Pleomorphic rhabdomyosarcoma, outcomes of patients with advanced disease treated with systemic agents: Retrospective study from the global pushing ultra-rare sarcomas towards hope (PUSH) consortium
Por:
Baldi, GG, Giani, C, Ljevar, S, Denu, RA, Napolitano, A, Han, I, Brunello, A, Bhadri, V, Sebio, A, Andelkovic, V, Campos, F, Scanferla, R, Abreu, MH, Desar, IME, Ogura, K, Wong, DD, Bae, S, Bakhshi, S, Lefler, DS, Marquina, G, Mazzocca, A, Nakazawa, MS, Thway, K, Kim, HS, Chiusole, B, Connolly, EA, Terés, R, Antonescu, C, Bellan, E, Bovee, JVMG, Davis, JL, Tos, APD, Di Blasi, E, Lazar, AJ, Sbaraglia, M, Schaefer, IM, Taverna, S, Gronchi, A, Miceli, R, Chen, TW, Stacchiotti, S
Publicada:
2 may 2026
Resumen:
Objectives: To report the outcomes in adult patients with advanced pleomorphic rhabdomyosarcoma (P-RMS) treated with systemic therapy. Methods: This global, multicenter, retrospective study conducted within the Pushing Ultra-Rare Sarcomas Towards Hope consortium (PUSH) included patients > 40 years with histologically confirmed advanced P-RMS, treated with at least one line of systemic therapy between 2013 and 2023. The primary endpoint was progression-free survival from first diagnosis of advanced disease, and from systemic treatment start (PFS-1 and PFS-2). Secondary endpoints included overall response rate (ORR), overall survival from first diagnosis of advanced disease and from treatment start (OS-1 and OS-2), and treatment-specific outcomes. Results: Seventy-seven patients were included from 21 sarcoma reference centers. At a median follow-up of 44 months (IQR: 17.0-74.8), 49 (64%) patients had died and 48 (62%) had progressed. The median OS-1 and PFS-1 were 13.6 (95% confidence interval (CI): 9.4-22.5) and 5.4 (95% CI: 4.2-7.3) months, respectively. Two-and three-year OS-1 were 32.5% and 30.3%. Anthracycline-based regimens (n = 42) achieved a 50% ORR, with mPFS-2 and mOS-2 of 5.2 and 19.2 months; gemcitabine-based regimens (n = 15) a 42% ORR, with mPFS-2 and mOS-2 of 3.7 and 7.8 months; pazopanib (n = 6) a 33% ORR, with mPFS-2 and mOS-2 of 2.4 and 4.2 months; PD-1 inhibitors (n = 2) induced one response lasting 53 months. Conclusions: This series of advanced P-RMS treated with systemic agents, the largest available to date, showed meaningful activity of anthracycline-and gemcitabine-based regimens, and anecdotal responses to pazopanib and PD-1 inhibitors. Further prospective validation is planned.
Filiaciones:
Baldi, GG:
Azienda USL Toscana Ctr, Hosp Prato, Dept Med Oncol, Prato, Italy
Giani, C:
Fdn IRCCS Ist Nazl Tumori, Dept Med Oncol, Via Giacomo Venezian 1, I-20133 Milan, 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
Napolitano, A:
Royal Marsden NHS Fdn Trust, Dept Med Oncol, London, England
Inst Canc Res, London, England
Han, I:
Seoul Natl Univ Hosp, Dept Orthopaed Surg, Seoul, South Korea
Brunello, A:
IRCCS, Ist Oncol Veneto IOV, Dept Oncol, Med Oncol Unit 1, Padua, Italy
Bhadri, V:
Chris OBrien Lifehouse, Dept Med Oncol, Sydney, Australia
Sebio, A:
Santa Creu & St Pau Hosp, Dept Med Oncol, IR St Pau, Barcelona, Spain
Andelkovic, V:
Princess Alexandra Hosp, Dept Med Oncol, Brisbane, Qld, Australia
Campos, F:
AC Camargo Canc Ctr, Dept Med Oncol, Sao Paulo, Brazil
Scanferla, R:
AOU Careggi, Dept Orthoped Oncol & Reconstruct Surg, Florence, Italy
Abreu, MH:
Portuguese Inst Oncol, Dept Med Oncol, Porto, Portugal
Desar, IME:
Radboud Univ Nijmegen, Med Ctr, Dept Med Oncol, Nijmegen, Netherlands
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, Vic, Australia
Bakhshi, S:
India Inst Med Sci, Dept Med Oncol, New Delhi, India
Lefler, DS:
Univ Penn, Dept Hematol Oncol, Philadelphia, PA USA
Marquina, G:
UCM, EURACAN referral Ctr, Sch Med, Dept Med Oncol,Hosp Clin San Carlos,IdISSC, Madrid, Spain
Mazzocca, A:
Fdn Policlin Univ, Dept Med Oncol, Campus Biomed, Rome, Italy
Nakazawa, MS:
Univ Texas MD Anderson Canc Ctr, Dept Sarcoma Med Oncol, Houston, TX USA
Thway, K:
Royal Marsden NHS Fdn Trust, Dept Med Oncol, London, England
Inst Canc Res, London, England
Kim, HS:
Seoul Natl Univ Hosp, Dept Orthopaed Surg, Seoul, South Korea
Chiusole, B:
IRCCS, Ist Oncol Veneto IOV, Dept Oncol, Med Oncol Unit 1, Padua, Italy
Connolly, EA:
Chris OBrien Lifehouse, Dept Med Oncol, Sydney, Australia
Terés, R:
Santa Creu & St Pau Hosp, Dept Med Oncol, IR St Pau, Barcelona, Spain
Antonescu, C:
Mem Sloan Kettering Canc Ctr, Dept Pathol, New York, NY USA
Bellan, E:
Azienda Osped Univ Padova, Dept Integrated Diagnost, Padua, Italy
Bovee, 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
Gronchi, A:
Fdn IRCCS Ist Nazl Tumori, Dept Sarcoma Surg, Milan, Italy
Miceli, R:
Fdn IRCCS Ist Nazl Tumori, Unit Biostat Clin Res, Milan, Italy
Chen, TW:
Natl Taiwan Univ Hosp, Dept Oncol, Taipei, Taiwan
Stacchiotti, S:
Fdn IRCCS Ist Nazl Tumori, Dept Med Oncol, Via Giacomo Venezian 1, I-20133 Milan, Italy
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