561
Views
8
CrossRef citations to date
0
Altmetric
Review

Treatment of pemphigus vulgaris: part 2 – emerging therapies

, &
Pages 1061-1071 | Received 05 Jul 2019, Accepted 23 Sep 2019, Published online: 10 Oct 2019

References

  • Kasperkiewicz M, Ellebrecht CT, Takahashi H, et al. Pemphigus. Nat Rev Dis Primers. 2017;3:17026.
  • Hsu DY, Brieva J, Sinha AA, et al. Comorbidities and inpatient mortality for pemphigus in the U.S.A. Br J Dermatol. 2016;174:1290–1298.
  • Martin LK, Werth VP, Villaneuva EV, et al. A systematic review of randomized controlled trials for pemphigus vulgaris and pemphigus foliaceus. J Am Acad Dermatol. 2011;64:903–908.
  • Ahmed AR, Nguyen T, Kaveri S, et al. First line treatment of pemphigus vulgaris with a novel protocol in patients with contraindications to systemic corticosteroids and immunosuppressive agents: preliminary retrospective study with a seven year follow-up. Int Immunopharmacol. 2016;34:25–31.
  • Joly P, Maho-Vaillant M, Prost-Squarcioni C, et al. First-line rituximab combined with short-term prednisone versus prednisone alone for the treatment of pemphigus (Ritux 3): a prospective, multicentre, parallel-group, open-label randomised trial. Lancet. 2017;389:2031–2040.
  • Cianchini G, Corona R, Frezzolini A, et al. Treatment of severe pemphigus with rituximab: report of 12 cases and a review of the literature. Arch Dermatol. 143,1033–1038 (2007).
  • Lunardon L, Tsai KJ, Propert KJ, et al. Adjuvant rituximab therapy of pemphigus: a single-center experience with 31 patients. Arch Dermatol. 2012;148:1031–1036.
  • Joly P, Mouquet H, Roujeau JC, et al. A single cycle of rituximab for the treatment of severe pemphigus. N Engl J Med. 2007;357:545–552.
  • Murrell DF, Pena S, Joly P, et al. Diagnosis and management of pemphigus: recommendations by an international panel of experts. J Am Acad Dermatol. 2018. S0190-9622(18)30207-X. DOI:10.1016/j.jaad.2018.02.021. Preprint e-pub.
  • Amber KT, Valdebran M, Grando SA. Non-desmoglein antibodies in patients with pemphigus vulgaris. Front Immunol. 2018;9:1190.
  • Sinha AA, Sajda T. The evolving story of autoantibodies in pemphigus vulgaris: development of the “super compensation hypothesis”. Front Med (Lausanne). 2018;5:218.
  • Broussard KC, Leung TG, Moradi A, et al. Autoimmune bullous diseases with skin and eye involvement: cicatricial pemphigoid, pemphigus vulgaris, and pemphigus paraneoplastica. Clin Dermatol. 2016;34:205–213.
  • Yamagami J. Recent advances in the understanding and treatment of pemphigus and pemphigoid. F1000Res. 2018;7:1360.
  • Hammers CM, Chen J, Lin C, et al. Persistence of anti-desmoglein 3 IgG(+) B-cell clones in pemphigus patients over years. J Invest Dermatol. 2015;135:742–749.
  • Musette P, Bouaziz JD. B Cell modulation strategies in autoimmune diseases: new concepts. Front Immunol. 2018;9:622.
  • Truhan AP, Ahmed AR. Corticosteroids: a review with emphasis on complications of prolonged systemic therapy. Ann Allergy. 1989;62:375–391.
  • Ahmed AR, Kaveri S. Reversing autoimmunity combination of rituximab and intravenous immunoglobulin. Front Immunol. 2018;9:1189.
  • Feliciani C, Joly P, Jonkman MF, et al. Management of bullous pemphigoid: the European dermatology forum consensus in collaboration with the European academy of dermatology and venereology. Br J Dermatol. 2015;172:867–877.
