Modulating IL-21-driven B cell responses in idiopathic inflammatory myopathies via inhibition of the JAK/STAT pathway

Abstract Background Idiopathic inflammatory myopathies (IIM) are autoimmune disorders characterized by muscle inflammation and autoreactive B cell responses. The Janus kinase (JAK)-signal transducer and activator of transcription (STAT) signaling pathway is essential for B cell functions, making it...

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Main Authors: Ana Merino-Vico, Merve Kocyigit, Giulia Frazzei, Lisa Landman, Louis Boon, Ester M. van Leeuwen, Ingrid E. Lundberg, Anneke J. van der Kooi, Joost Raaphorst, Jan Piet van Hamburg, Sander W. Tas
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Language:English
Published: BMC 2025-04-01
Series:Arthritis Research & Therapy
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Online Access:https://doi.org/10.1186/s13075-025-03547-2
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author Ana Merino-Vico
Merve Kocyigit
Giulia Frazzei
Lisa Landman
Louis Boon
Ester M. van Leeuwen
Ingrid E. Lundberg
Anneke J. van der Kooi
Joost Raaphorst
Jan Piet van Hamburg
Sander W. Tas
author_facet Ana Merino-Vico
Merve Kocyigit
Giulia Frazzei
Lisa Landman
Louis Boon
Ester M. van Leeuwen
Ingrid E. Lundberg
Anneke J. van der Kooi
Joost Raaphorst
Jan Piet van Hamburg
Sander W. Tas
author_sort Ana Merino-Vico
collection DOAJ
description Abstract Background Idiopathic inflammatory myopathies (IIM) are autoimmune disorders characterized by muscle inflammation and autoreactive B cell responses. The Janus kinase (JAK)-signal transducer and activator of transcription (STAT) signaling pathway is essential for B cell functions, making it a promising therapeutic target. This study explores the potential of tofacitinib, a JAK1/JAK3 inhibitor, to modulate B cell activity in IIM. Methods Peripheral B cell populations from dermatomyositis (DM), anti-synthetase syndrome (ASyS) and overlap myositis (OM) patients were analyzed by flow cytometry. Peripheral blood mononuclear cells (PBMC) or sorted memory B cells were cultured with tofacitinib and stimulated with combinations of CD40, IL-21, IL-2, BAFF and CpG. B cell proliferation, differentiation and (auto)antibody, cytokine/chemokine production were assessed by flow cytometry, Luminex, and ELISA/ELiA assays. Results The IIM peripheral B cell compartment had elevated transitional and naive B cells, with reduced Bmem frequencies compared to healthy donors. Tofacitinib significantly inhibited CD40/IL-21-induced B cell proliferation, plasmablast formation and function in PBMC and B cell-only cultures across all IIM subgroups, predominantly affecting the IL-21-induced differentiation and antibody production. Remarkably, tofacitinib reduced the levels of anti-Jo1 autoantibodies, as well as of CXCL10 and CXCL13 in ASyS memory B cell cultures. Conclusions These findings highlight the B cell involvement in IIM, evidenced by altered peripheral B cell composition in active disease and the effective inhibition of essential B cell responses, including proliferation, differentiation, and (auto)antibody production, by tofacitinib in vitro. This positions the JAK/STAT pathway as a promising new therapeutic target to modulate B cell activity in IIM.
