Health State Utility Data in Cystic Fibrosis: A Systematic Review.
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ABSTRACT: Introduction
Cystic fibrosis (CF) is a life-limiting, hereditable condition, with the highest prevalence in Europe. CF treatments have led to improvements in clinical symptoms, disease management and decelerated disease progression. However, little is known about the health state utility (HSU) associated with CF disease states, adverse events, and changes in disease severity. Although HSU data have contributed to existing health economic modelling studies, a lack of such data have been highlighted. This systematic review aims to provide a summary of HSU-related research in CF and highlight related research gaps.Methods
Online searches were performed in six databases and studies in any of the following categories were included: (1) estimation of HSUs in CF; (2) mapping studies between patient-reported outcome measures (PROMs) and HSUs; (3) economic evaluations on the management of CF that report primary HSU data; and (4) any CF clinical trial that reported HSU as an outcome.Results
A total of 17 studies were reviewed, of which 12 provided HSU values for specific CF populations. The remaining five articles provided HSU data that were broken down by CF relevant health states, including lung transplantations, pulmonary exacerbation (PEx) events and forced expiratory volume in 1 s (FEV1).Conclusion
Current HSU data in CF are limited and there is considerable scope for further research, both in providing HSU values for CF and in investigating methods for HSU elicitation/evaluation in CF populations.
SUBMITTER: Mohindru B
PROVIDER: S-EPMC7018933 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:INTRODUCTION:Increasingly, assessment of healthcare technologies and interventions requires the assessment of both costs and utilities. Health state utility values (HSUVs) are measured using a range of generic and condition-specific measures. While reviews have identified that generic measures of HSUVs may lack validity in adults with conditions that result in physical disability, there is little information available on the methods used to obtain HSUVs in children and adolescents with disabilities. The objectives of this systematic review are to describe the methods used to obtain HSUVs, including mode of administration and psychometric properties, and provide summary statistics for HSUVs among children and adolescents with disabilities. METHODS AND ANALYSIS:The following databases will be searched from inception for English-language studies of any design: PubMed, PsychInfo, Medline, Scopus, CINAHL Plus, Econlit and EMBASE databases. Two reviewers will independently screen titles, abstracts and full text articles for studies reporting HSUVs and/or data on the psychometric properties of preference-based measures for children and adolescents with disabilities aged up to 19 years. Two reviewers will independently extract data items including descriptors of the study methods and sample, instruments used to capture HSUVs, summary statistics for HSUVs and items relating to the quality of reporting. A descriptive summary of results from included studies and summary statistics for HSUVs will be presented. If sufficient data is identified, we will pool summary statistics for HSUVs according to the method used to obtain the HSUV using a random effects model. In addition, we will explore the determinants of the HSUVs using a meta-regression. ETHICS AND DISSEMINATION:Ethical approval will not be required as no original data will be collected as part of this review. The completed review will be submitted for publication in a peer-reviewed journal and presentation at conferences. PROSPERO REGISTRATION NUMBER:CRD42018086574.
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Project description:BackgroundUsing appropriate health state utility values (HSUVs) is critical for economic evaluation of new lung cancer interventions, such as low-dose computed tomography screening and immunotherapy. Therefore, we provide a systematic review and meta-analysis of community- and choice-based HSUVs for lung cancer.MethodsOn 6 March 2017, we conducted a systematic search of the following databases: Embase, Ovid MEDLINE, Web of Science, Cochrane CENTRAL, Google Scholar, and the School of Health and Related Research Health Utility Database. The search was updated on 17 April 2019. Studies reporting mean or median lung cancer-specific HSUVs including a measure of variance were included and assessed for relevance and validity. Studies with high relevance (i.e. community- and choice-based) were further analysed. Mean HSUVs were pooled using random-effects models for all stages, stages I-II, and stages III-IV. For studies with a control group, we calculated the disutility due to lung cancer. A sensitivity analysis included only the methodologically most comparable studies (i.e. using the EQ-5D instrument and matching tariff). Subgroup analyses were conducted by time to death, histology, sex, age, treatment modality, treatment line, and progression status.ResultsWe identified and analysed 27 studies of high relevance. The pooled HSUV was 0.68 (95% confidence interval [CI] 0.61-0.75) for all stages, 0.78 (95% CI 0.70-0.86) for stages I-II, and 0.69 (95% CI 0.65-0.73) for stages III-IV (p = 0.02 vs. stage I-II). Heterogeneity was present in each pooled analysis (p < 0.01; I2 = 92-99%). Disutility due to lung cancer ranged from 0.11 (95% CI 0.05-0.17) to 0.27 (95% CI 0.18-0.36). In the sensitivity analysis with the methodologically most comparable studies, stage-specific HSUVs varied by country. Such studies were only identified for Canada, China, Spain, the UK, the USA, Denmark, Germany, and Thailand. In the subgroup analysis by time to death, HSUVs for metastatic non-small-cell lung cancer ranged from 0.83 (95% CI 0.82-0.85) at ≥ 360 days from death to 0.56 (95% CI 0.46-0.66) at < 30 days from death. Among patients with metastatic non-small-cell lung cancer, HSUVs were lower for those receiving third- or fourth-line treatment and for those with progressed disease. Results of subgroup analyses by histology, sex, age, and treatment modality were ambiguous.ConclusionsThe presented evidence supports the use of stage- and country-specific HSUVs. However, such HSUVs are unavailable for most countries. Therefore, our pooled HSUVs may provide the best available stage-specific HSUVs for most countries. For metastatic non-small-cell lung cancer, adjusting for the decreased HSUVs in the last year of life may be considered, as may further stratification of HSUVs by treatment line or progression status. If required, HSUVs for other health states may be identified using our comprehensive breakdown of study characteristics.
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Project description:This multi-center study will compare multi-target DNA and quantitative FIT stool-based testing to colonoscopy in individuals with Cystic Fibrosis (CF) undergoing colon cancer screening with colonoscopy. The primary endpoint is detection of any adenomas, including advanced adenomas and colorectal cancer (CRC).
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