  • Mahoney MG, Wang Z, Rothenberger K, et al. Explanations for the clinical and microscopic localization of lesions in pemphigus foliaceus and vulgaris. J Clin Invest. 1999;103:461–468.
  • Amagai M, Tsunoda K, Zillikens D, et al. The clinical phenotype of pemphigus is defined by the anti-desmoglein autoantibody profile. J Am Acad Dermatol. 1999;40:167–170.
  • Schmidt E. Rituximab as first-line treatment of pemphigus. Lancet. 2017;389:1956–1958.
  • Kridin K. Emerging treatment options for the management of pemphigus vulgaris. Ther Clin Risk Manag. 2018;14:757–778.
  • Lever WF, Schaumburg-Lever G. Immunosuppressants and prednisone in pemphigus vulgaris: therapeutic results obtained in 63 patients between 1961 and 1975. Arch Dermatol. 1977;113:1236–1241.
  • Zhao CY, Murrell DF. Pemphigus vulgaris: an evidence-based treatment update. Drugs. 2015;75:271–284.
  • Clemens V, Regen F, Le Bret N, et al. Anti-inflammatory effects of minocycline are mediated by retinoid signaling. BMC Neurosci. 2018;19:58.
  • Olszewska M, Kolacinska-Strasz Z, Sulej J, et al. Efficacy and safety of cyclophosphamide, azathioprine, and cyclosporine (ciclosporin) as adjuvant drugs in pemphigus vulgaris. Am J Clin Dermatol. 2007;8:85–92.
  • Margolis DJ. A randomized trial and the treatment of pemphigus vulgaris. J Invest Dermatol. 2010;130:1964–1966.
  • Mabrouk D, Gurcan HM, Keskin DB, et al. Association between cancer and immunosuppressive therapy–analysis of selected studies in pemphigus and pemphigoid. Ann Pharmacother. 2010;44:1770–1776.
  • Bernatsky S, Clarke AE, Suissa S. Hematologic malignant neoplasms after drug exposure in rheumatoid arthritis. Arch Intern Med. 2008;168:378–381.
  • Vial T, Descotes J. Immunosuppressive drugs and cancer. Toxicology. 2003;185:229–240.
  • Cummins DL, Mimouni D, Anhalt GJ, et al. Oral cyclophosphamide for treatment of pemphigus vulgaris and foliaceus. J Am Acad Dermatol. 2003;49:276–280.
  • Cornaby C, Gibbons L, Mayhew V, et al. B cell epitope spreading: mechanisms and contribution to autoimmune diseases. Immunol Lett. 2015;163:56–68.
  • Housman G, Byler S, Heerboth S, et al. Drug resistance in cancer: an overview. Cancers (Basel). 2014;6:1769–1792.
  • Ahmed AR. Intravenous immunoglobulin therapy in the treatment of patients with pemphigus vulgaris unresponsive to conventional immunosuppressive treatment. J Am Acad Dermatol. 2001;45:679–690.
  • Ahmed AR. Treatment of autoimmune mucocutaneous blistering diseases with intravenous immunoglobulin therapy. Expert Opin Investig Drugs. 2004;13:1019–1032.
  • Akilesh S, Petkova S, Sproule TJ, et al. The MHC class I-like Fc receptor promotes humorally mediated autoimmune disease. J Clin Invest. 2004;113:1328–1333.
  • Ahmed AR, Dahl MV. Consensus statement on the use of intravenous immunoglobulin therapy in the treatment of autoimmune mucocutaneous blistering diseases. Arch Dermatol. 2003;139:1051–1059.
  • Kridin K, Sagi SZ, Bergman R. Mortality and cause of death in patients with pemphigus. Acta Derm Venereol. 2017;97:607–611.
  • Ahmed AR, Shetty S. The emerging role of rituximab in autoimmune blistering diseases. Am J Clin Dermatol. 2015;16:167–177.
