RESUMEN
BACKGROUND: Abdominal pain is a common and often debilitating issue for children and adolescents. In many cases, it is not caused by a specific somatic condition but rather emerges from a complex interplay of bio-psycho-social factors, leading to functional abdominal pain (FAP). Given the complex nature of FAP, understanding its origins and how to effectively manage this condition is crucial. Until now, however, no questionnaire exists that targets knowledge in this specific domain. To address this, the Abdominal Pain Knowledge Questionnaire (A-PKQ) was developed. METHODS: Two versions were created (one for children and one for parents) and tested in four gastroenterology clinics and one specialized pain clinic in Germany between November 2021 and February 2024. Children between 8 and 17 years of age (N = 128) and their accompanying parents (N = 131) participated in the study. Rasch analysis was used to test the performance of both versions of the questionnaire. RESULTS: The original questionnaires exhibited good model and item fit. Subsequently, both questionnaires were refined to improve usability, resulting in final versions containing 10 items each. These final versions also demonstrated good model and item fit, with items assessing a variety of relevant domains. CONCLUSION: The A-PKQ is an important contribution to improving assessment in clinical trials focused on pediatric functional abdominal pain.
RESUMEN
Ustekinumab is an effective therapy for adult Crohn's disease (CD), but data in paediatric CD patients are scarce. The aim of the study was to describe the real-life effectiveness and safety of ustekinumab in paediatric CD. This is a multicentre review of children with Crohn's disease treated with ustekinumab. The aim of our study was to describe the effectiveness and safety of ustekinumab in paediatric real-life practice. This is a study of the Paediatric IBD (inflammatory bowel disease) Porto group of ESPGHAN. Corticosteroid (CS)- and exclusive enteral nutrition (EEN)-free remission, defined as weighted Paediatric Crohn's Disease Activity Index (wPCDAI) < 12.5, and physician global assessment (PGA) were determined at weeks 12 and 52. A total of 101 children were included at a median age of 15.4 years (IQR 12.7-17.2) with a median follow-up of 7.4 months (IQR 5.6-11.8). Ninety-nine percent had received prior anti-TNF, 63% ≥ 2 anti-TNFα therapies and 22% vedolizumab. Baseline median wPCDAI was 39 (IQR 25-57.5) (71 (70%) patients with moderate-severe activity). Weeks 12 and 52 CS- and EEN-free remission were both 40.5%. Clinical response at week 6, iv induction route and older age at onset of ustekinumab treatment were predictive factors associated with clinical remission at week 12. Seven minor adverse events probably related to ustekinumab were reported. One patient died from an unrelated cause. Conclusion: Our results suggest that ustekinumab is effective and safe in children with chronically active or refractory CD. What is Known: ⢠Ustekinumab is an effective therapy for adult moderate to severe Crohn's disease (CD). ⢠Off-label use of ustekinumab in children is increasing especially in anti-TNF refractory CD. What is New: ⢠Is the largest cohort of real-world use of ustekinumab in paediatric CD to date. ⢠Clinical response at week 6, iv induction and older age at onset of ustekinumab were predictive factors associated with clinical response at week 12.