Number of Subjects in Study Archive: 135
Study Design: Observational
Conditions: Cystic Fibrosis, Liver Diseases, Liver Fibrosis
Division: DDN
Duration: March 2017 - May 2023
# Recruitment Centers: 8
Available Genotype Data: No
Image Summary: No
Transplant Type: None
Does it have dialysis patients: No
Clinical Trials URL:
https://clinicaltrials.gov/ct2/show/NCT03001388
Study Website: https://childrennetwork.org/Clinical-Studies
Data Package Version Number: 1 (October 7, 2024)
Noninvasive monitoring of liver fibrosis is an unmet critical need in cystic fibrosis (CF) liver disease. Liver biopsy is infrequently used in CF due to the patchy nature of liver involvement and is not suitable for longitudinal studies of progressive liver disease in CF. Indeed, unlike many other liver diseases, synthetic dysfunction is rare in CF liver disease. Portal hypertension due to hepatic fibrosis mediates most if not all of the complications seen in CF liver disease.
The Longitudinal Assessment of Transient Elastography in Cystic Fibrosis (ELASTIC-CF) study assessed if combining FibroScan® measurement of liver stiffness transient elastography with ultrasound would improve the prediction of the development of a nodular liver on ultrasound and development of portal hypertension over time in children and young adults with CF. Study visits occurred at baseline and for at least two annual follow-up visits.
This study is an ancillary study to the CFLD PUSH study. If requesting access to the ELASTIC-CF data, it is recommended to also request access to the CFLD PUSH data to obtain comprehensive information collected on study participants.
The specific aims and hypotheses for this study were:
The primary outcome measure was liver stiffness measurement (LSM) obtained via transient elastography using FibroScan. The secondary outcome measure was liver steatosis obtained via transient elastography and controlled attenuation parameter (CAP) to quantify liver steatosis.
Inclusion criteria:
Exclusion criteria:
The use of LSM and CAP has demonstrated excellent feasibility, reproducibility between two anatomical sites, and strong associations with biochemical and nodular ultrasound findings of liver disease. Most of the ELASTIC-CF participants (54.1%) had a normal (NL) ultrasound pattern, and 13.5%, 12.8%, and 19.5% had heterogenous (HTG), homogenous (HMG), and nodular (NOD) ultrasound patterns, respectively.
Participants with a NOD ultrasound pattern had a significantly higher median LSM compared with NL, HTG, and HMG patterns. Furthermore, the study further expands on a previously noted association between increased LSM and decreased platelet count among participants with CF. This study showed that LSM was associated with an increase in ultrasound spleen-size-for-age z-score (SSAZ) and decreased platelet count, suggesting that an elevated LSM may be used as a surrogate for portal hypertension. In addition, the study found that CAP was significantly associated with ultrasound patterns, consistent with previous reports. Median CAP in the HMG group was significantly higher and above the previously reported pediatric steatosis cutoff.