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Multicenter Study
. 2021 Apr 15;127(8):1311-1317.
doi: 10.1002/cncr.33365. Epub 2020 Dec 9.

Real-world outcomes of patients with locally advanced or metastatic epithelioid sarcoma

Mrinal M Gounder  1 , Priscilla Merriam  2 , Ravin Ratan  3 , Shreyaskumar R Patel  3 , Rashmi Chugh  4 , Victor M Villalobos  5   6 , Mark Thornton  7 , Brian A Van Tine  8 , Amr H Abdelhamid  2 , Jennifer Whalen  9 , Jay Yang  9 , Anand Rajarethinam  9 , Mei Sheng Duh  10 , Priyanka J Bobbili  10 , Lynn Huynh  10 , Todor I Totev  10 , Angela K Lax  10 , Shefali Agarwal  9 , George D Demetri  2   11
Affiliations
Multicenter Study

Real-world outcomes of patients with locally advanced or metastatic epithelioid sarcoma

Mrinal M Gounder et al. Cancer. .

Abstract

Background: Limited data are available on the real-world effectiveness and safety of systemic therapies for advanced (surgically unresectable and/or metastatic) epithelioid sarcoma (ES).

Methods: A retrospective medical records review was conducted in patients with advanced ES who were initiating first-line or ≥2 lines of systemic therapy (2000-2017) at 5 US cancer centers. The real-world overall response rate (rwORR), the duration of response (rwDOR), the disease control rate (rwDCR) (defined as stable disease for ≥32 weeks or any duration of response), and progression-free survival (rwPFS) were assessed by radiology reports. Overall survival (OS), rwDOR, and rwPFS were estimated from the time therapy was initiated using the Kaplan-Meier method. Serious adverse events were assessed.

Results: Of 74 patients (median age at diagnosis, 33 years; range, 10.6-76.3 years), 72% were male, and 85% had metastatic disease. The median number of lines of therapy was 2 (range, 1-7 lines of therapy), and 46 patients (62%) received ≥2 lines of systemic therapy. First-line regimens were usually anthracycline-based (54%) or gemcitabine-based (24%). For patients receiving first-line systemic therapy, the rwORR was 15%, the rwDCR was 20%, the median rwDOR was 3.3 months (95% CI, 2.1-5.2 months), the median rwPFS was 2.5 months (95% CI, 1.7, 6.9 months), and the median OS was 15.2 months (95% CI, 11.4-21.7 months). For those who received ≥2 lines of systemic therapy, the rwORR was 9%, the rwDCR was 20%, the median rwDOR was 4.5 months (95% CI, 0.7-5.6 months), and the median rwPFS was 6.0 months (95% CI, 3.2-7.4 months). Over one-half of patients (51.4%) experienced an adverse event, most frequently febrile neutropenia (14%), pain (10%), anemia, dyspnea, fever, thrombocytopenia, or transaminitis (5% each).

Conclusions: Systemic therapies demonstrate limited efficacy in patients with advanced ES and have associated toxicities.

Keywords: chemotherapy; epithelioid; natural history; personal medical records; review of reported cases; sarcoma; treatment efficacy.

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Conflict of interest statement

Mrinal M. Gounder is an employee of the Memorial Sloan Kettering Cancer Center, which received research support from Epizyme, Inc, for this study; he reports grants and personal fees from Epizyme, Inc, during the conduct of the study; and personal fees from Bayer, Springworks Therapeutics, Daiichi Sankyo, Karyopharm, Amgen, TRACON, Flatiron, Medscape, Physicians Education Resource, Guidepoint, GLG, and UpToDate, outside the submitted work; he was supported in part by a National Institutes of Health/National Cancer Institute Cancer Center Support Grant (P30CA008748). Priscilla Merriam, Amr H. Abdelhamid, and George D. Demetri are employees of the Dana‐Farber Cancer Institute and Harvard Medical School, which received research support from Epizyme, Inc, for this study. Amr H. Abdelhamid received a partial scholarship from Dubai Harvard Foundation for Medical Research to support his master's degree at Harvard Medical School. George D. Demetri was supported in part by the Ludwig Center at Harvard, the Dr. Miriam and Sheldon Adelson Medical Research Foundation, and Paul's Posse of the Pan‐Mass Challenge for Dana‐Farber. Ravin Ratan and Shreyaskumar R. Patel are employees of The University of Texas MD Anderson Cancer Center, which received research support from Epizyme, Inc, for this study. Rashmi Chugh is an employee of The University of Michigan Comprehensive Cancer Center, which received research support from Epizyme, Inc, for this study. At the time of study conduct, Victor M. Villalobos was an employee of the University of Colorado Cancer Center, which received research support from Epizyme, Inc, for this study. Mark Thornton is an employee of the Sarcoma Foundation of America, which received research support from Epizyme, Inc, for this study. Brian A. Van Tine is an employee of the Washington University School of Medicine, which received research funding from Epizyme, Inc, for this study. Jay Yang, ,Shefali S. Agarwal, Jennifer Whalen, and Anand Rajarethinam are employees of Epizyme, Inc, and own stock and stock options in the company. Mei Sheng Duh, Priyanka J. Bobbili, Lynn Huynh, Todor I. Totev, and Angela K. Lax are employees of Analysis Group, Inc, which received research funding and consulting fees for this study.

Figures

Figure 1
Figure 1
This Kaplan‐Meier curve illustrates overall survival according to first‐line therapy in patients with locally advanced or metastatic epithelioid sarcoma
See this image and copyright information in PMC

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