J Pediatr. 2023 Dec 20:113882. doi: 10.1016/j.jpeds.2023.113882. Online ahead of print.

Ugo Cucinotta ¹, Miriam Acunzo ², Elise Payen ³, Cécile Talbotec ³, Céline Chasport ⁴, Angela Alibrandi ⁵, Florence Lacaille ³, Cécile Lambe ³

Author information

¹Pediatric Gastroenterology and Nutrition, Necker Enfants Malades University Hospital, Paris, France; Pediatric Gastroenterology and Cystic Fibrosis Unit, Department of Human Pathology in Adulthood and Childhood "G. Barresi", University of Messina, Messina, Italy. Electronic address: ugocucinotta@gmail.com.

²Pediatric Gastroenterology and Nutrition, Necker Enfants Malades University Hospital, Paris, France; Department of Pediatrics Vittore Buzzi Children's Hospital, Milan, Italy.

³Pediatric Gastroenterology and Nutrition, Necker Enfants Malades University Hospital, Paris, France.

⁴Pharmacy, Hopital Universitaire Necker - Enfants Malades, Paris, France.

⁵Statistical and Mathematical Science Unit, Department of Economics, University of Messina, Messina, Italy.

Abstract

Objectives: To analyze the real-life health care costs of home parenteral nutrition (HPN) in children with short bowel syndrome with intestinal failure (SBS-IF) before and after treatment with teduglutide, and to compare those with costs of children with SBS-IF not treated with teduglutide.

Study design: All consecutive children with SBS-IF on HPN treated with subcutaneous teduglutide starting from 2018 through 2020 in a tertiary French referral center were retrospectively included. These patients were matched to children with SBS-IF on HPN followed during the same 3-year period who were eligible for the teduglutide but were not treated. HPN direct medical costs included: home-care visits, HPN bags, hospital admissions, and teduglutide. A comparison of costs before/after treatment, and between patients treated/not treated was performed.

Results: Sixty children were included: 30 (50%) treated with teduglutide and 30 (50%) untreated. In the treated group, the median total costs of HPN significantly decreased after 1 (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. Comparing patients treated and not treated, the total HPN costs/year/patient were similar at baseline (p=0.6) but were significantly lower in the teduglutide-treated group after 1 (p=0.006) and 2 years of treatment (p<0.001). When adding the cost of teduglutide into the analysis, the total cost increased significantly in the treated group, and remained much higher even after modeling a reduction in the cost of the drug to 1/3 the present cost and PN weaning (p<0.001).

Conclusions: Treatment with teduglutide is associated with a significant reduction in the annual costs of HPN but still remains expensive because of the drug itself. Finding cost saving strategies is essential.