Old medications and new targeted therapies in systemic sclerosis

Rheumatology (United Kingdom)

Volumn 54, Issue 11, 2015, Pages 1944-1953

  Nagaraja, Vivek ^a   Denton, Christopher P ^b   Khanna, Dinesh ^a  

^a UNIVERSITY OF MICHIGAN   (United States)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Digital ulcers; Immunosuppressive therapy; Interstitial lung disease; Pulmonary arterial hypertension; Raynaud's phenomenon; Renal crisis; Skin fibrosis; Systemic sclerosis; Treatment

Indexed keywords

ANGIOTENSIN RECEPTOR ANTAGONIST; CALCIUM CHANNEL BLOCKING AGENT; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; GLUCOCORTICOID; METHOTREXATE; MYCOPHENOLATE MOFETIL; PENICILLIN DERIVATIVE; PROSTAGLANDIN DERIVATIVE;

AUTOLOGOUS STEM CELL TRANSPLANTATION; FINGER ULCER; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; MOLECULARLY TARGETED THERAPY; PATHOGENESIS; PRIORITY JOURNAL; PULMONARY HYPERTENSION; RAYNAUD PHENOMENON; REVIEW; SCLERODERMA; SKIN FIBROSIS; SYSTEMIC SCLEROSIS;

EID: 84946706774     PISSN: 14620324     EISSN: 14620332     Source Type: Journal    
DOI: 10.1093/rheumatology/keu285     Document Type: Review

References (85)

