Janus Kinase Inhibitor Tofacitinib Shows Potent Efficacy in a Mouse Model of Autoimmune Lymphoproliferative Syndrome (ALPS)

Journal of Clinical Immunology

Volumn 35, Issue 7, 2015, Pages 661-667

  Yokoyama, Seiji ^a   Perera, Pin Yu ^b   Terawaki, Seigo ^a   Watanabe, Nobumasa ^a   Kaminuma, Osamu ^a   Waldmann, Thomas A ^c   Hiroi, Takachika ^a   Perera, Liyanage P ^c  

^a TOKYO METROPOLITAN INSTITUTE OF MEDICAL SCIENCE   (Japan)

^b VETERANS AFFAIRS MEDICAL CENTER   (United States)

^c NATIONAL CANCER INSTITUTE   (United States)

Author keywords

ALPS; Autoimmunity; lymphoproliferation; SLE; tofacitinib

Indexed keywords

IMMUNOGLOBULIN A; IMMUNOGLOBULIN G; TOFACITINIB; FAS ANTIGEN; FAS PROTEIN, MOUSE; IMMUNOGLOBULIN; JANUS KINASE 3; PIPERIDINE DERIVATIVE; PROTEIN KINASE INHIBITOR; PYRIMIDINE DERIVATIVE; PYRROLE DERIVATIVE;

ADULT; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; AUTOIMMUNE LYMPHOPROLIFERATIVE SYNDROME; BLOOD; CD4+ T LYMPHOCYTE; CD8+ T LYMPHOCYTE; EXPERIMENTAL MODEL; FAS GENE; GENE; IMMUNOGLOBULIN BLOOD LEVEL; LEUKOCYTE COUNT; LOSS OF FUNCTION MUTATION; MOUSE; NONHUMAN; PLASMA CELL; PLASMABLAST; PRIORITY JOURNAL; REGULATORY T LYMPHOCYTE; RHEUMATOID ARTHRITIS; SPLEEN; ANIMAL; ANTAGONISTS AND INHIBITORS; C57BL MOUSE; DISEASE MODEL; DRUG EFFECTS; GENETICS; HUMAN; IMMUNOLOGY; MURPHY ROTHS LARGE LYMPHOPROLIFERATIVE MOUSE; MUTATION; PHYSIOLOGY; T LYMPHOCYTE SUBPOPULATION;

ANIMALS; ANTIGENS, CD95; AUTOIMMUNE LYMPHOPROLIFERATIVE SYNDROME; DISEASE MODELS, ANIMAL; HUMANS; IMMUNOGLOBULINS; JANUS KINASE 3; MICE; MICE, INBRED C57BL; MICE, INBRED MRL LPR; MUTATION; PIPERIDINES; PROTEIN KINASE INHIBITORS; PYRIMIDINES; PYRROLES; T-LYMPHOCYTE SUBSETS; T-LYMPHOCYTES, REGULATORY;

EID: 84946486019     PISSN: 02719142     EISSN: 15732592     Source Type: Journal    
DOI: 10.1007/s10875-015-0203-z     Document Type: Article

References (29)

