Prospective Case-Control Safety Study of Bronchitol (inhaled mannitol) in Patients with Cystic Fibrosis from the UK CF Registry

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Effectiveness study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  BRONCHITOL
• Medical condition to be studied:
  Cystic fibrosis

Population studied

• Short description of the study population:
  Adult (≥ 18 years) patients in the CF registry database who were prescribed Bronchitol. The unexposed group were untreated patients.
• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with Cystic Fibrosis
• Estimated number of subjects:
  446

Study design details

• Main study objective:
  To assess the rates of identified and potential risk of Bronchitol in CF through a comparison of Bronchitol treated vs. non-treated patients in a cohort matched for key characteristics from the CF registry over a 1 to 5 year period. Safety analysis would be conducted on a 6 monthly basis for three years and then annually for two further years..
• Outcomes:
  To assess the rates of identified and potential risks of Bronchitol through a comparison of treated vs. non-treated patients, - To compare the rates of identified and potential risks against background rates in the general CF population.- To assess the rate of identified and potential risks in the population below 18 years of age- To compare the effect on lung function in treated vs. non-treated patients- To compare the effect on CF exacerbations in treated vs. non-treated patients
• Data analysis plan:
  At each analysis time a propensity score analysis will be performed. Here we will use logistic regression to estimate the probability of being treated with Bronchitol. This will generate a propensity score for each patient which will be used in subsequent analyses. Full Analysis PopulationAll patients (treated and untreated) from CF Registry will be included in the analyses. For this population, we will include:- Simple descriptive analyses (frequency and percentage) for AEs- Negative Binomial regression for the count of events for each AE with adjustment for propensity scores.- Cox regression for time to first event for each AE (Hazard Ratio), with adjustment for propensity scores.Cox regression will be used to study the time to first event for each AE. As for the negative binomial regression models these will be done both with and without adjustment for potential confounders.Random effects linear models will be used to study changes in FEV1 over time

Documents

Study results

5 yr PASS - NACF poster 2018 Final

Abstract 484 NACF 2018

Study publications

Carr S et al., Pediatric pulmonology Vol 53, Issue S2 Abstract 484