UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adults.

IF 3 2区医学 Q2 HEMATOLOGY

Haemophilia Pub Date : 2024-11-20 DOI:10.1111/hae.15125

Pratima Chowdary, Beatriz Duran, Paul Batty, Gillian Lowe, April Jones, Debra Pollard, Sara Boyce, Jayashree Motwani, Bahareh Amirloo, Kathryn Musgrave, David Hopper, Stephen Classey, Sarah Whitaker, Nicola Dunn, Annette Bowyer, Susan Shapiro

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引用次数: 0

Abstract

Introduction: 2022 was a landmark year with two adeno-associated viral vectors (AAVs) receiving conditional marketing authorization from EMA for the treatment of persons with severe haemophilia A and severe to moderately severe haemophilia B and a third in 2024. Gene therapy is a transformative, irreversible treatment with long-lasting effects, necessitating development of new clinical pathways to ensure optimal outcomes.

Aim: To develop a consensus framework and service specification for delivery of AAV gene therapy for haemophilia in adults within the UK using the hub-and-spoke model proposed by the European Association of Haemophilia and Allied Disorders and the European Haemophilia Consortium.

Methods: The UK Haemophilia Centre Doctors Organisation (UKHCDO) set up a working party to develop expert consensus guidance, working with NHS England to ensure alignment with NHS England commissioning and the national service specification.

Results: These guidelines detail the patient pathway, counselling and governance requirements for the hub-and-spoke model. The national service specification requires the hub site to manage governance for AAV-based gene therapy. Proposed regional and national multidisciplinary teams will harmonize clinical practices incorporating expertise from various specialities and professional groups. Key requirements identified include standardized documentation and multidisciplinary collaboration. Nationally agreed patient information and counselling checklists will streamline the informed consent process and facilitate data collection for long-term safety and efficacy monitoring.

Conclusion: These guidelines provide a structured framework for the delivery of liver-directed gene therapy. Whilst specific to the United Kingdom they provide a framework for the implementation of gene therapy in other countries for haemophilia and other monogenic disorders.

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英国血友病组织基因治疗工作组：成人血友病基因治疗常规临床实践实施指南。

导言：2022 年是具有里程碑意义的一年，两款腺相关病毒载体（AAV）获得了欧洲药品管理局（EMA）的有条件上市许可，用于治疗重度血友病 A 患者和重度至中度血友病 B 患者，第三款 AAV 将于 2024 年上市。目的：采用欧洲血友病及相关疾病协会（European Association of Haemophilia and Allied Disorders）和欧洲血友病联盟（European Haemophilia Consortium）提出的 "中心辐射"（hub-and-spoke）模式，制定英国成人血友病 AAV 基因疗法的共识框架和服务规范：方法：英国血友病中心医生组织（UKHCDO）成立了一个工作小组，与英格兰国家医疗服务体系合作制定专家共识指南，确保与英格兰国家医疗服务体系的委托和国家服务规范保持一致：这些指南详细介绍了中心辐射模式的患者路径、咨询和管理要求。国家服务规范要求中心站对基于 AAV 的基因治疗进行管理。拟议中的地区和国家多学科团队将协调临床实践，吸收各专科和专业团体的专业知识。已确定的主要要求包括标准化文件和多学科协作。全国统一的患者信息和咨询核对表将简化知情同意程序，促进长期安全性和疗效监测数据的收集：这些指南为肝脏定向基因疗法的实施提供了一个结构化框架。虽然这些指南仅适用于英国，但它们为其他国家实施血友病和其他单基因遗传疾病的基因疗法提供了框架。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Haemophilia 医学-血液学

CiteScore

6.50

自引率

28.20%

发文量

226

审稿时长

3-6 weeks

期刊介绍： Haemophilia is an international journal dedicated to the exchange of information regarding the comprehensive care of haemophilia. The Journal contains review articles, original scientific papers and case reports related to haemophilia care, with frequent supplements. Subjects covered include: clotting factor deficiencies, both inherited and acquired: haemophilia A, B, von Willebrand''s disease, deficiencies of factor V, VII, X and XI replacement therapy for clotting factor deficiencies component therapy in the developing world transfusion transmitted disease haemophilia care and paediatrics, orthopaedics, gynaecology and obstetrics nursing laboratory diagnosis carrier detection psycho-social concerns economic issues audit inherited platelet disorders.