Dataset Information

Assessment of Survival of Pediatric Patients With Hepatoblastoma Who Received Chemotherapy Following Liver Transplant or Liver Resection.

ABSTRACT: Importance:The incidence of hepatoblastoma is increasing, and liver transplant (LT) provides a potential cure for pediatric patients with unresectable hepatoblastoma; however, the use of LT for hepatoblastoma has not been examined in a modern cohort. Moreover, data are lacking on the association between the type of surgical management received and overall risk of death among pediatric patients with hepatoblastoma. Objectives:To examine the receipt of LT among pediatric patients with hepatoblastoma and to assess overall survival of pediatric patients with hepatoblastoma who were treated with chemotherapy after LT or liver resection (LR) using data from a national cancer registry. Design, Setting, and Participants:This cohort study used data for 443 pediatric patients with histologically confirmed hepatoblastoma who received chemotherapy and surgical therapies, as documented in the Surveillance, Epidemiology, and End Results database of the National Cancer Institute, from 2004 to 2016, with follow-up through December 31, 2018. Multivariable logistic regression was used to determine factors associated with the use of LT. Cox proportional hazards models were used to assess factors associated with overall survival. Data analysis was performed from April 18, 2019, to July 25, 2019. Main Outcomes and Measures:Overall survival. Results:Among 443 patients receiving chemotherapy (mean [SD] age, 1.8 [2.6] years; 167 [37.7%] female), 350 (79%) underwent LR and 93 (21%) underwent LT. Multivariable analysis showed that patients with multiple lesions were more likely to undergo LT than LR (31% vs 13%; P?

SUBMITTER: Feng J

PROVIDER: S-EPMC6784752 | biostudies-literature | 2019 Oct

REPOSITORIES: biostudies-literature

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Assessment of Survival of Pediatric Patients With Hepatoblastoma Who Received Chemotherapy Following Liver Transplant or Liver Resection.

Feng Jincheng J He Ying Y Wei Lai L Chen Dong D Yang Huifang H Tan Rumeng R Chen Zhishui Z

JAMA network open 20191002 10

<h4>Importance</h4>The incidence of hepatoblastoma is increasing, and liver transplant (LT) provides a potential cure for pediatric patients with unresectable hepatoblastoma; however, the use of LT for hepatoblastoma has not been examined in a modern cohort. Moreover, data are lacking on the association between the type of surgical management received and overall risk of death among pediatric patients with hepatoblastoma.<h4>Objectives</h4>To examine the receipt of LT among pediatric patients wi ...[more]

PMID: 31584686

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Project description:BackgroundHepatoblastoma (HB) is a prevalent form of liver cancer in pediatric patients, characterized by an embryonal malignant tumor. In the current study, a clinical prediction model was developed; that can effectively assess the likelihood of a patient's survival with HB.MethodsData from the Surveillance, Epidemiology, and End Results (SEER) database for cases of HB between 2010 and 2019 were used in this retrospective research. Information on clinicopathologic characteristics, therapeutic interventions, and survival outcomes were included in the data. The HB patients were randomly assigned to the training or validation cohort in a 7:3 ratio. Using univariate and multivariate Cox proportional hazards regression models, the prognostic indicators for overall survival (OS) and cancer-specific survival (CSS) were identified. The area under the receiver operating characteristic curve (AUC-ROC), calibration plots, and concordance index (C-index) were used to evaluate the accuracy and calibration of these models. The clinical utility of the models was examined using decision curve analysis (DCA).ResultsThe multivariate Cox regression analysis revealed multiple autonomous prognostic determinants for the OS and CSS, including age, surgical interventions, and chemotherapy administration. Significantly, tumor size was found to be a strong predictor of OS. AUC values of 0.915, 0.846, and 0.847 for 1-, 3-, and 5-year OS, respectively, indicated that the nomogram-based models were highly accurate at predicting outcomes. Similarly, the AUC values for CSS were 0.871, 0.814, and 0.825. The C-index measurements, which quantify the discriminatory performance of the models, produced CSS values of 0.836 and OS values of 0.864. Furthermore, the calibration plots accurately represented the actual survival rates. Concurrently, the DCA had validated the clinical relevance of the nomogram-based models.ConclusionsThe present study successfully developed and validated user-friendly nomogram-based models, allowing for accurate assessment of OS and CSS in pediatric HB patients. These tools enable personalized survival predictions, enhance risk stratification, and strengthen clinical decision-making for managing HB.

