Forty-eight months outcomes of teduglutide treatment in adult stable patients with short bowel syndrome and home parenteral nutrition dependence: A real-world Italian single-center observational cohort study

Aim of the study

This real-life study is designed to investigate the short and long-term efficacy and safety of teduglutide (TED) and its effects on the quality of life (QoL) in a cohort of adult, stable patients with short bowel syndrome and chronic intestinal failure receiving long-term parenteral support (PS).

Patient and Methods

A prospective, single-center study was conducted for individuals who began to take TED between March 2017 and August 2023.

Results

Ten patients were included in the analysis, among whom the median duration of TED administration was 48 (range, 12–71) months. Data relative to short-term clinical outcomes demonstrate that 4 of 10 (40%) patients were early responders to TED therapy, defined as a >20% reduction in PS requirement at 3 months. Six of the 10 (60%) were non-responders. Data relative to 48 months long-term clinical outcomes demonstrated the absence of late responder patients and underline that all 4 early TED responder patients continued to maintain a sustained reduction in PS. Indeed, two of the 4 patients (50%) responding to TED discontinued PS while the other 2 patients (50%) reduced the number of weekly PS infusions by approximately 50%. The physical and mental components of the QoL improved significantly (P < 0.05) in responsive patients but did not change in non-responsive ones. Predictability of response to TED therapy in this study seemed to be linked to 1) SBS type 3, which was completely absent in the non-responder patients (P < 0.0001), 2) the residual small bowel length was significantly (P < 0.02) higher in responder (102 ± 18 cm) compared to non-responder patients (67 ± 27 cm), and 3) SBS type 1 and 2, enterostomy, surgical complications are significantly predictive of no response. Crohn's disease is equally present in both responder and non-responder patients. The number of side effects observed in our experience was extremely small both at the 3rd (0.01 n°/d/patients) and at the 48th month (0.001 n°/d/patients). No endoscopic neoplastic lesions were observed up to the 48th month of TED therapy.

Conclusions

This real-world experience allows us to state that, after 48 months of treatment, TED: 1) is a safe therapy with minimal, rare side effects; 2) produces a rapid reduction in PS volumes in 40% of treated patients; 3) consents 20% of enrolled patients to be weaned off PS; 4) maintains a stable nutritional state and, finally, 5) significantly improves the QoL.