Efgartigimod alfa for the treatment of primary immune thrombocytopenia
Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia. Most patients with ITP have antiplatelet antibodies of the immunoglobulin G (IgG) subtype which through interaction with platelet and megakaryocyte glycoproteins result in increased p...
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Format: | Article |
Language: | English |
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SAGE Publishing
2023-05-01
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Series: | Therapeutic Advances in Hematology |
Online Access: | https://doi.org/10.1177/20406207231172831 |
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author | Catherine Broome |
author_facet | Catherine Broome |
author_sort | Catherine Broome |
collection | DOAJ |
description | Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia. Most patients with ITP have antiplatelet antibodies of the immunoglobulin G (IgG) subtype which through interaction with platelet and megakaryocyte glycoproteins result in increased platelet destruction and inhibition of platelet production. There are a variety of therapeutic options available for the treatment of ITP including corticosteroids, IVIgG, TPO-RA, rituximab, fostamatinib, and splenectomy. Long-term remissions with any of these therapies can vary widely and patients may require additional therapy. The neonatal Fc receptor (FcRn) plays a pivotal role in IgG and albumin physiology through recycling pathways. Efgartigimod is a human IgG1-derived fragment that has been modified by ABDEG technology to increase its affinity for FcRn at both physiologic and acidic pH. The binding of efgartigimod to FcRn blocks the interaction of IgG with FcRn facilitating increased lysosomal degradation of IgG and decreasing total IgG levels. Based on the mechanism of action and the known pathophysiology of ITP as well as the efficacy of other therapies such as intravenous immunoglobulin (IVIG), the use of efgartigimod in patients with ITP is attractive. This article will briefly discuss the pathophysiology of ITP, current treatments, and the data available on efgartigimod in ITP. |
first_indexed | 2024-04-09T13:25:06Z |
format | Article |
id | doaj.art-3153ab6427dd42aeb663e41fdcba33e7 |
institution | Directory Open Access Journal |
issn | 2040-6215 |
language | English |
last_indexed | 2024-04-09T13:25:06Z |
publishDate | 2023-05-01 |
publisher | SAGE Publishing |
record_format | Article |
series | Therapeutic Advances in Hematology |
spelling | doaj.art-3153ab6427dd42aeb663e41fdcba33e72023-05-10T11:34:41ZengSAGE PublishingTherapeutic Advances in Hematology2040-62152023-05-011410.1177/20406207231172831Efgartigimod alfa for the treatment of primary immune thrombocytopeniaCatherine BroomePrimary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia. Most patients with ITP have antiplatelet antibodies of the immunoglobulin G (IgG) subtype which through interaction with platelet and megakaryocyte glycoproteins result in increased platelet destruction and inhibition of platelet production. There are a variety of therapeutic options available for the treatment of ITP including corticosteroids, IVIgG, TPO-RA, rituximab, fostamatinib, and splenectomy. Long-term remissions with any of these therapies can vary widely and patients may require additional therapy. The neonatal Fc receptor (FcRn) plays a pivotal role in IgG and albumin physiology through recycling pathways. Efgartigimod is a human IgG1-derived fragment that has been modified by ABDEG technology to increase its affinity for FcRn at both physiologic and acidic pH. The binding of efgartigimod to FcRn blocks the interaction of IgG with FcRn facilitating increased lysosomal degradation of IgG and decreasing total IgG levels. Based on the mechanism of action and the known pathophysiology of ITP as well as the efficacy of other therapies such as intravenous immunoglobulin (IVIG), the use of efgartigimod in patients with ITP is attractive. This article will briefly discuss the pathophysiology of ITP, current treatments, and the data available on efgartigimod in ITP.https://doi.org/10.1177/20406207231172831 |
spellingShingle | Catherine Broome Efgartigimod alfa for the treatment of primary immune thrombocytopenia Therapeutic Advances in Hematology |
title | Efgartigimod alfa for the treatment of primary immune thrombocytopenia |
title_full | Efgartigimod alfa for the treatment of primary immune thrombocytopenia |
title_fullStr | Efgartigimod alfa for the treatment of primary immune thrombocytopenia |
title_full_unstemmed | Efgartigimod alfa for the treatment of primary immune thrombocytopenia |
title_short | Efgartigimod alfa for the treatment of primary immune thrombocytopenia |
title_sort | efgartigimod alfa for the treatment of primary immune thrombocytopenia |
url | https://doi.org/10.1177/20406207231172831 |
work_keys_str_mv | AT catherinebroome efgartigimodalfaforthetreatmentofprimaryimmunethrombocytopenia |