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Real-world efficacy of long-term teduglutide use in pediatric patients with short bowel syndrome
Claire Josey1, George Mazariegos2, Elizabeth King1
1Department of Pediatrics, University of Pittsburgh Medical Center (UPMC) Children's Hospital, Pittsburgh, PA, USA.
Insights
Teduglutide significantly reduced parenteral support dependency in pediatric patients with short bowel syndrome (SBS), with many achieving enteral autonomy. This study provides real-world evidence of teduglutide
Area of Science:
- Pediatric Gastroenterology
- Gastrointestinal Rehabilitation
- Pharmacological Interventions
Background:
- Short bowel syndrome (SBS) necessitates parenteral support (PS) for intestinal growth and absorption.
- Teduglutide is a therapeutic agent aimed at reducing PS dependency in SBS patients.
- Long-term outcomes of teduglutide in pediatric SBS require further investigation.
Purpose of the Study:
- To evaluate the long-term efficacy of teduglutide in a pediatric SBS population.
- To assess reductions in parenteral support (PS) volume and achievement of enteral autonomy.
- To describe patient characteristics and adverse events associated with teduglutide treatment.
Main Methods:
- Retrospective analysis of 27 pediatric SBS patients (ages 1-23 years) treated with teduglutide.
- Data collected included sex, age, intestinal length, BMI, PS regimens, and transplant status.
- Primary endpoint: ≥ 20% reduction in PS volume; secondary endpoint: enteral autonomy.
Main Results:
- 67% of patients achieved ≥ 20% reduction in PS volume; 30% reached enteral autonomy.
- Significant decreases observed in PS volume, duration, nonprotein calories, PNDI, and lipid dose.
- Pyrexia was the most frequent adverse event; 10 patients discontinued treatment, 4 due to related adverse events.
Conclusions:
- Teduglutide demonstrated significant efficacy in reducing PS dependency in pediatric SBS.
- Real-world data support teduglutide's role in improving outcomes for pediatric SBS patients.
- Further research may explore optimizing teduglutide treatment protocols and managing adverse events.
Background:
In patients with short bowel syndrome (SBS), teduglutide reduces dependency on parenteral support (PS) by promoting intestinal growth and absorption. We aim to describe long-term outcomes using teduglutide in a large pediatric intestinal rehabilitation and transplant center.
Material And Methods:
We performed a single-center, retrospective analysis of teduglutide use in patients with SBS ages 1-23 years. Sex, age, intestinal length, BMI, PS regimens, and isolated small bowel (ISB) transplant status are described. Subgroup analysis comparing younger and older patient cohorts was performed. Primary end point was a reduction in PS volume of ≥ 20 %.
Results:
27 patients (10 female; mean age 8.9 years) received subcutaneous teduglutide (0.05 mg/kg/d) once daily for mean 120.8 weeks. Mean bowel length was 56.9 (SD=52.1) cm. 5 were listed for ISB transplant, 2 were "inactive," and 20 were not listed. 2 patients were post-enterectomy. A decrease in PS volume of ≥ 20 % was experienced by 67 % of patients, with 30 % reaching enteral autonomy at mean 71.57 (SD=56.37) weeks (Fig. 1). Significant decreases in PS volume, hours and days, nonprotein calories, parenteral nutrition dependency index (PNDI), and lipid dose were observed (Table 2). Of patients listed at baseline, 2 remained listed, 2 were removed, and 1 was reclassified as "inactive." 1 was newly listed and 2 "inactive" patients were removed. 19 remained unlisted.The most frequent adverse event was pyrexia. 10 patients permanently discontinued treatment at mean 62.59 (SD=55.55) weeks, 4 due to related AEs.
Conclusion:
Pediatric SBS patients experienced significant decreases in PS dependency, providing real-world evidence of teduglutide efficacy.
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