Evidence map›Paper›PMID 37789059›Full record

ReviewNature reviews. Gastroenterology & hepatology2023

Precision medicine in monogenic inflammatory bowel disease: proposed mIBD REPORT standards.

Holm H Uhlig, Claire Booth, Judy Cho, Marla Dubinsky, Anne M Griffiths, Bodo Grimbacher, Sophie Hambleton, Ying Huang, Kelsey Jones, Jochen Kammermeier and 16 more

Open access · greenAbstract readReview
PubMed Publisher
In one paragraph

Review in Nature reviews. Gastroenterology & hepatology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed, 2 pooled it
3.1field-weighted citation impact, top 8% of its field
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

14 citing papers in PubMed, 2 syntheses or guidelines pooled it, 19 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

26 authors at 20 institutions in 12 countries.

Holm H UhligTranslational Gastroenterology Unit, University of Oxford, Oxford, UK. holm.uhlig@ndm.ox.ac.uk.ORCID 0000-0002-6111-7355
Claire BoothUCL Great Ormond Street Institute of Child Health, London, UK.
Judy ChoDepartment of Pathology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.ORCID 0000-0002-7959-0466
Marla DubinskyDepartment of Paediatric Gastroenterology, Susan and Leonard Feinstein IBD Clinical Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Anne M GriffithsSickKids Inflammatory Bowel Disease Centre and Cell Biology Program, Research Institute, Toronto, Canada.
Bodo GrimbacherInstitute for Immunodeficiency, Center for Chronic Immunodeficiency, Medical Center, Faculty of Medicine, Albert Ludwig University of Freiburg, Freiburg, Germany.
Sophie HambletonPrimary Immunodeficiency Group, Newcastle University Translational and Clinical Research Institute, Newcastle upon Tyne, UK.
Ying HuangDepartment of Gastroenterology, National Children's Medical Center, Children's Hospital of Fudan University, Shanghai, China.
Kelsey JonesPaediatric Gastroenterology, Great Ormond Street Hospital, London, UK.
Jochen KammermeierGastroenterology Department, Evelina London Children's Hospital, London, UK.ORCID 0000-0002-6046-8727
Hirokazu KaneganeDepartment of Child Health and Development, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University, Tokyo, Japan.
Sibylle KoletzkoDr. von Hauner Children's Hospital, Department of Paediatrics, University Hospital, LMU Munich, Munich, Germany.
Daniel KotlarzDr. von Hauner Children's Hospital, Department of Paediatrics, University Hospital, LMU Munich, Munich, Germany.
Christoph KleinDr. von Hauner Children's Hospital, Department of Paediatrics, University Hospital, LMU Munich, Munich, Germany.
Michael J LenardoMolecular Development of the Immune System Section, Laboratory of Immune System Biology, and Clinical Genomics Program, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Bernice LoResearch Branch, Sidra Medicine, Doha, Qatar.ORCID 0000-0002-1087-6845
Dermot P B McGovernF. Widjaja Foundation, Inflammatory Bowel and Immunobiology Research Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Ahmet ÖzenMarmara University Division of Allergy and Immunology, Istanbul, Turkey.
Lissy de RidderDepartment of Paediatric Gastroenterology, Erasmus University Medical Center Sophia Children's Hospital, Rotterdam, Netherlands.ORCID 0000-0002-6035-1182
Frank RuemmeleUniversité Paris Cité, APHP, Hôpital Necker Enfants Malades, Service de Gastroentérologie pédiatrique, Paris, France.
Dror S ShouvalInstitute of Gastroenterology, Nutrition and Liver Diseases, Schneider Children's Medical Center of Israel, Petah Tikva, Israel.
Scott B SnapperDivision of Gastroenterology and Nutrition, Boston Children's Hospital, Boston, MA, USA.
Simon P TravisTranslational Gastroenterology Unit, University of Oxford, Oxford, UK.ORCID 0000-0002-2690-4361
Dan TurnerShaare Zedek Medical Center, The Hebrew University of Jerusalem, Jerusalem, Israel.
David C WilsonChild Life and Health, Centre for Inflammation Research, University of Edinburgh, Edinburgh, UK.
Aleixo M MuiseSickKids Inflammatory Bowel Disease Centre and Cell Biology Program, Research Institute, Toronto, Canada.ORCID 0000-0001-9624-3346
Great Ormond Street Hospital · GBIcahn School of Medicine at Mount Sinai · USUniversity of Oxford · GBUniversity of Toronto · CABoston Children's Hospital · USCedars-Sinai Medical Center · USChildren's Hospital of Fudan University · CNErasmus MC · NLEvelina London Children's Healthcare · GBGerman Center for Pediatric and Adolescent Rheumatology · DEHamad bin Khalifa University · QAHebrew University of Jerusalem · ILHelmholtz Zentrum München · DEHôpital Necker-Enfants Malades · FRMarmara University · TRNational Institutes of Health · USNewcastle University · GBRoyal Hospital for Children · GBTel Aviv University · ILTokyo Medical and Dental University · JP

Funding

COngenital Diarrhea and Enteropathy (PediCODE) Consortium and BioRepositoryRC2DK118640 · NIDDK · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI James Richard Goldenring, Izumi Kaji · 2019 to 2026
$14.8M
Identifying Immune and Epithelial Network Signatures in Very Early Onset Inflammatory Bowel DiseaseRC2DK122532 · NIDDK · BOSTON CHILDREN'S HOSPITAL · PI KLEIN, CHRISTOPH, MUISE, ALEIXO M · 2020 to 2024
$9.1M
Medical Research Council MR/R008019/1NIDDK NIH HHS RC2 DK118640NIDDK NIH HHS RC2 DK122532
6 · The paper itself

Abstract

Owing to advances in genomics that enable differentiation of molecular aetiologies, patients with monogenic inflammatory bowel disease (mIBD) potentially have access to genotype-guided precision medicine. In this Expert Recommendation, we review the therapeutic research landscape of mIBD, the reported response to therapies, the medication-related risks and systematic bias in reporting. The mIBD field is characterized by the absence of randomized controlled trials and is dominated by retrospective observational data based on case series and case reports. More than 25 off-label therapeutics (including small-molecule inhibitors and biologics) as well as cellular therapies (including haematopoietic stem cell transplantation and gene therapy) have been reported. Heterogeneous reporting of outcomes impedes the generation of robust therapeutic evidence as the basis for clinical decision making in mIBD. We discuss therapeutic goals in mIBD and recommend standardized reporting (mIBD REPORT (monogenic Inflammatory Bowel Disease Report Extended Phenotype and Outcome of Treatments) standards) to stratify patients according to a genetic diagnosis and phenotype, to assess treatment effects and to record safety signals. Implementation of these pragmatic standards should help clinicians to assess the therapy responses of individual patients in clinical practice and improve comparability between observational retrospective studies and controlled prospective trials, supporting future meta-analysis.

Indexed as

Precision MedicineHumansProspective StudiesRetrospective Studies

Identifiers

PMID37789059
OpenAlexW4387312423

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.