1. Canale VC, Smith CH. Chronic lymphadenopathy simulating malignant lymphoma. J Pediatr. 1967;70:891–9.
2. Sneller MC, Straus SE, Jaffe ES, Jaffe JS, Fleisher TA, Stetler-Stevenson M, et al. A novel lymphoproliferative/autoimmune syndrome resembling murine lpr/gld disease. J Clin Invest. 1992;90:334–41.
3. Rieux-Laucat F, Le Deist F, Hivroz C, Roberts IA, Debatin KM, Fischer A, et al. Mutations in Fas associated with human lymphoproliferative syndrome and autoimmunity. Science. 1995;268:1347–9.
4. Fisher GH, Rosenburg FJ, Straus SE, Dale JK, Middleton LA, Lin AY, et al. Dominant interfering Fas gene mutations impair apoptosis in a human autoimmune lymphoproliferative syndrome. Cell. 1995;81:935–46.
5. Straus SE, Jaffe ES, Puck JM, Dale JK, Elkon KB, Rösen-Wolff A, et al. The development of lymphomas in families with autoimmune lymphoproliferative syndrome with germline Fas mutations and defective lymphocyte apoptosis. Blood. 2001;98:194–200.
6. Madkaikar M, Mhatre S, Gupta M, Ghosh K. Advances in autoimmune lymphoproliferative syndromes. Eur J Haematol. 2011;87:1–9.
7. Rao VK, Oliveira JB. How I treat autoimmune lymphoproliferative syndrome. Blood. 2011;118:5741–51.
8. Teachey DT, Greiner R, Seif A, Attiyeh E, Bleesing J, Choi J, et al. Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome. Br J Haematol. 2009;145:101–6.
9. Yokoyama S, Takada K, Hirasawa M, Perera LP, Hiroi T. Transgenic mice that overexpress human IL-15 in enterocytes recapitulate both B and T cell-mediated pathologic manifestations of celiac disease. J Clin Immunol. 2011;31:1038–44.
10. Watanabe-Fukunaga R, Brannan CI, Copeland NG, Jenkins NA, Nagata S. Lymphoproliferation disorder in mice explained by defects in Fas antigen that mediates apoptosis. Nature. 1992;356:314–7.
11. Teachey DT, Obzut DA, Axsom K, Choi JK, Goldsmith KC, Hall J, et al. Rapamycin improves lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome (ALPS). Blood. 2006;108:1965–71.
12. Dowdell KC, Pesnicak L, Hoffmann V, Steadman K, Remaley AT, Cohen JI, et al. Valproic acid (VPA), a histone deacetylase (HDAC) inhibitor, diminishes lymphoproliferation in the Fas-deficient MRL/lpr(-/-) murine model of autoimmune lymphoproliferative syndrome (ALPS). Exp Hematol. 2009;37:487–94.
13. Holzelova E, Vonarbourg C, Stolzenberg MC, Arkwright PD, Selz F, Prieur AM, et al. Autoimmune lymphoproliferative syndrome with somatic Fas mutations. N Engl J Med. 2004;351:1409–18.
14. Magerus-Chatinet A, Neven B, Stolzenberg MC, Daussy C, Arkwright PD, Lanzarotti N, et al. Onset of autoimmune lymphoproliferative syndrome (ALPS) in humans as a consequence of genetic defect accumulation. J Clin Invest. 2011;121:106–12.
15. Hauck F, Magerus-Chatinet A, Vicca S, Rensing-Ehl A, Roesen-Wolff A, Roesler J, et al. Somatic loss of heterozygosity, but not haploinsufficiency alone, leads to full-blown autoimmune lymphoproliferative syndrome in 1 of 12 family members with FAS start codon mutation. Clin Immunol. 2013;147:61–8.
16. Rensing-Ehl A, Völkl S, Speckmann C, Lorenz MR, Ritter J, Janda A, et al. Abnormally differentiated CD4+ or CD8+ T cells with phenotypic and genetic features of double negative T cells in human Fas deficiency. Blood. 2014;124:851–60.
17. Pesu M, Laurence A, Kishore N, Zwillich SH, Chan G, O’Shea JJ. Therapeutic targeting of Janus kinases. Immunol Rev. 2008;223:132–42.
18. Ghoreschi K, Jesson MI, Li X, Lee JL, Ghosh S, Alsup JW, et al. Modulation of innate and adaptive immune responses by tofacitinib (CP-690,550). J Immunol. 2011;186:4234–43.
19. Fleischmann R, Kremer J, Cush J, Schulze-Koops H, Connell CA, Bradley JD, et al. Placebo-controlled trial of tofacitinib monotherapy in rheumatoid arthritis. N Engl J Med. 2012;367:495–507.
20. Garbe K. Pfizer’s JAK inhibitor sails through phase 3 in rheumatoid arthritis. Nat Biotechnol. 2011;29:467–8.
21. Sewgobind VD, Quaedackers ME, van der Laan LJ, Kraaijeveld R, Korevaar SS, Chan G, et al. The Jak inhibitor CP-690,550 preserves the function of CD4CD25FoxP3 regulatory T cells and inhibits effector T cells. Am J Transplant. 2010;10:1785–95.
22. Bristeau-Leprince A, Mateo V, Lim A, Magerus-Chatinet A, Solary E, Fischer A, et al. Human TCR alpha/beta + CD4-CD8- double-negative T cells in patients with autoimmune lymphoproliferative syndrome express restricted Vbeta TCR diversity and are clonally related to CD8+ T cells. J Immunol. 2008;181:440–8.
23. Bleesing JJ, Brown MR, Dale JK, Straus SE, Lenardo MJ, Puck JM, et al. TCR-alpha/beta(+) CD4(-)CD8(-) T cells in humans with the autoimmune lymphoproliferative syndrome express a novel CD45 isoform that is analogous to murine B220 and represents a marker of altered O-glycan biosynthesis. Clin Immunol. 2001;100:314–24.
24. Marlies A, Udo G, Juergen B, Bernd S, Herrmann M, Haas JP. The expanded double negative T cell populations of a patient with ALPS are not clonally related to CD4+ or to CD8+ T cells. Autoimmunity. 2007;40:299–301.
25. Oliveira JB, Bleesing JJ, Dianzani U, Fleisher TA, Jaffe ES, Lenardo MJ, et al. Revised diagnostic criteria and classification for the autoimmune lymphoproliferative syndrome (ALPS): report from the 2009 NIH International Workshop. Blood. 2010;116:e35–40.
26. Price S, Shaw PA, Seitz A, Joshi G, Davis J, Niemela JE, et al. Natural history of autoimmune lymphoproliferative syndrome associated with FAS gene mutations. Blood. 2014;123:1989–99.
27. Boggio E, Clemente N, Mondino A, Cappellano G, Orilieri E, Gigliotti CL, et al. IL-17 protects T cells from apoptosis and contributes to development of ALPS-like phenotypes. Blood. 2014;123:1178–86.
28. Chavele KM, Merry E, Ehrenstein MR. Cutting edge: circulating plasmablasts induce the differentiation of human T follicular helper cells via IL-6 production. J Immunol. 2015;194:2482–24855.
29. Stranges PB, Watson J, Cooper CJ, Choisy-Rossi CM, Stonebraker AC, Beighton RA, et al. Elimination of antigen-presenting cells and autoreactive T cells by Fas contributes to prevention of autoimmunity. Immunity. 2007;26:629–41.