Hepatoblastoma In Children Research Articles

Clinical features and prognostic analysis of hepatoblastoma in children under six years old

Objective: To summarize and analyze the clinical features, treatment effects and related factors affecting the prognosis of hepatoblastoma (HB) in children under six years old. Methods: Clinical data of 382 children with HB under six years old who were pathologically diagnosed at the Pediatric Single Center of Beijing Tongren Hospital from May 2005 to May 2019 were analyzed retrospectively. The factors affecting the treatment effect and survival rate of HB were analyzed. The independent risk factors affecting the prognosis of HB were studied by Cox regression model. The χ(2) test was used to compare the enumeration data between groups. Kaplan-Meier method was used for survival analysis. Log-rank test was used to compare the survival rates among subgroups. Results: Children enrolled were with median age of 1.75 (0.08 ~ 5.92) years old and a male to female ratio of 1.5. Alpha-fetoprotein (AFP) median level was 197 406.5 μg/L at initial diagnosis, and the pathological tissue type was mainly epithelial (55.8%). Preoperative PRETEXT stage was mostly stage III (58.6%). 86 cases (22.5%) had portal vein or hepatic vein, and vena cava invasion. 73 cases (19.1%) had extrahepatic adjacent tissues and organs invasion. Twenty-four cases (6.3%) had tumor rupture and bleeding. 171 cases (44.8%) had distant metastases, and 96 cases (25.1%) had multiple intrahepatic lesions. Patients were followed-up to May 2020 (median follow-up time was 56 months). After comprehensive treatment, 218 cases were completely relieved, and 69 cases were partially relieved, and the treatment efficiency was 75.1%. Kaplan-Meier survival analysis showed that the 1, 3, and 5-years overall survival rates (OS) were 93.7%, 84.0%, and 73.9%, respectively, and the event-free survival rates were 90.5%, 79.2%, and 67.5%, respectively. Comparison of the clinical factors of 5-year OS showed that AFP < 100 μg/L (HR = 3.341, P = 0.005), PRETEXT stage IV (HR = 4.026, P = 0.001), vascular invasion (HR = 2.178, P = 0.019) and distant metastasis (HR = 2.634, P = 0.010) were independent risk factors in each subgroup affecting the prognosis of children with HB, and the difference was statistically significant. Conclusion: HB prognosis is related to AFP level, PRETEXT stage, presence or absence of vascular invasion and distant metastasis. Therefore, its survival and prognosis will be different in the presence of different risk factors.

In vitro cytotoxic effects of 5-Fluorouracil on isolated murine ovarian preantral follicles

5 fluorouracil (5FU), an antineoplastic drug, is often utilized in the therapeutic regimen for several types of cancer, including the hepatoblastoma in children. The effects of 5FU on the population of ovarian preantral follicles, which is the largest oocyte reservoir, is still poorly understood. The integrity of the ovarian preantral follicle pool is important for lifelong fertility. The better understanding of such effects may favor intervention strategies to protect fertility in 5FU-treated children and women coping with cancer. To analyze the effects of 5FU on isolated murine secondary follicles in vitro, ovaries were collected from young mice (28–30 days old), and secondary follicles were isolated and cultured for 12 days in basic culture medium, with or without 5FU at concentrations of 0.3 mM, 1 mM, 3 mM, 10 mM, and 30 mM. In the in vitro study, we analyzed the percentage of morphologically normal follicles, antrum formation, follicular diameter, and hormone production. On day 12, oocytes were recovered for in vitro maturation. 5FU treatment did not alter the percentage of morphologically normal follicles. On day 12, only 1, 10, and 30 mM 5FU significantly reduced the percentage of antrum. From day 4 onwards, 5FU treatments significantly reduced follicle diameter. The meiosis resumption rate was significantly lower in all 5FU treatments. 5FU concentrations ≥3 mM reduced estradiol levels. In conclusion, 5FU does not affect follicular morphology. However, 5FU deleteriously affects follicular growth, estradiol production, and oocyte maturation in isolated ovarian follicles.

Trans-Ancestry Mutation Landscape of Hepatoblastoma Genomes in Children.

