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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Main Author: Catherine Broome
Format: Article
Language:English
Published: SAGE Publishing 2023-05-01
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.
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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