1. Khanna D, Denton CP. Evidence-based management of rapidly progressing systemic sclerosis. Best Pract Res Clin Rheumatol 2010;24:387-400.
2. Avouac J, Fransen J, Walker UA et al. Preliminary criteria for the very early diagnosis of systemic sclerosis: results of a Delphi Consensus Study from EULAR Scleroderma Trials and Research Group. Ann Rheum Dis 2011;70: 476-81.
3. Khanna D, Merkel PA. Outcome measures in systemic sclerosis: an update on instruments and current research. Curr Rheumatol Rep 2007;9:151-7.
4. Clements PJ, Hurwitz EL, Wong WK et al. Skin thickness score as a predictor and correlate of outcome in systemic sclerosis: high-dose versus low-dose penicillamine trial. Arthritis Rheum 2000;43:2445-54.
5. Johnson SR, Feldman BM, Pope JE, Tomlinson GA. Shifting our thinking about uncommon disease trials: the case of methotrexate in scleroderma. J Rheumatol 2009; 36:323-9.
6. Pope JE, Bellamy N, Seibold JR et al. A randomized, controlled trial of methotrexate versus placebo in early diffuse scleroderma. Arthritis Rheum 2001;44: 1351-8.
7. Tashkin DP, Elashoff R, Clements PJ et al. Cyclophosphamide versus placebo in scleroderma lung disease. N Engl J Med 2006;354:2655-66.
8. Tashkin DP, Elashoff R, Clements PJ et al. Effects of 1-year treatment with cyclophosphamide on outcomes at 2 years in scleroderma lung disease. Am J Respir Crit Care Med 2007;176:1026-34.
9. D'Angelo S, Cuomo G, Paone C et al. Low-dose intravenous cyclophosphamide in systemic sclerosis: a preliminary safety study. Clin Rheumatol 2003;22:393-6.
10. Herrick AL, Lunt M, Whidby N et al. Observational study of treatment outcome in early diffuse cutaneous systemic sclerosis. J Rheumatol 2010;37:116-24.
11. Nihtyanova SI, Brough GM, Black CM, Denton CP. Mycophenolate mofetil in diffuse cutaneous systemic sclerosis-a retrospective analysis. Rheumatology 2007; 46:442-5.
12. Le EN, Wigley FM, Shah AA, Boin F, Hummers LK. Longterm experience of mycophenolate mofetil for treatment of diffuse cutaneous systemic sclerosis. Ann Rheum Dis 2011;70:1104-7.
13. Clements PJ, Furst DE, Wong WK et al. High-dose versus low-dose D-penicillamine in early diffuse systemic sclerosis: analysis of a two-year, double-blind, randomized, controlled clinical trial. Arthritis Rheum 1999;42:1194-203.
14. Black CM, Silman AJ, Herrick AI et al. Interferon-alpha does not improve outcome at one year in patients with diffuse cutaneous scleroderma: results of a randomized, double-blind, placebo-controlled trial. Arthritis Rheum 1999;42:299-305.
15. Khanna D, Clements PJ, Furst DE et al. Recombinant human relaxin in the treatment of systemic sclerosis with diffuse cutaneous involvement: a randomized, doubleblind, placebo-controlled trial. Arthritis Rheum 2009;60: 1102-11.
16. Denton CP, Merkel PA, Furst DE et al. Recombinant human anti-transforming growth factor beta1 antibody therapy in systemic sclerosis: a multicenter, randomized, placebo-controlled phase I/II trial of CAT-192. Arthritis Rheum 2007;56:323-33.
17. Denton CP, Engelhart M, Tvede N et al. An open-label pilot study of infliximab therapy in diffuse cutaneous systemic sclerosis. Ann Rheum Dis 2009;68:1433-9.
18. Pope J, McBain D, Petrlich L et al. Imatinib in active diffuse cutaneous systemic sclerosis: results of a sixmonth, randomized, double-blind, placebo-controlled, proof-of-concept pilot study at a single center. Arthritis Rheum 2011;63:3547-51.
19. Avouac J, Clements PJ, Khanna D, Furst DE, Allanore Y. Articular involvement in systemic sclerosis. Rheumatology 2012;51:1347-56.
20. Steen VD, Medsger TA. Changes in causes of death in systemic sclerosis, 1972-2002. Ann Rheum Dis 2007;66: 940-4.
21. Schurawitzki H, Stiglbauer R, Graninger W et al. Interstitial lung disease in progressive systemic sclerosis: high-resolution CT versus radiography. Radiology 1990; 176:755-9.