Hepatoblastoma (HB) is the most common malignant tumor in the liver of infants and young children. The incidence rate varies among different populations. However, genetic differences in HB patients with different epidemiological and ancestral backgrounds have not been found. In this study, we aim to analyze data from 16 patients treated at our center and collected published data from whole-exome sequencing studies on HB, and to explore the genetic differences between races. Data from a total of 75 HB patients of three races (24 Asian, 37 Caucasian and 14 Hispanic) were analyzed. We identified 16 genes with recurrent somatic mutations and 7 core pathway modules. Among them, the Wnt/β-catenin pathway had the highest mutation rate, and the mutation frequency in Caucasians and Hispanics was approximately twice as high as that in Asians. In addition, this study compared the characteristics of gene mutations between patients who underwent preoperative chemotherapy and those who did not and found that there was no significant difference in gene mutations between the two groups. We also preliminarily verified the function of cancer-associated candidate genes (CTNNB1 and KMT2D). In conclusion, we found ethnic differences in HB biology at the genomic level, which expands our understanding of the genetics of HB in children.

A new risk-stratification system for hepatoblastoma in children under six years old and the significance for prognosis evaluation\u2014a 14-year retrospective study from a single center

BackgroundThis study explores and analyzes the clinical characteristics and prognostic factors of hepatoblastoma (HB) in children under 6 years old and establishes a new risk-stratification system for individualized therapy.MethodsThe clinical data of 382 pediatric patients under 6 years old (231 males and 151 females) who had been diagnosed with HB by pathology between May 2005 and May 2019 were collected. By analyzing the risk factors influencing the survival rate of patients with HB, a new risk-stratification system was established, and it was compared with previous risk-stratification systems by a receiver operating characteristic (ROC) curve.ResultsAccording to a Kaplan-Meier survival analysis, the one-year, three-year, and five-year overall survival (OS) was 93.7, 84.0, and 73.9%, respectively, and the event-free survival (EFS) was 90.5, 79.2, and 67.5%, respectively.The independent risk factors influencing prognosis in pediatric patients with HB were alpha-fetoprotein (AFP) < 100 ng/ml or > 1000 ng/ml (HR = 3.341, P = 0.005); platelet count > 400 × 109/L (pooled hazard ratio [HR] = 2.123, P = 0.026); PRETEXT stage IV (HR = 4.026, P = 0.001); vascular involvement (HR = 2.178, P = 0.019); distant metastasis (HR = 2.634, P = 0.010);and multifocality (HR = 2.215, P = 0.012).A new risk-stratification system was established and divided into three groups: low risk, moderate risk, and high risk. There were statistical differences among the three groups (P = 0.002). Compared with the previous risk-staging systems, there was no significant difference in the survival rate. Although the effect in the guiding therapy was the same, the area under the curve for the ROC curve was 0.835 (95% CI: 0.784–0.885) for the new stratification system.ConclusionThis new risk-stratification system had a better predictive value for the prognosis of pediatric patients with HB than other stratification systems.

Analysis of survival rates and prognosis of hepatoblastoma in children: a retrospective study from a single center in China

Hepatoblastoma (HB) is the most common liver cancer in children, this study aims at analyzing the prognostic factors affecting the survival rates and summarizing the treatment experience. In this study, we reviewed patients with primary HB under the age of 14 years who underwent complete tumor resection from June 1997 to March 2019. The data of 72 patients were collected. Survival analysis was performed by Kaplan-Meier, multivariate Cox proportional hazards regression and linear mixed model for repeated measures (LMMRM). The 5-year and the 10-year event-free survival (EFS) of all patients were 78.2% and 73%, respectively. Both the 5-year and 10-year overall survival (OS) were 85.7%. Kaplan-Meier survival analysis showed that patients with tumor capsule infiltration (TCI) and patients with surgical margin less than 1 cm may also have a good prognosis. The Cox proportional hazards regression model analysis results were similar to the Kaplan-Meier analysis results. LMMRM analysis showed that there were significant differences in platelet, alpha-fetoprotein, C-reactive protein and hemoglobin values after surgery in the metastasis group (P < 0.05). This study suggests that patients with TCI or narrow surgical margin (<1 cm) may also have a good prognosis, and the risk stratification of HB can be used as the latest grading standard to evaluate the prognosis of patients.

Cesarean Section Is Associated with an Increased Risk of Acute Lymphoblastic Leukemia and Hepatoblastoma in Children from Minnesota.

