The substantial morbidity and mortality associated with refractory systemic JIA underlies the need for new treatment approaches. However, progress in this area has been limited by the difficulty of enrolling these patients in clinical trials with traditional designs, particularly in patients presenting with the life-threatening macrophage activation syndrome. At the NextGen 2022 conference, there was group consensus that using historical cohorts as a control group to avoid the need for a placebo-arm or drug withdrawal was highly desirable and might be acceptable for clinical trials in MAS to support medication efficacy and safety. However, if historic controls were used in a trial, it would be important to ensure that the historic cohort matches the study group in terms of clinical characteristics (such as disease severity and exposure to other medications), and that disease outcome in both groups is assessed using the same outcome measures. The discussions at the NextGen 2022 conference focused on the potential strategies to achieve these goals.

中文翻译：

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针对 SJIA 和 MAS 的第四次 NextGen 疗法：难治性 SJIA 临床试验第 3 部分：历史对照作为撤回设计研究的替代方案

与难治性系统性幼年特发性关节炎相关的高发病率和死亡率表明需要新的治疗方法。然而，由于难以将这些患者纳入传统设计的临床试验，特别是患有危及生命的巨噬细胞活化综合征的患者，该领域的进展受到限制。在 NextGen 2022 会议上，小组一致认为，使用历史队列作为对照组以避免安慰剂组或停药的需要是非常可取的，并且对于 MAS 的临床试验来说可能是可以接受的，以支持药物疗效和安全性。然而，如果在试验中使用历史对照，重要的是要确保历史队列在临床特征（例如疾病严重程度和接触其他药物）方面与研究组相匹配，并且两组的疾病结果是使用相同的结果测量进行评估。NextGen 2022 会议的讨论重点是实现这些目标的潜在策略。