Nonmetastatic Rhabdomyosarcoma in Children and Adolescents: Overall Results of the European Pediatric Soft Tissue Sarcoma Study Group RMS2005 Study

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Publikace nespadá pod Pedagogickou fakultu, ale pod Lékařskou fakultu. Oficiální stránka publikace je na webu muni.cz.
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BISOGNO Gianni MINARD-COLIN Veronique ZANETTI Ilaria FERRARI Andrea GALLEGO Soledad FAJARDO Raquel Davila MANDEVILLE Henry KELSEY Anna ALAGGIO Rita ORBACH Daniel TERWISSCHA van Scheltinga Sheila BURRIEZA Gabriela Guillen BEN-ARUSH Myriam GLOSLI Heidi MÚDRY Peter FERMAN Sima DEVALCK Christine DEFACHELLES Anne Sophie MERKS Johannes Hendrikus Maria JENNEY Meriel

Rok publikování 2023
Druh Článek v odborném periodiku
Časopis / Zdroj Journal of clinical oncology
Fakulta / Pracoviště MU

Lékařská fakulta

Citace
www https://ascopubs.org/doi/10.1200/JCO.22.02093
Doi http://dx.doi.org/10.1200/JCO.22.02093
Klíčová slova Nonmetastatic Rhabdomyosarcoma; Children
Popis Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported.The RMS2005 study included two phase III randomized trials for high-risk (HR) and observational trials for low (LR), standard (SR), and very high-risk (VHR) patients who have been partially reported. Herein, we present a comprehensive report of results achieved for the complete unselected nonmetastatic cohort and analyze the evolution of treatment in comparison with previous European protocols. After a median follow-up of 73.1 months, the 5-year event-free survival (EFS) and overall survival (OS) of the 1,733 patients enrolled were 70.7% (95% CI, 68.5 to 72.8) and 80.4% (95% CI, 78.4 to 82.3), respectively. The results by subgroup: LR (80 patients) EFS 93.7% (95% CI, 85.5 to 97.3), OS 96.7% (95% CI, 87.2 to 99.2); SR (652 patients) EFS 77.4% (95% CI, 73.9 to 80.5), OS 90.6% (95% CI, 87.9 to 92.7); HR (851 patients) EFS 67.3% (95% CI, 64.0 to 70.4), OS 76.7% (95% CI, 73.6 to 79.4); and VHR (150 patients) EFS 48.8% (95% CI, 40.4 to 56.7), OS 49.7% (95% CI, 40.8 to 57.9). The RMS2005 study demonstrated that 80% of children with localized rhabdomyosarcoma could be long-term survivors. The study has established the standard of care across the European pediatric Soft tissue sarcoma Study Group countries with the confirmation of a 22-week vincristine/actinomycin D regimen for LR patients, the reduction of the cumulative ifosfamide dose in the SR group, and for HR disease, the omission of doxorubicin and the addition of maintenance chemotherapy.
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