Abstract:
BACKGROUND:The efficacy of combination chemotherapy with methotrexate (MTX) and asparaginase is not well known in relapsed and refractory acute leukemia after contemporary therapy. PROCEDURE:A retrospective study of pediatric patients with relapsed or refractory acute myeloid leukemia (AML) who received MTX and asparaginase as a salvage therapy at St. Jude Children Research Hospital was performed. MTX was given intravenously followed by a dose of asparaginase intramuscularly or intravenously 24 hours later. The chemotherapy cycle was repeated every 7-10 days. Response, survival, and toxicities were evaluated. RESULTS:Fifteen patients, median age 10.5 years (range, 1.1-18.5 years), were treated. Median number of previous therapeutic regimens was three (range, 1-4). Six patients responded to treatment (three had morphologic complete remission with incomplete blood count recovery, two had partial remission, and one had stable disease for 16 months), and four are still alive. Three of six responders had monoblastic leukemia, and also developed tumor lysis syndrome. The 1- and 2-year overall survival rates are 35.6% and 17.8%, respectively. The most common adverse event was transient elevation of transaminases (nine patients). Two patients developed pancreatitis. Episodes of febrile neutropenia were rare (two patients), and most courses (75 out of 93 total courses) were given in an outpatient setting. CONCLUSIONS:Combination chemotherapy with MTX and asparaginase appears to be an effective salvage therapy and well tolerated in patients with relapsed or refractory childhood AML, even in those heavily pretreated with contemporary frontline or salvage therapy.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Buaboonnam J,Cao X,Pauley JL,Pui CH,Ribeiro RC,Rubnitz JE,Inaba Hdoi
10.1002/pbc.24470subject
Has Abstractpub_date
2013-07-01 00:00:00pages
1161-4issue
7eissn
1545-5009issn
1545-5017journal_volume
60pub_type
杂志文章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:A pediatric Phase I trial was performed to determine the maximum-tolerated dose, dose-limiting toxicities (DLTs), and pharmacokinetics (PK) of vorinostat and bortezomib, in patients with solid tumors. PROCEDURE:Oral vorinostat was administered on days 1-5 and 8-12 of a 21-day cycle (starting dose 180 mg/m(2...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24271
更新日期:2013-03-01 00:00:00
abstract:BACKGROUND:Histology after intensive induction chemotherapy is expected to become a beacon indicating when and how extensively radical surgery and lymph node dissection should be performed in advanced neuroblastoma. A thorough histologic review of surgical specimens was undertaken. PROCEDURE:All specimens from 34 pati...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20345
更新日期:2005-10-15 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:PURPOSE:To assess health and musculoskeletal function in survivors of pediatric sarcomas. PATIENTS AND METHODS:Thirty-two individuals treated for Ewing sarcoma family of tumors (ESFT), rhabdomyosarcoma (RMS), or non-rhabdomyosarcoma soft tissue sarcomas (NR-STS) with multi-modality therapy were enrolled on this cross-...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20871
更新日期:2007-02-01 00:00:00
abstract::We describe here the outcomes of reduced-toxicity alternate-donor stem cell transplant (SCT) with posttransplant cyclophosphamide (PTCy) for primary immunodeficiency disorders (PIDs) in eight children (haploidentical-seven and matched unrelated donor-one). The conditioning was with serotherapy (alemtuzumab-3/rabbit-an...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26783
更新日期:2018-01-01 00:00:00
abstract:BACKGROUND:Physical fitness is an important determinant of quality of life (QOL) after hematopoietic stem cell transplantation. Cardiac function can influence exercise performance. The aim of this study was to assess these factors and their interrelationship. PROCEDURE:Children underwent cardiopulmonary exercise testi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27499
更新日期:2019-01-01 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:A desperate need for novel therapies in pediatric ependymoma (EPN) exists, as chemotherapy remains ineffective and radiotherapy often fails. EPN have significant infiltration of immune cells, which correlates with outcome. Immune checkpoint inhibitors provide an avenue for new treatments. This study characte...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26960
更新日期:2018-05-01 00:00:00
abstract::Poverty is an important patient-reported outcome of therapy and a potential predictor of outcome disparities in pediatric cancer. We previously identified that nearly 30% of pediatric cancer families experience household material hardship (HMH), a concrete measure of poverty including food, energy, or housing insecuri...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.26743
更新日期:2018-01-01 00:00:00
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 ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24891
更新日期:2014-06-01 00:00:00
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:Posterior fossa syndrome (PFS) is defined as the temporary and complete loss of speech after posterior fossa surgery. The goal of this study was to identify incidence and risk factors for PFS and to determine accompanying neurobehavioral and psychologic problems. PROCEDURE:Between May 2007 and April 2009, c...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22730
更新日期:2011-02-01 00:00:00
abstract:BACKGROUND:Methylenetetrahydrofolate reductase (MTHFR) C677T and A1298C polymorphisms have been implicated in childhood acute lymphoblastic leukemia (ALL) risk, but previously published studies were inconsistent and recent meta-analyses were not adequate. PROCEDURES:In a meta-analysis of 21 publications with 4,706 cas...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,meta分析,评审
