Therapeutic options for systemic sclerosis related interstitial lung diseases

Respiratory Medicine

Volumn 104, Issue SUPPL. 1, 2010, Pages

  Mouthon, Luc ^a   Bérezné, Alice ^a   Guillevin, Loïc ^a   Valeyre, Dominique ^b  

^a UNIVERSITÉ PARIS DESCARTES   (France)

^b UNIVERSITÉ PARIS 13   (France)

Author keywords

Cyclophosphamide; Interstitial lung disease; Mycophenolate mofetil; Systemic sclerosis; Treatment

Indexed keywords

ACETYLCYSTEINE; ALPHA INTERFERON; AZATHIOPRINE; BOSENTAN; BUCILLAMINE; CHLORAMBUCIL; CORTICOSTEROID; CYCLOPHOSPHAMIDE; DASATINIB; FLUOROURACIL; GAMMA INTERFERON; IMATINIB; IMMUNOSUPPRESSIVE AGENT; METHYLPREDNISOLONE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PENICILLAMINE; PIRFENIDONE; PLACEBO; PREDNISOLONE; PREDNISONE; PROTEIN TYROSINE KINASE INHIBITOR; TRANSFORMING GROWTH FACTOR BETA ANTIBODY;

ANTIINFLAMMATORY ACTIVITY; ARTICLE; AUTOIMMUNE DISEASE; BRONCHIOLITIS OBLITERANS; CLINICAL TRIAL; CYTOPENIA; DIFFERENTIAL DIAGNOSIS; DISEASE COURSE; DOSE RESPONSE; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG MEGADOSE; DRUG PULSE THERAPY; DRUG TREATMENT FAILURE; DRUG WITHDRAWAL; FIBROSING ALVEOLITIS; GASTROINTESTINAL DISEASE; HIGH RESOLUTION COMPUTER TOMOGRAPHY; HISTOPATHOLOGY; HUMAN; INFECTION; INTERSTITIAL LUNG DISEASE; INTERSTITIAL PNEUMONIA; LOW DRUG DOSE; LUNG BIOPSY; LUNG TRANSPLANTATION; PRIORITY JOURNAL; PROGNOSIS; PROTEINURIA; SYSTEMIC SCLEROSIS; TREATMENT OUTCOME; UNSPECIFIED SIDE EFFECT;

CYCLOPHOSPHAMIDE; DISEASE PROGRESSION; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INJECTIONS, INTRAVENOUS; LUNG DISEASES, INTERSTITIAL; LUNG TRANSPLANTATION; PREDNISONE; PROGNOSIS; RARE DISEASES; SCLERODERMA, SYSTEMIC;

EID: 77953210442     PISSN: 09546111     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.rmed.2010.03.018     Document Type: Article

References (82)

