Cotrim Carina Costa, Vieira Messias André M, Jorge Rodrigo, Siqueira Rubens Camargo
Department of Ophthalmology, Otorhinolaryngology and Head and Neck Surgery, Ribeirão Preto Medical School, University of São Paulo, Brazil.
Stem Cells Int. 2020 Dec 10;2020:8828256. doi: 10.1155/2020/8828256. eCollection 2020.
To assess the therapeutic potential and the safety of intravitreous use of a bone marrow mononuclear fraction (BMMF) containing CD34+ cells in patients with Stargardt type macular dystrophy. The study was conducted on 10 patients with Stargardt dystrophy with worse eye visual acuity ≤ 20/125. A bone marrow aspirate was obtained from all patients, and after processing in the cell therapy center (CTC), 0.1 ml of the intravitreous BMMF suspension was injected into the eye with worse visual acuity. A sham injection was performed in the contralateral eye. The patients were evaluated at baseline and one, three, and six months after the injection. All of them were submitted to measurement of best corrected visual acuity (BCVA), microperimetry, multifocal electroretinography (mfERG) and full field electroretinography (ffERG), autofluorescence (AF), and optical coherence tomography (OCT). Fluorescein angiography was also performed before and six months after the injection. All patients completed the six-month period of evaluation. Mean visual acuity of the treated eye was 1.1 logMAR (20/250) before intravitreous (IV) injection, 0.96 logMAR (20/200+2) one month after injection, and 0.92 logMAR (20/160-1) 3 months after injection. In the untreated eye, mean VA was 1.0 logMAR (20/200) at baseline and 0.96 logMAR (20/200+2) and 0.94 logMAR (20/160-2) one and three months after injection, respectively. In the treated group, VA at baseline ranged from best acuity of 20/125-1 to worst acuity of 20/640+2, going through 20/100+2 and 20/400 during the first month. In the untreated group, BCVA ranged from 20/100+2 to 20/400 at baseline and from 20/100 to 20/400 after one month. The results for the treated group differed significantly at all follow-up times, whereas no significant difference was observed in the untreated group. Regarding the mean sensitivity of microperimetry, although there was improvement throughout all months, a significant difference occurred only during the first month. In the untreated eye, there was no significant difference in any analysis. Angiofluoresceinography did not reveal neovessel formation or tumor growth. The remaining exams were used in order to aid the diagnosis. The results indicate that the use of intravitreous BMMF in patients with Stargardt dystrophy is safe and is associated with a discrete improvement of BCVA and microperimetry in the treated eye compared to the untreated one.
评估玻璃体内注射含CD34+细胞的骨髓单个核细胞组分（BMMF）治疗斯塔加特型黄斑营养不良患者的治疗潜力和安全性。该研究纳入了10例斯塔加特营养不良且患眼最佳视力≤20/125的患者。从所有患者获取骨髓抽吸物，在细胞治疗中心（CTC）处理后，将0.1ml玻璃体内BMMF悬浮液注入视力较差的眼睛。对侧眼进行假注射。在注射前及注射后1个月、3个月和6个月对患者进行评估。所有患者均接受最佳矫正视力（BCVA）、微视野检查、多焦视网膜电图（mfERG）和全视野视网膜电图（ffERG）、自发荧光（AF）以及光学相干断层扫描（OCT）检查。在注射前和注射后6个月还进行了荧光素血管造影。所有患者均完成了6个月的评估期。治疗眼在玻璃体内（IV）注射前的平均视力为1.1 logMAR（20/250），注射后1个月为0.96 logMAR（20/200+2），注射后3个月为0.92 logMAR（20/160-1）。在未治疗眼，基线时平均视力为1.0 logMAR（20/200），注射后1个月和3个月分别为0.96 logMAR（20/200+2）和0.94 logMAR（20/160-2）。在治疗组中，基线时视力范围从最佳的20/125-1到最差的20/640+2，在第一个月经历了20/100+2和20/400。在未治疗组中，基线时BCVA范围为20/100+2至20/400，1个月后为20/100至20/400。治疗组在所有随访时间的结果均有显著差异，而未治疗组未观察到显著差异。关于微视野检查的平均敏感度，尽管在所有月份均有改善，但仅在第一个月出现显著差异。在未治疗眼中，任何分析均未发现显著差异。荧光素血管造影未显示新生血管形成或肿瘤生长。其余检查用于辅助诊断。结果表明，在斯塔加特营养不良患者中玻璃体内注射BMMF是安全的，与未治疗眼相比，治疗眼的BCVA和微视野检查有轻微改善。
