Humbert Marc, Singh Manjit, Furst Daniel E, Khanna Dinesh, Seibold James R
Faculté de Médecine, Université Paris-Sud.
Service de Pneumologie, DHU Thorax Innovation, AP-HP, Hôpital Bicêtre, Le Kremlin-Bicêtre.
Rheumatology (Oxford). 2017 Sep 1;56(suppl_5):v33-v37. doi: 10.1093/rheumatology/kex197.
There are proven successful approaches to clinical trial design in pulmonary arterial hypertension (PAH), which in turn have led to the licensing of a number of effective therapies. SSc has been included in trials of World Health Organization Group 1 PAH but has been under-represented. Responses in outcomes as diverse as exercise capacity, quality of life, durability of drug effect and survival have been reduced in comparison with those seen in idiopathic PAH. The PAH community has achieved international and interdisciplinary consensus guidelines for future studies. We consider the diverse outcome measures used in trials in the context of the complexities of scleroderma. An argument is advanced in favour of future trials focused exclusively on SSc but with adaptations of the core outcome measures and trial design templates applicable to more general studies of PAH.
在肺动脉高压（PAH）的临床试验设计中，已有经证实的成功方法，这些方法进而促成了多种有效疗法的获批。系统性硬化症（SSc）已被纳入世界卫生组织第1组PAH的试验，但所占比例较低。与特发性PAH相比，在运动能力、生活质量、药物疗效持久性和生存率等各种结果方面的反应有所降低。PAH领域已达成关于未来研究的国际和跨学科共识指南。我们在硬皮病复杂性的背景下考虑试验中使用的各种结果指标。有人提出支持未来专门针对SSc的试验，但要对核心结果指标和试验设计模板进行调整，使其适用于PAH更一般的研究。
