Off-Label Use and Safety of Drug Use in Vascular Anomalies.
Drug prescribing
Drug safety
Hemangiomas
Off-label drugs
Vascular anomalies
Journal
Dermatology (Basel, Switzerland)
ISSN: 1421-9832
Titre abrégé: Dermatology
Pays: Switzerland
ID NLM: 9203244
Informations de publication
Date de publication:
2021
2021
Historique:
received:
29
01
2021
accepted:
18
03
2021
pubmed:
7
4
2021
medline:
15
12
2021
entrez:
6
4
2021
Statut:
ppublish
Résumé
Off-label drug use is associated with an increased risk of adverse drug reactions. It is common in pediatrics and in rare diseases, which are two characteristics applying to vascular anomalies (VA). The aim of this work was to quantify off-label drug use in VA and assess its safety. A review was conducted to extract a list of drugs used in VA management. A drug was considered to have significant safety concerns if a black box warning was present or if a serious adverse drug reaction (SADR) was reported in at least 1% of the patients (SADR is defined as a noxious and unintended response to a drug that occurs at any dose and results in hospitalization, prolongation of existing hospitalization, congenital malformation, persistent or significant disability or incapacity, life-threatening condition, or death). The labelling status and safety of each drug was assessed based on the product monograph, Micromedex, and the FDA data. We found that 98.9% of the inventoried drugs were used off-label or unlicensed for VA management. Only the oral solution of propranolol hydrochloride (Hemangeol®) for the treatment of infantile hemangiomas is approved. Significant safety issues concerned 73% of the drugs and were more frequent among systemic than locally delivered drugs. Off-label drug use in VA is the rule and not the exception. Significant safety concerns are common. It is necessary to carefully weigh risk and benefits for every patient when using systemic and local treatments carrying safety concerns. Patients should be openly informed and involved in the decision-making process.
Sections du résumé
BACKGROUND
BACKGROUND
Off-label drug use is associated with an increased risk of adverse drug reactions. It is common in pediatrics and in rare diseases, which are two characteristics applying to vascular anomalies (VA).
OBJECTIVES
OBJECTIVE
The aim of this work was to quantify off-label drug use in VA and assess its safety.
METHODS
METHODS
A review was conducted to extract a list of drugs used in VA management. A drug was considered to have significant safety concerns if a black box warning was present or if a serious adverse drug reaction (SADR) was reported in at least 1% of the patients (SADR is defined as a noxious and unintended response to a drug that occurs at any dose and results in hospitalization, prolongation of existing hospitalization, congenital malformation, persistent or significant disability or incapacity, life-threatening condition, or death). The labelling status and safety of each drug was assessed based on the product monograph, Micromedex, and the FDA data.
RESULTS
RESULTS
We found that 98.9% of the inventoried drugs were used off-label or unlicensed for VA management. Only the oral solution of propranolol hydrochloride (Hemangeol®) for the treatment of infantile hemangiomas is approved. Significant safety issues concerned 73% of the drugs and were more frequent among systemic than locally delivered drugs.
CONCLUSIONS
CONCLUSIONS
Off-label drug use in VA is the rule and not the exception. Significant safety concerns are common. It is necessary to carefully weigh risk and benefits for every patient when using systemic and local treatments carrying safety concerns. Patients should be openly informed and involved in the decision-making process.
Identifiants
pubmed: 33823514
pii: 000515980
doi: 10.1159/000515980
doi:
Substances chimiques
Pharmaceutical Preparations
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
649-657Informations de copyright
© 2021 S. Karger AG, Basel.