1. Servettaz A., Agard C., Tamby M., Guilpain P., Guillevin L., and Mouthon L. Physiopathologie de la sclérodermie systémique: état des lieux sur une affection aux multiples facettes. Presse Med 35 (2006) 1903-1915
2. Tamby M.C., Chanseaud Y., Guillevin L., and Mouthon L. New insights into the pathogenesis of systemic sclerosis. Autoimmun Rev 2 3 (2003) 152-157
3. Mayes M.D., Lacey Jr. J.V., Beebe-Dimmer J., Gillespie B.W., Cooper B., Laing T.J., et al. Prevalence, incidence, survival, and disease characteristics of systemic sclerosis in a large US population. Arthritis Rheum 48 8 (2003) 2246-2255
4. Roberts-Thomson P.J., and Walker J.G. Scleroderma: it has been a long hard journey. Intern Med J 36 8 (2006) 519-523
5. Tamaki T., Mori S., and Takehara K. Epidemiological study of patients with systemic sclerosis in Tokyo. Arch Dermatol Res 283 6 (1991) 366-371
6. Magnant J., and Diot E. Systemic sclerosis: epidemiology and environmental factors. Presse Med 35 12 Pt 2 (2006) 1894-1901
7. Le Guern V., Mahr A., Mouthon L., Jeanneret D., Carzon M., and Guillevin L. Prevalence of systemic sclerosis in a French multi-ethnic county. Rheumatology (Oxford) 43 9 (2004) 1129-1137
8. LeRoy E.C., and Medsger Jr. T.A. Criteria for the classification of early systemic sclerosis. J Rheumatol 28 7 (2001) 1573-1576
9. LeRoy E.C., Black C., Fleischmajer R., Jablonska S., Krieg T., Medsger Jr. T.A., et al. Scleroderma (systemic sclerosis): classification, subsets and pathogenesis. J Rheumatol 15 2 (1988) 202-205
10. Hachulla E., Gressin V., Guillevin L., Carpentier P., Diot E., Sibilia J., et al. Early detection of pulmonary arterial hypertension in systemic sclerosis: a French nationwide prospective multicenter study. Arthritis Rheum 52 12 (2005) 3792-3800
11. Steen V.D., and Medsger Jr. T.A. Severe organ involvement in systemic sclerosis with diffuse scleroderma. Arthritis Rheum 43 11 (2000) 2437-2444
12. Casale R., Buonocore M., and Matucci-Cerinic M. Systemic sclerosis (scleroderma): an integrated challenge in rehabilitation. Arch Phys Med Rehabil 78 7 (1997) 767-773
13. Wells A.U., Cullinan P., Hansell D.M., Rubens M.B., Black C.M., Newman-Taylor A.J., et al. Fibrosing alveolitis associated with systemic sclerosis has a better prognosis than lone cryptogenetic fibrosing alveolitis. Am J Respir Crit Care Med 149 (1994) 1583-1590
14. Kim E.A., Lee K.S., Johkoh T., Kim T.S., Suh G.Y., Kwon O.J., et al. Interstitial lung diseases associated with collagen vascular diseases: radiologic and histopathologic findings. Radiographics 22 Spec No (2002) S151-S165
15. Bouros D., Wells A.U., Nicholson A.G., Colby T.V., Polychronopoulos V., Pantelidis P., et al. Histopathologic subsets of fibrosing alveolitis in patients with systemic sclerosis and their relationship to outcome. Am J Respir Crit Care Med 165 12 (2002) 1581-1586
16. American Thoracic Society/European Respiratory Society International Multidisciplinary Consensus Classification of the Idiopathic Interstitial Pneumonias. This joint statement of the American Thoracic Society (ATS), and the European Respiratory Society (ERS) was adopted by the ATS board of directors, June 2001 and by the ERS Executive Committee, June 2001. Am J Respir Crit Care Med 165 2 (2002) 277-304
17. Martinez F.J., and McCune W.J. Cyclophosphamide for scleroderma lung disease. N Engl J Med 354 25 (2006) 2707-2709
18. Remy-Jardin M., Remy J., Cortet B., Mauri F., and Delcambre B. Lung changes in rheumatoid arthritis: CT findings. Radiology 193 2 (1994) 375-382
19. Diot E., Boissinot E., Asquier E., Guilmot J.L., Lemarie E., Valat C., et al. Relationship between abnormalities on high-resolution CT and pulmonary function in systemic sclerosis. Chest 114 6 (1998) 1623-1629
20. Launay D., Remy-Jardin M., Michon-Pasturel U., Mastora I., Hachulla E., Lambert M., et al. High resolution computed tomography in fibrosing alveolitis associated with systemic sclerosis. J Rheumatol 33 9 (2006) 1789-1801
21. Behr J., Vogelmeier C., Beinert T., Meurer M., Krombach F., Konig G., et al. Bronchoalveolar lavage for evaluation and management of scleroderma disease of the lung. Am J Respir Crit Care Med 154 2 Pt 1 (1996) 400-406
22. Bolster M.B., Ludwicka A., Sutherland S.E., Strange C., and Silver R.M. Cytokine concentrations in bronchoalveolar lavage fluid of patients with systemic sclerosis. Arthritis Rheum 40 4 (1997) 743-751
