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

Abstract

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.

Keywords: Clinical nutrition; Crohn's disease; Gut healing; Parenteral nutrition; Short bowel syndrome.