PMID- 34596969
OWN - NLM
STAT- MEDLINE
DCOM- 20220415
LR  - 20220415
IS  - 1545-5017 (Electronic)
IS  - 1545-5009 (Linking)
VI  - 68
IP  - 12
DP  - 2021 Dec
TI  - Systemic, local, and sclerotherapy drugs: What do we know about drug prescribing 
      in vascular anomalies?
PG  - e29364
LID - 10.1002/pbc.29364 [doi]
AB  - Off-label drug prescribing, frequent in the treatment of vascular anomalies (VA), 
      relies on the quality of the literature reporting drug efficacy and safety. Our 
      objective is to review the level of evidence (LOE) surrounding drug use in VA, 
      which is more prevalent in pediatric care. A list of drugs used in VA was created 
      with a literature review in July 2020. For each drug listed, the article 
      displaying the highest LOE was determined and then compared between 
      efficacy/safety data, routes of administration, pharmacological categories and a 
      subset of VA. The influence of research quality on study results was also 
      explored. The median LOE for the 74 drugs identified poor methodological quality, 
      with a predominance of retrospective studies or case reports. Drug safety is 
      currently inadequately reported. This is alarming as many treatments display 
      significant safety concerns. Also, current literature displays major publication 
      bias that probably leads to overestimation of drug efficacy in VA.
CI  - (c) 2021 Wiley Periodicals LLC.
FAU - Marcoux, Simon
AU  - Marcoux S
AUID- ORCID: 0000-0001-7899-1754
AD  - Faculty of Medicine, Universite de Sherbrooke, Sherbrooke, Quebec, Canada.
AD  - Clinical Pharmacology Unit, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
FAU - Theoret, Yves
AU  - Theoret Y
AUID- ORCID: 0000-0002-2353-860X
AD  - Department of Pharmacology and Physiology, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Clinical Pharmacology Unit, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
FAU - Dubois, Josee
AU  - Dubois J
AUID- ORCID: 0000-0002-8373-3028
AD  - Department of Radiology, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Vascular Anomaly Team, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
FAU - Essouri, Sandrine
AU  - Essouri S
AUID- ORCID: 0000-0001-9144-0957
AD  - Vascular Anomaly Team, 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  - Research Center, CHU Sainte-Justine, Universite de Montreal, Montreal, Quebec, 
      Canada.
FAU - Pincivy, Alix
AU  - Pincivy A
AUID- ORCID: 0000-0002-1143-3536
AD  - Library, CHU Sainte-Justine, Universite de Montreal, Montreal, Quebec, Canada.
FAU - Coulombe, Jerome
AU  - Coulombe J
AUID- ORCID: 0000-0003-1261-3093
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 - McCuaig, Catherine
AU  - McCuaig C
AUID- ORCID: 0000-0003-0792-6247
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
AUID- ORCID: 0000-0002-2107-4880
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
AUID- ORCID: 0000-0002-5566-3552
AD  - Department of Radiology, Centre Hospitalier de l'Universite de Montreal (CHUM), 
      Montreal, Quebec, Canada.
FAU - Kleiber, Niina
AU  - Kleiber N
AUID- ORCID: 0000-0002-7730-0541
AD  - Department of Pharmacology and Physiology, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Clinical Pharmacology Unit, CHU Sainte-Justine, Universite de Montreal, Montreal, 
      Quebec, Canada.
AD  - Vascular Anomaly Team, 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  - Research Center, CHU Sainte-Justine, Universite de Montreal, Montreal, Quebec, 
      Canada.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20211001
PL  - United States
TA  - Pediatr Blood Cancer
JT  - Pediatric blood & cancer
JID - 101186624
RN  - 0 (Pharmaceutical Preparations)
SB  - IM
MH  - Child
MH  - Humans
MH  - Off-Label Use
MH  - Pharmaceutical Preparations
MH  - Retrospective Studies
MH  - *Sclerotherapy
MH  - *Vascular Malformations/drug therapy
OTO - NOTNLM
OT  - Kasabach-Merritt phenomenon
OT  - drug safety
OT  - hemangiomas
OT  - hematology (nonmalignant)
OT  - hematology/oncology
OT  - patient safety
OT  - pediatric hematology/oncology
OT  - pharmacology
OT  - pharmacovigilance
OT  - vascular malformations
OT  - vascular tumors
EDAT- 2021/10/02 06:00
MHDA- 2022/04/16 06:00
CRDT- 2021/10/01 12:39
PHST- 2021/08/08 00:00 [revised]
PHST- 2021/05/09 00:00 [received]
PHST- 2021/08/31 00:00 [accepted]
PHST- 2021/10/02 06:00 [pubmed]
PHST- 2022/04/16 06:00 [medline]
PHST- 2021/10/01 12:39 [entrez]
AID - 10.1002/pbc.29364 [doi]
PST - ppublish
SO  - Pediatr Blood Cancer. 2021 Dec;68(12):e29364. doi: 10.1002/pbc.29364. Epub 2021 
      Oct 1.