Is PDGFRB a therapeutic target in Soft Tissue Tumors?

Details

Supervisors

Demoulin, Jean-Baptiste

Faculty

Faculté de pharmacie et des sciences biomédicales

Degree label

Master [60] en sciences biomédicales

Abstract

This study evaluates the potential of PDGFRB as a therapeutic target in soft tissue tumors (STTs). It reviews published case series and reports on both pediatric and adult patients with PDGFRB-driven STTs who have been treated with tyrosine kinase inhibitors (TKIs). Clinical outcomes vary. However, several cases have reported significant tumor regression or disease stabilization with TKIs such as imatinib and sunitinib. These findings support the efficacy of PDGFRβ-directed therapies. The findings highlight the importance of a precision medicine approach for tumors with PDGFRB alterations. Identifying actionable genetic changes can guide the development of tailored therapies. However, no PDGFRB-specific TKIs are formally approved for these indications, so treatment often occurs off-label, which presents regulatory and access challenges. In the absence of large clinical trials, aggregated real-world case data are essential to inform medical practice. Our analysis emphasizes the need for international registries and collaborative studies to compile outcomes and develop evidence-based guidelines. Overall, the clinical and experimental data compiled in this study support targeting PDGFRβ as a promising strategy, which has significant implications for personalized oncology in managing these rare STTs.