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

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

Adhikari, Sugat; Sapkota, Surendra; Shrestha, Suraj; Karki, Kshitiz; Shrestha, Anjan

Orphanet journal of rare diseases, 06/2023, Volume: 18, Issue: 1

Journal Article

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.

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Adhikari, Sugat | Sapkota, Surendra | Shrestha, Suraj | Karki, Kshitiz | Shrestha, Anjan

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