Patient-reported Outcomes for Clinical Trials in Idiopathic Pulmonary Fibrosis: New Opportunities to Understand How Patients Feel and Function.
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ABSTRACT:
SUBMITTER: Holland AE
PROVIDER: S-EPMC7737578 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:Patient-reported outcomes (PROs) include questionnaires or surveys that ask patients for their perceptions about things like symptoms they are experiencing or quality of life. For incurable, morbid, life-shortening conditions like idiopathic pulmonary fibrosis (IPF), PROs are particularly germane: They elucidate for clinicians and researchers what it is like for patients to live with such a disease, and they may detect important treatment effects not captured by other metrics (eg, pulmonary physiology). However, a relative paucity of research on PROs in IPF has left significant knowledge gaps in this area and contributed to the timidity investigators have about using PROs as prominent outcomes in IPF drug trials. Additional research on existing instruments is needed to establish or bolster their basic psychometric properties in IPF. When PROs are used as end points in therapeutic trials, analyzing PRO response data can be challenging, but these challenges can be overcome with a transparent, thoughtful, and sophisticated statistical approach. In this article, we discuss some of the basics of PRO assessment, existing knowledge gaps in IPF-related PRO research, and the potential usefulness of using PROs in IPF trials and conclude by offering specific recommendations for an approach to analyzing repeated-measures PRO data from IPF trials.
| S-EPMC3425334 | biostudies-other
Project description:Rationale: Progression of idiopathic pulmonary fibrosis (IPF) is accompanied by worsening of symptoms, exercise capacity, and health-related quality of life. However, the utility of patient-reported outcomes as predictors of mortality remains uncertain.Objectives: To assess whether patient-reported outcomes are independently associated with mortality beyond clinical risk factors in patients with IPF.Methods: Data from the observational IPF Prospective Outcomes Registry were used to examine associations between patient-reported outcomes at enrollment and the composite outcome of death or lung transplant in the following year. Associations were examined using univariable models and models adjusted for age and clinical variables that have been associated with death or lung transplant in patients with IPF in this cohort (oxygen use, forced vital capacity % predicted, and diffusing capacity of the lungs for carbon monoxide % predicted at enrollment).Results: Among 662 patients, 45 died and 12 underwent lung transplant over 1 year. In the model adjusted for age and clinical variables that were associated with death or lung transplant, worse scores on the St. George's Respiratory Questionnaire (SGRQ) total score (hazard ratio [HR], 1.22 [95% confidence interval (CI), 1.01-1.48] per 10-point increase), SGRQ activity score (HR, 1.25 [95% CI, 1.02-1.54] per 10-point increase) and SGRQ symptoms score (HR, 1.17 [95% CI, 1.01-1.36] per 10-point increase) were associated with death or lung transplant over 1 year.Conclusions: Patient-reported outcomes that assess symptoms and physical activity are independently associated with mortality in patients with IPF.
| S-EPMC7258421 | biostudies-literature
Project description:BackgroundVarious patient reported outcome measures (PROMs) are used in idiopathic pulmonary fibrosis (IPF). We aimed to describe their psychometric properties, assess their relationship with 1-year mortality and determine their minimal clinically important differences (MCIDs).MethodsIn a prospective multicentre study, participants with IPF completed the King's Brief Interstitial Lung Disease Questionnaire (K-BILD), the modified Medical Research Council (mMRC) dyspnoea scale, St George's Respiratory Questionnaire (SGRQ) and University of California, San Diego shortness of breath questionnaire (UCSD-SOBQ) three-monthly intervals over a 12-month period. Forced vital capacity (FVC) was matched with questionnaires and mortality was captured. Anchor- and distribution-based methods were used to derive MCID.ResultsData were available from 238 participants. All PROMs had good internal consistency and high degree of correlations with other tools (except UCSD-SOBQ correlated poorly with FVC). There were significant associations with mortality for K-BILD (hazard ratio 16.67; 95% CI 2.38-100) and SGRQ (hazard ratio 4.65; 95% CI 1.32-16.62) but not with the other PROMs or FVC. The median MCID (range) for K-BILD was 6.3 (4.1-7.0), SGRQ was 7.0 (3.8-9.6), mMRC was 0.4 (0.1-0.5) and UCSD-SOBQ was 9.6 (4.1-14.2).ConclusionsThe K-BILD was related to other severity measures and had the strongest relationship with mortality.
