Understanding and optimizing health-related quality of life and physical functional capacity in idiopathic pulmonary fibrosis.
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SUBMITTER: Olson AL
PROVIDER: S-EPMC4876092 | biostudies-literature | 2016
REPOSITORIES: biostudies-literature
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Understanding and optimizing health-related quality of life and physical functional capacity in idiopathic pulmonary fibrosis.
Olson Amy L AL Brown Kevin K KK Swigris Jeffrey J JJ
Patient related outcome measures 20160517
Idiopathic pulmonary fibrosis (IPF) is a relentlessly progressive pulmonary disease characterized by the insidious onset of shortness of breath due to parenchymal scarring. As IPF progresses, breathlessness worsens, physical functional capacity declines, and health-related quality of life (HRQL) - the impact of health or disease on a person's satisfaction with their overall station in life - deteriorates. These two inextricably linked variables - breathlessness and physical functional capacity - ...[more]
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Project description:Despite major research efforts leading to the recent approval of pirfenidone and nintedanib, the dismal prognosis of idiopathic pulmonary fibrosis (IPF) remains unchanged. The elaboration of international diagnostic criteria and disease stratification models based on clinical, physiological, radiological, and histopathological features has improved the accuracy of IPF diagnosis and prediction of mortality risk. Nevertheless, given the marked heterogeneity in clinical phenotype and the considerable overlap of IPF with other fibrotic interstitial lung diseases (ILDs), about 10% of cases of pulmonary fibrosis remain unclassifiable. Moreover, currently available tools fail to detect early IPF, predict the highly variable course of the disease, and assess response to antifibrotic drugs. Recent advances in understanding the multiple interrelated pathogenic pathways underlying IPF have identified various molecular phenotypes resulting from complex interactions among genetic, epigenetic, transcriptional, post-transcriptional, metabolic, and environmental factors. These different disease endotypes appear to confer variable susceptibility to the condition, differing risks of rapid progression, and, possibly, altered responses to therapy. The development and validation of diagnostic and prognostic biomarkers are necessary to enable a more precise and earlier diagnosis of IPF and to improve prediction of future disease behaviour. The availability of approved antifibrotic therapies together with potential new drugs currently under evaluation also highlights the need for biomarkers able to predict and assess treatment responsiveness, thereby allowing individualised treatment based on risk of progression and drug response. This approach of disease stratification and personalised medicine is already used in the routine management of many cancers and provides a potential road map for guiding clinical care in IPF.
| S-EPMC4890320 | biostudies-literature
Project description:This is a time of substantial progress in the evaluation and care of patients with idiopathic pulmonary fibrosis (IPF). In addition to the approval and widespread availability of the first IPF-specific therapies, there have been improvements in imaging interpretation and lung biopsy methods to enable more expeditious and more accurate diagnosis. Recent advances in identifying genetic factors that underlie susceptibility to IPF and affect prognosis have raised the possibility of personalized therapeutic approaches in the future. Further, evolving work is elucidating novel mechanisms influencing epithelial, mesenchymal, and inflammatory cell responses during the injury-repair process, thus advancing understanding of disease pathogenesis. As analytic approaches mature, the field is now poised to harness the power of rapidly advancing "omics" technologies to further accelerate progress.
