Low-grade fibromyxoid sarcoma and sclerosing epithelioid fibrosarcoma, outcome of advanced disease: retrospective study from the Ultra-Rare Sarcoma Working Group

C Giani; R A Denu; S Ljevar; A Gronchi; A Napolitano; E Rosenbaum; A Salawu; J Bajpai; E A Connolly; A T J Lee; J C Trent; H Koseła-Paterczyk; Z Chia-Chen Li; K Ogura; E Palmerini; G G Baldi; A Brunello; F Campos; C M Cicala; R G Maki; A J Wagner; V Andelkovic; H H Loong; D D Wong; R L Jones; W D Tap; S M Taverna; A J Lazar; E G Demicco; A Hong; J V M G Bovee; A P Dei Tos; C D M Fletcher; D Baumhoer; M Sbaraglia; I M Schaefer; R Miceli; S Stacchiotti

doi:10.1016/j.esmoop.2024.103689

Low-grade fibromyxoid sarcoma and sclerosing epithelioid fibrosarcoma, outcome of advanced disease: retrospective study from the Ultra-Rare Sarcoma Working Group

ESMO Open. 2024 Sep;9(9):103689. doi: 10.1016/j.esmoop.2024.103689. Epub 2024 Sep 11.

Authors

C Giani¹, R A Denu², S Ljevar³, A Gronchi⁴, A Napolitano⁵, E Rosenbaum⁶, A Salawu⁷, J Bajpai⁸, E A Connolly⁹, A T J Lee¹⁰, J C Trent¹¹, H Koseła-Paterczyk¹², Z Chia-Chen Li¹³, K Ogura¹⁴, E Palmerini¹⁵, G G Baldi¹⁶, A Brunello¹⁷, F Campos¹⁸, C M Cicala¹⁹, R G Maki²⁰, A J Wagner²¹, V Andelkovic²², H H Loong²³, D D Wong²⁴, R L Jones⁵, W D Tap⁶, S M Taverna²⁵, A J Lazar²⁶, E G Demicco²⁷, A Hong⁹, J V M G Bovee²⁸, A P Dei Tos²⁹, C D M Fletcher³⁰, D Baumhoer³¹, M Sbaraglia²⁹, I M Schaefer³⁰, R Miceli³, S Stacchiotti³²

Affiliations

¹ Department of Medical Oncology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
² Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, USA.
³ Department of Clinical Epidemiology and Trial Organisation, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
⁴ Department of Sarcoma Surgery, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
⁵ Department of Medical Oncology, The Royal Marsden NHS, London, UK.
⁶ Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, USA.
⁷ Division of Medical Oncology and Hematology, Mount Sinai Hospital and Princess Margaret Cancer Centre, Toronto, Canada.
⁸ Department of Medical Oncology, Tata Memorial Centre, Homibhabha National University, Mumbai, India.
⁹ Central Clinical School, Faculty of Medicine and Health Science, The University of Sydney, Sydney, Australia.
¹⁰ Department of Medical Oncology, The Christie NHS Foundation Trust, Manchester, UK.
¹¹ Department of Medical Oncology, Sylvester Comprehensive Cancer Center, University of Miami, Miami, USA.
¹² Department of Medical Oncology, Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland.
¹³ Department of Medical Oncology, National Taiwan University Cancer Center, Taipei, Taiwan.
¹⁴ Department of Musculoskeletal Oncology, National Cancer Center Hospital, Tokyo, Japan.
¹⁵ Department of Medical Oncology, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.
¹⁶ Department of Medical Oncology, Hospital of Prato, Azienda USL Toscana Centro, Prato, Italy.
¹⁷ Department of Oncology, Medical Oncology 1 Unit, Istituto Oncologico Veneto IOV - IRCCS, Padua, Italy.
¹⁸ Department of Medical Oncology, A.C.Camargo Cancer Center, São Paulo, Brazil.
¹⁹ Department of Medical Oncology, Vall d'Hebron University Hospital, Vall d'Hebron Institute of Oncology (VHIO), Barcelona, Spain.
²⁰ Department of Medical Oncology, University of Pennsylvania Perelman School of Medicine, Philadelphia, USA.
²¹ Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, USA.
²² Department of Medical Oncology, Princess Alexandra Hospital (PAH), Woolloongabba, Australia.
²³ Department of Clinical Oncology, The Chinese University of Hong Kong, Prince of Wales Hospital, Hong Kong SAR, China.
²⁴ Department of Anatomical Pathology, PathWest, Sir Charles Gairdner Hospital, Perth, Australia.
²⁵ Technology Transfer Office, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
²⁶ Department of Pathology and Genomic Medicine, The University of Texas MD Anderson Cancer Center, Houston, USA.
²⁷ Department of Pathology and Laboratory Medicine, Mount Sinai Hospital and Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Canada.
²⁸ Department of Pathology, Leiden University Medical Center (LUMC), Leiden, The Netherlands.
²⁹ Department of Pathology, Azienda Ospedale-Università Padova, Padova, Italy.
³⁰ Department of Pathology, Brigham and Women's Hospital and Harvard Medical School, Boston, USA.
³¹ Department of Pathology, University Hospital Basel, Basel, Switzerland.
³² Department of Medical Oncology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy. Electronic address: [email protected].

