Prospective validation of the provisional criteria for the evaluation of response to therapy in childhood-onset systemic lupus erythematosus.
Prospective validation of the provisional criteria for the evaluation of response to therapy in childhood-onset systemic lupus erythematosus.
复制标题
儿童期发病的系统性红斑狼疮治疗反应评估临时标准的前瞻性验证。
DOI：
10.1002/acr.20103
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发表时间：
2010
影响因子：
4.7
通讯作者：
Giannini,EdwardH
中科院分区：
文献类型：
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作者：
Brunner,HermineI;Higgins,GloriaC;Wiers,Kristina;Lapidus,SiviaK;Olson,JudyannC;Onel,Karen;Punaro,Marilynn;Ying,Jun;Klein-Gitelman,MarisaS;Giannini,EdwardH
目的对系统性红斑狼疮（SLE）患儿治疗反应评价的临时标准进行前瞻性验证。方法在这项多中心研究中，儿童发病SLE患者（n = 98； 81名女孩，17名男孩，50%白人，88%非西班牙裔）每3个月随访7次（总访问量623次）。在每次访问时获得5个儿童期发病SLE核心反应变量：1)医生对总体疾病活动性的评估，2)家长对患者健康状况的评估，3)儿童健康问卷，4)蛋白尿，5)全球疾病活动性测量评分，由欧洲共识狼疮活动性测量（ECLAM）、系统性红斑狼疮疾病活动性指数（SLEDAI）或系统性狼疮活动性测量（SLAM）测量。医生评价的两次就诊之间病程的相关变化（临床相关改善、疾病无变化或恶化）作为标准。混合模型用于评估4个最高级别的治疗反应临时定义的诊断准确性。结果2次连续访视期间临床相关好转89次，无好转448次。无论选择何种全球疾病活动性指标（ECLAM、SLAM、SLEDAI），所有4个排名最高的定义的敏感性都很低（均≤31%），而它们的特异性都很好（均低于88%）。使用逻辑模型，可以开发具有80%敏感性和特异性的替代定义。结论儿童期起病SLE治疗反应的临时标准可能比以前报道的敏感性低得多。需要在临床试验中进一步验证，以进一步评估儿科风湿病国际试验组织/美国风湿病学会关于SLE儿童治疗反应的临时标准的测量特性。
ObjectiveTo prospectively validate the provisional criteria for the evaluation of response to therapy in children with systemic lupus erythematosus (SLE).MethodsIn this multicenter study, childhood‐onset SLE patients (n = 98; 81 girls, 17 boys, 50% white, 88% non‐Hispanic) were followed every 3 months for up to 7 visits (total number of visits 623). The 5 childhood‐onset SLE core response variables were obtained at the time of each visit: 1) physician assessment of overall disease activity, 2) parent assessment of patient well‐being, 3) Child Health Questionnaire, 4) proteinuria, and 5) global disease activity measure score, as measured by the European Consensus Lupus Activity Measure (ECLAM), the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI), or the Systemic Lupus Activity Measure (SLAM). Physician‐rated relevant changes in the disease course (clinically relevant improvement, no change in disease, or worsening) between visits served as the criterion standard. Mixed models were used to assess the diagnostic accuracy of the 4 highest‐ranked provisional definitions of response to therapy.ResultsThere were 89 episodes of clinically relevant improvement between 2 consecutive visits, and 448 episodes without improvement. Irrespective of the choice of the global disease activity measure (ECLAM, SLAM, SLEDAI), sensitivities of all 4 highest‐ranked definitions were low (all ≤31%), whereas their specificities were excellent (all >88%). Using logistic models, alternative definitions can be developed with both 80% sensitivity and specificity.ConclusionThe provisional criteria of response to therapy in childhood‐onset SLE may have considerably lower sensitivity than previously reported. Additional validation in clinical trials is necessary to further evaluate the measurement properties of the provisional Paediatric Rheumatology International Trials Organisation/American College of Rheumatology criteria for response to therapy in children with SLE.