Book Description

The objective of this thesis was to evaluate the effect of implementation of a multidisciplinary intestinal rehabilitation program (IRP) on clinical outcomes of pediatric intestinal failure (IF). This was attained by two comprehensive reviews and three related research projects that contribute both clinical and methodological knowledge. Pediatric IF is a complex medical condition. Advances have been made clinically in recent years, but research in the field has suffered from methodological challenges and poor quality. Many of these were outlined and solutions suggested in this thesis. The first project compared clinical outcomes of three eras of pediatric IF-management using univariate categorical and time-to-event analysis. While IRP-introduction led to an increased transplantation rate and improved clinical outcomes, further progress was made and the natural course of the disease changed with IRP-maturation and adoption of novel treatment strategies. The second project quantified the continuous change over time of pediatric IF over a 15-year time period and tested systematically introduced treatments using novel methods. Multivariate time series analysis was applied to assess the relationship between new treatment options and change in clinical outcomes. The study showed that IRP-introduction and omega-3 lipid emulsions independently decreased disease-specific mortality from liver failure and sepsis when adjusted for disease severity, while serial transverse enteroplasty and ethanol locks did not impact mortality significantly. With improved mortality of pediatric IF, assessment of morbidity became imperative. After having identified a severe lack in methodology to measure neonatal and infant health-related quality of life (NIHRQOL) in a systematic literature review, the conceptual meaning of NIHRQL was explored in the final project. It concluded that NIHRQOL is a multidimensional, multilayered and interconnected concept, in which the childâ s needs are fundamental factors, and the caregiverâ s and societyâ s ability to meet those needs characterize the interdependence between the child and its caregiving environment. In summary, the complexity of pediatric IF and related methodological challenges were identified. Methodological solutions were described and illustrated. Clinical advancements of pediatric IF were quantified, and IRP-introduction and omega-3 lipid emulsions recognized as strongest contributors. Finally, the meaning of NIHRQOL was explored and organized in a conceptual framework in this thesis.