Abstract

Background: To present findings from a retrospective study conducted by the Ultra-Rare Sarcoma Working Group on metastatic low-grade fibromyxoid sarcoma (LGFMS), sclerosing epithelioid fibrosarcoma (SEF), and hybrid (H)-LGFMS/SEF across 28 global centres.

Methods: Patients treated at participating institutions from January 2000 to September 2022 were retrospectively selected. Diagnosis was confirmed by expert pathologists. Primary endpoint was progression-free survival (PFS-1) from metastasis detection to first progression or death. PFS-2 was calculated from therapy initiation.

Results: A total of 101 patients were identified (32 LGFMS, 50 SEF, 19 H-LGFMS/SEF). Median (m) follow-up was 62.1 months. mPFS-1 was 28.7, 11.8, and 20.3 months for LGFMS, SEF, and H-LGFMS/SEF, respectively. mOS was 145.8, 41.9, and 113.5 months, respectively. Treatments included anthracycline-based chemotherapy, gemcitabine-based chemotherapy (G), pazopanib, trabectedin, others. mPFS-2 was: 20.1, 5.5, and 3.5 months in H-LGFMS/SEF, SEF, and LGFMS, respectively, with anthracyclines; 19.5, 7.7, and 6.9 months in LGFMS, SEF, and H-LGFMS/SEF, respectively, with pazopanib; 12.0, 9.7, and 3.1 months in H-LGFMS/SEF, LGFMS, and SEF, respectively. Occasional responses occurred with ifosfamide/oral cyclophosphamide, and prolonged stable disease with immune checkpoint inhibitors.

Conclusions: In this series, the largest available, metastatic LGFMS, SEF, and H-LGFMS/SEF showed different courses. Systemic agents have modest efficacy, informing future trials of novel agents for these tumours.

Keywords: advanced disease; low-grade fibromyxoid sarcoma; sclerosing epithelioid fibrosarcoma; systemic therapies; ultra-rare sarcomas.

Publication types

Multicenter Study

MeSH terms

Adolescent
Adult
Aged
Anthracyclines / therapeutic use
Antineoplastic Combined Chemotherapy Protocols / therapeutic use
Deoxycytidine / analogs & derivatives
Deoxycytidine / therapeutic use
Female
Fibrosarcoma* / drug therapy
Gemcitabine
Humans
Indazoles / therapeutic use
Male
Middle Aged
Neoplasm Grading
Pyrimidines / therapeutic use
Retrospective Studies
Soft Tissue Neoplasms / drug therapy
Soft Tissue Neoplasms / pathology
Sulfonamides / therapeutic use
Trabectedin / therapeutic use
Young Adult

Substances

Pyrimidines
pazopanib
Indazoles
Gemcitabine
Trabectedin
Sulfonamides
Anthracyclines
Deoxycytidine

Grants and funding

P30 CA008748/CA/NCI NIH HHS/United States