PMID- 33823514
OWN - NLM
STAT- MEDLINE
DCOM- 20211214
LR  - 20211214
IS  - 1421-9832 (Electronic)
IS  - 1018-8665 (Linking)
VI  - 237
IP  - 4
DP  - 2021
TI  - Off-Label Use and Safety of Drug Use in Vascular Anomalies.
PG  - 649-657
LID - 10.1159/000515980 [doi]
AB  - 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: The aim of this 
      work was to quantify off-label drug use in VA and assess its safety. 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: 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(R)) 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: 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.
CI  - (c) 2021 S. Karger AG, Basel.
FAU - Kleiber, Niina
AU  - Kleiber N
AD  - Department of Pharmacology and Physiology, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Research Center, CHU Sainte-Justine, Universite de Montreal, Montreal, Quebec, 
      Canada.
AD  - Division of General Pediatrics, Department of Pediatrics, CHU Sainte-Justine, 
      Universite de Montreal, Montreal, Quebec, Canada.
AD  - Vascular Anomaly Team, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Clinical Pharmacology Unit, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
FAU - Gariepy-Assal, Laurence
AU  - Gariepy-Assal L
AD  - Division of General Pediatrics, Department of Pediatrics, CHU Sainte-Justine, 
      Universite de Montreal, Montreal, Quebec, Canada.
FAU - Coulombe, Jerome
AU  - Coulombe J
AD  - Vascular Anomaly Team, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Division of Dermatology, Department of Pediatrics, CHU Sainte-Justine, Universite 
      de Montreal, Montreal, Quebec, Canada.
FAU - Marcoux, Simon
AU  - Marcoux S
AD  - Clinical Pharmacology Unit, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Faculte de medecine et des sciences de la sante, Universite de Sherbrooke, 
      Sherbrooke, Quebec, Canada.
FAU - Essouri, Sandrine
AU  - Essouri S
AD  - Research Center, CHU Sainte-Justine, Universite de Montreal, Montreal, Quebec, 
      Canada.
AD  - Division of General Pediatrics, Department of Pediatrics, CHU Sainte-Justine, 
      Universite de Montreal, Montreal, Quebec, Canada.
AD  - Vascular Anomaly Team, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
FAU - McCuaig, Catherine
AU  - McCuaig C
AD  - Vascular Anomaly Team, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Division of Dermatology, Department of Pediatrics, CHU Sainte-Justine, Universite 
      de Montreal, Montreal, Quebec, Canada.
FAU - Powell, Julie
AU  - Powell J
AD  - Vascular Anomaly Team, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Division of Dermatology, Department of Pediatrics, CHU Sainte-Justine, Universite 
      de Montreal, Montreal, Quebec, Canada.
FAU - Soulez, Gilles
AU  - Soulez G
AD  - Department of Radiology, Centre Hospitalier de l'Universite de Montreal (CHUM), 
      Montreal, Quebec, Canada.
FAU - Dubois, Josee
AU  - Dubois J
AD  - Vascular Anomaly Team, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Department of Radiology, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
LA  - eng
PT  - Journal Article
DEP - 20210406
PL  - Switzerland
TA  - Dermatology
JT  - Dermatology (Basel, Switzerland)
JID - 9203244
RN  - 0 (Pharmaceutical Preparations)
SB  - IM
MH  - Blood Vessels/*abnormalities
MH  - Congenital Abnormalities/drug therapy
MH  - *Drug Labeling
MH  - *Drug-Related Side Effects and Adverse Reactions
MH  - Humans
MH  - *Off-Label Use
MH  - Pharmaceutical Preparations
OTO - NOTNLM
OT  - Drug prescribing
OT  - Drug safety
OT  - Hemangiomas
OT  - Off-label drugs
OT  - Vascular anomalies
EDAT- 2021/04/07 06:00
MHDA- 2021/12/15 06:00
CRDT- 2021/04/06 20:21
PHST- 2021/01/29 00:00 [received]
PHST- 2021/03/18 00:00 [accepted]
PHST- 2021/04/07 06:00 [pubmed]
PHST- 2021/12/15 06:00 [medline]
PHST- 2021/04/06 20:21 [entrez]
AID - 000515980 [pii]
AID - 10.1159/000515980 [doi]
PST - ppublish
SO  - Dermatology. 2021;237(4):649-657. doi: 10.1159/000515980. Epub 2021 Apr 6.