Scleroderma Research Landscape Overview Powerpoint

Landscape of scleroderma review

1. Current Landscape of Clinical Research in Systemic Sclerosis

2. Unmet Needs Early diagnosis and prediction of progression Effective anti-fibrotic and immune-modulatory therapies Better biomarkers for disease activity and response

3. Key Ongoing Trials Nintedanib (SENSCIS): slowed FVC decline in SSc-ILD Tocilizumab (faSScinate, focuSSced): skin and lung benefit Lenabasum (RESOLVE-1): modest improvement in skin score Romilkimab: dual IL-4/IL-13 blockade under investigation

4. Pathophysiologic Targets Fibroblast and endothelial cross-talk via TGF-β, IL-6, and integrins JAK/STAT and PI3K pathways in immune regulation Macrophage activation and profibrotic signaling

5. Biomarkers and Precision Medicine Transcriptomic and proteomic profiling for patient stratification Gene signatures predictive of therapeutic response Circulating proteins: CXCL4, COMP, periostin

6. Imaging Innovations High-frequency ultrasound for dermal thickness SFDI and SLIM for skin optical property mapping MRI and shear-wave elastography for organ fibrosis

7. Translational Research Fibroblast heterogeneity in lesional vs non-lesional skin Macrophage-fibroblast co-culture models Single-cell and spatial transcriptomic profiling

8. Emerging Therapies Anti-TGF-β, integrin inhibitors, anti-CTGF agents Stem cell transplantation in severe disease Novel antifibrotics targeting ECM remodeling

9. Clinical Trial Design Evolution Use of mRSS, HAQ-DI, and composite endpoints Digital biomarkers and remote patient monitoring Adaptive and enrichment trial designs

10. Future Directions Artificial intelligence for image and omics integration Digital health and wearable-based monitoring Collaborative registries and patient-centric trials