RESUMO
BACKGROUND: Upadacitinib (UPA) is a novel selective JAK inhibitor approved for adults with ulcerative colitis (UC) and with positive phase 3 data for Crohn's disease (CD). Pediatric off-label use is common due to delays in pediatric approvals; real-world data on UPA are needed to understand the safety and effectiveness in pediatric IBD. METHODS: This is a single-center retrospective case series study of adolescents (12-17 years) with inflammatory bowel disease IBD on UPA. The primary outcome was postinduction steroid-free clinical remission (SF-CR) defined as Pediatric UC Activity Index (PUCAI) or Pediatric CD Activity Index (PCDAI) ≤10. Secondary outcomes include postinduction clinical response (decrease ≥12.5 in PUCAI/PCDAI), postinduction C-reactive protein (CRP) normalization, 6-month SF-CR, and intestinal ultrasound response and remission. Adverse events were recorded through last follow-up. RESULTS: Twenty patients (9 CD, 10 UC, 1 IBD-U; 55% female; median age 15 years, 90% ≥2 biologics) were treated with UPA for ≥12 weeks (median 51 [43-63] weeks). Upadacitinib was used as monotherapy in 55% and as combination with ustekinumab and vedolizumab in 35% and 10%, respectively. Week 12 SF-CR was achieved in 75% (15/20) and 80% (16/20) with CRP normalization. About 3/4 (14/19) achieved SF-CR at 6 months. Adverse event occurred in 2 patients (10%): Cytomegalovirus colitis requiring hospitalization and hyperlipidemia requiring no treatment. In the 75% with ultrasound monitoring, response and remission were achieved in 77% and 60%, respectively. CONCLUSION: While awaiting pediatric registration trials, our data suggest that UPA is effective in inducing and maintaining SF-CR in adolescents with highly-refractory IBD with an acceptable safety profile.
This case series presents novel data on the effectiveness and safety of upadacitinib in adolescent patients with IBD.
RESUMO
BACKGROUND AND AIMS: Inflammatory bowel disease (IBD) is a chronic inflammatory condition of the gastrointestinal tract that is associated with malnutrition. Malnutrition is associated with poor clinical outcomes in patients with IBD and therefore early identification of those at risk for malnutrition is crucial. We aimed to evaluate how frequently nutrition screening occurs in a large, tertiary care outpatient IBD center and to initiate an intervention to improve malnutrition screening for patients with IBD. METHODS: We used a traditional plan-do-study-act quality improvement technique to understand our current malnutrition screening practices and institute an intervention to improve screening. To do this, we utilized a modified Malnutrition Universal Screening Tool (mMUST) and integrated this into the electronic health record. We then evaluated the intervention and the impact on IBD related clinical outcomes. RESULTS: Prior to the intervention, few patients with IBD were screened for malnutrition. However, the number of patients screened for malnutrition significantly improved with the study intervention and those who were identified as high-risk had increased nutrition follow up including serum micronutrient evaluations and referral to a dedicated registered dietician. CONCLUSION: This study demonstrated the feasibility and impact of a malnutrition screening program in ambulatory IBD patients. Those patients identified as high risk for malnutrition who engaged in nutrition care had improved clinical outcomes including reduced hospitalizations and emergency room visits.
