Tyrosine kinase inhibitor therapy for systemic sclerosis: Quo Vadis?

Arthritis and Rheumatism

Volumn 63, Issue 11, 2011, Pages 3199-3203

  Mendoza, Fabian A ^a   Jiménez, Sergio A ^a  

^a THOMAS JEFFERSON UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ABELSON KINASE; CARBON MONOXIDE; IMATINIB; METHOTREXATE; PLACEBO; PLATELET DERIVED GROWTH FACTOR; PLATELET DERIVED GROWTH FACTOR RECEPTOR; PROTEIN TYROSINE KINASE INHIBITOR; SMAD1 PROTEIN; TISSUE INHIBITOR OF METALLOPROTEINASE 1; TRANSFORMING GROWTH FACTOR BETA1;

CLINICAL FEATURE; CLINICAL PROTOCOL; DIARRHEA; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; EDEMA; FLUID RETENTION; HUMAN; INTERSTITIAL LUNG DISEASE; LUNG CAPACITY; PRIORITY JOURNAL; PROTEINURIA; REVIEW; SIDE EFFECT; SIGNAL TRANSDUCTION; SYSTEMIC SCLEROSIS;

EARLY TERMINATION OF CLINICAL TRIALS; FEMALE; HUMANS; LUNG DISEASES, INTERSTITIAL; MALE; PIPERAZINES; PROTEIN KINASE INHIBITORS; PYRIMIDINES; SCLERODERMA, DIFFUSE; SCLERODERMA, SYSTEMIC;

EID: 80155206267     PISSN: 00043591     EISSN: 15290131     Source Type: Journal    
DOI: 10.1002/art.30545     Document Type: Review

References (41)

