TY - JOUR
T1 - Local therapy for rhabdomyosarcoma of the bladder and/or prostate without nodal or metastatic spread during the European paediatric Soft tissue sarcoma Study Group (EpSSG) RMS2005 study
AU - Smeulders, Naima
AU - Guerin, Florent
AU - Gaze, Mark N.
AU - Rogers, Timothy
AU - Terwisscha van Scheltinga, Sheila
AU - De Corti, Federica
AU - Chisholm, Julia
AU - Slater, Olga
AU - Minard-Colin, Veronique
AU - Coppadoro, Beatrice
AU - Zanetti, Ilaria
AU - Craigie, Ross
AU - Guillen Burrieza, Gabriela
AU - Dall'Igna, Patrizia
AU - Davila Fajardo, Raquel
AU - Lim, Pei S.
AU - Chargari, Cyrus
AU - Espenel, Sophie
AU - Luis Huertas, Ana L.
AU - Cho, Alexander
AU - Nguyen, Trung
AU - Rees, Helen
AU - Bisogno, Gianni
AU - Merks, Hans
AU - Martelli, Helene
N1 - Publisher Copyright:
© 2025
PY - 2025/12
Y1 - 2025/12
N2 - Background: During the EpSSG RMS2005 trial, organ-sparing surgery (OSS) with brachytherapy (BT) became the local therapy (LT) of choice for selected patients with bladder-prostate rhabdomyosarcoma (BP-RMS). We compare this LT technique with surgical resection and/or external-beam radiotherapy. Methods: Patients with BP-RMS without nodal or metastatic spread enrolled in RMS2005 were categorized by their LT, differentiating OSS from organ-depleting surgery (ODS) and BT from external-beam radiotherapy (EBRT). Progressive disease, relapse or death were considered events for progression-free survival (PFS) and all deaths for overall survival (OS). Results: The cohort comprised 176 patients, aged 10days-21.8years (median 2.5years). Median follow-up was 6.5years (22months-12.5years): 5year-PFS was 80.3 % (95 %CI:73.6–85.5 %); 5year-OS was 90.7 % (95 %CI:85.3–94.2 %). Patients selected for surgery alone or BT with/without OSS (BT+/-OSS) differed significantly in age, tumour size and location from those offered EBRT alone or any other surgery and radiotherapy. Nevertheless, 5year-PFS was similar for the LT groups. However, 5year-OS differed significantly, being highest in patients suitable for surgery alone (100 %; by ODS in 55 %) or BT+ /-OSS (98.1 %; 95 %CI:87.4–99.7 %). Patients with local tumour progression/relapse after EBRT failed salvage: 5year-OS was 81.8 % (95 %CI:58.5–92.8 %) for EBRT alone and 85.3 % (95 %CI:71.6–92.7 %) for surgery and radiotherapy. Postponing LT until after chemotherapy cycle 7 did not significantly impact 5year-PFS or OS. Conclusions: The risk of events was similar for different LT modalities; poor salvage after EBRT significantly reduced 5year-OS. Although not feasible for all, BT+ /-OSS offers an excellent prospect of cure, the best chance of organ retention while avoiding EBRT, and may be delayed for chemotherapy responsive tumours.
AB - Background: During the EpSSG RMS2005 trial, organ-sparing surgery (OSS) with brachytherapy (BT) became the local therapy (LT) of choice for selected patients with bladder-prostate rhabdomyosarcoma (BP-RMS). We compare this LT technique with surgical resection and/or external-beam radiotherapy. Methods: Patients with BP-RMS without nodal or metastatic spread enrolled in RMS2005 were categorized by their LT, differentiating OSS from organ-depleting surgery (ODS) and BT from external-beam radiotherapy (EBRT). Progressive disease, relapse or death were considered events for progression-free survival (PFS) and all deaths for overall survival (OS). Results: The cohort comprised 176 patients, aged 10days-21.8years (median 2.5years). Median follow-up was 6.5years (22months-12.5years): 5year-PFS was 80.3 % (95 %CI:73.6–85.5 %); 5year-OS was 90.7 % (95 %CI:85.3–94.2 %). Patients selected for surgery alone or BT with/without OSS (BT+/-OSS) differed significantly in age, tumour size and location from those offered EBRT alone or any other surgery and radiotherapy. Nevertheless, 5year-PFS was similar for the LT groups. However, 5year-OS differed significantly, being highest in patients suitable for surgery alone (100 %; by ODS in 55 %) or BT+ /-OSS (98.1 %; 95 %CI:87.4–99.7 %). Patients with local tumour progression/relapse after EBRT failed salvage: 5year-OS was 81.8 % (95 %CI:58.5–92.8 %) for EBRT alone and 85.3 % (95 %CI:71.6–92.7 %) for surgery and radiotherapy. Postponing LT until after chemotherapy cycle 7 did not significantly impact 5year-PFS or OS. Conclusions: The risk of events was similar for different LT modalities; poor salvage after EBRT significantly reduced 5year-OS. Although not feasible for all, BT+ /-OSS offers an excellent prospect of cure, the best chance of organ retention while avoiding EBRT, and may be delayed for chemotherapy responsive tumours.
KW - Bladder
KW - Brachytherapy
KW - Event-free survival
KW - External beam radiotherapy
KW - Local therapy
KW - Organ-depleting surgery
KW - Organ-sparing surgery
KW - Overall survival
KW - Prostate
KW - Rhabdomyosarcoma
UR - https://www.scopus.com/pages/publications/105011484313
U2 - 10.1016/j.ejcped.2025.100313
DO - 10.1016/j.ejcped.2025.100313
M3 - Article
AN - SCOPUS:105011484313
SN - 2772-610X
VL - 6
JO - EJC Paediatric Oncology
JF - EJC Paediatric Oncology
M1 - 100313
ER -