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

Denton, Christopher P and Yee, Philip and Kanitkar, Medha and Sims, Hannah and Clarke, Charlotte and Ahmed, Saiam and Ong, Voon H and Del Galdo, Francesco and Pauling, John D and Anderson, Marina E and Samaranayaka, Muditha and Hughes, Michael and Bhat, Smita and Griffiths, Bridget and Buch, Maya H and Herrick, Ariane L and D’Cruz, David and Vonk, Madelon C and Freemantle, Nick and Dehbi, Hakim-Moulay (2026) A randomised open-label pilot trial comparing mycophenolate mofetil with no immunosuppression in limited cutaneous systemic sclerosis (MINIMISE-Pilot). Rheumatology, 65 (3): keag108. ISSN 1462-0324

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Abstract

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.