Advanced Therapy Unit, Hospital Clinic, Escala 3, Planta 1 Criopreservació, C/Villarroel 170, 08036, Barcelona, Spain.
Fondazione Banca degli Occhi del Veneto, Venice, Italy.
Cell Tissue Bank. 2020 Jun;21(2):189-204. doi: 10.1007/s10561-020-09813-8. Epub 2020 Feb 4.
We evaluated the feasibility and performed a risk-benefit analysis of the storage and widespread distribution of stromal lenticules for clinical application using a new systematic tool (European Good Tissue and cells Practices II-EuroGTP II tool), specifically designed for assessing the risk, safety and efficacy of substances of human origin. Three types of potential tissue preparations for human stromal lenticules were evaluated: cryopreserved, dehydrated and decellularized. The tool helps to identify an overall risk score (0-2: negligible; 2-6: low; 6-22: moderate; > 22: high) and suggests risk reduction strategies. For all the three types of products, we found the level of risk to be as "moderate". A process validation, pre-clinical in vitro and in vivo evaluations and a clinical study limited to a restricted number of patients should therefore be performed in order to mitigate the risks. Our study allowed to establish critical points and steps necessary to implement a new process for safe stromal lenticule preparation by the eye banks to be used in additive keratoplasty. Moreover, it shows that the EuroGTP II tool is useful to assess and identify risk reduction strategies for introduction of new Tissue and Cellular Therapies and Products into the clinical practice.
我们使用新的系统工具（专门用于评估人体来源物质的风险、安全性和有效性的欧洲良好组织和细胞实践 II- EuroGTP II 工具）评估了储存和广泛分发用于临床应用的基质透镜的可行性，并进行了风险效益分析。评估了三种潜在的人类基质透镜组织制备类型：冷冻保存、脱水和去细胞化。该工具有助于确定总体风险评分（0-2：可忽略；2-6：低；6-22：中等；>22：高）并提出降低风险策略。对于所有三种类型的产品，我们发现风险水平为“中等”。因此，应进行工艺验证、临床前体外和体内评估以及仅限于少数患者的临床研究，以降低风险。我们的研究确定了实施新的安全基质透镜制备过程的关键点和步骤，该过程由眼库用于添加剂角膜移植。此外，它表明 EuroGTP II 工具可用于评估和确定引入新的组织和细胞疗法和产品进入临床实践的风险降低策略。