| S-EPMC8495510 | biostudies-literature
Project description:Over the past decade, several large registries of patients with idiopathic pulmonary fibrosis (IPF) have been established. These registries are collecting a wealth of longitudinal data on thousands of patients with this rare disease. The data collected in these registries will be complementary to data collected in clinical trials because the patient populations studied in registries have a broader spectrum of disease severity and comorbidities and can be followed for a longer period of time. Maintaining the quality and completeness of registry databases presents administrative and resourcing challenges, but it is important to ensuring the robustness of the analyses. Data from patient registries have already helped improve understanding of the clinical characteristics of patients with IPF, the impact that the disease has on their quality of life and survival, and current practices in diagnosis and management. In the future, analyses of biospecimens linked to detailed patient profiles will provide the opportunity to identify biomarkers linked to disease progression, facilitating the development of precision medicine approaches for prognosis and therapy in patients with IPF.
| S-EPMC6635784 | biostudies-literature
Project description:Over the past 20 years, there has been tremendous progress in the area of patient-reported outcomes (PROs). A PRO instrument is defined as any measure of a patient's health status that is elicited directly from the patient and assesses how the patient "feels or functions with respect to his or her health condition." The advances seen in clinical research regarding PROs has been mirrored in research in cystic fibrosis (CF). A large number of instruments have been used for both therapeutic and nontherapeutic clinical research for many chronic conditions. This review will summarize a history of the development of PROs and how PROs are viewed by the U.S. Food and Drug Administration. We will then review the current state of the art of patient-reported outcomes in CF, specifically addressing the evaluation of different PRO instruments in terms of their reliability and validity. Finally, we will delineate further areas for development of PROs in CF. We believe that the future of CF research will incorporate a more diverse selection of PRO outcome measures; these outcome measures ultimately may be incorporated into clinical care to standardize symptom assessment and provide information regarding the need for specific clinical interventions to improve the quality of care delivered to these patients.
| S-EPMC2647603 | biostudies-literature
Project description:Patient-reported outcomes (PROs) are reports of a person's health status that provide a global perspective of patient well-being. PROs can be classified into 4 primary domains: global, mental, physical, and social health. In this descriptive review, we focus on how PROs can be used in cardiac clinical trials, with an emphasis on cardiac surgical trials for patients with coronary heart disease and heart failure. We also highlight ongoing challenges and provide specific suggestions and novel opportunities to advance cardiac clinical trials. Current challenges include the long-term measurement of PROs in clinical trials beyond 1 year, inconsistency in the choice of the outcome measures among studies, and the lack of measurement of PROs across multiple domains. Opportunities for advancement include measuring PROs using consumer health informatics tools, including returning information back to participants in formats that they can understand using visualization. Future opportunities include quantifying cohort-specific minimal clinically important differences for PROs.
| S-EPMC8487900 | biostudies-literature
Project description:PURPOSE OF REVIEW:Idiopathic inflammatory myopathies (IIM) have considerable impact on patient symptoms and quality of life. We have reviewed the evolution of patient-centered care and use of patient-reported outcome measures (PROMs) for adults with IIM. RECENT FINDINGS:Use of PROMs in myositis care and research is limited, although the importance of incorporation into routine practice and trials has become increasingly recognized. Several key domains/measures have been identified including the patient global assessment of disease activity, physical function as measured by the health assessment questionnaire-disability index (HAQ-DI), Short Form Health Survey-36 (SF-36), or the Patient-Reported Outcome Measurement Information System ® (PROMIS®) in adult IIM. Data are limited for these instruments concerning their reliability, content and construct validity, and responsiveness. Incorporation of the patient perspective into clinical care and research may be used to address the unmet/unaddressed needs of the patient living with myositis. Several ongoing projects aim to bring validated PROMs to the IIM community.
| S-EPMC7050440 | biostudies-literature
Project description:Idiopathic pulmonary fibrosis (IPF) is a progressive and ultimately fatal disease which has a major impact on patients' quality of life (QOL). Except for lung transplantation, there is no curative treatment option. Fortunately, two disease-modifying drugs that slow down disease decline were recently approved. Though this is a major step forward, these drugs do not halt or reverse the disease, nor convincingly improve health-related QOL. In daily practice, disease behavior and response to therapy greatly vary among patients. It is assumed that this is related to the multiple biological pathways and complex interactions between genetic, molecular, and environmental factors that are involved in the pathogenesis of IPF. Recently, research in IPF has therefore started to focus on developing targeted therapy through identifying genetic risk factors and biomarkers. In this rapidly evolving field of personalized medicine, patient factors such as lifestyle, comorbidities, preferences, and experiences with medication should not be overlooked. This review describes recent insights and methods on how to integrate patient perspectives into personalized medicine. Furthermore, it provides an overview of the most used patient-reported outcome measures in IPF, to facilitate choices for both researchers and clinicians when incorporating the patient voice in their research and care. To enhance truly personalized treatment in IPF, biology should be combined with patient perspectives.
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Project description: Not available
| S-EPMC7159425 | biostudies-literature