A Systematic Review and Meta-Analysis of Studies of Defibrotide Prophylaxis for Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome.
Journal
Clinical drug investigation
ISSN: 1179-1918
Titre abrégé: Clin Drug Investig
Pays: New Zealand
ID NLM: 9504817
Informations de publication
Date de publication:
Jun 2022
Jun 2022
Historique:
accepted:
16
03
2022
pubmed:
21
5
2022
medline:
15
6
2022
entrez:
20
5
2022
Statut:
ppublish
Résumé
Defibrotide is approved to treat severe veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) after haematopoietic cell transplantation in patients aged > 1 month in the European Union and for VOD/SOS with renal/pulmonary dysfunction post-haematopoietic cell transplantation in the United States. This meta-analysis estimated the incidence and risk of VOD/SOS after intravenous defibrotide prophylaxis using the published literature. PubMed, Embase and Web of Science were searched through 30 November 2021 for defibrotide studies in VOD/SOS "prevention" or "prophylaxis," excluding phase I studies, case reports, studies with fewer than ten patients and reviews. The search identified 733 records; 24 met inclusion criteria, of which 20 (N = 3005) evaluated intravenous defibrotide for VOD/SOS prophylaxis. Overall VOD/SOS incidence with intravenous defibrotide was 5%, with incidences of 5% in adults and 8% in paediatric patients. In eight studies with data on intravenous defibrotide prophylaxis vs controls (e.g. heparin, no prophylaxis), VOD/SOS incidence in controls was 16%. The risk ratio for developing VOD/SOS with defibrotide prophylaxis vs controls was 0.30 (95% confidence interval 0.12-0.71; p = 0.006). This analysis suggests a low incidence of VOD/SOS following intravenous defibrotide prophylaxis, regardless of age group, and a lower relative risk for VOD/SOS with defibrotide prophylaxis vs controls in patient populations at high risk of VOD/SOS.
Sections du résumé
BACKGROUND AND OBJECTIVES
OBJECTIVE
Defibrotide is approved to treat severe veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) after haematopoietic cell transplantation in patients aged > 1 month in the European Union and for VOD/SOS with renal/pulmonary dysfunction post-haematopoietic cell transplantation in the United States. This meta-analysis estimated the incidence and risk of VOD/SOS after intravenous defibrotide prophylaxis using the published literature.
METHODS
METHODS
PubMed, Embase and Web of Science were searched through 30 November 2021 for defibrotide studies in VOD/SOS "prevention" or "prophylaxis," excluding phase I studies, case reports, studies with fewer than ten patients and reviews.
RESULTS
RESULTS
The search identified 733 records; 24 met inclusion criteria, of which 20 (N = 3005) evaluated intravenous defibrotide for VOD/SOS prophylaxis. Overall VOD/SOS incidence with intravenous defibrotide was 5%, with incidences of 5% in adults and 8% in paediatric patients. In eight studies with data on intravenous defibrotide prophylaxis vs controls (e.g. heparin, no prophylaxis), VOD/SOS incidence in controls was 16%. The risk ratio for developing VOD/SOS with defibrotide prophylaxis vs controls was 0.30 (95% confidence interval 0.12-0.71; p = 0.006).
CONCLUSIONS
CONCLUSIONS
This analysis suggests a low incidence of VOD/SOS following intravenous defibrotide prophylaxis, regardless of age group, and a lower relative risk for VOD/SOS with defibrotide prophylaxis vs controls in patient populations at high risk of VOD/SOS.
Identifiants
pubmed: 35594010
doi: 10.1007/s40261-022-01140-y
pii: 10.1007/s40261-022-01140-y
pmc: PMC9188533
doi:
Substances chimiques
Polydeoxyribonucleotides
0
defibrotide
438HCF2X0M
Types de publication
Journal Article
Meta-Analysis
Systematic Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
465-476Informations de copyright
© 2022. The Author(s).
Références
Clin Res Hepatol Gastroenterol. 2016 Sep;40(4):378-85
pubmed: 27038846
Bone Marrow Transplant. 2007 Jul;40(1):79-82
pubmed: 17502897
Biol Blood Marrow Transplant. 2010 Feb;16(2):157-68
pubmed: 19766729
Bone Marrow Transplant. 2018 Feb;53(2):138-145
pubmed: 28759025
Clin Transplant. 2012 Jul-Aug;26(4):511-9
pubmed: 22428954
Transplantation. 1987 Dec;44(6):778-83
pubmed: 3321587
Br J Haematol. 2009 Nov;147(4):554-60
pubmed: 19747363
Bone Marrow Transplant. 2016 Feb;51(2):227-31
pubmed: 26524264
Leukemia. 2003 Aug;17(8):1636-42
pubmed: 12886253
Adv Clin Exp Med. 2020 Mar;29(3):339-344
pubmed: 32207590
Biol Blood Marrow Transplant. 2018 Jul;24(7):1471-1475
pubmed: 29477779
Biol Blood Marrow Transplant. 2019 Jul;25(7):1271-1280
pubmed: 30797942
J Clin Exp Hepatol. 2014 Dec;4(4):332-46
pubmed: 25755580
Lancet. 2012 Apr 7;379(9823):1301-9
pubmed: 22364685
Blood Adv. 2018 Jun 26;2(12):1495-1509
pubmed: 29945939
Bone Marrow Transplant. 2006 Oct;38(8):547-53
pubmed: 16953210
Transfus Apher Sci. 2020 Aug;59(4):102827
pubmed: 32522474
Bone Marrow Transplant. 2016 Jul;51(7):906-12
pubmed: 27183098
Pediatr Blood Cancer. 2008 Apr;50(4):831-2
pubmed: 18286502
Bone Marrow Transplant. 2021 Feb;56(2):411-418
pubmed: 32839533
Ann Intern Med. 1993 Feb 15;118(4):255-67
pubmed: 8420443
Expert Opin Drug Saf. 2013 Jan;12(1):123-36
pubmed: 23228043
Lancet Haematol. 2020 Jan;7(1):e61-e72
pubmed: 31818728
Cochrane Database Syst Rev. 2015 May 27;(5):CD009311
pubmed: 26017019
Bone Marrow Transplant. 2021 Jan;56(1):175-184
pubmed: 32665674