In recent decades, Cesarean section (C-section) rates have increased. C-section is hypothesized to negatively impact the developing immune system by altering activation of the hypothalamic-pituitary-adrenal axis and the infant microbiome, among other mechanisms, thereby potentially modulating childhood cancer risk. Using linked birth and cancer registry data from Minnesota (1976-2014), we included individuals ages 0-14 at diagnosis with one of 19 cancers. Cases and controls were frequency matched by birth year. We used logistic regression to estimate ORs and 95% confidence intervals (95% CI) as the measure of association between C-section and cancer. We assessed sex-C-section interactions for each cancer and conducted stratified analyses in acute lymphoblastic leukemia (ALL) for birth year, age at diagnosis, and maternal race. There were 3,166 cases and 20,589 controls. One third (n = 1,174) of controls born during 2004-2014 were delivered via C-section compared with 42.2% of cases (n = 285). C-section was associated with ALL (n = 819; OR: 1.20; 95% CI: 1.01-1.43) and hepatoblastoma (n = 50; OR: 1.89; 95% CI: 1.03-3.48), particularly among females (ALL OR: 1.34; 95% CI: 1.04-1.72; hepatoblastoma OR: 3.87; 95% CI: 1.30-11.57). The risk of ALL was highest during 2005-2014 (OR: 1.62; 95% CI: 1.11-2.34) and among children ages 1-5 years (OR: 1.28; 95% CI: 1.02-1.61). C-section was associated with an increased risk of ALL and hepatoblastoma. These associations require investigation to determine causality and rule out confounding by indication or reverse causality. The mechanisms underlying these associations may depend on neonatal immune system processes altered during C-section deliveries.

Therapeutic effect and clinical cost of multi-disciplinary team model of hepatoblastoma in children

Objective To summarize and analyze the treatment process, long-term efficacy and clinical economics of children's hepatoblastoma (HB) in multi-disciplinary team (MDT) mode, so as to provide basis for the rational choice of diagnosis and treatment. Methods From January 2014 to February 2019, 13 cases of hepatoblastoma in children who completed the whole treatment course in the Pediatric Hematology Tumor Ward of Peking University First Hospital were collected and analyzed, and were followed up until June 30, 2020. There were 9 males and 4 females who were diagnosed and treated according to the MDT process in the hospital. The median age was 16 months (2-54 months), 69.23% (9/13) were under 2 years old. The characteristics, diagnosis and treatment process and treatment effect of the cases were summarized, and the cost of clinical treatment was analyzed. Results According to the pretreatment extent of disease(PRETEXT), there were 1, 9 and 3 children with stages Ⅱ, Ⅲ and Ⅳ. 76.92% (10/13) of the patients had the largest tumor diameter > 10 cm. All the patients received preoperative neoadjuvant chemotherapy (8 patients received 4 cycles of chemotherapy, and 6 patients changed the chemotherapy plan), surgical treatment and postoperative chemotherapy, the tumor volume decreased by more than 50% (64%-95%) in 12 cases, except 1 case with no significant increase of alpha fetal protein and multiple lesions.The median length of stay was 87 days (68-214 days), the median cost of stay was 200 000 yuan (115 000-500 000 yuan), the median length of stay was 7 days (5-17 days), the median cost of stay was 20 000 yuan (15 000-60 000 yuan), and the incidence of postoperative complications was 7.69% (1/13). All the patients were followed up for 16-69 months. All the patients survived. Conclusion Under the MDT mode, the treatment is seamless connection, the long-term prognosis of children with HB is good, and the total hospitalization cost and time are within the acceptable range. Standard preoperative neoadjuvant chemotherapy can significantly reduce the tumor, improve the resection rate, reduce postoperative complications, reduce the total individual expenditure, shorten the total hospital stay, and further improve the long-term disease-free survival rate.

A single-center retrospective analysis of childhood hepatoblastoma in China.

