Evidence map›Paper›PMID 33374831›Full record

ReviewJournal of clinical medicine2020

Pharmacological and Parenteral Nutrition-Based Interventions in Microvillus Inclusion Disease.

Changsen Leng, Edmond H H M Rings, Saskia N de Wildt, Sven C D van IJzendoorn

Registry-linked trialOpen access · goldAbstract readReview
In one paragraph

Review in Journal of clinical medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06721871 (Evaluation of Safety, Tolerability and Efficacy of Crofelemer Following Multiple Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Participants With Microvillus Inclusion Disease), which is not on this map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
1.1field-weighted citation impact, top 21% 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.

NCT06721871 phase2 / phase3active not recruitingnot on this mapstarted 2025, after this paper: background citation

Evaluation of Safety, Tolerability and Efficacy of Crofelemer Following Multiple Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Participants With Microvillus Inclusion Disease (MVID)

TypeinterventionalSponsorNapo Pharmaceuticals, Inc.Ran2025 to 2026Enrolled6ConditionsMicrovillus Inclusion Disease, Congenital Disorders, Rare DiseasesArmsCrofelemer Powder for Oral Solution, Placebo Powder for Oral Solution
3 · Its place in the literature

Who cites it

9 citing papers in PubMed, 14 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Microvillus inclusion disease: a short review of literature.Gastroenterology and hepatology from bed to bench · 2025
    Article
  5. Review
  6. Review
  7. Review
  8. Review
  9. Article
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

4 authors at 3 institutions in 3 countries.

Changsen LengDepartment of Biomedical Sciences of Cells and Systems, section Molecular Cell Biology, University of Groningen, University Medical Center Groningen, 9713 GZ Groningen, The Netherlands.
Edmond H H M RingsDepartment of Pediatrics, Erasmus University Medical Center, 3015 GD Rotterdam, The Netherlands.ORCID 0000-0001-8195-4356
Saskia N de WildtDepartment of Pharmacology and Toxicology, Radboud Institute Health Sciences, Radboud University Medical Center, 6525 GA Nijmegen, The Netherlands.
Sven C D van IJzendoornDepartment of Biomedical Sciences of Cells and Systems, section Molecular Cell Biology, University of Groningen, University Medical Center Groningen, 9713 GZ Groningen, The Netherlands.
University Medical Center Groningen · NLLeiden University Medical Center · NLRadboud University Nijmegen · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Microvillus inclusion disease (MVID) is a rare inherited and invariably fatal enteropathy, characterized by severe intractable secretory diarrhea and nutrient malabsorption. No cure exists, and patients typically die during infancy because of treatment-related complications. The need for alternative treatment strategies is evident. Several pharmacological interventions with variable successes have been tried and reported for individual patients as part of their clinical care. Unfortunately, these interventions and their outcomes have remained hidden in case reports and have not been reviewed. Further, recent advances regarding MVID pathogenesis have shed new light on the outcomes of these pharmacological interventions and offer suggestions for future clinical research and trials. Hence, an inventory of reported pharmacological interventions in MVID, their rationales and outcomes, and a discussion of these in the light of current knowledge is opportune. Together with a discussion on MVID-specific pharmacokinetic, -dynamic, and -genetic concerns that pose unique challenges regarding pharmacological strategies, we envision that this paper will aid researchers and clinicians in their efforts to develop pharmacological interventions to combat this devastating disease.

Indexed as

congenital diarrheal disordermalabsorptionmicrovillus inclusion diseaseMYO5Bmyosin Vb

Identifiers

PMID33374831
PMCPMC7794843
OpenAlexW3114972628

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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.