Bosma A L, Spuls P I, Garcia-Doval I, Naldi L, Prieto-Merino D, Tesch F, Apfelbacher C J, Arents B W M, Barbarot S, Baselga E, Deleuran M, Eichenfield L F, Gerbens L A A, Irvine A D, Manca A, Mendes-Bastos P, Middelkamp-Hup M A, Roberts A, Seneschal J, Svensson Å, Thyssen J P, Torres T, Vermeulen F M, Vestergaard C, von Kobyletzki L B, Wall D, Weidinger S, Schmitt J, Flohr C
Amsterdam UMC, Location AMC, University of Amsterdam, Department of Dermatology, Amsterdam Public health, Infection and Immunity, Amsterdam, the Netherlands.
Research Unit, Academia Española de Dermatología y Venereología, Madrid, Spain.
Br J Dermatol. 2020 Jun;182(6):1423-1429. doi: 10.1111/bjd.18452. Epub 2019 Nov 20.
A long-term prospective observational safety study is essential to characterize fully the safety profile of systemic immunomodulating therapies for patients with atopic eczema. The TREatment of ATopic eczema (TREAT) Registry Taskforce offers a large platform to conduct such research using national registries that collect the same data using a predefined core dataset.
To present a protocol for a safety study comparing dupilumab with other systemic immunomodulating therapies in children and adults with moderate-to-severe atopic eczema, to assess the long-term safety risk of these therapies in a routine clinical care setting.
We describe a registry-embedded international observational prospective cohort study. Adult and paediatric patients who start treatment with dupilumab or another systemic immunomodulating agent for their atopic eczema will be included. The primary end point is the incidence of malignancies (excluding nonmelanoma skin cancer) compared between the treatment groups. Secondary end points include other serious adverse events and adverse events of special interest, such as eye disorders and eosinophilia.
This protocol delineates a safety study for dupilumab in adult and paediatric patients with atopic eczema, using a standardized methodological approach across several national registries. The protocol could also be used for other novel systemic immunomodulating therapies, and could provide licensing and reimbursement authorities, pharmaceutical companies and clinicians with safety evidence from a routine clinical care setting. What's already known about this topic? There is a need for long-term data on the safety of systemic immunomodulating therapies in patients with atopic eczema. Regulatory bodies, such as the European Medicines Agency, increasingly stipulate the collection of such data as part of the licensing agreement for new treatments, to assess the new agent's long-term safety profile against established therapies. Large numbers of patients with a long duration of follow-up are necessary in order to detect rare events like malignancies. What does this study add? The TREAT Registry Taskforce offers a platform to conduct such research with a network of multiple national atopic eczema research registries. We present a protocol for an investigator-initiated multicentre safety study comparing dupilumab with other systemic immunomodulating therapies in adults and subsequently adolescents and children with moderate-to-severe atopic eczema. This protocol can be used as a framework for similar studies for other novel systemic immunomodulating therapies across both adult and paediatric populations.
一项长期前瞻性观察性安全性研究对于全面描述系统性免疫调节疗法治疗特应性皮炎患者的安全性至关重要。特应性皮炎治疗（TREAT）注册工作组提供了一个大型平台，可利用国家注册库开展此类研究，这些注册库使用预定义的核心数据集收集相同的数据。
提出一项安全性研究方案，比较度普利尤单抗与其他系统性免疫调节疗法治疗中重度特应性皮炎儿童和成人的安全性，评估这些疗法在常规临床护理环境中的长期安全风险。
我们描述了一项纳入注册库的国际观察性前瞻性队列研究。将纳入开始使用度普利尤单抗或其他系统性免疫调节药物治疗特应性皮炎的成人和儿科患者。主要终点是比较各治疗组之间恶性肿瘤（不包括非黑色素瘤皮肤癌）的发生率。次要终点包括其他严重不良事件和特别关注的不良事件，如眼部疾病和嗜酸性粒细胞增多。
本方案描述了一项针对成人和儿科特应性皮炎患者使用度普利尤单抗的安全性研究，采用了跨多个国家注册库的标准化方法。该方案也可用于其他新型系统性免疫调节疗法，并可为许可和报销机构、制药公司和临床医生提供来自常规临床护理环境的安全性证据。关于该主题已知的信息有哪些？需要有关系统性免疫调节疗法治疗特应性皮炎患者安全性的长期数据。监管机构，如欧洲药品管理局，越来越多地规定收集此类数据作为新疗法许可协议的一部分，以评估新药物相对于现有疗法的长期安全性。为了检测如恶性肿瘤等罕见事件，需要大量长期随访的患者。本研究增加了什么？TREAT注册工作组提供了一个平台，可通过多个国家特应性皮炎研究注册库网络开展此类研究。我们提出了一项由研究者发起的多中心安全性研究方案，比较度普利尤单抗与其他系统性免疫调节疗法治疗中重度特应性皮炎的成人患者，随后是青少年和儿童患者。该方案可作为针对成人和儿科人群的其他新型系统性免疫调节疗法类似研究的框架。
