The Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria Treatment
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare chronic bone marrow failure condition characterized by complement-mediated hemolytic anemia and thrombosis. While its initial clinical description occurred in 1882, somatic mutations in PIGA were discovered in the 1990s. With an improved understand...
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| Format: | Article |
| Language: | English |
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Galenos Publishing House
2025-06-01
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| Series: | Turkish Journal of Hematology |
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| Online Access: | https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-73483 |
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| author | Cameron Perry Xinyu Von Buttlar Swapna Thota |
| author_facet | Cameron Perry Xinyu Von Buttlar Swapna Thota |
| author_sort | Cameron Perry |
| collection | DOAJ |
| description | Paroxysmal nocturnal hemoglobinuria (PNH) is a rare chronic bone marrow failure condition characterized by complement-mediated hemolytic anemia and thrombosis. While its initial clinical description occurred in 1882, somatic mutations in PIGA were discovered in the 1990s. With an improved understanding of PNH biology, a focused effort on complement inhibitors led to the discovery of eculizumab, a C5 inhibitor initially approved by the US Food and Drug Administration in 2007. Terminal complement pathway inhibition reduced intravascular hemolysis, anemia, and thrombosis. Further advancements in drug development for PNH have included improved pharmacokinetics with ravulizumab in 2018 and the introduction of proximal complement inhibitors such as pegcetacoplan (2021), iptacopan (2023), danicopan (2024), and crovalimab (2024) to enhance patient outcomes. With these new proximal and distal complement inhibitors in the treatment landscape, it is timely for clinicians to review the evolving landscape of PNH treatments and patient selection. |
| format | Article |
| id | doaj-art-e3e72fb8e24e48f598538417f8265310 |
| institution | Kabale University |
| issn | 1308-5263 |
| language | English |
| publishDate | 2025-06-01 |
| publisher | Galenos Publishing House |
| record_format | Article |
| series | Turkish Journal of Hematology |
| spelling | doaj-art-e3e72fb8e24e48f598538417f82653102025-08-20T03:46:06ZengGalenos Publishing HouseTurkish Journal of Hematology1308-52632025-06-01422748110.4274/tjh.galenos.2025.2025.0054TJH-73483The Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria TreatmentCameron Perry0Xinyu Von Buttlar1Swapna Thota2University of Tennessee Health Science Center College of Medicine, Department of Hematology and Oncology, Memphis, United StatesUniversity of Tennessee Health Science Center, Department of Internal Medicine, Division of Hematology and Oncology, Memphis, United StatesUniversity of Tennessee Health Science Center, Department of Internal Medicine, Division of Hematology and Oncology, Memphis, United StatesParoxysmal nocturnal hemoglobinuria (PNH) is a rare chronic bone marrow failure condition characterized by complement-mediated hemolytic anemia and thrombosis. While its initial clinical description occurred in 1882, somatic mutations in PIGA were discovered in the 1990s. With an improved understanding of PNH biology, a focused effort on complement inhibitors led to the discovery of eculizumab, a C5 inhibitor initially approved by the US Food and Drug Administration in 2007. Terminal complement pathway inhibition reduced intravascular hemolysis, anemia, and thrombosis. Further advancements in drug development for PNH have included improved pharmacokinetics with ravulizumab in 2018 and the introduction of proximal complement inhibitors such as pegcetacoplan (2021), iptacopan (2023), danicopan (2024), and crovalimab (2024) to enhance patient outcomes. With these new proximal and distal complement inhibitors in the treatment landscape, it is timely for clinicians to review the evolving landscape of PNH treatments and patient selection.https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-73483paroxysmal nocturnal hemoglobinuriaaplastic anemianovel complement inhibitors |
| spellingShingle | Cameron Perry Xinyu Von Buttlar Swapna Thota The Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria Treatment Turkish Journal of Hematology paroxysmal nocturnal hemoglobinuria aplastic anemia novel complement inhibitors |
| title | The Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria Treatment |
| title_full | The Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria Treatment |
| title_fullStr | The Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria Treatment |
| title_full_unstemmed | The Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria Treatment |
| title_short | The Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria Treatment |
| title_sort | advancing landscape of paroxysmal nocturnal hemoglobinuria treatment |
| topic | paroxysmal nocturnal hemoglobinuria aplastic anemia novel complement inhibitors |
| url | https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-73483 |
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