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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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
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author Adhikari, Sugat
Sapkota, Surendra
Shrestha, Suraj
Karki, Kshitiz
Shrestha, Anjan
author_facet Adhikari, Sugat
Sapkota, Surendra
Shrestha, Suraj
Karki, Kshitiz
Shrestha, Anjan
author_sort Adhikari, Sugat
collection PubMed
description 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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spelling pubmed-103117882023-07-01 Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients Adhikari, Sugat Sapkota, Surendra Shrestha, Suraj Karki, Kshitiz Shrestha, Anjan Orphanet J Rare Dis Letter to the Editor 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. BioMed Central 2023-06-30 /pmc/articles/PMC10311788/ /pubmed/37391775 http://dx.doi.org/10.1186/s13023-023-02779-2 Text en © The Author(s) 2023 https://creativecommons.org/licenses/by/4.0/Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Letter to the Editor
Adhikari, Sugat
Sapkota, Surendra
Shrestha, Suraj
Karki, Kshitiz
Shrestha, Anjan
Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients
title Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients
title_full Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients
title_fullStr Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients
title_full_unstemmed Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients
title_short Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients
title_sort eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in nepalese patients
topic Letter to the Editor
url 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
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