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Transcriptome based individualized therapy of refractory pediatric sarcomas: feasibility, tolerability and efficacy

Bushra Weidenbusch1, Günther H S Richter1,2, Marie Sophie Kesper1,2

  • 1Department of Pediatrics and Children's Cancer Research Center, Kinderklinik München Schwabing, Klinikum rechts der Isar, Fakultät für Medizin, Technische Universität München, Munich, Germany.

Oncotarget
|May 15, 2018
PubMed

Insights

Gene expression profiling identified actionable targets in pediatric sarcomas. Targeted therapy significantly improved overall survival and progression-free survival in refractory cases, showing promise for future treatment strategies.

Area of Science:

  • Pediatric Oncology
  • Molecular Diagnostics
  • Cancer Therapeutics

Background:

  • Pediatric sarcoma survival rates have plateaued, particularly for adolescents and young adults (AYAs).
  • Refractory cases require novel treatment strategies beyond conventional therapies.
  • Gene expression profiling offers a method to characterize tumor transcriptomes and guide personalized treatment.

Purpose of the Study:

  • To assess the feasibility and efficacy of expression-based targeted therapy (TT) in pediatric sarcoma patients.
  • To identify actionable molecular targets in refractory pediatric sarcomas using gene expression profiling.
  • To compare outcomes, including survival and adverse events, between patients receiving TT and those not.

Main Methods:

  • Array-based gene expression profiling was performed on tumor biopsies from 20 pediatric sarcoma patients (ages 8-35).
  • Actionable targets and deregulated pathways were identified to guide individualized TT selection.
  • Patients receiving TT were compared to a control group (non TT) regarding disease status, survival, adverse events (AEs), and quality of life (QOL).

Main Results:

  • Actionable targets were identified in all analyzed pediatric sarcoma biopsies.
  • Nine patients received TT, while eleven did not; no significant differences in risk factors were observed between groups.
  • TT significantly improved overall survival (OS) and progression-free survival (PFS) compared to non TT (median OS: 8.83 vs. 4.93 months; median PFS: 6.17 vs. 1.6 months).
  • Quality of life (QOL) did not differ between groups. TT patients experienced fewer grade 1 AEs, with no difference in grade 2-4 AEs.

Conclusions:

  • Expression-based targeted therapy is a feasible approach for refractory pediatric sarcomas.
  • Targeted therapy demonstrated significant survival benefits in this patient cohort.
  • Further investigation is warranted to confirm the efficacy and benefits of targeted therapy in pediatric sarcoma treatment.
Keywords:
adolescents and young adultsexpression profilingpediatric cancersarcomatargeted therapy

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