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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11145001 | PMC |
| http://dx.doi.org/10.18553/jmcp.2024.30.6.618 | DOI Listing |
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Proximal complement inhibitors in paroxysmal nocturnal hemoglobinuria: an abundance of options in a rare disease.
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January 2025
Department of Pharmacy, Atrium Health, Levine Cancer Institute, Charlotte, NC, USA.
[New treatment strategies for paroxysmal nocturnal hemoglobinuria: the guideline update and novel complement-targeting drugs].
Rinsho Ketsueki
November 2024
Department of Hematology and Oncology, Graduate School of Medicine, Osaka University.
Article Synopsis
- The 2022 update of Japanese guidelines for paroxysmal nocturnal hemoglobinuria (PNH) introduced improved diagnostic criteria and a new disease severity classification, enhancing clinical management.
- It included 10 key clinical questions to guide treatment strategies and patient considerations, helping healthcare providers navigate PNH management more effectively.
- New therapies, such as pegcetacoplan and danicopan, were approved to address PNH, with a focus not just on hemolysis but also on managing residual anemia and fatigue, influenced by real-world evidence.
Paroxysmal Nocturnal Hemoglobinuria, Pathophysiology, Diagnostics, and Treatment.
Transfus Med Hemother
October 2024
Department of Hematology, Oncology, Elblandklinikum, Riesa, University Hospital, Dresden, Germany.
Article Synopsis
- Paroxysmal nocturnal hemoglobinuria (PNH) causes intravascular hemolysis due to a lack of complement regulation, leading to symptoms like anemia, pain, and fatigue, but can be treated with complement inhibitors to reduce disease-related complications and mortality.
- Eculizumab and ravulizumab, terminal complement inhibitors, improve survival rates, but many patients still experience issues from extravascular hemolysis, necessitating new treatment options.
- Proximal complement inhibitors (like pegcetacoplan, danicopan, and iptacopan) can help normalize hemoglobin levels and enhance quality of life for patients with significant extravascular hemolysis, though a clear treatment algorithm for choosing among
Navigating the Complement Pathway to Optimize PNH Treatment with Pegcetacoplan and Other Currently Approved Complement Inhibitors.
Int J Mol Sci
August 2024
Apellis Pharmaceuticals, Inc., Waltham, MA 02451, USA.
Article Synopsis
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder caused by a mutation in hematopoietic stem cells that leads to a lack of protective proteins on red blood cells, making them vulnerable to destruction.
- The resulting symptoms include anemia, fatigue, and increased risk of blood clots due to complement-mediated hemolysis.
- Current treatments focus on inhibiting different stages of the complement activation pathway to manage symptoms and enhance patient outcomes, with pegcetacoplan being highlighted as a notable C3-targeted therapy.
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