This study aimed to investigate the critical factors associated with prognosis for children with hepatoblastoma (HB) in mainland China combined with the aspect of health economics and management. This study retrospectively reviewed children with HB in Renji Hospital Affiliated to the Shanghai Jiao Tong University School of Medicine from January 2013 to December 2019. Descriptive analysis was used to describe the essential characteristics. Kaplan-Meier method and Cox proportional hazard models were used to estimate the survival rate and prognosis factors. For the 87 children with HB, the average survival was 2,002.8 days (95% CI: 1,798.7-2,206.9 days), the 1- and 5-year survival rates were 87.7% and 78.9%, respectively. Undergoing surgery and sex were independent prognostic factors of childhood HB. Children with HB undergoing hepatectomy (HR: 0.039) or liver transplantation (HR: 0.142) had a better prognosis, while boys were associated with a poorer prognosis (HR: 3.614). The average medical expenses for childhood HB were 40,217.5±3,862.0 CNY and liver transplantation cost more than hepatectomy. The results had a comparable survival rate with other studies globally. Surgical therapy and sex are associated with the prognosis of children with HB. The economic burden of childhood HB deserves to be further explored.

Pediatric Hepatocellular Carcinoma.

Pediatric hepatocellular carcinoma is rarely seen in childhood. It constitutes approximately 1% of childhood solid organ malignancies. Pediatric hepatocellular carcinoma is the second most common malignant liver tumor after hepatoblastoma in children. In this review, we aimed to review the diagnosis and treatment of pediatric hepatocellular carcinoma in the light of the latest literature. We reviewed the literature in terms of the diagnosis and treatment of pediatric hepatocellular carcinoma. Hepatocellular carcinoma (HCC) and hepatoblastoma constitute 0.5-1.5% of all childhood malignant tumors. HCC is responsible for 27% of all liver tumors and 4% of all pediatric liver transplantations. While 99.6% of HCC is seen in adults, only 0.4% of it is seen in pediatric patients. Etiological predisposition and biological behavior are different from adults. In a child with cirrhosis or liver disease, HCC should be suspected in the presence of a high level of AFP and an abnormal nodule on ultrasonography. Hepatoblastoma should be considered first in the differential diagnosis. Treatment of pediatric HCC is challenging. Complete surgical resection is essential for the cure. To this end, different neoadjuvant chemotherapy protocols have been designed to convert non-resectable tumors into resectable tumors. For tumors that cannot be resected, liver transplantation for each patient with childhood HCC should be decided individually.

Hepatoblastoma del adulto

Fundamento: el hepatoblastoma del adulto (HBA) es un tumor hepático poco frecuente y con un mal pronóstico, lo cual contrasta con el hepatoblastoma infantil (HBI). Esta patología aún no ha sido comprendida completamente y hasta la fecha, no se han reportado de forma adecuada más de 50 casos en la literatura médica.&#x0D; Objetivo: presentar el caso de un paciente que fue egresado de nuestro hospital con el diagnóstico de carcinoma hepatocelular, aproximadamente con 3 meses de anterioridad.&#x0D; Caso clínico: paciente masculino de 60 años con historia de alcoholismo y de ser un fumador inveterado. Fue ingresado en nuestro hospital por dolor abdominal, en el momento del examen físico, puso de manifiesto un tumor palpable en la región del hipocondrio derecho. Este paciente había sido egresado aproximadamente 3 meses atrás, con el diagnóstico de carcinoma hepatocelular, en el curso de una cirrosis hepática. El hombre falleció por causa de la progresión de la enfermedad y la autopsia reveló la existencia de un HBA.&#x0D; Conclusiones: el HBA es un tumor infrecuente, de grave pronóstico y muchos casos son asintomáticos hasta el momento del diagnóstico. Dicho tumor, por lo regular, presenta un gran tamaño. Las enzimas hepáticas, la alfafetoproteína y los estudios imagenológicos revelan el diagnóstico de un hepatocarcinoma, el cual es un tumor frecuente en los adultos. Asimismo, el estudio histológico confirma el diagnóstico. Debido a su mal pronóstico, y a las mejores perspectivas de tratamiento en niños, hoy en día es lógico utilizar el tratamiento pediátrico en los adultos. Se necesitan más estudios de investigación para el manejo óptimo del HBA.

