Abstract 5449
Background
Limited data are available on effectiveness and safety of systemic therapies for treatment of advanced (unresectable and/or metastatic) epithelioid sarcoma (ES). This natural history study collected real-world (rw) data on outcomes of ES patients (pts) receiving at least first-line (1L) or 2 or more lines (2L+) of systemic therapies.
Methods
Retrospective chart review was conducted in pts with advanced ES who initiated systemic therapy between 2000-2017 at 5 US cancer centers. Due to unavailability of RECIST assessment, rw overall response rate (rwORR) was assessed by review of radiology reports. rw disease control rate (rwDCR) was defined as percent of pts with response of any duration or stable disease ≥32 wks. Median rw duration of response (rwDOR), rw progression-free survival (rwPFS) and overall survival (OS) were estimated from start of therapy by Kaplan-Meier method. The index date for time-to-event endpoints was the start of first-line treatment. Adverse events (AEs) resulting in treatment modification, discontinuation, hospitalization, permanent sequelae or death, were abstracted.
Results
Of 74 eligible pts, 53 (71.6%) were male, and 63 (85.1%) had metastatic ES. INI1/BAF47 was not expressed in 90.2% of 41 tumors tested. Mean age at advanced ES diagnosis was 36.4 years. Anthracycline-based (54.1%) and gemcitabine-based (24.3%) regimens were most common in 1L. Median (range) number of lines of therapy received was 2 (1-7). 1L rwORR was 14.9%, rwDCR was 20.3%, rwDOR was 14.5 wks, and median OS was 66.3 wks. The table shows 1L and 2L+ results. Over 50% of pts had an AE; most frequently febrile neutropenia (13.5%), pain (9.5%), anemia, dyspnea, fever, thrombocytopenia and transaminitis (5.4% each).Table:
1687P Real-world outcomes by line of therapy
1L N = 74 | 2L+ N = 46 | |
---|---|---|
rwORR (95% CI), % | 14.9 (7.7-25.0) | 9.4 (4.4-17.1) |
Median rwDOR (95% CI), wks | 14.5 (9.1-22.6) | 19.6 (3.1-24.3) |
rwDCR (95% CI), % | 20.3 (11.8-31.2) | 19.8 (12.4-29.2) |
Median rwPFS (95% CI), wks | 11.0 (7.3-29.9) | 26.0 (13.9-32.0) |
Median OS (95% CI), wks | 66.3 (49.6-94.1) | 43.3 (33.6-78.3) |
Conclusions
Currently available systemic therapies are not specific for advanced ES, have a limited response durability and low tolerability. This is the largest US-based rw study that provides benchmarking treatment efficacy and safety data for development of standard of care therapies for ES.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
Epizyme, Inc.
Funding
Epizyme, Inc.
Disclosure
M.M. Gounder: Advisory / Consultancy, Travel / Accommodation / Expenses: Epizyme; Honoraria (self), Advisory / Consultancy: Karyopharm; Honoraria (self), Advisory / Consultancy: Daiichi; Honoraria (self): TRACON; Honoraria (self): Amgen. P. Merriam: Advisory / Consultancy: Lilly. S.R. Patel: Advisory / Consultancy, Research grant / Funding (self): Janssen; Advisory / Consultancy: PharmaMar; Advisory / Consultancy: MJ Hennessey/Onc Live; Advisory / Consultancy: Novartis; Advisory / Consultancy: Immune D; Research grant / Funding (self): Eisai; Research grant / Funding (self): Blueprint Medicines; Research grant / Funding (self): Bavarian Nordic. R. Chugh: Advisory / Consultancy: Epizyme, Janssen ,EMD Serono; Research grant / Funding (institution): AADi (Institution), Novartis (Institution), Lilly (Institution), Medivation (Institution), Advenchen (Institution), Epizyme (Institution), Plexiconn (Institution), Pfizer (Institution); Research grant / Funding (self): Portoia Pharmaceuticals. M. Thorton: Research grant / Funding (self): Epizyme; Advisory / Consultancy: Biologics Consulting Group; Shareholder / Stockholder / Stock options: Ziopharm, Novavax, Zogenix. B.A. Van Tine: Advisory / Consultancy, Research grant / Funding (institution): Epizyme. A.H. Abdelhamid: Research grant / Funding (institution): Dubai Harvard Foundation. P. Joshi: Full / Part-time employment: