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血管性水肿控制试验对变化的敏感性及最小临床重要差异

Sensitivity to change and minimal clinically important difference of the angioedema control test.

作者信息

Fijen Lauré M, Vera Carolina, Buttgereit Thomas, Bonnekoh Hanna, Maurer Marcus, Magerl Markus, Weller Karsten

机构信息

Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands.

Institute of Allergology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.

出版信息

Clin Transl Allergy. 2023 Sep;13(9):e12295. doi: 10.1002/clt2.12295.

DOI:10.1002/clt2.12295
PMID:37746798
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10472988/
Abstract

BACKGROUND

The Angioedema Control Test (AECT) is a patient-reported outcome measure developed and validated for the assessment of disease control in patients with recurrent angioedema. Its sensitivity to change and minimal clinically important difference (MCID) have hitherto not been established.

METHODS

Patients with recurrent angioedema due to chronic spontaneous urticaria, hereditary angioedema, or acquired C1-inhibitor deficiency were repeatedly asked to complete the AECT along with the Angioedema Quality of Life Questionnaire (AE-QoL), Dermatology Life Quality Index (DLQI), and anchors for disease control and whether treatment was sufficient during routine care visits. The sensitivity to the change of the AECT was determined by correlating changes in its scores over time with changes in the applied anchors. The MCID was determined using anchor-based and distributional criterion-based approaches.

RESULTS

Eighty-six cases were used for this analysis. Changes in AECT scores correlated well with AE-QoL changes (but less with changes in the DLQI) as well as other applied anchors, demonstrating its sensitivity to change. The MCID was found to be three points for improvement of angioedema control. The available number of cases with meaningful deterioration in our dataset was too low to reach a definite conclusion on the MCID for deterioration of angioedema control.

CONCLUSION

The AECT is a valuable tool to assess changes in disease control in patients with recurrent angioedema over time. The lowest AECT score change that reflects a meaningful improvement of disease control to patients (MCID) is three points.

摘要

背景

血管性水肿控制测试(AECT)是一种由患者报告的结局指标,已开发并验证用于评估复发性血管性水肿患者的疾病控制情况。其对变化的敏感性和最小临床重要差异(MCID)迄今尚未确定。

方法

反复要求因慢性自发性荨麻疹、遗传性血管性水肿或获得性C1抑制剂缺乏而导致复发性血管性水肿的患者在常规护理就诊期间完成AECT,同时完成血管性水肿生活质量问卷(AE-QoL)、皮肤病生活质量指数(DLQI),以及关于疾病控制和治疗是否充分的指标。通过将AECT分数随时间的变化与所应用指标的变化进行关联,来确定AECT对变化的敏感性。使用基于指标和基于分布标准的方法来确定MCID。

结果

86例病例用于该分析。AECT分数的变化与AE-QoL的变化(但与DLQI的变化相关性较小)以及其他应用指标密切相关,表明其对变化具有敏感性。发现血管性水肿控制改善的MCID为3分。在我们的数据集中,血管性水肿控制出现有意义恶化的可用病例数过低,无法就血管性水肿控制恶化的MCID得出明确结论。

结论

AECT是评估复发性血管性水肿患者疾病控制随时间变化的有价值工具。反映对患者疾病控制有意义改善的最低AECT分数变化(MCID)为3分。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/35d9/10472988/8f10fde8631c/CLT2-13-e12295-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/35d9/10472988/3c4817d717c1/CLT2-13-e12295-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/35d9/10472988/8f10fde8631c/CLT2-13-e12295-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/35d9/10472988/3c4817d717c1/CLT2-13-e12295-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/35d9/10472988/8f10fde8631c/CLT2-13-e12295-g002.jpg

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