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Gamma-glutamyl transferase and disease course in pediatric-onset primary sclerosing cholangitis: A single-center
Besrat Berhane1, Patrick F van Rheenen1, Henkjan J Verkade1
1Pediatric Gastroenterology & Hepatology, Department of Pediatrics, Beatrix Children's Hospital, University Medical Center Groningen University of Groningen Groningen The Netherlands.
Insights
A significant decrease in gamma-glutamyl transferase (GGT) levels within one year of diagnosis in pediatric-onset primary sclerosing cholangitis (PSC) patients is linked to fewer complications. This finding supports GGT as a predictive marker for the disease course in pediatric PSC.
Area of Science:
- Hepatology
- Pediatric Gastroenterology
- Biomarker Research
Background:
- Pediatric-onset primary sclerosing cholangitis (PSC) carries a risk of long-term hepatic complications, with liver transplantation as the only cure.
- Predictive markers are needed to forecast the clinical course of pediatric PSC, as complications often manifest over many years.
- Gamma-glutamyl transferase (GGT) has emerged as a potential surrogate marker for predicting the disease trajectory in PSC.
Purpose of the Study:
- To evaluate the predictive capability of gamma-glutamyl transferase (GGT) levels in forecasting the clinical course of pediatric-onset primary sclerosing cholangitis (PSC).
- To determine if a decrease in GGT levels within the first year of diagnosis is associated with a reduced incidence of complications in pediatric PSC patients.
Main Methods:
- A retrospective single-center cohort study analyzed longitudinal data from pediatric PSC patients diagnosed between 2000 and 2020.
- Patients were categorized based on GGT decrease from baseline: >25% (n=36) versus ≤25% (n=7).
- Kaplan-Meier analysis and log-rank testing assessed the occurrence of portal hypertensive/biliary complications, malignancies, liver transplantation, or death.
Main Results:
- The group with a >25% GGT decrease showed a significantly lower probability of developing complications within 5 years post-diagnosis (20%) compared to the ≤25% decrease group (50%) (p=0.031).
- Median age at diagnosis was comparable between groups (12.1 years for >25% GGT decrease vs. 15.2 years for ≤25% GGT decrease; p=0.078).
- Medication use did not correlate with the development of complications.
Conclusions:
- A GGT decrease exceeding 25% within one year of diagnosis in pediatric-onset PSC is associated with a reduced risk of complications within the subsequent five years.
- These findings reinforce the hypothesis that early changes in GGT levels serve as a valuable predictive indicator for the disease progression in pediatric PSC.
- The study highlights the potential utility of monitoring GGT trends for better patient management and risk stratification in pediatric PSC.
Background And Aims:
Patients with pediatric-onset primary sclerosing cholangitis (PSC) are at risk of developing hepatic complications with liver transplantation as only curative treatment. Complications usually occur over many years, underlining the need for reliable surrogate markers to predict the clinical course. Recently, gamma-glutamyl transferase (GGT) has been suggested to allow prediction of the clinical course. In a single-center cohort study, we tested the potency of GGT in this respect.
Methods:
We used longitudinal data of patients from our academic center, diagnosed with pediatric-onset PSC between 2000 and 2020. Patients with a GGT decrease from baseline >25% (n = 36) were compared with those who did not have this decrease (n = 7). We performed Kaplan-Meier analysis and log-rank testing to assess the occurrence of portal hypertensive or biliary complications, hepatobiliary malignancies, liver transplantation, or death.
Results:
The median age diagnosis was 15.2 years and 12.1 years in the group with ≤25% decrease of GGT and the group with >25% decrease, respectively (p = 0.078). The probability of developing ≥1 complications in the first 5 years after diagnosis was 50% in the group with ≤25% decrease of GGT and 20% in the group with >25% decrease of GGT (p = 0.031). The use of medication was not associated with the development of complications.
Conclusion:
In a retrospective cohort study, we report that a GGT decrease of >25% within 1 year of diagnosis of pediatric-onset PSC is associated with a lower occurrence of complications within 5 years. Our results provide further support for the recently hypothesized predictive value of first-year GGT change in predicting the disease course in pediatric-onset PSC.
