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Teduglutide in pediatric patients under 10 kg with short bowel syndrome on parenteral support: An open-label study
Kouji Masumoto1, Mitsuru Muto2, Takato Sasaki1
1Department of Pediatric Surgery, Institute of Medicine, University of Tsukuba, Tsukuba, Japan.
Insights
Teduglutide demonstrated safety and efficacy in Japanese pediatric patients with short bowel syndrome-intestinal failure (SBS-IF) weighing under 10kg. The treatment led to significant reductions in parenteral support and no adverse impact on growth.
Area of Science:
- Gastroenterology
- Pediatric Medicine
- Pharmacology
Background:
- Short bowel syndrome-associated intestinal failure (SBS-IF) necessitates parenteral support (PS).
- Teduglutide, a GLP-2 analog, is approved in Japan for SBS-IF but data in low-body-weight pediatric patients are limited.
- This study focuses on Japanese pediatric patients with SBS-IF weighing less than 10 kg.
Purpose of the Study:
- To evaluate the safety and efficacy of teduglutide in Japanese pediatric patients with SBS-IF and low body weight (<10 kg).
- To assess changes in parenteral support requirements and growth parameters following teduglutide treatment.
Main Methods:
- Phase 3, open-label study involving Japanese pediatric patients with SBS-IF (<10 kg body weight).
- Patients received teduglutide 0.05 mg/kg/day subcutaneously for 24 weeks within 28-week cycles.
- Safety (adverse events) and efficacy (PS requirements, growth parameters) were monitored.
Main Results:
- Three patients completed the study with a mean exposure of 48.9 weeks.
- All patients experienced treatment-emergent adverse events, none drug-related, leading to discontinuation, or death.
- Significant reductions in mean PS volume (13.1%) and caloric intake (46.6%) were observed.
- Weight and height/length-for-age z-scores showed increases from baseline.
Conclusions:
- Teduglutide treatment in pediatric patients with SBS-IF (<10 kg) showed no new safety concerns.
- Clinically meaningful reductions in parenteral support were achieved.
- No adverse effects on growth parameters were observed, indicating a positive safety and efficacy profile.
Background:
Patients with short bowel syndrome (SBS)-associated intestinal failure (SBS-IF) are dependent on parenteral support (PS). In Japan, teduglutide is the only GLP-2 analog medicine indicated for these patients. There are limited data for pediatric patients, especially those with a low body weight. This study evaluated the safety and efficacy of teduglutide in Japanese pediatric patients with SBS-IF (dependence on PS to provide ≥30% of fluid or caloric intake needs) who weighed less than 10 kg.
Methods:
This phase 3, open-label study enrolled Japanese pediatric patients with SBS-IF. Patients weighing less than 10 kg received teduglutide 0.05 mg/kg/day subcutaneously in 28-week treatment cycles (24 weeks of teduglutide treatment and 4 weeks of follow-up). Adverse events, changes in PS requirements, and growth parameters were assessed.
Results:
Three patients completed the study, with a mean teduglutide exposure duration of 48.9 weeks. All patients experienced treatment-emergent adverse events (TEAEs), but none were related to the study drug or led to death or treatment discontinuation. Clinically meaningful reductions in mean PS volume (13.1%) and mean PS caloric intake (46.6%) were observed at the end of treatment from baseline. One patient achieved a reduction in PS volume of ≥20% at the end of treatment from baseline. Growth parameters showed increases in weight and height/length-for-age z-scores at the end of treatment from baseline.
Conclusion:
In the three patients with SBS-IF who weighed less than 10 kg, no new safety signals were observed following teduglutide treatment. Clinically meaningful reductions in PS were noted and there was no adverse impact on growth parameters.
Trial Registration:
ClinicalTrials.gov registration number: NCT05027308.
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