 20 μV) 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.
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.
评估单次玻璃体内注射自体骨髓源性单核细胞治疗色素性视网膜炎或圆锥-杆营养不良患者的短期（10 个月）安全性。
前瞻性、I 期、非随机、开放性研究，纳入 3 名色素性视网膜炎患者和 2 名圆锥-杆营养不良患者，最佳矫正视力（BCVA）为 20/200 或更差，符合早期治疗糖尿病性视网膜病变研究标准。对每位患者的 1 只眼玻璃体内注射 10×106 个自体骨髓源性单核细胞（0.1ml）后，于基线及 1、7、13、18、22 和 40 周进行 BCVA、全视野视网膜电图（金标准闪光视觉诱发电位）、动态视野（Goldman）、荧光素和吲哚青绿血管造影及光学相干断层扫描检查。
未观察到与注射相关的不良事件。4 例患者于注射后 1 周视力提高 1 行，且随访期间一直保持该视力水平。3 例患者在所有研究访视时视网膜电图均无反应，而 1 例患者暗适应标准闪光刺激的视网膜电图 a 波振幅约为 35μV，在随访期间一直可记录到，1 例患者仅在第 7、13、22 和 40 周可记录到较小的反应（a 波振幅约为 20μV）。任何患者在任何时间点的视野检查均未出现金标准 V5e 刺激的缩小。光学相干断层扫描或荧光素和吲哚青绿血管造影未见其他变化。
在晚期色素性视网膜炎或圆锥-杆营养不良眼中，玻璃体内注射自体骨髓源性单核细胞在 10 个月内无结构或功能毒性。需要进一步研究以探讨自体骨髓源性单核细胞治疗在视网膜营养不良管理中的作用。
