New insights on patient-reported outcome measures in idiopathic pulmonary fibrosis: only PROMises?

Abstract

In a chronic, progressive and ultimately fatal disease like idiopathic pulmonary fibrosis (IPF), the maintenance of patients' quality of life should be regarded as a major aim of treatment. Although better knowledge and two antifibrotic drugs are now available in IPF, the individual response to treatment and its acceptance remain poorly explored. This review summarizes recent advances in research on patient-reported outcomes and their measures, indispensable instruments to investigate how patients feel and function, and how the disease impacts their lives. In IPF, there is a paucity of specific well-validated patient-reported outcome measures (PROMs). The use of generic PROMs in past IPF trials revealed a poor correlation of such questionnaires with established endpoints of treatment response. Several attempts are currently ongoing to develop specific IPF PROMs. The King's Brief Interstitial Lung Disease health status questionnaire and the Tool to Assess Quality of Life in IPF are promising questionnaires developed by using institutional recommendations and are currently being validated in large cohorts. Well-validated relevant PROMs can be employed for multiple purposes: as outcome measures for daily care or for driving therapeutic decisions, as efficacy endpoints in clinical trials, or as tools to collect useful data for healthcare policy makers in order to improve access and quality of care.