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 kinase, combined with vinblastine resulted in tumor regression. Furthermore, this patient experienced almost immediate progression of disease after holding vemurafenib for only 2-3 weeks, suggesting that the tumor response is vemurafenib dependent. This population of patients may benefit from targeted therapy and testing of individual tumors for BRAF mutations is justified.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Skrypek M,Foreman N,Guillaume D,Moertel Cdoi
10.1002/pbc.25084subject
Has Abstractpub_date
2014-11-01 00:00:00pages
2099-100issue
11eissn
1545-5009issn
1545-5017journal_volume
61pub_type
杂志文章abstract::Time-to-antibiotic administration (TTA) has been suggested as a quality-of-care (QOC) measure for pediatric oncology patients with febrile neutropenia (FN). Unknown, however, is to what extent pediatric oncology centers utilize TTA. Therefore, we designed and administered an electronic survey (68% response rate) of pr...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23148
更新日期:2012-02-01 00:00:00
abstract::Torsades de Pointes (TdP) is a life-threatening ventricular arrhythmia that can be associated with metabolic abnormalities, exposure to arrhythmogenic medications, and congenital long-QT syndrome. This report describes a patient with ALL and multiple complications of therapy who developed TdP. The patient had no evide...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20712
更新日期:2007-12-01 00:00:00
abstract::The molecular detection of minimal residual disease (MRD) is standard of care in acute lymphoblastic leukemia to personalize the stratification of patients to appropriate intensity chemotherapy regimens. High-throughput sequencing (HTS) techniques are driving changes to MRD methodologies. Our study demonstrates HTS ca...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27787
更新日期:2019-08-01 00:00:00
abstract:BACKGROUND:Severe pandemic influenza A (H1N1) infection can lead to acute respiratory failure (ARF) with associated high mortality. Children with malignancy may be at higher risk of H1N1-associated ARF because of underlying primary disease or immunosuppression associated with chemotherapy. PROCEDURE:We describe the cl...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22973
更新日期:2011-10-01 00:00:00
abstract:BACKGROUND: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 the Histiocy...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21838
更新日期:2009-05-01 00:00:00
abstract:BACKGROUND:Little is known about the clinical significance of coronary artery dilation (CAD) and left ventricular hypertrophy (LVH) in patients with sickle cell disease (SCD). PROCEDURE:In a retrospective cohort, we studied the prevalence of CAD and LVH in 101 children with SCD in comparison to 93 healthy African-Amer...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25239
更新日期:2015-01-01 00:00:00
abstract:BACKGROUND:This study was conducted to evaluate oral health and dental anomalies in children treated for acute lymphoblastic leukemia (ALL) and to compare results with those of a group of healthy children matched for sex and age. PROCEDURE:Fifty-six children treated for ALL and 56 healthy controls were examined for de...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22108
更新日期:2009-09-01 00:00:00
abstract:BACKGROUND:A majority of Fanconi anemia (FA) patients will experience bone marrow failure (BMF) and androgen therapy (most often oxymetholone) may be utilized as a treatment to improve BMF-related cytopenias. However, oxymetholone is associated with toxicities making identification of other agents of interest. In this ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24617
更新日期:2014-01-01 00:00:00
abstract::This study describes hydroxyurea use among children ages 1 to 17 with sickle cell anemia (SCA) enrolled in at least one year of Medicaid in six states from 2005 to 2012. Administrative claims were used to summarize the number of days' supply of hydroxyurea dispensed by state and year. A total of 7963 children with SCA...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27721
更新日期:2019-06-01 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::The provision of tele-practice symptom management is often without the provision of evidence-based guidelines. Under the auspices of the Pediatric Oncology Group of Ontario, a nursing task force was established to appraise the evidence and develop guidelines. Promising new efforts to enhance symptom management through...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,实务指引,评审
doi:10.1002/pbc.22993
更新日期:2011-10-01 00:00:00
abstract::Radiation necrosis is a potentially debilitating side effect of therapy necessary to treat pediatric central nervous system tumors. Clinical signs of cerebral radiation necrosis (CRN) are similar to symptoms of disease progression and require close monitoring. The case of an infant diagnosed with a malignant rhabdoid ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28705
更新日期:2021-01-01 00:00:00
abstract::In Mexico, childhood cancer incidence and mortality have increased in the last decade. Through government actions since 2005, the Popular Medical Insurance (PMI) program for childhood cancer was created. The objective of PMI was to offer early cancer diagnosis, standardized treatment regimens, and numerous pediatric o...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.26366
更新日期:2017-06-01 00:00:00
abstract::Hemophagocytic lymphohistioytosis (HLH) is a severe, life-threatening hyperinflammatory disorder that requires prompt diagnosis and treatment. Approximately, 25-50% of patients with HLH fail to achieve remission with established regimens that include dexamethasone and etoposide, or methylprednisolone and antithymocyte...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.26308
