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000130973 1001_ $$0P:(DE-He78)fae1bf941c5fd76cf5356ecfa1243cc4$$aWorst, Barbara$$b0$$eFirst author$$udkfz
000130973 245__ $$aNext-generation personalised medicine for high-risk paediatric cancer patients - The INFORM pilot study.
000130973 260__ $$aAmsterdam [u.a.]$$bElsevier$$c2016
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000130973 520__ $$aThe Individualized Therapy for Relapsed Malignancies in Childhood (INFORM) precision medicine study is a nationwide German program for children with high-risk relapsed/refractory malignancies, which aims to identify therapeutic targets on an individualised basis. In a pilot phase, reported here, we developed the logistical and analytical pipelines necessary for rapid and comprehensive molecular profiling in a clinical setting. Fifty-seven patients from 20 centers were prospectively recruited. Malignancies investigated included sarcomas (n = 25), brain tumours (n = 23), and others (n = 9). Whole-exome, low-coverage whole-genome, and RNA sequencing were complemented with methylation and expression microarray analyses. Alterations were assessed for potential targetability according to a customised prioritisation algorithm and subsequently discussed in an interdisciplinary molecular tumour board. Next-generation sequencing data were generated for 52 patients, with the full analysis possible in 46 of 52. Turnaround time from sample receipt until first report averaged 28 d. Twenty-six patients (50%) harbored a potentially druggable alteration with a prioritisation score of intermediate or higher (level 4 of 7). Common targets included receptor tyrosine kinases, phosphoinositide 3-kinase-mammalian target of rapamycin pathway, mitogen-activated protein kinase pathway, and cell cycle control. Ten patients received a targeted therapy based on these findings, with responses observed in some previously treatment-refractory tumours. Comparative primary relapse analysis revealed substantial tumour evolution as well as one case of unsuspected secondary malignancy, highlighting the importance of re-biopsy at relapse. This study demonstrates the feasibility of comprehensive, real-time molecular profiling for high-risk paediatric cancer patients. This extended proof-of-concept, with examples of treatment consequences, expands upon previous personalised oncology endeavors, and presents a model with considerable interest and practical relevance in the burgeoning era of personalised medicine.
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000130973 7001_ $$aWitt, Ruth$$b4
000130973 7001_ $$aFreitag, Angelika$$b5
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000130973 7001_ $$aTaylor, Lenka$$b15
000130973 7001_ $$aBorst, Tobias$$b16
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000130973 7001_ $$aBartram, Claus R$$b18
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000130973 7001_ $$aKulozik, Andreas E$$b21
000130973 7001_ $$avon Stackelberg, Arend$$b22
000130973 7001_ $$aMeisel, Roland$$b23
000130973 7001_ $$aBorkhardt, Arndt$$b24
000130973 7001_ $$aReinhardt, Dirk$$b25
000130973 7001_ $$aKlusmann, Jan-Henning$$b26
000130973 7001_ $$aFleischhack, Gudrun$$b27
000130973 7001_ $$aTippelt, Stephan$$b28
000130973 7001_ $$aDirksen, Uta$$b29
000130973 7001_ $$aJürgens, Heribert$$b30
000130973 7001_ $$aKramm, Christof M$$b31
000130973 7001_ $$avon Bueren, Andre O$$b32
000130973 7001_ $$0P:(DE-He78)91f32735ee876c579d63c05a7f4778dd$$aWestermann, Frank$$b33$$udkfz
000130973 7001_ $$aFischer, Matthias$$b34
000130973 7001_ $$aBurkhardt, Birgit$$b35
000130973 7001_ $$aWößmann, Wilhelm$$b36
000130973 7001_ $$aNathrath, Michaela$$b37
000130973 7001_ $$aBielack, Stefan S$$b38
000130973 7001_ $$aFrühwald, Michael C$$b39
000130973 7001_ $$0P:(DE-HGF)0$$aFulda, Simone$$b40
000130973 7001_ $$aKlingebiel, Thomas$$b41
000130973 7001_ $$aKoscielniak, Ewa$$b42
000130973 7001_ $$0P:(DE-He78)0321c153233e619c095d93d9d5546c9d$$aSchwab, Matthias$$b43$$udkfz
000130973 7001_ $$aTremmel, Roman$$b44
000130973 7001_ $$aDriever, Pablo Hernáiz$$b45
000130973 7001_ $$aSchulte, Johannes H$$b46
000130973 7001_ $$0P:(DE-He78)fc949170377b58098e46141d95c72661$$aBrors, Benedikt$$b47$$udkfz
000130973 7001_ $$0P:(DE-He78)a8a10626a848d31e70cfd96a133cc144$$avon Deimling, Andreas$$b48$$udkfz
000130973 7001_ $$0P:(DE-He78)e13b4363c5fe858044ef8a39c02c870c$$aLichter, Peter$$b49$$udkfz
000130973 7001_ $$aEggert, Angelika$$b50
000130973 7001_ $$0P:(DE-He78)51bf9ae9cb5771b30c483e5597ef606c$$aCapper, David$$b51$$udkfz
000130973 7001_ $$0P:(DE-He78)f746aa965c4e1af518b016de3aaff5d9$$aPfister, Stefan$$b52$$udkfz
000130973 7001_ $$0P:(DE-He78)551bb92841f634070997aa168d818492$$aJones, David$$b53$$udkfz
000130973 7001_ $$0P:(DE-He78)143af26de9d57bf624771616318aaf7c$$aWitt, Olaf$$b54$$eLast author$$udkfz
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