Real-world effectiveness and safety of teduglutide in adult and pediatric patients with short bowel syndrome in Argentina

Table 1 Study population baseline characteristics, n (%)

	Adults (n = 21)	Pediatric (n = 24)
Age (years)
mean (SD)	42.9 (17.6)	9.7 (4.6)
Range (minimum-maximum)	19-75	3-17
Sex
Female	11 (52.4)	5 (20.8)
Male	10 (47.6)	19 (79.2)
Weight and BMI
Weight (kg), mean (SD)	57.5 (12.8)	27.3 (12.7)
BMI, mean (SD)	21.9 (3.4)	16.4 (1.9)
Time of parenteral support (years), mean (SD)	7 (5.9)	7.8 (4.7)
Cause of major intestinal resection
Vascular disease	9 (42.9)	0 (0)
Postoperative complication	5 (23.8)	0 (0)
Volvulus	3 (14.3)	8 (33.3)
Intestinal atresia	2 (9.5)	7 (29.2)
Gastroschisis	0 (0)	5 (20.8)
Injury/traumatic	2 (9.5)	1 (5.6)
Necrotizing enterocolitis	0 (0)	1 (4.2)
Other cause	0 (0)	2 (8.4)
Anatomy type
Type 1	2 (9.5)	1 (4.2)
Type 2	13 (61.9)	16 (66.7)
Type 3	6 (28.9)	7 (29.2)
Colon in continuity
Yes	19 (90.5)	22 (91.7)
No	2 (9.5)	2 (8.3)
Remanent colon
> 0%-25%	2 (9.5)	2 (8.3)
> 25%-50%	13 (61.9)	6 (25)
> 50%-75%	0 (0)	6 (25)
> 75%-100%	6 (28.6)	9 (37.5)
Other or not available	0 (0)	1 (4.2)
Remanent small bowel length (cm), mean (SD)	48.2 (40.6)	43 (39.8)
Ostomy
Colostomy	0 (0)	2 (8.3)
Jejunostomy	1 (4.8)	1 (4.2)
Ileostomy	1 (4.8)	0 (0)

BMI: Body mass index.

Table 2 Effects on the reduction of parenteral support in study cohorts

	Weekly PS volume (mL)	P value	Number of days per week with PS	P value
Adult cohort
Baseline	12000 (4663)	NA	4 (4-7)	NA
Week 12	8700 (3884)	0.00071	4 (4-5)	0.00482
Week 24	5705 (4514)	0.00014	3 (0-4)	0.00019
Pediatric cohort
Baseline	9090 (4954)	NA	6 (5-7)	NA
Week 12	7266 (5082)	0.00044	6 (4-6)	0.00143
Week 24	5491 (4433)	0.00002	5 (2-5)	0.00008

Weekly volume expressed as mean (SD); number of days per week expressed as median (1^st and 3^rd quartile). P value calculated by non-parametric method for paired data vs baseline; NA: Not applicable; PS: Parenteral support.

Table 3 Analysis of patients weaned from parenteral support after 24 weeks, n (%)

	Not weaned (n = 33)	Weaned (n = 12)	P value1
Study cohort
Adult	15 (45.5)	6 (50)
Pediatric	18 (54.5)	6 (50)	0.19
Anatomy type
Type 1	2 (6.1)	1 (8.3)
Type 2	23 (69.7)	6 (50)	0.12
Type 3	8 (24.2)	5 (41.7)
Time from PS to treatment, mean (SD)	7.0 (5.9)	7.5 (6.4)	0.76
Remanent small bowel length (cm), mean (SD)	40.9 (40)	57.9 (38)	0.11
Weekly baseline PS volume (mL), mean (SD)	11541 (4634)	7441 (4850)	0.02

¹Adjusted P value calculated by logistic regression.

PS: Parenteral support.

Table 4 Treatment-emergent adverse events ≥ 5% overall, n (%)

	Adults (n = 21)		Pediatric (n = 24)
	All TEAE	Severe TEAE	All TEAE	Severe TEAE
Infections and infestations
Device related infection	0 (0)	0 (0)	2 (8.3)	1 (4.1)
Upper respiratory tract infection	0 (0)	0 (0)	2 (8.3)	0 (0)
Gastrointestinal disorders
Abdominal pain	1 (4.7)	0 (0)	4 (16.6)	0 (0)
Abdominal distension	2 (9.5)	0 (0)	0 (0)	0 (0)
General disorders and administration site conditions
Injection site pain or erythema1	0 (0)	0 (0)	2 (8.3)	0 (0)
Injury, poisoning and procedural complications
Stoma complications	1 (4.7)	0 (0)	2 (8.3)	1 (4.1)

¹Considered adverse event of special interest (AESI).

Treatment-emergent adverse event with incidence ≥ 5% in the overall study population (n = 45); one in adults and one in pediatric considered AESI. TEAE: Treatment-emergent adverse event.

Table 5 Serious treatment-emergent adverse events, n (%)

	Adults (n = 21)		Pediatric (n = 24)
	All TEAE	Severe TEAE	All TEAE	Severe TEAE
Infections and infestations
Catheter site infection	0 (0)	0 (0)	1 (4.1)	0 (0)
Device related infection	0 (0)	0 (0)	2 (8.3)	1 (4.1)
Influenza	0 (0)	0 (0)	1 (4.1)	0 (0)
Rhinovirus infection	0 (0)	0 (0)	1 (4.1)	0 (0)
Vascular device infection	0 (0)	0 (0)	1 (4.1)	0 (0)
General disorders and administration site conditions
Vascular device occlusion	0 (0)	0 (0)	1 (4.1)	0 (0)
Metabolism and nutrition disorders
Hypolabuminaemia	0 (0)	0 (0)	1 (4.1)	0 (0)
Lactic acidosis	0 (0)	0 (0)	1 (4.1)	0 (0)
Injury, poisoning and procedural complications
Stoma complications1	1 (4.7)	0 (0)	2 (8.3)	1 (4.1)
Hepatobiliary disorders
Cholestasis	0 (0)	0 (0)	1 (4.1)	0 (0)
Hyperbilirubinemia	0 (0)	0 (0)	1 (4.1)	0 (0)
Blood and lymphatic system disorders
Anaemia	0 (0)	0 (0)	1 (4.1)	0 (0)
Investigations
Liver function test abnormal	0 (0)	0 (0)	1 (4.1)	0 (0)
Nervous system disorders
Seizure	0 (0)	0 (0)	1 (4.1)	0 (0)

¹One in adults and one in pediatric considered adverse event of special interest (AESI); considered AESI.

All serious treatment-emergent adverse event in the overall study population (n = 45). TEAE: Treatment-emergent adverse event.