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Table 1 Randomized controlled trials evaluating immunosuppressive/immunomodulatory drugs in patients with SSc.

From: Molecular targets for therapy in systemic sclerosis

Reference Treatment Number and main inclusion criteria of SSc patients Study duration Clinical effect
Furst DE et al.
Arthritis Rheum
1989;32:584
Chlorambucil p.o. 0.05-0.1 mg/kg/day versus placebo 65 SSc 3 years NS effect
O'Dell JR et al. J Rheumatol
1989;32:584
Total lymphoid irradiation versus untreated control 6 SSc with internal organ involvement Follow-up of 1-4 years NS effect
Casaes JA et al.
Ann Rheum Dis
1990;49:926
5-fluorouracil i.v. 4 × 12 mg/kg daily, followed by 4 × 6 mg/kg every two days and maintenance therapy with 12.5 mg/kg weekly versus placebo 70 SSc (diffuse or limited with visceral involvement) 6 months Significant improvement in skin score, Raynaud's score and patient's general assesment scores
Sharada B et al.
Rheumatol Int
1994;14:91
Dexamethasone i.v. 100 mg/month versus placebo 35 diffuse SSc 6 months Significant improvement in skin score
Van den Hoogen FH et al.
Br J Rheumatol
1996;35:364
Methotrexate i.m. 15 mg/week versus placebo 29 SSc with <3 years of skin involvement or with disease progression 24 weeks Trend towards improvement in skin score (p = 0.06 in comparison with placebo)
Clements PJ et al. Arthritis Rheum
2001;44:1351
D-penicillamine p.o. high (750-1000 mg/d) versus low (125 mg every second day) dose 134 early diffuse SSc 2 years NS effect
Pope JE et al.
Arthritis Rheum
2001;44:1351
Methotrexate p.o. 15 mg/week versus placebo 71 early diffuse SSc 1 year Improvement in skin scores, borderline significance
Tashkin DP et al. N Eng J Med
2006;354:2655
Cyclophosphamide p.o. 1-2 mg/kg/d versus placebo 154 SSc with early (<7 years duration) SSc, symptomatic SLD and alveolitis 1 year Significant improvement in lung volumes, dyspnoea and some measures of health related quality of life.
Significant improvement in skin score in diffuse SSc as a secondary outcome measure
Hoyles Rk et al.
Arthritis Rheum
2006;54:3962
Cyclophosphamide 6 × i.v. 600 mg/m2/month plus oral predonisone 20 mg every second day followed by azathioprine (p.o. 2.5 mg/kg/day) versus placebo 45 SSc with SLD 1 year Trend towards improvement of FVC (p = 0.08), but low power
Nadashkevich O et al.
Clin Rheumatol
2006;25:205
Cyclophosphamide p.o. 2 mg/kg/d for 12 months, then 1 mg/kg/d for another 6 months versus asathioprine 2.5 mg/kg/d for 12 months and then 2 mg/kg/d for 18 months 60 early diffuse SSc (<12 months duration) 18 months Significant improvement in skin score, lung function tests and frequency of attacks of Raynaud's phenomenon
  1. i.v. = intravenous, i.m. = intramuscular, p.o. = peroral, NS = not significant, SLD = scleroderma interstitial lung diseases, SSc = systemic sclerosis. This list is not complete and is only a selection of potential molecules for targeted therapies. Modified from Distler O: Screloderma-modern aspects of pathogenesis, diagnosis and therapy. UNI-MED 2009.