Frieri, Camilla (2022) Development of the anti-Factor B iptacopan as single-agent treatment for paroxysmal nocturnal hemoglobinuria. [Tesi di dottorato]
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| Item Type: | Tesi di dottorato |
|---|---|
| Resource language: | English |
| Title: | Development of the anti-Factor B iptacopan as single-agent treatment for paroxysmal nocturnal hemoglobinuria |
| Creators: | Creators Email Frieri, Camilla camillafrieri@gmail.com |
| Date: | 5 March 2022 |
| Number of Pages: | 56 |
| Institution: | Università degli Studi di Napoli Federico II |
| Department: | Medicina Clinica e Chirurgia |
| Dottorato: | Terapie avanzate biomediche e chirurgiche |
| Ciclo di dottorato: | 34 |
| Coordinatore del Corso di dottorato: | nome email DI MINNO, GIOVANNI UNSPECIFIED |
| Tutor: | nome email RISITANO, ANTONIO MARIA UNSPECIFIED |
| Date: | 5 March 2022 |
| Number of Pages: | 56 |
| Keywords: | pnh, novel complement inhibitors |
| Settori scientifico-disciplinari del MIUR: | Area 06 - Scienze mediche > MED/15 - Malattie del sangue |
| Date Deposited: | 17 Mar 2022 12:03 |
| Last Modified: | 28 Feb 2024 14:14 |
| URI: | http://www.fedoa.unina.it/id/eprint/14579 |
Collection description
Eculizumab, the first anti-C5 monoclonal antibody approved for patients with paroxysmal nocturnal hemoglobinuria (PNH), has revolutionized the natural history of this disease, blocking intravascular hemolysis, reducing the risk of thrombo-embolic events, resulting in a significant improvement in survival and quality of life. However, the hematological response to eculizumab is extremely heterogeneous, with only one-third of PNH patients reaching normal hemoglobin levels. We know that different factors may be contributing to residual anemia during treatment with eculizumab, such as chronic intravascular hemolysis, C3-mediated extravascular hemolysis and underlying bone marrow failure. In PNH patients on eculizumab treatment, the combined therapy with LNP023/ iptacopan has been shown to be effective in terms of hematological response, leading to the normalization of hemoglobin values and reducing intravascular hemolysis.The same results were confirmed during the extension period in monotherapy.
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