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.

Abstract

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.