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, however, the effect of deferasirox on proximal tubule function is unclear. We report one pediatric patient with reversible Fanconi syndrome associated with long-term deferasirox therapy and one patient with mild proximal tubular dysfunction. Kidney and proximal tubular function should be periodically monitored in patients receiving deferasirox throughout their course of therapy.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Rheault MN,Bechtel H,Neglia JP,Kashtan CEdoi
10.1002/pbc.22711subject
Has Abstractpub_date
2011-04-01 00:00:00pages
674-6issue
4eissn
1545-5009issn
1545-5017journal_volume
56pub_type
杂志文章abstract:BACKGROUND:Morphological evaluation of early response to chemotherapy and measurement of minimal residual disease by flow cytometry or PCR are being used for evaluation of prognosis and treatment stratification in children with acute lymphoblastic leukaemia (ALL). PROCEDURE:In a series of 14 consecutive bone marrow in...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23081
更新日期:2011-08-01 00:00:00
abstract:BACKGROUND:Neuroblastoma is the most common extracranial pediatric solid cancer. Lung metastasis is rarely detected in children with newly diagnosed neuroblastoma. We aimed to describe the incidence, clinical characteristics, and outcome of patients with lung metastasis at initial diagnosis using a large international ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21684
更新日期:2008-11-01 00:00:00
abstract:BACKGROUND:Patients with sickle cell disease (SCD) receiving chronic blood transfusions are at risk of developing iron overload and organ toxicity. Chelation therapy with either subcutaneous (SQ) desferrioxamine (DFO) or oral deferasirox is effective in preventing and reducing iron overload but poses significant challe...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22660
更新日期:2010-12-15 00:00:00
abstract::High dose acetaminophen (HDAC) with N-acetylcysteine (NAC) has been effective in adults with advanced malignancies. We report HDAC with NAC in a child with progressive hepatoblastoma, confirmed at biopsy of an unresectable hepatic mass. Alpha-fetoprotein (AFP) increased despite four courses of doxorubicin and one cour...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20330
更新日期:2005-08-01 00:00:00
abstract::While encapsulated bacterial agents, particularly Streptococcus pneumoniae, are recognized as important microbes that are associated with serious illness in hosts with sickle cell disease (SCD), multiple pathogens are implicated in infectious manifestations of SCD. Variations in clinical practice have been an obstacle...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.25911
更新日期:2016-05-01 00:00:00
abstract::Primary cutaneous lymphomas are rare in children and mostly represented by mycosis fungoides and CD30(+) lymphoproliferative disorders. Most pediatric cutaneous lymphomas have similar clinical/pathological features as their adult counterparts, particularly the T-cell subtypes. With regard to outcome, adult cutaneous m...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.26076
更新日期:2016-11-01 00:00:00
abstract:BACKGROUND:Although weight loss and lack of linear growth occur in children with cancer, growth history is not included in research that aims to determine nutritional status in children newly diagnosed with cancer. Therefore, this study aimed to determine weight loss and lack of linear growth in this patient group. PR...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.25301
更新日期:2015-02-01 00:00:00
abstract::Anaphylactic/anaphylactoid reaction to methotrexate (MTX) is uncommon. It may occur with the first dose (non-allergic reactions) or after a previous exposure to the drug (allergic or specific reactions). Desensitization has been shown effective in children with allergic-type reactions permitting the continuation of hi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22616
更新日期:2010-09-01 00:00:00
abstract:BACKGROUND:Pediatric nonrhabdomyosarcoma soft tissue sarcomas (NRSTS) may rarely occur in visceral tissues, and little is known about their clinical history. The present study retrospectively analyzed a group of patients prospectively registered in Italian pediatric protocols conducted between 1979 and 2004. METHODS:I...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26490
更新日期:2017-09-01 00:00:00
abstract:BACKGROUND:Chronic myeloid leukemia (CML) is a rare disease in children and represents approximately 2% of all childhood leukemia. This results in difficulty creating large cohorts of patients for pediatric CML research. The Glivec International Patient Assistance Program (GIPAP) is a patient-access program sponsored b...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25120
更新日期:2014-10-01 00:00:00
abstract::Both hepatoblastoma and hypoplastic kidneys are rare in children. A review of all patients with hepatoblastoma treated at our institution between 1993 and 2011 revealed three cases of hepatoblastoma occurring in children with hypoplastic kidneys and significantly impaired renal function. Two patients were treated with...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24936
更新日期:2014-08-01 00:00:00
