THE EUROPEAN Alliance of Associations for Rheumatology (EULAR) updated its recommendations for systemic sclerosis (SSc) in 2023, incorporating four new therapies. Autologous hematopoietic stem cell transplantation (aHSCT) has emerged as the most effective treatment for selected patients regarding overall and event-free survival, yet its high toxicity necessitates new approaches. Two abstracts on novel approaches were presented at the EULAR 2023 Congress, held on 12–15 June in Vienna, Austria.
The first abstract, presented by Jörg Henes on behalf of the AST MOMA investigators, detailed a prospective, open-label study assessing aHSCT’s feasibility in patients with impaired lung or heart function, as well as evaluating a reduced-toxicity regimen. Patients were stratified by their manifestations and underwent mobilisation with reduced doses of cyclophosphamide (2×1,000 mg) plus lenograstim before CD34+ selection. For those with active alveolitis, cyclophosphamide was increased to 2×1,500 mg. Patients with heart involvement received a conditioning regimen including thiotepa, a half dose of cyclophosphamide, and rATG before stem cell reinfusion. The primary endpoint was overall survival. Over a 3-year follow-up, eight of the 35 patients died. The overall response rate was 71.4% after 12 months and 60% after 36 months. Progressive disease was reported in four patients, with one relapse in the first year and four later relapses. Despite a treatment-related mortality rate of 11.4%, the overall survival rate after 3 years was comparable to existing data. This study is significant as it is the first to use a reduced cyclophosphamide regimen in patients with cardiac involvement, showing a comparable outcome in overall survival with reduced toxicity.
The second abstract was presented by Panagiota Xanthouli from the EDITA trial, focusing on SSc patients with mild pulmonary arterial hypertension (PAH) treated with ambrisentan. Previous data indicated significant declines in pulmonary vascular resistance but not in mean pulmonary arterial pressure (mPAP) when compared to placebo after 6 months. This study assessed the long-term effects of ambrisentan, and results showed significant mPAP improvement in the ambrisentan group compared to a control group with no vasodilative treatment. Additionally, four control group patients developed new PAH with mPAP >20 mmHg, whereas none did in the ambrisentan group. These findings suggest that continued PAH therapy with ambrisentan protects SSc patients from deteriorating haemodynamics.
Overall, these studies highlight critical advancements in SSc treatment, potentially influencing future standards of care.
Reference
Pecher AC et al. Highdose chemotherapy and transplantation of 34+ selected stem cells for progressive systemic sclerosis with or without cardiac involvement or alveolitis – modification according to manifestation (AST MOMA). OP0212. European Alliance of Associations for Rheumatology (EULAR) 2024, 12-15 June, 2024.
