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Real-World Data on Cabozantinib in Advanced Osteosarcoma and Ewing Sarcoma Patients: A Study from the Hellenic Group
Stefania Kokkali1,2, Anastasios Kyriazoglou3, Elpida Mangou1
1Department of Medical Oncology, Saint-Savvas Anticancer Hospital, 11522 Athens, Greece.
Abstract:
Advanced osteosarcomas (OSs) and Ewing sarcomas (ESs) tend to have poor prognosis with limited therapeutic options beyond first-line therapy. Aberrant angiogenesis and MET signaling play an important role in preclinical models. The anti-angiogenic drug cabozantinib was tested in a phase 2 trial of advanced OS and ES and was associated with clinical benefits. We retrospectively analyzed the off-label use of cabozantinib in adult patients with advanced OS and ES/primitive neuroectodermal tumors (PNETs) in three centers of the Hellenic Group of Sarcoma and Rare Cancers (HGSRC). Between April 2019 and January 2022, 16 patients started taking 60 mg of cabozantinib for advanced bone sarcoma or PNET. Median age at cabozantinib initiation was 31 years (17-83). All patients had received peri-operative chemotherapy for primary sarcoma and between 0 and 4 lines of treatment (median; 2.5) for advanced disease. The most common adverse effects included fatigue, anorexia, hypertransaminasemia, weight loss, and diarrhea. One toxic death was noted (cerebral hemorrhage). Dose reduction to 40 mg was required in 31.3% of the patients. No objective response was noted, and 9/16 patients exhibited stable disease outcomes. Progression-free survival varied from 1 to 8 (median; 5) months. Our study demonstrates that cabozantinib has antitumor activity in this population. In the real-life setting, we observed similar adverse events as in the CABONE study and in other neoplasms.
Insights
Cabozantinib shows antitumor activity in advanced osteosarcomas (OS) and Ewing sarcomas (ES). This real-world study observed stable disease in most patients, with manageable side effects similar to prior trials.
Area of Science:
- Oncology
- Medical Research
- Pharmacology
Background:
- Advanced osteosarcomas (OS) and Ewing sarcomas (ES) have poor prognoses and limited treatment options.
- Aberrant angiogenesis and MET signaling are implicated in OS and ES progression.
- Cabozantinib, an anti-angiogenic drug, showed promise in a phase 2 trial for advanced OS and ES.
Purpose of the Study:
- To retrospectively analyze the off-label use of cabozantinib in adult patients with advanced OS and ES/primitive neuroectodermal tumors (PNETs).
- To evaluate the clinical benefits and safety profile of cabozantinib in a real-world setting.
Main Methods:
- Retrospective analysis of 16 adult patients with advanced bone sarcoma or PNET treated with cabozantinib (60 mg initially).
- Patients received prior chemotherapy for primary sarcoma and 0-4 lines for advanced disease.
- Data collected on adverse events, objective response, stable disease, and progression-free survival.
Main Results:
- No objective responses were observed; 9 out of 16 patients achieved stable disease.
- Median progression-free survival was 5 months (range: 1-8 months).
- Common adverse events included fatigue, anorexia, hypertransaminasemia, weight loss, and diarrhea; one toxic death (cerebral hemorrhage) occurred. Dose reduction was needed in 31.3%.
Conclusions:
- Cabozantinib demonstrates antitumor activity in patients with advanced osteosarcoma and Ewing sarcoma/PNET in a real-world setting.
- Observed adverse events were consistent with previous studies, highlighting the need for careful monitoring.
- Further investigation is warranted to optimize cabozantinib use in these rare cancers.
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