Project description:Objective: To explore a CT-based radiomics model for preoperative prediction of event-free survival (EFS) in patients with hepatoblastoma and to compare its performance with that of a clinicopathologic model. Patients and Methods: Eighty-eight patients with histologically confirmed hepatoblastoma (mean age: 2.28 ± 2.72 years) were recruited from two institutions between 2002 and 2019 for this retrospective study. They were divided into a training cohort (65 patients from institution A) and a validation cohort (23 patients from institution B). Radiomics features were extracted manually from pretreatment CT images in the portal venous (PV) phase. The least absolute shrinkage and selection operator (LASSO) Cox regression model was applied to construct a "radiomics signature" and radiomics score (Rad-score) for EFS prediction. Then, a nomogram incorporating the Rad-score, updated staging system, and significant variables of clinicopathologic risk (age, alpha-fetoprotein (AFP) level, histology subtype, tumor diameter) as the radiomic model, clinicopathologic model, and combined clinicopathologic-radiomic model were built for EFS estimation in the training cohort, the performance of which was assessed in an external-validation cohort with respect to clinical usefulness, discrimination, and calibration. Results: Nine survival-relevant features were selected for a radiomics signature and Rad-score building. Multivariable analysis revealed that histology subtype (P = 0.01), PV (P = 0.001) invasion, and metastasis (P = 0.047) were independent risk factors of EFS. Patients were divided into low- and high-risk groups based on the Rad-score with a cutoff of 0.08 according to survival outcome. The radiomics signature-incorporated nomogram showed good performance (P < 0.001) for EFS estimation (C-Index: 0.810; 95% CI: 0.738-0.882), which was comparable with that of the clinicopathological model for EFS estimation (C-Index: 0.81 vs. 0.85). The radiomics-based nomogram failed to show incremental prognostic value compared with that using the clinicopathologic model. The combined model (radiomics signature plus clinicopathologic parameters) showed significant improvement in the discriminatory accuracy, along with good calibration and greater net clinical benefit, of EFS (C-Index: 0.88; 95% CI: 0.829-0.933). Conclusion: The radiomics signature can be used as a prognostic indicator for EFS in patients with hepatoblastoma. A combination of the radiomics signature and clinicopathologic risk factors showed better performance in terms of EFS prediction in patients with hepatoblastoma, which enabled precise clinical decision-making.

Project description:BackgroundThe effectiveness of adjuvant transarterial chemoembolization (TACE) for intrahepatic cholangiocarcinoma (ICC) after hepatectomy remains unclear. This study was performed to identify ICC patients who would benefit from adjuvant TACE.Patients and methodsThe study included 553 patients who underwent hepatectomy for ICC between January 2008 and February 2011 at the Eastern Hepatobiliary Surgery Hospital and who were treated with or without TACE (122 with TACE and 431 without TACE). Survival risk stratification was performed using the established prognostic nomogram (ICC nomogram). The predictive performance was evaluated by concordance index and calibration. The tumor recurrence and overall survival (OS) rates were analyzed by the Kaplan-Meier method before and after propensity score matching (PSM).ResultsThe predictive performance of the ICC nomogram was demonstrated by the well-fitted calibration curves and an optimal c-index of 0.71 for OS prediction. In the whole cohort, the 5-year recurrence and OS rates between the TACE and non-TACE groups were significantly different (5-year recurrence: 72.9% vs. 78.1%; OS: 38.4% vs. 29.7%). After 1:1 PSM, the TACE and non-TACE groups (122 patients each) had similar 5-year recurrence and OS rates (5-year recurrence: 72.9% vs. 74.2%; OS: 38.4% vs. 36.0%). By survival risk stratification based on ICC nomogram, only the patients in the lowest tertile (nomogram scores ?77) benefited from adjuvant TACE (TACE vs. non-TACE groups: 90.4% vs. 95.9% for 5-year recurrence; 21.3% vs. 6.2% for 5-year OS).ConclusionAdjuvant TACE following liver resection might be suitable for ICC patients with high ICC nomogram scores (?77).Implications for practiceThe accurate predictive performance of the established prognostic nomogram for intrahepatic cholangiocarcinoma (ICC) following liver resection was reconfirmed in an independent cohort with 553 patients. Based on the survival risk stratification using the nomogram, adjuvant transarterial chemoembolization following liver resection might be suitable only for ICC patients with high scores from the nomogram.

Dataset Information

Assessment of Survival of Pediatric Patients With Hepatoblastoma Who Received Chemotherapy Following Liver Transplant or Liver Resection.

Publications

Assessment of Survival of Pediatric Patients With Hepatoblastoma Who Received Chemotherapy Following Liver Transplant or Liver Resection.

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