23. Steen V.D., and Medsger T.A. Case-control study of corticosteroids and other drugs that either precipitate or protect from the development of scleroderma renal crisis. Arthritis Rheum 41 9 (1998) 1613-1619
24. Kuwana M., Kaburaki J., Arnett F.C., Howard R.F., Medsger Jr. T.A., and Wright T.M. Influence of ethnic background on clinical and serologic features in patients with systemic sclerosis and anti-DNA topoisomerase I antibody. Arthritis Rheum 42 3 (1999) 465-474
25. Steen V.D., Conte C., Owens G.R., and Medsger T.A. Severe restrictive lung disease in systemic sclerosis. Arthritis Rheum 37 9 (1994) 1283-1289
26. Jacobsen S., Halberg P., Ullman S., Hoier-Madsen M., Petersen J., Mortensen J., et al. A longitudinal study of pulmonary function in Danish patients with systemic sclerosis. Clin Rheumatol 16 4 (1997) 384-390
27. Peters-Golden M., Wise R.A., Schneider P., Hichberg M., Stevens M.B., and Wigley F. Chemical and demographic predictors of loss of pulmonary function in systemic sclerosis. Medicine 63 (1984) 221-231
28. Peters-Golden M., Wise R.A., Hochberg M., Stevens M.B., and Wigley F.M. Incidence of lung cancer in systemic sclerosis. J Rheumatol 12 6 (1985) 1136-1139
29. Chatterjee S., Dombi G.W., Severson R.K., and Mayes M.D. Risk of malignancy in scleroderma: a population-based cohort study. Arthritis Rheum 52 8 (2005) 2415-2424
30. Mouthon L., Berezne A., Poiraudeau S., and Guillevin L. Therapeutic management of systemic sclerosis. Presse Med 35 12 Pt 2 (2006) 1975-1982
31. Grassegger A., Schuler G., Hessenberger G., Walder-Hantich B., Jabkowski J., MacHeiner W., et al. Interferon-gamma in the treatment of systemic sclerosis: a randomized controlled multicentre trial. Br J Dermatol 139 4 (1998) 639-648
32. Black C.M., Silman A.J., Herrick A.I., Denton C.P., Wilson H., Newman J., 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 42 2 (1999) 299-305
33. Clements P.J., Furst D.E., Wong W.K., Mayes M., White B., Wigley F., 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 42 6 (1999) 1194-1203
34. Seibold J.R., Korn J.H., Simms R., Clements P.J., Moreland L.W., Mayes M.D., et al. Recombinant human relaxin in the treatment of scleroderma. A randomized, double-blind, placebo-controlled trial. Ann Intern Med 132 11 (2000) 871-879
35. Steen V.D., Owens G.R., Redmond C., Rodnan G.P., and Medsger T.A. The effect of D-penicillamine on pulmonary findings in systemic sclerosis. Arthritis Rheum 28 8 (1985) 882-888
36. Steen V.D., Blair S., and Medsger T.A. The toxicity of D-penicillamine in systemic sclerosis. Ann Intern Med 104 5 (1986) 699-705
37. Granstein R.D., Flotte T.J., and Amento E.P. Interferons and collagen production. J Invest Dermatol 95 (1990) 75s
38. Kahan A., Amor B., Menkes C.J., and Strauch G. Recombinant interferon gamma in the treatment of systemic sclerosis. Am J Med 87 (1989) 273-277
39. Freundlich B., Jimenez S.A., Steen V.D., Medsger T.A., Szkolnicki M., and Jaffe H.S. Treatment of systemic sclerosis with recombinant interferon-gamma. A phase I/II clinical trial. Arthritis Rheum 35 10 (1992) 1134-1142
40. Hein R., Behr J., Hundgen M., Hunzelmann N., Meurer M., Braun-Falco O., et al. Treatment of systemic sclerosis with gamma-interferon. Br J Dermatol 126 5 (1992) 496-501
41. Bletry O., Laraki R., Cabane J., Frances C., Piette J.C., Soubrier M., et al. Long-term results of the treatment of diffuse systemic scleroderma with interferon-gamma. Rev Med Interne 14 10 (1993) 978
42. Vlachoyiannopoulos P.G., Tsifetaki N., Dimitriou I., Galaris D., Papiris S.A., and Moutsopoulos H.M. Safety and efficacy of recombinant gamma interferon in the treatment of systemic sclerosis. Ann Rheum Dis 55 10 (1996) 761-768
43. Polisson R.P., Gilkeson G.S., Pyun E.H., Pisetsky D.S., Smith E.A., and Simon L.S. A multicenter trial of recombinant human interferon gamma in patients with systemic sclerosis: effects on cutaneous fibrosis and interleukin 2 receptor levels. J Rheumatol 23 (1996) 654-658
44. DeMarco P.J., Weisman M.H., Seibold J.R., Furst D.E., Wong W.K., Hurwitz E.L., 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 46 11 (2002) 2983-2989
