Clinical Features and Natural History of Paediatric Patients with Ulcerative Proctitis: A Multicentre Study from the Paediatric IBD Porto Group of ESPGHAN

Author:

Tal Noa12,Tzivinikos Christos3,Gasparetto Marco4ORCID,Serban Daniela E5ORCID,Zifman Eyal6,Hojsak Iva7,Ledder Oren8ORCID,Yerushalmy Feler Anat29,Rolandsdotter Helena1011,Aloi Marina12ORCID,Bramuzzo Matteo13ORCID,Buderus Stephan14,Lionetti Paolo15ORCID,Norsa Lorenzo16ORCID,Norden Christoph17,Urlep Darja18ORCID,Romano Claudio19,Shaoul Ron20,Martinez-Vinson Christine21,Karoliny Anna22,De Greef Elisabeth23,Kang Ben24ORCID,VIčková Eva25,Alvisi Patrizia26,Kori Michal27,Tavares Marta28,Weiss Batia29,Hussey Seamus30ORCID,Qamhawi Maria E31,Palomino Pérez Laura M32,Henderson Paul33ORCID,Parmar Raj34,Miele Erasmo35,Rinawi Firas3637,Lozano-Ruf Ana38,Zamvar Veena39,Kolho Kaija-Leena4041ORCID,Shouval Dror S12ORCID

Affiliation:

1. Institute of Gastroenterology, Nutrition and Liver Disease, Schneider Children’s Medical Center , Petah Tikva , Israel

2. Faculty of Medicine, Tel Aviv University , Tel Aviv , Israel

3. Paediatric Gastroenterology Department, Al Jalila Children’s Specialty Hospital, Mohammed Bin Rashid University , Dubai , United Arab Emirates

4. Barts Health NHS Trust, The Royal London Children’s Hospital, Department of Paediatric Gastroenterology, Queen Mary University of London, Centre for Immunobiology, Blizard Institute , London , UK

5. 2nd Clinic of Pediatrics, ‘Iuliu Hatieganu’ University of Medicine and Pharmacy, Emergency Clinical Hospital for Children , Cluj-Napoca , Romania

6. Pediatric Gastroenterology Unit, Meir Medical Center , Kfar-Saba , Israel

7. Referral Center for Pediatric Gastroenterology and Nutrition, Children’s Hospital Zagreb , Zagreb , Croatia

8. The Juliet Keidan Institute of Pediatric Gastroenterology & Nutrition, Shaare Zedek Medical Center, The Hebrew University of Jerusalem , Jerusalem , Israel

9. Pediatric Gastroenterology Institute, ‘Dana-Dwek’ Children’s Hospital, Tel Aviv Sourasky Medical Center , Tel Aviv , Israel

10. Department of Clinical Science and Education, Södersjukhuset, Karolinska Institutet , Stockholm , Sweden

11. Sachs’ Children and Youth Hospital, Department of Gastroenterology, Södersjukhuset , Stockholm , Sweden

12. Pediatric Gastroenterology, Hepatology and Nutrition Institute, Sapienza University of Rome , Rome , Italy

13. Institute for Maternal and Child Health, IRCCS ‘Burlo Garofolo’ , Trieste , Italy

14. Department of Pediatrics, St-Marien-Hospital , Bonn , Germany

15. Department NEUROFARBA, University of Florence, Meyer Children’s Hospital , Florence , Italy

16. Pediatric Hepatology, Gastroenterology and Transplantation Unit, ASST Papa Giovanni XXIII , Bergamo , Italy

17. Department of Pediatrics, Hvidovre University Hospital , Copenhagen, Hvidovre , Denmark

18. Pediatric Gastroenterology and Liver Unit, University Children’s Hospital of the University Medical Centre Ljubljana , Ljubljana , Slovenia

19. Pediatric Gastroenterology and Cystic Fibrosis Unit, Department of Human Pathology in Adulthood and Childhood ‘G. Barresi’, University of Messina , Messina , Italy

20. Pediatric Gastroenterology and Nutrition Institute, Ruth Children’s Hospital of Haifa, Rambam Medical Center, Faculty of Medicine, Technion , Haifa , Israel

