Introduction
Kaftrio improves spirometry, respiratory symptoms, reduces pulmonary exacerbations in Cystic Fibrosis Phe508del minimal function genotype adults1. Reduction in medication prescriptions such as mucolytics has previously not been demonstrated, which can reduce cost and treatment burden for patients.
Aims and Objectives
Assess prescription of oral (carbocysteine) and nebulized (Dornase alfa, Hypertonic Saline[HTS]) mucolytics following one year of Kaftrio administration.
Methods
Retrospective observational analysis of mucolytic prescriptions in 99 Phe508del homozygous patients. The age, gender, and presciprtion status of the three mucolytics was recorded at baseline and after one year of Kaftrio use. McNemar?s test used to assess for change in the use of each mucolytic individually, and a Wilcoxon Signed-Rank test to asses for a change in total mucolytic use. A Bonferroni Correction was employed to account for multiple comparison.
Results
Final analysis performed on 99 individuals (53% female, age range 19-59, mean 30.4 years, median 29 years). We found a significant decrease in the use of Dornase (p=4x10-7) and HTS (p=8x10-7), but not carbocisteine once we accounted for multiple tests (p=0.02). This may partially reflect lower baseline carbocisteine use. There was a significant decrease in overall mucolytic prescription (p=9x10-9)
Conclusion
Kaftrio reduces the prescription of Dornase alfa and HTS and overall mucolytic prescription in these patients. No significant reduction in carbocisteine presciption, though limited by sample size. This reinforces the role of Kaftrio in reducing the treatment burden in cystic fibrosis patients.