Abstract
Objective
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.
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.
| Original language | English |
|---|---|
| Article number | 152638 |
| Journal | Seminars in arthritis and rheumatism |
| Volume | 71 |
| Early online date | 31 Jan 2025 |
| DOIs | |
| Publication status | E-pub ahead of print - 31 Jan 2025 |