更新日期:2017-04-01 00:00:00
abstract::Epidural spinal cord compression as the initial presentation of acute lymphoblastic leukemia (ALL) is a rare and serious complication. Extramedullary disease is rarely reported in patients with ALL. The most common sites are bone, followed by soft tissue, skin and lymph nodes. We describe a child with common B-lineage...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22682
更新日期:2010-10-01 00:00:00
abstract:BACKGROUND:There are no tests to identify critically ill children at high risk of deep venous thrombosis (DVT). In this exploratory study, we aimed to identify proteins that are associated with incident DVT in critically ill adolescents. PROCEDURE:Plasma samples were obtained from critically ill adolescents within 24 ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.28159
更新日期:2020-04-01 00:00:00
abstract::Sickle cell disease (SCD) is an inherited red blood cell disorder that occurs worldwide and results in significant morbidity and mortality. In addition to affected children who are born in the US, an increasing number of children with SCD are migrating to the US with their families or through international adoption. C...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27000
更新日期:2018-06-01 00:00:00
abstract::Enrollment of patients in sickle cell intervention trials has been challenging due to difficulty in obtaining consent from a legal guardian and lack of collaboration between emergency medicine and hematology. We utilized education and preconsent in a pediatric multisite sickle cell intervention trial to overcome these...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.26013
更新日期:2016-09-01 00:00:00
abstract::Malignant mesothelioma (MM) is a highly aggressive malignancy that is extremely rare in children. This case report documents a 7-year-old male without previous asbestos exposure with peritoneal MM that initially responded to chemotherapy with cisplatin and gemcitabine but ultimately metastasized to his chest. He was d...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24284
更新日期:2013-01-01 00:00:00
abstract::A 4(3/4)-year-old male with Down syndrome (DS) presented with unsteady gait and fatigue. Neuroimaging revealed a cerebellar mass with concomitant obstructive hydrocephalus and additional metastatic lesions. He was successfully treated and is still in complete remission 5 years from diagnosis. The present case illustra...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22109
更新日期:2009-12-01 00:00:00
abstract:BACKGROUND:Patients with sickle cell anemia (SCA) experience a broad range of psychiatric disorders, placing them at risk for more complicated and longer hospitalizations for vaso-occlusive crises (VOC). The current study examined the frequency of psychiatric disorders in SCA patients (ages birth to 20 years) admitted ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23117
更新日期:2012-02-01 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:The male excess in childhood cancer incidence is well-established; however, the underlying biologic mechanisms remain unknown. Examining the association between male sex and childhood cancer by single year of age and tumor type may highlight important periods of risk such as variation in growth and hormonal ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27620
更新日期:2019-06-01 00:00:00
abstract:BACKGROUND:The death of a child is a devastating event that results in profound grief and significant psychosocial and physical morbidities in parents. The parental grief journey is a complex phenomenon necessitating the utilization of newer models of bereavement with a focus on relationships and exploration of parents...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26046
更新日期:2016-09-01 00:00:00
abstract:BACKGROUND:We have previously shown that aerosol interleukin-2 (IL-2) increased the number of intravenously injected human natural killer (NK) cells in the lungs. In this study we investigated whether this increase was secondary to NK cell proliferation and determined the site of the proliferation. MATERIALS AND METHO...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25019
更新日期:2014-08-01 00:00:00
abstract:BACKGROUND:Only a few studies have assessed cardiovascular risk factors (CRFs) in childhood cancer survivors. We determined the prevalence of CRFs in long-term survivors of acute lymphoblastic leukemia (ALL) and Wilms tumor. PROCEDURE:Adult survivors of ALL and Wilms tumor treated with radiotherapy and chemotherapy (R...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22518
更新日期:2010-10-01 00:00:00
abstract::Scrotal ulcers are a rare manifestation in patients with acute promyelocytic leukemia. Fournier's gangrene (FG) is even rarer. We describe three adolescents and young adults who developed scrotal ulcerations during induction with all-trans-retinoic acid. One patient developed FG. These lesions are predominantly seen i...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21549
更新日期:2008-08-01 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:Symptoms of epidural compression (SEC) in children with neuroblastoma (particularly infants) may be misinterpreted, leading to delay in diagnosis. PATIENTS AND METHODS:Clinical, imaging and follow-up data of 34 infants with neuroblastoma and SEC diagnosed between 2000 and 2011 at Italian AIEOP centers were ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.25028
更新日期:2014-08-01 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