Similar to the 2009 recommendations, there is still not sufficient data, to make specific recommendation for paediatric patients. It would be important to have studies at least for the effective paediatric dose of each medication, to be safely applied. It should be recognized that there are several other promising therapies, including immunosuppressive drugs or new biological agents which could not be included in the present recommendations because the evidence for their efficacy was considered insufficient at the time of developing these recommendations. The results of RCT evaluating new therapies in SSc patients which were published after closure of the systematic literature research are presented in table S4 in online Supplement. The of these trials evaluated the efficacy of sildenafil in DUs healing in SSc patients and is addressed in the comment following recommendation concerning treatment of DUs.[39] Another double-blind, phase 2 RCT involved 87 SSc patients with early diffuse SSc and elevated acute phase reactants. Treatment with tocilizumab (s.c. 162 mg per week) was associated with a favourable trends in skin score improvement as compared with placebo after 24 weeks (p=0.09) and 48 weeks (p=0.06). In addition, encouraging changes in FVC were noted. In view of promising effects of tocilizumab on skin and lung involvement, it is concluded that further studies are warranted before definitive conclusions can be made about its risks and benefits in SSc. [101] The results of another RCT, the Scleroderma Lung Study 2 comparing mycophenolate mofetil with CYC in patients with SSc-related ILD are expected to be published soon. The preliminary results of this study, recently published as an abstract of the 2015 ACR annual congress, indicate that mycophenolate mofetil (up to 3 g/day orally for 2 years) was comparable with oral CYC (2 mg/kg/day for one year followed by matching placebo for the second year) with regard to FVC course at
Similar to the 2009 recommendations, there is still not sufficient data, to make specific recommendation for paediatric patients. It would be important to have studies at least for the effective paediatric dose of each medication, to be safely applied. It should be recognized that there are several other promising therapies, including immunosuppressive drugs or new biological agents which could not be included in the present recommendations because the evidence for their efficacy was considered insufficient at the time of developing these recommendations. The results of RCT evaluating new therapies in SSc patients which were published after closure of the systematic literature research are presented in table S4 in online Supplement. The of these trials evaluated the efficacy of sildenafil in DUs healing in SSc patients and is addressed in the comment following recommendation concerning treatment of DUs.[39] Another double-blind, phase 2 RCT involved 87 SSc patients with early diffuse SSc and elevated acute phase reactants. Treatment with tocilizumab (s.c. 162 mg per week) was associated with a favourable trends in skin score improvement as compared with placebo after 24 weeks (p=0.09) and 48 weeks (p=0.06). In addition, encouraging changes in FVC were noted. In view of promising effects of tocilizumab on skin and lung involvement, it is concluded that further studies are warranted before definitive conclusions can be made about its risks and benefits in SSc. [101] The results of another RCT, the Scleroderma Lung Study 2 comparing mycophenolate mofetil with CYC in patients with SSc-related ILD are expected to be published soon. The preliminary results of this study, recently published as an abstract of the 2015 ACR annual congress, indicate that mycophenolate mofetil (up to 3 g/day orally for 2 years) was comparable with oral CYC (2 mg/kg/day for one year followed by matching placebo for the second year) with regard to FVC course at
Updated_EULAR_recommendations_SSc_R1_Clean_copy.docx