Rare inherited coagulation disorders: no longer orphan and neglected.
coagulation rebalancing agents
nonfactor products
plasma-derived factors
prophylaxis
recessive coagulation disorders
recombinant coagulant factors
Journal
Research and practice in thrombosis and haemostasis
ISSN: 2475-0379
Titre abrégé: Res Pract Thromb Haemost
Pays: United States
ID NLM: 101703775
Informations de publication
Date de publication:
May 2024
May 2024
Historique:
received:
20
03
2024
revised:
03
05
2024
accepted:
18
05
2024
medline:
18
7
2024
pubmed:
18
7
2024
entrez:
18
7
2024
Statut:
epublish
Résumé
Rare inherited coagulation disorders due to the deficiency or dysfunction of coagulation factors have until recently received less clinical attention than hemophilias and von Willebrand disease. This situation has changed in the last decades, mainly due to therapeutic progress with the availability of more and safer products for replacement therapy produced by plasma fractionation or recombinant DNA technology. This narrative review, based on the latest literature and expert opinion, emphasizes the progress achieved for each of the rare deficiencies, mentions the still unmet therapeutic needs, and sketches the perspectives for further progress.
Identifiants
pubmed: 39022653
doi: 10.1016/j.rpth.2024.102460
pii: S2475-0379(24)00149-3
pmc: PMC11253144
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
102460Informations de copyright
© 2024 The Author(s).