Related Experiment Video
Updated: Oct 5, 2025
Laparoscopic Choledochal Cyst Excision and Roux-en-Y Choledochojejunostomy in Adults
Published on: February 28, 2025
Diagnosis of Primary Sclerosing Cholangitis Beyond Childhood is Associated with Worse Outcomes
Stefani Tica1, Saad Alghamdi2, Christopher Tait2
1Department of Pediatrics, Washington University in St. Louis, St. Louis Children's Hospital, St. Louis, MO, USA.
Insights
Adults with primary sclerosing cholangitis (PSC) experience worse outcomes than children, despite similar initial disease severity. Higher rates of portal hypertension and complications contribute to poorer prognosis in adult PSC patients.
Area of Science:
- Hepatology
- Gastroenterology
- Pediatric Gastroenterology
Background:
- Primary sclerosing cholangitis (PSC) affects both adults and children, but differences in disease progression and outcomes are not fully understood.
- Understanding these differences is crucial for tailoring clinical management and evaluating the applicability of adult clinical trial data to pediatric populations.
Purpose of the Study:
- To compare the clinical characteristics, disease severity, and outcomes of adult versus pediatric-onset primary sclerosing cholangitis (PSC).
- To identify factors influencing prognosis in different age groups diagnosed with PSC.
Main Methods:
- A retrospective analysis of PSC patients diagnosed between 2000-2013 at a single center.
- Comparison of demographic, clinical, and laboratory data between pediatric (0-18 years) and adult (19+ years) PSC cohorts.
- Definition of adverse outcomes including PSC-related death, liver transplant, or malignancy, and assessment of event-free survival.
Main Results:
- While initial liver disease severity markers were comparable, adult PSC patients exhibited higher incidences of adverse outcomes (P=0.02) and portal hypertension (PHT).
- Pediatric PSC patients (0-18 years) demonstrated significantly better event-free survival compared to those over 40 years (P=0.03).
- Adults had increased rates of biliary complications, ascites, variceal bleeding, and extra-hepatic comorbidities, with PHT adversely affecting outcomes in both groups.
Conclusions:
- Adult-onset PSC is associated with a worse prognosis compared to pediatric-onset PSC, even with similar initial disease biomarkers.
- Higher prevalence of PHT, biliary complications, extra-hepatic comorbidities, and longer follow-up durations in adults may explain the poorer outcomes.
- Findings suggest distinct disease trajectories and management considerations for adult and pediatric PSC patients.
Background:
The elucidation of differences between adult and pediatric-onset primary sclerosing cholangitis (PSC) may inform clinical decision making, and whether results of adult PSC clinical trials can be extrapolated to pediatric subjects.
Methods:
A single-center retrospective analysis of PSC subjects diagnosed during the epoch 2000-13 was conducted. Demographic, clinical, and laboratory data were compared between PSC subjects diagnosed between 0-18 (pediatric) and 19+ (adult) years of age. An adverse outcome was defined as PSC-related death, liver transplant, or malignancy. Survival without any of these was defined as event-free survival.
Results:
Analyses of 28 pediatric-diagnosed and 59 adult-diagnosed subjects revealed that incidence of early portal hypertension (PHT; P = 0.2), laboratory parameters of liver disease severity, and fibrosis grade at diagnosis were comparable between adult and pediatric PSC subjects. Adult-diagnosed PSC subjects had higher incidences of adverse outcomes compared to pediatric-diagnosed PSC subjects (P = 0.02). The age group 0-18 years (n = 30) had significantly better event-free survival compared to the age group more than 40 years (n = 25; P = 0.03). The prevalence of PHT in adult PSC subjects was 2.6 that of pediatric PSC subjects. PHT adversely affected outcomes in both adult (P < 0.001) and pediatric (P = 0.01) subjects. Adult PSC subjects were more likely to develop biliary complications (BCs; P = 0.001), ascites (P = 0.004), and variceal bleed (P = 0.03). Adult PSC subjects were more likely to have extra-hepatic co-morbidities (P < 0.001). Adult subjects had a longer follow-up duration compared to pediatric subjects (P = 0.06).
Conclusion:
Despite having a comparable clinical, laboratory, and histologic biomarkers of liver disease severity at the time of diagnosis, adult PSC subjects had a worse outcome compared to pediatric PSC subjects. Possible reasons for this finding include higher incidence of PHT, BCs, extra-hepatic co-morbidities, and longer duration of follow-up.
Related Concept Videos
01:29Chronic Pancreatitis II: Collaborative Care
Assessment:
01:17Chronic Bowel Disorders: Introduction
Irritable Bowel Syndrome (IBS) is a common disorder affecting the gastrointestinal tract. The distinctive feature is recurrent abdominal pain associated with altered bowel movements, manifesting as constipation, diarrhea, or fluctuating between both. The...
01:24Chronic Pancreatitis I: Introduction
Pancreatitis is the inflammation of the pancreas, which occurs when the immune system becomes active and causes swelling, pain, and disruptions in organ function. Pancreatitis can manifest as either an acute or chronic condition.
Acute pancreatitis arises suddenly and lasts for a brief duration, while chronic pancreatitis is a long-term affliction...
01:26Diseases of the Liver and Gallbladder
Cirrhosis is characterized by the scarring of hepatic lobules in the liver, which are replaced by fibrous tissue, affecting the liver's normal functioning. NAFLD, on the other hand, is caused by an excessive build-up of fat in the liver, not...
01:26Esophageal Strictures-II: Clinical Features and Management
Healthcare providers should gather a comprehensive medical history and conduct a physical examination for diagnosis. If esophageal stricture is...
01:22Effect of Hepatic Disease on Pharmacokinetics: Pathophysiologic Assessment and Liver Function Test