Saketkoo Lesley Ann, Scholand Mary Beth, Lammi Matthew R, Russell Anne-Marie
New Orleans Scleroderma and Sarcoidosis Patient Care and Research Center, New Orleans, LA, USA.
Interstitial Lung Disease Clinic Programs, Comprehensive Pulmonary Hypertension Center, University Medical Center, New Orleans, LA, USA.
J Scleroderma Relat Disord. 2020 Mar;5(2 Suppl):48-60. doi: 10.1177/2397198320904178. Epub 2020 Mar 5.
Systemic sclerosis (SSc) is a progressive vasculopathic, fibrosing autoimmune condition, portending significant mortality; wherein interstitial lung disease (ILD) is the leading cause of death. Although lacking a definitive cure, therapeutics for (SSc-ILD) that stave progression exist with further promising primary and adjuvant compounds in development, as well as interventions to reduce symptom burden and increase quality of life. To date, there has been a significant but varied history related to systemic sclerosis-related interstitial lung disease trial design and endpoint designation. This is especially true of endpoints measuring patient-reported perceptions of efficacy and tolerability. This article describes the underpinnings and complexity of the science, methodology, and current state of patient-reported outcome measures used in (SSc-ILD) systemic sclerosis-related interstitial lung disease in clinical practice and trials.
系统性硬化症（SSc）是一种进行性血管病变、纤维化的自身免疫性疾病，预示着较高的死亡率；其中间质性肺病（ILD）是主要死因。虽然尚无根治方法，但有延缓系统性硬化症相关间质性肺病（SSc-ILD）进展的疗法，还有更多有前景的一线和辅助化合物正在研发中，以及减轻症状负担和提高生活质量的干预措施。迄今为止，与系统性硬化症相关间质性肺病的试验设计和终点设定相关的历史颇为丰富但各不相同。在测量患者报告的疗效和耐受性认知的终点方面尤其如此。本文描述了在临床实践和试验中用于系统性硬化症相关间质性肺病（SSc-ILD）的患者报告结局测量的科学基础、复杂性、方法及现状。
