Abstract:
BACKGROUND:Distinguishing alveolar rhabdomyosarcoma (ARMS) from embryonal rhabdomyosarcoma (ERMS) has historically been of prognostic and therapeutic importance. However, classification has been complicated by shifting histologic criteria required for an ARMS diagnosis. Children's Oncology Group (COG) studies after IRS-IV, which included the height of this diagnostic shift, showed both an increased number of ARMS and an increase in the proportion of fusion-negative ARMS. Following diagnostic standardization and histologic re-review of ARMS cases enrolled during this era, analysis of low-risk (D9602) and intermediate-risk (D9803) rhabdomyosarcoma (RMS) studies showed that fusion status rather than histology best predicts prognosis for patients with RMS. This analysis remains to be completed for patients with high-risk RMS. PROCEDURE:We re-reviewed cases on high-risk COG studies D9802 and ARST0431 with an enrollment diagnosis of ARMS. We compared the event-free survival (EFS) and overall survival by histology, PAX-FOXO1 fusion, and clinical risk factors (Oberlin score) for patients with metastatic RMS using the log-rank test. RESULTS:Histology re-review resulted in reclassification as ERMS for 12% of D9802 cases and 5% of ARST0431 cases. Fusion-negative RMS had a superior EFS to fusion-positive RMS; however, poorer outcome for metastatic RMS was most related to clinical risk factors including age, primary site, and number of metastatic sites. CONCLUSIONS:In contrast to low- or intermediate-risk RMS, in metastatic RMS, clinical risk factors have the most impact on patient outcome. PAX-FOXO1 fusion is more common in patients with a high Oberlin score, but fusion status is not an independent biomarker of prognosis.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Rudzinski ER,Anderson JR,Chi YY,Gastier-Foster JM,Astbury C,Barr FG,Skapek SX,Hawkins DS,Weigel BJ,Pappo A,Meyer WH,Arnold MA,Teot LA,Parham DMdoi
10.1002/pbc.26645subject
Has Abstractpub_date
2017-12-01 00:00:00issue
12eissn
1545-5009issn
1545-5017journal_volume
64pub_type
杂志文章abstract::Risk-adapted, response-based therapies for pediatric Hodgkin lymphoma have resulted in 5-year survival exceeding 90%. Although high-dose chemotherapy and autologous hematopoietic stem cell transplantation (AHSCT) are considered standard for most patients with relapsed or refractory Hodgkin lymphoma, a subset of childr...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24851
更新日期:2014-04-01 00:00:00
abstract:BACKGROUND:Coping with end-stage pediatric cancer patients and the related bereavement is a challenge for all the caregivers involved. PROCEDURE:Forty-seven cancer patients who died in 2006 were assessed as concerns the main place of care in the end stage of their disease, their symptoms, the palliative treatments rec...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22284
更新日期:2010-01-01 00:00:00
abstract:BACKGROUND:Hematopoietic stem cell transplantation (HSCT) provides a curative therapy for children severely affected by sickle cell disease (SCD). Rejection-free survival after matched sibling donor (MSD) HSCT is very high, but adoption of HSCT as a curative SCD therapy has been slow. In this study, we assess providers...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28861
更新日期:2021-03-01 00:00:00
abstract:BACKGROUND:Clinical outcomes of children with sickle cell disease (SCD) who undergo total or partial splenectomy (PS) are poorly defined. The purpose of this retrospective study was to initiate an Internet-based registry to facilitate analysis of clinical outcomes for these children. We hypothesized that both surgical ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.24057
更新日期:2012-07-15 00:00:00
abstract:BACKGROUND:This study evaluated the outcome of retinoblastoma patients, when employing a telemedicine-based twinning program in Jordan. PROCEDURE:This cohort study included patients at the King Hussein Cancer Centre (KHCC; Amman, Jordan) who received consultations for retinoblastoma from March 2003 to September 2006. ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21489
更新日期:2008-08-01 00:00:00
abstract::2-Chlorodeoxyadenosine (2-CdA) has been successfully used in children to treat refractory Langerhans cell histiocytosis and juvenile xanthogranuloma (JXG) as salvage therapy. Although 2-CdA is generally well-tolerated, with temporary myelosuppression as the primary dose-limiting toxicity, prolonged myelosuppressive, a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23087
更新日期:2012-02-01 00:00:00
abstract:BACKGROUND:Sickle cell disease (SCD) is characterized by frequent disease-related events that require acute care. It is unknown to what extent patients utilize multiple hospitals for acute care. We examined the continuity pattern of acute care visits to the hospital or emergency department. We hypothesized that among p...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24028
更新日期:2012-10-01 00:00:00
abstract:PURPOSE:The Children's Oncology Group conducted a phase II trial of 21-day continuous infusion topotecan to determine the response rate in pediatric patients with recurrent or refractory malignant solid tumors. PROCEDURE:Patients with Ewing sarcoma family of tumors (ESFT), osteosarcoma (OS), soft tissue sarcomas (STS)...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20739
更新日期:2006-11-01 00:00:00
abstract:BACKGROUND:Ensuring adequate parental consent is a key issue of ethical practice in pediatric oncology. In Germany, however, knowledge about parental comprehension and satisfaction with the informed consent procedure is limited, and representative data on parents' perspectives are still missing. Based on data collected...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24330
更新日期:2013-03-01 00:00:00
abstract:BACKGROUND:For children with juvenile myelomonocytic leukemia (JMML) who undergo stem cell transplantation (SCT), the role of immunological interventions including withdrawal of immunosuppressive therapy (IST) and donor lymphocyte infusion (DLI) for treatment of disease recurrence remains uncertain. PROCEDURE:We analy...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24259
