Quantifying the natural variation in lesion counts over time in untreated hidradenitis suppurativa: Implications for outcome measures and trial design.
Quantifying the natural variation in lesion counts over time in untreated hidradenitis suppurativa: Implications for outcome measures and trial design.
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DOI：
10.1016/j.jdin.2020.09.005
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发表时间：
2020-12
影响因子：
--
通讯作者：
Krueger JG
中科院分区：
文献类型：
--
作者：
Frew JW;Jiang CS;Singh N;Navrazhina K;Vaughan R;Krueger JG
化脓性汗腺炎（HS）在临床试验中表现出较高的安慰剂反应率，这可能是由于该疾病的自然变异性。尚未对未经治疗的疾病的病变计数的变异性进行量化。量化未经处理的 HS 的变异性。对来自 PIONEER 研究安慰剂组的个体患者数据进行了分析，并检查了受试者内变异系数的测量结果。变异性按疾病相关变量（Hurley 分期、BMI、性别、吸烟、家族史）和身体部位进行分层。对受试者内变异系数的分析表明，在 PIONEER I 和 II 中，一半参与者的中间分布（受试者脓肿和结节计数的 75% 和 25% 百分位之间的差异）大于其中位脓肿和结节计数的 33% 和 40%，并且 25% 的受试者的中间分布大于其中位脓肿和结节计数的 70% 和 78%，分别。 Hurley 2 期参与者的受试者内变异明显大于 Hurley 3 期患者。腋窝和腹股沟区域的变化比其他解剖位置更大。局限性包括使用预先收集的临床试验数据。未经治疗的 HS 中病变计数的受试者内部变异性比之前估计的要大。这对 HS 的结果测量和未来临床试验的进行具有深远的影响。
Hidradenitis suppurativa (HS) demonstrates high placebo response rates in clinical trials, possibly due to the natural variability of the disease. No quantification of variability in lesion counts of untreated disease has been undertaken. To quantify the variability of untreated HS. Deidentified individual patient data from the placebo arms of PIONEER studies were analyzed, and measurements of within-subject coefficients of variation were examined. Variability was stratified by disease-associated variables (Hurley stage, BMI, sex, smoking, family history) and body site. Analysis of within-subject coefficients of variation demonstrated that half of the participants had a middle spread [difference between 75th and 25th percentiles of the subject's abscess and nodule counts] greater than 33% and 40% of their median abscess and nodule counts, and 25% of the subjects had a middle spread greater than 70% and 78% of their median abscess and nodule counts in PIONEER I and II, respectively. Hurley stage 2 participants had significantly greater within-subject variation than Hurley stage 3 patients. Variation was greater in the axillary and groin regions than in other anatomical locations. Limitations include the use of precollected clinical trial data. The within-subject variability of the lesion counts in untreated HS was greater than previously appreciated. This has profound effects on outcome measures and the conduct of future clinical trials of HS.