Translational autoimmunity in pemphigus and the role of novel Bruton tyrosine kinase inhibitors

Egami, 2020, Autoimmune bullous skin diseases, pemphigus and pemphigoid, J. Allergy Clin. Immunol., 145, 10.1016/j.jaci.2020.02.013 Didona, 2019, Pemphigus: current and future therapeutic strategies, Front. Immunol., 10, 10.3389/fimmu.2019.01418 Yamagami, 2021, A multicenter, open-label, uncontrolled, single-arm phase 2 study of tirabrutinib, an oral Bruton's tyrosine kinase inhibitor, in pemphigus, J. Dermatol. Sci., 103, 135, 10.1016/j.jdermsci.2021.07.002 Di Paolo, 2011, Specific Btk inhibition suppresses B cell-and myeloid cell-mediated arthritis, Nat. Chem. Biol., 7, 10.1038/nchembio.481 Honigberg, 2010, The Bruton tyrosine kinase inhibitor PCI-32765 blocks B-cell activation and is efficacious in models of autoimmune disease and B-cell malignancy, Proc. Natl Acad. Sci. U. S. A., 107, 13075, 10.1073/pnas.1004594107 Xu, 2012, RN486, a selective Bruton's tyrosine kinase inhibitor, abrogates immune hypersensitivity responses and arthritis in rodents, J. Pharmacol. Exp. Therapeut., 341, 90, 10.1124/jpet.111.187740 Haselmayer, 2019, Efficacy and pharmacodynamic modeling of the BTK inhibitor evobrutinib in autoimmune disease models, J. Immunol., 202, 2888, 10.4049/jimmunol.1800583 Hutcheson, 2012, Modulating proximal cell signaling by targeting Btk ameliorates humoral autoimmunity and end-organ disease in murine lupus, Arthritis Res. Ther., 14, 10.1186/ar4086 Richmond, 2014, Immunology and skin in health and disease, Cold Spring Harb. Perspect. Med., 4, 10.1101/cshperspect.a015339 Singh, 2018, Role of Bruton's tyrosine kinase in B cells and malignancies, Mol. Cancer, 17 P, 2019 LLC, 2019 Angst, 2020, Discovery of LOU064 (Remibrutinib), a potent and highly selective covalent inhibitor of Bruton's tyrosine kinase, J. Med. Chem., 63, 5102, 10.1021/acs.jmedchem.9b01916 Pisanti, 1974, Pemphigus vulgaris: incidence in Jews of different ethnic groups, according to age, sex, and initial lesion, Oral Surg, Oral Med. Oral Pathol., 38, 382, 10.1016/0030-4220(74)90365-X Kridin, 2017, Pemphigus vulgaris and pemphigus foliaceus: differences in epidemiology and mortality, Acta Derm. Venereol., 97, 1095, 10.2340/00015555-2706 Simon, 1980, Pemphigus in hartford county, Connecticut, from 1972 to 1977, Arch. Dermatol., 116, 1035, 10.1001/archderm.1980.01640330073017 Kridin, 2018, Pemphigus group: overview, epidemiology, mortality, and comorbidities, Immunol. Res., 66, 255, 10.1007/s12026-018-8986-7 Sun, 2019, Investigation of the predisposing factor of pemphigus and its clinical subtype through a genome-wide association and next generation sequence analysis, J. Eur. Acad. Dermatol. Venereol., 33, 410, 10.1111/jdv.15227 del Mar Sáez-de-Ocariz, 2005, HLA-DRB1*0101 is associated with foliaceous pemphigus in Mexicans, Int. J. Dermatol., 44, 350, 10.1111/j.1365-4632.2005.02038.x Crofford, 2016, The role of Bruton's tyrosine kinase in autoimmunity and implications for therapy, Expet Rev. Clin. Immunol., 12, 763, 10.1586/1744666X.2016.1152888 Rip, 2018, The role of bruton's tyrosine kinase in immune cell signaling and systemic autoimmunity, Crit. Rev. Immunol., 38, 17, 10.1615/CritRevImmunol.2018025184 Quaresma, 2019, Organization of the skin immune system and compartmentalized immune responses in infectious diseases, Clin. Microbiol. Rev., 32, 10.1128/CMR.00034-18 Chang, 2011, The Bruton tyrosine kinase inhibitor PCI-32765 ameliorates autoimmune arthritis by inhibition of multiple effector cells, Arthritis Res. Ther., 13, 10.1186/ar3400 Lorenzo-Vizcaya, 2020, Bruton's tyrosine kinase inhibitors: a new therapeutic target for the treatment of SLE?, ImmunoTargets Ther., 9, 105, 10.2147/ITT.S240874 Bisconte, 2015 Herter, 2018, PRN473, an inhibitor of Bruton's tyrosine kinase, inhibits neutrophil recruitment via inhibition of macrophage antigen 1 