35 What is known about the pharmacology of intramuscular therapeutics in duchenne muscular dystrophy? A systematic review

2Clinical Research Facility, Alder Hey Children’s HospitalIntroductionIn 2018 the Centers for Disease Control published updated Standards of Care for Duchenne Muscular Dystrophy – newly included was the recommendation for all patients with DMD who received steroids to receive prescriptions for intra...

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Bibliographic Details
Published inArchives of disease in childhood Vol. 108; no. 6; p. A12
Main Authors Roberts, Eve, Yi Gan, Jia, Hawcutt, Daniel, Spinty, Stefan
Format Journal Article
LanguageEnglish
Published London BMJ Publishing Group Ltd and Royal College of Paediatrics and Child Health 01.06.2023
BMJ Publishing Group LTD
Subjects
Duchenne's muscular dystrophy
Dystrophy
General abstracts
Hydrocortisone
Immunogenicity
Kinetics
Muscular dystrophy
Patients
Pharmacodynamics
Pharmacokinetics
Pharmacology
Steroid hormones
Structure-function relationships
Systematic review
Vaccination
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Summary:2Clinical Research Facility, Alder Hey Children’s HospitalIntroductionIn 2018 the Centers for Disease Control published updated Standards of Care for Duchenne Muscular Dystrophy – newly included was the recommendation for all patients with DMD who received steroids to receive prescriptions for intramuscular (IM) hydrocortisone for emergency administration at home.The aim of this systematic review was to assess the current understanding of the pharmacodynamics and kinetics of intramuscular therapies in patients affected by DMD.MethodsA systematic review was conducted according to Cochrane methodology. Medline, EMBASE and PubMed databases were searched. Two independent reviewers reviewed the abstract of each identified paper. Where there was any discrepancy in the decision to include or exclude a paper, a third reviewer arbitrated.ResultsThe search returned a total of 98 papers. 96 papers were excluded: 61 described animal or in-vitro studies, whilst the remaining studies did not study an intramuscular pharmacological intervention or were review articles.Of the two included articles, one compared the immunogenicity of intramuscular and subcutaneous administration of influenza vaccination, and the other studied ten patients with DMD who were injected with two different doses of plasmidic DNA. Neither study reported on the pharmacodynamics or kinetics of the interventions.ConclusionsThere is very limited evidence into the pharmaco-kinetics and -dynamics of IM therapies for children affected by muscular dystrophy. Given the recognised changes in the muscle structure and function, studies to explore if this causes clinically significant changes in boys with DMD are required.
ISSN:0003-9888
1468-2044
DOI:10.1136/archdischild-2023-ESDPPP.35