Multi‐Targeted Kinase Inhibitor Therapy in Pediatric Bone and Soft Tissue Sarcoma Patients—A Single Centre Experience
ABSTRACT Background Patients with relapsed or refractory soft tissue and bone sarcomas have dismal outcomes. Multi‐targeted kinase inhibitors (mTKI) have proven to be potent agents in several malignancies, both as primer therapy and as a salvage option. Our aim was to evaluate the clinical outcomes...
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| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Wiley
2025-05-01
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| Series: | Cancer Medicine |
| Subjects: | |
| Online Access: | https://doi.org/10.1002/cam4.70951 |
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| Summary: | ABSTRACT Background Patients with relapsed or refractory soft tissue and bone sarcomas have dismal outcomes. Multi‐targeted kinase inhibitors (mTKI) have proven to be potent agents in several malignancies, both as primer therapy and as a salvage option. Our aim was to evaluate the clinical outcomes of mTKI treatment in a heterogeneous group of pediatric sarcoma patients retrospectively. Procedures A total of 18 patients were treated with sorafenib, regorafenib, or pazopanib; 13 of them had osteosarcoma (OSC), 3 had synovial sarcoma (SySa), and 1–1 patient had chondrosarcoma and rhabdomyosarcoma. Indication for mTKI treatment was primarily progressive, inoperable, relapsed, or chemotherapy‐resistant disease after completion of first‐ and second‐line chemotherapy. Results At the time of the beginning of mTKI treatment, the median age was 16.5 years, and the median time to progression from initiation of mTKI was 4 months. The overall response rate was 16%. We conducted a comparison of the survival outcomes of OSC patients receiving mTKIs against a retrospective, non‐randomized control group. Overall survival was evaluated from the time of progression or relapse after second‐line treatment to the time of death. The log‐rank test revealed a significant difference in the survival distribution between patients receiving mTKIs and those who did not (chi2(1) = 8.13 p = 0.004). We observed benefits from mTKI treatment in 3 SySa patients, with pazopanib demonstrating effectiveness and no progression observed thus far. Conclusions Our findings suggest that mTKIs are well‐tolerated and can serve as a therapeutic option for refractory bone sarcomas as palliative treatment, aiming to slow disease progression and uphold a good quality of life. |
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| ISSN: | 2045-7634 |