1. Gabrieli A, Avvediemento EV, Krieg T,. Mechanisms of disease: scleroderma. N Engl J Med 2009; 360: 1989-2003.
2. Jimenez SA, Derk CT,. Following the molecular pathways toward an understanding of the pathogenesis of systemic sclerosis. Ann Int Med 2004; 140: 37-50. (Pubitemid 38055920)
3. Varga J, Abraham D,. Systemic sclerosis: a prototypic multisystem fibrotic disorder. J Clin Invest 2007; 117: 557-67. (Pubitemid 46348510)
4. Denton CP, Black CM, Abraham DJ,. Mechanisms and consequences of fibrosis in systemic sclerosis. Nat Clin Pract Rheumatol 2006; 2: 134-44. (Pubitemid 43771821)
5. Varga JA, Trojanowska M,. Fibrosis in systemic sclerosis. Rheum Dis Clin North Am 2008; 34: 115-43, vii. (Pubitemid 351329455)
6. Katsumoto TR, Whitfield ML, Connolly MK,. The pathogenesis of systemic sclerosis. Annu Rev Pathol 2011; 28: 509-37.
7. Rosenbloom J, Jimenez SA,. Molecular ablation of TGFβ signaling pathways by tyrosine kinase inhibition: the coming of a promising new era in the treatment of tissue fibrosis [editorial]. Arthritis Rheum 2008; 58: 2219-24.
8. Distler J, Distler O,. Novel treatment approaches to fibrosis in scleroderma. Rheum Dis Clin North Am 2008; 34: 145-59, vii. (Pubitemid 351329462)
9. Varga J, Pasche B,. Antitransforming growth factor-β therapy in fibrosis: recent progress and implications for systemic sclerosis. Curr Opin Rheumatol 2008; 20: 720-8.
10. Ong VH, Denton CP,. Innovative therapies for systemic sclerosis. Curr Opin Rheumatol 2010; 22: 264-72.
11. Iwamoto N, Distler JH, Distler O,. Tyrosine kinase inhibitors in the treatment of systemic sclerosis: from animal models to clinical trials. Curr Rheumatol Rep 2011; 13: 21-7.
12. Krieg T, Abraham D, Lafyatis R,. Fibrosis in connective tissue disease: the role of the myofibroblast and fibroblast-epithelial cell interactions. Arthritis Res Ther 2007; 9 Suppl 2: S4.
13. Abraham DJ, Eckes B, Rajkumar V, Krieg T,. New developments in fibroblast and myofibroblast biology: implications for fibrosis and scleroderma. Curr Rheumatol Rep 2007; 9: 136-43. (Pubitemid 46932720)
14. Kalluri R, Nielson EG,. Epithelial-mesenchymal transition and its implications for fibrosis. J Clin Invest 2003; 112: 1776-84. (Pubitemid 38063698)
15. Thiery JP, Acloque H, Huang RY, Nieto MA,. Epithelial-mesenchymal transitions in development and disease. Cell 2009; 139: 871-90.
16. Chaudhuri V, Zhou L, Karasek M,. Inflammatory cytokines induce the transformation of human dermal microvascular endothelial cells into myofibroblasts: a potential role in skin fibrogenesis. J Cutan Pathol 2007; 34: 146-53. (Pubitemid 46145554)
17. Piera-Velazquez S, Li Z, Jimenez SA,. Role of endothelial-mesenchymal transition (EndoMT) in the pathogenesis of fibrotic disorders. Am J Pathol Rev 2011; 179: 1074-84.
18. Xu J, Lamouille S, Derynck R,. TGF-β-induced epithelial to mesenchymal transition. Cell Res 2009; 19: 156-72.
19. Li Z, Jimenez SA,. Protein kinase CÎ and c-Abl kinase are required for transforming growth factor β induction of endothelial- mesenchymal transition in vitro. Arthritis Rheum 2011; 63: 2473-83.
20. Medici D, Potenta S, Kalluri R,. Transforming growth factor-β2 promotes Snail-mediated endothelial-mesenchymal transition through convergence of Smad-dependent and Smad-independent signalling. Biochem J 2011; 437: 515-20.
21. Varga J, Whitfield ML,. Transforming growth factor-β in systemic sclerosis (scleroderma). Front Biosci 2009; 1: 226-35.
22. Daniels CE, Wilkes MC, Edens M, Kottom TJ, Murphy SJ, Limper AH, et al. Imatinib mesylate inhibits the profibrogenic activity of TGF-β and prevents bleomycin-mediated lung fibrosis. J Clin Invest 2004; 114: 1308-16. (Pubitemid 40385302)
23. Bhattachavya S, Ishida W, Wu M, Wilkes M, Mori Y, Hinchcliff M, et al. A non-Smad mechanism of fibroblast activation by transforming growth factor-β via c-Abl and Egr-1: selective modulation by imatinib mesylate. Oncogene 2009; 28: 1285-97.
24. Jimenez SA, Castro SV, Piera-Velazquez M,. Role of growth factors in the pathogenesis of tissue fibrosis in systemic sclerosis. Curr Rheumatol Rev 2010; 6: 283-94.
25. Trojanowska M,. Role of PDGF in fibrotic diseases and systemic sclerosis. Rheumatology (Oxford) 2008; 47 Suppl 5: v2-4.
26. Barst RJ,. PDGF signaling in pulmonary arterial hypertension. J Clin Invest 2005; 115: 2691-4. (Pubitemid 41434394)
27. Druker B, Tamura S, Buchdunger E, Ohno S, Segal GM, Fanning S, et al. Effects of a selective inhibitor of the Abl tyrosine kinase on the growth of Bcr-Abl positive cells. Nat Med 1996; 2: 561-6.
28. Deininger MW, Goldman JM, Lydon N, Melo JV,. The tyrosine kinase inhibitor CGP57148B selectively inhibits the growth of BCR-ABL positive cells. Blood 1997; 90: 3691-8. (Pubitemid 27473440)
29. Gambacorti-Passerini C, le Coutre P, Mologni L, Fanelli M, Bertazzoli C, Marchesi E, et al. Inhibition of the ABL kinase activity blocks the proliferation of BCR/ABL+ leukemic cells and induces apoptosis. Blood Cells Mol Dis 1997; 23: 380-94. (Pubitemid 28123720)
30. Druker B, Lydon NB,. Lessons learned from the development of an Abl tyrosine kinase inhibitor for chronic myelogenous leukemia. J Clin Invest 2000; 105: 3-7. (Pubitemid 30036380)
31. Wang S, Wilkes MC, Leof EB, Hirschberg R,. Imatinib mesylate blocks a non-Smad TGF-β pathway and reduces renal fibrogenesis in vivo. FASEB J 2005; 19: 1-11. (Pubitemid 40069914)
32. Rosenbloom J, Castro SV, Jimenez SA,. Fibrotic diseases: cellular and molecular mechanisms and novel therapies. Ann Intern Med 2010; 152: 159-66.
33. Distler JH, Jungel A, Huber LC, Schulze-Horsel U, Zwerina J, Gay RE, et al. Imatinib mesylate reduces production of extracellular matrix and prevents development of experimental dermal fibrosis. Arthritis Rheum 2007; 56: 311-22. (Pubitemid 46106205)
34. Van Daele PL, Dik WA, Thio HB, van Hal PT, van Laar JA, Hooijkaas H, et al. Is imatinib mesylate a promising drug in systemic sclerosis? Arthritis Rheum 2008; 58: 2549-52.
35. Chung L, Fiorentino DF, BenBarak MJ, Adler AS, Mariano MM, Paniagua RT, et al. Molecular framework for response to imatinib mesylate in systemic sclerosis. Arthritis Rheum 2009; 60: 584-91.
36. Akhmetshina A, Venalis P, Dees C, Busch N, Zwerina J, Schett G, et al. Treatment with imatinib prevents fibrosis in different preclinical models of systemic sclerosis and induces regression of established fibrosis. Arthritis Rheum 2009; 60: 219-24.
37. Spiera RF, Gordon JK, Mersten JN, Magro CM, Mehta M, Wildman HF, et al. Imatinib mesylate (Gleevec) in the treatment of diffuse cutaneous systemic sclerosis: results of a 1-year, phase IIa, single-arm, open-label clinical trial. Ann Rheum Dis 2011; 70: 1003-9.
38. Khanna D, Saggar R, Mayes MD, Abtin F, Clements PJ, Maranian P, et al. A one-year, phase I/IIa, open-label pilot trial of imatinib mesylate in the treatment of systemic sclerosis-associated active interstitial lung disease. Arthritis Rheum 2011; 63: 3540-6.
39. Pope J, McBain D, Petrlich L, Watson S, Vanderhoek L, de Leon F, et al. Imatinib in active diffuse cutaneous systemic sclerosis: results of a six-month, randomized, double-blind, placebo-controlled, proof-of-concept pilot study at a single center. Arthritis Rheum 2011; 63: 3547-51.
40. Van Hest RM, Schnog JB, Van't Veer MB, Cornelissen JJ,. Extremely slow methotrexate elimination in a patient with t(9;22) positive acute lymphoblastic leukemia treated with imatinib. Am J Hematol 2008; 83: 757-8.
41. Distler O, Distler JH, Varga J, Denton CP, Lafyatis RA, Wigley FM, et al. A multi-center, open-label, proof of concept study of imatinib mesylate demonstrates no benefit for the treatment of fibrosis in patients with early, diffuse systemic sclerosis [abstract]. Arthritis Rheum 2010; 62 Suppl: S233-4.