Multi-Targeted Kinase Inhibitor Therapy in Pediatric Bone and Soft Tissue Sarcoma Patients-A Single Centre Experience

Area of Science:

• Oncology
• Pediatric Hematology/Oncology
• Pharmacology

Background:

• Relapsed or refractory soft tissue and bone sarcomas in pediatric patients have poor prognoses.
• Multi-targeted kinase inhibitors (mTKIs) are effective in various malignancies as both initial and salvage therapies.

Purpose of the Study:

• To retrospectively evaluate the clinical outcomes of multi-targeted kinase inhibitor (mTKI) treatment in a diverse group of pediatric sarcoma patients.
• To assess the efficacy and safety of mTKIs in pediatric patients with relapsed or refractory sarcomas.

Main Methods:

• Eighteen pediatric patients with osteosarcoma, synovial sarcoma, chondrosarcoma, or rhabdomyosarcoma received sorafenib, regorafenib, or pazopanib.
• Treatment was indicated for progressive, inoperable, relapsed, or chemotherapy-resistant disease after first- and second-line chemotherapy.
• Survival outcomes were compared to a retrospective, non-randomized control group for osteosarcoma patients.

Main Results:

• The median age of patients at the start of mTKI treatment was 16.5 years, with a median time to progression of 4 months.
• The overall response rate was 16%.
• A significant difference in survival distribution was observed between osteosarcoma patients receiving mTKIs and controls (p=0.004), indicating improved survival with mTKIs.

Conclusions:

• Multi-targeted kinase inhibitors (mTKIs) are well-tolerated in pediatric sarcoma patients.
• mTKIs represent a viable therapeutic option for refractory bone sarcomas, serving as palliative treatment to slow disease progression.
• Treatment with mTKIs can help maintain a good quality of life for pediatric patients with advanced sarcomas.