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@ARTICLE{Horak:169175,
author = {P. Horak$^*$ and C. Heining$^*$ and S. Kreutzfeldt$^*$ and
B. Hutter$^*$ and A. Mock$^*$ and J. Hüllein$^*$ and M. A.
Fröhlich$^*$ and S. Uhrig$^*$ and A. Jahn$^*$ and A.
Rump$^*$ and L. Gieldon and L. Möhrmann$^*$ and D. Hanf$^*$
and M.-V. Teleanu$^*$ and C. Heilig$^*$ and D. Lipka$^*$ and
M. Allgauer and L. Ruhnke$^*$ and A. Lassmann and V. Endris
and O. Neumann and R. Penzel and K. Beck$^*$ and D.
Richter$^*$ and U. Winter$^*$ and S. Wolf$^*$ and K.
Pfütze$^*$ and C. Geörg$^*$ and B. Meissburger$^*$ and I.
Buchhalter$^*$ and M. Augustin and W. E. Aulitzky and P.
Hohenberger and M. Kroiss and P. Schirmacher$^*$ and R.
Schlenk$^*$ and U. Keilholz$^*$ and F. Klauschen$^*$ and G.
Folprecht and S. Bauer$^*$ and J. Siveke$^*$ and C. H.
Brandts$^*$ and T. Kindler$^*$ and M. Börries$^*$ and A. L.
Illert$^*$ and N. von Bubnoff and P. J. Jost and K.
Spiekermann$^*$ and M. Bitzer$^*$ and K. Schulze Osthoff$^*$
and C. von Kalle and B. Klink$^*$ and B. Brors$^*$ and A.
Stenzinger$^*$ and E. Schrock$^*$ and D. Hübschmann$^*$ and
W. Weichert$^*$ and H. Glimm$^*$ and S. Fröhling$^*$},
title = {{C}omprehensive {G}enomic and {T}ranscriptomic {A}nalysis
for {G}uiding {T}herapeutic {D}ecisions in {P}atients with
{R}are {C}ancers.},
journal = {Cancer discovery},
volume = {11},
number = {11},
issn = {2159-8290},
address = {Philadelphia, Pa.},
reportid = {DKFZ-2021-01304},
pages = {2780-2795},
year = {2021},
note = {2021 Nov;11(11):2780-2795 / #EA:B340#LA:B340#},
abstract = {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.},
cin = {B340 / HD01 / DD01 / BE01 / ED01 / FM01 / FR01 / MU01 /
TU01 / W190 / W610 / D210 / B330 / B080},
ddc = {610},
cid = {I:(DE-He78)B340-20160331 / I:(DE-He78)HD01-20160331 /
I:(DE-He78)DD01-20160331 / I:(DE-He78)BE01-20160331 /
I:(DE-He78)ED01-20160331 / I:(DE-He78)FM01-20160331 /
I:(DE-He78)FR01-20160331 / I:(DE-He78)MU01-20160331 /
I:(DE-He78)TU01-20160331 / I:(DE-He78)W190-20160331 /
I:(DE-He78)W610-20160331 / I:(DE-He78)D210-20160331 /
I:(DE-He78)B330-20160331 / I:(DE-He78)B080-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:34112699},
doi = {10.1158/2159-8290.CD-21-0126},
url = {https://inrepo02.dkfz.de/record/169175},
}