: Objectives To elicit and quantify expert opinion concerning the relative merits of two treatments for a rare inflammatory disease: Juvenile dermatomyositis (JDM). The formal expression of expert opinion reported in this paper will be used in a Bayesian analysis of a forthcoming randomised controlled trial known as BARJDM (baricitinib for juvenile dermatomyositis). Methods A Bayesian prior elicitation meeting was convened, following a previously described methodological template. Opinion was sought on the probability that a patient in the BARJDM trial would achieve clinically inactive disease, off glucocorticoids (GC) within a 12-month period with either methotrexate (standard of care); or baricitinib (a Janus kinase inhibitor, JAKi), with GC schedules identical in both arms of the trial. Experts’ views were discussed and refined following presentation and further discussion of summated published data regarding efficacy of methotrexate or JAKi for JDM. Results Ten UK paediatric rheumatology consultants (including one adolescent paediatric rheumatologist) participated in the elicitation meeting. All had expertise in JDM, leading active National Health Service clinics for this disease. Consensus expert prior opinion was that the most likely probability of clinically inactive disease off GC within 12 months was 0.55 on baricitinib and 0.23 on methotrexate, with a greater degree of uncertainty for baricitinib. Conclusion Experts currently think that baricitinib is superior to MTX for the treatment of JDM, although there is uncertainty around this. BARJDM will therefore integrate randomised trial data with this expert prior opinion to derive a posterior distribution for the relative efficacy of baricitinib compared with MTX.

中文翻译：

---

征求专家事先意见来设计青少年皮肌炎的 BARJDM 试验


：目的 就青少年皮肌炎（JDM）这一罕见炎症性疾病的两种治疗方法的相对优点，征求并量化专家意见。本文报告的专家意见的正式表达将用于即将进行的名为 BARJDM（巴瑞克替尼治疗青少年皮肌炎）的随机对照试验的贝叶斯分析。方法 按照先前描述的方法模板，召开了贝叶斯事先启发会议。就 BARJDM 试验中的患者在停用糖皮质激素 (GC) 后 12 个月内使用甲氨蝶呤（标准护理）或使用甲氨蝶呤（标准护理）后达到临床非活动性疾病的可能性征求了意见。或 baricitinib（一种 Janus 激酶抑制剂，JAKi），试验的两个组的 GC 时间表相同。在介绍和进一步讨论有关甲氨蝶呤或 JAKi 对 JDM 疗效的已发表数据汇总后，专家们的观点得到了讨论和完善。结果 十名英国儿科风湿病学顾问（包括一名青少年儿科风湿病学家）参加了启发会议。所有人都拥有 JDM 方面的专业知识，领导活跃的国家卫生服务诊所治疗这种疾病。专家先前的共识意见是，巴瑞替尼治疗后 12 个月内出现临床非活动性疾病的最可能概率为 0.55，甲氨蝶呤治疗为 0.23，巴瑞替尼治疗的不确定性更大。结论 目前专家认为巴瑞克替尼治疗 JDM 优于 MTX，尽管这一点还存在不确定性。因此，BARJDM 将把随机试验数据与专家先前的意见相结合，得出巴瑞克替尼与 MTX 相比相对疗效的后验分布。