22. Warrick JH, Bhalla M, Schabel SI, Silver RM. High resolution computed tomography in early scleroderma lung disease. J Rheumatol 1991;18:1520-8.
23. Steen VD, Medsger TA Jr. Severe organ involvement in systemic sclerosis with diffuse scleroderma. Arthritis Rheum 2000;43:2437-44.
24. Khanna D, Tseng CH, Farmani N et al. Clinical course of lung physiology in patients with scleroderma and interstitial lung disease: analysis of the Scleroderma Lung Study Placebo Group. Arthritis Rheum 2011;63: 3078-85.
25. Goh NS, Desai SR, Veeraraghavan S et al. Interstitial lung disease in systemic sclerosis: a simple staging system. Am J Respir Crit Care Med 2008;177: 1248-54.
26. Hoyles RK, Ellis RW, Wellsbury J et al. A multicenter, prospective, randomized, double-blind, placebocontrolled trial of corticosteroids and intravenous cyclophosphamide followed by oral azathioprine for the treatment of pulmonary fibrosis in scleroderma. Arthritis Rheum 2006;54:3962-70.
27. Khanna D, Yan X, Tashkin DP et al. Impact of oral cyclophosphamide on health-related quality of life in patients with active scleroderma lung disease: results from the scleroderma lung study. Arthritis Rheum 2007;56: 1676-84.
28. Kowal-Bielecka O, Landewe R, Avouac J et al. EULAR recommendations for the treatment of systemic sclerosis: a report from the EULAR Scleroderma Trials and Research group (EUSTAR). Ann Rheum Dis 2009;68:620-8.
29. Derk CT, Grace E, Shenin M et al. A prospective openlabel study of mycophenolate mofetil for the treatment of diffuse systemic sclerosis. Rheumatology 2009;48: 1595-9.
30. Gerbino AJ, Goss CH, Molitor JA. Effect of mycophenolate mofetil on pulmonary function in scleroderma-associated interstitial lung disease. Chest 2008;133:455-60.
31. Pakas I, Ioannidis JP, Malagari K et al. Cyclophosphamide with low or high dose prednisolone for systemic sclerosis lung disease. J Rheumatol 2002;29:298-304.
32. Steen VD, Medsger TA Jr. Case-control study of corticosteroids and other drugs that either precipitate or protect from the development of scleroderma renal crisis. Arthritis Rheum 1998;41:1613-9.
33. Aggarwal R, Nagaraja V, Khanna D. B-cell targeted therapies in systemic sclerosis and inflammatory myopathies. In: Bosch X, Ramos-Casals M, Khamashta MA, eds. Drugs Targeting B-Cells in Autoimmune Diseases. Basel, Switzerland: Springer, 2014:153-80.
34. Seibold JR, Denton CP, Furst DE et al. Randomized, prospective, placebo-controlled trial of bosentan in interstitial lung disease secondary to systemic sclerosis. Arthritis Rheum 2010;62:2101-8.
35. Khanna D, Saggar R, Mayes MD 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.
36. Khanna DT, Tashkin DP. Treatment of interstitial lung disease. In: Varga J, Denton CP, Wigley FM, eds. Scleroderma: From Pathogenesis to comprehensive Management. New York: Springer, 2012.
37. Goh NS, Veeraraghavan S, Desai SR et al. Bronchoalveolar lavage cellular profiles in patients with systemic sclerosis-associated interstitial lung disease are not predictive of disease progression. Arthritis Rheum 2007;56:2005-12.
38. Burt RK, Shah SJ, Dill K et al. Autologous nonmyeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trial. Lancet 2011;378:498-506.
39. Van Laar JM, Farge D, Sont JK et al. Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. JAMA 2014;311:2490-8.
40. Hachulla E, Carpentier P, Gressin V et al. Risk factors for death and the 3-year survival of patients with systemic sclerosis: the French ItinerAIR-Sclerodermie study. Rheumatology 2009;48:304-8.
41. Badesch DB, Tapson VF, McGoon MD et al. Continuous intravenous epoprostenol for pulmonary hypertension due to the scleroderma spectrum of disease. A randomized, controlled trial. Ann Intern Med 2000;132:425-34.
42. Oudiz RJ, Schilz RJ, Barst RJ et al. Treprostinil, a prostacyclin analogue, in pulmonary arterial hypertension associated with connective tissue disease. Chest 2004; 126:420-7.
