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.
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