How I treat paroxysmal nocturnal hemoglobinuria.
Journal
Blood
ISSN: 1528-0020
Titre abrégé: Blood
Pays: United States
ID NLM: 7603509
Informations de publication
Date de publication:
11 03 2021
11 03 2021
Historique:
received:
01
07
2020
accepted:
28
08
2020
pubmed:
30
1
2021
medline:
27
7
2021
entrez:
29
1
2021
Statut:
ppublish
Résumé
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, complement-mediated hemolytic anemia with protean manifestations. PNH can present as a hemolytic anemia, a form of bone marrow failure, a thrombophilia, or any combination of the above. Terminal complement inhibition is highly effective for treating intravascular hemolysis from PNH and virtually eliminates the risk of thrombosis, but is not effective for treating bone marrow failure. Here, I present a variety of clinical vignettes that highlight the clinical heterogeneity of PNH and the attributes and limitations of the 2 US Food and Drug Administration-approved C5 inhibitors (eculizumab and ravulizumab) to treat PNH. I review the concept of pharmacokinetic and pharmacodynamic breakthrough hemolysis and briefly discuss new complement inhibitors upstream of C5 that are in clinical development. Last, I discuss the rare indications for bone marrow transplantation in patients with PNH.
Identifiants
pubmed: 33512400
pii: S0006-4971(21)00169-5
doi: 10.1182/blood.2019003812
pmc: PMC7955407
doi:
Substances chimiques
Antibodies, Monoclonal, Humanized
0
Complement Inactivating Agents
0
eculizumab
A3ULP0F556
ravulizumab
C3VX249T6L
Types de publication
Case Reports
Journal Article
Research Support, N.I.H., Extramural
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
1304-1309Informations de copyright
© 2021 by The American Society of Hematology.
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