signalling, Br. J. Pharmacol., 175, 429, 10.1111/bph.14090 Goodale, 2020, Efficacy of a Bruton's tyrosine kinase Inhibitor (PRN 473) in the treatment of canine pemphigus foliaceus, Vet. Dermatol., 31, 291, 10.1111/vde.12841 Schmidt, 2019, Pemphigus, Lancet, 394, 882, 10.1016/S0140-6736(19)31778-7 Pollmann, 2018, Pemphigus: a comprehensive review on pathogenesis, clinical presentation and novel therapeutic approaches, Clin. Rev. Allergy Immunol., 54, 1, 10.1007/s12016-017-8662-z Kitajima, 2014, 150th anniversary series: desmosomes and autoimmune disease, perspective of dynamic desmosome remodeling and its impairments in pemphigus, Cell Commun. Adhes., 21, 269, 10.3109/15419061.2014.943397 Walter, 2019, Role of Dsg1- and Dsg3-mediated signaling in pemphigus autoantibody-induced loss of keratinocyte cohesion, Front. Immunol., 10, 1128, 10.3389/fimmu.2019.01128 Bieber, 2020, Milestones in personalized medicine in pemphigus and pemphigoid, Front. Immunol., 11, 591971, 10.3389/fimmu.2020.591971 Hammers, 2020, Recent advances in understanding pemphigus and bullous pemphigoid, J. Invest. Dermatol., 140, 733, 10.1016/j.jid.2019.11.005 Hammers, 2016, Mechanisms of disease: pemphigus and bullous pemphigoid, Annu. Rev. Pathol., 11, 175, 10.1146/annurev-pathol-012615-044313 Berkowitz, 2005, Desmosome signaling. Inhibition of p38MAPK prevents pemphigus vulgaris IgG-induced cytoskeleton reorganization, J. Biol. Chem., 280, 23778, 10.1074/jbc.M501365200 Jolly, 2010, P38MAPK signaling and Desmoglein-3 internalization are linked events in pemphigus acantholysis, J. Biol. Chem., 285, 8936, 10.1074/jbc.M109.087999 Lee, 2009, Biphasic activation of p38MAPK suggests that apoptosis is a downstream event in pemphigus acantholysis, J. Biol. Chem., 284, 12524, 10.1074/jbc.M808204200 Berkowitz, 2008, Induction of p38MAPK and HSP27 phosphorylation in pemphigus patient skin, J. Invest. Dermatol., 128, 738, 10.1038/sj.jid.5701080 Lee, 2017, Successful use of Bruton's kinase inhibitor, ibrutinib, to control paraneoplastic pemphigus in a patient with paraneoplastic autoimmune multiorgan syndrome and chronic lymphocytic leukaemia, Australas. J. Dermatol., 58, e240, 10.1111/ajd.12615 Ito, 2018, Paraneoplastic pemphigus associated with B-cell chronic lymphocytic leukemia treated with Ibrutinib and rituximab, Intern. Med., 57, 2395, 10.2169/internalmedicine.0578-17 Wieczorek, 2016, Paraneoplastic pemphigus: a short review, Clin. Cosmet. Invest. Dermatol., 9, 291, 10.2147/CCID.S100802 Frew, 2011, Current management strategies in paraneoplastic pemphigus (paraneoplastic autoimmune multiorgan syndrome), Dermatol. Clin., 29, 607, 10.1016/j.det.2011.06.016 Smith, 2017, A phase I trial of PRN1008, a novel reversible covalent inhibitor of Bruton's tyrosine kinase, in healthy volunteers, Br. J. Clin. Pharmacol., 83, 2367, 10.1111/bcp.13351 Bilgic Temel, 2019, Pharmacological advances in pemphigus, Curr. Opin. Pharmacol., 46, 44, 10.1016/j.coph.2019.01.001 Goodale, 2020, Open trial of Bruton's tyrosine kinase inhibitor (PRN1008) in the treatment of canine pemphigus foliaceus, Vet. Dermatol., 31, 410, 10.1111/vde.12878 Murrell, 2021, Proof of concept for the clinical effects of oral rilzabrutinib, the first Bruton tyrosine kinase inhibitor for pemphigus vulgaris: the phase II BELIEVE study, Br. J. Dermatol., 185, 745, 10.1111/bjd.20431 Yuko, 2019, vol. 9, 2 Gabizon, 2020, A fast and clean BTK inhibitor, J. Med. Chem., 63, 5100, 10.1021/acs.jmedchem.0c00597 MacGlashan, 2010, Expression of CD203c and CD63 in human basophils: relationship to differential regulation of piecemeal and anaphylactic degranulation processes, Clin. Exp. Allergy, 40, 1365, 10.1111/j.1365-2222.2010.03572.x Kaul, 2021, Remibrutinib (LOU064): a selective potent oral BTK inhibitor with promising clinical safety and pharmacodynamics in a randomized phase I trial, Clin. Transl. Sci., 14, 1756, 10.1111/cts.13005