% IMPORTANT: The following is UTF-8 encoded. This means that in the presence % of non-ASCII characters, it will not work with BibTeX 0.99 or older. % Instead, you should use an up-to-date BibTeX implementation like “bibtex8” or % “biber”. @ARTICLE{Budczies:120516, author = {J. Budczies$^*$ and G. Mechtersheimer and C. Denkert$^*$ and F. Klauschen and S. S. Mughal and P. Chudasama$^*$ and M. Bockmayr and K. Jöhrens and V. Endris and A. Lier and F. Lasitschka and R. Penzel and M. Dietel$^*$ and B. Brors$^*$ and S. Gröschel$^*$ and H. Glimm$^*$ and P. Schirmacher$^*$ and M. Renner and S. Fröhling$^*$ and A. Stenzinger$^*$}, title = {{PD}-{L}1 ({CD}274) copy number gain, expression, and immune cell infiltration as candidate predictors for response to immune checkpoint inhibitors in soft-tissue sarcoma.}, journal = {OncoImmunology}, volume = {6}, number = {3}, issn = {2162-402X}, address = {Austin, Tex.}, publisher = {Landes Bioscience}, reportid = {DKFZ-2017-00945}, pages = {e1279777 -}, year = {2017}, abstract = {Soft-tissue sarcomas (STS) are rare malignancies that account for $1\%$ of adult cancers and comprise more than 50 entities. Current therapeutic options for advanced-stage STS are limited. Immune checkpoint inhibitors targeting the PD-1/PD-L1 signaling axis are being explored as new treatment modality in STS; however, the determinants of response to these agents are largely unknown. Using the sarcoma data set of The Cancer Genome Altas (TCGA) and an independent cohort of untreated high-grade STS, we analyzed DNA copy number status and mRNA expression of PD-L1 in a total of 335 STS cases. Copy number gains (CNG) were detected in 54 TCGA cases $(21.1\%),$ of which 21 $(8.2\%)$ harbored focal PD-L1 CNG and that were most prevalent in myxofibrosarcoma $(35\%)$ and undifferentiated pleomorphic sarcoma $(34\%).$ In the untreated high-grade STS cohort, we detected CNG in six cases $(7.6\%).$ Analysis of co-amplified genes identified a 5.6-Mb core region comprising 27 genes, including JAK2. Patients with PD-L1 CNG had higher PD-L1 expression compared with STS without CNG (fold change, 1.8; p = 0.02), an effect that was most pronounced in the setting of focal PD-L1 CNG (fold change, 3.0; p = 0.0027). STS with PD-L1 CNG showed a significantly higher mutational load compared with tumors with a diploid PD-L1 locus (median number of mutated genes; 58 vs. 40; p = 3.6E-06), and PD-L1 CNG were associated with inferior survival (HR = 1.82; p = 0.025). In contrast, T-cell infiltrates quantified by mRNA expression of CD3Z were associated with improved survival (HR = 0.88; p = 0.024) and consequently influenced the prognostic power of PD-L1 CNG, with low CD3Z levels conferring poor survival in cases with PD-L1 CNG (HR = 1.8; p = 0.049). These data demonstrate that PD-L1 GNG and elevated expression of PD-L1 occur in a substantial proportion of STS, have prognostic impact that is modulated by T-cell infiltrates, and thus warrant investigation as response predictors for immune checkpoint inhibition.}, cin = {G100 / G200 / G240 / L201 / L101}, ddc = {610}, cid = {I:(DE-He78)G100-20160331 / I:(DE-He78)G200-20160331 / I:(DE-He78)G240-20160331 / I:(DE-He78)L201-20160331 / I:(DE-He78)L101-20160331}, pnm = {317 - Translational cancer research (POF3-317)}, pid = {G:(DE-HGF)POF3-317}, typ = {PUB:(DE-HGF)16}, pubmed = {pmid:28405504}, pmc = {pmc:PMC5384369}, doi = {10.1080/2162402X.2017.1279777}, url = {https://inrepo02.dkfz.de/record/120516}, }