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-intensive Medical Research Council (MRC) regimen. The purpose of this study was to describe early post-treatment cardiac function as a potential indicator of acute and long-term risk associated with this approach. PROCEDURE:A multi-center retrospective cohort analysis was conducted of AML survivors diagnosed from 2004 to 2009 and treated with MRC-based regimens. Change in left ventricular shortening fraction (LVSF) on echocardiogram was determined from baseline to latest post-treatment/pre-relapse value; associations with potential predictors were examined. RESULTS:This cohort of pediatric survivors (n = 52) was assessed at a median interval of 13 months from end of treatment. Mean cumulative anthracycline dose was 339 ± 14 mg/m(2) . Mean baseline and post-treatment LVSF were 39.3 ± 0.8% and 35.4 ± 0.9%, respectively; mean percent change for individuals was -8.4 ± 2.8% (P < 0.001). Cardiac-directed medications were initiated in four patients (7.7%). Decline in LVSF was significantly associated with cumulative anthracycline dose, increasing BMI and Hispanic ethnicity. CONCLUSION:Early, significant decline in LVSF was observed following treatment with these MRC-based regimens. Elevated BMI and Hispanic ethnicity were identified as new independent risk factors. Children and adolescents so treated are at substantial risk for late-onset cardiomyopathy, require monitoring with annual echocardiogram per current COG survivorship guidelines, and are good candidates for appropriate cardioprotection strategies.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Orgel E,Zung L,Ji L,Finklestein J,Feusner J,Freyer DRdoi
10.1002/pbc.24498subject
Has Abstractpub_date
2013-09-01 00:00:00pages
1528-33issue
9eissn
1545-5009issn
1545-5017journal_volume
60pub_type
临床试验,杂志文章,多中心研究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 famil...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24020
更新日期:2012-09-01 00:00:00
abstract::Rhabdomyosarcoma arising within a congenital cystic adenomatoid malformation (CCAM) is an unusual entity. The patient underwent a lobectomy of his right lower lobe of lung due to a CCAM at the age of two. One year later, he developed a solid embryonal rhabdomyosarcoma at the same location. He received 1-year period ch...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20481
更新日期:2005-11-01 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: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::In HLH-94, the first prospective international treatment study for hemophagocytic lymphohistiocytosis (HLH), diagnosis was based on five criteria (fever, splenomegaly, bicytopenia, hypertriglyceridemia and/or hypofibrinogenemia, and hemophagocytosis). In HLH-2004 three additional criteria are introduced; low/absent NK...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21039
更新日期:2007-02-01 00:00:00
abstract:BACKGROUND:ABT-751, an orally bioavailable sulfonamide, binds beta-tubulin to inhibit microtubule polymerization. We described response and event-free survival (EFS) in children with neuroblastoma and other solid tumors receiving ABT-751, assessed in vitro cytotoxicity of ABT-751 and evaluated the effect of ABT-751 on ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22267
更新日期:2010-01-01 00:00:00
abstract:BACKGROUND:Tumor cells frequently contaminate autologous stem cell products in a variety of malignancies, but their clinical significance remains controversial. We retrospectively monitored tumor contamination in stem cell harvests from patients with Ewing family of tumors (EFT) all harboring the specific translocation...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20022
更新日期:2004-05-01 00:00:00
abstract::Almost all of what is known about neurologic and cognitive development in hemophilia derives from the Hemophilia Growth and Development Study, conducted during an era when treatment regimens and comorbidities differed significantly from the current environment. Results suggested hemophilia and human immunodeficiency v...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.28004
更新日期:2020-01-01 00:00:00
abstract:BACKGROUND:Accurate risk stratification of Langerhans cell histiocytosis (LCH) is essential as management can range from conservative in single system, low risk for central nervous system (CNS) involvement lesions to intensive chemotherapy for multisystem or high-risk disease. Additionally, being able to differentiate ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28891
更新日期:2021-01-14 00:00:00
abstract:BACKGROUND:Retinoblastoma (Rb) is the most common intraocular primary malignancy in children. In industrialised countries, the cure rate is about 95%. We present the results of a prospective study on the management of Rb in the paediatric oncology unit of Gabriel Touré Teaching Hospital and African Institute of Tropica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27101
更新日期:2018-08-01 00:00:00
abstract::Hepatoblastoma is a malignant pediatric liver tumor. The currently used diagnostic serum marker for hepatoblastoma, α-fetoprotein (AFP), is not always reliable in infants with hepatoblastoma, due to the physiologically elevated levels of AFP in this age group. In this report, we show that Delta-like 1 homolog (DLK1), ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24024
更新日期:2012-10-01 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::Deferasirox (Exjade®, Novartis) is a widely used oral iron chelator for the treatment of patients with iron overload due to chronic transfusion therapy for diseases such as β-thalassemia and sickle cell disease. Renal side effects of deferasirox are common and include non-progressive increases in serum creatinine, how...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22711
更新日期:2011-04-01 00:00:00
abstract:INTRODUCTION:The National Heart, Lung, and Blood Institute guidelines for sickle cell disease (SCD) pain crisis management recommend opioids within 60 minutes of emergency department (ED) registration and every 30 minutes thereafter until acute pain is managed. These guidelines are based on expert opinion without publi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28268
更新日期:2020-09-01 00:00:00
abstract::The identification and referral of candidate patients for phase I trials relies heavily on pediatric oncologists who must balance their own perceptions of phase I trials with the desires of the patient and his/her family. A survey was sent to 419 physicians practicing pediatric oncology at 30 different institutions. R...
