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Comprehensive Genomic and Transcriptomic Analysis for Guiding Therapeutic Decisions in Patients with Rare Cancers.
59 auth. P. Horák, C. Heining, S. Kreutzfeldt, B. Hutter, A. Mock, Jennifer Hullein, Martina Frohlich, S. Uhrig, A. Jahn, A. Rump, L. Gieldon, L. Mohrmann, D. Hanf, V. Teleanu, C. Heilig, ... D. Lipka, Michael Allgauer, L. Ruhnke, A. Lassmann, V. Endris, O. Neumann, R. Penzel, K. Beck, D. Richter, U. Winter, S. Wolf, Katrin Pfutze, C. Georg, B. Meissburger, I. Buchhalter, M. Augustin, W. Aulitzky, P. Hohenberger, M. Kroiss, P. Schirmacher, R. Schlenk, U. Keilholz, F. Klauschen, G. Folprecht, S. Bauer, J. Siveke, C. Brandts, T. Kindler, M. Boerries, A. Illert, N. von Bubnoff, P. Jost, K. Spiekermann, M. Bitzer, K. Schulze-Osthoff, C. von Kalle, B. Klink, B. Brors, A. Stenzinger, E. Schrock, Daniel Hubschmann, W. Weichert, H. Glimm, Stefan Frohling
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 Germ…
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. Based on 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 to 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.
Published in Cancer Discovery
17
7 2021