43. Khanna D, Gladue H, Channick R et al. Recommendations for screening and detection of connective tissue diseaseassociated pulmonary arterial hypertension. Arthritis Rheum 2013;65:3194-201.
44. Galie N, Corris PA, Frost A et al. Updated treatment algorithm of pulmonary arterial hypertension. J Am Coll Cardiol 2013;62(Suppl):D60-72.
45. McLaughlin VV, Gaine SP, Howard LS et al. Treatment goals of pulmonary hypertension. J Am Coll Cardiol 2013; 62(Suppl):D73-81.
46. Ingraham K, Steen VD. Morbidity of digital tip ulcerations in scleroderma. Arthritis Rheum 2006;54(Suppl 9):P578.
47. Herrick AL. Pathogenesis of Raynaud's phenomenon. Rheumatology 2005;44:587-96.
48. Thompson AE, Shea B, Welch V, Fenlon D, Pope JE. Calcium-channel blockers for Raynaud's phenomenon in systemic sclerosis. Arthritis Rheum 2001;44:1841-7.
49. Rademaker M, Cooke ED, Almond NE et al. Comparison of intravenous infusions of iloprost and oral nifedipine in treatment of Raynaud's phenomenon in patients with systemic sclerosis: a double blind randomised study. BMJ 1989;298:561-4.
50. Teh LS, Manning J, Moore T et al. Sustained-release transdermal glyceryl trinitrate patches as a treatment for primary and secondary Raynaud's phenomenon. Br J Rheumatol 1995;34:636-41.
51. Wigley FM, Seibold JR, Wise RA, McCloskey DA, Dole WP. Intravenous iloprost treatment of Raynaud's phenomenon and ischemic ulcers secondary to systemic sclerosis. J Rheumatol 1992;19:1407-14.
52. Biondi ML, Marasini B, Bassani C, Agastoni A. Increased plasma endothelin levels in patients with Raynaud's phenomenon. N Engl J Med 1991;324:1139-40.
53. Korn JH, Mayes M, Matucci Cerinic M et al. Digital ulcers in systemic sclerosis: prevention by treatment with bosentan, an oral endothelin receptor antagonist. Arthritis Rheum 2004;50:3985-93.
54. Matucci-Cerinic M, Denton CP, Furst DE et al. Bosentan treatment of digital ulcers related to systemic sclerosis: results from the RAPIDS-2 randomised, double-blind, placebo-controlled trial. Ann Rheum Dis 2011;70:32-8.
55. Fries R, Shariat K, von Wilmowsky H, Bohm M. Sildenafil in the treatment of Raynaud's phenomenon resistant to vasodilatory therapy. Circulation 2005;112:2980-5.
56. Herrick AL, van den Hoogen F, Gabrielli A et al. Modifiedrelease sildenafil reduces Raynaud's phenomenon attack frequency in limited cutaneous systemic sclerosis. Arthritis Rheum 2011;63:775-82.
57. Brueckner CS, Becker MO, Kroencke T et al. Effect of sildenafil on digital ulcers in systemic sclerosis: analysis from a single centre pilot study. Ann Rheum Dis 2010;69: 1475-8.
58. Gore J, Silver R. Oral sildenafil for the treatment of Raynaud's phenomenon and digital ulcers secondary to systemic sclerosis. Ann Rheum Dis 2005;64:1387.
59. Shenoy PD, Kumar S, Jha LK et al. Efficacy of tadalafil in secondary Raynaud's phenomenon resistant to vasodilator therapy: a double-blind randomized cross-over trial. Rheumatology 2010;49:2420-8.
60. Coleiro B, Marshall SE, Denton CP et al. Treatment of Raynaud's phenomenon with the selective serotonin reuptake inhibitor fluoxetine. Rheumatology 2001;40: 1038-43.
61. Gliddon AE, Dore CJ, Black CM et al. Prevention of vascular damage in scleroderma and autoimmune Raynaud's phenomenon: a multicenter, randomized, double-blind, placebo-controlled trial of the angiotensin-converting enzyme inhibitor quinapril. Arthritis Rheum 2007;56: 3837-46.
62. Tosi S, Marchesoni A, Messina K et al. Treatment of Raynaud's phenomenon with captopril. Drugs Exp Clin Res 1987;13:37-42.
63. Dziadzio M, Denton CP, Smith R et al. Losartan therapy for Raynaud's phenomenon and scleroderma: clinical and biochemical findings in a fifteen-week, randomized, parallel-group, controlled trial. Arthritis Rheum 1999;42: 2646-55.
