Data Resource Profile: The UK Cystic Fibrosis Registry

Taylor-Robinson, David; Archangelidi, Olga; Carr, S.B.; Cosgriff, Rebecca; Gunn, E.; Keogh, Ruth H.; MacDougall, Amy; Newsome, Simon; Schlüter, Daniela K; Stanojevic, Sanja; Bilton, Diana

doi:10.1093/ije/dyx196

Cited by 66 publications

(71 citation statements)

References 12 publications

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“…For rare diseases, this is particularly important [26,27]. CF is a characteristic example of a rare disease that has benefited greatly from large long-standing registries [28][29][30]. However, registries usually collect a minimal dataset and need to be completed in addition to regular clinical forms increasing the workload of healthcare professional.…”

Section: Discussionmentioning

confidence: 99%

Standardised clinical data from patients with primary ciliary dyskinesia: FOLLOW-PCD

Goutaki

Papon

Boon³

et al. 2020

ERJ Open Res

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Clinical data on primary ciliary dyskinesia (PCD) are limited, heterogeneous and mostly derived from retrospective chart reviews, leading to missing data and unreliable symptoms and results of physical examinations. We need standardised prospective data collection to study phenotypes, severity and prognosis and improve standards of care.A large, international and multidisciplinary group of PCD experts developed FOLLOW-PCD, a standardised clinical PCD form and patient questionnaire. We identified existing forms for clinical data collection via the Better Experimental Approaches to Treat PCD (BEAT-PCD) COST Action network and a literature review. We selected and revised the content items with the working group and patient representatives. We then revised several drafts in an adapted Delphi process, refining the content and structure.FOLLOW-PCD has a modular structure, to allow flexible use based on local practice and research focus. It includes patient-completed versions for the modules on symptoms and lifestyle. The form allows a comprehensive standardised clinical assessment at baseline and for annual reviews and a short documentation for routine follow-up. It can either be completed using printable paper forms or using an online REDCap database.Data collected in FOLLOW-PCD version 1.0 is available in real-time for national and international monitoring and research. The form will be adapted in the future after extensive piloting in different settings and we encourage the translation of the patient questionnaires to multiple languages. FOLLOW-PCD will facilitate quality research based on prospective standardised data from routine care, which can be pooled between centres, to provide first-line and real-time evidence for clinical decision-making.

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Section: Discussionmentioning

confidence: 99%

Standardised clinical data from patients with primary ciliary dyskinesia: FOLLOW-PCD

Goutaki

Papon

Boon³

et al. 2020

ERJ Open Res

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“…Such longitudinal measurements will allow for more reflective health economic evaluation of interventions. Another avenue for elicitation/evaluation of HSU data could be the CF Trust registry, which recently launched a study looking at quality of life in CF adults [49,50]. Studies on HSUs using the EQ-5D would also allow research to further explore problems around the ceiling effects of the instrument that have been mentioned in NICE appraisals of Orkambi ® [51] as well as the published literature [18].…”

Section: Discussionmentioning

confidence: 99%

Health State Utility Data in Cystic Fibrosis: A Systematic Review

Mohindru

Turner

Sach

et al. 2019

PharmacoEconomics Open

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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 (FEV 1). 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.

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“…The UK CF registry collects data from patient annual reviews and is estimated to capture 99% of the current UK CF population 12. The stepwise introduction of NBS through regional programmes and the capture of all individuals with CF in the registry, those diagnosed by NBS and those diagnosed clinically, enabled us to evaluate NBS as a natural experiment.…”

Section: Methodsmentioning

confidence: 99%

Impact of newborn screening on outcomes and social inequalities in cystic fibrosis: a UK CF registry-based study

Schlüter

Southern

Dryden

et al. 2019

Thorax

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BackgroundNewborn bloodspot screening (NBS) for cystic fibrosis (CF) was introduced across the UK in 2007 but the impact on clinical outcomes and health inequalities for children with CF is unclear.MethodsWe undertook longitudinal analyses of UK CF registry data on over 3000 children with CF born between 2000 and 2015. Clinical outcomes were the trajectories of percent predicted forced expiratory volume in one second (%FEV1) from age 5, weight for age and body mass index (BMI) SD-scores from age one, and time to chronic Pseudomonas aeruginosa (cPA) infection. Using mixed effects and time-to-event models we assessed the association of NBS with outcomes and potential interactions with childhood socioeconomic conditions, while adjusting for confounders.ResultsNBS was associated with higher average lung function trajectory (+1.56 FEV1 percentage points 95% CI 0.1 to 3.02, n=2216), delayed onset of cPA, and higher average weight trajectory intercept at age one (+0.16 SD; 95% CI 0.07 to 0.26, n=3267) but negative rate of weight change thereafter (−0.02 SD per year; 95% CI −0.03 to −0.00). We found no significant association of NBS with BMI or rate of change of lung function. There was no clear evidence of an impact of NBS on health inequalities early in life.ConclusionsChildren diagnosed with CF by NBS in the UK have better lung function and increased early weight but NBS does not appear to have narrowed early health inequalities.

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Data Resource Profile: The UK Cystic Fibrosis Registry

Cited by 66 publications

References 12 publications

Standardised clinical data from patients with primary ciliary dyskinesia: FOLLOW-PCD

Standardised clinical data from patients with primary ciliary dyskinesia: FOLLOW-PCD

Health State Utility Data in Cystic Fibrosis: A Systematic Review

Impact of newborn screening on outcomes and social inequalities in cystic fibrosis: a UK CF registry-based study

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