Abstract:
BACKGROUND:Extracranial malignant rhabdoid tumor (MRT) is a rare pediatric cancer with a poor prognosis. The kidney is the most common site. Isolated reports have shown improvements in patient survival, but no specific treatment regimen has shown efficacy over others. PROCEDURE:Retrospective review of patients diagnosed with extracranial MRT at Children's Hospital Los Angeles between 1983 and 2012. RESULTS:The median age at presentation for the 21 patients was 13 months (range, 0-108 months). Ten patients had renal primary tumors. The median time to progression was 4 months (range, 0.4-7 months). The 5-year event free survival (EFS) and overall survival (OS) of the entire cohort was 38 ± 10.6%. After 2002, patients diagnosed with extracranial MRT were administered a chemotherapy regimen of vincristine, doxorubicin and high dose cyclophosphamide (VDC). The OS for the patients diagnosed before and after 2002 were 20 ± 12% and 54 ± 15%, respectively. Of the 13 patients who received VDC containing regimen, eight patients achieved a complete radiological remission; five of these patients are long-term survivors. Four patients who received autologous bone marrow transplantation were alive at last follow-up. All patients with unresectable primary tumors died. Patients who had disease progression or relapse did not survive. CONCLUSIONS:Patients with extracranial MRT have a poor prognosis. Treatment with high dose alkylator therapy followed by consolidation with high dose chemotherapy and autologous bone marrow transplant for those patients in radiographic complete remission appears to have a beneficial effect on survival.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Venkatramani R,Shoureshi P,Malvar J,Zhou S,Mascarenhas Ldoi
10.1002/pbc.25093subject
Has Abstractpub_date
2014-08-01 00:00:00pages
1357-61issue
8eissn
1545-5009issn
1545-5017journal_volume
61pub_type
临床试验,杂志文章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: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:Information on the epidemiology of pediatric liver tumors in Latin America is limited. PURPOSE:To describe the incidence of liver tumors in a pediatric registry in Argentina according to geographic region, national trends over 16 years, and survival related to stage, age, sex, and care center. METHODS:Newl...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28583
更新日期:2020-11-01 00:00:00
abstract:OBJECTIVE:To investigate the effect of Helicobacter pylori eradication on platelet recovery in childhood chronic idiopathic thrombocytopenic purpura (ITP). PATIENTS AND METHODS:A multi-center randomized controlled trial was conducted. Patients aged 4-18 years, diagnosed with chronic ITP, defined by platelet count belo...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1002/pbc.21991
更新日期:2009-07-01 00:00:00
abstract::A contemporaneous presentation of a second breast cancer in a mother and an extremity rhabdomyosarcoma (RMS) in her daughter led to the diagnosis of the Li Fraumeni syndrome (LFS). Although the association between LFS and RMS in young patients is well recognised 1 there are no guidelines as to how this knowledge shoul...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20795
更新日期:2007-03-01 00:00:00
abstract:BACKGROUND:Retinoblastoma occurs in approximately 4 per million children per year in the United States, usually before 2 years of age. In developed countries, 95% of children with tumor in one eye can be cured with enucleation, while children with tumor in both eyes require individualized therapy to preserve vision. Al...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21155
更新日期:2008-02-01 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: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:BACKGROUND:Neuroblastoma (NB) is the most common solid extracranial tumor in childhood. Despite advances in therapy, the prognosis is poor and optimized therapies are urgently needed. Therefore, we investigated the antitumor potential of interleukin-15 (IL-15)-activated cytokine-induced killer (CIK) cells against diffe...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26147
更新日期:2016-12-01 00:00:00
abstract::Although awareness of implicit bias and its influence on providers and patients is increasing, the effects of implicit bias on the field of pediatric hematology-oncology are less clear. This Special Report reviews the literature on implicit bias in pediatrics and medical oncology and further provides case examples and...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.28204
更新日期:2020-05-01 00:00:00
abstract:BACKGROUND:Childhood germ cell tumors (cGCTs), believed to arise from transformed primordial germ cells by an unknown mechanism, provide a unique model system for investigating cell signaling, pluripotency, and the microenvironment of neoplastic stem cells (NSCs) in vivo. This is the first report of proteomics of cGCTs...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23282