45. Teixeira L., Mouthon L., Mahr A., Berezne A., Agard C., Mehrenberger M., et al. Mortality and risk factors of scleroderma renal crisis: a French retrospective study of 50 patients. Ann Rheum Dis 67 1 (2008) 110-116
46. Berezne A., Valeyre D., Ranque B., Guillevin L., and Mouthon L. Interstitial lung disease associated with systemic sclerosis: what is the evidence for efficacy of cyclophosphamide?. Ann N Y Acad Sci 1110 (2007) 271-284
47. Casas J.A., Saway P.A., Villarreal I., Nolte C., Menajovsky B.L., Escudero E.E., et al. 5-fluorouracil in the treatment of scleroderma: a randomised, double blind, placebo controlled international collaborative study. Ann Rheum Dis 49 11 (1990) 926-928
48. Clements P., Lachenbruch P., Furst D., and Paulus H. The course of skin involvement in systemic sclerosis over three years in a trial of chlorambucil versus placebo. Arthritis Rheum 36 11 (1993) 1575-1579
49. Calguneri M., Apras S., Ozbalkan Z., Ertenli I., Kiraz S., Ozturk M.A., et al. The efficacy of oral cyclophosphamide plus prednisolone in early diffuse systemic sclerosis. Clin Rheumatol 22 4-5 (2003) 289-294
50. Silver R.M., Warrick J.H., Kinsella M.B., Staudt L.S., Baumann M.H., and Strange C. Cyclophosphamide and low-dose prednisone therapy in patients with systemic sclerosis (scleroderma) with interstitial lung disease. J Rheumatol 20 (1993) 838-844
51. Akesson A., Scheja A., Lundin A., and Wollheim F.A. Improved pulmonary function in systemic sclerosis after treatment with cyclophosphamide. Arthritis Rheum 37 5 (1994) 729-735
52. Steen V.D., Lanz J.K., Conte C., Owens G.R., and Medsger T.A. Therapy for severe interstitial lung disease in systemic sclerosis. A retrospective study. Arthritis Rheum 37 9 (1994) 1290-1296
53. White B., Moore W.C., Wigley F.M., Xiao H.Q., and Wise R.A. Cyclophosphamide is associated with pulmonary function and survival benefit in patients with scleroderma and alveolitis. Ann Intern Med 132 12 (2000) 947-954
54. D'Angelo S., Cuomo G., Paone C., Colutta E., La Montagna G., and Valentini G. Low-dose intravenous cyclophosphamide in systemic sclerosis: a preliminary safety study. Clin Rheumatol 22 6 (2003) 393-396
55. Casale R., Generini S., Luppi F., Pignone A., and Matucci-Cerinic M. Pulse cyclophosphamide decreases sympathetic postganglionic activity, controls alveolitis, and normalizes vascular tone dysfunction (Raynaud's phenomenon) in a case of early systemic sclerosis. Arthritis Rheum 51 4 (2004) 665-669
56. Airo P., Danieli E., Parrinello G., Antonioli C.M., Cavazzana I., Toniati P., et al. Intravenous cyclophosphamide therapy for systemic sclerosis. A single-center experience and review of the literature with pooled analysis of lung function test results. Clin Exp Rheumatol 22 5 (2004) 573-578
57. Nadashkevich O., Davis P., Fritzler M., and Kovalenko W. A randomized unblinded trial of cyclophosphamide versus azathioprine in the treatment of systemic sclerosis. Clin Rheumatol 25 2 (2006) 205-212
58. Beretta L., Caronni M., Raimondi M., Ponti A., Viscuso T., Origgi L., et al. Oral cyclophosphamide improves pulmonary function in scleroderma patients with fibrosing alveolitis: experience in one centre. Clin Rheumatol 26 2 (2007) 168-172
59. Schnabel A., Reuter M., and Gross W.L. Intravenous pulse cyclophosphamide in the treatment of interstitial lung disease due to collagen vascular diseases. Arthritis Rheum 41 7 (1998) 1215-1220
60. Davas E.M., Peppas C., Maragou M., Alvanou E., Hondros D., and Dantis P.C. Intravenous cyclophosphamide pulse therapy for the treatment of lung disease associated with scleroderma. Clin Rheumatol 18 6 (1999) 455-461
61. Pakas I., Ioannidis J.P., Malagari K., Skopouli F.N., Moutsopoulos H.M., and Vlachoyiannopoulos P.G. Cyclophosphamide with low or high dose prednisolone for systemic sclerosis lung disease. J Rheumatol 29 2 (2002) 298-304
62. Giacomelli R., Valentini G., Salsano F., Cipriani P., Sambo P., Conforti M.L., et al. Cyclophosphamide pulse regimen in the treatment of alveolitis in systemic sclerosis. J Rheumatol 29 4 (2002) 731-736
63. Griffiths B., Miles S., Moss H., Robertson R., Veale D., and Emery P. Systemic sclerosis and interstitial lung disease: a pilot study using pulse intravenous methylprednisolone and cyclophosphamide to assess the effect on high resolution computed tomography scan and lung function. J Rheumatol 29 11 (2002) 2371-2378
