Sorafenib and cisplatin/doxorubicin (PLADO) in pediatric hepatocellular carcinoma

PURPOSE: Overall survival is poor in children with primary unresectable hepatocellular carcinoma. Sorafenib has been shown to significantly improve progression-free survival in adult hepatocellular carcinoma (HCC) patients. We evaluated the experience of PLADO (cisplatin 80 mg/m(2) /day, doxorubicin...

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Hauptverfasser: Schmid, Irene (VerfasserIn) , Scheurlen, Wolfram (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: 2012
In: Pediatric blood & cancer
Year: 2011, Jahrgang: 58, Heft: 4, Pages: 539-544
ISSN:1545-5017
DOI:10.1002/pbc.23295
Online-Zugang:Verlag, Volltext: http://dx.doi.org/10.1002/pbc.23295
Volltext
Verfasserangaben:Irene Schmid, Beate Häberle, Michael H. Albert, Selim Corbacioglu, Birgit Fröhlich, Norbert Graf, Birgit Kammer, Udo Kontny, Ivo Leuschner, Hans-Gerhard Scheel-Walter, Wolfram Scheurlen, Sebastian Werner, Thomas Wiesel, and Dietrich von Schweinitz

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245 1 0 |a Sorafenib and cisplatin/doxorubicin (PLADO) in pediatric hepatocellular carcinoma  |c Irene Schmid, Beate Häberle, Michael H. Albert, Selim Corbacioglu, Birgit Fröhlich, Norbert Graf, Birgit Kammer, Udo Kontny, Ivo Leuschner, Hans-Gerhard Scheel-Walter, Wolfram Scheurlen, Sebastian Werner, Thomas Wiesel, and Dietrich von Schweinitz 
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520 |a PURPOSE: Overall survival is poor in children with primary unresectable hepatocellular carcinoma. Sorafenib has been shown to significantly improve progression-free survival in adult hepatocellular carcinoma (HCC) patients. We evaluated the experience of PLADO (cisplatin 80 mg/m(2) /day, doxorubicin 2 × 30 mg/m(2) /day) in combination with sorafenib in pediatric HCC patients. - PATIENTS AND METHODS: Clinical data of 12 patients (7-16 years), 7 with unresectable tumor, were retrospectively assessed. - RESULTS: In total 6/12 (50%) patients are in complete remission after a median follow-up of 20 months (4 with PLADO/sorafenib/resection, 2 with liver transplantation after local relapse). Of the seven patients with unresectable tumor, PLADO/sorafenib resulted in partial response (PR) in four, stable disease (SD) in two, and progression in one. Three are alive in CR after complete resection after 12 (alternative therapy after two cycles PLADO/sorafenib), 12 and 18 months (six cycles PLADO/sorafenib), respectively. All four patients with elevated alpha-fetoprotein levels had a marked drop after two cycles. Of the five patients with primary complete tumor resection one is alive disease-free at 27 months. Four had local or metastatic relapses (13, 7, 12, and 13 months), two of whom were rescued by liver transplantation (CR after 25 and 32 months). The main toxicity attributable to sorafenib was a hand-foot skin reaction (HFSR) in seven patients. - CONCLUSION: Sorafenib in combination with PLADO may be a promising approach in pediatric HCC; HFSR was the most important toxicity. Data based on prospective studies are needed to evaluate pharmacokinetics, resectability rates, and survival in pediatric HCC treated with sorafenib. 
534 |c 2011 
650 4 |a Adolescent 
650 4 |a Antineoplastic Combined Chemotherapy Protocols 
650 4 |a Benzenesulfonates 
650 4 |a Carcinoma, Hepatocellular 
650 4 |a Child 
650 4 |a Cisplatin 
650 4 |a Disease-Free Survival 
650 4 |a Doxorubicin 
650 4 |a Female 
650 4 |a Follow-Up Studies 
650 4 |a Humans 
650 4 |a Liver Neoplasms 
650 4 |a Liver Transplantation 
650 4 |a Male 
650 4 |a Niacinamide 
650 4 |a Phenylurea Compounds 
650 4 |a Prospective Studies 
650 4 |a Protein Kinase Inhibitors 
650 4 |a Pyridines 
650 4 |a Retrospective Studies 
650 4 |a Sorafenib 
650 4 |a Survival Rate 
650 4 |a Transplantation, Homologous 
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