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PD-1 blockade using pembrolizumab in adolescent and young adult patients with advanced bone and soft tissue sarcoma
Tahlia Scheinberg1,2,3, Anna Lomax1, Martin Tattersall1,2
1Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.
Background:
Sarcomas represent 10%-15% of cancers in adolescent and young adult (AYA) patients, and survival for those with metastatic disease or relapse is poor. Immunotherapy with checkpoint inhibition has improved outcomes in multiple tumor types, but data in advanced sarcomas, particularly within the AYA population, are limited.
Aim:
We aim to evaluate response and toxicity for AYA patients with sarcoma treated with pembrolizumab.
Methods And Results:
We retrospectively reviewed AYA patients with advanced bone and soft tissue sarcoma who received self-funded pembrolizumab between May 2015 and January 2019. Eighteen patients were identified. One patient with Ewing sarcoma had a sustained complete response to therapy. Two patients with alveolar soft part sarcoma received a clinical benefit from pembrolizumab: one had a radiological partial response with an excellent clinical response and one patient achieved stable disease. Four patients died of disease prior to first scheduled assessment and thus were not evaluable. The remaining eleven patients had progressive disease.
Conclusion:
The role of immunotherapy in AYA sarcoma warrants further investigation. Biomarkers of response need to be further evaluated in order to guide patient selection.
Insights
Immunotherapy with pembrolizumab showed limited efficacy in advanced sarcomas for adolescent and young adult (AYA) patients. Further research is needed to identify biomarkers for patient selection in sarcoma treatment.
Area of Science:
- Oncology
- Immunotherapy
- Sarcoma Research
Background:
- Sarcomas account for 10%-15% of adolescent and young adult (AYA) cancers.
- Survival rates for advanced or relapsed sarcomas in AYAs are poor.
- Checkpoint inhibition immunotherapy shows promise but lacks data in advanced sarcomas, especially in AYAs.
Purpose of the Study:
- To evaluate the response and toxicity of pembrolizumab in AYA patients with sarcoma.
- To assess the efficacy of immunotherapy in this specific patient population.
Main Methods:
- Retrospective review of AYA patients with advanced bone and soft tissue sarcoma.
- Patients received self-funded pembrolizumab between May 2015 and January 2019.
- Eighteen patients were identified and analyzed for treatment outcomes.
Main Results:
- One patient with Ewing sarcoma achieved a complete response.
- Two patients with alveolar soft part sarcoma showed clinical benefit (partial response or stable disease).
- Eleven patients experienced progressive disease, and four were not evaluable due to early mortality.
Conclusions:
- Immunotherapy's role in AYA sarcoma requires further investigation.
- Biomarkers are crucial for selecting appropriate patients for immunotherapy.
- Pembrolizumab demonstrated limited efficacy in this advanced sarcoma cohort.
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