Department of Dermatology, Ghent University Hospital, Ghent, Belgium.
JAMA Dermatol. 2022 Nov 1;158(11):1304-1314. doi: 10.1001/jamadermatol.2022.3511.
The minimally important difference (MID) represents the point at which a difference in an outcome measure (eg, Dermatology Life Quality Index) is important enough that it warrants a change in treatment, and, to the authors' knowledge, the robustness and limitations of MIDs have not been thoroughly evaluated in skin diseases. The MID is increasingly used in clinical trials to demonstrate that an intervention is worthwhile for patients; furthermore, MIDs also contribute to sample size calculations in clinical trials, influence treatment guidelines, and can guide clinicians to modify treatment.
To evaluate the credibility and generalization of MIDs for patient-reported outcome measures (PROMs) in skin disorders.
A systematic search was conducted in PubMed and Embase for all original articles using the MID concept for skin disorders from inception to December 29, 2021. The credibility of MIDs obtained via an anchor-based approach (eg, global rating of change scale) was assessed with a previously developed credibility instrument. The validity of generalizing established MIDs to other patient groups was evaluated based on the diagnosis and the patient characteristics.
A total of 126 articles were selected, and 84 different MIDs were identified for PROMs. A total of 13 of 84 MIDs (15.5%) for PROMs displayed acceptable credibility. The anchors used had varying capacity to assess minimal important changes from a patient's perspective and were deemed inappropriate for this purpose in 52 of 84 cases (61.9%). Correlations between the anchors and PROMs were frequently not determined (39 of 84; 46.4%). The time interval for anchor questions assessing a change in the experienced disease burden was not optimal for 10 of 32 transition anchors (>3 months), introducing potential recall bias. Previously reported MIDs were widely used to examine relevant changes in other study populations. However, the diagnosis and disease severity were different from the original MID population in 39 of 70 (55.7%) and 45 of 70 (64.3%) cases, respectively.
In this scoping review, only a minority of MIDs for PROMs demonstrated sufficient credibility in dermatology. Inappropriate generalization of previously reported MIDs to patient populations with different disease characteristics was found to be a major concern. Furthermore, the study supported the use of multiple anchors and encouraged consistent reporting of the correlation between changes in the anchor and changes in the outcome measures.
最小有意义差异（MID）代表了结果测量（例如皮肤病生活质量指数）中的差异重要到足以改变治疗的程度，据作者所知，MID 的稳健性和局限性尚未在皮肤病中得到彻底评估。MID 越来越多地用于临床试验，以证明干预对患者是有价值的；此外，MID 还为临床试验中的样本量计算提供了依据，影响治疗指南，并指导临床医生修改治疗方案。
评估用于皮肤疾病患者报告结局测量（PROM）的 MID 的可信度和可推广性。
从建立到 2021 年 12 月 29 日，在 PubMed 和 Embase 中进行了系统检索，检索了所有使用 MID 概念的用于皮肤疾病的原始文章。通过锚定方法（例如，变化整体评估量表）获得的 MID 的可信度，使用先前开发的可信度工具进行了评估。根据诊断和患者特征，评估了将已建立的 MID 推广到其他患者群体的有效性。
共选择了 126 篇文章，确定了 84 种用于 PROM 的不同 MID。84 个 PROM 中的 13 个（15.5%）MID 显示出可接受的可信度。用于评估患者视角下最小重要变化的锚定具有不同的能力，其中 52 个案例（61.9%）被认为不适合该目的。在 84 个案例中，有 39 个案例（46.4%）没有确定锚定与 PROM 之间的相关性。评估经历疾病负担变化的锚定问题的时间间隔对于 10 个过渡锚定中的 32 个（>3 个月）并不理想，这引入了潜在的回忆偏差。先前报告的 MID 广泛用于研究其他患者群体中的相关变化。然而，在 70 个案例中，诊断和疾病严重程度分别与原始 MID 人群不同，分别为 39 个（55.7%）和 45 个（64.3%）。
在这项范围界定审查中，只有少数 PROM 的 MID 在皮肤病学中表现出足够的可信度。发现先前报告的 MID 不适当地推广到具有不同疾病特征的患者群体是一个主要问题。此外，该研究支持使用多个锚定，并鼓励一致报告锚定变化与结局测量变化之间的相关性。
