SynthesisBMC gastroenterology2026
Targeted therapies and their outcomes in monogenic very early onset inflammatory bowel disease: a systematic review.
Synthesis in BMC gastroenterology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed.
- Clinical features and prognostic analysis of very early-onset inflammatory bowel disease.BMC pediatrics · 2026Article
- Immune-microbe interactions in pediatric ulcerative colitis: clinical features, pathogenesis, and novel targeted therapeutic strategies.Frontiers in immunology · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundVery early onset inflammatory bowel disease (VEO-IBD) is a rare, severe condition with over 80 monogenic causes. These often resist conventional therapies, necessitating targeted treatments such as biologics and hematopoietic stem cell transplantation (HSCT). This study aimed to systematically review therapeutic outcomes.
methodsThis systematic review followed PRISMA guidelines. We searched MEDLINE/PubMed, Web of Science, and Scopus (up to July 25, 2025) for studies on outcomes of targeted therapies in genetically confirmed monogenic VEO-IBD. Eligible studies included case reports, case series, and observational studies reporting remission, exacerbation, or mortality, with quality assessed using Joanna Briggs Institute checklists.
resultsOf 447 records, 62 studies (31 case reports, 21 observational studies, 10 case series) involving 355 patients were included. HSCT (4 studies) achieved survival rates of 64%-100% and remission rates up to 92%, with pooled individual-level data indicating 82% survival and 69% remission. Common complications included graft-versus-host disease (46%) and infections (64%). Of 59 patients receiving biologics or small-molecule agents, anti-TNF therapy showed minimal benefit, especially in IL-10 signaling defects, while based on case repots, other targeted therapies demonstrated favorable responses in specific genetic contexts, including ustekinumab (DUOX2, TGFBR2), anakinra (IL10RA), vedolizumab (CYBB), canakinumab (MVK), and abatacept (CTLA4).
conclusionsHSCT is effective for monogenic VEO-IBD, especially IL10R deficiency, with substantial survival and remission rates despite notable risks, while biologics yield inconsistent results. These findings reinforce early genetic diagnosis in guiding treatment and support the integration of precision medicine into the management of monogenic VEO-IBD, but also highlight the need for genotype-stratified prospective studies. REGISTRATION: This review was registered on PROSPERO (CRD420251118080).
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.