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Abstract：
In order to treat a pediatric patient with ligneous conjunctivitis secondary to congenital plasminogen deficiency, a supply of topically administered replacement human plasminogen has been required. In the absence of market approval, this blood-derived drug is managed by a temporary authorisation for nominative use, allowing monthly hospital dispensations. To ensure regulatory compliance and proper use of the drug, it took two years of interactions between various hospital departments and the laboratory to define the pharmaceutical supply chain in our hospital and allow the patient to receive treatment. The main difficulties lie in respecting the cold chain of this drug stored frozen in the bottles not ready for use. Transportation from the laboratory to the patient\'s home via the hospital pharmacy is carried out in calibrated conditions, ensuring a temperature below -20°C for 72h. Reception and dispensing steps were combined into a single pharmaceutical service in order to optimise transport time while ensuring the safety and traceability of the drug lots. Each month, a date is scheduled between the hospital pharmacy, the laboratory and the family to ensure that delivery and dispensing take place on the same day. Appropriate use and handling are explained to the family. However, two issues remain to be addressed by the manufacturer to facilitate future use of human plasminogen: the thermostability problem, which does not allow stays away from home longer than three days, and self-administration by the child, which is unlikely to be feasible due to handling difficulties.
摘要：
为了治疗先天性纤溶酶原缺乏症继发的结膜炎患儿，需要提供局部给药的替代人纤溶酶原。在没有市场认可的情况下,这种血液衍生药物由临时授权管理，允许每月住院。为了确保遵守法规和正确使用药物，医院各部门和实验室之间的互动花了两年时间来定义我们医院的药品供应链，并允许患者接受治疗。主要困难在于尊重冷冻储存在未准备好使用的瓶子中的这种药物的冷链。通过医院药房从实验室到患者家的运输在校准条件下进行，确保温度低于-20°C持续72h。为了优化运输时间，同时确保药品批次的安全性和可追溯性，将接收和分配步骤合并到单个药品服务中。每个月,医院药房之间有一个日期，实验室和家庭，以确保交付和分配在同一天进行。向家人解释了适当的使用和处理方式。然而,为了促进人类纤溶酶原的未来使用，制造商仍需要解决两个问题：热稳定性问题，不允许离家超过三天，和孩子的自我管理，由于处理困难，这不太可能可行。
