Pharmacological and Parenteral Nutrition-Based Interventions in Microvillus Inclusion Disease

CS Leng, E Rings, Saskia de Wildt, SCD van Ijzendoorn

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4 Citations (Scopus)
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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.

Original languageEnglish
Article number22
Pages (from-to)1-17
Number of pages17
JournalJournal of Clinical Medicine
Volume10
Issue number1
DOIs
Publication statusPublished - 2021

Bibliographical note

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© 2020 by the authors. Submitted for possible open access.

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