Hepatocellular carcinoma in children and effect of living‐donor liver transplantation on outcome

Abstract

Hepatocellular carcinoma (HCC) is primarily observed in the older children and in most cases it develops in association with liver cirrhosis. Liver transplantation offers a good chance for long-term cure. To evaluate the outcome of children with HCC and the impact of living-donor orthotopic liver transplantation (OLT) on survival a retrospective review of radiographic, laboratory, pathologic, and therapeutic data in 13 children (six female and seven male) with chronic liver disease accompanied with HCC were studied. The patients were divided into two groups according to therapeutic modality: transplanted and non-transplanted patients. Kaplan-Meier survival curves in various therapeutic groups were plotted. The mean age of patients was 6.4 +/- 4.8 yr. Pediatric end-stage liver disease score was adapted to model for end-stage liver disease score for HCC and ranged between 1-44 and 18-44, respectively. The underlying liver diseases were tyrosinemia type 1 (n = 6), chronic hepatitis B infection (n = 6), glycogen storage disease type 1 (n = 1). Alfa-feto protein levels were elevated in all patients except one. Median number of tumor nodules was three (1-10), median maximal diameter of tumor nodules was 3.4 cm (0.5-8). Eleven patients were eligible for OLT whereas two patients were not eligible. Seven of the 11 patients considered for transplantation underwent living-donor OLT. Remaining four patients died while waiting on cadaveric transplant list. Overall 1 and 4-yr survival rates for all patients were 53.3 and 26.6%, respectively, and were found significantly higher in transplanted children than non-transplanted children (72%, 72% vs. 33% and 16.6%). No patient had tumor recurrence at median of 36-month follow-up after OLT. OLT is a life-saving procedure for children with chronic liver disease accompanying with HCC. Living-donor OLT avoids the risk of tumor progression and transplant ineligibility in these children.

Hepatic stress in hereditary tyrosinemia type 1 (HT1) activates the AKT survival pathway in the fah−/− knockout mice model

“…NTBC (2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione), a therapeutic inhibitor of the upstream enzyme 4-hydroxyphenylpyruvate dioxygenase (HPPD) (Fig. 1), prevents acute liver failure, a major cause of early death in HT1 patients [13][14][15] (HCC) despite NTBC treatment [16][17][18][19], making HT1 the genetic disease with the highest susceptibility for a primary liver cancer [20]. NTBC discontinuation in adult fah À/À HT1 mice was reported to induce resistance to cell death in vivo [21], as shown by survival to lethal doses of homogentisate (HGA).…”

Liver transplantation for adenomatosis: European experience

“…1 HCC Gordon-Burroughs et al (35) (2014) 1 HCC Fernandez-Vega et al (36) (2014) 1 No Patients with GSD IA Matern et al (14) (1999) 14 No Labrune (15) (2002) 1 HCC Lerut et al (13) (2003) 1 HCC Franco et al (37) (2005) 1 HCC Arikan et al (38) (2006) 13 No Davis and Weinstein (39) (2008) 4 No Reddy et al (16) (2009) 1 HCC Manzia et al (40) (2011) 2 HCC…”

Liver Transplantation in Children With Glycogen Storage Diseases: Risk Assessment and Necessity of This Procedure

“…По опыту ряда авторов, лабораторные показатели функции печени находятся в пределах нормы, несмотря на наличие аденомы печени и массивную гепатомега-лию, а альфа-фетапротеин повышается не во всех случаях злокачественной трансформации [3,17,26]. Однажды обнаруженную аденому печени реко-мендовано наблюдать УЗИ органов брюшной поло-сти каждые 3-6 месяцев [41,42].…”

“…Несмотря на описанную связь гепатоцел-люлярной карциномы и аденомы печени, наблю-даемых у пациентов с гликогенозом I типа, только у одного ребенка из 25 была обнаружена ГЦК в удаленной печени, и еще у одного реципиента была обнаружена дисплазия средней степени [3,16]. По-сле трансплантации печени у всех пациентов c бо-лезнью Гирке были полностью купированы метабо-лические нарушения: гипогликемия, лактат-ацидоз, гиперурикемия и гиперлипидемия.…”