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000136976 1001_ $$aWeidenbusch, Bushra$$b0
000136976 245__ $$aTranscriptome based individualized therapy of refractory pediatric sarcomas: feasibility, tolerability and efficacy.
000136976 260__ $$a[S.l.]$$bImpact Journals LLC$$c2018
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000136976 520__ $$aSurvival rates of pediatric sarcoma patients stagnated during the last two decades, especially in adolescents and young adults (AYAs). Targeted therapies offer new options in refractory cases. Gene expression profiling provides a robust method to characterize the transcriptome of each patient's tumor and guide the choice of therapy. Twenty patients with refractory pediatric sarcomas (age 8-35 years) were assessed with array profiling: ten had Ewing sarcoma, five osteosarcoma, and five soft tissue sarcoma. Overexpressed genes and deregulated pathways were identified as actionable targets and an individualized combination of targeted therapies was recommended. Disease status, survival, adverse events (AEs), and quality of life (QOL) were assessed in patients receiving targeted therapy (TT) and compared to patients without targeted therapy (non TT). Actionable targets were identified in all analyzed biopsies. Targeted therapy was administered in nine patients, while eleven received no targeted therapy. No significant difference in risk factors between these two groups was detected. Overall survival (OS) and progression free survival (PFS) were significantly higher in the TT group (OS: P=0.0014, PFS: P=0.0011). Median OS was 8.83 versus 4.93 months and median PFS was 6.17 versus 1.6 months in TT versus non TT group, respectively. QOL did not differ at baseline as well as at four week intervals between the two groups. TT patients had less grade 1 AEs (P=0.009). The frequency of grade 2-4 AEs did not differ. Overall, expression based targeted therapy is a feasible and likely beneficial approach in patients with refractory pediatric sarcomas that warrants further study.
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000136976 7001_ $$aRichter, Günther H S$$b1
000136976 7001_ $$aKesper, Marie Sophie$$b2
000136976 7001_ $$aGuggemoos, Monika$$b3
000136976 7001_ $$aGall, Katja$$b4
000136976 7001_ $$aPrexler, Carolin$$b5
000136976 7001_ $$aKazantsev, Ilya$$b6
000136976 7001_ $$aSipol, Alexandra$$b7
000136976 7001_ $$aLindner, Lars$$b8
000136976 7001_ $$aNathrath, Michaela$$b9
000136976 7001_ $$0P:(DE-He78)143af26de9d57bf624771616318aaf7c$$aWitt, Olaf$$b10$$udkfz
000136976 7001_ $$aSpecht, Katja$$b11
000136976 7001_ $$aBeitinger, Frigga$$b12
000136976 7001_ $$aKnebel, Carolin$$b13
000136976 7001_ $$aHosie, Stuart$$b14
000136976 7001_ $$avon Eisenhardt-Rothe, Rüdiger$$b15
000136976 7001_ $$aWeichert, Wilko$$b16
000136976 7001_ $$aLuettichau, Irene Teichert-von$$b17
000136976 7001_ $$aBurdach, Stefan$$b18
000136976 773__ $$0PERI:(DE-600)2560162-3$$a10.18632/oncotarget.25087$$gVol. 9, no. 29$$n29$$p20747-20760$$tOncoTarget$$v9$$x1949-2553$$y2018
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000136976 9141_ $$y2018
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