Abstract:
BACKGROUND:Atypical teratoid/rhabdoid tumor (AT/RT) is a highly malignant embryonal brain tumor that occurs mainly in early childhood. Although most of the tumors are characterized by inactivating mutations of the tumor suppressor gene, SMARCB1, the biological basis of its tumorigenesis and aggressiveness is still unknown. PROCEDURE:We performed high-throughput copy number variation analysis of primary cell lines generated from primary and relapsed tumors from one of our patients to identify new genes involved in AT/RT biology. The expression of the identified gene was validated in 29 AT/RT samples by gene expression profiling, quantitative real-time polymerase chain reaction, and immunohistochemistry (IHC). Furthermore, we investigated the function of this gene by mutating it in rhabdoid tumor cells. RESULTS:TEAD4 amplification was detected in the primary cell lines and its overexpression was confirmed at mRNA and protein levels in an independent cohort of AT/RT samples. TEAD4's co-activator, YAP1, and the downstream targets, MYC and CCND1, were also found to be upregulated in AT/RT when compared to medulloblastoma. IHC showed TEAD4 and YAP1 overexpression in all samples. Cell proliferation and migration were significantly reduced in TEAD4-mutated cells. CONCLUSIONS:We report the overexpression of TEAD4 in AT/RT, which is a key component of Hippo pathway. Recent reports revealed that dysregulation of the Hippo pathway is implicated in tumorigenesis and poor prognosis of several human cancers. Our results suggest that TEAD4 plays a role in the pathophysiology of AT/RT, which represents a new insight into the biology of this aggressive tumor.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Suzuki M,Kondo A,Ogino I,Arai H,Tomita T,Sredni STdoi
10.1002/pbc.26398subject
Has Abstractpub_date
2017-07-01 00:00:00issue
7eissn
1545-5009issn
1545-5017journal_volume
64pub_type
杂志文章abstract:BACKGROUND:Pheochromocytomas (PCs) are neuroendocrine tumors arising from the chromaffin cells of the adrenal gland, and paragangliomas (PGLs) are their extra-adrenal counterparts arising from ganglia along the sympathetic/parasympathetic chain. Surgery is the cornerstone of treatment. A sporatic or inherited germline ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.28332
更新日期:2020-08-01 00:00:00
abstract::A newborn with unresectable kaposiform hemangioendothelioma associated with Kasabach Merritt phenomenon, unresponsive to vincristine and prednisone, received second-line treatment with propranolol at a dose of 2 mg/kg/day, starting at 2 months of life and continued for 13 months. There was only slight reduction in tum...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25979
更新日期:2016-07-01 00:00:00
abstract:BACKGROUND:Sickle cell anaemia (SCA) is the leading genetic disorder in Nigeria. Elevated velocities ≥170 cm/sec occur in about a third of Nigerian children with SCA. Chronic blood transfusion for stroke prevention is faced with a myriad of challenges in our practice. OBJECTIVES:To evaluate the effectiveness of hydrox...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25529
更新日期:2015-09-01 00:00:00
abstract:OBJECTIVE:The objective of this single-center observational study was to determine the clinical and hematologic responses to intravenous ferric carboxymaltose (FCM) in a cohort of pediatric patients with poor response to oral iron therapy. The occurrence of adverse events was systematically recorded for up to 96 hours ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28614
更新日期:2020-10-01 00:00:00
abstract::Antibody therapy has become standard of care for adult B cell lymphoma patients. It is a potentially less toxic and more targeted approach for lymphoma therapy and should therefore be applied to treat pediatric B cell lymphoma patients as well. In pediatric lymphoma patients, however, clinical experience with monoclon...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.25193
更新日期:2014-12-01 00:00:00
abstract::Survivors of childhood cancer are at risk for dental late effects. This systematic review summarizes associations between treatment exposures and dental late effects among survivors of childhood cancer. We included investigations with at least 20 study participants conducted for 2 or more years after completion of chi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24842
更新日期:2014-03-01 00:00:00
abstract::Congenital amegakaryocytic thrombocytopenia (CAMT) is characterised by neonatal thrombocytopenia, with reduced or absent bone marrow megakaryocytes, leading eventually to pancytopenia. The mean age for progression to bone marrow failure is four years, with the earliest reported being six months. We describe a CAMT pat...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24566
