An interesting study (C Tetard et al. JPGN 2020; 71: 778-781. Reduced Intestinal Inflammation With Lumacaftor/Ivacaftor in Adolescents With Cystic Fibrosis) describes the association of Lumacaftor/Ivacaftor and improvement in intestinal inflammation based on calprotectin levels.
This was a small study with 15 patients, with 9 patients having very elevated fecal calprotectin (FC) (>250) and the median level was 713 mcg/g for the entire cohort at baseline. After treatment with lumacaftor/Ivacaftor, median FC level was 102 mcg/g.
This pilot study is intriguing and makes the point that intestinal inflammation is commonly seen in children with cystic fibrosis. However, this study has some striking differences from prior studies.
- H Ellemunter et al. JPGN 2017; 65: 438-442. Fecal Calprotectin in Cystic Fibrosis and Its Relation to Disease Parameters: A Longitudinal Analysis for 12 Years This study with 171 patients and more than 2400 FC values showed that ~60% had elevated FC (>50) but these values were much lower. The cohort had a median value of 61. Patients with pancreatic insufficiency (compared with pancreatic sufficiency) had higher FC (68 vs 29).
- R Enaud et al. J. Clin. Med. 2019, 8(5), 645; https://doi.org/10.3390/jcm805064 Intestinal Inflammation in Children with Cystic Fibrosis Is Associated with Crohn’s-Like Microbiota Disturbances In this study with 20 patients, only 7 had FC values above 250.
My take: This study suggests that Lumacaftor/Ivacaftor is associated with a significant decrease in intestinal inflammation. When one looks at prior studies, the FC values presented in this study at baseline are much greater which could indicate significant selection bias.
Related blog posts:
- Big Advance in Cystic Fibrosis –Who Will Benefit?
- Lumacaftor-Ivacaftor for Cystic Fibrosis
- CFTR Modulators for Cystic Fibrosis
- FDA Expands Use of Cystic Fibrosis Medication | gutsandgrowth
- “Origins of Cystic Fibrosis Lung Disease”
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