Abstract:
:A 4-year-old male presented with rapid-onset cranial nerve palsy and ataxia. Brain magnetic resonance imaging (MRI) revealed a pontine mass lesion with discordant conventional and advanced imaging. A stereotactic core biopsy revealed glioblastoma with immunostaining suggestive of histone H3K27M and TP53 mutation, consistent with diffuse intrinsic pontine glioma. MRI 3 months after radiotherapy revealed extensive new leptomeningeal metastatic disease involving both the supra- and infratentorial brain, as well as the imaged portion of the spine. Tissue procured at the time of needle biopsy has undergone striking in vivo expansion as an orthotopic xenograft.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Tinkle CL,Orr BA,Lucas JT Jr,Klimo P,Patay Z,Baker SJ,Broniscer A,Qaddoumi Idoi
10.1002/pbc.26416subject
Has Abstractpub_date
2017-08-01 00:00:00issue
8eissn
1545-5009issn
1545-5017journal_volume
64pub_type
杂志文章abstract:PURPOSE:We conducted a pediatric phase I study to estimate the maximum tolerated dose (MTD), dose-limiting toxicities (DLT), and pharmacokinetic properties of vorinostat, a histone deacetylase (HDAC) inhibitor, when given in combination with temozolomide in children with refractory or recurrent CNS malignancies. PATIE...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.24541
更新日期:2013-09-01 00:00:00
abstract::Primary choriocarcinoma of the liver is an extremely rare childhood malignancy frequently associated with clinical instability at initial presentation. It often mimics other benign and malignant childhood liver tumors. Prompt diagnosis and initiation of treatment are necessary to attain a successful outcome. We descri...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20623
更新日期:2007-07-01 00:00:00
abstract:BACKGROUND:Hepatic veno-occlusive disease (VOD) is one of the most serious complications in stem cell transplantation (SCT). Although plasma protein C activity decreases in VOD after SCT, the timeframe of plasma protein C activity decreases during SCT is not known. PROCEDURE:We examined levels of plasma protein C seri...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22314
更新日期:2010-03-01 00:00:00
abstract:BACKGROUND:This study aimed to compare the health related quality of life (HRQoL) of children and adolescents after malignant bone tumor surgery of the leg with healthy controls. PROCEDURE:Patients between 8 and 25 years old were cross-sectional recruited. Patients under 16 years of age received the TNO (Netherlands O...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.22439
更新日期:2010-05-01 00:00:00
abstract:PURPOSE:To assess local control, event-free survival (EFS), and overall survival (OS) rates in 71 patients with localized, completely resected (Group I) alveolar rhabdomyosarcoma (ALV RMS) and their relation to radiation therapy (RT) on IRSG Protocols III and IV, 1984-1997. METHODS:Chart review and standard statistica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22520
更新日期:2010-10-01 00:00:00
abstract::Malignant degeneration frequently arises from preexisting plexiform neurofibroma in patients with neurofibromatosis type 1 (NF1). Image guided biopsy for diagnostic purposes, such as with CT guidance, can be technically challenging in these patients, as CT cannot distinguish malignant from benign areas within the same...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24668
更新日期:2013-12-01 00:00:00
abstract::Treatment with the nucleoside analog cytarabine has been shown to mimic changes in gene expression associated with downregulation of the EWS-FLI1 oncogene in Ewing sarcoma cell lines, selectively inhibit their growth in vitro, and cause tumor regression in athymic nude mice. For this report cytarabine was studied in v...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22355
更新日期:2010-12-01 00:00:00
abstract:BACKGROUND:The aim of this study is to evaluate the relationship between the latency to diagnosis (LD) and the time to completion of chemotherapy (TCC) with clinical outcomes in children with osteosarcoma. METHODS:We performed a retrospective analysis of all patients who received treatment for osteosarcoma in two tert...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.28180
更新日期:2020-04-01 00:00:00
abstract:BACKGROUND:Most patients with stage IV Wilms tumor (WT) and pulmonary metastases are treated with surgery, local radiotherapy (RT), and whole-lung irradiation (WLI). The Children's Oncology Group is studying whether WLI should only be given if metastatic lung lesions persist following induction chemotherapy. We hypothe...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.25007