更新日期:2013-09-01 00:00:00
abstract::Acute splenic sequestration crisis (ASSC) is a hematological emergency in young children with sickle cell disease (SCD), characterized by worsening anemia and splenomegaly, usually with reticulocytosis and thrombocytopenia. Transient aplastic crisis (TAC) due to parvovirus B19 infection occurs in older children with S...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22035
更新日期:2009-09-01 00:00:00
abstract::Juvenile myelomonocytic leukemia (JMML) is a rare myelodysplastic/myeloproliferative disorder of early childhood characterized by mutations of the RAS-RAF-MAP kinase signaling pathway. We report the case of a child with a diagnosis of JMML carrying two mutations of NRAS gene (c.37G>C and c.38G>A) independently occurri...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23401
更新日期:2012-09-01 00:00:00
abstract::It is unclear if dosing intervals for Erwinase can be extended with intramuscular (i.m.) versus intravenous (i.v.) dosing. Children with acute lymphoblastic leukemia received Erwinase at 30 000-42 000 IU/m2 i.v. or i.m. I.m. Erwinase (n = 22) achieved activity above 0.1 IU/mL for longer than i.v. Erwinase (n = 33) (3....
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28244
更新日期:2020-07-01 00:00:00
abstract::We report successful bone marrow transplantation in an 11-year-old male with chronic myeloid leukemia from his HLA-identical sibling selected by preimplantation HLA testing. Because collection of cord blood failed, the transplantation was performed when the donor reached the age of 19 months, and sufficient bone marro...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23007
更新日期:2011-08-01 00:00:00
abstract::Beckwith-Wiedemann syndrome (BWS) is a genetic overgrowth and cancer predisposition syndrome, associated with both benign and malignant adrenal findings. Literature review and an institutional case series elucidate the wide spectrum of adrenal findings in BWS patients. The altered expression of the 11p15 region is lik...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.26432
更新日期:2017-08-01 00:00:00
abstract:BACKGROUND:Cancer among infants (<1 year old) has unique epidemiologic, clinical, and genetic characteristics compared with cancer in older children. Nonetheless, data on secular trends in infant cancer incidence and survival in the United States is sparse. METHODS:Population-based data from nine areas of the Surveill...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28917
更新日期:2021-01-20 00:00:00
abstract:BACKGROUND:Patients with Langerhans cell histiocytosis (LCH) may develop neurodegeneration and significant CNS sequelae, affecting a significant proportion of the patients. We here aimed to investigate the neuropsychological consequences in more detail. METHODS:Using an extensive neuropsychological test battery, we ev...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21656
更新日期:2008-11-01 00:00:00
abstract::Severe congenital neutropenia (SCN) is a bone marrow failure disease with an autosomal dominant inheritance from mutations in ELANE. Here, we report a 7-week-old Korean male with SCN. His elder sister died from pneumonia at 2 years. Direct sequencing of ELANE in the proband identified a heterozygous novel frameshift m...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25654
更新日期:2015-12-01 00:00:00
abstract:BACKGROUND:Recent studies suggest that SLCO1B1 c.521T > C variant decreases the clearance of methotrexate (MTX) and elevates its plasma concentration, hence leucovorin doses may need to be adjusted. However, high leucovorin doses may affect the cure rate in childhood acute lymphoblastic leukemia (ALL). Hitherto neither...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25191
更新日期:2014-12-01 00:00:00
abstract:BACKGROUND:Worldwide, hemoglobinopathies affect millions of children. Identification of hemoglobin disorders in most sub-Saharan African countries is delayed until clinical signs of the disease are present. Limited studies have been conducted to understand their prevalence and clinical presentation among newborns in re...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.26735
更新日期:2018-01-01 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:One of the most important adverse prognostic factors for adult renal cell carcinoma (RCC) is the retroperitoneal lymph node involvement. The aim of this article is to study the prognostic significance of local lymph node involvement in pediatric RCC and the role of retroperitoneal lymph node dissection (RLND...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21652
