PMID- 36799567
OWN - NLM
STAT- MEDLINE
DCOM- 20240108
LR  - 20240108
IS  - 1536-4844 (Electronic)
IS  - 1078-0998 (Print)
IS  - 1078-0998 (Linking)
VI  - 30
IP  - 1
DP  - 2024 Jan 5
TI  - Efficacy and Tolerance of Thalidomide in Patients With Very Early Onset 
      Inflammatory Bowel Disease.
PG  - 20-28
LID - 10.1093/ibd/izad018 [doi]
AB  - BACKGROUND: Few drugs have been studied for patients with very early onset 
      inflammatory bowel disease (VEOIBD). This study aimed to evaluate the efficacy 
      and tolerance of thalidomide in children with VEOIBD compared with children with 
      pediatric-onset IBD (pIBD). METHODS: A retrospective cohort study with a control 
      group was conducted. Propensity score 1:1 matching was used to identify control 
      subjects. The treatment persistence; the causes of drug withdrawal; the rate of 
      clinical remission and mucosal healing at 1, 2, and 3 years; and adverse events 
      (AEs) were evaluated in children with VEOIBD treated with thalidomide and 
      compared with children with pIBD. RESULTS: Thirty-nine courses of treatment with 
      thalidomide in VEOIBD and pIBD patients were compared. The treatment persistence 
      at 1, 2, and 3 years was 68.2% (95% confidence interval [CI], 50.8%-80.6%), 57.0% 
      (95% CI, 39.6%-71.1%), and 50.9% (95% CI, 33.7%-65.8%) for VEOIBD patients and 
      81.7% (95% CI, 65.3%-90.9%), 60.0% (95% CI, 41.7%-74.3%) and 33.0% (95% CI, 
      17.4%-49.5%) for pIBD patients, respectively (P = .12). A significantly higher 
      proportion of VEOIBD patients discontinued therapy due to lack of efficacy (48.2% 
      vs 17.2%; P = .03), while AEs were the main reason for discontinuation in pIBD 
      patients. Clinical remission and mucosal healing rates did not significantly 
      differ between VEOIBD and pIBD patients. A significatively lower number of VEOIBD 
      patients experienced AEs compared with pIBD patients (14 [35.9%] vs 30 [76.9%]; P 
      = .0005). CONCLUSIONS: Thalidomide is an effective and tolerated treatment in 
      children with VEOIBD. Discontinuation due to lack of efficacy is more frequent, 
      but AEs are less common than in children with pIBD.
CI  - (c) 2023 Crohn's & Colitis Foundation. Published by Oxford University Press on 
      behalf of Crohn's & Colitis Foundation.
FAU - Bramuzzo, Matteo
AU  - Bramuzzo M
AUID- ORCID: 0000-0002-5249-8248
AD  - Institute for Maternal and Child Health, IRCCS Burlo Garofolo, Trieste, Italy.
FAU - Giudici, Fabiola
AU  - Giudici F
AD  - Bureau de biostatistique et d'epidemiologie, Gustave Roussy and Universite 
      Paris-Saclay, Paris, France.
FAU - Arrigo, Serena
AU  - Arrigo S
AD  - Pediatric Gastroenterology and Endoscopy, IRCCS Istituto Giannina Gaslini, Genoa, 
      Italy.
FAU - Lionetti, Paolo
AU  - Lionetti P
AUID- ORCID: 0000-0003-1056-4400
AD  - Department NEUROFARBA, Meyer Children's Hospital, University of Florence, 
      Florence, Italy.
FAU - Zuin, Giovanna
AU  - Zuin G
AD  - Pediatrics, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy.
FAU - Romano, Claudio
AU  - Romano C
AD  - Pediatric Gastroenterology and Cystic Fibrosis Unit, Department of Human 
      Pathology in Adulthood and Childhood G. Barresi, University of Messina, Messina, 
      Italy.
FAU - Graziano, Francesco
AU  - Graziano F
AUID- ORCID: 0000-0002-4896-1842
AD  - Pediatric Unit, Villa Sofia Cervello Hospital, Palermo, Italy.
FAU - Faraci, Simona
AU  - Faraci S
AD  - Digestive Endoscopy and Surgery Unit, Bambino Gesu Children's Hospital, Rome, 
      Italy.
FAU - Alvisi, Patrizia
AU  - Alvisi P
AD  - Pediatric Gastroenterology Unit, Maggiore Hospital, Bologna, Italy.
FAU - Signa, Sara
AU  - Signa S
AD  - Pediatric Gastroenterology and Endoscopy, IRCCS Istituto Giannina Gaslini, Genoa, 
      Italy.
FAU - Scarallo, Luca
AU  - Scarallo L
AD  - Department NEUROFARBA, Meyer Children's Hospital, University of Florence, 
      Florence, Italy.
FAU - Martelossi, Stefano
AU  - Martelossi S
AD  - Pediatric Unit, Ca' Foncello's Hospital, Treviso, Italy.
FAU - Di Leo, Grazia
AU  - Di Leo G
AD  - Institute for Maternal and Child Health, IRCCS Burlo Garofolo, Trieste, Italy.
LA  - eng
PT  - Journal Article
PL  - England
TA  - Inflamm Bowel Dis
JT  - Inflammatory bowel diseases
JID - 9508162
RN  - 4Z8R6ORS6L (Thalidomide)
SB  - IM
MH  - Child
MH  - Humans
MH  - *Thalidomide/adverse effects
MH  - Retrospective Studies
MH  - Age of Onset
MH  - Drug Tolerance
MH  - *Inflammatory Bowel Diseases/drug therapy
PMC - PMC10769807
OAB - Thalidomide is a valid therapeutic option in children with very early onset 
      inflammatory bowel diseases unresponsive to conventional therapies. 
      Discontinuation due to lack of efficacy is more frequent, but adverse events are 
      less common than in children with pediatric-onset inflammatory bowel disease.
OABL- eng
OTO - NOTNLM
OT  - children
OT  - inflammatory bowel disease
OT  - thalidomide
OT  - therapy
OT  - very early onset
COIS- The authors declare no conflict of interest.
EDAT- 2023/02/18 06:00
MHDA- 2024/01/08 06:43
PMCR- 2023/02/17
CRDT- 2023/02/17 08:52
PHST- 2022/08/29 00:00 [received]
PHST- 2024/01/08 06:43 [medline]
PHST- 2023/02/18 06:00 [pubmed]
PHST- 2023/02/17 08:52 [entrez]
PHST- 2023/02/17 00:00 [pmc-release]
AID - 7044214 [pii]
AID - izad018 [pii]
AID - 10.1093/ibd/izad018 [doi]
PST - ppublish
SO  - Inflamm Bowel Dis. 2024 Jan 5;30(1):20-28. doi: 10.1093/ibd/izad018.