21. Service de Gastroentérologie et Nutrition Pédiatriques, Hôpital Universitaire Robert-Debré , Paris , France

22. Heim Pál National Pediatric Institute , Budapest , Hungary

23. Department of Paediatric Gastroenterology and Nutrition, Kidz Health Castle UZ Brussels, Free University Brussels , Brussels , Belgium

24. Department of Pediatrics, School of Medicine, Kyungpook National University , Daegu , Korea

25. Department of Pediatrics, 2nd Medical Faculty, Charles University and University Hospital Motol , Prague , Czech Republic

26. Pediatric Gastroenterology Unit, Maggiore Hospital , Bologna , Italy

27. Pdiatric Gastroenterology, Kaplan Medical Centre, Rehovot and the Faculty of Medicine, Hebrew University of Jerusalem , Jerusalem , Israel

28. Department of Pediatric Gastroenterology, Centro Materno Infantil do Norte, Centro Hospitalar e Universitário de Porto , Porto , Portugal

29. Division of Pediatric Gastroenterology and Nutrition, Edmond and Lily Safra Children’s Hospital , Ramat Gan , Israel

30. Children’s Health Ireland, UCD and RCSI , Dublin , Ireland

31. Department of Gastroenterology, Hepatology and Nutrition, Astrid Lindgren Children’s Hospital, Karolinska University Hospital , Stockholm Sweden

32. Gastroenterology and Nutrition Department, Hospital Infantil Universitario Niño Jesús , Madrid , Spain

33. Department of Paediatric Gastroenterology and Nutrition, Royal Hospital for Children and Young People , Edinburgh , UK

34. Department of Pediatric Gastroenterology, Great North Children’s Hospital , Newcastle , UK

35. Department of Translational Medical Science, Section of Pediatrics, University of Naples ‘Federico II’ , Naples , Italy

36. Pediatric Gastroenterology Unit, Emek Medical Center , Afula , Israel

37. The Ruth and Bruce Rappaport Faculty of Medicine, Technion – Israel Institute of Technology , Haifa , Israel

38. Department of Pediatric Gastroenterology, Hepatology and Nutrition, Hospital Sant Joan de Déu , Barcelona , Spain

39. Department of Paediatric Gastroenterology, Leeds Children’s Hospital, Leeds Teaching Hospitals NHS Trust , Leeds , UK

40. Children’s Hospital, University of Helsinki , Helsinki , Finland

41. Faculty of Medicine and Health Technology, University of Tampere , Tampere , Finland

Abstract

Abstract Background and Aims Ulcerative proctitis [UP] is an uncommon presentation in paediatric patients with ulcerative colitis. We aimed to characterize the clinical features and natural history of UP in children, and to identify predictors of poor outcomes. Methods This was a retrospective study involving 37 sites affiliated with the IBD Porto Group of ESPGHAN. Data were collected from patients aged <18 years diagnosed with UP between January 1, 2016 and December 31, 2020. Results We identified 196 patients with UP (median age at diagnosis 14.6 years [interquartile range, IQR 12.5–16.0]), with a median follow-up of 2.7 years [IQR 1.7–3.8]. The most common presenting symptoms were bloody stools [95%], abdominal pain [61%] and diarrhoea [47%]. At diagnosis, the median paediatric ulcerative colitis activity index [PUCAI] score was 25 [IQR 20–35], but most patients exhibited moderate–severe endoscopic inflammation. By the end of induction, 5-aminosalicylic acid administration orally, topically or both resulted in clinical remission rates of 48%, 48%, and 73%, respectively. The rates of treatment escalation to biologics at 1, 3, and 5 years were 10%, 22%, and 43%, respectively. In multivariate analysis, the PUCAI score at diagnosis was significantly associated with initiation of systemic steroids, or biologics, and subsequent acute severe colitis events and inflammatory bowel disease-associated admission, with a score ≥35 providing an increased risk for poor outcomes. By the end of follow-up, 3.1% of patients underwent colectomy. Patients with UP that experienced proximal disease progression during follow-up [48%] had significantly higher rates of a caecal patch at diagnosis and higher PUCAI score by the end of induction, compared to those without progression. Conclusion Paediatric patients with UP exhibit high rates of treatment escalation and proximal disease extension.

Publisher

Oxford University Press (OUP)

Subject

Gastroenterology,General Medicine

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