Abstract:
:Embryonal rhabdomyosarcoma (ERMS) is the most common childhood sarcoma and is a component of the familial pleuropulmonary blastoma (PPB)-predisposition syndrome. Using the PPB model, we hypothesized that DICER1 mutations would be found in familial and sporadic forms of ERMS. Blood samples from four children with familial PPB and ERMS, and 52 sporadic ERMS tumors were tested for DICER1 mutations. Germline DICER1 mutations were found in all four patients with familial PPB and 2 of 52 (3.8%) sporadic ERMS had somatic mutations. Our findings confirm the pathogenetic relationship between ERMS and PPB suggesting that ERMS may result from abnormal miRNA regulation.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Doros L,Yang J,Dehner L,Rossi CT,Skiver K,Jarzembowski JA,Messinger Y,Schultz KA,Williams G,André N,Hill DAdoi
10.1002/pbc.24020subject
Has Abstractpub_date
2012-09-01 00:00:00pages
558-60issue
3eissn
1545-5009issn
1545-5017journal_volume
59pub_type
杂志文章abstract:BACKGROUND:Numerous recent reports have described the use of recombinant factor VIIa (rFVIIa) in non-hemophilia bleeding situations for achievement of hemostasis. However, its use in clinical situations other than hemophilia patients with inhibitors has been complicated by some reports of thrombotic events. rFVIIa has ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.22160
更新日期:2009-12-01 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::A 12-year-old boy in third remission of an acute lymphoblastic leukaemia developed infection of lung and paranasal sinuses with Aspergillus flavus in neutropenia. Because of the high risk of leukaemia-relapse bone marrow transplantation (BMT) from a matched unrelated donor was carried out despite invasive pulmonary as...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20302
更新日期:2005-06-15 00:00:00
abstract:BACKGROUND:Little is known about the effects of chemotherapy on patient antibody titers to vaccine-preventable infectious diseases; thus, there is no standard protocol for revaccinating post-chemotherapy patients. PROCEDURES:To assess losses of detectable antibody titers due to chemotherapy, we retrospectively examine...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21277
更新日期:2007-10-15 00:00:00
abstract:BACKGROUND:Robatumumab (19D12; MK-7454 otherwise known as SCH717454) is a fully human antibody that binds to and inhibits insulin-like growth factor receptor-1 (IGF-1R). This multiinstitutional study (P04720) determined the safety and clinical efficacy of robatumumab in three separate patient groups with resectable ost...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.26087
更新日期:2016-10-01 00:00:00
abstract:BACKGROUND:(131) I-metaiodobenzylguanidine ((131) I-MIBG) is a targeted radiopharmaceutical for patients with neuroblastoma. Despite its tumor-specific uptake, the treatment with (131) I-MIBG results in whole-body radiation exposure. Our aim was to correlate whole-body radiation dose (WBD) from (131) I-MIBG with tumor ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25816
更新日期:2016-03-01 00:00:00
abstract:BACKGROUND:MOPP (mechlorethamine, vincristine, procarbazine, prednisone) and ABVD (doxorubicin, bleomycin, vinblastine, and dacarbazine) are effective therapies for Hodgkin disease (HD) that may cause long-term toxicities in children. APE (cytosine arabinoside, cisplatin, etoposide) is a non-cross-resistant regimen wit...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20563
更新日期:2006-03-01 00:00:00
abstract::Clinically apparent jaundice is unusual in patients with beta-thalassemia major. Co-inheritance of Gilbert syndrome has been reported to cause hyperbilirubinemia in these subjects. Crigler-Najjar syndrome is another rare disorder of bilirubin metabolism caused by mutation in the gene coding the enzyme UGT1A1. We repor...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22313
更新日期:2010-04-01 00:00:00
abstract::We report a case of recurrent squamous cell carcinoma (SCC) of the scalp with deep cerebral invasion in a 15-year-old girl. Plain films and CT showed extensive, full thickness, and skull destruction at the vertex. Gadolinium-enhanced MRI revealed neoplastic invasion of the meninges and both cerebral hemispheres down t...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20427
更新日期:2005-12-01 00:00:00
abstract:AIM:Osteopontin (OPN) has been investigated as a biomarker for cancer and nonmalignant diseases during the last decades. Data about OPN as a potential biomarker in childhood diseases are still sparse, and reference values are not available in children. We aimed to establish reference values for children from birth to y...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28272
更新日期:2020-07-01 00:00:00
abstract:BACKGROUND:Kaposi sarcoma (KS) is the most common HIV-associated malignancy in sub-Saharan Africa. The presentation and outcomes of pediatric KS are not well understood. PROCEDURE:We performed a retrospective cohort analysis of 81 HIV-infected children with KS at the Baylor Children's Clinical Centres of Excellence in...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.24516
更新日期:2013-08-01 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:BACKGROUND:The purpose of this study was to evaluate a reduced irradiation dose strategy for newly diagnosed primary central nervous system (CNS) germinomas. METHODS:Twenty patients with histologically diagnosed localized pure germinoma (n = 19) or germinoma with a mature teratoma component (n = 1) received four cycle...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22468
更新日期:2010-07-15 00:00:00
