Università degli Studi di Genova and Istituto Giannina Gaslini, Genoa, Italy.
Istituto Giannina Gaslini, Genoa, Italy.
Ann Rheum Dis. 2016 Mar;75(3):481-9. doi: 10.1136/annrheumdis-2015-208982.
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.
为了制定系统性幼年特发性关节炎（JIA）患者中巨噬细胞活化综合征（MAS）的分类标准。采用专家共识和真实患者数据分析相结合的多步骤方法。首先，一个由 28 名专家组成的小组根据疾病发作时的临床和实验室特征，将 428 名患者的病例分为有或没有 MAS。这 428 个病例包括 161 名患有系统性 JIA 相关 MAS 的患者和 267 名可能与 MAS 混淆的患者（有活性的系统性 JIA 而无 MAS 证据或全身感染）。然后，通过评估使用标准进行分类与专家共识分类之间的一致性，评估候选标准对个体患者分类为 MAS 或非 MAS 的能力。最终标准在共识会议上进行选择。专家们就 428 个病例中的 391 个病例（91.4%）的分类达成了共识。对总共 982 个候选标准进行了统计学检验。在共识会议上评估了表现最佳的 37 个标准和从文献中获得的 8 个标准。在会议期间，专家们达成了 82%的共识MAS 分类标准。在验证分析中，这些标准的敏感性为 0.73，特异性为 0.99。使用标准进行的分类（MAS 或非 MAS）与治疗医生的原始诊断之间的一致性很高（κ=0.76）。我们已经制定了一套用于诊断系统性 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适当招募患者参加研究，提高对系统性 JIA 中 MAS 的认识，并加强发现有效疗法的努力。
