Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients

Autor: Sugat Adhikari, Surendra Sapkota, Suraj Shrestha, Kshitiz Karki, Anjan Shrestha
Jazyk: angličtina
Rok vydání: 2023
Předmět:
Zdroj: Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-4 (2023)
Druh dokumentu: article
ISSN: 1750-1172
DOI: 10.1186/s13023-023-02779-2
Popis: Abstract 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.
Databáze: Directory of Open Access Journals
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