A randomised open-label pilot trial comparing mycophenolate mofetil with no immunosuppression in limited cutaneous systemic sclerosis (MINIMISE-Pilot)

OBJECTIVES: Mycophenolate mofetil (MMF) is routinely used in early diffuse cutaneous systemic sclerosis (dcSSc) but not in limited cutaneous (lc)SSc. This may miss an opportunity to slow disease progression. MINIMISE-Pilot tested the feasibility of an open-label event-driven randomised trial of MMF vs no immunosuppression in lcSSc. METHODS: We tested the feasibility of a trial evaluating the impact of MMF on a novel event-driven composite endpoint. The MINIMISE endpoint measures time to worsening of lcSSc determined by progressive lung fibrosis, pulmonary hypertension, scleroderma renal crisis, heart failure, severe gut involvement, major digital vascular complications or death. Prespecified 'Stop-Go' criteria were agreed. Subjects were stratified by ACA status. RESULTS: Recruitment was challenging. A total of 53 subjects were screened and 43 were randomised, 21 to the MMF arm. Since recruitment was