Novel patient-derived xenograft and cell line models for therapeutic testing of pediatric liver cancer.
Journal of Hepatology 65(2):325 (2016)
Pediatric liver cancer is a rare but serious disease whose incidence is rising, and for which the therapeutic options are limited. Development of more targeted, less toxic therapies is hindered by the lack of an experimental animal model that captures the heterogeneity and metastatic capability ...
Relapsed hepatoblastoma confined to the lung is effectively treated with pulmonary metastasectomy.
Journal of Pediatric Surgery 51(4):525 (2016)
In children diagnosed with hepatoblastoma (HB), the lungs are the most common site of metastasis at both initial presentation and relapse. Previous studies have encouraged pulmonary metastasectomy to achieve a disease-free state after resection of the primary hepatic lesion. However, there is no...
Identifying gene disruptions in novel balanced de novo constitutional translocations in childhood cancer patients by whole-genome sequencing.
Genetics in Medicine 17(10):831 (2015)
We applied whole-genome sequencing (WGS) to children diagnosed with neoplasms and found to carry apparently balanced constitutional translocations to discover novel genic disruptions.
We applied the structural variation (SV) calling programs CREST, BreakDancer, SV-STAT, and CGAP-CNV, and we deve...
Reply to: No correlation between estimated and actual glomerular filtration rates in pediatric oncology patients.
Pediatric Blood & Cancer 62(7):1300 (2015)
Focal nodular hyperplasia in children: an institutional experience with review of the literature.
Journal of Pediatric Surgery 50(3):382 (2015)
Focal nodular hyperplasia (FNH) is uncommonly diagnosed in pediatric patients and may be difficult to distinguish from a malignancy. We present a review of all children with a tissue diagnosis of FNH at our institution, describe the diagnostic modalities, and provide recommendations for diagnosi...
Relapse surveillance in AFP-positive hepatoblastoma: re-evaluating the role of imaging.
Pediatric Radiology 44(10):1275 (2014)
Children with hepatoblastoma routinely undergo repetitive surveillance imaging, with CT scans for several years after therapy, increasing the risk of radiation-induced cancer.
The purpose of this study was to determine the utility of surveillance CT scans compared to serum alpha-fetoprotein (AFP...
A phase I trial of imetelstat in children with refractory or recurrent solid tumors: a Children's Oncology Group Phase I Consortium Study (ADVL1112).
Clinical Cancer Research 19(23):6578 (2013)
Imetelstat is a covalently-lipidated 13-mer thiophosphoramidate oligonucleotide that acts as a potent specific inhibitor of telomerase. It binds with high affinity to the template region of the RNA component of human telomerase (hTERC) and is a competitive inhibitor of telomerase enzymatic activ...
Extrarenal Wilms tumor: a case report and review of the literature.
Journal of Pediatric Surgery 48(6):E33 (2013)
Extrarenal Wilms tumors are extremely rare with only isolated case reports in the pediatric literature. We present the case of a 2-year old boy who presented with a large abdominal mass and constipation. Pathologic diagnosis of the tumor was extrarenal Wilms tumor (ERWT) with favorable histology...
Phase I trial of capecitabine rapidly disintegrating tablets and concomitant radiation therapy in children with newly diagnosed brainstem gliomas and high-grade gliomas.
Neuro-Oncology 15(6):759 (2013)
We conducted a phase I study to estimate the maximum tolerated dose and describe the dose-limiting toxicities and pharmacokinetics of oral capecitabine rapidly disintegrating tablets given concurrently with radiation therapy to children with newly diagnosed brainstem or high-grade gliomas.
Unusually early presentation of small-bowel adenocarcinoma in a patient with Peutz-Jeghers syndrome.
Journal of Pediatric Hematology/Oncology 35(4):323 (2013)
Peutz-Jeghers syndrome (PJS) is an autosomal dominant cancer predisposition syndrome characterized by melanotic macules and hamartomatous polyps. Small-bowel surveillance in the pediatric PJS population is not designed to identify small-bowel malignancy, which is thought to arise in adulthood. A...
