Naima Smeulders , Florent Guerin , Mark N. Gaze , Timothy Rogers , Sheila Terwisscha van Scheltinga , Federica De Corti , Julia Chisholm , Olga Slater , Veronique Minard-Colin , Beatrice Coppadoro , Ilaria Zanetti , Ross Craigie , Gabriela Guillen Burrieza , Patrizia Dall'Igna , Raquel Davila Fajardo , Pei S. Lim , Cyrus Chargari , Sophie Espenel , Ana L. Luis Huertas , Alexander Cho , Helene Martelli
{"title":"在欧洲儿科软组织肉瘤研究组（EpSSG） RMS2005研究中，膀胱和/或前列腺横纹肌肉瘤的局部治疗无淋巴结或转移性扩散","authors":"Naima Smeulders , Florent Guerin , Mark N. Gaze , Timothy Rogers , Sheila Terwisscha van Scheltinga , Federica De Corti , Julia Chisholm , Olga Slater , Veronique Minard-Colin , Beatrice Coppadoro , Ilaria Zanetti , Ross Craigie , Gabriela Guillen Burrieza , Patrizia Dall'Igna , Raquel Davila Fajardo , Pei S. Lim , Cyrus Chargari , Sophie Espenel , Ana L. Luis Huertas , Alexander Cho , Helene Martelli","doi":"10.1016/j.ejcped.2025.100313","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Methods</h3><div>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).</div></div><div><h3>Results</h3><div>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 %).</div><div>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.</div><div>Postponing LT until after chemotherapy cycle 7 did not significantly impact 5year-PFS or OS.</div></div><div><h3>Conclusions</h3><div>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.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100313"},"PeriodicalIF":0.0000,"publicationDate":"2025-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"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\",\"authors\":\"Naima Smeulders , Florent Guerin , Mark N. Gaze , Timothy Rogers , Sheila Terwisscha van Scheltinga , Federica De Corti , Julia Chisholm , Olga Slater , Veronique Minard-Colin , Beatrice Coppadoro , Ilaria Zanetti , Ross Craigie , Gabriela Guillen Burrieza , Patrizia Dall'Igna , Raquel Davila Fajardo , Pei S. Lim , Cyrus Chargari , Sophie Espenel , Ana L. Luis Huertas , Alexander Cho , Helene Martelli\",\"doi\":\"10.1016/j.ejcped.2025.100313\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Background</h3><div>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.</div></div><div><h3>Methods</h3><div>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).</div></div><div><h3>Results</h3><div>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 %).</div><div>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.</div><div>Postponing LT until after chemotherapy cycle 7 did not significantly impact 5year-PFS or OS.</div></div><div><h3>Conclusions</h3><div>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.</div></div>\",\"PeriodicalId\":94314,\"journal\":{\"name\":\"EJC paediatric oncology\",\"volume\":\"6 \",\"pages\":\"Article 100313\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-07-24\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"EJC paediatric oncology\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2772610X25001023\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"EJC paediatric oncology","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2772610X25001023","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
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
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.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
