European standard clinical practice recommendations for children and adolescents with Rhabdomyosarcoma a joint EpSSG, CWS and ERN PaedCan project

Rhabdomyosarcoma (RMS) is a heterogeneous group of malignancies with specific histopathological characteristics. Distinct molecular findings help to classify RMS into PAX::FOXO1 fusion gene positive and fusion gene negative subtypes. Further new molecular subtypes give insight into the heterogeneity...

Full description

Saved in:
Bibliographic Details
Main Authors: Johannes H.M. Merks, Eva Brack, Martin Ebinger, Véronique Minard-Colin, Anne-Sophie Defachelles, Raquel Hladun, Timothy Rogers, Jörg Fuchs, Jan Godzinski, Sheila Terwisscha van Scheltinga, Gabriela Guillén Burrieza, Henry Mandeville, Beate Timmermann, Raquel Davila Fajardo, Rick R. van Rijn, Jürgen Schäfer, Helen Rees, Andrea Ferrari, Rita Alaggio, Anna Kelsey, Julia C. Chisholm, Christian Vokuhl, Monika Sparber-Sauer, Gianni Bisogno
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:EJC Paediatric Oncology
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2772610X25000169
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Rhabdomyosarcoma (RMS) is a heterogeneous group of malignancies with specific histopathological characteristics. Distinct molecular findings help to classify RMS into PAX::FOXO1 fusion gene positive and fusion gene negative subtypes. Further new molecular subtypes give insight into the heterogeneity of these rare tumours. Multiple international clinical trials have been conducted to improve the prognosis for paediatric, adolescent, and young adult patients with RMS. The overall cure rate is around 70 % but varies dramatically between the low risk localised and very high-risk metastatic patients. New treatment approaches are needed in the High-Risk and Very High-Risk RMS disease to improve patients’ outcome. State of the art diagnostics and staging is crucial and the multimodal treatment approach in specialized paediatric-oncology centres is highly recommended. Treatment includes chemotherapy with vincristine, actinomycin, and ifosfamide with or without anthracyclines. Local treatment with the aim of microscopically complete resection and/or radiotherapy is warranted depending on patient and tumour characteristics. Maintenance treatment is recommended to (Very) High Risk groups. This consensus summarizes the standard of care diagnostic work up, multimodal treatment and surveillance recommendations for paediatric, adolescent, and young adult patients with RMS. The guideline was developed as a joint project by the European paediatric Soft tissue sarcoma Study Group (EpSSG) and the Cooperative Weichteilsarkom Studiengruppe (CWS) summarized as the European RMS working group supported by European Reference Network on Paediatric Cancer (ERN PaedCan).
ISSN:2772-610X