Teduglutide effective in children with SBS, but still costly

Use of teduglutide is effective against short bowel syndrome (SBS) in paediatric patients, although some children need a longer duration of treatment to achieve enteral autonomy, according to a Spain study presented at ESPGHAN 2023. Predictive factors of response to treatment have not been identified.

“Teduglutide is a glucagon-like-peptide-2 analog that reduces the need for parenteral nutrition (PN) in patients with SBS,” said the researchers, led by Marta German Diaz, paediatric gastroenterology, Hospital Universitario 12 de Octubre, Madrid, Spain. “Data about long-term therapy with teduglutide in children are still scarce.”

In this national multicentre and prospective study, Diaz and her team described the real-life experience with teduglutide in children with SBS over the last 5 years in Spain. Patients with SBS were treated with the study drug for at least 3 months.

The researchers collected the following data: demographic and medical background, anthropometrics, laboratory assessments, adverse events, and PN requirements. Data was collected using REDCap. Additionally, treatment response was defined as >20 percent reduction in PN requirement.

Thirty-one patients (median age 2.3 years at the start of treatment, 64.5 percent males) from seven centres were included in this study. Their most common cause of intestinal failure was SBS (87.1 percent), followed by motility disorders (12.9 percent), while the most frequent cause of SBS was necrotizing enterocolitis (40.7 percent). [ESPGHAN 2023, abstract EP016]

The median residual bowel length was 29 cm among patients, of whom 18.5 percent presented with ileocecal valve. The median duration of home PN prior to teduglutide initiation was 22.6 months. The median duration of treatment was 19 months, but 23 patients (74.2 percent) received teduglutide for more than 1 year, while nine lasted for more than 3 years.

Of these children, 24 (83 percent) reduced their weekly PN energy by >20 percent, and 28 (97 percent) reduced their weekly PN volume by >20 percent. Ten patients (32.2 percent) who responded to treatment were weaned off PN, with a median treatment duration of 6 months.

Teduglutide treatment was discontinued in six patients during follow-up due to intestinal transplantation (n=3), lack of response (n=1), and other reasons (n=2). Fortunately, major adverse events did not occur in these patients. Finally, the researchers did not identify any predictive factors of treatment response.

Apart from its effectiveness, teduglutide is also associated with a significant decrease in the annual costs of home PN, as shown in a study in Italy. However, treatment remains costly, and cost-saving strategies are needed for its use. [ESPGHAN 2023, abstract EP015]

“Teduglutide … may reduce or even wean PN, but this new treatment is expensive,” said the investigators, led by Ugo Cucinotta, paediatric gastroenterology, Department of Human Pathology in Adulthood and Childhood, University of Messina, Messina, Italy.

The study included 60 children, of whom 30 (50 percent) received teduglutide and the other half (50 percent) did not. The median total costs of home PN significantly decreased in the treated group after 1 year (p<0.001) and 2 years of treatment (p<0.001), from 59.454 euros/year/patient to 43.885 euros/year/patient and 34.973 euros/year/patient, respectively.

“When adding teduglutide in the analysis, the total healthcare cost increased significantly in the treated group compared to not treated group, even assuming a reduction in the cost of the drug to 1/3 (p<0.001),” the investigators said.