Patient-centered Outcomes Research in Interstitial Lung Disease: An Official American Thoracic Society Research Statement

Aronson, Kerri I.; Danoff, Sonye K.; Russell, Anne-Marie; Ryerson, Christopher J.; Suzuki, Atsushi; Wijsenbeek, Marlies; Bianchi, Pauline; Corte, Tamera J.; Lee, Joyce; Lindell, Kathleen O.; Maher, Toby M.; Martínez, Fernando J.; Meek, Paula; Raghu, Ganesh; Rouland, Glenda; Rudell, Rick; Safford, Monika M.; Sheth, Jamie; Swigris, Jeffrey J.

doi:10.1164/rccm.202105-1193st

Cited by 53 publications

(42 citation statements)

References 226 publications

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“…Several validated questionnaires are available to assess HRQoL in patients with IPF, such as an IPF-specific version of the St George's Respiratory Questionnaire (SGRQ-I), K-BILD and Living with IPF [ 29 ], although less is known for progressive fibrosing ILDs. K-BILD and, recently, the Living with Pulmonary Fibrosis questionnaire are potential tools to assess HRQoL in these patients [ 8 , 30 ].…”

Section: Discussionmentioning

confidence: 99%

The psychometric properties of the King's Brief Interstitial Lung Disease questionnaire and thresholds for meaningful treatment response in patients with progressive fibrosing interstitial lung diseases

Birring

Bushnell²,

Baldwin

et al. 2021

Eur Respir J

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There is a lack of fully validated patient-reported outcome measures for progressive fibrosing interstitial lung disease (PF-ILD). We aimed to validate the King's Brief Interstitial Lung Disease questionnaire (KBILD) for measuring health-related quality of life (HRQoL) in these patients. We also estimated the meaningful change threshold for interpreting stabilisation of HRQoL as a clinical endpoint in PF-ILD, where the current goal of treatment is disease stability and slowing progression. This analysis evaluated data from 663 patients with PF-ILD other than idiopathic pulmonary fibrosis from the INBUILD trial. Validation of the measurement properties was assessed for internal consistency, test-retest reliability, construct validity, known-groups validity, and responsiveness. We calculated meaningful change thresholds for treatment response using anchor-based (within-patient) and distribution-based methods. The KBILD had strong internal consistency (Cronbach alpha was 0.94 for total score, 0.88 for KBILD domain breathlessness and activities, 0.91 for psychological and 0.79 for chest symptoms). Test-retest reliability intraclass correlation coefficient was 0.74 for KBILD total score. The KBILD demonstrated weak correlations with forced vital capacity (FVC)% predicted. Known-groups validity showed significant differences in KBILD scores for patient groups with different disease severity based on use of supplemental oxygen or baseline FVC% predicted (<70 or >70%). We estimated a meaningful change threshold of ≥–2 for the KBILD total score for defining patients who remain stable/improved versus those with progressive deterioration. Our results validate the KBILD as a tool for assessing HRQoL in patients with PF-ILD and set a meaningful change threshold of ≥–2 for KBILD total score.

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Section: Discussionmentioning

confidence: 99%

The psychometric properties of the King's Brief Interstitial Lung Disease questionnaire and thresholds for meaningful treatment response in patients with progressive fibrosing interstitial lung diseases

Birring

Bushnell²,

Baldwin

et al. 2021

Eur Respir J

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“…These strategies center around the need to address mistrust and misconception, promote increased information and awareness of the benefits of trial participation, and ensure that investigators and study personnel are fully trained in cultural and racial competencies (108,112). Ensuring feasibility studies are undertaken prior to commencement of large trials and involving patients in the design stages of trials will also help to address several of these challenges (113). Global approach to treatment of sarcoidosis.…”

