Greene Adina, Hwang Angelina S, Amjad Sarah, Kechter Jacob A, Mangold Aaron R, Chen Stella X
University of Arizona College of Medicine, Phoenix, Arizona.
Mayo Clinic Department of Dermatology, Scottsdale, Arizona.
Int J Womens Dermatol. 2025 May 16;11(2):e204. doi: 10.1097/JW9.0000000000000204. eCollection 2025 Jun.
Hidradenitis suppurativa (HS) is a chronic, recurrent inflammatory condition and is associated with significant psychosocial impacts on patient quality of life.
This study aimed to characterize the utilization of patient-reported outcomes (PROs) in HS clinical trials and their concordance with trial primary endpoints.
A systematic review of clinical trials was performed using the publicly available U.S. National Library of Medicine (ClinicalTrials.gov) in June 2023 utilizing the search terms hidradenitis, hidradenitis suppurativa, and suppurativa hidradenitis. Study title, start year, trial status, intervention, design, location, and primary and secondary outcomes were collected. To assess for concordance of patient and provider-reported outcomes, we identified published placebo-controlled trials that included Dermatology Life Quality Index (DLQI) and/or a numeric rating scale (NRS) for pain, the 2 most utilized PROs.
One hundred sixty-four HS clinical trials were identified, of which 115 were interventional studies. A total of 65.2% ( = 107) of HS trials included at least one PRO. Pain NRS (42.7%, = 70) and DLQI (42.1%, = 69) were the most frequently used PRO instruments. The use of PROs in clinical trials increased over time, with 50% of trials between 2020 and 2023 utilizing PROs. Of 11 published HS trials, 82% ( = 9) trials showed concordance of provider-assessed and PROs in at least one treatment arm.
Significance and standard deviations of PROs were rarely reported, preventing the calculation of significance and is a limitation of this review.
While lesion counts provide single snapshots of disease, PROs can capture perceptions of lesions not actively present. PROs are frequently concordant with both achievement and nonachievement of primary endpoints in clinical trials.
化脓性汗腺炎（HS）是一种慢性复发性炎症性疾病，对患者生活质量有重大的社会心理影响。
本研究旨在描述HS临床试验中患者报告结局（PROs）的使用情况及其与试验主要终点的一致性。
2023年6月，利用公开可用的美国国立医学图书馆（ClinicalTrials.gov），使用搜索词“汗腺炎”“化脓性汗腺炎”和“化脓性汗腺”对临床试验进行系统评价。收集研究标题、起始年份、试验状态、干预措施、设计、地点以及主要和次要结局。为了评估患者报告结局与提供者报告结局的一致性，我们确定了已发表的安慰剂对照试验，这些试验包括皮肤病生活质量指数（DLQI）和/或疼痛数字评定量表（NRS），这是最常用的两种PROs。
共识别出164项HS临床试验，其中115项为干预性研究。共有65.2%（n = 107）的HS试验纳入了至少一项PRO。疼痛NRS（42.7%，n = 70）和DLQI（42.1%，n = 69）是最常用的PRO工具。随着时间的推移，PROs在临床试验中的使用有所增加，2020年至2023年间有50%的试验使用了PROs。在11项已发表的HS试验中，82%（n = 9）的试验在至少一个治疗组中显示出提供者评估与PROs的一致性。
PROs的显著性和标准差很少报告，无法计算显著性，这是本综述的一个局限性。
虽然病变计数提供了疾病的单一快照，但PROs可以捕捉对未积极出现的病变的感知。PROs在临床试验中通常与主要终点的达成和未达成情况一致。
