Preliminary results of the registry of european children treated with teduglutide

Introduction: Teduglutide is now an available treatment in Europe to improve intestinal absorption for children with short bowel syndrome (SBS). The extent of response of children to teduglutide and, and the profile of candidates expected to respond have not been clarified yet. For this reason, we established a European registry on the real-life use of teduglutide with the aim of measuring treatment response and predictors of response, safety and adverse events and cost effectiveness of the drug. Methods: A multi-center, observational, longitudinal, retrospective and prospective cohort study was designed. A registry was created in order to collect all data from previously treated patients and enrolling prospectively children starting teduglutide from June 2021. Response to treatment was analized based on different parameters. Results: Children enrolled in the registry are currently 73 from six countries in Europe. We were able to perform analysis on 69/72 who reached a minimum of 6 months treatments. Median age at Teduglutide start was 5.4 years old (IQR: 3, 9). More than a half (64%) were preterm. The most preminent coause of SBS was necrotizing enterocolitis (NEC) (27.5%). The median length of residual small bowel was 26 cm (IQR: 14, 55), 16% had a class I SBS, 59% class II, and 24% class III. Ending stoma was present in a minority of patients 19%. Median parenteral nutrition dependency index (PNDI) was 60% before starting treatment. Reduction of at least 20%, 50% or 75% of PNDI was achieved in 73%, 46% and 33% respectively. The analysis on growth pattern was performed on 46 (67%) patients which achieved the 12-month follow-up. Patients achieving a sustained response of over 75% of PN reduction were 19 (41%) and showed a mean increase of 7.8% of body weight after 3 months of treatment, 14% after 6 months, 23.7% after 9 month and 29.7% after 12 months of treatment, which were significantly higher (p: 0.035) than their non responder pairs (figure 1). When considering a lower response rate of > 20% or >50% PN no significant weight increase difference was observed. Conclusions: Teduglutide is an effective treatment for children with SBS with nearly one third of patients achieving a stable reduction of more than 75% of PNDI. Children with SBS on home PN display an important weight for age retardation before starting Teduglutide treatment. Children achieving an almost complete PN weaning after Teduglutide show a higher weight gain despite a lowering PN support in the first year after treatment. Monitoring weight gain during the first year on Teduglutide treatment may help identifying children which will most benefit from Teduglutide treatment.