| S-EPMC6378692 | biostudies-literature
Project description:BackgroundIdiopathic pulmonary fibrosis (IPF) profoundly affects the quality of patients' lives. A systematic review was performed to evaluate critically the published literature and to examine what is known about health-related quality of life (HRQL) in patients with IPF.MethodsThe MEDLINE, EMBASE, Health and Psychosocial Instruments, and Cochrane Library databases were searched to 1 April 2004. Abstracts and bibliographies of published articles were scanned and contact was made with investigators. Included studies analysed HRQL (or quality of life) in at least 10 patients with IPF. Two reviewers independently selected studies, evaluated their quality according to predetermined criteria, and abstracted data on study design, patients' demographic and clinical characteristics, and quality of life outcome measures.ResultsSeven studies met the inclusion criteria. The studies enrolled 512 patients with IPF and used three different instruments to measure HRQL. All studies had important limitations in methodological quality; none measured longitudinal changes in HRQL over time. Patients reported substantially impaired HRQL, especially in domains that measured physical health and level of independence. Patients with IPF appear to have similar impairments in HRQL to those with chronic obstructive pulmonary disease. Measures of dyspnoea were moderately correlated with scores from domains that measured physical health (R2 = 0.03-0.66) and energy/fatigue/pep (R2 = 0.19-0.55), but measures of pulmonary function and gas exchange did not correlate as strongly with these and other domains.ConclusionStudies of HRQL in patients with IPF suggest that, in addition to the obvious effect on physical health, general health, energy level, respiratory symptoms, and level of independence are also impaired. Variability in HRQL among patients is not fully explained by measures of dyspnoea or pulmonary function, suggesting that HRQL measures provide unique information. More research is needed to identify or design appropriate measurement instruments for patients with IPF and to examine changes in HRQL over time or in response to specific treatments.
| S-EPMC1747452 | biostudies-literature
Project description:BackgroundIdiopathic pulmonary fibrosis (IPF) is one of the common types of interstitial lung disease having high prevalence and mortality worldwide. As a result of patient-centred hindering factors of adherence to centre-based pulmonary rehabilitation (PR), home-based PR is an alternate mode of rehabilitating individuals with IPF. This systematic review will evaluate the effectiveness of unsupervised home-based PR on functional capacity and health-related quality of life (HRQoL) in individuals with IPF.MethodsClinically stable, high resolution computed tomography and physician diagnosed IPF participants having modified Medical Research Council score below 5 will be considered for the systematic review. Studies involving home-based PR as an intervention to treat individuals with IPF will be considered. Randomised controlled trials and quasi-randomised studies (with two groups followed up) are eligible to be included. Outcomes of our interest are functional capacity (6-min walk distance, shuttle walk test and incremental shuttle walk test) and secondary outcome measure would include assessment of quality of life and adverse effects of intervention. Electronic databases such as SCOPUS, Medline (PubMed and Web of Science), PEDRo and CINAHL will be searched using database specific terms. Additionally, forward and backward citations of included studies will be searched to identify potential records. Two review authors, independently, will conduct the screening, data extraction using a customised standard tool, and critical appraisal using Cochrane Risk of Bias 2 tool of included studies. If data permits, meta-analysis will be conducted. In case of substantial heterogeneity, we will do a narrative synthesis. Subgroup analysis will be undertaken based on various contextual and interventional factors.DiscussionThis review will provide comprehensive evidence on the effectiveness of unsupervised home-based PR to physiotherapists, policy makers and researchers who are interested in IPF management. Findings from this review may guide the development and evaluation of more robust evidence based home-based PR that aimed to improve functional capacity among people with IPF.Systematic review registrationPROSPERO CRD42020213883.
| S-EPMC8591795 | biostudies-literature
Project description:No therapy is known to improve health-related quality of life (HRQoL) or dyspnoea in patients with idiopathic pulmonary fibrosis. The present study investigated longitudinal changes in HRQoL and dyspnoea and explored the effects of bosentan on these end-points during the Bosentan Use in Interstitial Lung Disease (BUILD)-1 trial. In total, 154 subjects received oral bosentan (n = 71) or placebo (n = 83). Changes in HRQoL and dyspnoea from baseline to month (M) 6 and up to M12 were measured using the St George's Respiratory Questionnaire (SGRQ), 36-item short-form health survey (SF-36), Transition Dyspnoea Index and Borg dyspnoea index. Overall, minimal changes occurred in measures of HRQoL and dyspnoea among placebo-treated subjects during the study. The effects of bosentan treatment on HRQoL and dyspnoea in the all-treated population were minimal. However, in the subset of subjects who had undergone surgical lung biopsy for diagnosis of idiopathic pulmonary fibrosis, treatment effects were observed up to M12 in the impact domain of the SGRQ and the physical functioning, general health and role emotional domains of the SF-36. HRQoL and dyspnoea changed minimally during the course of the present study. Observations from exploratory analyses suggested benefits of bosentan on HRQoL among patients who had undergone surgical lung biopsy for diagnosis, and they merit further investigation.
