Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs.
Journal
Journal of managed care & specialty pharmacy
ISSN: 2376-1032
Titre abrégé: J Manag Care Spec Pharm
Pays: United States
ID NLM: 101644425
Informations de publication
Date de publication:
Dec 2020
Dec 2020
Historique:
entrez:
28
12
2020
pubmed:
29
12
2020
medline:
27
8
2021
Statut:
ppublish
Résumé
The current standard of care for paroxysmal nocturnal hemoglobinuria (PNH) are the C5 inhibitors eculizumab and ravulizumab, both monoclonal antibodies designed to target the complement protein C5, thereby preventing its cleavage and the formation of the terminal attack complex. C5 inhibitors have yielded substantial improvements in the treatment of PNH and changed the mortality and morbidity, as well as health-related quality of life of patients with the disease. These treatments target underlying intravascular hemolysis; however, they do not address extravascular hemolysis, resulting in incomplete response and remaining symptoms in some patients. Therefore, despite treatment with a C5 inhibitor, some patients still experience anemia with associated fatigue, transfusion needs, and impaired health-related quality of life.
Identifiants
pubmed: 33356783
doi: 10.18553/jmcp.2020.26.12-b.s14
pmc: PMC10410676
doi:
Substances chimiques
Antibodies, Monoclonal, Humanized
0
Complement C5
0
eculizumab
A3ULP0F556
ravulizumab
C3VX249T6L
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
S14-S20Références
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