abstract:BACKGROUND:Children with cancer may be at an increased risk of infection with hepatitis B virus (HBV) when levels of hepatitis B antibodies are reduced owing to chemotherapy-induced immunosuppression. This study evaluated the changes in HBV antibody status and HBV infections after chemotherapy in children with acute ly...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27904
更新日期:2019-12-01 00:00:00
abstract:BACKGROUND:Soft tissue sarcomas (STS) are a heterogeneous group of mesenchymal malignancies that occur throughout the lifespan. The impact of age on disease features and outcome is unclear. METHODS:We analyzed the clinical features and outcome of all STS cases registered between 1973 and 2006 in the SEER database. RE...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23252
更新日期:2011-12-01 00:00:00
abstract:BACKGROUND:The aim of this study was to evaluate the value of follow-up investigations of T-cell acute lymphoblastic leukemia (T-ALL) and T-cell non-Hodgkin's lymphoma (T-NHL), including cerebrospinal fluid (CSF) examination, bone marrow (BM) aspiration, peripheral blood (PB) count, serum lactate dehydrogenase (LDH) an...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20813
更新日期:2007-04-01 00:00:00
abstract:OBJECTIVE:To examine the level of children's understanding of informed consent in clinical trials and factors that may influence these processes. DESIGN:Twenty nine children who were included in clinical trials for treatment of cancer or HIV, were offered the possibility to complete a semidirective interview, with par...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.21359
更新日期:2008-05-01 00:00:00
abstract:BACKGROUND:In the pediatric population, pathologic bleeding is often challenging to identify. The pediatric bleeding questionnaire (PBQ) was developed as a screening tool for von Willebrand disease (VWD) but was designed to be self-completed by children above 12 years of age. The study objective was to determine whethe...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27709
更新日期:2019-06-01 00:00:00
abstract::Pediatric renal cell carcinoma (RCC) is a rare cancer that can be associated with inherited diseases including tuberous sclerosis complex (TSC) caused by germline mutations in TSC1 or TSC2. Somatic mutations in TSC1 and TSC2 have also been reported in adult RCC, which predict response to mTOR inhibitors. Here, we pres...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26286
更新日期:2017-05-01 00:00:00
abstract:BACKGROUND:Silent cerebral infarcts are both morbid and progressive in children with sickle cell disease (SCD). While blood transfusion therapy is effective primary and secondary stroke prevention, the efficacy and acceptance of blood transfusion therapy for children with silent cerebral infarcts is unknown. The overal...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.21338
更新日期:2008-03-01 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::High-risk (HR) acute lymphoblastic leukemia (ALL) remains one of the greatest challenges in pediatric oncology. Relapsed ALL is a leading cause of death in young people, and further improvements in outcome will required the development of therapeutic approaches directed against rational therapeutic targets, as escalat...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.22996
更新日期:2011-06-01 00:00:00
abstract:BACKGROUND:Methylenetetrahydrofolate reductase (MTHFR) C677T and A1298C polymorphisms have been implicated in childhood acute lymphoblastic leukemia (ALL) risk, but previously published studies were inconsistent and recent meta-analyses were not adequate. PROCEDURES:In a meta-analysis of 21 publications with 4,706 cas...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,meta分析,评审
doi:10.1002/pbc.23137
更新日期:2012-04-01 00:00:00
abstract:BACKGROUND:Based on the presumed clinical similarity between the two most severe sickle cell disease (SCD) genotypes, hemoglobin (Hb) Sβ0 thalassemia and HbSS, randomized controlled trials (RCTs) have included both genotypes. Our group has demonstrated that healthcare providers inadequately distinguish the two diagnose...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27352
更新日期:2018-11-01 00:00:00
abstract:BACKGROUND:Children less than 5 years of age with malignant central nervous system (CNS) tumors, continue to have a high rate of morbidity and mortality following administration of conventional therapy. In an attempt to avoid the neurologic sequelae associated with craniospinal radiation, strategies such as high-dose c...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21381
更新日期:2008-05-01 00:00:00
abstract:BACKGROUND:Deaths during induction chemotherapy for pediatric acute lymphoblastic leukemia (ALL) account for one-tenth of ALL-associated mortality and half of ALL treatment-related mortality. We sought to ascertain patient- and hospital-level factors associated with induction mortality. PROCEDURE:We performed a retros...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24855
更新日期:2014-05-01 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: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:BACKGROUND:Anthracycline agents are used for treatment of acute myeloid leukemia (AML) but may cause late-onset cardiomyopathy. Current frontline therapy for AML in North America, as reflected in the approach of the Children's Oncology Group (COG) and other pediatric consortia, is adapted from the anthracyline-intensiv...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.24498
更新日期:2013-09-01 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