The Effect of Intravenous Trehalose Administration in a Patient with Multiple Sulfatase Deficiency

Overview

Journal Arch Med Sci

Specialty General Medicine

Date 2023 Sep 21

PMID 37732042

Authors

Shabnam Radbakhsh

Moein Mobini

Eric Gumpricht

Maciej Banach

Tannaz Jamialahmadi

Amirhossein Sahebkar

Affiliations

Soon will be listed here.

Abstract

Introduction: We describe the case of a female child with multiple sulfatase deficiency (MSD) who received intravenous (IV) trehalose (15 g/week) for 3 months.

Methods: The efficacy of trehalose was evaluated by comparing serum biomarkers, a quality-of-life questionnaire (HRQoL), and imaging (brain MRI and ultrasonography of liver and spleen) at weeks 0 (W0) and 12 (W12).

Results: The improvement in quality of life assessments along with a slight decrease in the spleen dimensions were observed after 3-month intervention.

Conclusions: Future research with a larger MSD population and a longer-term follow-up is warranted to determine whether trehalose can improve MSD patient health and clinical outcomes.

Citing Articles

Effect of trehalose on mortality and disease severity in ICU-admitted patients: Protocol for a triple-blind, randomized, placebo-controlled clinical trial.

Sahranavard M, Hosseinjani H, Emadzadeh M, Jamialahmadi T, Sahebkar A Contemp Clin Trials Commun. 2024; 40:101324.

PMID: 39021672 PMC: 11252791. DOI: 10.1016/j.conctc.2024.101324.

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