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 . 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.

中文翻译：

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Stargardt型黄斑营养不良患者的玻璃体内使用含CD34 +细胞的骨髓单核部分（BMMF）

评估Stargardt型黄斑营养不良患者体内含CD34 +细胞的骨髓单核部分（BMMF）的治疗潜力和玻璃体内安全性。该研究是针对10例患有Stargardt营养不良的病。从所有患者获得骨髓抽吸物，并在细胞治疗中心（CTC）进行处理后，将0.1 ml玻璃体内BMMF悬浮液注入视力较差的眼睛。在对侧眼中进行假注射。在基线以及注射后1、3和6个月对患者进行评估。他们都接受了最佳矫正视力（BCVA），显微视野，多焦点视网膜电图（mfERG）和全视野视网膜电图（ffERG），自发荧光（AF）和光学相干断层扫描（OCT）的测量。注射前和注射后六个月还进行了荧光素血管造影。所有患者均完成了六个月的评估。玻璃体内注射（IV）之前，治疗眼的平均视力为1.1 logMAR（20/250），为0。注射后1个月为96 logMAR（20/200 + 2），注射后3个月为0.92 logMAR（20 / 160-1）。在未经治疗的眼中，注射后一个月和三个月的平均VA分别为基线时的1.0 logMAR（20/200）和0.96 logMAR（20/200 + 2）和0.94 logMAR（20 / 160-2）。在治疗组中，基线时的VA范围从最佳视力20 / 125-1到最差视力20/640 + 2，在第一个月内经历了20/100 + 2和20/400。在未经治疗的组中，BCVA的基线范围为20/100 + 2至20/400，一个月后为20/100至20/400。治疗组的结果在所有随访时间均存在显着差异，而未治疗组未观察到显着差异。关于微视野测定法的平均灵敏度，尽管所有月份都有改善，仅在第一个月发生了显着差异。在未经处理的眼睛中，任何分析均无显着差异。血管荧光素造影未显示新血管形成或肿瘤生长。其余的检查用于帮助诊断。结果表明，与未治疗的眼睛相比，在患有Stargardt营养不良的患者中使用玻璃体内BMMF是安全的，并且与治疗后的眼睛中BCVA和微孔率的离散改善有关。