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. 2023 Jun 30;18(1):172.
doi: 10.1186/s13023-023-02779-2.

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

Sugat Adhikari  1 , Surendra Sapkota  2 , Suraj Shrestha  3 , Kshitiz Karki  4 , Anjan Shrestha  5
Affiliations

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

Sugat Adhikari et al. Orphanet J Rare Dis. .

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.

Keywords: Catastrophic health expenditure; Eculizumab; Low and middle-income countries; Nepal; Orphan drug; Paroxysmal nocturnal hemoglobinuria.

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Conflict of interest statement

None to declare.

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