New concepts and outcomes for children with hepatoblastoma based on the experience of a tertiary center over the last 21 years

Tannuri, Ana Cristina Aoun; Cristófani, Lílian Maria; Teixeira, Ricardo Rodrigues; Filho, Vicente Odone; Tannuri, Uenis

doi:10.6061/clinics/2015(06)01

Cited by 18 publications

(9 citation statements)

References 26 publications

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“…Hepatoblastoma (HB) is rare; nevertheless, it is the most common primary malignant liver tumor in children younger than 5 years 1 2 . Multiple studies have reported HB 5-year survival rates of 60% to 87.7% 3 4 5 with a multidisciplinary treatment approach involving chemotherapy, surgical resection and liver transplantation. Despite this improvement, the outcome for patients with recurrent HB continues to be dismal.…”

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confidence: 99%

First Experience of Ultrasound-guided Percutaneous Ablation for Recurrent Hepatoblastoma after Liver Resection in Children

Liu

Zhou

Huang

et al. 2015

Sci Rep

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This study aimed to summarize the first experience with ultrasound-guided percutaneous ablation treatment (PAT) for recurrent hepatoblastoma (HB) after liver resection in children. From August 2013 to October 2014, PAT was used to treat 5 children with a total of 8 recurrent HB (mean size, 1.4 ± 0.8 cm; size range, 0.7–3.1 cm), including 4 patients with 7 tumors in the liver and 1 patient with 1 tumor in the lung. Technical success was achieved in all patients (5/5, 100%). The complete ablation rate after the first ablation session was 80% (4/5) on a patient-by-patient basis and 87.5% (7/8) on a tumor-by-tumor basis. Only 1 patient developed a fever with temperature >39 °C; it lasted 4 days after radiofrequency ablation (RFA) and was resolved by conservative therapy. During the follow-up period, new intrahepatic recurrences after PAT were detected in two patients. One died due to tumor progression 4 months after ablation. The median overall survival time after PAT was 13.8 months. PAT is a safe and promising therapy for children with recurrent HB after liver resection, and further investigation in large-scale randomized clinical trials is required to determine its role in the treatment of this disease.

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confidence: 99%

First Experience of Ultrasound-guided Percutaneous Ablation for Recurrent Hepatoblastoma after Liver Resection in Children

Liu

Zhou

Huang

et al. 2015

Sci Rep

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“…Hepatoblastomas are malignant liver cancers that consist of fetal liver cells, more mature liver cells and bile duct cells [Roebuck and Perilongo, ]. Ninety percent of hepatoblastomas occur before age 4 years, at a mean age of 22 months and median age of 16 months, and only exceptionally at an older age [Surveillance Epidemiology, and End Results (SEER) Program, ; Tannuri et al, ]. In BWS all hepatoblastoma occurred before 30 months (Table ) [Tannuri et al, ].…”

Section: Discussionmentioning

confidence: 99%

“…Ninety percent of hepatoblastomas occur before age 4 years, at a mean age of 22 months and median age of 16 months, and only exceptionally at an older age [Surveillance Epidemiology, and End Results (SEER) Program, ; Tannuri et al, ]. In BWS all hepatoblastoma occurred before 30 months (Table ) [Tannuri et al, ]. We have been unable to find a reliable description of an exception.…”

Section: Discussionmentioning

confidence: 99%

Phenotype, cancer risk, and surveillance in Beckwith–Wiedemann syndrome depending on molecular genetic subgroups