更新日期:2014-08-01 00:00:00
abstract:BACKGROUND:Sickle cell disease (SCD) is an inherited blood disorder characterized by a chronic hemolytic anemia that can contribute to fatigue and global cognitive impairment in patients. The study objective was to report on the feasibility, reliability, and validity of the PedsQL™ Multidimensional Fatigue Scale in SCD...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.24776
更新日期:2014-01-01 00:00:00
abstract:BACKGROUND:Dexamethasone is more efficacious than prednisone in the treatment of acute lymphoblastic leukemia (ALL), but has also been associated with greater toxicity. We compared neuropsychological outcomes for patients treated on DFCI ALL Consortium Protocol 00-01, which included a randomized comparison of the two s...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.24666
更新日期:2013-11-01 00:00:00
abstract::Immunotherapy with the anti-GD2 monoclonal antibody ch14.18, or dinutuximab, represents an important therapeutic advance in the treatment of pediatric high-risk neuroblastoma and is now considered part of standard of care in this patient population. To date, transverse myelitis as a result of dinutuximab therapy has n...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26732
更新日期:2018-01-01 00:00:00
abstract::To characterize radiation necrosis following hypofractionated brainstem re-irradiation in pediatric patients, we reviewed 23 cases with 28 tumors invading or abutting brainstem and treated with hypofractionated re-irradiation from 2004 to 2014. Re-irradiation delivered total doses of 16-30 Gy in two to five fractions....
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26341
更新日期:2017-05-01 00:00:00
abstract:BACKGROUND:Juvenile xanthogranuloma (JXG) is the most common non-Langerhans cell histiocytosis in children. The mortality and morbidity of JXG with extracutaneous lesions remain unclear. METHODS:Data of patients aged < 18 years who were diagnosed with JXG between 2001 and 2010 were retrospectively collected through a ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28381
更新日期:2020-07-01 00:00:00
abstract::Hepatoblastoma (HB) and biliary atresia (BA) are both rare conditions that occurred in the patient described. This is the second such case in the literature. An explanation for this apparent coincidence could possibly be found in the existance of pluripotent liver stem cells. In humans, small epithelial cells (SEC) be...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20115
更新日期:2004-10-01 00:00:00
abstract:BACKGROUND:The use of carboplatin for the treatment of pediatric low grade gliomas (PLGG) is often limited by the development of carboplatin hypersensitivity. Reported rates of carboplatin hypersensitivity reactions vary between 6% and 32% in these patients. Here we report the frequency of carboplatin hypersensitivity ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25686
更新日期:2016-01-01 00:00:00
abstract::Here, we present the case of a pediatric patient with newly diagnosed hepatocellular carcinoma causing central biliary obstruction and persistently elevated bilirubin of 3.0-4.3 mg/dl despite placement of bilateral internal-external biliary drains. The tumor was not resectable, and the patient was not a candidate for ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26330
更新日期:2017-06-01 00:00:00
abstract:BACKGROUND:The outcome for patients with Ewing sarcoma family of tumors (ESFTs) of bone with metastases at diagnosis remains poor despite new approaches to treatment. We evaluated whether a dose-intensity chemotherapy regimen improved survival for patients with ESFTs of bone with metastases at diagnosis. METHODS:We en...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.21233
更新日期:2007-12-01 00:00:00
abstract:BACKGROUND:The Wnt/β-catenin pathway plays a central role in the pathogenesis of most hepatoblastomas (HBs), that is, up to 60-80% carry activating CTNNB1 mutations. HBs can however also be the first manifestation of familial adenomatous polyposis (FAP). As this is a severe disease, it is important for the patient and ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26991
更新日期:2018-06-01 00:00:00
