Division of Rheumatology and Center for Pediatric Clinical Effectiveness, Roberts Center for Pediatric Research, Children's Hospital of Philadelphia, 2716 South Street, Room 11121, Philadelphia, PA, 19104, USA.
Center for Clinical Epidemiology and Biostatistics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, USA.
Trials. 2023 Feb 8;24(1):100. doi: 10.1186/s13063-022-07038-6.
The effectiveness of biologic therapies, primarily tumor necrosis factor inhibitors (TNFi), for children with spondyloarthritis (SpA) has made inactive disease a realistic patient outcome. However, biologic therapies are costly, primarily delivered by subcutaneous or intravenous route, and have non-trivial side effects. Many patients and families want to know if biologic medications can be discontinued after inactive disease is achieved. It remains unclear whether medication dose should remain unchanged, tapered (increase the time between doses), or discontinued once when inactive disease is attained.
The Biologic Abatement and Capturing Kids' Outcomes and Flare Frequency in Juvenile SpA (BACK-OFF JSpA) trial is a multicenter pragmatic trial that will randomize 198 participants ages 8-21 years old with SpA and sustained inactive disease on standard TNFi dosing to (1) continue standard TNFi dosing, (2) fixed longer dosing intervals of TNFi, or (3) stop TNFi. The trial will compare the hazard rate of protocol-defined flare and participants' emotional health among the 3 groups over 12 months. Innovative aspects of this trial are the involvement of patient and parent stakeholders in the design and conduct of the study as well as an electronic health record-based enhanced recruitment strategy.
This is the first randomized pragmatic trial to assess the efficacy of TNFi de-escalation strategies in children with JSpA with sustained inactive disease. This research will improve the evidence base that patients, caregivers, and rheumatologists use to make shared decisions about continued treatment versus de-escalation of TNFi therapy in this population.
ClinicalTrials.gov NCT04891640. Registered on 18 May 2021.
生物疗法（主要是肿瘤坏死因子抑制剂[TNFi]）对患有脊柱关节炎（SpA）的儿童的有效性使得疾病无活动成为现实的患者结局。然而，生物疗法成本高昂，主要通过皮下或静脉途径给药，并且具有不可忽视的副作用。许多患者和家属想知道在达到疾病无活动后是否可以停用生物药物。目前尚不清楚一旦达到疾病无活动，药物剂量是否应保持不变、逐渐减少（增加剂量之间的时间间隔）还是停用。
生物药物消除和捕获儿童青少年脊柱关节炎结局和复发频率（BACK-OFF JSpA）试验是一项多中心实用临床试验，将随机纳入 198 名年龄在 8-21 岁之间、正在接受标准 TNFi 剂量治疗且疾病持续无活动的 SpA 患者，将其分为（1）继续标准 TNFi 剂量治疗，（2）固定更长 TNFi 给药间隔，或（3）停用 TNFi。该试验将在 12 个月内比较 3 组中方案定义的复发的危险率和参与者的心理健康。该试验的创新之处在于患者和家长利益相关者参与了研究的设计和实施，以及采用了电子病历为基础的增强招募策略。
这是第一项评估在疾病持续无活动的青少年 JSpA 患者中降低 TNFi 治疗强度策略的有效性的随机实用试验。这项研究将改善患者、护理人员和风湿病学家用于在该人群中做出关于继续治疗或降低 TNFi 治疗强度的共享决策的证据基础。
ClinicalTrials.gov NCT04891640。于 2021 年 5 月 18 日注册。