Section: Challenges Affecting Drug Trials and Drug Development In Sar...mentioning

confidence: 99%

Sarcoidosis: Updates on therapeutic drug trials and novel treatment approaches

et al. 2022

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Sarcoidosis is a systemic granulomatous inflammatory disease of unknown etiology. It affects the lungs in over 90% of patients yet extra-pulmonary and multi-organ involvement is common. Spontaneous remission of disease occurs commonly, nonetheless, over 50% of patients will require treatment and up to 30% of patients will develop a chronic progressive non-remitting disease with marked pulmonary fibrosis leading to significant morbidity and death. Guidelines outlining an immunosuppressive treatment approach to sarcoidosis were recently published, however, the strength of evidence behind many of the guideline recommended drugs is weak. None of the drugs currently used for the treatment of sarcoidosis have been rigorously studied and prescription of these drugs is often based on off-label” indications informed by experience with other diseases. Indeed, only two medications [prednisone and repository corticotropin (RCI) injection] currently used in the treatment of sarcoidosis are approved by the United States Food and Drug Administration. This situation results in significant reimbursement challenges especially for the more advanced (and often more effective) drugs that are favored for severe and refractory forms of disease causing an over-reliance on corticosteroids known to be associated with significant dose and duration dependent toxicities. This past decade has seen a renewed interest in developing new drugs and exploring novel therapeutic pathways for the treatment of sarcoidosis. Several of these trials are active randomized controlled trials (RCTs) designed to recruit relatively large numbers of patients with a goal to determine the safety, efficacy, and tolerability of these new molecules and therapeutic approaches. While it is an exciting time, it is also necessary to exercise caution. Resources including research dollars and most importantly, patient populations available for trials are limited and thus necessitate that several of the challenges facing drug trials and drug development in sarcoidosis are addressed. This will ensure that currently available resources are judiciously utilized. Our paper reviews the ongoing and anticipated drug trials in sarcoidosis and addresses the challenges facing these and future trials. We also review several recently completed trials and draw lessons that should be applied in future.

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“…Several patient-reported outcome measures (PROMs) have been adapted for use in ILDs. The PROMs utilized in ILD research and practice are mainly categorized into three groups: (1) disease-specific HRQOL, (2) generic HRQOL, and (3) domainspecific instruments (69). These instruments are ideally chosen as endpoints in research according to the objective of the study and characteristics of the study population.…”

Section: Cross Cultural Adaptation In Ildmentioning

confidence: 99%

Health Related Quality of Life in Interstitial Lung Disease: Can We Use the Same Concepts Around the World?

Aronson

Suzuki

2021

Front. Med.

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Health-Related Quality of Life (HRQOL) is increasingly viewed as an important patient-centered outcome by leading health organizations, clinicians, and patients alike. This is especially true in the interstitial lung disease community where patients often struggle with progressive and debilitating disease with few therapeutic options. In order to test the effectiveness of new pharmacologic therapies and non-pharmacologic interventions globally in ILD, this will require expansion of clinical research studies to a multinational level and HRQOL will be an important endpoint to many. In order to successfully expand trials across multiple nations and compare the results of studies between different communities we must recognize that there are differences in the concepts of HRQOL across the world and have strategies to address these differences. In this review, we will describe the different global influences on HRQOL both generally and in the context of ILD, discuss the processes of linguistic translation and cross-cultural adaptation of HRQOL Patient Reported Outcome Measures (PROMs), and highlight the gaps and opportunities for improving HRQOL measurement in ILD across the world.

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Patient-centered Outcomes Research in Interstitial Lung Disease: An Official American Thoracic Society Research Statement

Cited by 53 publications

References 226 publications

The psychometric properties of the King's Brief Interstitial Lung Disease questionnaire and thresholds for meaningful treatment response in patients with progressive fibrosing interstitial lung diseases

The psychometric properties of the King's Brief Interstitial Lung Disease questionnaire and thresholds for meaningful treatment response in patients with progressive fibrosing interstitial lung diseases

Sarcoidosis: Updates on therapeutic drug trials and novel treatment approaches

Health Related Quality of Life in Interstitial Lung Disease: Can We Use the Same Concepts Around the World?

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