Schmid, Irene and Haeberle, Beate and Albert, Michael H. and Corbacioglu, Selim and Froehlich, Birgit and Graf, Norbert and Kammer, Birgit and Kontny, Udo and Leuschner, Ivo and Scheel-Walter, Hans-Gerhard and Scheurlen, Wolfram and Werner, Sebastian and Wiesel, Thomas and von Schweinitz, Dietrich (2012) Sorafenib and cisplatin/doxorubicin (PLADO) in pediatric hepatocellular carcinoma. PEDIATRIC BLOOD & CANCER, 58 (4). pp. 539-544. ISSN 1545-5009, 1545-5017
Full text not available from this repository. (Request a copy)Abstract
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/m2/day, doxorubicin 2 x 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. Pediatr Blood Cancer 2012; 58: 539-544. (C) 2011 Wiley Periodicals, Inc.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | INTERNATIONAL-SOCIETY; RAF/MEK/ERK PATHWAY; PRECLINICAL MODELS; ANTITUMOR-ACTIVITY; ONCOLOGY-GROUP; CHILDREN; ANGIOGENESIS; COMBINATION; HEPATOBLASTOMA; BLOCKS; cisplatin; doxorubicin; hepatocellular carcinoma; pediatric; PLADO; sorafenib |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 15 May 2020 10:07 |
| Last Modified: | 15 May 2020 10:07 |
| URI: | https://pred.uni-regensburg.de/id/eprint/19003 |
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