Systemic treatment of advanced clear cell sarcoma: results from a retrospective international series from the World Sarcoma Network.

BACKGROUND

Clear cell sarcoma (CCS) is a translocated aggressive malignancy with a high incidence of metastases and poor prognosis. There are few studies describing the activity of systemic therapy in CCS. We report a multi-institutional retrospective study of the outcomes of patients with advanced CCS treated with systemic therapy within the World Sarcoma Network (WSN).

MATERIALS AND METHODS

Patients with molecularly confirmed locally advanced or metastatic CCS treated with systemic therapy from June 1985 to May 2021 were included. Baseline demographic and treatment information, including response by Response Evaluation Criteria in Solid Tumours (RECIST) 1.1, was retrospectively collected by local investigators. Descriptive statistics were carried out.

RESULTS

Fifty-five patients from 10 institutions were included. At diagnosis, the median age was 30 (15-73) years and 24% (n = 13/55) had metastatic disease. The median age at diagnosis was 30 (15-73) years. Most primary tumours were at aponeurosis (n = 9/55, 16%) or non-aponeurosis limb sites (n = 17/55, 31%). The most common fusion was EWSR1-ATF1 (n = 24/55, 44%). The median number of systemic therapies was 1 (range 1-7). The best response rate was seen for patients treated with sunitinib (30%, n = 3/10), with a median progression-free survival of 4 [95% confidence interval (CI) 1-7] months. The median overall survival for patients with advanced/metastatic disease was 15 months (95% CI 3-27 months).

CONCLUSIONS

Soft tissue sarcoma-type systemic therapies have limited benefit in advanced CCS and response rate was poor. International, multicentre prospective translational studies are required to identify new treatments for this ultra-rare subtype, and access to early clinical trial enrolment remains key for patients with CCS.

Informations de copyright

Copyright © 2022 The Author(s). Published by Elsevier Ltd.. All rights reserved.

Déclaration de conflit d'intérêts

Disclosure AS has received consulting fees from Merck and Medison. RLJ is the recipient of grants/research support from MSD and GSK and is the recipient of consultation fees from Adaptimmune, Athenex, Blueprint, Clinigen, Eisai, Epizyme, Daichii, Deciphera, Immunedesign, Lilly, Merck, Pharmamar, and UptoDate. PR has received honoraria for lectures and advisory boards from BMS, MSD, Novartis, Pierre Fabre, Sanofi, Merck, Philogen, and Blueprint Medicines outside of the scope of the trial. AMC has received honoraria as invited speaker from BMS, MSD, Novartis, and Pierre Fabre. FD has received honoraria from PharmaMar, GSK, and Bayer, and travel grants from PharmaMar. GGB has received honoraria from Eli Lilly, Eisai, PharmaMar, GSK, and MSD; travel grants from PharmaMar, Pfizer, and Eli Lilly; and personal fees from AboutEvents, EditaMed, and Eli Lilly. All other authors have declared no conflicts of interest.