The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry data

Granger, Emily, Keogh, Ruth H and Frost, Freddy ORCID: 0000-0002-3902-6502 (2022) 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, 8 (4). pp. 170-2022.

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Official URL: http://dx.doi.org/10.1183/23120541.00170-2022

Abstract

<h4>Introduction</h4>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 <i>versus</i> non-use in CFRD.<h4>Methods</h4>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 (FEV<sub>1</sub> %) 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.<h4>Results</h4>We identified 1613 individuals diagnosed with CFRD between 2008 and 2016 and included 1196 and 1192 in the FEV<sub>1</sub> % and BMI outcome analyses respectively. We found no evidence of an effect of insulin on FEV<sub>1</sub> % 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.<h4>Conclusion</h4>Using well-established national registry data, we found no evidence of long-term treatment effects for insulin on FEV<sub>1</sub> % or BMI in people with incident CFRD.

Item Type:	Article
Uncontrolled Keywords:	Clinical Research, Cystic Fibrosis, Rare Diseases, Diabetes, Nutrition, Lung, Metabolic and endocrine
Divisions:	Faculty of Health and Life Sciences Faculty of Health and Life Sciences > Institute of Life Courses and Medical Sciences
Depositing User:	Symplectic Admin
Date Deposited:	25 Nov 2022 09:46
Last Modified:	02 Apr 2024 09:25
DOI:	10.1183/23120541.00170-2022
Related URLs:	Author Publisher
URI:	https://livrepository.liverpool.ac.uk/id/eprint/3166364