Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

Abstract

Patient-reported outcome measures (PROMs), tools to assess patient self-report of health status, are now increasingly used in research, care and policymaking. While there are two well-developed disease-specific PROMs for interstitial lung diseases (ILD) and idiopathic pulmonary fibrosis (IPF), many unmet and urgent needs remain. In December 2019, 64 international ILD experts convened in Erice, Italy to deliberate on many topics, including PROMs in ILD. This review summarises the history of PROMs in ILD, shortcomings of the existing tools, challenges of development, validation and implementation of their use in clinical trials, and the discussion held during the meeting. Development of disease-specific PROMs for ILD including IPF with robust methodology and validation in concordance with guidance from regulatory authorities have increased user confidence in PROMs. Minimal clinically important difference for bidirectional changes may need to be developed. Cross-cultural validation and linguistic adaptations are necessary in addition to robust psychometric properties for effective PROM use in multinational clinical trials. PROM burden of use should be reduced through appropriate use of digital technologies and computerised adaptive testing. Active patient engagement in all stages from development, testing, choosing and implementation of PROMs can help improve probability of success and further growth.

The Burden of Progressive-Fibrosing Interstitial Lung Diseases

“…Currently, there are also an absence of dedicated PROMs and PREMs in this field (9). Whilst tools such as the L-PF have demonstrated applicability and sensitivity to change in PF-ILD in the context of the INBUILD trial, and are expected to be used to measure HRQoL in PF-ILD in future (9,32), current tools have their limitations in measuring preservation of QoL in this chronic disease (9,55) Consequently, further work is needed to develop PROMs/PREMs specific to this disease.…”

Responsiveness and meaningful change thresholds of the Living with Pulmonary Fibrosis (L-PF) questionnaire Dyspnoea and Cough scores in patients with progressive fibrosing interstitial lung diseases

“…There is no consensus on the best approach to estimating meaningful change thresholds for patientreported outcomes. 13 18 Food and Drug Administration guidance recommends that anchor-based approaches incorporate 'patient ratings' of change 12 ; however, such transition items, which require patients to assess their current state, recall their prior state and mentally subtract the difference (eg, "Is your shortness of breath a lot better/the same/a lot worse? "), are fraught with problems.…”

“…There is no consensus on the best approach to estimating meaningful change thresholds for patient-reported outcomes. 13 18 Food and Drug Administration guidance recommends that anchor-based approaches incorporate ‘patient ratings’ of change 12 ; however, such transition items, which require patients to assess their current state, recall their prior state and mentally subtract the difference (eg, “Is your shortness of breath a lot better/the same/a lot worse?”), are fraught with problems. Ideally, the correlation between the transition item and baseline score is equal and opposite to the correlation between the transition item and the score at follow-up, but with recall periods of longer than 4 weeks, transition ratings tend to be (inappropriately) highly correlated with the patient’s current state.…”

“…10 The responsiveness of a patient-reported outcome, that is, its capacity to detect change in the target construct, as determined by clinically relevant outcomes or patients' perceptions (anchors) is an important aspect of its validation. [11][12][13] In this study, we evaluated the responsiveness of the L-PF Dyspnoea and Cough domain scores and estimated meaningful change thresholds (minimal clinically important differences) in these scores in patients with progressive fibrosing ILDs other than IPF.…”

Patient-reported outcomes measures (PROMs) and patient-reported experience measures (PREMs) of COVID-19 telerehabilitation: Prospective pilot program

“…[10,11] Therefore, the use of Patient-Reported Outcomes Measures (PROMs) [11] and Patient-Reported Experience Measures (PREMs) [10] are recommended to assess the patient's perspective after a medical action. PROMs [11] are tools to assess the patient's perception of the impact of health care intervention on their condition (e.g., health-related quality of life or dyspnea scale), while PREMs [10] assess the patient's perception of how the health care is received (e.g., Am I satisfied with the follow-up?). [10,11] Therefore, a prospective pilot study to evaluate the feasibility and patient outcomes of the COVID-19 telerehabilitation program using a mobile application was carried out.…”