Metastatic adult-type non-rhabdomyosarcoma soft tissue sarcomas in children and adolescents: A cohort study from the European paediatric Soft tissue sarcoma Study Group |
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| Authors: | Andrea Ferrari MD Daniel Orbach MD Michela Casanova MD Max M van Noesel MD Pablo Berlanga MD Bernadette Brennan MD Nadege Corradini MD Reineke A Schoot MD Gema L Ramirez-Villar MD Lisa Lyngsie Hjalgrim MD Rita Alaggio MD Gabriela Guillen Burrieza MD Akmal Safwat MD Alison L Cameron MD Rick R van Rijn MD Veronique Minard-Colin MD Ilaria Zanetti BSc Gianni Bisogno MD Julia C Chisholm MD Johannes H M Merks MD |
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| Institution: | 1. Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milano, Italy;2. SIREDO Oncology Center, Institut Curie, PSL University, Paris, France;3. Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands;4. Department of Pediatric and Adolescent Oncology, Gustave-Roussy, Cancer Campus, Université Paris-Saclay, Villejuif, France;5. Pediatric Oncology, Royal Manchester Children's Hospital, Manchester, UK;6. Department of Pediatric Oncology, Institut d’Hematologie et d’Oncologie Pédiatrique,/Centre Léon Bérard, Lyon, France;7. Pediatric Oncology Unit, Hospital Universitario Virgen del Rocío, Sevilla, Spain;8. Department of Pediatrics and Adolescent Medicine, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark;9. Pathology Department, Ospedale Pediatrico Bambino Gesù IRCCS, Rome, Italy;10. Surgical Oncology and Neonatal Surgery, Pediatric Surgery Department, Hospital Infantil Universitari Vall d’Hebron, Barcelona, Spain;11. Oncology Department and Danish Center for Particle Therapy, Aarhus University Hospital, Aarhus, Denmark;12. Bristol Haematology and Oncology Centre, University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, UK;13. Department of Radiology and Nuclear Medicine, UMC University of Amsterdam, Amsterdam, Netherlands;14. Pediatric Hematology Oncology Division, University Hospital of Padua, Padua, Italy;15. Children and Young People's Unit, The Royal Marsden Hospital and Institute of Cancer Research, Sutton, UK;16. Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands
Division of Imaging and Oncology, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands |
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| Abstract: | Background Limited data exist on the clinical behavior of pediatric non-rhabdomyosarcoma soft tissue sarcomas (NRSTS) with distant metastases at onset, and a clear standard of care has not yet been defined. Methods This cohort study reports on pediatric adult-type metastatic NRSTS enrolled in two concurrent prospective European studies, i.e., the randomized BERNIE study and the single-arm MTS 2008 study developed by the European paediatric Soft tissue sarcoma Study Group. Treatment programs were originally designed for patients with metastatic rhabdomyosarcoma, i.e., nine courses of multidrug chemotherapy (with or without bevacizumab in the BERNIE study), followed by 12 cycles of maintenance therapy, whereas radiotherapy and/or surgery (on primary tumor and/or metastases) were delayed until after seven courses of chemotherapy had been administered. Results The study included 61 patients <21 years old treated from July 2008 to December 2016. The lung was the site of metastases in 75% of the cases. All patients received multi-agent chemotherapy, 44% had local therapy to primary tumor, and 18% had treatment of metastases. Median time to progression/relapse was 6 months. A high rate of tumor progression was observed during the initial part of the chemotherapy program. With a median follow-up of 41.5 months (range, 2–111 months), 3-year event-free survival and overall survival were 15.4% (95% confidence interval CI], 7.6–25.7) and 34.9% (95% CI, 22.7–47.5), respectively. There were no statistically significant differences in outcome depending on the type of treatment administered. Conclusions The study confirmed the overall poor outcome for patients with metastatic NRSTS, whose treatment remains a challenge. Plain Language Summary - Pediatric non-rhabdomyosarcoma soft tissue sarcomas form a heterogeneous group of rare tumors.
- Although recent international studies have defined the standard of care for patients with localized disease, limited data are available on the clinical behavior of patients with distant metastases.
- This study on 61 metastatic cases treated on two prospective European protocols confirms that the chances of survival of such patients are often dismal and a standard treatment is still lacking.
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| Keywords: | adolescents children metastases non-rhabdomyosarcoma soft tissue sarcomas outcome prognostic factors treatment |
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