背景:急性非动脉炎性缺血性视神经病变(NA-AION)的有效治疗方法尚未被知晓或证实。以前的研究表明,同种异体骨髓间充质干细胞具有神经保护作用。这项研究旨在报告对接受玻璃体腔注射同种异体骨髓间充质干细胞(BM-MSCs)(MSV®)治疗的急性非动脉炎性视神经病变(NA-AION)患者的临床试验结果。
方法:我们进行了前瞻性,非随机化,临床II期研究(EudraCT编号2016-003029-40;ClinicalTrials.govRegistryNCT03173638),纳入5例在症状发作后2周内诊断为急性单侧NA-AION的患者,并接受玻璃体内注射同种异体BM-MSCs(0.05ml;细胞浓度:1.5×106cells/mL).患者接受了定期眼科检查,并随访了一年。
结果:在本试验中,同种异体BM-MSCs似乎是安全的,因为没有患者出现急性或慢性眼内炎症或眼压明显变化的迹象,尽管一名患者出现了视网膜前膜。在另一位有意义的患者中,注射后不久形成的后牙聚集体在几周内自发消失。留下囊下白内障。4例患者视力改善,3例患者的视觉诱发电位记录中P100的振幅增加。随访期间视网膜神经纤维层和黄斑神经节细胞层厚度明显下降。
结论:除了一个患者的视网膜前膜的发展,玻璃体内应用同种异体BM-MSCs在眼内表现出良好的耐受性.因此,不仅NA-AION,而且BM-MSCs也值得获得更多的临床试验资源和更大的随机多中心试验,这将为玻璃体内注射同种异体BM-MSCs在急性NA-AION中的安全性和潜在治疗效果提供更有力的证据.
背景:玻璃体内间质干细胞治疗急性非动脉炎性前部缺血性视神经病变(神经干细胞)的安全性评估。NCT03173638。注册2017年6月2日https://clinicaltrials.gov/ct2/show/NCT03173638。
An effective treatment for acute non-arteritic ischemic optic neuropathy (NA-AION) has not been known or proven yet. Previous studies have suggested a neuroprotective effect of allogeneic bone marrow-derived mesenchymal stem cells. This
study aims to report the results of a clinical
trial on patients with acute non-arteritic optic neuropathy (NA-AION) treated with an intravitreal injection of allogeneic bone marrow-derived mesenchymal stem cells (BM-MSCs) (MSV®).
We conducted a prospective, non-randomized, clinical phase-II
study (Eudra CT number 2016-003029-40; ClinicalTrials.gov Registry NCT03173638) that included 5 patients with acute unilateral NA-AION diagnosed within 2 weeks after symptom onset and who received an intravitreal injection of allogeneic BM-MSCs (0.05 ml; cell concentration: 1.5 × 106cells/mL). The patients underwent regular ophthalmological examinations and were followed for one year.
In this
trial, allogeneic BM-MSCs appeared to be safe as no patients developed signs of acute nor chronic intraocular inflammation or a significant change in intraocular pressure, although an epiretinal membrane was developed in one patient. A retrolental aggregate formed shortly after the injection spontaneously disappeared within a few weeks in another phakic patient, leaving a subcapsular cataract. Visual improvement was noted in 4 patients, and amplitudes of P100 on the visually evoked potentials recordings increased in three patients. The retinal nerve fiber layer and macular ganglion cell layer thicknesses significantly decreased during the follow-up.
Besides the development of an epiretinal membrane in one patient, the intravitreal application of allogeneic BM-MSCs appeared to be intraocularly well tolerated. Consequently, not only NA-AION but also BM-MSCs deserve more clinical investigational resources and a larger randomized multicenter
trial that would provide stronger evidence both about safety and the potential therapeutic efficacy of intravitreally injected allogeneic BM-MSCs in acute NA-AION.
Safety Assessment of Intravitreal Mesenchymal Stem Cells for Acute Non-Arteritic Anterior Ischemic Optic Neuropathy (NEUROSTEM). NCT03173638. Registered June 02, 2017 https://clinicaltrials.gov/ct2/show/NCT03173638 .