WHAT IS KNOWN ABOUT THE PHARMACOLOGY OF INTRAMUSCULAR THERAPEUTICS IN DUCHENNE MUSCULAR DYSTROPHY? A SYSTEMATIC REVIEW

Roberts, Eve, Gan, Jia Yi, Hawcutt, Daniel ORCID: 0000-0002-8120-6507 and Spinty, Stefan (2023) WHAT IS KNOWN ABOUT THE PHARMACOLOGY OF INTRAMUSCULAR THERAPEUTICS IN DUCHENNE MUSCULAR DYSTROPHY? A SYSTEMATIC REVIEW. .

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Abstract

<jats:sec><jats:title>Introduction</jats:title><jats:p>Off-label use is still inevitable for paediatric drug treatment. The aim of this study was to analyse the licensing status of drug prescriptions in German paediatric (specialised) outpatient clinics and to determine changes over a 10-year time course.</jats:p></jats:sec><jats:sec><jats:title>Methods</jats:title><jats:p>Cross-sectional, retrospective, monocentric studies were conducted in 2009 and 2019 to assess drug prescriptions regarding their licensing status in 10 (one general and nine specialised) outpatient clinics in Germany. Prevalence and relative frequency of off-label prescriptions were calculated, reasons for off-label prescribing analysed and logistic regression performed to determine influencing factors.</jats:p></jats:sec><jats:sec><jats:title>Results</jats:title><jats:p>751 prescriptions of 296 patients in 2009 and 1438 prescriptions of 786 patients in 2019 were examined and classified according to their licensing status. Relative frequency of off-label prescriptions was around 45% without significant change over that decade. Prevalence of off-label prescriptions was 60.1% in 2009 and 53.1% in 2019 and therefore significantly higher in 2009 (p=0.037). The number of prescriptions per patient was significantly higher in 2009 (2.5 ± 2.3 vs. 1.8 ± 1.5, p&lt;0.000), too. Comparison revealed the same high-ranking reasons in every study: off-label use due to indication, overdosing and missing paediatric information.</jats:p></jats:sec><jats:sec><jats:title>Conclusions</jats:title><jats:p>Off-label prescribing still plays an important role in clinical daily routine in paediatrics. Despite numerous regulatory efforts and incentives, no substantial reduction in off-label prescribing could be determined since 2009. Further efforts are needed to generate more evidence-based knowledge about paediatric pharmacotherapy and to treat children as best as possible.</jats:p></jats:sec>

Item Type:	Conference or Workshop Item (Unspecified)
Uncontrolled Keywords:	6 Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals
Divisions:	Faculty of Health and Life Sciences Faculty of Health and Life Sciences > Institute of Life Courses and Medical Sciences
Depositing User:	Symplectic Admin
Date Deposited:	06 Nov 2023 16:33
Last Modified:	17 Mar 2024 18:14
DOI:	10.1136/archdischild-2023-ESDPPP.33
Related URLs:	Author Publisher
URI:	https://livrepository.liverpool.ac.uk/id/eprint/3176608