更新日期:2013-01-01 00:00:00
abstract:BACKGROUND:The aim of the study was to investigate factors affecting response to everolimus, a mammalian-target-of-rapamycin (mTOR) inhibitor, of subependymal giant cell astrocytomas (SEGA) in patients with tuberous sclerosis complex (TSC). METHODS:The study group consisted of 15 children with a diagnosis of TSC-relat...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.25368
更新日期:2015-04-01 00:00:00
abstract:BACKGROUND:Clear cell sarcoma of the kidney (CCSK) is known for its propensity to metastasize to bone, but it also spreads to other sites including the brain. This study was undertaken to describe the treatment and outcomes of patients with recurrent CCSK involving the brain. METHODS:A retrospective records review was...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21131
更新日期:2008-02-01 00:00:00
abstract::Global variations in the incidence of pediatric cancers have been described; however, the causes of such differences are not known. We investigated the relationship between the incidence of embryonal tumors and human development index on a global scale. Increasing incidence of neuroblastoma correlates significantly wi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26244
更新日期:2017-02-01 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::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::A major hallmark of NF1 is the development of benign tumors, including peripheral neurofibromas, plexiform neurofibromas, gliomas of the optic tract, other low grade gliomas, and pheochromocytomas. Hepatoblastoma have not been previously reported in patients with neurofibromatosis type 1. We present a case of a 9-mont...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20663
更新日期:2007-09-01 00:00:00
abstract::Beckwith-Wiedemann syndrome (BWS) is a genetic overgrowth and cancer predisposition syndrome, associated with both benign and malignant adrenal findings. Literature review and an institutional case series elucidate the wide spectrum of adrenal findings in BWS patients. The altered expression of the 11p15 region is lik...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.26432
更新日期:2017-08-01 00:00:00
abstract:BACKGROUND:The treatment of central nervous system (CNS) germ cell tumors (GCT) remains controversial. The purpose of this study was to demonstrate efficacy of a chemotherapy only strategy, with less morbidity, when compared to regimens with irradiation. METHODS:Between January 2001 and December 2004 newly diagnosed p...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.22381
更新日期:2010-03-01 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: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: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:Alvespimycin (17-DMAG, KOS-1022), a potent small-molecule inhibitor of the protein chaperone Hsp90, is being developed as an anticancer agent because of the multiple Hsp90 client proteins involved in cancer cell growth and survival. PROCEDURES:Alvespimycin was tested against the in vitro panel of the Pediat...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21508
更新日期:2008-07-01 00:00:00
abstract:BACKGROUND:Langerhans cell histiocytosis (LCH) is a rare disease, frequently affecting young children. PROCEDURE:We performed a retrospective study in patients younger than 16 years old manifesting with skin symptoms, and documented their different cutaneous lesions and systemic symptoms. We compared subgroups of chil...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25834
更新日期:2016-03-01 00:00:00
abstract::We report two survivors of Wilms tumor (WT) who developed inflammatory myofibroblastic tumor (IMT). The first patient had IMT in the mainstem bronchus 1 year after WT therapy that included vincristine, dactinomycin, doxorubicin (VDA) and whole lung radiation therapy (XRT). The second patient had IMT in the cecum 7 yea...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25345
更新日期:2015-06-01 00:00:00
abstract:BACKGROUND:Pediatric venous thromboembolism (VTE) is an increasingly common problem. We hypothesized that VTE occurs most commonly in tertiary care settings and that the pattern of associated illnesses may have changed from earlier reports. METHODS:The Kids' Inpatient Database 2006 was utilized to identify children ≤ ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23388
更新日期:2012-08-01 00:00:00
abstract:BACKGROUND:Although "aspirin resistance" (AR-inadequate platelet inhibition as suggested by in vitro testing of aspirin-treated patients) has been widely studied in adults and linked to increased risk of adverse outcomes, its prevalence and clinical significance are largely unknown in children. PROCEDURE:To determine ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21465
更新日期:2008-07-01 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:BACKGROUND:Hypogammaglobulinemia (hypo-IgG) is common early post-HSCT in children, occasionally necessitating long-term immunoglobulin (Ig) G replacement therapy. IgG replacement may not reduce mortality, although infectious complications are decreased PROCEDURE:Clinical data and samples from 86 children were analyzed...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25409
更新日期:2015-05-01 00:00:00
abstract:BACKGROUND:Burnout is a work-related syndrome consisting of emotional exhaustion, depersonalization, and diminished feelings of personal accomplishment. Physicians who care for patients with life-threatening illnesses are at high risk for developing burnout. This survey evaluates the prevalence of burnout among pediatr...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23121
更新日期:2011-12-15 00:00:00
abstract::Inherited bone marrow failure syndromes (IBMFS) are rare cancer predisposition syndromes with an especially high risk of transformation to myelodysplastic syndrome (MDS) and/or acute myeloid leukemia (AML). We performed a retrospective systematic review of reported MDS/AML arising in the eight most common IBMFS to det...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.26714
更新日期:2017-12-01 00:00:00