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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| Tipologia del documento: | Tesi di dottorato |
|---|---|
| Lingua: | English |
| Titolo: | Development of the anti-Factor B iptacopan as single-agent treatment for paroxysmal nocturnal hemoglobinuria |
| Autori: | Autore Email Frieri, Camilla camillafrieri@gmail.com |
| Data: | 5 Marzo 2022 |
| Numero di pagine: | 56 |
| Istituzione: | Università degli Studi di Napoli Federico II |
| Dipartimento: | Medicina Clinica e Chirurgia |
| Dottorato: | Terapie avanzate biomediche e chirurgiche |
| Ciclo di dottorato: | 34 |
| Coordinatore del Corso di dottorato: | nome email DI MINNO, GIOVANNI [non definito] |
| Tutor: | nome email RISITANO, ANTONIO MARIA [non definito] |
| Data: | 5 Marzo 2022 |
| Numero di pagine: | 56 |
| Parole chiave: | pnh, novel complement inhibitors |
| Settori scientifico-disciplinari del MIUR: | Area 06 - Scienze mediche > MED/15 - Malattie del sangue |
| Depositato il: | 17 Mar 2022 12:03 |
| Ultima modifica: | 28 Feb 2024 14:14 |
| URI: | http://www.fedoa.unina.it/id/eprint/14579 |
Abstract
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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