Treatment options in locally advanced/metastatic BS and STS are limited. PEM has shown first signs of promising activity in some histologic subtypes. In this named patient use BS and STS patients who either failed standard therapy or where no standard therapy was established were treated with PEM.
This retrospective analysis includes efficacy/safety data from 18 pts. with advanced/metastatic BS/STS treated with PEM 200mg d1, q21d between May 2016 and April 2017.
10 pts. were female (56%), 8 pts. male (44%). Median age was 45 yrs. (range 18-84 yrs.). Extent of disease at initial diagnosis was localized in 15 pts. (83%) and advanced/metastatic in 3 pts. (17%). The median number of previous lines of systemic treatment before PEM was 3 (range 0-7 lines). In total, 71 cycles of PEM were administered (median 3 cycles per pt., range 1-11 cycles). Immune-related side effects were hypothyroidism in two pts. and uveitis in one pt. PD-L1 assessment on tumor samples is ongoing.Table:
|patient ID||histology||n PEM cycles||status||status PEM|
|7||dedifferentiated LPS||7||0||0 PR|
|11||myxoid LPS||3||0||0 PR|
|13||dedifferentiated LPS||5||0||0 SD|
|15||epitheloid sarcoma||3||0||0 PR|
|18||myxoid LPS||2||0||0 ie|
Abbreviation: OSA = osteosarcoma, EMC = extraskeletal myxoid chondrosarcoma, LPS = liposarcoma, status 0 = alive, * = dead; status PEM 0 = PEM ongoing, 1 = PEM discontinued to PD (progressive disease); NED = no evidence of disease, PR = partial remission, SD = stable disease, ie = response in evaluation.
In this unselected cohort, PEM seems to have some activity in advanced/metastatic BS/STS. However, longer follow up of treated patients and prospective clinical trials of PEM in BS/STS patients will define the value of PEM in this patient cohort. Updated efficacy and toxicity data as well as PD-L1 expression levels will be presented at the meeting.
Clinical trial identification
Legal entity responsible for the study
Thomas Brodowicz, Clinical Division of Oncology, Medical University of Vienna
S. Schur: Personal fees from Eli Lilly (advisory board) outside the submitted work. T. Brodowicz: Personal fees from Roche and PharmaMar for lecture fees and from Amgen, Bayer, Novartis, Eisai and Eli Lilly for lecture fees and advisory boards. All other authors have declared no conflicts of interest.