Liver function tests in F508del homozygous paediatric patients with cystic fibrosis taking lumacaftor/ivacaftor combination therapy
Aims
In clinical trials, elevated liver transaminase levels in patients taking lumacaftor/ivacaftor therapy were reported. Our aim was to assess in clinical practice, whether F508del homozygous paediatric CF patients had a derangement of liver functions tests (LFTs) while on lumacaftor/ivacaftor therapy.
Methods
A retrospective chart review audit in a single CF centre.
Results
Thirty-nine (43%) patients out of 91 CF clinic patients met criteria to start treatment. We observed a statistically significant decrease in ALT, ALP, GGT and total bilirubin levels, and no change in AST levels during first 3 months of treatment. In two patients (5%) AST levels rose to greater than three times the upper limit of normal (ULN) during treatment, however, these levels then decreased with continued use. A similar trend of improved LFTs was seen in a subgroup of patients with pre-existing liver disease (6/15.4% of patients). No patients died or experienced hepatic encephalopathy.
Conclusion
Our results were unexpected and encouraging. They suggest that, although the clinical trials report a risk of derangement of LFTs, the risk appears to be low.
History
Comments
The original article is available at www.imj.iePublished Citation
Paluck F. et al. Liver function tests in F508del homozygous paediatric patients with cystic fibrosis taking lumacaftor/ivacaftor combination therapy. Ir Med J. 2021;114(2):259Publication Date
February 2021Department/Unit
- Paediatrics
Publisher
Irish Medical AssociationVersion
- Published Version (Version of Record)