Patient-reported outcome measures in systemic sclerosis-related interstitial lung disease for clinical practice and clinical trials

被引:17
|
作者
Saketkoo, Lesley Ann [1 ,2 ,3 ,4 ]
Scholand, Mary Beth [5 ]
Lammi, Matthew R. [1 ,2 ,3 ]
Russell, Anne-Marie [6 ,7 ]
机构
[1] New Orleans Scleroderma & Sarcoidosis Patient Car, New Orleans, LA USA
[2] Univ Med Ctr, Comprehens Pulm Hypertens Ctr, Interstitial Lung Dis Clin Programs, New Orleans, LA USA
[3] Louisiana State Univ, Sch Med, Div Pulm Dis, New Orleans, LA USA
[4] Tulane Univ, Sch Med, 1430 Tulane Ave, New Orleans, LA 70112 USA
[5] Univ Utah, Div Pulm Med, Salt Lake City, UT USA
[6] Imperial Coll London, Natl Heart & Lung Inst, London, England
[7] Imperial Coll Healthcare NHS Trust, London, England
基金
美国国家卫生研究院;
关键词
Systemic sclerosis; scleroderma; pulmonary fibrosis; interstitial lung disease; health-related quality of life; dyspnea; cough; patient-reported; outcomes; QUALITY-OF-LIFE; STEM-CELL TRANSPLANTATION; IDIOPATHIC PULMONARY-FIBROSIS; SCLERODERMA LUNG; OPEN-LABEL; MYCOPHENOLATE-MOFETIL; HEALTH-CARE; PULSE CYCLOPHOSPHAMIDE; IMATINIB MESYLATE; RESPONSE CRITERIA;
D O I
10.1177/2397198320904178
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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.
引用
收藏
页码:48 / 60
页数:13
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