Abstract:
:We present three pediatric patients with BRAFV600E mutant high-grade gliomas treated by vemurafenib on a nominative authorization level at our institution. One patient with anaplastic ganglioglioma experienced confirmed partial tumor response and significant clinical improvement and she is alive 20 months after start of treatment. A second patient with ganglioglioma responded transiently to re-introduction of vemurafenib after immunotherapy. Pharmacokinetic studies suggest that maximum concentration and exposure of vemurafenib at steady-state is dose-dependent and similar in children to that reported in adults. These cases suggest that BRAFV600 is an oncogenic driver in pediatric gliomas. Further exploration in clinical studies is ongoing.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Bautista F,Paci A,Minard-Colin V,Dufour C,Grill J,Lacroix L,Varlet P,Valteau-Couanet D,Geoerger Bdoi
10.1002/pbc.24891subject
Has Abstractpub_date
2014-06-01 00:00:00pages
1101-3issue
6eissn
1545-5009issn
1545-5017journal_volume
61pub_type
杂志文章abstract::Hepatic sinusoidal obstructive syndrome (HSOS) is a frequent complication in patients undergoing haematopoietic stem cell transplant (HSCT), and more rarely, in paediatric patients receiving conventional chemotherapy for solid tumours. Its diagnosis relies on a combination of clinical signs and symptoms such as hepato...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24859
更新日期:2014-05-01 00:00:00
abstract:BACKGROUND:The level of minimal residual disease (MRD) prior to allogeneic hematopoietic stem cell transplantation (HSCT) has been shown to be an independent prognostic factor for outcome of pediatric patients with high-risk acute lymphoblastic leukemia (ALL). Retrospective studies which used (semi-) quantitation of cl...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.20794
更新日期:2007-01-01 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::The treatment of malignancy in cancer predisposition syndromes that also confer exquisite sensitivity to standard chemotherapy and radiation regimens remains a challenge. Bloom syndrome is one such disorder that is caused by a defect in DNA repair, predisposing to the development of early-onset age-related medical con...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28815
更新日期:2021-02-01 00:00:00
abstract:BACKGROUND:Drug shortages require clinical teams to decide how to allocate drugs in limited supply among their patients. Ethical frameworks are invaluable for promoting rational approaches to drug distribution, but gaps remain between ethical theory and clinical application. The goal of this work was to explore how dec...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26331
更新日期:2017-07-01 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:Insomnia is common among adolescent and young adult (AYA) cancer survivors. Cognitive-behavioral therapy for insomnia (CBT-I) is considered the gold standard treatment. Standard CBT-I was designed for adults and not adapted to the unique medical, psychosocial, and developmental needs of AYA cancer survivors,...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28506
更新日期:2020-09-01 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:While adolescents and adults with sickle cell disease (SCD) have reported using religion to cope with SCD, there is no data examining religious coping in young children with SCD. The purpose of this qualitative study was to: (1) describe the types of religious coping used by children with SCD; (2) describe t...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23038
更新日期:2012-02-01 00:00:00
abstract:BACKGROUND:Pediatric aggressive fibromatosis (AF) is a rare, benign tumor with locally infiltrative growth. Therefore, how to prevent reoccurrence while maintaining the mandible contour and continuity as much as possible is very important when the mandible is involved. PROCEDURE:We selected 10 pediatric patients with ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24145
更新日期:2012-10-01 00:00:00
abstract:BACKGROUND:Children treated with cranial radiotherapy (CRT) for leukemia are at risk of developing central nervous system injuries. Magnetic resonance imaging (MRI) represents the examination method of choice for evaluating radiation-induced brain complications. The purpose of this report is to describe the spectrum of...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22923
更新日期:2011-08-01 00:00:00
abstract:BACKGROUND:The identification of hemophagocytosis (HPC) in tissue or bone marrow (BM) represents only one of 5/8 criteria needed for the diagnosis of hemophagocytic lymphohistiocytosis (HLH). Yet, confirmation of HPC in bone marrow aspirates (BMA) is often relied upon to make therapeutic decisions. There is no standard...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21564
更新日期:2008-09-01 00:00:00
abstract::The Standards, Options, and Recommendations (SOR) project undertaken by the French National Federation of Cancer Centers (FNCLCC) to develop and disseminate clinical practice guidelines in oncology has now been taken over by the French National Cancer Institute. In 2007, the SOR updated the information related to the ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.21953
更新日期:2009-07-01 00:00:00
abstract::A teenage boy presented with a CD30-positive anaplastic large cell lymphoma (ALCL) affecting his scapula and was successfully treated with chemotherapy. His clinical features and outcome were compared with other cases described in the literature. A further review of 11 ALCL cases with bony involvement treated in the U...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20542
更新日期:2007-04-01 00:00:00
