TY - JOUR
T1 - Dual Biologic or Small Molecule Therapy in Refractory Pediatric Inflammatory Bowel Disease (DOUBLE-PIBD)
T2 - A Multicenter Study from the Pediatric IBD Porto Group of ESPGHAN
AU - Yerushalmy-Feler, Anat
AU - Olbjorn, Christine
AU - Kolho, Kaija Leena
AU - Aloi, Marina
AU - Musto, Francesca
AU - Martin-De-Carpi, Javier
AU - Lozano-Ruf, Ana
AU - Yogev, Dotan
AU - Matar, Manar
AU - Scarallo, Luca
AU - Bramuzzo, Matteo
AU - de Ridder, Lissy
AU - Kang, Ben
AU - Norden, Christoph
AU - Wilson, David C.
AU - Tzivinikos, Christos
AU - Turner, Dan
AU - Cohen, Shlomi
N1 - Publisher Copyright: © 2023 The Author(s).
PY - 2024/2/1
Y1 - 2024/2/1
N2 - Background: Current data on dual biologic therapy in children are limited. This multicenter study aimed to evaluate the effectiveness and safety of dual therapy in pediatric patients with inflammatory bowel disease (IBD). Methods: A retrospective study from 14 centers affiliated with the Pediatric IBD Interest and Porto Groups of the European Society for Paediatric Gastroenterology, Hepatology and Nutrition. Included were children with IBD who underwent combinations of biologic agents or biologic and small molecule therapy for at least 3 months. Demographic, clinical, laboratory, endoscopic, and imaging data were collected. Adverse events were recorded. Results: Sixty-two children (35 Crohn's disease, 27 ulcerative colitis; median age 15.5 [interquartile range, 13.1-16.8] years) were included. They had all failed previous biologic therapies, and 47 (76%) failed at least 2 biologic agents. The dual therapy included an anti-tumor necrosis factor agent and vedolizumab in 30 children (48%), anti-tumor necrosis factor and ustekinumab in 21 (34%) children, vedolizumab and ustekinumab in 8 (13%) children, and tofacitinib with a biologic in 3 (5%) children. Clinical remission was observed in 21 (35%), 30 (50%), and 38 (63%) children at 3, 6, and 12 months, respectively. Normalization of C-reactive protein and decrease in fecal calprotectin to <250 μg/g were achieved in 75% and 64%, respectively, at 12 months of follow-up. Twenty-nine (47%) children sustained adverse events, 8 of which were regarded as serious and led to discontinuation of therapy in 6. Conclusions: Dual biologic therapy may be effective in children with refractory IBD. The potential efficacy should be weighed against the risk of serious adverse events.
AB - Background: Current data on dual biologic therapy in children are limited. This multicenter study aimed to evaluate the effectiveness and safety of dual therapy in pediatric patients with inflammatory bowel disease (IBD). Methods: A retrospective study from 14 centers affiliated with the Pediatric IBD Interest and Porto Groups of the European Society for Paediatric Gastroenterology, Hepatology and Nutrition. Included were children with IBD who underwent combinations of biologic agents or biologic and small molecule therapy for at least 3 months. Demographic, clinical, laboratory, endoscopic, and imaging data were collected. Adverse events were recorded. Results: Sixty-two children (35 Crohn's disease, 27 ulcerative colitis; median age 15.5 [interquartile range, 13.1-16.8] years) were included. They had all failed previous biologic therapies, and 47 (76%) failed at least 2 biologic agents. The dual therapy included an anti-tumor necrosis factor agent and vedolizumab in 30 children (48%), anti-tumor necrosis factor and ustekinumab in 21 (34%) children, vedolizumab and ustekinumab in 8 (13%) children, and tofacitinib with a biologic in 3 (5%) children. Clinical remission was observed in 21 (35%), 30 (50%), and 38 (63%) children at 3, 6, and 12 months, respectively. Normalization of C-reactive protein and decrease in fecal calprotectin to <250 μg/g were achieved in 75% and 64%, respectively, at 12 months of follow-up. Twenty-nine (47%) children sustained adverse events, 8 of which were regarded as serious and led to discontinuation of therapy in 6. Conclusions: Dual biologic therapy may be effective in children with refractory IBD. The potential efficacy should be weighed against the risk of serious adverse events.
KW - Crohn's disease
KW - dual biologics
KW - ulcerative colitis
UR - http://www.scopus.com/inward/record.url?scp=85167874218&partnerID=8YFLogxK
U2 - https://doi.org/10.1093/ibd/izad064
DO - https://doi.org/10.1093/ibd/izad064
M3 - مقالة
C2 - 37042978
SN - 1078-0998
VL - 30
SP - 159
EP - 166
JO - Inflammatory Bowel Diseases
JF - Inflammatory Bowel Diseases
IS - 2
ER -