Benefit assessment of novel systemic therapies for bone and soft tissue sarcomas: a cross-sectional study

Qiang He1,2, Zhen Wang3, Jie Mei1

  • 1Department of Orthopedics, Nanjing University of Chinese Medicine Affiliated Nanjing Hospital of Traditional Chinese Medicine, Nanjing, China.

Abstract

Insights

Novel drug therapies for bone and soft tissue sarcomas show limited clinical benefit. A decade of research reveals few treatments offer substantial patient advantages, highlighting challenges in sarcoma drug development.

Area of Science:

  • Oncology
  • Clinical Pharmacology

Background:

  • Bone and soft tissue sarcomas are rare and heterogeneous cancers.
  • Drug development for these malignancies has been historically challenging.

Purpose of the Study:

  • To evaluate the clinical benefits of novel systemic therapies for sarcoma over the past decade.
  • To apply the European Society of Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS) and the American Society of Clinical Oncology-Value Framework (ASCO-VF) to assess treatment efficacy.

Main Methods:

  • A systematic review of randomized controlled trials (RCTs) published between 2013 and 2023 was conducted.
  • Searches were performed on PubMed and Embase databases for systemic sarcoma therapies.
  • Included RCTs were scored using ESMO-MCBS v1.1 and ASCO-VF v2.

Main Results:

  • Out of 52 included RCTs, 17 (32.7%) demonstrated positive results favoring the experimental treatment.
  • Only 3 of the 17 positive trials (21.4% by ESMO-MCBS, 27.3% by ASCO-VF) met criteria for meaningful clinical benefit.
  • A weak correlation and agreement were found between the ESMO-MCBS and ASCO-VF scoring systems.

Conclusions:

  • Few novel systemic therapies for bone and soft tissue sarcomas have shown significant patient benefit in the last decade.
  • The heterogeneity of sarcomas presents ongoing challenges for developing effective treatments.
  • Further research is needed to improve clinical outcomes for sarcoma patients.