| S-EPMC2848824 | biostudies-literature
Project description:BackgroundPatients with interstitial lung disease (ILD) have poor health-related quality of life (HRQL). However, whether HRQL differs among different subtypes of ILD is unclear. The aim of this study was to determine whether HRQL was different among patients with idiopathic pulmonary fibrosis (IPF) and chronic hypersensitivity pneumonitis (CHP).MethodsWe identified patients from an ongoing longitudinal cohort of patients with ILD. HRQL was assessed using the Short Form (SF)-36 medical outcomes form (version 2.0). Regression analysis was used to determine the association between clinical covariates and HRQL, primarily the physical component summary (PCS) and mental component summary (MCS) score. A multivariate regression model was created to identify potential covariates that could help explain the association between the ILD subtype and HRQL.ResultsPatients with IPF (n = 102) were older, more likely to be men, and more likely to have smoked. Pulmonary function was similar between the groups. The patients with CHP (n = 69) had worse HRQL across all eight domains of the SF-36, as well as the PCS and MCS, compared with patients with IPF (P < .01-.09). This pattern remained after controlling for age and pulmonary function (P < .01-.02). Covariates explaining part of the relationship between disease subtype and PCS score included severity of dyspnea (P < .01) and fatigue (P < .01). Covariates explaining part of the relationship between disease subtype and MCS score included severity of dyspnea (P < .01), female sex (P = .02), and fatigue (P = .02).ConclusionsHRQL is worse in CHP compared with IPF. HRQL differences between ILD subtypes are explained in part by differences in sex, dyspnea, and fatigue.
| S-EPMC4042514 | biostudies-literature
Project description:BackgroundFew studies have investigated the relationship between physiological outcomes and quality of life in patients with idiopathic pulmonary fibrosis (IPF). Health-related quality of life (HRQL) data may provide a valuable patient-centred outcome for the evaluation of interventions. Determination of which physiological and functional variables are associated with HRQL may facilitate interpretation of disease progression and impacts of therapy.ObjectivesTo determine whether an association between Medical Outcomes Short-Form 36 and St George's Respiratory Questionnaire scores and other markers of disease severity exist, and to identify which physiological and functional variables are independently associated with HRQL.MethodsIn a cross-sectional study, 137 patients with IPF completed HRQL surveys and underwent clinical assessment. Multiple linear regression analysis was used to determine the relationship between age, forced vital capacity, diffusing capacity of carbon monoxide, 6 min walk distance (6MWD) and oxygen requirement, and HRQL scores.Results6MWD was the only functional measure of disease severity significantly associated with all domain scores of the St George's Respiratory Questionnaire and the Medical Outcomes Short-Form 36 physical component summary score.Conclusions6MWD was associated with HRQL among patients with IPF. This highlights the importance of 6MWD not only as a prognostic indicator in IPF, but also a predictor of HRQL and a meaningful outcome for patients.
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Project description:INTRODUCTION:Forced vital capacity is the only registrational endpoint in idiopathic pulmonary fibrosis clinical trials. As most new treatments will be administered on top of standard of care, estimating treatment response will become more challenging. We developed a simulation model to quantify variability associated with forced vital capacity decline. METHODS:The model is based on publicly available clinical trial summary and home spirometry data. A single, illustrative trial setting is reported. Model assumptions are 400 subjects randomised 1:1 to investigational drug or placebo over 52 weeks, 50% of each group receiving standard of care (all-comer population), and a 90-mL treatment difference in annual forced vital capacity decline. Longitudinal profiles were simulated and the impact of varying clinical scenarios evaluated. RESULTS:Power to detect a significant treatment difference was 87-97%, depending on the analysis method. Repeated measures analysis generally outperformed analysis of covariance and mixed linear models, particularly with missing data (as simulated data were non-linear). A 15% yearly random dropout rate led to 0.6-5% power loss. Forced vital capacity decline-related dropout introduced greater power loss (up to 12%), as did subjects starting/stopping standard of care or investigational drug. Power was substantially lower for a 26-week trial due to the smaller assumed treatment effect at week 26 (sample size would need doubling to reach a power similar to that of a 52-week trial). CONCLUSIONS:Our model quantifies forced vital capacity decline and associated variability, with all the caveats of background therapy, permitting robust power calculations to inform future idiopathic pulmonary fibrosis clinical trial design. FUNDING:Galapagos NV (Mechelen, Belgium).
