Le Reun Corinne, Yasmeen Najeeda, Cullen Alexis E, Sawyer Laura, Ostrovskaya Olga, Barion Francesca
Symmetron Limited, Devonshire Square, London, EC2M 4PL, UK.
argenx, Boston, USA.
Adv Ther. 2025 Apr;42(4):1642-1691. doi: 10.1007/s12325-025-03118-6. Epub 2025 Feb 28.
Pemphigus diseases are a family of chronic, autoimmune, blistering skin conditions. Despite advances in treatment approaches, more effective and safer therapies for pemphigus are urgently needed. Trials investigating novel therapeutics must be designed to yield evidence that can be compared to existing data, necessitating a comprehensive understanding of the clinical trial landscape. We aimed to perform a mapping review to assess the comparability of randomized controlled trials (RCTs) evaluating existing treatments for pemphigus vulgaris (PV) and pemphigus foliaceous (PF).
Embase, MEDLINE, and Cochrane Library were systematically searched from inception to July 2023, supplemented with conference abstracts, clinical trial registries, and grey literature searches, for RCTs evaluating pharmacotherapies in adults with moderate-to-severe PV or PF. Comparability of study populations (demographic and clinical characteristics), interventions and comparators (dose, administration route, regimen), and outcomes (definition, time point, measure) across trials was assessed.
Fifteen RCTs were eligible for inclusion. Substantial heterogeneity was observed in participant age, sex, and disease duration at baseline, and none of the studies used the same criteria to assess illness severity. Doses and regimens differed across trials assessing the same interventions. Across 16 outcome measures extracted, clinical remission outcomes had limited comparability across studies and were often not defined according to published guidelines. Cumulative corticosteroid dose during the study period had the highest comparability. Health-related quality of life data and serious adverse events were infrequently reported.
The lack of comparability across studies has major implications for developers of new treatments for PV and PF and for decision-makers who must evaluate the efficacy, safety, and cost-effectiveness of these treatments relative to existing therapeutics.
天疱疮疾病是一类慢性自身免疫性水疱性皮肤病。尽管治疗方法取得了进展，但仍迫切需要更有效、更安全的天疱疮治疗方法。研究新型疗法的试验必须设计得能够产生可与现有数据进行比较的证据，这就需要全面了解临床试验情况。我们旨在进行一项图谱综述，以评估评估寻常型天疱疮（PV）和落叶型天疱疮（PF）现有治疗方法的随机对照试验（RCT）的可比性。
从创刊至2023年7月，系统检索了Embase、MEDLINE和Cochrane图书馆，并补充了会议摘要、临床试验注册库和灰色文献检索，以查找评估中重度PV或PF成人药物治疗的RCT。评估了各试验之间研究人群（人口统计学和临床特征）、干预措施和对照（剂量、给药途径、方案）以及结局（定义、时间点、测量方法）的可比性。
15项RCT符合纳入标准。在基线时观察到参与者年龄、性别和疾病持续时间存在显著异质性，且没有一项研究使用相同的标准来评估疾病严重程度。评估相同干预措施的试验中，剂量和方案各不相同。在提取的16项结局指标中，临床缓解结局在各研究之间的可比性有限，且往往未根据已发表的指南进行定义。研究期间的累积皮质类固醇剂量可比性最高。很少报告与健康相关的生活质量数据和严重不良事件。
研究之间缺乏可比性对PV和PF新治疗方法的开发者以及必须评估这些治疗方法相对于现有疗法的疗效、安全性和成本效益的决策者具有重大影响。
