Evidence map›Paper›PMID 30120816›Full record

ArticleActa physiologica (Oxford, England)2019

Vamorolone treatment improves skeletal muscle outcome in a critical illness myopathy rat model.

Hazem Akkad, Nicola Cacciani, Monica Llano-Diez, Rebeca Corpeno Kalamgi, Tamara Tchkonia, James L Kirkland, Lars Larsson

2 registry-linked trialsOpen access · greenAbstract read
In one paragraph

Article in Acta physiologica (Oxford, England), 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 15 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed, 1 pooled it
1.8field-weighted citation impact, top 15% 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.

NCT05166109 phase2completednot on this mapstarted 2022, after this paper: background citation

A Phase II Pilot Trial of Vamorolone vs. Placebo for the Treatment of Becker Muscular Dystrophy

TypeinterventionalSponsorReveraGen BioPharma, Inc.Ran2022 to 2025Enrolled46ConditionsBecker Muscular DystrophyArmsVamorolone, Placebo
NCT05185622 phase2completednot on this mapstarted 2022, after this paper: background citation

A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

TypeinterventionalSponsorSanthera PharmaceuticalsRan2022 to 2024Enrolled54ConditionsDuchenne Muscular DystrophyArmsVamorolone
3 · Its place in the literature

Who cites it

15 citing papers in PubMed, 1 synthesis or guideline pooled it, 25 citations in OpenAlex.

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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors at 3 institutions in 2 countries.

Hazem AkkadDepartment of Physiology and Pharmacology, Karolinska Institutet, Stockholm, Sweden.
Nicola CaccianiDepartment of Physiology and Pharmacology, Karolinska Institutet, Stockholm, Sweden.
Monica Llano-DiezDepartment of Physiology and Pharmacology, Karolinska Institutet, Stockholm, Sweden.
Rebeca Corpeno KalamgiDepartment of Physiology and Pharmacology, Karolinska Institutet, Stockholm, Sweden.
Tamara TchkoniaRobert and Arlene Kogod Center on Aging, Mayo Clinic, Rochester, Minnesota.
James L KirklandRobert and Arlene Kogod Center on Aging, Mayo Clinic, Rochester, Minnesota.
Lars LarssonDepartment of Physiology and Pharmacology, Karolinska Institutet, Stockholm, Sweden.ORCID 0000-0003-3722-035X
Karolinska Institutet · SEMayo Clinic in Florida · USPennsylvania State University · US

Funding

Targeting Cellular Senescence to Extend HealthspanP01AG062413 · NIA · MAYO CLINIC ROCHESTER · PI Sundeep Khosla · 2019 to 2026
$28.7M
Effect of Aging on Preadipocyte DifferentiationR37AG013925 · NIA · MAYO CLINIC ROCHESTER · PI KIRKLAND, JAMES L. · 2016 to 2025
$6.6M
Translational Geroscience NetworkR33AG061456 · NIA · MAYO CLINIC ROCHESTER · PI JAMES L. KIRKLAND, STEPHEN B. KRITCHEVSKY · 2019 to 2026
$6.2M
Familjen Erling-Perssons StiftelseKarolinska InstitutetNIA NIH HHS P01 AG062413NIA NIH HHS R33 AG061456NIA NIH HHS R37 AG013925Stockholms Läns Landsting 20150423Stockholms Läns Landsting 20170133Vetenskapsrådet 8651
6 · The paper itself

Abstract

aimCritical illness myopathy (CIM) is a consequence of modern critical care, leading to skeletal muscle atrophy/paralysis with negative consequences for mortality/morbidity and health care costs. Glucocorticoids (GCs) have been proposed to trigger CIM. Here, we compare outcomes of two GCs, the commonly used prednisolone and the newly developed dissociative vamorolone in response to the intensive care unit (ICU) condition for 5 days, ie, sedation, immobilization, and mechanical ventilation.

methodsRats were divided into a 0-day sham-operated control group, and three groups exposed to 5 days ICU condition during treatment with prednisolone (PRED) or vamorolone (VAM) or none of these GCs (ICU-group). Survival, body and muscle weights, cytokine concentrations, regulation of muscle contraction in single fast- and slow-twitch muscle fibres, myofibrillar protein expression and protein degradation pathways were studied.

resultsCritical illness myopathy geno- and pheno-types were confirmed in the ICU group. However, VAM and PRED groups showed reduced atrophy/weakness than the ICU group, and muscle specific differences with more severe negative effects on fast-twitch muscle fibres in the PRED than the other groups.

conclusionThese results show that vamorolone provides a GC intervention superior to typical GCs in improving CIM outcomes. Further, the findings do not support the notion that moderate-dose GC treatment represents a factor triggering CIM.

Indexed as

AnimalsAnti-Inflammatory AgentsContractile ProteinsCritical IllnessCytokinesDisease Models, AnimalFemaleMuscle ContractionMuscle, SkeletalMuscular DiseasesPrednisolonePregnadienediolsRatsRats, Sprague-DawleyAnti-Inflammatory AgentsContractile ProteinsCytokinesPrednisolonePregnadienediolsVBP15 compoundGlucocorticoidsICUmuscle wastingprednisolonevamorolone

Identifiers

PMID30120816
PMCPMC8424699
OpenAlexW2886306865

What OpenQuestion holds

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LicenceTDM
Read underepoch 390

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.