The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry data
Introduction Cystic fibrosis-related diabetes (CFRD) is a common complication of cystic fibrosis and is associated with deleterious clinical outcomes. Insulin is recommended as a treatment by international guidelines. However, there are scarce clinical trial data to support the use of insulin, and l...
| Main Authors: | , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
European Respiratory Society
2022-11-01
|
| Series: | ERJ Open Research |
| Online Access: | http://openres.ersjournals.com/content/8/4/00170-2022.full |
| _version_ | 1797809454267236352 |
|---|---|
| author | Emily Granger Ruth H. Keogh Freddy Frost |
| author_facet | Emily Granger Ruth H. Keogh Freddy Frost |
| author_sort | Emily Granger |
| collection | DOAJ |
| description | Introduction
Cystic fibrosis-related diabetes (CFRD) is a common complication of cystic fibrosis and is associated with deleterious clinical outcomes. Insulin is recommended as a treatment by international guidelines. However, there are scarce clinical trial data to support the use of insulin, and little is known about the long-term outcomes of treatment. The aim of this study was to compare the long-term impacts of insulin use versus non-use in CFRD.
Methods
We used data from the national UK Cystic Fibrosis Registry and adopted a target trial framework. Eligible individuals included those 12 years and older with a new diagnosis of CFRD. Outcomes were change in % predicted forced expiratory volume in 1 s (FEV1 %) and body mass index z-scores (BMI) over a 5-year follow-up period. Treatment strategies were to receive insulin or not for the duration of follow-up. Treatment effect estimates were obtained using two methods to control for confounding: inverse-probability-of-treatment weighted estimation of marginal structural models and the G-formula.
Results
We identified 1613 individuals diagnosed with CFRD between 2008 and 2016 and included 1196 and 1192 in the FEV1 % and BMI outcome analyses respectively. We found no evidence of an effect of insulin on FEV1 % over the 5-year study period. Similarly, we found no overall effect of insulin on BMI; however, there was some evidence for a positive treatment effect in patients with lower baseline BMI.
Conclusion
Using well-established national registry data, we found no evidence of long-term treatment effects for insulin on FEV1 % or BMI in people with incident CFRD. |
| first_indexed | 2024-03-13T06:53:03Z |
| format | Article |
| id | doaj.art-b9f3ee6615b24f84bb3fc81d5f3a9f1c |
| institution | Directory Open Access Journal |
| issn | 2312-0541 |
| language | English |
| last_indexed | 2024-03-13T06:53:03Z |
| publishDate | 2022-11-01 |
| publisher | European Respiratory Society |
| record_format | Article |
| series | ERJ Open Research |
| spelling | doaj.art-b9f3ee6615b24f84bb3fc81d5f3a9f1c2023-06-07T13:30:36ZengEuropean Respiratory SocietyERJ Open Research2312-05412022-11-018410.1183/23120541.00170-202200170-2022The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry dataEmily Granger0Ruth H. Keogh1Freddy Frost2 Dept of Medical Statistics, Faculty of Epidemiology and Population Health, London School of Hygiene and Tropical Medicine, London, UK Dept of Medical Statistics, Faculty of Epidemiology and Population Health, London School of Hygiene and Tropical Medicine, London, UK Institute of Infection, Veterinary and Ecological Sciences, University of Liverpool, Liverpool, UK Introduction Cystic fibrosis-related diabetes (CFRD) is a common complication of cystic fibrosis and is associated with deleterious clinical outcomes. Insulin is recommended as a treatment by international guidelines. However, there are scarce clinical trial data to support the use of insulin, and little is known about the long-term outcomes of treatment. The aim of this study was to compare the long-term impacts of insulin use versus non-use in CFRD. Methods We used data from the national UK Cystic Fibrosis Registry and adopted a target trial framework. Eligible individuals included those 12 years and older with a new diagnosis of CFRD. Outcomes were change in % predicted forced expiratory volume in 1 s (FEV1 %) and body mass index z-scores (BMI) over a 5-year follow-up period. Treatment strategies were to receive insulin or not for the duration of follow-up. Treatment effect estimates were obtained using two methods to control for confounding: inverse-probability-of-treatment weighted estimation of marginal structural models and the G-formula. Results We identified 1613 individuals diagnosed with CFRD between 2008 and 2016 and included 1196 and 1192 in the FEV1 % and BMI outcome analyses respectively. We found no evidence of an effect of insulin on FEV1 % over the 5-year study period. Similarly, we found no overall effect of insulin on BMI; however, there was some evidence for a positive treatment effect in patients with lower baseline BMI. Conclusion Using well-established national registry data, we found no evidence of long-term treatment effects for insulin on FEV1 % or BMI in people with incident CFRD.http://openres.ersjournals.com/content/8/4/00170-2022.full |
| spellingShingle | Emily Granger Ruth H. Keogh Freddy Frost The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry data ERJ Open Research |
| title | The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry data |
| title_full | The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry data |
| title_fullStr | The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry data |
| title_full_unstemmed | The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry data |
| title_short | The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry data |
| title_sort | long term effects of insulin use in incident cystic fibrosis related diabetes a target trial emulated using longitudinal national registry data |
| url | http://openres.ersjournals.com/content/8/4/00170-2022.full |
| work_keys_str_mv | AT emilygranger thelongtermeffectsofinsulinuseinincidentcysticfibrosisrelateddiabetesatargettrialemulatedusinglongitudinalnationalregistrydata AT ruthhkeogh thelongtermeffectsofinsulinuseinincidentcysticfibrosisrelateddiabetesatargettrialemulatedusinglongitudinalnationalregistrydata AT freddyfrost thelongtermeffectsofinsulinuseinincidentcysticfibrosisrelateddiabetesatargettrialemulatedusinglongitudinalnationalregistrydata AT emilygranger longtermeffectsofinsulinuseinincidentcysticfibrosisrelateddiabetesatargettrialemulatedusinglongitudinalnationalregistrydata AT ruthhkeogh longtermeffectsofinsulinuseinincidentcysticfibrosisrelateddiabetesatargettrialemulatedusinglongitudinalnationalregistrydata AT freddyfrost longtermeffectsofinsulinuseinincidentcysticfibrosisrelateddiabetesatargettrialemulatedusinglongitudinalnationalregistrydata |