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Multi-Targeted TKIs in Patients with Advanced Ewing Sarcoma: A Systematic Review and Single-Arm Meta-Analysis.

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  • 1Department of Hematology/Oncology, University of Virginia, Charlottesville, VA 22903, USA.

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|February 13, 2026
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Summary
This summary is machine-generated.

Tyrosine kinase inhibitors (TKIs) show anti-tumor activity in relapsed Ewing sarcoma, with an objective response rate of 23%. Further research is needed to fully understand TKI efficacy and toxicity in this patient population.

Keywords:
Ewing sarcomaTKImultiply refractory diseasetyrosine kinase inhibitor

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Area of Science:

  • Oncology
  • Pharmacology

Background:

  • Standard treatment for multiply relapsed Ewing sarcoma is not well-established.
  • Tyrosine kinase inhibitors (TKIs) with anti-angiogenic properties show promise.
  • A systematic review and meta-analysis was conducted to evaluate TKIs in Ewing sarcoma.

Purpose of the Study:

  • To explore the efficacy and safety of tyrosine kinase inhibitors (TKIs) in patients with Ewing sarcoma.
  • To analyze objective response rate (ORR) and disease control rate (DCR) of TKIs.
  • To compare outcomes between clinical trials and real-world studies.

Main Methods:

  • Systematic review and meta-analysis of PubMed, Embase, and Cochrane databases.
  • Inclusion of clinical trials and cohort studies of advanced Ewing sarcoma patients with prior therapy.
  • Pooled analysis using random effects models to determine ORR and DCR.

Main Results:

  • 14 studies with 257 patients evaluated various TKIs.
  • Pooled ORR was 23% (95% CI, 11.2-37.1%) and DCR was 61.1% (95% CI, 47.3-74.2%).
  • Single-agent cabozantinib and regorafenib showed superior outcomes among FDA-approved TKIs.

Conclusions:

  • Anti-angiogenic TKIs demonstrate significant anti-tumoral activity in Ewing sarcoma.
  • Consistent efficacy observed in both clinical trials and real-world data.
  • Limitations in study design and population data impact definitive conclusions on toxicity.