Teduglutide therapy in children, six years of experience in a specialized Intestinal Rehabilitation Unit

Introduction: Management of intestinal failure (IF) secondary to short bowel syndrome aims to achieve enteral autonomy and to prevent parenteral nutrition (PN) related complications. Intestinal rehabilitation is accomplished using a combination of different strategies (medical, surgical, and nutritional). A semisynthetic GLP-2 analogue, teduglutide (TED), is an enterohormone that has been recently added to the IF treatment options for children; experience with its use is still limited. Aim: to describe the results of using TED treatment in children with Type III-IF (IF) due to short gut syndrome. Methods: Retrospective descriptive study in patients < 18 years treated with TED 0.05 mg/kg/day from 1/2017 to 1/2022 performed at a single center in Argentina. SPSS v24.0 was used for statistical analysis, continuous variables were expressed as median and interquartile range (M/IQR), and Wilcoxon test was used to compare variables between initial and end points. Results: 10 patients are described, 5 with neonatal onset of IF (congenital GI anomalies) and 5 with childhood onset of IF (3 volvulus, 1 post-surgical complications and 1 trauma). M/IQR time of IF before TED initiation was 6 (2.5/11.7) years; M/IQR residual intestinal length was 18(8.5/21.5) cm, 3 patients had ICV and intact colon. M/IQR time of TED treatment was 1.7 (0.8/4.1) years. At last follow up 4 patients had electively discontinued parenteral nutrition (PN) at weeks 24, 29, 48 and 186 of TED therapy, and one discontinued at week 168 due to loss of conventional vascular access; one of them electively discontinued TED at week 260, and other 2 are on TED alternating days. The additional 5 patients continue on PN and daily TED, with a reduction on PN support of 26.5% (expressed as ratio between non protein energy intake/ resting energy expenditure) from baseline: M 0.85 (IQR:0.7/1.23) to 0.71 (IQR: 0.52/0.91) (p=0.059); and number of days of PN infusion/week from 6 (IQR: 5/6.5) to 4.8(4/5.5) (p 0.068). Overall Nutritional data is presented in table 1. Adverse events occurred were: abdominal pain in 8 patients, pain/bruises at injection site in 5, transient diarrhea in 5 mild hyperamylasemia in 4, low serum bicarbonate in 4, mild anemia in 3, hypomagnesemia in 2, low Vitamin D levels in 8, recurrent lower GI bleeding in 1 (due to anastomotic ulcers, diagnosed prior to TED initiation), positive fecal occult blood with negative endoscopy findings in 1, cholesterolosis in 1 and cholecystitis in 1. Conclusion: the use TED therapy allowed achieving intestinal rehabilitation or reduction on PN support with no impact on the nutritional status, in a group of patients unable to obtain further progress with standard medical rehabilitation. The adverse events were similar to those reported in the adult population.