In the Journals

Gattex reduces need for parenteral support in children with short bowel syndrome

Gattex, a recombinant human glucagon-like peptide-2, helped pediatric patients with short bowel syndrome-associated intestinal failure reduce their need for parenteral support, according to results of a phase 3 trial.

The drug, manufactured by Takeda, is also known as teduglutide.

Andrew A. Grimm, MD, PhD, of Human Genetic Therapies at Shire, a Takeda company, and colleagues wrote that parenteral support, like parenteral nutrition or IV fluids, over a long-term period can be lifesaving for patients with short bowel syndrome-associated intestinal failure (SBS-IF), but comes with some potential complications, including liver disease, blood stream infections and central venous thrombosis.

“Enhancing intestinal adaptation minimizes dependence on parenteral support, thereby reducing the risk of complications and potentially improving quality of life,” they wrote. “Teduglutide enables [parenteral support] reductions in heterogeneous populations of adult and pediatric patients with SBS-IF.”

Infographic on the efficacy of Gattex for short bowel syndrome
Gattex, a recombinant human glucagon-like peptide-2, helped pediatric patients with short bowel syndrome-associated intestinal failure reduce their need for parenteral support, according to results of a phase 3 trial.

The 24-month, phase 3 trial comprised 59 patients in three arms. One group received 0.025 mg/kg teduglutide once daily (n= 24), the second received 0.05 mg/kg teduglutide once daily (n = 26) and group three received nonblinded standard of care (n = 9).

The primary efficacy outcome was the number of patients who achieved a reduction in parenteral support of at least 20% from baseline to week 24. Researchers also assessed the drug’s safety using treatment-emergent adverse events and growth parameters as the main safety endpoints.

Investigators found that 13 patients in the 0.025 mg/kg group (54.2%) and 18 patients in the 0.05 mg/kg group (69.2%) achieved the primary efficacy outcome compared with 1 patient in the standard of care group (11.1%). Patients in both teduglutide groups also showed clinically significant reductions in parenteral support volume, parenteral calories, days per weeks and hours per day of parenteral support infusions and increase in enteral nutrition and plasma citrulline at week 24. Two patients in the 0.025 mg/kg group and three patients in the 0.05 mg/kg group achieved enteral autonomy.

In the teduglutide groups, 98% of patients reported treatment-emergent adverse events compared with 100% of patients in the standard of care group. The most common adverse events were pyrexia and vomiting.

“The post hoc statistical analysis of the primary efficacy endpoint and change in mean parenteral support volume supports the clinically meaningful improvements observed in the teduglutide groups,” the researchers wrote. “Parenteral support reductions in response to treatment with teduglutide are expected to reduce the long-term risks associated with parenteral support and may also improve quality of life of children with SBS-IF.” by Alex Young

Disclosures: Grimm is an employee of Takeda. Please see the full study for all other authors’ relevant financial disclosures.

 

Gattex, a recombinant human glucagon-like peptide-2, helped pediatric patients with short bowel syndrome-associated intestinal failure reduce their need for parenteral support, according to results of a phase 3 trial.

The drug, manufactured by Takeda, is also known as teduglutide.

Andrew A. Grimm, MD, PhD, of Human Genetic Therapies at Shire, a Takeda company, and colleagues wrote that parenteral support, like parenteral nutrition or IV fluids, over a long-term period can be lifesaving for patients with short bowel syndrome-associated intestinal failure (SBS-IF), but comes with some potential complications, including liver disease, blood stream infections and central venous thrombosis.

“Enhancing intestinal adaptation minimizes dependence on parenteral support, thereby reducing the risk of complications and potentially improving quality of life,” they wrote. “Teduglutide enables [parenteral support] reductions in heterogeneous populations of adult and pediatric patients with SBS-IF.”

Infographic on the efficacy of Gattex for short bowel syndrome
Gattex, a recombinant human glucagon-like peptide-2, helped pediatric patients with short bowel syndrome-associated intestinal failure reduce their need for parenteral support, according to results of a phase 3 trial.

The 24-month, phase 3 trial comprised 59 patients in three arms. One group received 0.025 mg/kg teduglutide once daily (n= 24), the second received 0.05 mg/kg teduglutide once daily (n = 26) and group three received nonblinded standard of care (n = 9).

The primary efficacy outcome was the number of patients who achieved a reduction in parenteral support of at least 20% from baseline to week 24. Researchers also assessed the drug’s safety using treatment-emergent adverse events and growth parameters as the main safety endpoints.

Investigators found that 13 patients in the 0.025 mg/kg group (54.2%) and 18 patients in the 0.05 mg/kg group (69.2%) achieved the primary efficacy outcome compared with 1 patient in the standard of care group (11.1%). Patients in both teduglutide groups also showed clinically significant reductions in parenteral support volume, parenteral calories, days per weeks and hours per day of parenteral support infusions and increase in enteral nutrition and plasma citrulline at week 24. Two patients in the 0.025 mg/kg group and three patients in the 0.05 mg/kg group achieved enteral autonomy.

In the teduglutide groups, 98% of patients reported treatment-emergent adverse events compared with 100% of patients in the standard of care group. The most common adverse events were pyrexia and vomiting.

“The post hoc statistical analysis of the primary efficacy endpoint and change in mean parenteral support volume supports the clinically meaningful improvements observed in the teduglutide groups,” the researchers wrote. “Parenteral support reductions in response to treatment with teduglutide are expected to reduce the long-term risks associated with parenteral support and may also improve quality of life of children with SBS-IF.” by Alex Young

Disclosures: Grimm is an employee of Takeda. Please see the full study for all other authors’ relevant financial disclosures.

 

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