Epizyme. J. Whalen: Full / Part-time employment: Epizyme. J. Yang: Shareholder / Stockholder / Stock options, Full / Part-time employment: Epizyme. A. Rajarethinam: Shareholder / Stockholder / Stock options, Full / Part-time employment: Epizyme. M.S. Duh: Research grant / Funding (self): Epizyme, Novartis, GlaxoSmithKline, Janssen, Pfizer, Bayer, Taiho Pharmaceutical; Speaker Bureau / Expert testimony: AbbVie. P.J. Bobbili: Research grant / Funding (self): Epizyme, Novartis, AstraZeneca, GlaxoSmithKline. C.L. Cavanaugh: Research grant / Funding (institution): Epizyme, GlaxoSmithKline, NovoNordisk, Merck, Eisai, AstraZeneca. L. Huynh: Research grant / Funding (institution): Epizyme, Novartis, Pfizer, Taiho Pharmaceutical; Travel / Accommodation / Expenses: Novartis. T. Totev: Research grant / Funding (institution): Epizyme, Novartis, Takeda (formerly Shire). G. Demetri: Shareholder / Stockholder / Stock options: Blueprint Medicines, Merrimack G1 Therapeutics; Caris Life Sciences; Chapions Oncology; Besson Phramceuticals; Advisory / Consultancy: Novartis, Pfizer, EMD Serono, Jannsen, Ignyta, Loxo, Mirati Therapeutics, Epizyme, PharmaMar, Daiichi Sankyo, WIRB-copernicus group, Ziopharm Oncology, Polaris, Bluprint Medicines, Merrimack, G1 Therapeutics, Caris Life Sciences, Champions Oncology, Besso; Research grant / Funding (institution): Bayer, Novartis, Pfizer, Jannsen, Ignyta, Loxo, AbbVie, Epizyme, AdaptImmune.; Licensing / Royalties: Novartis. All other authors have declared no conflicts of interest.
Resources from the same session
1728 - A phase III trial evaluating olanzapine 5 mg for the prevention of chemotherapy-induced nausea and vomiting in patients receiving cisplatin: J-FORCE Study
Presenter: Hironobu Hashimoto
Session: Poster Display session 1
Resources:
Abstract
920 - Efficacy of intravenous (IV) NEPA, a fixed NK1/5-HT3 receptor antagonist (RA) combination, for prevention of CINV following cisplatin- and anthracycline cyclophosphamide (AC)-based chemotherapy (CT)
Presenter: Lee Schwartzberg
Session: Poster Display session 1
Resources:
Abstract
5146 - Efficacy of olanzapine combination in prevention of nausea & vomiting in highly emetogenic chemotherapy
Presenter: Smitha Saldanha
Session: Poster Display session 1
Resources:
Abstract
1947 - Patient-reported outcome data during real-world use of NEPA for prevention of chemotherapy-induced nausea and vomiting in high-risk platin-receiving patients - A prospective multicenter trial
Presenter: Meinolf Karthaus
Session: Poster Display session 1
Resources:
Abstract
6163 - A study evaluating steroid induced metabolic syndrome after antiemetic dexamethasone therapy in patients received high emetic risk chemotherapy
Presenter: Hee Jun Kim
Session: Poster Display session 1
Resources:
Abstract
2154 - High incidence of nausea during initial and repeated courses if intravenous chemotherapy in patients receiving guideline consistent antiemetic prophylaxis - a prospective, observational, real world study.
Presenter: Teresa Smit
Session: Poster Display session 1
Resources:
Abstract
1637 - "Randomised controlled trial of Scalp Cooling (SC) for the prevention of Chemotherapy Induced Alopecia (CIA)”
Presenter: Jyoti Bajpai
Session: Poster Display session 1
Resources:
Abstract
5351 - Performance of the ‘4S rule’ to predict short-term outcomes in cancer outpatients with unsuspected pulmonary embolism.
Presenter: David Pesántez Coronel
Session: Poster Display session 1
Resources:
Abstract
1189 - Prevalence of venous thromboembolism based on intensive screening for patients with advanced solid tumor in prospective observational study
Presenter: Shota Omori
Session: Poster Display session 1
Resources:
Abstract
4340 - Short-term outcomes of cancer patients with pulmonary embolism according to the setting (hospital-acquired vs. outpatient) at diagnosis.
Presenter: Diego Muñoz Guglielmetti
Session: Poster Display session 1
Resources:
Abstract