Outcome of Very Early Onset Inflammatory Bowel Disease Associated With Primary Sclerosing Cholangitis: A Multicenter Study From the Pediatric IBD Porto Group of ESPGHAN

Giulia Catassi; Giulia D'Arcangelo; Lorenzo Norsa; Matteo Bramuzzo; Iva Hojsak; Kaija-Leena Kolho; Claudio Romano; Marco Gasparetto; Angelo Di Giorgio; Seamus Hussey; Anat Yerushalmy-Feler; Dan Turner; Manar Matar; Batia Weiss; Anna Karoliny; Patrizia Alvisi; Christos Tzivinikos; Marina Aloi

doi:10.1093/ibd/izad218

Outcome of Very Early Onset Inflammatory Bowel Disease Associated With Primary Sclerosing Cholangitis: A Multicenter Study From the Pediatric IBD Porto Group of ESPGHAN

Inflamm Bowel Dis. 2023 Sep 28:izad218. doi: 10.1093/ibd/izad218. Online ahead of print.

Authors

Giulia Catassi¹, Giulia D'Arcangelo¹, Lorenzo Norsa², Matteo Bramuzzo³, Iva Hojsak⁴, Kaija-Leena Kolho⁵, Claudio Romano⁶, Marco Gasparetto⁷, Angelo Di Giorgio², Seamus Hussey⁸, Anat Yerushalmy-Feler⁹, Dan Turner¹⁰, Manar Matar¹¹, Batia Weiss¹², Anna Karoliny¹³, Patrizia Alvisi¹⁴, Christos Tzivinikos¹⁵, Marina Aloi¹

Affiliations

¹ Pediatric Gastroenterology and Liver Unit, Sapienza University of Rome, Umberto I Hospital, Rome, Italy.
² Pediatric Hepatology Gastroenterology and Transplantation, Papa Giovanni XXIII Hospital, Bergamo, Italy.
³ Institute for Maternal and Child Health, IRCCS "Burlo Garofolo," Trieste, Italy.
⁴ University Children's Hospital Zagreb, University of Zagreb Medical School, Zagreb, Croatia.
⁵ Children's Hospital, Helsinki University Hospital and University of Helsinki, Helsinki, Finland.
⁶ Pediatric Gastroenterology and Nutrition Unit, Edmond and Lily Safra Children's Hospital, Tel-Hashomer, Israel.
⁷ Department of Paediatric Gastroenterology, Barts Health Trust, The Royal London Children's Hospital, London, UK.
⁸ National Children's Research Centre, Royal College of Surgeons of Ireland and University College Dublin, Dublin, Ireland.
⁹ Pediatric Gastroenterology Institute "Dana-Dwek" Children's Hospital, Tel Aviv University, Tel Aviv, Israel.
¹⁰ Shaare Zedek Medical Center, the Hebrew University of Jerusalem, Israel.
¹¹ Institute of Gastroenterology, Nutrition and Liver Diseases, Schneider Children's Medical Center, Petach Tikva, Israel.
¹² Pediatric Gastroenterology and Cystic Fibrosis Unit, University of Messina, Messina, Italy.
¹³ Heim Pal National Pediatric Institute, Budapest, Hungary.
¹⁴ Pediatric Unit, Maggiore Hospital, Largo Bartolo Nigrisoli, 2, 40133 Bologna, Italy.
¹⁵ Department of Pediatric Gastroenterology, Al Jalila Children's Specialty Hospital, Dubai, United Arab Emirates.

PMID: 37768032
DOI: 10.1093/ibd/izad218

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.

Keywords: phenotype; primary sclerosing cholangitis; very early onset inflammatory bowel disease; very early onset primary sclerosing cholangitis.

Plain language summary

Very early onset primary sclerosing cholangitis associated with IBD (VEO-PSC-IBD) often presents with autoimmune features and shows a milder PSC disease course than later-onset disease. These findings highlight the significance of studying the distinctive genetic and pathophysiological factors specific to VEO disease.