Maas

Vansenne

Kadouch

et al. 2016

American J of Med Genetics Pt A

176

255

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Patients with Beckwith-Wiedemann syndrome (BWS) have an increased risk to develop cancer in childhood, especially Wilms tumor and hepatoblastoma. The risk varies depending on the cause of BWS. We obtained clinical and molecular data in our cohort of children with BWS, including tumor occurrences, and correlated phenotype and genotype. We obtained similar data from larger cohorts reported in the literature. Phenotype, genotype and tumor occurrence were available in 229 of our own patients. Minor differences in phenotype existed depending on genotype/epigenotype, similar to earlier studies. By adding patients from the literature, we obtained data on genotype and tumor occurrence of in total 1,971 BWS patients. Tumor risks were highest in the IC1 (H19/IGF2:IG-DMR) hypermethylation subgroup (28%) and pUPD subgroup (16%) and were lower in the KCNQ1OT1:TSS-DMR (IC2) subgroup (2.6%), CDKN1C (6.9%) subgroup, and the group in whom no molecular defect was detectable (6.7%). Wilms tumors (median age 24 months) were frequent in the IC1 (24%) and pUPD (7.9%) subgroups. Hepatoblastoma occurred mostly in the pUPD (3.5%) and IC2 (0.7%) subgroups, never in the IC1 and CDKN1C subgroups, and always before 30 months of age. In the CDKN1C subgroup 2.8% of patients developed neuroblastoma. We conclude tumor risks in BWS differ markedly depending on molecular background. We propose a differentiated surveillance protocol, based on tumor risks in the various molecular subgroups causing BWS. © 2016 Wiley Periodicals, Inc.

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“…The incidence is slowly but steadily increasing at a rate of 1.2-1.5 cases/million/year, partly due to prematurity and very low birth weight [ 1 , 2 ]. The development of adjuvant, neo-adjuvant chemotherapy as well as the new surgical resection methods significantly improved the patients’ survival rate in the last decades [ 3 ]. Despite these significant achievements in HB treatment, the prognosis of patients with metastasis and those who are at a stage of pretreatment extent of disease (PRETEXT) IV remains unfavorable [ 4 ].…”

Section: Introductionmentioning

confidence: 99%

Central role of mTORC1 downstream of YAP/TAZ in hepatoblastoma development

Calvisi

Kiss

et al. 2017

Oncotarget

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Hepatoblastoma (HB) is the most common type of liver malignancy in children. Recent studies suggest that activation of Yes-associated protein (YAP) is a major molecular event in HB development, as activated YAP synergizes with mutant β-catenin to promote HB formation in mice (YAP/β-catenin). However, how YAP regulates HB development remains poorly defined. Similarly, de-regulation of mammalian target of rapamycin complex 1 (mTORC1) signaling has been implicated in multiple tumor types, but its functional role in HB development is scarcely understood. In the present study, we found that mTORC1 is activated in human HB cells and YAP/β-catenin-induced mouse HB tumor tissues. mTOR inhibitor MLN0128 significantly inhibits human HB cell growth in vitro. Furthermore, ablation of Raptor, the unique subunit of mTORC1, strongly delayed YAP/β-catenin-induced HB development in mice. At the molecular level, we found that expression of the amino acid transporter SLC38A1 is induced in mouse HB tissues, and amino acid deprivation leads to mTORC1 suppression in HB cell lines. Silencing of YAP and/or its paralog, transcriptional co-activator with PDZ binding motif (TAZ), decreased SLC38A1 expression as well as mTORC1 activation in HB cells. Furthermore, a frequent and concomitant upregulation of mTORC1 and SLC38A1 was detected in a collection of human HB specimens. Altogether, our study demonstrates the key role of mTORC1 in HB development. YAP and TAZ promote HB development via inducing SLC38A1 expression, whose upregulation leads to mTORC1 activation. Targeting mTOR pathway or amino acid transporters may represent novel therapeutic strategies for the treatment of human HB.

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New concepts and outcomes for children with hepatoblastoma based on the experience of a tertiary center over the last 21 years

Cited by 18 publications

References 26 publications

First Experience of Ultrasound-guided Percutaneous Ablation for Recurrent Hepatoblastoma after Liver Resection in Children

First Experience of Ultrasound-guided Percutaneous Ablation for Recurrent Hepatoblastoma after Liver Resection in Children

Phenotype, cancer risk, and surveillance in Beckwith–Wiedemann syndrome depending on molecular genetic subgroups

Central role of mTORC1 downstream of YAP/TAZ in hepatoblastoma development

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