64. Drake DB, Kesler RW, Morgan RF. Digital sympathectomy for refractory Raynaud's phenomenon in an adolescent. J Rheumatol 1992;19:1286-8.
65. Tomaino MM, Goitz RJ, Medsger TA. Surgery for ischemic pain and Raynaud's phenomenon in scleroderma: a description of treatment protocol and evaluation of results. Microsurgery 2001;21:75-9.
66. Yee AM, Hotchkiss RN, Paget SA. Adventitial stripping: a digit saving procedure in refractory Raynaud's phenomenon. J Rheumatol 1998;25:269-76.
67. Kotsis SV, Chung KC. A systematic review of the outcomes of digital sympathectomy for treatment of chronic digital ischemia. J Rheumatol 2003;30: 1788-92.
68. Iorio ML, Masden DL, Higgins JP. Botulinum toxin A treatment of Raynaud's phenomenon: a review. Semin Arthritis Rheum 2012;41:599-603.
69. Steen VD, Costantino JP, Shapiro AP, Medsger TA Jr. Outcome of renal crisis in systemic sclerosis: relation to availability of angiotensin converting enzyme (ACE) inhibitors. Ann Intern Med 1990;113:352-7.
70. Steen VD, Medsger TA Jr. Long-term outcomes of scleroderma renal crisis. Ann Intern Med 2000;133:600-3.
71. Denton CP, Black CM. Scleroderma-clinical and pathological advances. Best Pract Res Clin Rheumatol 2004;18: 271-90.
72. Hudson M, Baron M, Tatibouet S et al. Exposure to ACE inhibitors prior to the onset of scleroderma renal crisis-results from the International Scleroderma Renal Crisis Survey. Semin Arthritis Rheum 2014;43: 666-72.
73. Hudson M, Baron M, Lo E et al. An international, webbased, prospective cohort study to determine whether the use of ACE inhibitors prior to the onset of scleroderma renal crisis is associated with worse outcomes-methodology and preliminary results. Int J Rheumatol 2010;2010: 347402.
74. Penn H, Quillinan N, Khan K et al. Targeting the endothelin axis in scleroderma renal crisis: rationale and feasibility. QJM 2013;106:839-48.
75. DeMarco PJ, Weisman MH, Seibold JR et al. Predictors and outcomes of scleroderma renal crisis: the high-dose versus low-dose D-penicillamine in early diffuse systemic sclerosis trial. Arthritis Rheum 2002;46:2983-9.
76. Helfrich DJ, Banner B, Steen VD, Medsger TA Jr. Normotensive renal failure in systemic sclerosis. Arthritis Rheum 1989;32:1128-34.
77. Teixeira L, Servettaz A, Mehrenberger M et al. [Scleroderma renal crisis]. Presse Med 2006;35(12 Pt 2): 1966-74.
78. Santiago B, Gutierrez-Canas I, Dotor J et al. Topical application of a peptide inhibitor of transforming growth factor-beta1 ameliorates bleomycin-induced skin fibrosis. J Invest Dermatol 2005;125:450-5.
79. Stevenson JP, Kindler HL, Papasavvas E et al. Immunological effects of the TGFbeta-blocking antibody GC1008 in malignant pleural mesothelioma patients. Oncoimmunology 2013;2:e26218.
80. Tager AM, LaCamera P, Shea BS et al. The lysophosphatidic acid receptor LPA1 links pulmonary fibrosis to lung injury by mediating fibroblast recruitment and vascular leak. Nat Med 2008;14:45-54.
81. Gonzalez EG, Selvi E, Balistreri E et al. Synthetic cannabinoid ajulemic acid exerts potent antifibrotic effects in experimental models of systemic sclerosis. Ann Rheum Dis 2012;71:1545-51.
82. Henderson NC, Arnold TD, Katamura Y et al. Targeting of av integrin identifies a core molecular pathway that regulates fibrosis in several organs. Nat Med 2013;19:1617-24.
83. Haque SU, Dashwood MR, Heetun M et al. Efficacy of the specific endothelin a receptor antagonist zibotentan (ZD4054) in colorectal cancer: a preclinical study. Mol Cancer Ther 2013;12:1556-67.
84. Quillinan NP, McIntosh D, Vernes J, Haq S, Denton CP. Treatment of diffuse systemic sclerosis with hyperimmune caprine serum (AIMSPRO): a phase II double-blind placebo-controlled trial. Ann Rheum Dis 2014;73:56-61.
85. Dees C, Akhmetshina A, Zerr P et al. Platelet-derived serotonin links vascular disease and tissue fibrosis. J Exp Med 2011;208:961-72.