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Clinical Studies

Relapsed rhabdomyosarcoma: treatment recommendations from the European pediatric soft tissue sarcoma study group (EpSSG)

British Journal of Cancer (2025)Cite this article

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Abstract

At least one-third of patients with localized rhabdomyosarcoma (RMS) and 60–70% of patients with metastatic RMS experience progressive disease or relapse. Following relapse, outcomes generally remain poor with limited treatment options and a high risk of subsequent recurrence. Optimal treatment requires a multidisciplinary approach incorporating chemotherapy with local control. Given the complexity of managing relapsed RMS and the challenges in developing effective treatment strategies, we aim to present clear and practical recommendations on the management of these patients across Europe. These recommendations were developed collaboratively by a group of pediatric and adolescent sarcoma experts from the European paediatric Soft Tissue Sarcoma Study Group. A careful review of the literature was performed to ensure that wherever possible recommendations are supported by the results of clinical trials or substantive retrospective reports. Such recommendations provide a standardized approach to managing relapsed cases, improving patient outcomes and offering a framework for clinicians to make informed decisions.

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Funding

This systematic review was not funded. JCC is supported by The Giant Pledge through the Royal Marsden Cancer Charity. This work represents independent research supported by the National Institute for Health Research (NIHR) Biomedical Research Center at The Royal Marsden NHS Foundation Trust and the Institute of Cancer Research, London. The views expressed are those of the authors and not necessarily those of the NIHR or the Department of Health and Social Care.

Author information

Author notes

  1. These authors contributed equally: J. C. Chisholm, S. A. Gatz.

Authors and Affiliations

  1. Department of Pediatric and AYA Oncology, Oscar Lambret Center, Lille, France

    A. S. Defachelles

  2. Department of Oncology, University Children’s Hospital Zürich, Zürich, Switzerland

    W. B. Breunis

  3. Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy

    M. Casanova

  4. Department of Paediatric Oncology, Gustave Roussy, Paris, France

    V. Minard-Collin

  5. Pediatric Oncology and Hematology Department, Hospital Universitari Vall d’Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain

    R. Hjadun

  6. The Institute of Cancer Research, London, UK

    A. Wasti

  7. Radiation Oncology department, University Medical Center Utrecht, Utrecht, the Netherlands

    R. Dávila Fajardo

  8. Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands

    R. Dávila Fajardo, S. Terwisscha van Scheltinga & J. H. M. Merks

  9. KickCancer Foundation, Brussels, Belgium

    D. Heenen

  10. Department of Hematology and Oncology, University Children’s Hospital Tübingen, Tübingen, Germany

    A. T. Heinz & M. Sparber-Sauer

  11. Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin (Olgahospital), Pädiatrie 5 (Pädiatrische Onkologie, Hämatologie, Immunologie), Klinikum der Landeshauptstadt Stuttgart, Stuttgart, Germany

    A. T. Heinz & M. Sparber-Sauer

  12. Division of Imaging and Oncology, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands

    J. H. M. Merks

  13. Children and Young People’s Unit, Royal Marsden Hospital and Institute of Cancer Research, Sutton, UK

    J. C. Chisholm

  14. Department of Cancer and Genomic Sciences, College of Medicine and Health, University of Birmingham, Birmingham, UK

    S. A. Gatz

  15. Department of Paediatric Oncology, Birmingham Women’s and Children’s NHS Foundation Trust, Birmingham, UK

    S. A. Gatz

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  1. A. S. Defachelles
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Contributions

Conceptualization, ASD, WBB, MC, VMC, JHMM, JCC, and SAG; writing-original draft preparation ASD; writing-review and editing ASD, WBB, JCC, and SAG. DH provided feedback from a patient/parent perspective. MC, VMC, JHMM, RH; AW, RDF, STvS, ATH, and MSp critically reviewed and all authors approved the final manuscript. Prior presentation: Presented at the annual conference of the International Society of European Pediatric Oncology (SIOPe), Valencia, Spain in May 2023 in the ITCC-EpSSG-CWS joint session.

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Correspondence to A. S. Defachelles.

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Competing interests

SAG has/has had an advisory role for EMD Serono/MERCK KGaA, AMGEN, and GILEAD; signed a consultancy agreement with AstraZeneca and Schroedinger Therapeutics; and received research funding from AstraZeneca (own grant and fee to institution), GSK (fee to institution), and BAYER (grant outside of this project). MC has/has had advisory roles AstraZeneca/Alexion, Roche; Merck, Servier; Speaker fee and travel expenses from Bayer. JHMM has/had advisory roles for MERCK, GSK, and BAYER. MSp had advisory roles for SOBI, Roche (hemophilia) and BAYER (hemophilia and funding outside this project). All other authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. The other authors declare no competing interests.

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Defachelles, A.S., Breunis, W.B., Casanova, M. et al. Relapsed rhabdomyosarcoma: treatment recommendations from the European pediatric soft tissue sarcoma study group (EpSSG). Br J Cancer (2025). https://doi.org/10.1038/s41416-025-03130-1

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