4CPS-096 Teduglutide versus home parenteral nutrition in paediatric population with short bowel syndrome

<h3>Background and Importance</h3> Long-term home parenteral nutrition(HPN) in paediatric population with short bowel syndrome (SBS), which incidence is estimated to be 24.5 per 100,000 live births, has significant survival improvement, but it is associated with complications and impact on life quality. Teduglutide may decrease HPN support and achieve enteral autonomy(EA) by accelerating intestinal adaptation. <h3>Aim and Objectives</h3> To evaluate effectiveness and incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) gained of teduglutide plus HPN versus HPN in paediatric patients with SBS using healthcare system perspective. <h3>Material and Methods</h3> Observational, descriptive and retrospective study from May 2018 to August 2024 in children(neonates-16 years) with SBS receiving HPN and teduglutide in a tertiary care hospital. Data collected from the electronic medical records and logistics management programme: age; sex, weight; teduglutide: start/end date, posology, annual acquisition cost/patient with and without repackaging; HPN: start/end date, units/week 12 months prior to teduglutide initiation(PRE-TEDU) and last 12 months with teduglutide(POST-TEDU), annual acquisition cost/patient. QALYs and direct non-acquisition costs(drug complications, medical visits and intestinal transplantation) were obtained from <i>Gattini et al.</i>¹ Quantitative variables were expressed as median and range, and qualitative variables as number and percentage(%). <h3>Results</h3> During the study period, 6 patients were included; 3(50%) female; median age 8.50(4–16) years, median weight 21(17–51)kg. Teduglutide: first dose 4.71(2.04–8.80)years from HPN onset; median duration 3.99(0.49–6.26) years; median dose 0.05 (0.02–0.05) mg/kg/day; median annual direct cost/patient 35,020€(17,829€-131,586€) with repackaging and 216,899€ (51,643€-234,974€) without repackaging. Five(83.33%) patients continued with treatment. In effectiveness terms, median reduction in HPN number POST-TEDU versus PRE-TEDU was 225(0–365); 2 patients (33%) achieved EA POST-TEDU. The incremental cost involved 23,805€ (−16,293€-81,966€) and 187,668€ (51,643€-191,879€) with and without repackaging in POST-TEDU period. In cost-utility analysis, using an additional 1.80(1.70–1.89) QALY, an ICER of 13,225€(−9,052€-45,537€)/QALY and 104,260€(28,691–108,038€)/QALY with and without repackaging was obtained. <h3>Conclusion and Relevance</h3> Compared to HPN, repackaged teduglutide allows EA to be achieved with a cost-effectiveness criterion below the threshold of 30,000€/QALY gained, starting at least 2 years after PN initiation. Future studies with larger sample size and utility national data would be necessary. <h3>References and/or Acknowledgements</h3> https://doi.org/10.1016/j.clnu.2023.10.001 <h3>Conflict of Interest</h3> No conflict of interest