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

J 2023

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

BISOGNO, Gianni, Veronique MINARD-COLIN, Ilaria ZANETTI, Andrea FERRARI, Soledad GALLEGO et. al.

Basic information

Original name

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

Authors

Edition

Journal of clinical oncology, PHILADELPHIA, LIPPINCOTT WILLIAMS & WILKINS, 2023, 0732-183X

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30204 Oncology

Country of publisher

United States of America

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 45.300 in 2022

RIV identification code

RIV/00216224:14110/23:00133412

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1200/JCO.22.02093

UT WoS

001030226300009

Keywords in English

Nonmetastatic Rhabdomyosarcoma; Children

Abstract

V originále

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.

Links

MUNI/A/1395/2022, interní kód MU

Name: Personalizovaná léčba v dětské onkologii: multimodální theranostický přístup a „N-of-1 clinical trials“

Investor: Masaryk University

Citovat

BISOGNO, Gianni, Veronique MINARD-COLIN, Ilaria ZANETTI, Andrea FERRARI, Soledad GALLEGO, Raquel Davila FAJARDO, Henry MANDEVILLE, Anna KELSEY, Rita ALAGGIO, Daniel ORBACH, van Scheltinga Sheila TERWISSCHA, Gabriela Guillen BURRIEZA, Myriam BEN-ARUSH, Heidi GLOSLI, Peter MÚDRY, Sima FERMAN, Christine DEVALCK, Anne Sophie DEFACHELLES, Johannes Hendrikus Maria MERKS and Meriel JENNEY. Nonmetastatic Rhabdomyosarcoma in Children and Adolescents: Overall Results of the European Pediatric Soft Tissue Sarcoma Study Group RMS2005 Study. Journal of clinical oncology. PHILADELPHIA: LIPPINCOTT WILLIAMS & WILKINS, 2023, vol. 41, No 13, p. 2342-2350. ISSN 0732-183X. Available from: https://dx.doi.org/10.1200/JCO.22.02093.

@article{2369017,
   author = {Bisogno, Gianni and MinardandColin, Veronique and Zanetti, Ilaria and Ferrari, Andrea and Gallego, Soledad and Fajardo, Raquel Davila and Mandeville, Henry and Kelsey, Anna and Alaggio, Rita and Orbach, Daniel and Terwisscha, van Scheltinga Sheila and Burrieza, Gabriela Guillen and BenandArush, Myriam and Glosli, Heidi and Múdry, Peter and Ferman, Sima and Devalck, Christine and Defachelles, Anne Sophie and Merks, Johannes Hendrikus Maria and Jenney, Meriel},
   article_location = {PHILADELPHIA},
   article_number = {13},
   doi = {http://dx.doi.org/10.1200/JCO.22.02093},
   keywords = {Nonmetastatic Rhabdomyosarcoma; Children},
   language = {eng},
   issn = {0732-183X},
   journal = {Journal of clinical oncology},
   title = {Nonmetastatic Rhabdomyosarcoma in Children and Adolescents: Overall Results of the European Pediatric Soft Tissue Sarcoma Study Group RMS2005 Study},
   url = {https://ascopubs.org/doi/10.1200/JCO.22.02093},
   volume = {41},
   year = {2023}
}

TY  - JOUR
ID  - 2369017
AU  - 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
PY  - 2023
TI  - Nonmetastatic Rhabdomyosarcoma in Children and Adolescents: Overall Results of the European Pediatric Soft Tissue Sarcoma Study Group RMS2005 Study
JF  - Journal of clinical oncology
VL  - 41
IS  - 13
SP  - 2342-2350
EP  - 2342-2350
PB  - LIPPINCOTT WILLIAMS & WILKINS
SN  - 0732183X
KW  - Nonmetastatic Rhabdomyosarcoma
KW  - Children
UR  - https://ascopubs.org/doi/10.1200/JCO.22.02093
N2  - 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.
ER  -

BISOGNO, Gianni, Veronique MINARD-COLIN, Ilaria ZANETTI, Andrea FERRARI, Soledad GALLEGO, Raquel Davila FAJARDO, Henry MANDEVILLE, Anna KELSEY, Rita ALAGGIO, Daniel ORBACH, van Scheltinga Sheila TERWISSCHA, Gabriela Guillen BURRIEZA, Myriam BEN-ARUSH, Heidi GLOSLI, Peter MÚDRY, Sima FERMAN, Christine DEVALCK, Anne Sophie DEFACHELLES, Johannes Hendrikus Maria MERKS and Meriel JENNEY. Nonmetastatic Rhabdomyosarcoma in Children and Adolescents: Overall Results of the European Pediatric Soft Tissue Sarcoma Study Group RMS2005 Study. \textit{Journal of clinical oncology}. PHILADELPHIA: LIPPINCOTT WILLIAMS \&{} WILKINS, 2023, vol.~41, No~13, p.~2342-2350. ISSN~0732-183X. Available from: https://dx.doi.org/10.1200/JCO.22.02093.

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