Abstract：
Guidelines for mycosis fungoides and Sézary syndrome clinical trials were published in 2011 to standardize endpoint criteria and trial design. Our retrospective cohort study of mycosis fungoides/Sézary syndrome clinical trials registered on ClinicalTrials.gov and pivotal trials supporting drug approvals and label extensions evaluates adherence to these guidelines. Sixty-three trials met our inclusion criteria. In a subpopulation of trials, mean adherence to the guidelines was approximately 60%. When comparing trials that began in the first 6 years after their publication with those that started after, we found no difference in mean adherence (4.12 vs 3.41) (P = .15). Among the 8 pivotal trials supporting new mycosis fungoides or Sézary syndrome systemic therapies from 1990 to 2020, systemic trials published after 2011 were more likely to randomize patients (100 vs 0%, P = .036), perform superiority testing (100 vs 0%, P = .036), and use an intention-to-treat analysis (100 vs 0%, P = .036). The design of trials registered on Clinicaltrials.gov did not change significantly between the first 6 years after the publication of the guidelines and after. This demonstrates that the guidelines are still not consistently implemented across all trials. However, registrational trials were more likely to implement the recommendations.
摘要：
2011年发布了关于霉菌病（MF）和Sézary综合征（SS）临床试验的指南，以标准化终点标准和试验设计。我们在ClinicalTrials.gov上注册的MF/SS临床试验的回顾性队列研究以及支持药物批准和标签扩展的关键试验评估了对这些指南的依从性。63项试验符合我们的纳入标准。在试验的亚群中，对指南的平均依从性约为60%.当比较在其发表后的前六年开始的试验与之后开始的试验时，我们发现平均依从性没有差异(4.12vs.3.41)(P=0.15)。在1990-2020年支持新的MF或SS系统疗法的8项关键试验中，2011年后发表的系统试验更有可能随机分组患者（100％与0%,P=0.036),执行优势测试(100%与0%,P=0.036),并使用意向治疗分析(100%与0%,P=0.036)。在Clinicaltrials.gov上注册的试验设计在指南发布后的前六年与之后没有显着变化。这表明指南在所有试验中仍未得到一致实施。然而,注册试验更有可能实施建议.
