Delivery of AAV-based gene therapy through haemophilia centres-A need for re-evaluation of infrastructure and comprehensive care: A Joint publication of EAHAD and EHC
Delivery of AAV-based gene therapy through haemophilia centres-A need for re-evaluation of infrastructure and comprehensive care: A Joint publication of EAHAD and EHC
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DOI：
10.1111/hae.14420
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发表时间：
2021-09-22
期刊：
影响因子：
3.9
通讯作者：
Peyvandi, Flora
中科院分区：
文献类型：
--
作者：
Miesbach, Wolfgang;Chowdary, Pratima;Peyvandi, Flora
简介 基于腺相关病毒 (AAV) 的血友病基因治疗对血友病中心的现有结构提出了挑战，需要重新思考当前的合作和信息交换，以确保一个适合先进疗法的系统，以最大限度地提高效益并最大限度地降低风险。在欧洲，欧洲血友病网络 (EUHANET) 向血友病中心提供基于患者和设施数量的认证流程。目的和方法欧洲血友病及相关疾病协会 (EAHAD) 和欧洲血友病联盟 (EHC) 联合出版物描述了参与基因治疗护理的中心的标准，需要重新评估综合护理的基础设施，并提供了如何在血友病中心未来工作中实施这些标准的展望。结果 血友病治疗中心的核心定义仍然存在，但可以发挥其他作用。可修改的“中心辐射”模型解决了与基因治疗相关的所有方面，包括基因治疗产品的制备和管理、凝血和免疫参数的测定、关节评分和功能以及肝脏健康。这还包括如何跟踪患者进行长期安全性和有效性监测的策略。结论 我们提出了一种可修改的、网络化的“中心辐射”模型，具有长期的安全性和有效性监测系统。这种方法将逐步发展，目标是使血友病中心更有资格提供基因治疗，并使所有血友病患者都能获得基因治疗，无论其国家或来源中心如何。
Introduction Adeno-associated virus (AAV)-based gene therapy for haemophilia presents a challenge to the existing structure of haemophilia centres and requires a rethink of current collaboration and information exchange with the aim of ensuring a system that is fit-for-purpose for advanced therapies to maximise benefits and minimise risks. In Europe, a certification process based on the number of patients and facilities is offered to the haemophilia centres by European Haemophilia Network (EUHANET). Aim and methods This joint European Association for Haemophilia and Allied Disorders (EAHAD) and European Haemophilia Consortium (EHC) publication describes criteria for centres participating in gene therapy care that require a reassessment of the infrastructure of comprehensive care and provides an outlook on how these criteria can be implemented in the future work of haemophilia centres. Results The core definition of a haemophilia treatment centre remains, but additional roles could be implemented. A modifiable 'hub-and-spoke' model addresses all aspects associated with gene therapy, including preparation and administration of the gene therapy product, determination of coagulation and immunological parameters, joint score and function, and liver health. This will also include the strategy on how to follow-up patients for a long-term safety and efficacy surveillance. Conclusion We propose a modifiable, networked 'hub and spoke' model with a long term safety and efficacy surveillance system. This approach will be progressively developed with the goal of making haemophilia centres better qualified to deliver gene therapy and to make gene therapy accessible to all persons with haemophilia, irrespective of their country or centre of origin.