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Metastatic Rhabdomyosarcoma: Results of the European Paediatric Soft Tissue Sarcoma Study Group MTS 2008 Study and Pooled Analysis With the Concurrent BERNIE Study

Authors :
Reineke A. Schoot
Julia C. Chisholm
Michela Casanova
Veronique Minard-Colin
Birgit Geoerger
Alison L. Cameron
Beatrice Coppadoro
Ilaria Zanetti
Daniel Orbach
Anna Kelsey
Timothy Rogers
Cecile Guizani
Markus Elze
Myriam Ben-Arush
Kieran McHugh
Rick R. van Rijn
Sima Ferman
Soledad Gallego
Andrea Ferrari
Meriel Jenney
Gianni Bisogno
Johannes H.M. Merks
Institut Català de la Salut
[Schoot RA] Princess Máxima Centre for Paediatric Oncology, Utrecht, the Netherlands. [Chisholm JC] Children and Young Peoples Unit, Royal Marsden Hospital and Institute of Cancer Research, Sutton, Surrey, United Kingdom. [Casanova M] Paediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy. [Minard-Colin V] Gustave-Roussy Cancer Campus, Department of Paediatric and Adolescent Oncology, Université Paris-Saclay, Villejuif, France. [Geoerger B] Gustave-Roussy Cancer Campus, Department of Paediatric and Adolescent Oncology, Université Paris-Saclay, Villejuif, France. Gustave-Roussy Cancer Campus, INSERM U1015, Université Paris Saclay, Villejuif, France. [Cameron AL] Bristol Haematology and Oncology Centre, University Hospitals Bristol. [Gallego S] Servei d’Oncologia i Hematologia Pediàtriques, Vall d’Hebron Hospital Universitari, Barcelona, Spain
Vall d'Hebron Barcelona Hospital Campus
Radiology and Nuclear Medicine
Other Research
Source :
Scientia, Journal of clinical oncology, 19(32):JCO.21.02981, 3730-3740. American Society of Clinical Oncology
Publication Year :
2022
Publisher :
American Society of Clinical Oncology, 2022.

Abstract

PURPOSE Outcome for patients with metastatic rhabdomyosarcoma (RMS) is poor. This study presents the results of the MTS 2008 study with a pooled analysis including patients from the concurrent BERNIE study. PATIENTS AND METHODS In MTS 2008, patients with metastatic RMS received four cycles of ifosfamide, vincristine, and actinomycin D (IVA) plus doxorubicin, five cycles of IVA, and 12 cycles of maintenance chemotherapy (low-dose cyclophosphamide and vinorelbine). The BERNIE study randomly assigned patients to the addition or not of bevacizumab to the same chemotherapy. Local therapy (surgery/radiotherapy) was given to the primary tumor and all metastatic sites when feasible. RESULTS MTS 2008 included 270 patients (median age, 9.6 years; range, 0.07-20.8 years). With a median follow-up of 50.3 months, 3-year event-free survival (EFS) and overall survival (OS) were 34.9% (95% CI, 29.1 to 40.8) and 47.9% (95% CI, 41.6 to 53.9), respectively. In pooled analyses on 372 patients with a median follow-up of 55.2 months, 3-year EFS and OS were 35.5% (95% CI, 30.4 to 40.6) and 49.3% (95% CI, 43.9 to 54.5), respectively. Patients with ≤ 2 Oberlin risk factors (ORFs) had better outcome than those with ≥ 3 ORFs: 3-year EFS was 46.1% versus 12.5% ( P < .0001) and 3-year OS 60.0% versus 26.0% ( P < .0001). Induction chemotherapy and maintenance appeared tolerable; however, about two third of patients needed dose adjustments during maintenance. CONCLUSION Outcome remains poor for patients with metastatic RMS and multiple ORFs. Because of the design of the studies, it was not possible to determine whether the intensive induction regimen and/or the addition of maintenance treatment resulted in apparent improvement of outcome compared with historical cohorts. Further studies, with novel treatment approaches are urgently needed, to improve outcome for the group of patients with adverse prognostic factors.

Details

Language :
English
ISSN :
0732183X
Database :
OpenAIRE
Journal :
Scientia, Journal of clinical oncology, 19(32):JCO.21.02981, 3730-3740. American Society of Clinical Oncology
Accession number :
edsair.doi.dedup.....ba487c58a9b7c2c5a505d6da4056f724