CFTR Modulators and Gastrointestinal Complications

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

47023 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-10-01

Study Completion Date

2024-02-10

Brief Summary

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To elucidate the similarities and distinctions in non-pulmonary manifestations of cystic fibrosis (CF) including distal intestinal obstruction syndrome (DIOS) incidence and pancreatic enzyme replacement therapy (PERT) use between US and UK CF populations in a parallel study using data from the UK and US CF registries. To assess how CFTR modulators impacted upon recorded PERT use and incidence of DIOS.

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Detailed Description

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Cystic fibrosis (CF) is a autosomal recessive multi-system disorder caused by mutations to the cystic fibrosis transmembrane conductance regulator (CFTR) gene. As well as the documented respiratory complications of CF, gastrointestinal manifestations are of clinical importance. CFTR mutations in the gastrointestinal tract are responsible for pancreatic exocrine insufficiency in around 85% of people with CF (pwCF). In addition a severe gastrointestinal complication in CF is distal intestinal obstructive syndrome (DIOS), affecting 5.7% pwCF in the UK (2.5% in \<16 years and 7.7% in adults) and 2.1% in the US (\<18 years 1.7%, adult 2.4%) in 2019.

This is a parallel data registry study using data from the UK and US CF registries, with data provided for the time period 2007-2018. As such no individual participants will be recruited to the study. The CFTR modulators to be studies are Ivacaftor and lumacaftor/ivacaftor.

Study aims;

* Describe DIOS events and PERT usage in UK and US registries
* Determine the effect of CFTR modulators on the incidence of DIOS and use of PERT: population time series
* Determine the effect of CFTR modulators on the incidence of DIOS and use of PERT: patient-level time series

The outcomes of the above aims will be used to generate hypotheses regarding the effect of the newer CFTR modulators such as Symdeko/Symkevi and Tricaftor/Kaftrio on PERT usage and DIOS incidence in CF registry data post 2018. This will form the basis of future studies.

Conditions

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Cystic Fibrosis Gastrointestinal System Disease Distal Intestinal Obstruction Syndrome Pancreatic Enzyme Abnormality Pancreatic Disease Lung Diseases Digestive System Disease

Study Design

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Observational Model Type

OTHER

Study Time Perspective

RETROSPECTIVE

Study Groups

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Cystic fibrosis registry (UK)

People with cystic fibrosis registered in the UK CF registry

No intervention

Intervention Type OTHER

Registry study: No intervention

Cystic fibrosis registry (US)

People with cystic fibrosis registered in the US CF registry

No intervention

Intervention Type OTHER

Registry study: No intervention

Interventions

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No intervention

Registry study: No intervention

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* All patients with CF of any genotype on the UK and US cystic fibrosis registries from the period 2007 - 2018.

Exclusion Criteria

\- Patients whose CFTR modulator status is unknown or only have one year of CFTR data recorded on the registry will be excluded from the analysis of the effects of CFTR modulators. This is to account for the fact that DIOS data is annualised on the registries, therefore there is no certainty at what time of year DIOS was diagnosed in relation to commencing CFTR modulator therapy.

Eligible Sex

ALL

Accepts Healthy Volunteers

No