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Localized incompletely resected standard risk rhabdomyosarcoma in children and adolescents: Results from the European Paediatric Soft Tissue Sarcoma Study Group RMS 2005 trial

HC. Mandeville, G. Bisogno, V. Minard-Colin, R. Alaggio, M. Ben-Arush, C. Chargari, B. Coppadoro, R. Craigie, C. Devalck, S. Ferman, A. Ferrari, H. Glosli, RH. Alvaro, M. Hol, P. Mudry, D. Orbach, MR. Albiac, JHM. Merks, MEM. Jenney

. 2024 ; 130 (23) : 4071-4084. [pub] 20240726

Jazyk angličtina Země Spojené státy americké

Typ dokumentu časopisecké články, multicentrická studie

Perzistentní odkaz   https://www.medvik.cz/link/bmc25003314

Grantová podpora
Fondazione Città della Speranza
Alice's Arc Children's Cancer Charity

BACKGROUND: The authors report the prospective evaluation of reduced dose alkylator chemotherapy combined with radiotherapy for European Pediatric Soft Tissue Sarcoma Study Group (EpSSG) standard risk nonalveolar rhabdomyosarcoma (NA-RMS). PATIENTS AND METHODS: Localized node negative Intergroup Rhabdomyosarcoma Study (IRS) II/III NA-RMS at favorable sites (subgroup C), <25 years old, received five cycles of ifosfamide, vincristine, and dactinomycin (IVA) chemotherapy (30 g/m2 ifosfamide) and four cycles of vincristine and dactinomycin (if receiving radiotherapy), or nine cycles of IVA (54 g/m2 ifosfamide) ± radiotherapy. Delayed primary tumor excision was considered for IRS III tumors. The primary end points were event-free survival (EFS) and overall survival (OS). RESULTS: From October 2005 to December 2016, 359 evaluable patients were recruited: orbit, 164 (45.7%); head and neck nonparameningeal, 77 (21.4%); and genitourinary non-bladder/prostate, 118 (32.9%). EFS and OS were 77.4% (95% confidence interval [CI], 72.5-81.6) and 93.5% (95% CI, 90.1-95.8), respectively. Lower dose alkylator chemotherapy and radiotherapy achieved 5-year OS of 93.7% but the difference with higher dose alkylator chemotherapy +/- radiotherapy was not significant (p = 0.8003). Adjuvant radiotherapy improved EFS with 5-year estimates of 84.7% versus 65.2% for nonirradiated (p < .0001), but not OS (p = .9298). Omitting radiotherapy for orbital tumors reduced OS (5-year was 87.1% vs. 97.3% for irradiated, p = .0257). Following R0 resection (n = 60), radiotherapy did not significantly improve EFS or OS. CONCLUSIONS: Radiotherapy for local tumor control allows for reduction of cumulative dose of alkylators in EpSSG standard risk subgroup C RMS patients. The omission of radiotherapy did not affect OS in all patients except those with orbital RMS and was associated with inferior EFS.

Centre for Rare Disorders Division of Paediatric and Adolescent Medicine Oslo University Hospital Oslo Norway

Children's Hospital for Wales Heath Park Cardiff UK

Department of Paediatric Surgery Royal Manchester Children's Hospital Manchester UK

Department of Pediatric and Adolescent Oncology INSERM U1015 Gustave Roussy Université Paris Saclay Villejuif France

Department of Pediatric Oncology and Haematology Hospital Universitari Vall d'Hebron Barcelona Spain

Department of Radiotherapy Hospital Universitari Vall d'Hebron Barcelona Spain

Hematology Oncology Division Department of Women's and Children's Health University of Padova Padova Italy

Hôpital Universitaire des Enfants Reine Fabiola ULB Brussels Belgium

Paediatric Oncology Unit Fondazione IRCCS Istituto Nazionale Tumori Milano Italy

Pathology Unit Department of Laboratories Bambino Gesu Children's Hospital IRCCS Rome Italy

Pediatric Oncology Department National Cancer Institute Rio de Janeiro Brazil

Pediatric Oncology Department University Hospital Brno and Faculty of Medicine Masaryk University Brno Czech Republic

Princess Máxima Center for Pediatric Oncology Utrecht the Netherlands

Radiation Oncology Gustave Roussy Cancer Campus Villejuif France

Ruth Rappaport Children's Hospital Rambam Medical Center Joan and Sanford Weill Pediatric Hematology Oncology and Bone Marrow Transplantation Division Haifa Israel

SIREDO Oncology Center Institut Curie PSL University Paris France

The Royal Marsden Hospital and The Institute of Cancer Research Surrey UK

Citace poskytuje Crossref.org

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$a BACKGROUND: The authors report the prospective evaluation of reduced dose alkylator chemotherapy combined with radiotherapy for European Pediatric Soft Tissue Sarcoma Study Group (EpSSG) standard risk nonalveolar rhabdomyosarcoma (NA-RMS). PATIENTS AND METHODS: Localized node negative Intergroup Rhabdomyosarcoma Study (IRS) II/III NA-RMS at favorable sites (subgroup C), <25 years old, received five cycles of ifosfamide, vincristine, and dactinomycin (IVA) chemotherapy (30 g/m2 ifosfamide) and four cycles of vincristine and dactinomycin (if receiving radiotherapy), or nine cycles of IVA (54 g/m2 ifosfamide) ± radiotherapy. Delayed primary tumor excision was considered for IRS III tumors. The primary end points were event-free survival (EFS) and overall survival (OS). RESULTS: From October 2005 to December 2016, 359 evaluable patients were recruited: orbit, 164 (45.7%); head and neck nonparameningeal, 77 (21.4%); and genitourinary non-bladder/prostate, 118 (32.9%). EFS and OS were 77.4% (95% confidence interval [CI], 72.5-81.6) and 93.5% (95% CI, 90.1-95.8), respectively. Lower dose alkylator chemotherapy and radiotherapy achieved 5-year OS of 93.7% but the difference with higher dose alkylator chemotherapy +/- radiotherapy was not significant (p = 0.8003). Adjuvant radiotherapy improved EFS with 5-year estimates of 84.7% versus 65.2% for nonirradiated (p < .0001), but not OS (p = .9298). Omitting radiotherapy for orbital tumors reduced OS (5-year was 87.1% vs. 97.3% for irradiated, p = .0257). Following R0 resection (n = 60), radiotherapy did not significantly improve EFS or OS. CONCLUSIONS: Radiotherapy for local tumor control allows for reduction of cumulative dose of alkylators in EpSSG standard risk subgroup C RMS patients. The omission of radiotherapy did not affect OS in all patients except those with orbital RMS and was associated with inferior EFS.
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