目的:开发并进行IgG4相关疾病损伤指数(IgG4-RDDI)的初步验证。
方法:由中国IgG4-RD联盟(CIC)的专家制定了评估IgG4-RD患者器官损害的指标草案。初步的DI是用德尔菲法精制的,最终版本是通过协商一致产生的。然后选择了40例代表四种临床情况的IgG4-RD病例,在至少3年的随访中,每个患者有两个评估时间点.来自全国35家医院的48位风湿病学家被邀请使用CICIgG4-RDDI评估器官损伤。使用组内相关系数(ICC)和Kendall-W一致系数(KW)来评估评估者间的可靠性。通过计算评估者的敏感性和特异性来测试IgG4-RDDI的标准有效性。
结果:IgG4-RDDI是由14个器官系统域组成的累积指数,共39个项目。IgG4-RDDI能够区分活动性疾病亚组和稳定疾病亚组的稳定和增加的损伤。就基线得分和所有评估者后来的观察而言,所有评估者在基线和后期观察时的评分总体一致性均令人满意.两个时间点的ICC分别为0.69和0.70,KW分别为0.74和0.73。在亚组分析中,所有亚组的ICC和KW分别大于0.55和0.61。标准效度分析显示出良好的表现,灵敏度为0.86(95%CI0.82至0.88),特异性为0.79(95%CI0.76~0.82),曲线下面积为0.88(95%CI0.85~0.91)。
结论:IgG4-RDDI是分析疾病结局的有用方法,具有良好的可操作性和可信性。预计DI将成为IgG4-RD患者的治疗试验和预后研究的有用工具。
OBJECTIVE: To develop and conduct an initial validation of the Damage Index for IgG4-related disease (IgG4-RD DI).
METHODS: A draft of index items for assessing organ damages in patients with IgG4-RD was generated by experts from the Chinese IgG4-RD Consortium (CIC). The preliminary DI was refined using the Delphi method, and a final version was generated by
consensus. 40 IgG4-RD cases representing four types of clinical scenarios were then selected, each with two time points of assessment for at least 3 years of follow-up. 48 rheumatologists from 35 hospitals nationwide were invited to evaluate organ damage using the CIC IgG4-RD DI. The intraclass correlation coefficient (ICC) and the Kendall-W coefficient of concordance (KW) were used to assess the inter-rater reliability. The criterion validity of IgG4-RD DI was tested by calculating the sensitivity and specificity of raters.
RESULTS: IgG4-RD DI is a cumulative index consisting of 14 domains of organ systems, including a total of 39 items. The IgG4-RD DI was capable of distinguishing stable and increased damage across the active disease subgroup and stable disease subgroup. In terms of scores at baseline and later observations by all raters, overall consistency in scores at baseline and later observations by all raters was satisfactory. ICC at the two time points was 0.69 and 0.70, and the KW was 0.74 and 0.73, respectively. In subgroup analysis, ICC and KW in all subgroups were over 0.55 and 0.61, respectively. The analysis of criterion validity showed a good performance with a sensitivity of 0.86 (95% CI 0.82 to 0.88), a specificity of 0.79 (95% CI 0.76 to 0.82) and an area under the curve of 0.88 (95% CI 0.85 to 0.91).
CONCLUSIONS: The IgG4-RD DI is a useful approach to analyse disease outcomes, and it has good operability and credibility. It is anticipated that the DI will become a useful tool for therapeutic trials and studies of prognosis in patients with IgG4-RD.