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.24522
更新日期:2013-08-01 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::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:We report a 13-year-old male with Diamond Blackfan anemia and short stature. He had a normal biochemical response to growth hormone (GH) stimulation, but his bone age was delayed, his insulin-like growth factor 1 (IGF-1) was low, and he had a poor growth velocity. He was started on daily GH injections. METH...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20075
更新日期:2004-10-01 00:00:00
abstract:BACKGROUND:Pediatric cancer-induced financial burden is source of stress for parents, particularly mothers, single parents, and parents with lower incomes. This financial burden has been linked to poorer family quality of life (QOL) in terms of new onset material hardships, and could also affect individual QOL in terms...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28093
更新日期:2020-03-01 00:00:00
abstract::Melanotic neuroectodermal tumor of infancy (MNTI) is a rare tumor arising mainly in the orofacial bones. Among more than 200 cases described in articles, only seven cases involved orbital regions. We present a case of newborn with recurrent MNTI in the lateral wall of right orbit. The patient underwent complete surgic...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21607
更新日期:2008-09-01 00:00:00
abstract:BACKGROUND:Stroke is a severe clinical disorder in sickle cell disease (SCD), and few studies have evaluated transcranial Doppler (TCD) flow velocities in hemoglobin SC disease (HbSC). The guidelines for stroke risk are based on evaluations in sickle cell anemia (SCA) or HbS/β thalassemia. PROCEDURE:In this study, we ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26342
更新日期:2017-05-01 00:00:00
abstract:BACKGROUND:We sought to assess patterns of failure in pediatric patients with intracranial germ cell tumors (GCT) treated with intensity-modulated radiation therapy with dose painting (DP-IMRT). PROCEDURE:Between July 2007 and October 2013, 11 patients with localized GCT-five germinomas and six nongerminoma GCT (NGGCT...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25867
更新日期:2016-04-01 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:Patients with cancer who receive intensive chemotherapeutic regimens are subject to profound immunosuppression and are susceptible to an extended array of pathogens. PROCEDURE:The infectious causes of symptomatic gastroenteritis as evidenced by diarrhea +/- fever, vomiting, and abdominal colic in children f...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.10394
更新日期:2004-04-01 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 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
abstract:BACKGROUND:Hepatoblastoma is a rare malignancy of childhood. The scarcity of adequate cell models has limited our understanding of this tumor. Here we describe and characterize a new human liver tumor cell line, Hep293TT, derived from an aggressive childhood hepatoblastoma. PROCEDURES:Hep293TT cells were established u...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22187
更新日期:2009-12-01 00:00:00
abstract::Opsoclonus, myoclonus, ataxia syndrome (OMA) is a severe neurologic disorder often associated with neuroblastoma. It is challenging to treat and can have long-term neurologic sequelae. Current recommended therapies include intravenous immunoglobulin, corticosteroids, rituximab, and chemotherapy (cyclophosphamide). We ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27110
更新日期:2018-08-01 00:00:00
abstract::Guidelines for management of chronic idiopathic thrombocytopenic purpura (ITP) in childhood are still based on expert opinions and therefore remain controversial. Splenectomy is an established option for chronic ITP in adults, but splenectomy in childhood is complex, due to higher probability for spontaneous recovery ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20979
更新日期:2006-10-15 00:00:00
abstract:BACKGROUND:Sleep disordered breathing (SDB) is emerging as a significant health condition for children. The purpose of this study is to evaluate SDB symptoms in childhood cancer survivors and identify associations with quality of life (QOL) and psychological symptoms. PROCEDURE:A sample of 62 survivors aged 8-18 years...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25394
更新日期:2015-04-01 00:00:00