  • Ahmed AR, Shetty S. A comprehensive analysis of treatment outcomes in patients with pemphigus vulgaris treated with rituximab. Autoimmun Rev. 2015;14:323–331.
  • Agrawal A, Hall R, Banez L, et al. Comparison of rituximab and conventional adjuvant therapy for pemphigus vulgaris: a retrospective analysis. PLoS One. 2018;13(9):e0198074.
  • Zhu HQ, Xu RC, Chen YY, et al. Impaired function of CD19(+) CD24(hi) CD38(hi) regulatory B cells in patients with pemphigus. Br J Dermatol. 2015;172:101–110.
  • Colliou N, Picard D, Caillot F, et al. Long-term remissions of severe pemphigus after rituximab therapy are associated with prolonged failure of desmoglein B cell response. Sci Transl Med. 2013;5:175ra30.
  • Mimouni D, Anhalt GJ, Cummins DL, et al. Treatment of pemphigus vulgaris and pemphigus foliaceus with mycophenolate mofetil. Arch Dermatol. 2003;139:739–742.
  • Lee J, Werth VP, Hall RP 3rd, et al. Perspective from the 5th International pemphigus and pemphigoid foundation scientific conference. Front Med (Lausanne). 2018;5:306.
  • Werth V, Culton D, Blumberg L, et al. 538 FcRn blockade with SYNT001 for the treatment of pemphigus. J Invest Dermatol. 2018;138:S92.
  • Hebert V, Boulard C, Houivet E, et al. Large international validation of ABSIS and PDAI pemphigus severity scores. J Invest Dermatol. 2019;139:31–37.
  • Koneczny I. A new classification system for IgG4 autoantibodies. Front Immunol. 2018;9:97.
  • Ulrichts P, Guglietta A, Dreier T, et al. Neonatal Fc receptor antagonist efgartigimod safely and sustainably reduces IgGs in humans. J Clin Invest. 2018;128:4372–4386.
  • Bayry J, Kaveri SV. Kill ‘Em All: efgartigimod immunotherapy for autoimmune diseases. Trends Pharmacol Sci. 2018;39:919–922.
  • Kil LP, de Bruijn MJ, van Nimwegen M, et al. Btk levels set the threshold for B-cell activation and negative selection of autoreactive B cells in mice. Blood. 2012;119:3744–3756.
  • Smith PF, Krishnarajah J, Nunn PA, et al. A phase I trial of PRN1008, a novel reversible covalent inhibitor of Bruton’s tyrosine kinase, in healthy volunteers. Br J Clin Pharmacol. 2017;83:2367–2376.
  • Miklos D, Cutler CS, Arora M, et al. Ibrutinib for chronic graft-versus-host disease after failure of prior therapy. Blood. 2017;130:2243–2250.
  • Shah B, Zhao X, Silva AS, et al. Resistance to ibrutinib in B cell malignancies: one size does not fit all. Trends Cancer. 2018;4:197–206.
  • Zhao X, Lwin T, Silva A, et al. Unification of de novo and acquired ibrutinib resistance in mantle cell lymphoma. Nat Commun. 2017;8:14920.
  • Qian H, Kusuhara M, Li X, et al. B-cell activating factor detected on both naive and memory B cells in bullous pemphigoid. Exp Dermatol. 2014;23:596–605.
  • Dorner T, Posch MG, Li Y, et al. Treatment of primary Sjogren’s syndrome with ianalumab (VAY736) targeting B cells by BAFF receptor blockade coupled with enhanced, antibody-dependent cellular cytotoxicity. Ann Rheum Dis. 2019;78:641–647.
  • St Clair EW, Levesque MC, Prak ET, et al. Rituximab therapy for primary Sjogren’s syndrome: an open-label clinical trial and mechanistic analysis. Arthritis Rheum. 2013;65:1097–1106.
  • Carter LM, Isenberg DA, Ehrenstein MR. Elevated serum BAFF levels are associated with rising anti-double-stranded DNA antibody levels and disease flare following B cell depletion therapy in systemic lupus erythematosus. Arthritis Rheum. 2013;65:2672–2679.