更新日期:2008-10-01 00:00:00
abstract:BACKGROUND:Morphological evaluation of early response to chemotherapy and measurement of minimal residual disease by flow cytometry or PCR are being used for evaluation of prognosis and treatment stratification in children with acute lymphoblastic leukaemia (ALL). PROCEDURE:In a series of 14 consecutive bone marrow in...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23081
更新日期:2011-08-01 00:00:00
abstract:BACKGROUND:Children with sickle cell disease (SCD) and abnormal transcranial Doppler (TCD) ultrasonography have a high risk of stroke, but this risk is greatly reduced when chronic transfusion therapy is administered. The change in TCD velocities during chronic transfusion therapy and rate and frequency of normalizatio...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22951
更新日期:2011-05-01 00:00:00
abstract::With improvements in therapy for childhood cancer, the expectation that most childhood cancer patients will survive and enter adulthood is a reality. There is clear evidence that survivors are at risk for adverse health-related long-term sequelae associated with their cancer and its treatment, requiring appropriate he...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20611
更新日期:2006-02-01 00:00:00
abstract::Angiotensin converting enzyme (ACE) gene insertion(I)/deletion(D) polymorphism influences the outcome of a number of cardiovascular diseases. ACE I/D polymorphism was investigated by PCR in 207 pediatric cancer patients and 144 controls. ACE I/D distribution of patients and controls was similar. The frequency of the D...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20400
更新日期:2005-08-01 00:00:00
abstract:BACKGROUND:Cancer patients undergoing chemotherapy often develop anemia, which can increase the risk for transfusions and fatigue. The recombinant erythropoiesis-stimulating agent darbepoetin alfa can effectively treat chemotherapy-induced anemia (CIA) in adults, but limited data are available regarding its use in pedi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21079
更新日期:2007-10-15 00:00:00
abstract:BACKGROUND:PG11047 is a novel conformationally restricted analog of the natural polyamine, spermine that lowers cellular endogenous polyamine levels and competitively inhibits natural polyamine functions leading to cancer cell growth inhibition. The activity of PG11047 was evaluated against the PPTP's in vitro and in v...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22797
更新日期:2011-08-01 00:00:00
abstract:BACKGROUND:Outcomes for patients with oncologic disease and/or after hematopoietic stem cell transplant (HSCT) requiring intensive care unit admission have improved, but indications for and outcomes after extracorporeal membrane oxygenation (ECMO) support in this population are poorly characterized. PROCEDURE:We analy...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.28403
更新日期:2020-10-01 00:00:00
abstract:BACKGROUND:Degenerative-like neuro-Langerhans cell histiocytosis (DN-LCH) is a rare complication of LCH marked by progressive cerebellar ataxia. No treatment has so far been shown to slow this progression. PROCEDURE:All-trans retinoic acid (ATRA) was administered orally at a dose of 45 mg/m(2) daily for 6 weeks and th...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.20040
更新日期:2004-07-01 00:00:00
abstract:BACKGROUND:Central nervous system irradiation (CNS-RT) has played a central role in the cure of acute lymphoblastic leukemia (ALL), but due to the risk of long-term toxicity, it is now considered a less-favorable method of CNS-directed therapy. PROCEDURES:Retrospectively, we estimated the effect of CNS involvement and...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26191
更新日期:2017-02-01 00:00:00
abstract:BACKGROUND:Iron overload is well documented in patients with β-thalassemia major, and patients who have undergone hematopoietic stem cell transplantation (HSCT) remain at risk as a result of pre- and immediate post-HSCT transfusions. PROCEDURE:This is a prospective, randomized, 1-year clinical trial that compares the ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.26213
更新日期:2017-01-01 00:00:00
abstract::Under and overweight, usually secondary to energy imbalance, are common complications of childhood chronic disease. A simple energy balance paradigm has been helpful in understanding the etiology of underweight and overweight in chronic disease, including malignant disease, particularly when measurements of total ener...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.21412
更新日期:2008-02-01 00:00:00