AHSCT in SSc — SCE Rheumatology MCQ

Correct answer: D — Early diffuse cutaneous systemic sclerosis with rapid skin and interstitial lung disease progression despite appropriate immunosuppression, a poor prognosis, and no advanced cardiorespiratory disease

AHSCT is considered for highly selected patients with early, severe diffuse cutaneous systemic sclerosis that is rapidly progressive and confers a poor prognosis, particularly when progression continues despite appropriate immunosuppression. Rigorous cardiopulmonary assessment is essential because advanced cardiac or pulmonary vascular disease markedly increases transplant toxicity; therefore option E is unsuitable. Stable limited disease, treatment-responsive early diffuse disease, and late irreversible organ damage without active progression do not provide an appropriate risk–benefit balance. ASTIS demonstrated a long-term event-free survival advantage over intravenous cyclophosphamide, although early treatment-related mortality was increased. SCOT demonstrated superiority for its global rank composite outcome and later event-free and overall survival. Anti-topoisomerase I positivity may identify a higher-risk phenotype but is not, by itself, an indication for transplantation.

Reference: European Alliance of Associations for Rheumatology. EULAR recommendations for the treatment of systemic sclerosis: 2023 update, section 9: People with poor prognosis. Published 2024. https://www.eular.org/document/download/1021/cb11a479-9d7a-4b7e-8020-b8cc1dc69bb8/932