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

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 activat...

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Detalles Bibliográficos
Autores principales: Adhikari, Sugat, Sapkota, Surendra, Shrestha, Suraj, Karki, Kshitiz, Shrestha, Anjan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Letter to the Editor
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10311788/
https://www.ncbi.nlm.nih.gov/pubmed/37391775
http://dx.doi.org/10.1186/s13023-023-02779-2
Descripción
Sumario: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. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s13023-023-02779-2.

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