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Regorafenib in Pediatric Patients With Advanced Osteosarcoma: A Case Series from a Single Institution
Yumi Matsuyama1, Kunihiro Asanuma2, Tomohito Hagi1
1Department of Orthopaedic Surgery, Mie University Graduate School of Medicine, Tsu, Japan.
Background/Aim:
Pediatric osteosarcoma is a rare and aggressive malignancy with limited treatment options in cases of progression or metastasis. Regorafenib is an oral multikinase inhibitor targeting vascular endothelial growth factor receptor (VEGFR), platelet-derived growth factor receptor (PDGFR), and other kinases, currently approved in Japan for certain gastrointestinal cancers. Its clinical use in osteosarcoma, especially in pediatric patients, has not been well established.
Patients And Methods:
We retrospectively analyzed six pediatric patients with advanced osteosarcoma who were treated with regorafenib at our institution. All patients had previously received standard treatment, including neoadjuvant and adjuvant chemotherapy as well as surgery, and were considered to have disease progression or resistance prior to regorafenib administration. Clinical background, treatment response, adverse events, and progression-free survival (PFS) were evaluated. Tumor response was assessed using RECIST criteria, and adverse events were graded according to CTCAE v5.0.
Results:
The average age was 16 years (range=12-18 years), and all patients had received prior surgery and chemotherapy. Regorafenib was administered at adult equivalent doses, with dose reductions required in 3 of 6 patients. Four patients (66.7%) achieved stable disease for at least eight weeks. The longest PFS exceeded 12 months. Common toxicities included diarrhea and hematuria; no grade ≧3 adverse events were observed. All patients underwent genomic testing to guide treatment planning.
Conclusion:
This case series suggests that regorafenib may provide disease control with acceptable safety in pediatric patients with advanced osteosarcoma. Further studies are needed to evaluate its efficacy and tolerability in this population as well as in adult patients.
Insights
Regorafenib shows promise in controlling advanced pediatric osteosarcoma, with four of six patients achieving stable disease. This oral multikinase inhibitor demonstrated acceptable safety in a small case series.
Area of Science:
- Pediatric Oncology
- Medical Oncology
- Pharmacology
Background:
- Pediatric osteosarcoma is a rare, aggressive cancer with limited treatment options for advanced or metastatic disease.
- Regorafenib, an oral multikinase inhibitor, is approved for some gastrointestinal cancers but its use in pediatric osteosarcoma is not well-established.
Purpose of the Study:
- To evaluate the efficacy and safety of regorafenib in pediatric patients with advanced osteosarcoma.
- To assess treatment response, adverse events, and progression-free survival (PFS) in this patient population.
Main Methods:
- Retrospective analysis of six pediatric patients with advanced osteosarcoma treated with regorafenib.
- Patients had previously undergone standard treatments including chemotherapy and surgery.
- Tumor response assessed by RECIST criteria; adverse events graded by CTCAE v5.0.
Main Results:
- Four out of six patients (66.7%) achieved stable disease for at least eight weeks.
- The longest progression-free survival (PFS) exceeded 12 months.
- Common toxicities included diarrhea and hematuria; no severe (grade ≧3) adverse events were observed.
Conclusions:
- Regorafenib may offer disease control with acceptable safety in pediatric patients with advanced osteosarcoma.
- Further research is warranted to confirm efficacy and tolerability in both pediatric and adult populations.
