Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia
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- dc.contributor.author Newland, Adrian C.
- dc.contributor.author Sánchez González, Blanca
- dc.contributor.author Rejtő, László
- dc.contributor.author Egyed, Miklos
- dc.contributor.author Romanyuk, Nataliya
- dc.contributor.author Godar, Marie
- dc.contributor.author Verschueren, Katrien
- dc.contributor.author Gandini, Domenica
- dc.contributor.author Ulrichts, Peter
- dc.contributor.author Beauchamp, Jon
- dc.contributor.author Dreier, Torsten
- dc.contributor.author Ward, E. Sally
- dc.contributor.author Michel, Marc
- dc.contributor.author Liebman, Howard A.
- dc.contributor.author de Haard, Hans
- dc.contributor.author Leupin, Nicolas
- dc.contributor.author Kuter, David J.
- dc.date.accessioned 2021-02-15T07:57:49Z
- dc.date.available 2021-02-15T07:57:49Z
- dc.date.issued 2020
- dc.description.abstract Primary immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder, characterized by a low platelet count (<100 × 109 /L) in the absence of other causes associated with thrombocytopenia. In most patients, IgG autoantibodies directed against platelet receptors can be detected. They accelerate platelet clearance and destruction, inhibit platelet production, and impair platelet function, resulting in increased risk of bleeding and impaired quality of life. Efgartigimod is a human IgG1 antibody Fc-fragment, a natural ligand of the neonatal Fc receptor (FcRn), engineered for increased affinity to FcRn, while preserving its characteristic pH-dependent binding. Efgartigimod blocks FcRn, preventing IgG recycling, and causing targeted IgG degradation. In this Phase 2 study, 38 patients were randomized 1:1:1 to receive four weekly intravenous infusions of either placebo (N = 12) or efgartigimod at a dose of 5 mg/kg (N = 13) or 10 mg/kg (N = 13). This short treatment cycle of efgartigimod in patients with ITP, predominantly refractory to previous lines of therapy, was shown to be well tolerated, and demonstrated a favorable safety profile consistent with Phase 1 data. Efgartigimod induced a rapid reduction of total IgG levels (up to 63.7% mean change from baseline), which was associated with clinically relevant increases in platelet counts (46% patients on efgartigimod vs 25% on placebo achieved a platelet count of ≥50 × 109 /L on at least two occasions, and 38% vs 0% achieved ≥50 × 109 /L for at least 10 cumulative days), and a reduced proportion of patients with bleeding. Taken together, these data warrant further evaluation of FcRn antagonism as a novel therapeutic approach in ITP.
- dc.format.mimetype application/pdf
- dc.identifier.citation Newland AC, Sánchez-González B, Rejtő L, Egyed M, Romanyuk N, Godar M. et al. Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. Am J Hematol. 2020 Feb; 95(2): 178-87. DOI: 10.1002/ajh.25680
- dc.identifier.doi http://dx.doi.org/10.1002/ajh.25680
- dc.identifier.issn 0361-8609
- dc.identifier.uri http://hdl.handle.net/10230/46475
- dc.language.iso eng
- dc.publisher Wiley-Blackwell
- dc.rights Copyright © 2019 The Authors. American Journal of Hematology published by Wiley Periodicals, Inc. This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
- dc.rights.accessRights info:eu-repo/semantics/openAccess
- dc.rights.uri http://creativecommons.org/licenses/by-nc-nd/4.0/
- dc.subject.other Trombocitopènia
- dc.subject.other Plaquetes sanguínies--Trastorns
- dc.subject.other Immunoglobulina G
- dc.title Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia
- dc.type info:eu-repo/semantics/article
- dc.type.version info:eu-repo/semantics/publishedVersion