doi:10.1002/pbc.23137
更新日期:2012-04-01 00:00:00
abstract:BACKGROUND:Although intra-retinal tumor has long been staged presurgically according to the Reese-Ellsworth (R-E) system, retinoblastoma differs from other pediatric neoplasms in never having had a widely accepted classification system that encompasses the entire spectrum of the disease. Comparisons among studies that ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.20606
更新日期:2006-11-01 00:00:00
abstract::To assess safety and tolerability, we administered valacyclovir, an oral anti-viral medication that inhibits erythrocyte sickling in vitro, to 14 subjects with sickle-cell anemia for 1 week at a standard dose of 1,000 mg every 8 hr. No clinically significant adverse effects occurred. In 11 subjects in steady state, th...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22809
更新日期:2011-05-01 00:00:00
abstract::Undifferentiated embryonal sarcoma of the liver (UESL) is a rare aggressive mesenchymal pediatric tumor. Previously, reported outcomes have been very poor. Here, we report a single-center experience of five patients with UESL treated with upfront gross total resection and adjuvant chemotherapy. We have a median follow...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26154
更新日期:2016-12-01 00:00:00
abstract:OBJECTIVES:The aim of this study was to assess glomerular and tubular renal function after HSCT in children in a prospective trial. METHODS:Renal function was assessed prospectively before HSCT (on day -10), on days +30, +100, and at least 6 months after transplantation in 34 patients (21 females/13 males) with a mean...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22030
更新日期:2009-08-01 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::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:BACKGROUND:Despite advances in therapy, outcome in many high-grade pediatric central nervous system (CNS) tumors remains poor. The focus of neuro-oncology research has thus turned towards identifying novel therapeutic targets. Poly(ADP-ribose) polymerase-1 (PARP1) is a DNA repair protein that has been studied in a vari...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22141
更新日期:2009-12-15 00:00:00
abstract:BACKGROUND:Hepatic veno-occlusive disease (VOD) is one of the most serious complications in stem cell transplantation (SCT). Although plasma protein C activity decreases in VOD after SCT, the timeframe of plasma protein C activity decreases during SCT is not known. PROCEDURE:We examined levels of plasma protein C seri...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22314
更新日期:2010-03-01 00:00:00
abstract:BACKGROUND:Survival of osteosarcoma patients has reached a plateau with the addition of chemotherapy and in part predicted based on histologic response. Risk-adapted therapy might be an alternative approach. We aimed to identify risk groups using clinical variables available at time of diagnosis in order to better pred...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21843
更新日期:2009-03-01 00:00:00
abstract:BACKGROUND:Systematic assessment of emotional distress is recommended in after care. Yet, it is unclear if parent report may be used as a proxy of child report. The aim of this study was to assess agreements and differences and explore possible moderators of disagreement between child and parent ratings. METHODS:Sixty...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.26840
更新日期:2018-02-01 00:00:00
abstract:INTRODUCTION:Our objectives were to design and validate methods to identify relapse and hematopoietic stem cell transplantation (HSCT) in children with acute lymphoblastic leukemia (ALL) using administrative data representing hospitalizations at US pediatric institutions. METHODS:We developed daily billing and ICD-9 c...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28315
更新日期:2020-05-11 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::Chronic granulomatous disease (CGD) is an immune deficiency characterized by defective neutrophil function and increased risk of life-threatening infections. Allogeneic hematopoietic cell transplantation is curative for CGD, and conditioning regimen impacts transplant-related outcomes. We report a single-center prospe...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.28030
更新日期:2020-01-01 00:00:00
abstract:PURPOSE:This pilot study was done to determine the feasibility and accuracy of University of Florida/National Cancer Institute (UF/NCI) phantoms and Monte Carlo (MC) retrospective dosimetry and had two aims: (1) to determine the anatomic accuracy of UF/NCI phantoms by comparing 3D organ doses in National Wilms Tumor St...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27395
更新日期:2018-12-01 00:00:00
abstract::This is the case report of a 14-year young female who was diagnosed with solitary bone plasmacytoma (SBP) of proximal tibia and was treated by local involved field radiotherapy. We present the clinical, radiological and pathological findings of the case and review of the available treatment options and prognosis of th...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22687
更新日期:2011-01-01 00:00:00
abstract::Immunotherapy with the anti-GD2 monoclonal antibody ch14.18, or dinutuximab, represents an important therapeutic advance in the treatment of pediatric high-risk neuroblastoma and is now considered part of standard of care in this patient population. To date, transverse myelitis as a result of dinutuximab therapy has n...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26732
更新日期:2018-01-01 00:00:00