Università degli Studi di Genova and Istituto Giannina Gaslini, Genoa, Italy.
Istituto Giannina Gaslini, Genoa, Italy.
Arthritis Rheumatol. 2016 Mar;68(3):566-76. doi: 10.1002/art.39332. Epub 2016 Feb 9.
To develop criteria for the classification of macrophage activation syndrome (MAS) in patients with systemic juvenile idiopathic arthritis (JIA).
A multistep process, based on a combination of expert consensus and analysis of real patient data, was conducted. A panel of 28 experts was first asked to classify 428 patient profiles as having or not having MAS, based on clinical and laboratory features at the time of disease onset. The 428 profiles comprised 161 patients with systemic JIA-associated MAS and 267 patients with a condition that could potentially be confused with MAS (active systemic JIA without evidence of MAS, or systemic infection). Next, the ability of candidate criteria to classify individual patients as having MAS or not having MAS was assessed by evaluating the agreement between the classification yielded using the criteria and the consensus classification of the experts. The final criteria were selected in a consensus conference.
Experts achieved consensus on the classification of 391 of the 428 patient profiles (91.4%). A total of 982 candidate criteria were tested statistically. The 37 best-performing criteria and 8 criteria obtained from the literature were evaluated at the consensus conference. During the conference, 82% consensus among experts was reached on the final MAS classification criteria. In validation analyses, these criteria had a sensitivity of 0.73 and a specificity of 0.99. Agreement between the classification (MAS or not MAS) obtained using the criteria and the original diagnosis made by the treating physician was high (κ = 0.76).
We have developed a set of classification criteria for MAS complicating systemic JIA and provided preliminary evidence of its validity. Use of these criteria will potentially improve understanding of MAS in systemic JIA and enhance efforts to discover effective therapies, by ensuring appropriate patient enrollment in studies.
制定用于系统性幼年特发性关节炎(JIA)患者的巨噬细胞活化综合征(MAS)分类标准。
采用多步骤流程,结合专家共识和真实患者数据分析。首先,由 28 名专家组成的专家组根据发病时的临床和实验室特征,对 428 份患者资料进行 MAS 有或无的分类。这 428 份资料包括 161 例伴有系统性 JIA 相关 MAS 的患者和 267 例可能与 MAS 混淆的患者(有活动系统性 JIA 但无 MAS 证据,或系统性感染)。然后,通过评估使用标准得出的分类与专家共识分类之间的一致性,评估候选标准分类个体患者 MAS 有无的能力。最后在共识会议上选择最终标准。
专家组就 428 份患者资料中的 391 份(91.4%)达成分类共识。对总共 982 项候选标准进行了统计学检验。在共识会议上评估了 37 项表现最佳的标准和 8 项来自文献的标准。会议期间,专家组就最终 MAS 分类标准达成了 82%的共识。在验证分析中,这些标准的敏感性为 0.73,特异性为 0.99。使用标准得出的分类(MAS 或非 MAS)与治疗医生的原始诊断之间具有高度一致性(κ=0.76)。
我们制定了一套用于系统性 JIA 并发 MAS 的分类标准,并提供了其有效性的初步证据。使用这些标准将有可能通过确保在研究中适当纳入患者,提高对系统性 JIA 中 MAS 的认识并加强寻找有效疗法的努力。