Haematological Toxicities Following Treatment of Childhood Hepatoblastoma with Intensive Multiagent Chemotherapy

Background: Hepatoblastoma is a rare malignant liver tumour that occurs almost exclusively in childhood. Although surgical resection is the foundation of curative therapy, with the use of effective neoadjuvant and adjuvant chemotherapy the 5 years overall survival of patient with hepatoblastoma had recently reached up to 80% to 90 %. But the frequency and severity of haematological toxicity is one of the major concerns of intensive chemotherapy. Objective: To evaluate the frequency and severity of haematological toxicities following treatment of childhood hepatoblastoma with two different regimens of multi-agent chemotherapy. Methodology: This was a comparative observational study conducted at Bangabandhu Sheikh Mujib Medical university in 24 childhood hepatoblastoma patients who received either cisplatin/carboplatin /doxorubicin (Group A; n=14) or cisplatin/ vincristine/5- fluorouracil (Group B; n= 10) to between March 2010 and July 2014. Results: Grade 3 or 4 anaemia and thrombocytopenia, and Grade 4 neutropenia were observed in 64.3% vs 60%, 35.7% vs 20% and 57.1% vs 20% respectively in group A and Group B. Febrile neutropenia occurred in 100% vs 70% and septicaemia developed in 92.9% vs 70%. Red cell concentrates and Platelet transfusions were required in 35.7% vs 50% and 14.3% vs 10% respectively in two groups. Deaths due to haematological toxicities occurred in 21.4% in Group A and 21% in Group B. Conclusion: We found similar toxicity profiles for two chemotherapy regimens except for higher rates of febrile neutropenia and septicaemia in patients treated with cisplatin/carboplatin /doxorubicin compared to cisplatin/ vincristine/5- fluorouracil.

Malignant Hypertension Secondary to Renin-Producing Hepatoblastoma

AbstractClinical presentation of hepatoblastoma is often insidious and its paraneoplastic manifestations are not uncommon. We report an extremely rare case of a renin-secreting hepatoblastoma inducing malignant hypertension remarked in our clinic. The hepatic malignancy classified as PRETEXT IV and a multidisciplinary approach according to SIOPEL III was made with a poor outcome. Hepatoblastoma in children should be considered as possible etiology of hypertension in children.

Đặc điểm bệnh u nguyên bào gan và kết quả điều trị ở trẻ em tại bệnh viện Nhi Đồng 2

CHARACTERISTICS AND TREATMENT OF HEPATOBLASTOMA IN CHILDREN AT PEDIATRIC HOSPITAL 2 Objective: To determine the epidemiological, clinical, paraclinical characteristics and evaluate the result in treatment of hepatoblastoma (HB) at Department of Hematology-Oncology, Children’s Hospital 2 from January 2011 to end of May 2019. Method: Cases series study in combination of retrospective and prospective. Results: From January 2011 to end of May 2019, there were 67 patients diagnosed with HB. The mean age at diagnosis was 22.7 ± 22.9 months with the most prevalent age group ranging from 6 months to 3 years. The male to female ratio was 1.8/1. The most common clinical symptoms were abdominal distention and hepatomegaly, approximately 7.5% HB cases were detected accidentally by ultrasound. Nearly 60% and 80% patients presented with anemia and thrombocytosis at admission, respectively.Almost tumors were unifocal locating in the right lobe of the liver and 58.2% cases belonged to standard risk group. Assessing treatment protocol in 54 HB patients demonstrate that number of patients undergoing hepatectomy after preoperative chemotherapy accounted for 82%. The most frequent acuteside effects after chemotherapy were myelotoxicity and infection. The proportion of fatal and relapsed cases was 31.5% and 12.9% respectively. Overall and event-free survival rate of the study after 3 years was 65.7% and 68.1% individually. Conclusion: Hepatoblastoma is a rare malignant childhood disease, pretty good response to chemotherapy followed by surgery. However mortality rate is rather high compared to worldwide reasearchs. Thus development of liver transplant is necessary to improve survival rate. Keywords: Hepatoblastoma, chemothearapy, surgery, liver transplant.

Liver transplantation for hepatoblastoma in children

To improve the outcomes in children with hepatoblastoma. There were 160 children with focal liver lesions who underwent surgery at the department of liver transplantation in 2008-2019. Patients with malignant tumors made up 77% (n=123). Hepatoblastoma (HB) prevailed (86%, n=106). Liver transplantation was performed in 19 (18%) patients with HB. Median follow-up after transplantation was 24.3 months by December 2019. Follow-up period did not exceed 4 years in more than 2/3 of patients. Overall and disease-free 10-year survival was 87.1% and 82.7%, respectively. Similar values were observed after resections (91.1% and 86.6%). At the same time, actuarial 4-year survival after liver transplantation for HB was 68%. Improvement of treatment outcomes may be achieved through multidisciplinary interaction ensuring timely drug therapy and liver transplantation.

