Precision Dosing of Anti-TNF Therapy in Pediatric Inflammatory Bowel Disease.

PURPOSE OF THE REVIEW: This review focuses on recent advancements in anti-TNF therapeutic drug monitoring (TDM), pharmacogenetics and personalized drug selection for children with inflammatory bowel disease (IBD). RECENT FINDINGS: Several real-world studies and one clinical trial in children have demonstrated that proactive TDM, targeting higher exposure concentrations (> 5 µg/mL), can improve disease remission rates and enhance durability of the anti-TNF biologics. Recent data from both adult and pediatric IBD patients have revealed an association between a genetic polymorphism (HLA-DQA1*05) and the development of auto-drug antibodies. The impact of this association on clinical outcomes, considering more routine use proactive TDM and dose optimization in children, is still under investigation. Additionally, recent studies have identified potential inflammatory signatures and biomarkers that may serve as companion diagnostics for anti-TNF biologics. The effective management of anti-TNF therapies in children with IBD requires evidence-based precision dosing strategies, including routine TDM and proactive pharmacodynamic assessments.

Racial Disparities in Pediatric Inflammatory Bowel Disease Care: Differences in Outcomes and Health Service Utilization Between Black and White Children.

OBJECTIVE: To describe racial inequities in pediatric inflammatory bowel disease care and explore potential drivers. METHODS: We undertook a single-center, comparative cohort study of newly diagnosed Black and non-Hispanic White patients with inflammatory bowel disease, aged <21 years, from January 2013 through 2020. Primary outcome was corticosteroid-free remission (CSFR) at 1 year. Other longitudinal outcomes included sustained CSFR, time to anti-tumor necrosis factor therapy, and evaluation of health service utilization. RESULTS: Among 519 children (89% White, 11% Black), 73% presented with Crohn's disease and 27% with ulcerative colitis. Disease phenotype did not differ by race. More patients from Black families had public insurance (58% vs 30%, P < .001). Black patients were less likely to achieve CSFR 1-year post diagnosis (OR: 0.52, 95% CI:0.3-0.9) and less likely to achieve sustained CSFR (OR: 0.48, 95% CI: 0.25-0.92). When adjusted by insurance type, differences by race to 1-year CSFR were no longer significant (aOR: 0.58; 95% CI: 0.33, 1.04; P = .07). Black patients were more likely to transition from remission to a worsened state, and less likely to transition to remission. We found no differences in biologic therapy utilization or surgical outcomes by race. Black patients had fewer gastroenterology clinic visits and 2-fold increased odds for emergency department visits. CONCLUSIONS: We observed no differences by race in phenotypic presentation and medication usage. Black patients had half the odds of achieving clinical remission, but a degree of this was mediated by insurance status. Understanding the cause of such differences will require further exploration of social determinants of health.

Yield and Costs of Evaluating Children With Cyclic Vomiting Syndrome.

BACKGROUND AND OBJECTIVES: Cyclic vomiting syndrome (CVS) clinical guidelines recommend an algorithm of basic testing for standard patients, and more targeted testing, including laboratory and imaging studies, in the presence of specific red flags. The cost-effectiveness of this screening of children with suspected CVS is lacking. The objectives of this study are to determine whether screening studies in CVS patients results in diagnostic change and to estimate their healthcare cost. METHOD: Charts of patients (1-18 years) with suspected CVS were retrospectively reviewed at a single center. Results and cost of laboratory and imaging studies were analyzed. RESULTS: A total of 503 charts were reviewed from electronic medical records with the International Classification of Diseases-9 (ICD-9) code 536.2 or search terms "CVS, cyclic vomiting, persistent emesis/vomiting, hyperemesis, or intractable/ periodic vomiting." Of these, 165 (33%) had a diagnosis of CVS and 135 (82%) children (mean age 7.7 ±â€Š4.3; 73 (54%) girls) met CVS criteria based on North American Society for Pediatric Gastroenterology, Hepatology and Nutrition diagnostic criteria. Of those meeting CVS criteria, 6 (4%) had a change in management based on the CVS screening evaluation. The mean cost of screening per patient that met CVS criteria was $6125.02 and the estimated total cost for all patients who met CVS criteria was $826,877.88. CONCLUSIONS: The screening metabolic laboratory results, pelvic ultrasound, magnetic resonance imaging, and upper endoscopy resulted in a diagnosis change in few patients screened for CVS. Most children who met criteria for CVS did not benefit from screening evaluation as results did not change clinical diagnosis or management, and were associated with higher cost.