International multicenter retrospective study on pleomorphic rhabdomyosarcoma (P-RMS), a PUSH platform study: outcome of primary localized disease
Por:
Giani, C, Baldi, GG, Ljevar, S, Denu, RA, Andelkovic, V, Han, I, Brunello, A, Napolitano, A, Bhadri, VA, Scanferla, R, Garcia, AS, Abreu, MH, Campos, F, Ogura, K, Wong, DD, Bae, S, Marquina, G, Mazzocca, A, Yang, V, Nakazawa, MS, Kim, HS, Chiusole, B, Thway, K, Antonescu, C, Bellan, E, Bovée, JVMG, Davis, JL, Tos, APD, Di Blasi, E, Lazar, AJ, Sbaraglia, M, Schaefer, IM, Taverna, S, Chen, TWW, Miceli, R, Gronchi, A, Stacchiotti, S
Publicada:
1 feb 2026
Ahead of Print:
1 ene 2026
Resumen:
Background The purpose of this study was to report the outcome of primary localized pleomorphic rhabdomyosarcoma (P-RMS). Patients and methods Patients with primary localized P-RMS, aged >40 years, and surgically treated with curative intent from January 2013 to December 2023 were enrolled from 25 institutions across 12 countries. Pathological inclusion criteria were predefined by expert pathologists per established World Health Organization guidelines. Primary endpoint was overall survival (OS). Secondary endpoints were disease-free survival (DFS), crude cumulative incidence (CCI) of local recurrence (LR), CCI of distant metastases (DM), post-relapse OS, and post-metastasis OS. Results A total of 93 patients were eligible and included in the present study. At a median follow-up of 39.8 months (interquartile range 22.83-85.69 months), 32/93 (34%) patients died and 53/93 (57%) recurred. The corresponding 5-year OS and DFS were 57.7% [95% confidence interval (CI) 46.7% to 71.2%] and 35.8% (95% CI 26.3% to 48.9%), respectively. Younger age, R0 surgical margins, and administration of radiotherapy were positive prognostic factors in multivariable analysis for DFS. LR and DM were developed in 14/93 (15%) and 39/93 (42%) patients, respectively. The corresponding 3-year CCI-LR and DM were 16% (95% CI 8.9% to 25%) and 44% (95% CI 33% to 55%). Post-metastasis median OS was 14.3 months (95% CI 9.4 months-not reached). Conclusions While P-RMS is known to be an aggressive histologic sarcoma subtype, one-third of patients with primary localized disease may be cured. The contribution of chemotherapy and radiotherapy to the cure rate remains unclear. Post-metastatic outcomes mirror those observed in other sarcomas.
Filiaciones:
Giani, C:
Fdn IRCCS Ist Nazl Tumori, Dept Med Oncol, Via Giacomo Venezian 1, I-20133 Milan, Italy
Baldi, GG:
Hosp Prato, Azienda USL Toscana Ctr, Dept Med Oncol, Prato, Italy
Ljevar, S:
Fdn IRCCS Ist Nazl Tumori, Unit Biostat Clin Res, Milan, Italy
Denu, RA:
Univ Texas MD Anderson Canc Ctr, Dept Sarcoma Med Oncol, Houston, TX USA
Andelkovic, V:
Princess Alexandra Hosp, Dept Med Oncol, Brisbane, Australia
Han, I:
Seoul Natl Univ Hosp, Dept Med Oncol, Seoul, South Korea
Brunello, A:
Ist Oncol Veneto IOV IRCCS, Dept Oncol, Med Oncol 1 Unit, Padua, Italy
Napolitano, A:
Royal Marsden NHS & Inst Canc Res, Dept Med Oncol, London, England
Bhadri, VA:
Chris OBrien Lifehouse, Dept Med Oncol, Sydney, Australia
Scanferla, R:
AOU Careggi, Dept Orthoped Oncol & Reconstruct Surg, Florence, Italy
Garcia, AS:
St Pau Hosp, Dept Med Oncol, Barcelona, Spain
Abreu, MH:
Portuguese Inst Oncol, Dept Med Oncol, Porto, Portugal
Campos, F:
A C Camargo Canc Ctr, Dept Med Oncol, Sao Paulo, Brazil
Ogura, K:
Natl Canc Ctr, Dept Musculoskeletal Oncol & Rehabil Med, Tokyo, Japan
Wong, DD:
Sir Charles Gairdner Hosp, Dept Anat Pathol, PathWest, Perth, Australia
Bae, S:
Peter MacCallum Canc Ctr, Dept Med Oncol, Melbourne, Australia
Marquina, G:
UCM, Hosp Clin San Carlos, Dept Med Oncol, Sch Med,IdissC,EURACAN,Referral Ctr, Madrid, Spain
Mazzocca, A:
Fdn Policlin Univ Campus Biomed, Dept Med Oncol, Rome, Italy
Yang, V:
Natl Canc Ctr, Dept Med Oncol, Singapore, Singapore
Nakazawa, MS:
Univ Texas MD Anderson Canc Ctr, Dept Sarcoma Med Oncol, Houston, TX USA
Kim, HS:
Seoul Natl Univ Hosp, Dept Orthopaed Surg, Seoul, South Korea
Chiusole, B:
Ist Oncol Veneto IOV IRCCS, Dept Oncol, Med Oncol 1 Unit, Padua, Italy
Thway, K:
Royal Marsden NHS & Inst Canc Res, Dept Med Oncol, London, England
Antonescu, C:
Mem Sloan Kettering Canc Ctr, Dept Pathol, New York, NY USA
Bellan, E:
Azienda Osped Univ Padova, Dept Integrated Diagnost, Padua, Italy
Bovée, JVMG:
Leiden Univ, Med Ctr, Dept Pathol, Leiden, Netherlands
Davis, JL:
Indiana Univ Sch Med, Dept Pathol, Indianapolis, IN USA
Tos, APD:
Azienda Osped Univ Padova, Dept Integrated Diagnost, Padua, Italy
Di Blasi, E:
Fdn IRCCS Ist Nazl Tumori, Dept Pathol, Milan, Italy
Lazar, AJ:
Univ Texas MD Anderson Canc Ctr, Dept Pathol, Houston, TX USA
Univ Texas MD Anderson Canc Ctr, Dept Genom Med, Houston, TX USA
Sbaraglia, M:
Azienda Osped Univ Padova, Dept Integrated Diagnost, Padua, Italy
Schaefer, IM:
Harvard Med Sch, Brigham & Womens Hosp, Dept Pathol, Boston, MA USA
Taverna, S:
Fdn IRCCS Ist Nazl Tumori, Technol Transfer Off, Milan, Italy
Chen, TWW:
Natl Taiwan Univ Hosp, Dept Oncol, Taipei, Taiwan
Miceli, R:
Fdn IRCCS Ist Nazl Tumori, Unit Biostat Clin Res, Milan, Italy
Gronchi, A:
Fdn IRCCS Ist Nazl Tumori, Dept Sarcoma Surg, Milan, Italy
Stacchiotti, S:
Fdn IRCCS Ist Nazl Tumori, Dept Med Oncol, Via Giacomo Venezian 1, I-20133 Milan, Italy
Green Submitted, gold
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