Impact of microscopically margin-positive resection on survival in children with hepatoblastoma after hepatectomy: a retrospective cohort study.

Impact of R1 (microscopically margin-positive) resection on survival of patients with hepatoblastoma (HB) remains debatable. This study aimed to compare the long-term outcomes of R0 (microscopically margin-negative) and R1 resection for HB in children after hepatectomy. We retrospectively reviewed files of children with HB who underwent resection at our institution between September 1, 2005, and November 30, 2017. Survival analyses and prognostic factors were evaluated using Kaplan-Meier curves and Cox regression models. Of 259 patients, 218 (84.2%) underwent R0 and 41 (15.8%) R1 resection. After adjusting for confounding factors, R1 resection demonstrated non-significantly lower overall survival (OS: hazard ratio [HR] = 0.75; 95% CI 0.34-1.64) and shorter event-free survival (EFS: HR = 0.97; 95% CI 0.53-1.78) rates than R0 resection. However, stratified analysis showed significantly increased risk of poor OS and EFS in patients with metastasis and mixed epithelial/mesenchymal pathologic subtype in R1 compared with R0 resection (P values for interactions < 0.05). There was no significant difference between R0 resection with metastasis and R1 resection with metastasis in the incidence of local recurrence (P = 0.494); however, a significant difference in the incidence of local recurrence was seen between R0 and R1 resection for subgroups with mixed pathologic subtypes (P = 0.035). With effective chemotherapy, microscopic margin status may not be associated with survival outcome in children with HB undergoing hepatectomy. However, stratified analysis showed that R1 resection might be associated with decreased survival in children with mixed epithelial/mesenchymal HB, compared with R0 resection, and not affect survival outcomes in those with an epithelial subtype and without metastasis.

Incidence trends and survival prediction of hepatoblastoma in children: a population-based study

BackgroundHepatoblastoma is a rare disease that nevertheless accounts for the majority of liver malignancies in children. Due to limited epidemiological data, therapy for hepatoblastoma tends to be individualized. This study aimed to evaluate incidence trends of hepatoblastoma and to develop a nomogram to predict the survival of children with newly diagnosed hepatoblastoma on a population-based level.MethodsIndividuals up to 18 years of age with hepatoblastoma recorded in 18 registries of the Surveillance, Epidemiology, and End Results (SEER) database between 2004 and 2015 were examined. Joinpoint regression analyses were applied to assess incidence trends in annual percentage change (APC). Multivariable Cox regression was used to identify factors associated with overall survival (OS). A nomogram was constructed to predict OS in individual cases based on independent predictors. Concordance index (C-index) and calibration curves were used to evaluate predictive performance.ResultsBetween 2004 and 2015, hepatoblastoma incidence increased significantly (APC, 2.2%; 95% confidence interval [CI] 0.5% to 3.8%, P < 0.05). In particular, this increase was observed among 2- to 4-year-old patients, males, and African–Americans. The 5- and 10-year OS rates were 81.5% and 81.0%, respectively. Age of 2 to 4 years, African–American ethnicity, and no surgery were independent predictors for short OS. Distant disease at presentation was found not to be an independent factor of survival. The nomogram had a C-index of 0.79 (95% CI 0.74–0.84) with appropriate calibration curve fitting.ConclusionsWe constructed a nomogram that integrates common factors associated with survival for hepatoblastoma patients. It provides accurate prognostic prediction for children with hepatoblastoma.

HEPATOBLASTOMA- MULTIMODALITY TREATMENT AND OUTCOMES - A TERTIARY CENTRE EXPERIENCE

Hepatoblastoma (HBL) is the most common primary liver tumor in (1) children and accounts for 1% of all pediatric cancers . Preterm is a risk factor although most cases are sporadic, but some are associated with constitutional genetic abnormalities and malformations, such as the Beckwith-Wiedemann syndrome and familial adenomatous polyposis (2,3) . Over the last three decades, the annual incidence of (4) Hepatoblastoma in children has gradually increased . Extremely premature babies with a low birth weight have been reported to have a greatly increased risk of developing Hepatoblastoma. The increased survival rates of these premature babies might account for the increased annual incidence of Hepatoblastomas. Upto 60% of ( 5 ) hepatoblastoma are unresectable on presentation .Due to chemoresponsiveness of hepatoblastoma neoadjuvant chemotherapy is used to downsize the tumor for surgical resection. This retrospective study reviews our experience with multimodality treatment in

