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
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.
Authors
- Andrea Napolitano (ORCID: https://orcid.org/0000-0002-7509-1555)
- Raúl Terés
- Alessandro Mazzocca
- Daniel Wong (ORCID: https://orcid.org/0000-0001-5069-1557)
- Elena Di Blasi (ORCID: https://orcid.org/0009-0005-1861-9340)
- M. Sbaraglia
- Silva Ljevar (ORCID: https://orcid.org/0000-0002-1151-1477)
- Elizabeth A. Connolly (ORCID: https://orcid.org/0000-0002-5579-3728)
- Roberto Scanferla (ORCID: https://orcid.org/0000-0002-4271-8278)
- GLORIA MARQUINA
- Benedetta Chiusole (ORCID: https://orcid.org/0000-0001-6449-6402)
- Koichi Ogura (ORCID: https://orcid.org/0000-0001-6956-3117)
- Claudia Giani (ORCID: https://orcid.org/0000-0001-8630-4037)
- Giacomo Giulio Baldi (ORCID: https://orcid.org/0000-0003-0798-7752)
- Silvia Taverna
- Judith V.M.G. Bovée (ORCID: https://orcid.org/0000-0003-1155-0481)
- Michael S. Nakazawa (ORCID: https://orcid.org/0000-0002-6272-5467)
- Elena Bellan (ORCID: https://orcid.org/0000-0003-3294-6055)
- Silvia Stacchiotti
- Prof Khin Thway
- Miceli Rosalba
- Antonella Brunello
- Ilkyu Han
- Ingrid M.E. Desar
- Miguel H. Abreu
- Jessica L. Davis
- Susie Bae
- Angelo P. Dei Tos
- Daniel S. Lefler
- Fernando Campos
- Alessandro Gronchi
- Alexander J. Lazar
- Inga-Marie Schaefer
- Tom W. Chen
- Vivek Bhadri
- Han-Soo Kim
- Vladimir Andelkovic
- Sameer Bakhshi
- Ryan A. Denu
- Cristina Antonescu
- Ana Sebio
Institutions
- Brigham and Women's Hospital (US)
- Royal Marsden NHS Foundation Trust (GB)
- Memorial Sloan Kettering Cancer Center (US)
- The University of Texas MD Anderson Cancer Center (US)
- Harvard University (US)
- University of Padua (IT)
- Institute of Cancer Research (GB)
- Radboud University Nijmegen (NL)
- Leiden University Medical Center (NL)
- Peter MacCallum Cancer Centre (AU)
- Hospital de Sant Pau (ES)
- Princess Alexandra Hospital (AU)
- Sir Charles Gairdner Hospital (AU)
- Seoul National University Hospital (KR)
- Radboud University Medical Center (NL)
- Hospital Clínico San Carlos (ES)
- AC Camargo Hospital (BR)
- Chris O’Brien Lifehouse (AU)
- Instituto de Investigación Sanitaria del Hospital Clínico San Carlos (ES)
- Instituto Português de Oncologia Francisco Gentil (PT)
- Istituto Oncologico Veneto (IT)
- Azienda Ospedaliero-Universitaria Careggi (IT)
- National Taiwan University Hospital (TW)
- Campus Bio Medico University Hospital (IT)
- Azienda Ospedale - Università Padova (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- Fondazione IRCCS Istituto Nazionale dei Tumori (IT)
- Hospital of Prato (IT)
- Indiana University School of Medicine
- Indiana University – Purdue University Indianapolis (US)
- All India Institute of Medical Sciences (IN)
- University of Pennsylvania (US)
Publication Details
- Journal
- European Journal of Cancer
- Published
- 2026-03-08
- DOI
- https://doi.org/10.1016/j.ejca.2026.116666
- Primary Topic
- Tuberous Sclerosis Complex Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Ministero della Salute