Inflamm Bowel Dis. 2024 Oct 3;30(10):1662-1669.doi: 10.1093/ibd/izad218.

Giulia Catassi ¹, Giulia D'Arcangelo ¹, Lorenzo Norsa ², Matteo Bramuzzo ³, Iva Hojsak ⁴, Kaija-Leena Kolho ⁵, Claudio Romano ⁶, Marco Gasparetto ⁷, Angelo Di Giorgio ², Seamus Hussey ⁸, Anat Yerushalmy-Feler ⁹, Dan Turner ¹⁰, Manar Matar ¹¹, Batia Weiss ⁶, Anna Karoliny ¹², Patrizia Alvisi ¹³, Christos Tzivinikos ¹⁴, Marina Aloi ¹

Author information

¹Pediatric Gastroenterology and Liver Unit, Sapienza University of Rome, Umberto I Hospital, Rome, Italy.

²Pediatric Hepatology Gastroenterology and Transplantation, Papa Giovanni XXIII Hospital, Bergamo, Italy.

³Institute for Maternal and Child Health, IRCCS "Burlo Garofolo," Trieste, Italy.

⁴University Children's Hospital Zagreb, University of Zagreb Medical School, Zagreb, Croatia.

⁵Children's Hospital, Helsinki University Hospital and University of Helsinki, Helsinki, Finland.

⁶Pediatric Gastroenterology and Cystic Fibrosis Unit, University of Messina, Messina, Italy.

⁷Department of Paediatric Gastroenterology, Barts Health Trust, The Royal London Children's Hospital, London, UK.

⁸National Children's Research Centre, Royal College of Surgeons of Ireland and University College Dublin, Dublin, Ireland.

⁹Pediatric Gastroenterology Institute "Dana-Dwek" Children's Hospital, Tel Aviv University, Tel Aviv, Israel.

¹⁰Shaare Zedek Medical Center, the Hebrew University of Jerusalem, Israel.

¹¹Institute of Gastroenterology, Nutrition and Liver Diseases, Schneider Children's Medical Center, Petach Tikva, Israel.

¹²Heim Pal National Pediatric Institute, Budapest, Hungary.

¹³Pediatric Unit, Maggiore Hospital, Largo Bartolo Nigrisoli, 2, 40133 Bologna, Italy.

¹⁴Department of Pediatric Gastroenterology, Al Jalila Children's Specialty Hospital, Dubai, United Arab Emirates.

Abstract

Background: Whether primary sclerosing cholangitis related to inflammatory bowel disease (PSC-IBD) diagnosed before 6 years (ie, VEO-IBD) has a distinct phenotype and disease course is uninvestigated. We aimed to analyze the characteristics and natural history of VEO-PSC-IBD, compared with early and adolescent-onset PSC-IBD.

Methods: This is a multicenter, retrospective, case-control study from 15 centers affiliated with the Porto and Interest IBD group of ESPGHAN. Demographic, clinical, laboratory, endoscopic, and imaging data were collected at baseline and every 6 months. Inflammatory bowel disease-related (clinical remission, need for systemic steroids and biologics, and surgery) and PSC-related (biliary and portal hypertensive complications, need for treatment escalation and liver transplantation, cholangiocarcinoma, or death) outcomes were compared between the 2 groups.

Results: Sixty-nine children were included, with a median follow-up of 3.63 years (interquartile range, 1-11): 28 with VEO-PSC-IBD (23 UC [82%], 2 IBD-U [7%] and 3 [11%] CD), and 41 with PSC-IBD (37 UC [90%], 3 IBDU [7.5%] and 1 [2.5%] CD). Most patients with UC presented with pancolitis (92% in VEO-PSC-UC vs 85% in PSC-UC, P = .2). A higher number of patients with VEO-PSC-IBD were diagnosed with PSC/autoimmune hepatitis overlap syndrome than older children (24 [92%] vs 27 [67.5%] PSC-IBD, P = .03), whereas no other differences were found for PSC-related variables. Time to biliary strictures and infective cholangitis was lower in the VEO-PSC-IBD group (P = .01 and P = .04, respectively), while no difference was found for other outcomes. No cases of cholangiocarcinoma were reported.

Conclusions: Primary sclerosing cholangitis related to inflammatory bowel disease has similar baseline characteristics whether diagnosed as VEO-IBD or thereafter. A milder disease course in terms of biliary complications characterizes VEO-PSC-IBD.