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

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 year...

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Autores: 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
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2026
País:España
Institución:Institut d’Investigació Biomèdica Sant Pau (IIB Sant Pau)
Repositorio:r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau
OAI Identifier:oai:dnet:r-iibsantpa_::fc612b3e99745e3f0d1695656b60a206
Acceso en línea:https://iibsantpau.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=21482
Access Level:acceso abierto
Palabra clave:Sarcoma
Advanced soft tissue sarcoma
Ultra-rare sarcoma
Rhabdomyosarcoma
Pleomorphic rhabdomyosarcoma
Systemic therapy
Anthracycline
Gemcitabine
Pazopanib
Immunotherapy
Descripción
Sumario: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.

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