Spierings, J;
Bandini, G;
Allanore, Y;
Papa, ND;
Denton, CP;
Distler, O;
Furst, DE;
... Hughes, M; + view all
(2025)
Management of disease progression after autologous hematopoietic stem cell transplantation in systemic sclerosis: Results from an international questionnaire-based study.
Seminars in Arthritis and Rheumatism
, 71
, Article 152638. 10.1016/j.semarthrit.2025.152638.
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Abstract
OBJECTIVES: Autologous stem cell transplantation (AHSCT) is an established treatment in diffuse cutaneous systemic sclerosis (dcSSc). Optimal management of disease progression after AHSCT in dcSSc has not been defined. The aim of this study was to explore the experience and preferences of SSc experts on post-AHSCT management. METHODS: An online questionnaire study was conducted containing 17 questions concerning respondent demographics, definition of SSc progression after AHSCT, diagnostic work-up and treatment preferences. RESULTS: In total, 69 respondents from 21 countries completed the questionnaire. The majority (89.7 %) works at a university hospital, and were involved in decisions regarding AHSCT in patients with SSc (71 %). Most have 1 to 5 patients who underwent AHSCT under their care. They defined failure to improve after AHSCT as: an increase in mRSS, new onset or worsening of interstitial lung disease (ILD), new onset scleroderma renal crisis (SRC) or inflammatory arthritis. Progression after initial response was defined as: increase in mRSS, new or worsening of ILD, new SRC, inflammatory arthritis, new pulmonary arterial hypertension, digital vasculopathy or impaired physical functioning. The most frequent therapy in case of AHSCT failure was mycophenolate mofetil (N = 55, 88.7 %), rituximab (N = 54, 87.1 %), nintedanib (N = 39, 62.9 %) or/and tocilizumab (N = 36, 58.1 %). Combination therapy with more than one of these agents was considered by most respondents (N = 61, 88.4 %). CONCLUSION: Our study benchmarks the unique combined experiences of post-AHSCT management among SSc experts. We summarize preferences regarding definition of AHSCT failure and progression after response, as well as approach to diagnostic work-up and treatment.
| Type: | Article |
|---|---|
| Title: | Management of disease progression after autologous hematopoietic stem cell transplantation in systemic sclerosis: Results from an international questionnaire-based study |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.semarthrit.2025.152638 |
| Publisher version: | https://doi.org/10.1016/j.semarthrit.2025.152638 |
| Language: | English |
| Additional information: | © 2025 The Author(s). Published by Elsevier Inc. under a Creative Commons license (http://creativecommons.org/licenses/by/4.0/). |
| Keywords: | Systemic sclerosis, Scleroderma, Autologous hematopoietic stem cell transplantation, Stem cell therapy, Disease progression, maintenance therapy |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inflammation |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10204835 |
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