Six-Month Safety and Effectiveness of Teduglutide in Patients with Short Bowel Syndrome in Japan: Interim Analysis of Post-marketing Surveillance

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INTRODUCTION: Patients with intestinal failure caused by short bowel syndrome (SBS) are dependent on parenteral support (PS) for nutrition. Teduglutide, an analogue of glucagon-like peptide 2, has been shown to decrease reliance on PS with an acceptable safety profile in multiregional phase 3 clinical trials, but currently there are no reports examining treatment outcomes with teduglutide in the real-world population in Japan. This interim analysis of a 36-month post-marketing surveillance (PMS) reports the safety and effectiveness of teduglutide in adults and pediatric patients with SBS in Japan. METHODS: This 6-month analysis was conducted as part of a prospective, all-case, 36-month PMS of teduglutide (Takeda Pharmaceutical Company Limited) conducted in patients with SBS across 102 sites in Japan. All patients who started teduglutide treatment from August 2021 to August 2022 were enrolled. Safety and effectiveness were evaluated after 6 months of teduglutide treatment and compared with baseline. RESULTS: A total of 123 patients aged 2-83 years were included in the safety analysis, including 27 children (aged < 15 years). A total of 51 patients (44 adults, 7 children) reported 117 adverse drug reactions (ADRs). In adults, the most common ADRs were abdominal distension (10.42%), abdominal pain (10.42%), stoma complication (8.33%), and nausea (7.29%). In children, the most common ADR was abdominal pain (11.11%). In the effectiveness analysis, 11.9% (12/101) of patients were able to wean off PS and 42.4% (39/92) were able to achieve at least 20% reduction in their PS volume. The overall mean (standard deviation) change from baseline in the z-score of body weight in children (n = 20) was 0.185 (0.2964). CONCLUSION: In this interim analysis of a 36-month PMS, teduglutide demonstrated an acceptable safety profile and promising effectiveness in patients with SBS. TRIAL REGISTRATION: ClinicalTrials.gov identifier, NCT05023382.

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