Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients.
Catastrophic health expenditure
Eculizumab
Low and middle-income countries
Nepal
Orphan drug
Paroxysmal nocturnal hemoglobinuria
Journal
Orphanet journal of rare diseases
ISSN: 1750-1172
Titre abrégé: Orphanet J Rare Dis
Pays: England
ID NLM: 101266602
Informations de publication
Date de publication:
30 Jun 2023
30 Jun 2023
Historique:
received:
12
11
2022
accepted:
18
06
2023
medline:
3
7
2023
pubmed:
1
7
2023
entrez:
30
6
2023
Statut:
epublish
Résumé
Paroxysmal nocturnal hemoglobinuria (PNH) results from a mutation in the phosphatidylinositol glycan class-A gene which causes uncontrolled complement activation with resultant intravascular hemolysis and its sequelae. Eculizumab is a terminal complement inhibitor that blocks this complement activation and has revolutionized the treatment of PNH but comes with an enormous price which can have catastrophic health expenditure in low-middle income countries (LMIC) like Nepal. Here, we discuss the potential way forwards in the treatment of PNH in Nepal and other LMICs.
Identifiants
pubmed: 37391775
doi: 10.1186/s13023-023-02779-2
pii: 10.1186/s13023-023-02779-2
pmc: PMC10311788
doi:
Substances chimiques
eculizumab
A3ULP0F556
Antibodies, Monoclonal, Humanized
0
Types de publication
Letter
Langues
eng
Sous-ensembles de citation
IM
Pagination
172Informations de copyright
© 2023. The Author(s).
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