Details der Publikation - Sorafenib and cisplatin/doxorubicin (PLADO) in pediatric hepatocellular carcinoma

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

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..

Medienart:	E-Artikel

Erscheinungsjahr:	2012
Erschienen:	2012

Enthalten in:	Zur Gesamtaufnahme - volume:58
Enthalten in:	Pediatric blood & cancer - 58(2012), 4, Seite 539-544

Sprache:	Englisch

Beteiligte Personen:	Schmid, Irene [VerfasserIn] Scheurlen, Wolfram [VerfasserIn]

Links:	Volltext

Themen:	Adolescent Antineoplastic Combined Chemotherapy Protocols Benzenesulfonates Carcinoma, Hepatocellular Child Cisplatin Disease-Free Survival Doxorubicin Female Follow-Up Studies Humans Liver Neoplasms Liver Transplantation Male Niacinamide Phenylurea Compounds Prospective Studies Protein Kinase Inhibitors Pyridines Retrospective Studies Sorafenib Survival Rate Transplantation, Homologous

Anmerkungen:	First published: 15 September 2011 Gesehen am 09.07.2019

Umfang:	6

doi:	10.1002/pbc.23295

funding:
Förderinstitution / Projekttitel:

PPN (Katalog-ID):	166882518X

Internformat


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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.
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650		4	\|a Benzenesulfonates
650		4	\|a Carcinoma, Hepatocellular
650		4	\|a Child
650		4	\|a Cisplatin
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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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