A 45-year-old woman with diffuse cutaneous systemic sclerosis develops progressive dyspnoea. High-resolution CT chest shows basilar honeycombing and ground-glass opacities. PFTs show restrictive pattern. Anti-topoisomerase-I (anti-Scl-70) antibody is positive. Which treatment has shown the best evidence for slowing SSc-ILD progression in clinical trials?
- A Cyclophosphamide (SLS-I trial evidence)
- B Nintedanib (SENSCIS trial) ✓
- C Mycophenolate mofetil (SLS-II trial)
- D Pirfenidone
Explanation
The SENSCIS trial demonstrated that nintedanib, a tyrosine kinase inhibitor that targets TGF-beta and PDGF pathways, reduced the annual rate of decline in FVC in SSc-ILD (the primary endpoint) compared to placebo. MMF (SLS-II) showed non-inferiority to cyclophosphamide but did not show superiority; cyclophosphamide (SLS-I) showed modest short-term FVC benefit but not long-term. Nintedanib is FDA-approved specifically for SSc-ILD and is now considered the gold standard, though MMF is still used as an immunosuppressive backbone. Pirfenidone lacks robust trial evidence in SSc-ILD specifically.
Reference: Harrison's Principles of Internal Medicine, 21st ed.
High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP
Written and medically reviewed by the StethoPrep medical team.