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Pazopanib in patients with primary multi-metastatic bone Ewing sarcoma
Anna Raciborska1, Katarzyna Bilska1, Jadwiga Węcławek-Tompol2
1Department of Oncology and Surgical Oncology for Children and Youth, Institute of Mother and Child, Warsaw, Poland.
Background:
Despite the use of different treatment regimens, patients with primary multi-metastatic Ewing sarcoma disease have a dismal outcome. Lately, pazopanib has been proposed as an effective salvage regimen for soft tissue sarcoma (STS), including extraosseous Ewing sarcoma (ESS). Thus, we sought to evaluate this approach for young patients with primary multi-metastatic bone Ewing sarcoma.
Materials And Methods:
Eleven patients with primary multi-metastatic bone Ewing sarcoma (metastasis to the bone and/or bone marrow), received standard first-line treatment in parallel with pazopanib. All patients had standard tumor imaging and laboratory evaluation. All toxicities were documented.
Results:
Pazopanib was administered throughout the whole treatment period (paused during the surgical procedure) and after its completion, on average 1.7 years (range 0.9 to 5.1). At the time of the beginning of pazopanib, the median age was 14.2 years (range 5.1 to 17.8 years). The primary tumor was operated on in five patients. Ten patients received concurrent radiation therapy, and 3 autologous hematopoietic stem cell transplantation. Significant toxicities have not been observed. One patient (9.1%) progressed. Two patients had relapse (18.2%), and one patient died (9.1%). Ten patients (90.9%) are alive with a median time follow-up 2.6 years (range 1.2 to 9.2 years). The estimated 2-year event-free survival and overall survival for the whole group were 68.2% and 85.7%, respectively.
Conclusions:
Pazopanib was well-tolerated in young patients, even when it was administered with chemotherapy and radiation therapy together. Pazopanib turned out to be effective in patients with primary multi-metastatic Ewing sarcoma and particularly could be considered as an option for them. This regimen deserves further investigation.
Insights
Pazopanib shows promise as a well-tolerated and effective treatment for young patients with multi-metastatic bone Ewing sarcoma, improving survival outcomes. Further investigation into this salvage regimen is warranted.
Area of Science:
- Pediatric Oncology
- Medical Oncology
- Sarcoma Research
Background:
- Primary multi-metastatic Ewing sarcoma in young patients has a poor prognosis despite various treatment regimens.
- Pazopanib, a tyrosine kinase inhibitor, has demonstrated efficacy in treating soft tissue sarcomas (STS), including extraosseous Ewing sarcoma (ESS).
- The study evaluates pazopanib as a potential salvage therapy for primary multi-metastatic bone Ewing sarcoma in pediatric patients.
Purpose of the Study:
- To assess the safety and efficacy of pazopanib in young patients with primary multi-metastatic bone Ewing sarcoma.
- To evaluate pazopanib's tolerability when administered concurrently with standard treatments like chemotherapy and radiation therapy.
- To determine the survival outcomes and toxicity profile of pazopanib in this patient population.
Main Methods:
- Eleven pediatric patients with primary multi-metastatic bone Ewing sarcoma received pazopanib alongside standard first-line treatments.
- Patients underwent regular tumor imaging and laboratory evaluations to monitor disease status and treatment response.
- Toxicities associated with pazopanib administration were meticulously documented throughout the study period.
Main Results:
- Pazopanib was well-tolerated in young patients, with no significant observed toxicities, even when combined with chemotherapy and radiation.
- The estimated 2-year event-free survival was 68.2%, and overall survival was 85.7%.
- Ten out of eleven patients (90.9%) remained alive at a median follow-up of 2.6 years, with only one progression and two relapses.
Conclusions:
- Pazopanib is a well-tolerated and effective treatment option for young patients with primary multi-metastatic Ewing sarcoma.
- The combination of pazopanib with standard therapies warrants further investigation for improved outcomes in this challenging disease.
- Pazopanib should be considered a viable salvage regimen for pediatric patients diagnosed with multi-metastatic bone Ewing sarcoma.