abstract::Gemtuzumab ozogamicin (GO) is a humanized anti-CD33 antibody used for treating patients with CD33+ acute myeloid leukemia (AML). We report three young children (two infants and one toddler) with AML treated with GO 9 mg/m(2). Two received two doses at diagnosis alone with conventional chemotherapy and one received one...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22404
更新日期:2010-07-15 00:00:00
abstract::Peripheral neuropathy is a well-known side effect of vincristine, a micro-tubule inhibitor commonly used to treat malignancies. Severe neurologic adverse events can occur in patients with Charcot-Marie-Tooth disease (CMT) treated with vincristine. Voriconazole is an antifungal agent used increasingly in children with ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21786
更新日期:2009-02-01 00:00:00
abstract::The youngest siblings may be both emotionally vulnerable and often neglected members of the family of a childhood cancer patient. The prompt identification of signs of distress in these subjects allows trained caregivers to intervene with personalized, age-appropriate, attention, and care. A narrative approach, based ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21056
更新日期:2008-03-01 00:00:00
abstract:BACKGROUND AND AIM:We reviewed the results and pattern of failure of the consensus HB/HCC 1996 treatment protocol for pediatric hepatoblastoma (HB) in Hong Kong. The role of SIOPEL and Children's Hepatic tumors International Collaboration (CHIC) risk stratification was evaluated. METHODS:Patients enrolled on the proto...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27482
更新日期:2019-01-01 00:00:00
abstract:INTRODUCTION:The rate of bacterial infections in children with sickle cell disease (SCD) has decreased in recent years, mainly due to penicillin prophylaxis and vaccination. OBJECTIVES:To determine the rate of severe bacterial infection (SBI) in a cohort of children with SCD and to describe low-risk factors for confir...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27667
更新日期:2019-06-01 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::Chronic myeloid leukemia (CML) is a rare disease in children. Different from that in adults, childhood CML involves transformative events occurring over a short time period. CML transformation to lymphoid blast phase (BP) is associated with copy number abnormalities, characteristic of BCR-ABL1 positive acute lymphobla...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27570
更新日期:2019-04-01 00:00:00
abstract::Germline DICER1 pathogenic variants predispose to numerous benign and malignant tumors. In this report, we describe DICER1 gene analysis in an adolescent diagnosed with multinodular goiter, ovarian Sertoli-Leydig cell tumor, and lung cyst. DICER1 mutational screening at the DNA level failed to detect any pathogenic va...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27005
更新日期:2018-06-01 00:00:00
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:BACKGROUND:Knowledge about their past medical history is central for childhood cancer survivors to ensure informed decisions in their health management. Knowledge about information provision and information needs in this population is still scarce. We thus aimed to assess: (1) the information survivors reported to have...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24762
更新日期:2014-02-01 00:00:00
abstract:BACKGROUND:We examined patterns of failure in pediatric patients with thoracic sarcoma and pulmonary metastases treated with intensity-modulated radiation therapy with dose-painting (DP-IMRT). PROCEDURE:Eleven pediatric patients, five with Ewing sarcoma family tumors (ESFT) and six with rhabdomyosarcoma (RMS), with pr...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.24502
更新日期:2013-10-01 00:00:00
abstract:BACKGROUND:The aim was to evaluate self-reported reproductive characteristics and markers of ovarian function in a nationwide cohort of female survivors of childhood acute lymphoblastic leukemia (ALL), because prior investigations have produced conflicting data. PROCEDURE:Self-reported reproductive characteristics wer...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28894
更新日期:2021-01-18 00:00:00
abstract::Twenty-eight patients were maintained on subcutaneous immunoglobulin replacement for persistent B-cell aplasia and agammaglobulinemia following CD19-targeted chimeric antigen receptor T-cell therapy for B-cell lymphoblastic leukemia. Patients were transitioned from intravenous to subcutaneous immunoglobulin replacemen...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28092
更新日期:2020-03-01 00:00:00
abstract:BACKGROUND:The role of the neutropenic diet in the development of infections in oncology and stem cell transplant (SCT) patients is controversial. There is no data on the use of the neutropenic diet among pediatric oncologists. METHODS:A self-administered electronic survey was sent to 1,639 pediatric oncologists at 19...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25104
更新日期:2014-10-01 00:00:00
abstract::Although limited data are available about cyclophosphamide disposition in patients with renal insufficiency, nothing has been reported in anephric patients. We characterized cyclophosphamide pharmacokinetics in an anephric child with bilateral Wilms tumor, both on (day 1) and off (day 2) hemodialysis. The median cyclo...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20558
更新日期:2006-01-01 00:00:00
abstract::The efficacy of hydroxyurea in hemoglobin SC (HbSC) patients is not well documented. We describe the long-term response to hydroxyurea in children with clinically severe HbSC. In 15 patients, hydroxyurea resulted in a significant increase in mean corpuscular volume (MCV) and fetal hemoglobin (HbF) and a significant de...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24283
更新日期:2013-02-01 00:00:00