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Horak, Peter; Heining, Christoph; Kreutzfeldt, Simon; Hutter, Barbara; Mock, Andreas; Huellein, Jennifer; Froehlich, Martina; Uhrig, Sebastian; Jahn, Arne; Rump, Andreas; Gieldon, Laura; Moehrmann, Lino; Hanf, Dorothea; Teleanu, Veronica; Heilig, Christoph E.; Lipka, Daniel B.; Allgaeuer, Michael; Ruhnke, Leo; Lassmann, Andreas; Endris, Volker; Neumann, Olaf; Penzel, Roland; Beck, Katja; Richter, Daniela; Winter, Ulrike; Wolf, Stephan; Pfuetze, Katrin; Geoerg, Christina; Meissburger, Bettina; Buchhalter, Ivo; Augustin, Marinela; Aulitzky, Walter E.; Hohenberger, Peter; Kroiss, Matthias; Schirmacher, Peter; Schlenk, Richard F.; Keilholz, Ulrich; Klauschen, Frederick; Folprecht, Gunnar; Bauer, Sebastian; Siveke, Jens Thomas; Brandts, Christian H.; Kindler, Thomas; Boerries, Melanie; Illert, Anna L.; Bubnoff, Nikolas von; Jost, Philipp J.; Spiekermann, Karsten; Bitzer, Michael; Schulze-Osthoff, Klaus; Kalle, Christof von; Klink, Barbara; Brors, Benedikt; Stenzinger, Albrecht; Schroeck, Evelin; Huebschmann, Daniel; Weichert, Wilko; Glimm, Hanno and Froehling, Stefan (2021): Comprehensive Genomic and Transcriptomic Analysis for Guiding Therapeutic Decisions in Patients with Rare Cancers. In: Cancer Discovery, Vol. 11, No. 11: pp. 2780-2795

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The clinical relevance of comprehensive molecular analysis in rare cancers is not established. We analyzed the molecular profiles and clinical outcomes of 1,310 patients (rare cancers, 75.5%) enrolled in a prospective observational study by the German Cancer Consortium that applies whole-genome/exome and RNA sequencing to inform the care of adults with incurable cancers. On the basis of 472 single and six composite biomarkers, a cross-institutional molecular tumor board provided evidence-based management recommendations, including diagnostic reevaluation, genetic counseling, and experimental treatment, in 88% of cases. Recommended therapies were administered in 362 of 1,138 patients (31.8%) and resulted in significantly improved overall response and disease control rates (23.9% and 55.3%) compared with previous therapies, translating into a progression-free survival ratio >1.3 in 35.7% of patients. These data demonstrate the benefit of molecular stratification in rare cancers and represent a resource that may promote clinical trial access and drug approvals in this underserved patient population. SIGNIFICANCE: Rare cancers are difficult to treat;in particular, molecular pathogenesis-oriented medical therapies are often lacking. This study shows that whole-genome/exome and RNA sequencing enables molecularly informed treatments that lead to clinical benefit in a substantial proportion of patients with advanced rare cancers and paves the way for future clinical trials.

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