Retrospective Analysis of Childhood Hepatoblastoma in a Single Centre in China

AimsTo investigate the protocol efficacy and prognostic factors for paediatric hepatoblastoma in a multidisciplinary model in our centre. Materials and methodsConsecutive hepatoblastoma patients (<18 years old) treated at Shanghai Children's Medical Center in China from August 2011 to October 2017 were analysed retrospectively for clinical features, chemotherapy courses, surgical treatment and outcomes. ResultsOne hundred and four cases of paediatric hepatoblastoma (64 males, 40 females; median age at diagnosis 1.64 years) had a median follow-up of 30.68 months (range 8.3–73.3 months). First complete remission was achieved in 95 cases, 85 of which achieved continuous complete remission. Another three cases were lost to follow-up after a median of 24.73 months in complete remission. Seven cases relapsed later, with two achieving a second complete remission and four deaths. Nine cases did not achieve complete remission and five of them died. In general, the 5-year overall survival rate and 5-year event-free survival (EFS) rate were 86.3 ± 5.0% and 81.8 ± 4.3%, respectively. Thirty-two cases were classified as standard risk and 72 as high risk with 5-year EFS of 96.8 ± 3.2% and 75.7 ± 5.7% (P = 0.029) and 5-year overall survival of 100% and 80.5 ± 7.0%, respectively. The mean platelet count (P = 0.0036), lactate dehydrogenase (P = 0.0443) and ferritin level (P = 0.0006) at diagnosis were much higher in the high-risk group than in the standard-risk group. Univariate analysis showed that patients <5 years of age (P = 0.018), with higher α-fetoprotein (AFP) level (>100 ng/ml, P = 0.008), without metastases at diagnosis (P = 0.001) and postoperative AFP recovery after no more than three chemocycles (P = 0.014) had better overall survival. In addition, the above factors, except metastases at diagnosis and risk group, were associated with prognosis in the multivariate analysis. ConclusionsThe result of this protocol had similar overall survival and EFS rates compared with those in developed countries. Normal postoperative AFP levels after three chemocycles has prognostic value.

Short-term prognosis of childhood hepatoblastoma in relation to ERCC1 C118T single nucleotide polymorphism and VEGF expression.

To evaluate the short-term prognosis of pediatric hepatoblastoma (HB) in relation to excision repair cross-complementation gene 1 (ERCC1) C118T single nucleotide polymorphism (SNP) and VEGF expression. ERCC1 C118T SNP and VEGF expression were detected and investigated in 31 children with HB undergoing platinum-based chemotherapy, to analyze their relationship with short-term pediatric HB prognosis. CC (38.7%; 12/31), CT (35.5%; 11/31), and TT (25.8%; 8/31) ERCC1 C118T mutation types were identified. The Kaplan-Meier survival curve analysis showed that the CC group had a better short-term prognosis than the CT + TT group (p = 0.010). VEGF was overexpressed in 14 cases (45.2%) and underexpressed in 17 cases (54.8%). The Kaplan-Meier survival curve analysis showed that the high VEGF expression group showed poorer short-term prognosis than the lower VEGF expression group (p = 0.004). In this study, ERCC1 C118T SNP in children with HB was mainly found to be mutant type CT + TT. Compared to wild type CC, children with the mutant type CT + TT exhibited better treatment efficacy and remission with platinum-based chemotherapy as well as better survival rates. Moreover, the short-term prognosis of children with low VEGF expression was better than in those with high expression.

Defining an optimal time window to screen for hepatoblastoma in children with Beckwith-Wiedemann syndrome.

Patients with Beckwith-Wiedemann spectrum (BWSp) undergo quarterly alpha-fetoprotein measurement for hepatoblastoma (HB) screening up to 4 years of age, paralleling the epidemiology of nonsyndromic HB. However, specific data on the timing of HB development in BWSp are lacking. Here we compare the timing of presentation of HBs in BWSp with a control cohort of consecutive HB cases, demonstrating that halving screening duration of screening procedures in BWSp likely will not impact its effectiveness.