64. Mittal G., Udwadia Z., and Joshi V.R. Cyclophosphamide pulse regimen in the treatment of alveolitis in systemic sclerosis. J Rheumatol 30 5 (2003) 1121-1122 [author reply 1122]
65. Kowal-Bielecka O., Kowal K., Rojewska J., Bodzenta-Lukaszyk A., Siergiejko Z., Sierakowska M., et al. Cyclophosphamide reduces neutrophilic alveolitis in patients with scleroderma lung disease: a retrospective analysis of serial bronchoalveolar lavage investigations. Ann Rheum Dis 64 9 (2005) 1343-1346
66. Ostojic P., and Damjanov N. Improvement of lung function in patients with systemic sclerosis after 6 months cyclophosphamide pulse therapy. Clin Rheumatol (2006) 1-3
67. Yiannopoulos G., Pastromas V., Antonopoulos I., Katsiberis G., Kalliolias G., Liossis S.N., et al. Combination of intravenous pulses of cyclophosphamide and methylprednizolone in patients with systemic sclerosis and interstitial lung disease. Rheumatol Int 27 4 (2007) 357-361
68. Tashkin D.P., Elashoff R., Clements P.J., Goldin J., Roth M.D., Furst D.E., et al. Cyclophosphamide versus placebo in scleroderma lung disease. N Engl J Med 354 25 (2006) 2655-2666
69. Clements PJ, Furst D, Silver R, Tashkin D, Roth M, Goldin J, et al. Outcomes at 24-months are better for patients who received one-year of cyclophosphamide (CYC) therapy for SSc alveolitis than in those who received one-year of placebo (PBO) in the scleroderma lung study; 2006.
70. Hoyles R.K., Ellis R.W., Wellsbury J., Lees B., Newlands P., Goh N.S., et al. A multicenter, prospective, randomized, double-blind, placebo-controlled trial of corticosteroids and intravenous cyclophosphamide followed by oral azathioprine for the treatment of pulmonary fibrosis in scleroderma. Arthritis Rheum 54 12 (2006) 3962-3970
71. Berezne A., Ranque B., Valeyre D., Brauner M., Allanore Y., Launay D., et al. Therapeutic strategy combining intravenous cyclophosphamide followed by oral azathioprine to treat worsening interstitial lung disease associated with systemic sclerosis: a retrospective multicenter open-label study. J Rheumatol 35 6 (2008) 1064-1072
72. Nihtyanova S.I., Brough G.M., Black C.M., and Denton C.P. Mycophenolate mofetil in diffuse cutaneous systemic sclerosis-a retrospective analysis. Rheumatology (Oxford) 46 3 (2007) 442-445
73. Gerbino A.J., Goss C.H., and Molitor J.A. Effect of mycophenolate mofetil on pulmonary function in scleroderma-associated interstitial lung disease. Chest 133 2 (2008) 455-460
74. Schachna L., Medsger Jr. T.A., Dauber J.H., Wigley F.M., Braunstein N.A., White B., et al. Lung transplantation in scleroderma compared with idiopathic pulmonary fibrosis and idiopathic pulmonary arterial hypertension. Arthritis Rheum 54 12 (2006) 3954-3961
75. Denton C.P., Merkel P.A., Furst D.E., Khanna D., Emery P., Hsu V.M., 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 56 1 (2007) 323-333
76. Azuma A. Shared mechanisms of lung injury and subsequent fibrosis: role of surfactant proteins in the pathogenesis of interstitial pneumonia in Hermansky-Pudlak Syndrome. Intern Med 44 6 (2005) 529-530
77. Demedts M., Behr J., Buhl R., Costabel U., Dekhuijzen R., Jansen H.M., et al. High-dose acetylcysteine in idiopathic pulmonary fibrosis. N Engl J Med 353 21 (2005) 2229-2242
78. Steen V.D., and Medsger T.A. Changes in causes of death in systemic sclerosis, 1972-2002. Ann Rheum Dis 66 7 (2007) 940-944
79. Simeon-Aznar C.P., Fonollosa-Pla V., Tolosa-Vilella C., Selva O.C.A., Solans-Laque R., Palliza E., et al. Intravenous cyclophosphamide pulse therapy in the treatment of systemic sclerosis-related interstitial lung disease: a long term study. Open Respir Med J 2 (2008) 39-45
80. Berezné A., Valeyre D., Le Guern V., Brauner M., Mahr A., Constans J., et al. Therapeutic strategy combining intravenous cyclophosphamide (CYC) and azathioprine to treat severe systemic sclerosis (SSc): a prospective open-label study including 52 patients. Arthr Rheum 52 (2005) [Abstract 352]
81. Varai G., Earle L., Jimenez S.A., Steiner R.M., and Varga J. A pilot study of intermittent intravenous cyclophosphamide for the treatment of systemic sclerosis associated lung disease. J Rheumatol 25 7 (1998) 1325-1329
82. Akesson A. Cyclophosphamide therapy for scleroderma. Curr Opin Rheumatol 10 6 (1998) 579-583