Snyder Corey L, Gibson Ruby S, Chen Stella X, Porter Martina L, Kimball Alexa B
Department of Dermatology, Beth Israel Deaconess Medical Center, Clinical Laboratory for Epidemiology and Applied Research in Skin (CLEARS), Boston, Massachusetts.
Department of Dermatology, University of Texas Southwestern Medical School, Dallas, Texas.
Int J Womens Dermatol. 2024 Jun 7;10(2):e152. doi: 10.1097/JW9.0000000000000152. eCollection 2024 Jun.
In some hidradenitis suppurativa (HS) clinical trial study arms, there is an unexpected decline in efficacy between the penultimate visit and the prespecified primary endpoint week, which we have termed a "wobble."
We aimed to establish how often study arms in HS programs wobble.
In a retrospective review, we identified HS clinical trials listed on ClinicalTrials.gov testing systemic, nonantibiotic medications that utilized Hidradenitis Suppurativa Clinical Response (HiSCR) as an outcome measure. We identified study arms demonstrating greater improvement in a visit prior to the primary endpoint week. Baseline subject characteristics were compared between studies with HiSCR wobble and no HiSCR wobble.
A total of 21 studies (randomized control trial [RCT], = 14; open-label, = 7) with 35 study drug arms (RCT, = 27; open-label, = 8) and 14 placebo arms were identified. HiSCR wobble occurred significantly more often in RCT compared to open-label study drug arms (11/27 [40.7%] vs 0/8 [0%]). In RCT study arms with HiSCR wobble, baseline draining fistula counts were significantly lower (2.3 vs 3.2), and numerically fewer Hurley stage 3 patients (33.2% vs 42.5%), lower weighted total abscess and nodule counts (12.1 vs 12.6), lower weighted dermatology life quality index scores (12.5 vs 14.5), and a higher proportion of female patients (63.9% vs 58.3%) were observed.
Include low number of HS clinical trials and insufficient data reported in many studies to assess for wobble, degree of wobble, and to compare all baseline characteristics.
Nonlinear improvement in study arm response occurs in some HS RCTs. Potential contributing factors include a higher proportion of less severe patients at baseline and more female patients.
在一些化脓性汗腺炎（HS）临床试验研究组中，在倒数第二次访视和预先设定的主要终点周之间疗效出现意外下降，我们将其称为“波动”。
我们旨在确定HS项目研究组中出现波动的频率。
在一项回顾性研究中，我们在ClinicalTrials.gov上识别了测试全身性非抗生素药物且将化脓性汗腺炎临床反应（HiSCR）作为结局指标的HS临床试验。我们确定了在主要终点周之前的一次访视中显示出更大改善的研究组。比较了出现HiSCR波动和未出现HiSCR波动的研究之间的基线受试者特征。
共识别出21项研究（随机对照试验[RCT]，n = 14；开放标签，n = 7），有35个研究药物组（RCT，n = 27；开放标签，n = 8）和14个安慰剂组。与开放标签研究药物组相比，RCT中HiSCR波动出现的频率显著更高（11/27 [40.7%]对0/8 [0%]）。在出现HiSCR波动的RCT研究组中，基线引流瘘管计数显著更低（2.3对3.2），Hurley 3期患者在数量上更少（33.2%对42.5%），加权总脓肿和结节计数更低（12.1对12.6），加权皮肤病生活质量指数评分更低（12.5对14.5），并且女性患者比例更高（63.9%对58.3%）。
包括HS临床试验数量少，许多研究报告的数据不足，无法评估波动情况、波动程度以及比较所有基线特征。
一些HS RCT中研究组反应存在非线性改善。潜在的促成因素包括基线时病情较轻患者比例较高以及女性患者较多。