Population pharmacokinetics of doxorubicin: establishment of a NONMEM model for adults and children older than 3 years.
Cancer Chemotherapy and Pharmacology 71(3):749 (2013)
The aim of the current investigation was to develop a population pharmacokinetic model for doxorubicin and doxorubicinol that could provide improved estimated values for the pharmacokinetic parameters clearance of doxorubicin, volume of distribution of the central compartment, clearance of doxor...
A phase I trial of vorinostat and bortezomib in children with refractory or recurrent solid tumors: a Children's Oncology Group phase I consortium study (ADVL0916).
Pediatric Blood & Cancer 60(3):390 (2013)
A pediatric Phase I trial was performed to determine the maximum-tolerated dose, dose-limiting toxicities (DLTs), and pharmacokinetics (PK) of vorinostat and bortezomib, in patients with solid tumors.
Oral vorinostat was administered on days 1-5 and 8-12 of a 21-day cycle (starting dose 180 mg/m...
Renal and hepatic tumors in the neonatal period.
Seminars in Fetal and Neonatal Medicine 17(4):216 (2012)
Renal and hepatic tumors in neonates are extremely rare. Nevertheless it is important for clinicians to be familiar with them. Both renal and hepatic neonatal tumors are heterogeneous collections of several tumor types. Some renal and hepatic tumors are benign and may require no interventions wh...
Vascular-targeted photothermal therapy of an orthotopic murine glioma model.
Nanomedicine 7(8):1133 (2012)
To develop nanoshells for vascular-targeted photothermal therapy of glioma.
The ability of nanoshells conjugated to VEGF and/or poly(ethylene glycol) (PEG) to thermally ablate VEGF receptor-2-positive endothelial cells upon near-infrared laser irradiation was evaluated in vitro. Subsequent in vi...
Mutations of PTCH1, MLL2, and MLL3 are not frequent events in hepatoblastoma.
Pediatric Blood & Cancer 58(6):1006 (2012)
Targeted therapy in bone and soft tissue sarcoma in children and adolescents.
Current Oncology Reports 14(2):197 (2012)
Pediatric soft-tissue and bone sarcomas are a heterogeneous group of tumors of mesenchymal origin which affect approximately 1,500 children in the United States each year. Using multimodal therapy (surgery, radiation, and chemotherapy),the overall 5-year survival rate for children with soft-tiss...
Nanoshell-mediated photothermal therapy improves survival in a murine glioma model.
Journal of Neuro-Oncology 104(1):55 (2011)
We are developing a novel treatment for high-grade gliomas using near infrared-absorbing silica-gold nanoshells that are thermally activated upon exposure to a near infrared laser, thereby irreversibly damaging cancerous cells. The goal of this work was to determine the efficacy of nanoshell-med...
Plasma and cerebrospinal fluid pharmacokinetics of ABT-888 after oral administration in non-human primates.
Cancer Chemotherapy and Pharmacology 65(3):419 (2010)
ABT-888 inhibits poly(ADP-ribose) polymerase (PARP) and may enhance the efficacy of chemotherapy and radiation in CNS tumors. We studied the plasma and cerebrospinal fluid (CSF) pharmacokinetics (PK) of ABT-888 in a non-human primate (NHP) model that is highly predictive of human CSF penetration...
Impact of body composition on pharmacokinetics of doxorubicin in children: a Glaser Pediatric Research Network study.
Cancer Chemotherapy and Pharmacology 64(2):243 (2009)
We studied the relationship between doxorubicin pharmacokinetics and body composition in children with cancer.
Children between 1 and 21 years of age, receiving doxorubicin as an infusion of any duration <24 h on either a 1-day or 2-day schedule were eligible if they had no significant abnormali...
Severe neurologic side effects in patients being treated for hemophagocytic lymphohistiocytosis.
Pediatric Blood & Cancer 52(5):621 (2009)
Hemophagocytic Lymphohistiocytosis (HLH) is characterized by uncontrolled inflammation that is generally fatal without immune modulating chemotherapy. At Texas Children's Hospital, we have observed significant central nervous system (CNS) toxicity in several patients treated for HLH according to...