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Safe and Successful Surgical Outcome in Persons with Hemophilia A with and without Inhibitors Treated with Emicizumab: A Large, Single Center, Real-World Experience

Emicizumab is a humanized recombinant bispecific antibody, bridging together activated factor IX (FIXa) and factor X (FX), thus mimicking the activity of FVIII in vivo. Emicizumab is designed for long-term prophylaxis in patients with severe hemophilia A with and without inhibitors. This approach pr...

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Autores principales: Castaman, Giancarlo, Linari, Silvia, Pieri, Lisa, Carulli, Christian, Prosperi, Paolo, Tonelli, Paolo, Demartis, Francesco, Fjerza, Rajmonda, Attanasio, Monica, Coppo, Mirella, Salvianti, Francesca
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10055920/
https://www.ncbi.nlm.nih.gov/pubmed/36983317
http://dx.doi.org/10.3390/jcm12062317
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author Castaman, Giancarlo
Linari, Silvia
Pieri, Lisa
Carulli, Christian
Prosperi, Paolo
Tonelli, Paolo
Demartis, Francesco
Fjerza, Rajmonda
Attanasio, Monica
Coppo, Mirella
Salvianti, Francesca
author_facet Castaman, Giancarlo
Linari, Silvia
Pieri, Lisa
Carulli, Christian
Prosperi, Paolo
Tonelli, Paolo
Demartis, Francesco
Fjerza, Rajmonda
Attanasio, Monica
Coppo, Mirella
Salvianti, Francesca
author_sort Castaman, Giancarlo
collection PubMed
description Emicizumab is a humanized recombinant bispecific antibody, bridging together activated factor IX (FIXa) and factor X (FX), thus mimicking the activity of FVIII in vivo. Emicizumab is designed for long-term prophylaxis in patients with severe hemophilia A with and without inhibitors. This approach provides constant protection, with significant reduction in bleeding rate and improved quality of life. However, protection provided by emicizumab is not absolute, and clotting factor concentrates (FVIII, rFVIIa, aPCC) may be necessary for post-traumatic bleeding or surgery, with a potential thrombotic risk or difficulty in preventing bleeding. Real world evidence is still scanty, especially for managing major surgery. In this study, 75 surgeries were managed in 28 patients (27 major procedures in 15 patients and 48 minor procedures in 20 patients. In 17 patients without inhibitors, 30 minor surgeries were carried out by using FVIII in 5, with only a bleeding event, which was successfully treated with FVIII concentrate. Six major surgeries were uneventfully performed with FVIII concentrate. Eleven PWHA and high-titer inhibitors underwent 39 surgical procedures (18 minor and 21 major surgeries). Minor surgeries were mostly performed without prophylaxis with rFVIIa, with only a single bleeding complication. All 21 major surgeries were covered with a homogeneous protocol using rFVIIa. In four instances, bleeding complications occurred, treated with rFVIIa. Of them, a single patient only failed to respond and died because of an uncontrollable bleeding from a large ruptured retroperitoneal pseudotumor. Surgery in patients with emicizumab can be safely carried out with the use of appropriate replacement therapy protocols.
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spelling pubmed-100559202023-03-30 Safe and Successful Surgical Outcome in Persons with Hemophilia A with and without Inhibitors Treated with Emicizumab: A Large, Single Center, Real-World Experience Castaman, Giancarlo Linari, Silvia Pieri, Lisa Carulli, Christian Prosperi, Paolo Tonelli, Paolo Demartis, Francesco Fjerza, Rajmonda Attanasio, Monica Coppo, Mirella Salvianti, Francesca J Clin Med Article Emicizumab is a humanized recombinant bispecific antibody, bridging together activated factor IX (FIXa) and factor X (FX), thus mimicking the activity of FVIII in vivo. Emicizumab is designed for long-term prophylaxis in patients with severe hemophilia A with and without inhibitors. This approach provides constant protection, with significant reduction in bleeding rate and improved quality of life. However, protection provided by emicizumab is not absolute, and clotting factor concentrates (FVIII, rFVIIa, aPCC) may be necessary for post-traumatic bleeding or surgery, with a potential thrombotic risk or difficulty in preventing bleeding. Real world evidence is still scanty, especially for managing major surgery. In this study, 75 surgeries were managed in 28 patients (27 major procedures in 15 patients and 48 minor procedures in 20 patients. In 17 patients without inhibitors, 30 minor surgeries were carried out by using FVIII in 5, with only a bleeding event, which was successfully treated with FVIII concentrate. Six major surgeries were uneventfully performed with FVIII concentrate. Eleven PWHA and high-titer inhibitors underwent 39 surgical procedures (18 minor and 21 major surgeries). Minor surgeries were mostly performed without prophylaxis with rFVIIa, with only a single bleeding complication. All 21 major surgeries were covered with a homogeneous protocol using rFVIIa. In four instances, bleeding complications occurred, treated with rFVIIa. Of them, a single patient only failed to respond and died because of an uncontrollable bleeding from a large ruptured retroperitoneal pseudotumor. Surgery in patients with emicizumab can be safely carried out with the use of appropriate replacement therapy protocols. MDPI 2023-03-16 /pmc/articles/PMC10055920/ /pubmed/36983317 http://dx.doi.org/10.3390/jcm12062317 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
Castaman, Giancarlo
Linari, Silvia
Pieri, Lisa
Carulli, Christian
Prosperi, Paolo
Tonelli, Paolo
Demartis, Francesco
Fjerza, Rajmonda
Attanasio, Monica
Coppo, Mirella
Salvianti, Francesca
Safe and Successful Surgical Outcome in Persons with Hemophilia A with and without Inhibitors Treated with Emicizumab: A Large, Single Center, Real-World Experience
title Safe and Successful Surgical Outcome in Persons with Hemophilia A with and without Inhibitors Treated with Emicizumab: A Large, Single Center, Real-World Experience
title_full Safe and Successful Surgical Outcome in Persons with Hemophilia A with and without Inhibitors Treated with Emicizumab: A Large, Single Center, Real-World Experience
title_fullStr Safe and Successful Surgical Outcome in Persons with Hemophilia A with and without Inhibitors Treated with Emicizumab: A Large, Single Center, Real-World Experience
title_full_unstemmed Safe and Successful Surgical Outcome in Persons with Hemophilia A with and without Inhibitors Treated with Emicizumab: A Large, Single Center, Real-World Experience
title_short Safe and Successful Surgical Outcome in Persons with Hemophilia A with and without Inhibitors Treated with Emicizumab: A Large, Single Center, Real-World Experience
title_sort safe and successful surgical outcome in persons with hemophilia a with and without inhibitors treated with emicizumab: a large, single center, real-world experience
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10055920/
https://www.ncbi.nlm.nih.gov/pubmed/36983317
http://dx.doi.org/10.3390/jcm12062317
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