Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients
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 complemen...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2023-06-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-023-02779-2 |
| _version_ | 1827910588667265024 |
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| author | Sugat Adhikari Surendra Sapkota Suraj Shrestha Kshitiz Karki Anjan Shrestha |
| author_facet | Sugat Adhikari Surendra Sapkota Suraj Shrestha Kshitiz Karki Anjan Shrestha |
| author_sort | Sugat Adhikari |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-13T01:52:54Z |
| format | Article |
| id | doaj.art-d0bb79ecc1fe4de48f75c5c2a4df956d |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-03-13T01:52:54Z |
| publishDate | 2023-06-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-d0bb79ecc1fe4de48f75c5c2a4df956d2023-07-02T11:26:42ZengBMCOrphanet Journal of Rare Diseases1750-11722023-06-011811410.1186/s13023-023-02779-2Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patientsSugat Adhikari0Surendra Sapkota1Suraj Shrestha2Kshitiz Karki3Anjan Shrestha4Shreegaun Primary Health Care CenterDepartment of Internal Medicine, Ascension Saint Agnes HospitalMaharajgunj Medical College, Institute of MedicineAnnapurna Neurological Institute and Allied SciencesDepartment of Hematology, Tribhuvan University Teaching HospitalAbstract 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.https://doi.org/10.1186/s13023-023-02779-2Catastrophic health expenditureEculizumabLow and middle-income countriesOrphan drugParoxysmal nocturnal hemoglobinuriaNepal |
| spellingShingle | Sugat Adhikari Surendra Sapkota Suraj Shrestha Kshitiz Karki Anjan Shrestha Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients Orphanet Journal of Rare Diseases Catastrophic health expenditure Eculizumab Low and middle-income countries Orphan drug Paroxysmal nocturnal hemoglobinuria Nepal |
| 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 | Catastrophic health expenditure Eculizumab Low and middle-income countries Orphan drug Paroxysmal nocturnal hemoglobinuria Nepal |
| url | https://doi.org/10.1186/s13023-023-02779-2 |
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