更新日期:2012-05-01 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::This report by the Radiation Oncology Discipline of Children's Oncology Group (COG) describes the practice patterns of pediatric image-guided radiotherapy (IGRT) based on a member survey and provides practice recommendations accordingly. The survey comprised of 11 vignettes asking clinicians about their recommended tr...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28629
更新日期:2020-10-01 00:00:00
abstract:BACKGROUND:Specialized pediatric cancer centers (PCCs) are thought to be essential to obtain state-of-the-art care for children and adolescents. We determined the proportion of childhood cancer patients not treated in a PCC, and described their characteristics and place of treatment. PROCEDURE:The Swiss Childhood Canc...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.22426
更新日期:2010-05-01 00:00:00
abstract:BACKGROUND:We report on the treatment of children and adolescents with acute lymphoblastic leukemia (ALL) in first relapse. The protocol focused on: (1) Intensive chemotherapy preceding allogeneic stem cell transplantation (SCT) in early bone marrow relapse; (2) Rotational chemotherapy in late relapse, without donor; (...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.22946
更新日期:2011-08-01 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::We conducted a retrospective chart review to determine prevalence of, risk factors for, and liver toxicity associated with Transfusion Related Iron Overload (TRIO) in pediatric cancer patients, and report our experience with Iron Chelation Therapy (ICT). Total number of transfusions was identified as the major risk fa...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25189
更新日期:2014-12-01 00:00:00
abstract::Familial adenomatous polyposis (FAP) due to APC mutation is associated with an increased risk of hepatoblastoma. All cases of hepatoblastoma in patients with FAP reported in the literature were reviewed. One hundred and nine patients were identified. Thirty-five patients (of 49 with data) were diagnosed with hepatobla...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.27103
更新日期:2018-08-01 00:00:00
abstract::The commonly accepted definition of Fanconi anemia (FA) relying on DNA repair deficiency is submitted to a critical review starting from the early reports pointing to mitomycin C bioactivation and to the toxicity mechanisms of diepoxybutane and a group of nitrogen mustards causing DNA crosslinks in FA cells. A critica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.25452
更新日期:2015-07-01 00:00:00
abstract:BACKGROUND:Alveolar soft part sarcomas (ASPS) are generally chemo- and radio-resistant mesenchymal tumours, with no standardized treatment guidelines. We describe the clinical behaviour of paediatric ASPS and compare these features to previously reported adult series. PATIENTS AND METHODS:The clinical data of 51 child...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24683
更新日期:2013-11-01 00:00:00
abstract:BACKGROUND:Little is known about the visual outcome of children affected by an optic pathway glioma (OPG). PROCEDURES:We evaluated the long-term visual outcome of 32 consecutive children affected by OPG without neurofibromatosis type-1 referred to the Pediatric Department of Padua University and managed according to s...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.22748
更新日期:2010-12-01 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: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:Fever and neutropenia (F&N) is a pediatric oncology emergency due to the risk of disseminated infection. Quality improvement (QI) efforts to improve time to antibiotics for F&N in the emergency department have been documented, but the issue has not been studied in the established inpatient setting. PROCEDUR...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25712
更新日期:2016-01-01 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::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:BACKGROUND:The optimal dose and schedule of mycophenolate mofetil (MMF) in pediatric allogeneic stem cell transplant recipients remains to be determined. We previously reported safety and pharmacokinetics of MMF at 900 mg/m2 q6h dosing. This study was conducted to investigate the efficacy of tacrolimus plus q8h MMF dos...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.27091
更新日期:2018-08-01 00:00:00
abstract::Phytosterolemia is a rare autosomal recessive sterol storage disease caused by mutations in ABCG5 and ABCG8 genes. A 9-year-old Turkish boy who was presented with exclusively hematologic abnormalities had elevated plant sterol levels. Sequencing of ABCG5 and ABCG8 genes revealed a novel homozygous IVS10-1 G>T mutation...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24934
更新日期:2014-08-01 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::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