PMID- 34091545
OWN - NLM
STAT- MEDLINE
DCOM- 20211022
LR  - 20221219
IS  - 1536-4801 (Electronic)
IS  - 0277-2116 (Linking)
VI  - 73
IP  - 3
DP  - 2021 Sep 1
TI  - Tofacitinib Therapy in Children and Young Adults With Pediatric-onset Medically 
      Refractory Inflammatory Bowel Disease.
PG  - e57-e62
LID - 10.1097/MPG.0000000000003190 [doi]
AB  - OBJECTIVES: Tofacitinib, a selective Janus kinase inhibitor, effectively induces 
      and maintains remission in adults with inflammatory bowel disease (IBD), but data 
      are limited in children. This study aimed to evaluate the efficacy and safety of 
      tofacitinib for medically refractory pediatric-onset IBD. METHODS: This 
      single-center retrospective study included subjects ages 21 years and younger who 
      started tofacitinib for medically refractory IBD. Clinical activity indices, 
      clinical response, steroid-free remission, biochemical response, and adverse 
      events (AEs) were evaluated over 52 weeks. RESULTS: Twenty-one subjects, 18 with 
      ulcerative colitis or indeterminate IBD, received tofacitinib. At the end of the 
      12-week induction period, 9 out of 21 (42.9%) subjects showed clinical response 
      and 7 out of 21 (33.3%) were in steroid-free remission. Of evaluable subjects at 
      52 weeks, 7 out of 17 (41.2%) showed clinical response and were in steroid-free 
      remission. Of those remaining on tofacitinib at 1 year, none required concomitant 
      systemic corticosteroids. Tofacitinib was discontinued in 8 subjects because of 
      refractory disease, including 8 who ultimately underwent colectomy, and in 1 
      subject who developed a sterile intra-abdominal abscess. There were no instances 
      of thrombi, zoster reactivation, or clinically significant hyperlipidemia, all of 
      which were AEs of interest. CONCLUSIONS: There is limited experience with 
      tofacitinib in pediatric IBD. In this cohort, tofacitinib induced rapid clinical 
      response with sustained efficacy in nearly half of subjects. This study provides 
      encouraging evidence for the efficacy and safety of tofacitinib as part of the 
      treatment paradigm for young individuals with moderate-to-severe IBD. Larger, 
      well-powered, prospective studies are warranted.
CI  - Copyright (c) 2021 by European Society for Pediatric Gastroenterology, Hepatology, 
      and Nutrition and North American Society for Pediatric Gastroenterology, 
      Hepatology, and Nutrition.
FAU - Moore, Hillary
AU  - Moore H
AD  - Division of Gastroenterology, Hepatology, and Nutrition, The Children's Hospital 
      of Philadelphia.
AD  - Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, 
      Philadelphia, PA.
FAU - Dubes, Lucie
AU  - Dubes L
AD  - Division of Gastroenterology, Hepatology, and Nutrition, The Children's Hospital 
      of Philadelphia.
FAU - Fusillo, Steven
AU  - Fusillo S
AD  - Division of Gastroenterology, Hepatology, and Nutrition, The Children's Hospital 
      of Philadelphia.
AD  - Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, 
      Philadelphia, PA.
FAU - Baldassano, Robert
AU  - Baldassano R
AD  - Division of Gastroenterology, Hepatology, and Nutrition, The Children's Hospital 
      of Philadelphia.
AD  - Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, 
      Philadelphia, PA.
FAU - Stein, Ronen
AU  - Stein R
AD  - Division of Gastroenterology, Hepatology, and Nutrition, The Children's Hospital 
      of Philadelphia.
AD  - Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, 
      Philadelphia, PA.
LA  - eng
PT  - Journal Article
PL  - United States
TA  - J Pediatr Gastroenterol Nutr
JT  - Journal of pediatric gastroenterology and nutrition
JID - 8211545
RN  - 0 (Piperidines)
RN  - 0 (Pyrimidines)
RN  - 87LA6FU830 (tofacitinib)
SB  - IM
MH  - Adult
MH  - Child
MH  - *Colitis, Ulcerative/drug therapy
MH  - Humans
MH  - *Inflammatory Bowel Diseases/drug therapy
MH  - *Piperidines/adverse effects
MH  - Pyrimidines/adverse effects
MH  - Retrospective Studies
MH  - Young Adult
COIS- The authors report no conflicts of interest.
EDAT- 2021/06/07 06:00
MHDA- 2021/10/28 06:00
CRDT- 2021/06/06 21:09
PHST- 2021/06/07 06:00 [pubmed]
PHST- 2021/10/28 06:00 [medline]
PHST- 2021/06/06 21:09 [entrez]
AID - 00005176-202109000-00014 [pii]
AID - 10.1097/MPG.0000000000003190 [doi]
PST - ppublish
SO  - J Pediatr Gastroenterol Nutr. 2021 Sep 1;73(3):e57-e62. doi: 
      10.1097/MPG.0000000000003190.