abstract:BACKGROUND:Cancer diagnoses pose challenges to adolescents' and young adults' (AYA) physical, mental, and emotional health, and developmental tasks. In order for AYAs, caregivers, clinicians, and other collaborators to optimize health outcomes (coproduction of health), understanding what living well means for AYAs who ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.28599
更新日期:2020-10-01 00:00:00
abstract::Osteoporosis and osteopenia are long-term side effects of bone marrow transplant (BMT). The purpose of this study was to determine the prevalence of bone mineral density (BMD) abnormalities in pediatric patients prior to BMT. Forty-four pediatric patients were evaluated with DEXA scans. The average Z-score was -0.37. ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22102
更新日期:2009-10-01 00:00:00
abstract:BACKGROUND:Fever in severe chemotherapy-induced neutropenia (FN) is the most frequent manifestation of a potentially lethal complication of current intensive chemotherapy regimens. This study aimed at establishing models predicting the risk of FN, and of FN with bacteremia, in pediatric cancer patients. METHODS:In a s...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21726
更新日期:2008-12-01 00:00:00
abstract::Opportunities for cancer prevention in adolescents range from limitation of sun exposure to the use of human papillomavirus vaccines. Those who develop malignant disease experience longer waiting times for diagnosis and treatment than do children, especially when referred to adult treatment centers, and they are less ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20634
更新日期:2006-03-01 00:00:00
abstract::The BRAF V600E missense mutation is known to be present in a subset of central nervous system tumors. We report a patient with a BRAF V600E mutated pilomyxoid astrocytoma who failed multiple conventional chemotherapy regimens. Treatment with vemurafenib, a molecularly targeted therapy against the mutant BRAF V600E kin...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25084
更新日期:2014-11-01 00:00:00
abstract:BACKGROUND:Sorafenib is an inhibitor of multiple kinases (e.g., VEGF receptors, PDGFR, FLT3, RET, BRAF, KIT) and is approved by FDA for treatment of two adult cancers. The activity of sorafenib was evaluated against the PPTP's in vitro and in vivo panels. PROCEDURES:Sorafenib was evaluated against the PPTP in vitro pa...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22712
更新日期:2010-12-01 00:00:00
abstract:BACKGROUND:Pneumocystis jirovecii, formerly carinii, pneumonia (PCP) poses a life-threatening risk to oncology patients. The use of trimethoprim-sulfamethoxazole (TMP-SMZ) prophylaxis virtually eliminates the risk of infection; however, many patients cannot tolerate TMP-SMZ. We performed a retrospective analysis to det...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21287
更新日期:2008-04-01 00:00:00
abstract:BACKGROUND:In malaria-endemic countries in West Africa, sickle cell disease (SCD) contributes to childhood mortality. Historically, Liberia had regions wherein hemoglobin S and beta-thalassemia trait were mutually exclusive. Data on hemoglobinopathies in the Monrovia, the capital, are outdated and do not reflect urban ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25875
更新日期:2016-04-01 00:00:00
abstract:BACKGROUND:Ewing tumors are the most frequent malignant tumors of the chest wall in children and young adults. Surgical management of these tumors can be challenging. Optimal local control remains controversial. The aim of this study was to analyze treatment, outcome, and surgical procedures in patients with thoracic t...
journal_title:Pediatric blood & cancer
pub_type: 更正并重新发布的文章,杂志文章
doi:10.1002/pbc.27884
更新日期:2019-08-01 00:00:00
abstract:BACKGROUND/OBJECTIVES:Sickle cell disease (SCD) is an important, hidden cause of childhood mortality worldwide. It is most prevalent in sub-Saharan Africa where national newborn screening programs remain unavailable and most children in rural areas are never diagnosed. We conducted a study at a rural district hospital ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28620
更新日期:2020-11-01 00:00:00
abstract:BACKGROUND:The Paediatric Oncology Centres (POCs) treating childhood cancer in South East England produce unified supportive care guidelines for use in the secondary pediatric (shared care) units. This study evaluated the adherence to current guidelines for febrile neutropenia (FN) and documented outcome in terms of ba...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21041
更新日期:2007-10-15 00:00:00