Pediatric Short Bowel Syndrome: Real-World Evidence on Incidence and Hospital Resource Use From a Finnish Data Lake.

OBJECTIVES: Little is known about the epidemiology and healthcare burden of pediatric intestinal failure (IF). We aimed to assess the incidence, prevalence, healthcare resource utilization (HCRU), and related costs of pediatric short bowel syndrome (SBS) using follow-up data from the largest hospital district in Finland. METHODS: This retrospective registry study utilized electronic healthcare data covering all pediatric patients with SBS-IF born between 2010 and 2019 at the Hospital District of Helsinki and Uusimaa in Finland. Patients were followed from birth until the end of 2020 and compared to control patients, all from the same hospital system. RESULTS: In total, 38 patients with SBS-IF and 1:5 matched controls were included, with median follow-up time of almost 6 years from birth. Over half of the patients were born early preterm (gestational age ≤30 weeks). The incidence of pediatric SBS-IF was 24 per 100,000 live births. The HCRU was higher compared to controls and most of the inpatient days incurred during the first year of the SBS-IF patients' life. The average hospital-based HCRU costs were €221,000 for the first year and €57,000 for whole follow-up annually. The costs were higher for the early preterm patients and accumulated mainly from inpatient days. CONCLUSIONS: SBS-IF is a rare disease with a relatively low number of patients treated at each hospital district. The burden on the hospital system, as well as the patient's family, is especially high at the onset as the newborns with SBS-IF spend a significant part of their first year of life in the hospital.

Dose-escalation of adalimumab, golimumab or ustekinumab in inflammatory bowel diseases: characterization and implications in real-life clinical practice.

OBJECTIVES: Dose-escalation is a common practice to optimize treatment with subcutaneously administered biologicals in Crohn's disease (CD) and ulcerative colitis (UC). However, limited data is available on the extent of dose-escalation in real-life. Here, we analyzed treatment persistence, dose-escalation, concomitant corticosteroid use, and costs of adalimumab, golimumab, and ustekinumab in inflammatory bowel diseases (IBD). METHODS: This was a nationwide, retrospective, non-interventional registry study. All adult patients who were diagnosed with CD or UC and had purchased adalimumab, golimumab, or ustekinumab from Finnish pharmacies between 2008 and 2018 were included in the study and followed up for 24 months after treatment initiation. RESULTS: A total of 2884 patients were included in the analyses. For adalimumab, treatment persistence was higher for CD patients compared to UC patients both at months 12 (46.2% versus 37.1%; p < .0001) and 24 (26.1% versus 19.7%; p < 0.0001). For golimumab (UC), treatment persistence was 48.3% at month 12 and 28.1% at month 24. The 12-month treatment persistence rate for patients on ustekinumab (CD) was 47.1%. Cumulative doses exceeding the regular dosing according to the summary of product characteristics (SPC), was observed for adalimumab in CD during the first 6 months of treatment (62.9% of the treatment periods), golimumab in the later stages of the UC treatment (52-54% of treatment periods at months 7-24), and ustekinumab during the first 6 months (70.7%). CONCLUSIONS: Based on this study, dose-escalation of subcutaneously administered biologicals is a common clinical practice in IBD. This has implications for treatment costs, use of concomitant medications, and treatment outcomes.