  • Mahevas M, Patin P, Huetz F, et al. B cell depletion in immune thrombocytopenia reveals splenic long-lived plasma cells. J Clin Invest. 2013;123:432–442.
  • Kraaij T, Kamerling SW, de Rooij EN, et al. The NET-effect of combining rituximab with belimumab in severe systemic lupus erythematosus. J Autoimmun. 2018;91:45–54.
  • Nagel A, Podstawa E, Eickmann M, et al. Rituximab mediates a strong elevation of B-cell-activating factor associated with increased pathogen-specific IgG but not autoantibodies in pemphigus vulgaris. J Invest Dermatol. 2009;129:2202–2210.
  • Tangye SG, Bryant VL, Cuss AK, et al. BAFF, APRIL and human B cell disorders. Semin Immunol. 2006;18:305–317.
  • Asashima N, Fujimoto M, Watanabe R, et al. Serum levels of BAFF are increased in bullous pemphigoid but not in pemphigus vulgaris. Br J Dermatol. 2006;155:330–336.
  • Torkamani N, Kamali SE, Naseri M. Investigation of serum APRIL and BAFF levels in pemphigus vulgaris patients in Southern Iran. Iran J of Dermatol. 2011;14:16–19.
  • Michalovich D, Nejentsev S. Activated PI3 kinase delta syndrome: from genetics to therapy. Front Immunol. 2018;9:369.
  • Gadina M, Gazaniga N, Vian L, et al. Small molecules to the rescue: inhibition of cytokine signaling in immune-mediated diseases. J Autoimmun. 2017;85:20–31.
  • Winkler DG, Faia KL, DiNitto JP, et al. PI3K-δ and PI3K-γ inhibition by IPI-145 abrogates immune responses and suppresses activity in autoimmune and inflammatory disease models. Chem Biol. 2013;20:1364–1374.
  • Ellebrecht CT, Bhoj VG, Nace A, et al. Reengineering chimeric antigen receptor T cells for targeted therapy of autoimmune disease. Science. 2016;353:179–184.
  • Ellebrecht CT, Payne AS. Setting the target for pemphigus vulgaris therapy. JCI Insight. 2017;2:e92021.
  • Siddiqi HF, Staser KW, Nambudiri VE. Research Techniques made simple: CAR T-cell therapy. J Invest Dermatol. 2018;138:2501–2504.e1.
  • Rubin CB, Elenitsas R, Taylor L, et al. Evaluating the skin in patients undergoing chimeric antigen receptor modified T-cell therapy. J Am Acad Dermatol. 2016;75:1054–1057.
  • Mao X, Payne AS. Seeking approval: present and future therapies for pemphigus vulgaris. Curr Opin Invest Drugs. 2008;9:497–504.
  • Wucherpfennig KW, Yu B, Bhol K, et al. Structural basis for major histocompatibility complex (MHC)-linked susceptibility to autoimmunity: charged residues of a single MHC binding pocket confer selective presentation of self-peptides in pemphigus vulgaris. Proc Natl Acad Sci U S A. 1995;92:11935–11939.
  • Anhalt G, Werth V, Strober B. et al., editors. An open-label phase I clinical study to assess the safety of PI-0824 in patients with pemphigus vulgaris. J Invest Dermatol. 2005;125.
  • Stern JN, Illes Z, Reddy J, et al. Amelioration of proteolipid protein 139-151-induced encephalomyelitis in SJL mice by modified amino acid copolymers and their mechanisms. Proc Natl Acad Sci U S A. 2004;101:11743–11748.
  • Kovalchin J, Krieger J, Collins K, et al. Safety, pharmacokinetic, and pharmacodynamic evaluations of PI-2301, a potent immunomodulator, in a first-in-human, single-ascending-dose study in healthy volunteers. J Clin Pharmacol. 2011;51:649–660.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.