2. Osteosarcoma

  1. Ross Pinkerton MB, BCh, BaO, MD Executive Director, Division of Oncology2,
  2. Ananth Shankar MD, FRCPCH Consultant in Paediatric and Adolescent Oncology3 and
  3. Katherine K. Matthay BA, MD Mildred V. Strouss Professor of Translational Oncology, Director, Pediatric Hematology-Oncology4
  1. Katherine K. Matthay

Published Online: 8 MAY 2013

DOI: 10.1002/9781118625309.ch2

Evidence-Based Pediatric Oncology

Evidence-Based Pediatric Oncology

How to Cite

Matthay, K. K. (2013) Osteosarcoma, in Evidence-Based Pediatric Oncology (eds R. Pinkerton, A. Shankar and K. K. Matthay), John Wiley & Sons, Ltd, Oxford. doi: 10.1002/9781118625309.ch2

Editor Information

  1. 2

    Royal Children's Hospital, Children's Health Queensland, Brisbane, QLD, Australia

  2. 3

    University College London Hospitals NHS Foundation Trust London, UK

  3. 4

    Department of Pediatrics, UCSF School of Medicine and, UCSF Benioff Children's Hospital, San Francisco, CA, USA

Author Information

  1. UCSF School of Medicine, San Francisco, CA, USA

Publication History

  1. Published Online: 8 MAY 2013
  2. Published Print: 20 MAY 2013

ISBN Information

Print ISBN: 9780470659649

Online ISBN: 9781118625309



  • biological markers;
  • children;
  • L-MTP-PE;
  • oncology;
  • osteosarcoma;
  • randomized trial


The current dilemma in osteosarcoma management surrounds the role of a novel biological agent, liposomal muramyl tripeptide phosphatidyl ethanolamine (L-MTP-PE, mifurmatide). Interest in L-MTP-PE was initially generated as preclinical data demonstrated responses in metastatic pulmonary osteosarcoma in animal models. A large phase III randomized trial was conducted by the Pediatric Oncology Group (POG)/Children's Oncology Group (COG) in the US to provide evidence of efficacy. The interpretation of the published reports of the study has, however, caused controversy. The context is that prior to these publications, there has been no significant improvement in survival for patients with osteosarcoma during the last two decades. Does adjuvant use of L-MTP-PE represent a breakthrough? Before addressing this question, the chapter discusses progress in osteosarcoma management to date. It provides a summary of previous studies and presents three new studies.