| S-EPMC6822798 | biostudies-literature
Project description:BackgroundIdiopathic pulmonary fibrosis (IPF) is a chronic disease with a high symptom burden and poor survival that influences patients' health-related quality of life (HRQOL). We aimed to evaluate IPF patients' symptoms and HRQOL in a well-documented clinical cohort during their last two years of life.MethodsIn April 2015, we sent the Modified Medical Research Council Dyspnea Scale (MMRC), the modified Edmonton Symptom Assessment Scale (ESAS) and a self-rating HRQOL questionnaire (RAND-36) to 300 IPF patients, of which 247 (82%) responded. Thereafter, follow-up questionnaires were sent every six months for two years.ResultsNinety-two patients died by August 2017. Among these patients, HRQOL was found to be considerably low already two years before death. The most prominent declines in HRQOL occurred in physical function, vitality, emotional role and social functioning (p?<?0.001). The proportion of patients with MMRC scores ?3 increased near death. Breathlessness and fatigue were the most severe symptoms. Symptom severity for the following symptoms increased significantly and reached the highest mean scores during the last six months of life (numeric rating scale/standard deviation): breathlessness (7.1/2.8), tiredness (7.0/2.3), dry mouth (6.0/3.0), cough (5.8/2.9), and pain with movement (5.0/3.5).ConclusionsTo our knowledge this is the first study demonstrating, that IPF patients experience remarkably low HRQOL already two years before death, especially regarding physical role. In addition, they suffer from severe breathlessness and fatigue. Furthermore, physical, social and emotional wellbeing deteriorate, and symptom burden increases near death. Regular symptom and HRQOL measurements are essential to assess palliative care needs in patients with IPF.
| S-EPMC6247520 | biostudies-literature
Project description:BACKGROUND:The INSIGHTS-IPF registry provides one of the largest data sets of clinical data and self-reported patient related outcomes including health related quality of life (QoL) on patients with idiopathic pulmonary fibrosis (IPF). We aimed to describe associations of various QoL instruments between each other and with patient characteristics at baseline. METHODS:Six hundred twenty-three IPF patients with available QoL data (St George's Respiratory Questionnaire SGRQ, UCSD Shortness-of-Breath Questionnaire SoB, EuroQol visual analogue scale and index EQ-5D, Well-being Index WHO-5) were analysed. Mean age was 69.6 ± 8.7 years, 77% were males, mean disease duration 2.0 ± 3.3 years, FVC pred was 67.5 ± 17.8%, DLCO pred 35.6 ± 17%. RESULTS:Mean points were SGRQ total 48.3, UCSD SoB 47.8, EQ-5D VAS 66.8, and WHO-5 13.9. These instruments had a high or very high correlation (exception WHO-5 to EQ-5D VAS with moderate correlation). On bivariate analysis, QoL by SGRQ total was statistically significantly associated with clinical symptoms (NYHA; p < 0.001), number of comorbidities (p < 0.05), hospitalisation rate (p < 0.01) and disease severity (as measured by GAP score, CPI, FVC and 6-min walk test; p < 0.05 each). Multivariate analyses showed a significant association between QoL (by SGRQ total) and IPF duration, FVC, age, NYHA class and indication for long-term oxygen treatment. CONCLUSIONS:Overall, IPF patients under real-life conditions have lower QoL compared to those in clinical studies. There is a meaningful relationship between QoL and various patient characteristics. TRIAL REGISTRATION:The INSIGHTS-IPF registry is registered at Clinicaltrials.gov ( NCT01695408 ).
| S-EPMC5512739 | biostudies-literature