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 v...
| Publicado en: | Rheumatology Vol. 65; no. 3; pp. 1 - 12 |
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| Autores principales: | , , , , , , , , , |
| Formato: | research tables/charts randomized controlled trial Journal Article |
| Publicado: |
Oxford University Press / USA
Mar2026
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| Acceso en línea: | Ver este registro en EBSCOhost |
| fields | @attributes: recordID: 1 pdfLink: plink: https://search.ebscohost.com/login.aspx?direct=true&db=ccm&AN=192667771&site=ehost-live header: @attributes: shortDbName: ccm uiTerm: 192667771 longDbName: CINAHL Complete uiTag: AN controlInfo: bkinfo: dissinfo: jinfo: jid: 14620324 DN9 jtl: Rheumatology issn: 14620324 maglogo: N pubinfo: dt: Mar2026 vid: 65 iid: 3 pid: 622 pub: Oxford University Press / USA artinfo: ui: 192667771 192667771 192667771 10.1093/rheumatology/keag108 192667771 ppf: 1 ppct: 11 formats: tig: atl: A randomised open-label pilot trial comparing mycophenolate mofetil with no immunosuppression in limited cutaneous systemic sclerosis (MINIMISE-Pilot). aug: au: Denton, Christopher P Yee, Philip Kanitkar, Medha Sims, Hannah Clarke, Charlotte Ahmed, Saiam Ong, Voon H Galdo, Francesco Del Pauling, John D Anderson, Marina E affil: Division of Medicine, University College London, London, UKRoyal Free London NHS Foundation Trust, London, UK sug: subj: Scleroderma, Systemic Drug Therapy Mycophenolate Mofetil Therapeutic Use Immunosuppression Disease Progression Research Subject Recruitment Human Funding Source United Kingdom Female Male Middle Age Aged Randomized Controlled Trials Random Assignment Pilot Studies Comparative Studies Multicenter Studies Questionnaires Descriptive Statistics Middle Aged: 45-64 years Aged: 65+ years Female Male ab: 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 <60 participants, MINIMISE-Pilot was terminated based upon the prespecified threshold for continuation. During the treatment period there were no clinical worsening endpoints. Adherence to MMF was generally high, with 19 participants (95%) being 100% adherent at week 1, decreasing to 9 participants (64%) at week 24. Conclusion MINIMISE-Pilot achieved its goal as a feasibility trial, leading to early termination of the study due to low recruitment. The rationale and concept for this study remain very strong. However, our findings suggest that a randomised prospective trial across 12 sites in the UK with relatively short follow-up duration is not feasible. This will inform the design of future studies testing the benefit of MMF in lcSSc. Trial registration Eudract (https://eudract.ema.europa.eu/) 2019-004139-21 pubtype: Academic Journal doctype: research tables/charts randomized controlled trial Journal Article ougenre: Article language: English refInfo: holdings: @attributes: islocal: N |
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