P210 Long-term outcome of very early onset inflammatory bowel disease associated with primary sclerosing cholangitis: a multicenter study from the Pediatric IBD Porto Group of ESPGHAN
Catassi, G.(1);D'Arcangelo, G.(2);Norsa, L.(3);Bramuzzo, M.(4);Hojsak, I.(5);Kolho, K.L.(6);Romano, C.(7);Gasparetto, M.(8);Di Giorgio, A.(3);Hussey, S.(9);Yerushalmy-Feler, A.(10);Turner, D.(11);Matar, M.(12); Vais, B.(13);Karoliny, A.(14);Alvisi, P.(15);Tzivinikos, C.(16);Aloi, M.(2)*;
(1)SapienzaUniversity of Rome, Pediatric Gastroenterology Unit, Rome, Italy;(2)Sapienza University of Rome, Pediatric Gastroenterology Unit, Rome, Italy;(3)Papa Giovanni XXIII Hospital, Paediatric Hepatology Gastroenterology and Transplantation, Bergamo, Italy;(4)IRCCS “Burlo Garofolo”, Institute for Maternal and Child Health, Trieste, Italy;(5)University Children’s Hospital Zagreb- University of Zagreb Medical School, Pediatric Gastroenterology Unit, Zagreb, Croatia;(6)5Children’s Hospital- Helsinki University Hospital and University of Helsinki, Pediatric Gastroenterology Unit, Helsinki, Finland;(7)University of Messina, Pediatric Gastroenterology and Cystic Fibrosis Unit, Messina, Italy;(8)The Royal London Children’s Hospital- London- UK. Queen Mary University of London- Blizard Institute- Centre for Immunobiology, Pediatric Gastroenterology, London, United Kingdom;(9)National Children’s Research Centre- Royal College of Surgeons of Ireland and University College Dublin, Pediatric Gastroenterology Unit, Dublin, Ireland;(10)Institute “Dana-Dwek” Children’s Hospital- Tel Aviv University, Pediatric Gastroenterology Unit, Tel Aviv, Israel;(11)Shaare Zedek Medical Center- the Hebrew University of Jerusalem, Pediatric Gastroenterology Unit, Jerusalem, Israel;(12)Sheba Medical Center- Tel Aviv University, Pediatric Gastroenterology Unit, Tel Aviv, Israel;(13)Edmond and Lily Safra Children’s Hospital, Pediatric Gastroenterology and Nutrition Unit, Tel-Hashomer, Israel;(14)Heim Pal National Pediatric Institute, Pediatric Gastroenterology Unit, Budapest, Hungary;(15)Maggiore Hospital, Pediatric Gastroenterology Unit, Bologna, Italy;(16)Al Jalila Children’s Specialty Hospital, Pediatric Gastroenterology Unit, Dubai, United Arab Emirates;
Background
Primary sclerosing cholangitis related to inflammatory bowel disease (PSC-IBD) diagnosed under the age of 6 years (i.e., VEO-IBD) may have unique characteristics and disease course. We aimed to analyze the characteristics and natural history of children with VEO PSC-IBD and compare them to children diagnosed with PSC-IBD at an older age.
Methods
This was a multicenter, retrospective, study evaluating patients diagnosed with both IBD and PSC before (VEO-PSC-IBD) or after the age of 6 years (PSC-IBD), followed at 14 centers affiliated with the Porto IBD Interest group of ESPGHAN. Demographic, clinical, laboratory, endoscopic, and imaging data were collected at baseline and every six months thereafter for a minimum follow-up of 12 months. IBD-related (clinical remission, need for systemic steroids, therapy escalation and surgery) and PSC-related outcomes (biliary and portal hypertensive complications, need for treatment escalation and liver transplantation, cholangiocarcinoma, and death) were analyzed at 1, 3, and 5 years.
Results
A total of 69 children with both IBD and PSC were included: 28 with VEO PSC-IBD [median age 5.2 (3.7-5.9) years, 21 UC (75%), 4 IBDU (14%) and 3 (15%) CD] and 41 with PSC-IBD [median age 15.7 (13.3-16.9) years, 34 UC (83%), 6 IBDU (1.5%) and 1 (0.2%) CD]. IBD was diagnosed prior to PSC in 28 of the 69 patients (40%), simultaneously in 30 (43%), and after PSC in 11 (16%), with no significant differences between VEO PSC-IBD and PSC-IBD. Most patients with UC presented with extensive disease at diagnosis (89% in VEO PSC-UC vs. 89% in PSC-UC, p = 0.72). Both groups presented most often with mild intestinal disease at diagnosis (mean PUCAI of VEO IBD-PSC 34±16, vs 31±19 of IBD-PSC, p=0.11; mean PCDAI 31±33 vs. 21±27, respectively, p=0.14). A higher number of VEO-IBD-PSC patients were diagnosed with autoimmune sclerosing cholangitis than older children [24 (86%) vs. 27 (66%) PSC-IBD, p=0.04], whereas no other differences were found for PSC-related variables. During follow-up, no significant differences were found in major IBD outcomes. The risk of developing biliary strictures and escalating therapy to vancomycin from ursodeoxycholic acid was lower in the VEO-PSC-IBD group (Figure 1 and 2, Log-rank p=0.02 and p=0.02), while no difference was found for portal hypertension and liver transplantation at 5-year follow-up. No cases of cholangiocarcinoma or death were reported.
Conclusion
IBD-PSC has similar baseline characteristics whether diagnosed as VEO-IBD or thereafter. However, a milder disease course in terms of biliary complications and the need for PSC-related therapy escalation, with an overall mild intestinal disease at presentation in all patients, characterize this specific subcohort of patients.
