TY - JOUR
T1 - Real-world outcomes of patients with locally advanced or metastatic epithelioid sarcoma
AU - Gounder, Mrinal M.
AU - Merriam, Priscilla
AU - Ratan, Ravin
AU - Patel, Shreyaskumar R.
AU - Chugh, Rashmi
AU - Villalobos, Victor M.
AU - Thornton, Mark
AU - Van Tine, Brian A.
AU - Abdelhamid, Amr H.
AU - Whalen, Jennifer
AU - Yang, Jay
AU - Rajarethinam, Anand
AU - Duh, Mei Sheng
AU - Bobbili, Priyanka J.
AU - Huynh, Lynn
AU - Totev, Todor I.
AU - Lax, Angela K.
AU - Agarwal, Shefali
AU - Demetri, George D.
N1 - Funding Information:
This work was supported by Epizyme, Inc.
Funding Information:
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.
Publisher Copyright:
© 2020 Epizyme, Inc. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society
PY - 2021/4/15
Y1 - 2021/4/15
N2 - 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.
AB - 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.
KW - chemotherapy
KW - epithelioid
KW - natural history
KW - personal medical records
KW - review of reported cases
KW - sarcoma
KW - treatment efficacy
UR - http://www.scopus.com/inward/record.url?scp=85097304603&partnerID=8YFLogxK
U2 - 10.1002/cncr.33365
DO - 10.1002/cncr.33365
M3 - Article
C2 - 33296083
AN - SCOPUS:85097304603
SN - 0008-543X
VL - 127
SP - 1311
EP - 1317
JO - Cancer
JF - Cancer
IS - 8
ER -