OUTCOMES IN SYSTEMIC SCLEROSIS PATIENTS TREATED WITH RITUXIMAB AND MYCOPHENOLATE MOFETIL COMBINATION THERAPY COMPARED TO AUTOLOGOUS HEMATOLOGICAL STEM CELL TRANSPLANTATION
Authors: Shiri Keret et al.
Autologous hematological stem cell transplantation (AHSCT) is a grade A therapy for early diffuse systemic sclerosis (SSc), validated in three randomized controlled trials (RCT). The efficacy of rituximab on skin and lung involvement in SSc has been demonstrated in the DESIRES RCT. The combination of rituximab with mycophenolate mofetil (MMF) is a potential potent regimen for progressive SSc.
This cohort study retrospectively compared the outcomes of 21 SSc patients treated with a RTX/MMF combination with that of 16 SSc patients treated with AHSCT.
Clinical improvement (defined as a decrease in mRSS by more than 25% or an increase in FVC by more than 10%) at 12 months was seen in 86% of patients in the combination group, compared to 81% in the AHSCT group (p=0.7). In both groups, a statistically significant increase in FVC values was demonstrated (in a linear mixed FVC model 10% in the RTX/MMF combo group, 19% in the AHSCT group). In both groups, there was a significant and similar reduction in mRSS at 12 and 24 months.
This small retrospective study indicates that the combination of MMF and RTX may evolve as a potent alternative to stem cell transplantation in patients with early diffuse SSc. Data on survival and quality of life were not presented. These results must, however, be replicated prospectively in a RCT before the combination therapy can be adopted into clinical routine.