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spelling doaj-art-05c19caae36e4a6e95db817412f7fe4e2025-08-20T03:07:43ZengBMCArthritis Research & Therapy1478-63622025-04-0127111210.1186/s13075-025-03547-2Modulating IL-21-driven B cell responses in idiopathic inflammatory myopathies via inhibition of the JAK/STAT pathwayAna Merino-Vico0Merve Kocyigit1Giulia Frazzei2Lisa Landman3Louis Boon4Ester M. van Leeuwen5Ingrid E. Lundberg6Anneke J. van der Kooi7Joost Raaphorst8Jan Piet van Hamburg9Sander W. Tas10Department of Rheumatology and Clinical Immunology, Amsterdam Rheumatology and Immunology Center, Amsterdam University Medical Center, University of AmsterdamDepartment of Rheumatology and Clinical Immunology, Amsterdam Rheumatology and Immunology Center, Amsterdam University Medical Center, University of AmsterdamDepartment of Rheumatology and Clinical Immunology, Amsterdam Rheumatology and Immunology Center, Amsterdam University Medical Center, University of AmsterdamDepartment of Rheumatology and Clinical Immunology, Amsterdam Rheumatology and Immunology Center, Amsterdam University Medical Center, University of AmsterdamJJP BiologicsDepartment of Experimental Immunology, Amsterdam University Medical Center, University of AmsterdamDivision of Rheumatology, Department of Medicine, Karolinska Institutet and Rheumatology, Karolinska University HospitalDepartment of Rheumatology and Clinical Immunology, Amsterdam Rheumatology and Immunology Center, Amsterdam University Medical Center, University of AmsterdamDepartment of Rheumatology and Clinical Immunology, Amsterdam Rheumatology and Immunology Center, Amsterdam University Medical Center, University of AmsterdamDepartment of Rheumatology and Clinical Immunology, Amsterdam Rheumatology and Immunology Center, Amsterdam University Medical Center, University of AmsterdamDepartment of Rheumatology and Clinical Immunology, Amsterdam Rheumatology and Immunology Center, Amsterdam University Medical Center, University of AmsterdamAbstract Background Idiopathic inflammatory myopathies (IIM) are autoimmune disorders characterized by muscle inflammation and autoreactive B cell responses. The Janus kinase (JAK)-signal transducer and activator of transcription (STAT) signaling pathway is essential for B cell functions, making it a promising therapeutic target. This study explores the potential of tofacitinib, a JAK1/JAK3 inhibitor, to modulate B cell activity in IIM. Methods Peripheral B cell populations from dermatomyositis (DM), anti-synthetase syndrome (ASyS) and overlap myositis (OM) patients were analyzed by flow cytometry. Peripheral blood mononuclear cells (PBMC) or sorted memory B cells were cultured with tofacitinib and stimulated with combinations of CD40, IL-21, IL-2, BAFF and CpG. B cell proliferation, differentiation and (auto)antibody, cytokine/chemokine production were assessed by flow cytometry, Luminex, and ELISA/ELiA assays. Results The IIM peripheral B cell compartment had elevated transitional and naive B cells, with reduced Bmem frequencies compared to healthy donors. Tofacitinib significantly inhibited CD40/IL-21-induced B cell proliferation, plasmablast formation and function in PBMC and B cell-only cultures across all IIM subgroups, predominantly affecting the IL-21-induced differentiation and antibody production. Remarkably, tofacitinib reduced the levels of anti-Jo1 autoantibodies, as well as of CXCL10 and CXCL13 in ASyS memory B cell cultures. Conclusions These findings highlight the B cell involvement in IIM, evidenced by altered peripheral B cell composition in active disease and the effective inhibition of essential B cell responses, including proliferation, differentiation, and (auto)antibody production, by tofacitinib in vitro. This positions the JAK/STAT pathway as a promising new therapeutic target to modulate B cell activity in IIM.https://doi.org/10.1186/s13075-025-03547-2B cellMyositisJAKPlasma cellTofacitinib
spellingShingle Ana Merino-Vico
Merve Kocyigit
Giulia Frazzei
Lisa Landman
Louis Boon
Ester M. van Leeuwen
Ingrid E. Lundberg
Anneke J. van der Kooi
Joost Raaphorst
Jan Piet van Hamburg
Sander W. Tas
Modulating IL-21-driven B cell responses in idiopathic inflammatory myopathies via inhibition of the JAK/STAT pathway
Arthritis Research & Therapy
B cell
Myositis
JAK
Plasma cell
Tofacitinib
title Modulating IL-21-driven B cell responses in idiopathic inflammatory myopathies via inhibition of the JAK/STAT pathway
title_full Modulating IL-21-driven B cell responses in idiopathic inflammatory myopathies via inhibition of the JAK/STAT pathway
title_fullStr Modulating IL-21-driven B cell responses in idiopathic inflammatory myopathies via inhibition of the JAK/STAT pathway
title_full_unstemmed Modulating IL-21-driven B cell responses in idiopathic inflammatory myopathies via inhibition of the JAK/STAT pathway
title_short Modulating IL-21-driven B cell responses in idiopathic inflammatory myopathies via inhibition of the JAK/STAT pathway
title_sort modulating il 21 driven b cell responses in idiopathic inflammatory myopathies via inhibition of the jak stat pathway
topic B cell
Myositis
JAK
Plasma cell
Tofacitinib
url https://doi.org/10.1186/s13075-025-03547-2
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