abstract::Optic pathway gliomas represent approximately 5% of all pediatric intracranial tumors. While these tumors are most frequently low grade astrocytomas, they follow a highly variable clinical course, and accordingly, there is much debate regarding their optimal management. Their propensity to occur in very young children...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20655
更新日期:2006-05-01 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:BACKGROUND:Contemporary descriptions of classical Hodgkin lymphoma (cHL) are lacking from sub-Saharan Africa where human immunodeficiency virus (HIV) and Epstein-Barr virus (EBV) are prevalent. METHODS:We describe a prospective cHL cohort in Malawi enrolled from 2013 to 2015. Patients received standardized treatment a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26302
更新日期:2017-05-01 00:00:00
abstract:BACKGROUND:Choroid plexus tumors (CPT) are rare, and predominate in early childhood. An association with the Li-Fraumeni syndrome (LFS) has been reported, but the biological and clinical implications of this association remain poorly defined. We have investigated the clinical features and overall survival of all CPT pa...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23349
更新日期:2012-06-01 00:00:00
abstract:INTRODUCTION:Understanding factors that affect the decisions of caregivers of African children to enroll their children in clinical trials would lead to more fully informed consent. METHODS:During the NOHARM study (NCT01976416), a placebo-controlled clinical trial of hydroxyurea for Ugandan children with sickle cell a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27830
更新日期:2020-02-01 00:00:00
abstract:BACKGROUND:Ispinesib is a highly specific inhibitor of kinesin spindle protein (KSP, HsEg5), a mitotic kinesin required for separation of the spindle poles. Here we report the activity of ispinesib against the in vitro and in vivo panels of the Pediatric Preclinical Testing Program (PPTP). PROCEDURES:Ispinesib was tes...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22056
更新日期:2009-12-15 00:00:00
abstract:BACKGROUND:Veno-occlusive disease (VOD) is a major complication following hematopoietic stem cell transplantation (HSCT). Its diagnosis is based on clinical criteria, which have a limited sensitivity. Increased plasminogen activator inhibitor-1 (PAI-1) levels have been suggested as a marker of VOD. We aimed to prospect...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23213
更新日期:2012-05-01 00:00:00
abstract::We report a case of recurrent squamous cell carcinoma (SCC) of the scalp with deep cerebral invasion in a 15-year-old girl. Plain films and CT showed extensive, full thickness, and skull destruction at the vertex. Gadolinium-enhanced MRI revealed neoplastic invasion of the meninges and both cerebral hemispheres down t...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20427
更新日期:2005-12-01 00:00:00
abstract::Pediatric cancer programs in low-income countries (LIC) can improve outcomes. However, treatment must be tailored to the patient's living conditions and the availability of supportive care. In some cases, a more intense regimen will decrease survival since the increase in death from toxicity may exceed any decrease in...
journal_title:Pediatric blood & cancer
pub_type:
doi:10.1002/pbc.20989
更新日期:2007-04-01 00:00:00
abstract:BACKGROUND:Prophylactic use of cranial radiation therapy (CRT) in young children with acute lymphoblastic leukemia (ALL) is associated with significant long-term morbidity. Therefore, current treatment protocols for pediatric B-precursor ALL have abandoned prophylactic CRT in favor of intrathecal chemotherapy, combined...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.10392
更新日期:2004-01-01 00:00:00
abstract:BACKGROUND:Based on the presumed clinical similarity between the two most severe sickle cell disease (SCD) genotypes, hemoglobin (Hb) Sβ0 thalassemia and HbSS, randomized controlled trials (RCTs) have included both genotypes. Our group has demonstrated that healthcare providers inadequately distinguish the two diagnose...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27352
更新日期:2018-11-01 00:00:00
abstract:BACKGROUND:Fanconi anaemia is a genetic disease characterized by congenital abnormalities, progressive bone marrow failure, and a higher predisposition of oral squamous cell carcinoma. The purpose of this study was to evaluate the prevalence of oral mucosa lesions in patients with Fanconi anaemia without hematopoietic ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25417
更新日期:2015-06-01 00:00:00
abstract:BACKGROUND:Ewing sarcoma (EWS) is rarely diagnosed as a second malignancy. We sought to describe a cohort of patients with secondary EWS and investigate if patient characteristics and survival differ between patients with secondary and primary EWS. PROCEDURE:Patients with EWS or peripheral primitive neuroectodermal tu...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24251
更新日期:2013-04-01 00:00:00
abstract:BACKGROUND:A majority of Fanconi anemia (FA) patients will experience bone marrow failure (BMF) and androgen therapy (most often oxymetholone) may be utilized as a treatment to improve BMF-related cytopenias. However, oxymetholone is associated with toxicities making identification of other agents of interest. In this ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24617
更新日期:2014-01-01 00:00:00
abstract:BACKGROUND:Children with sickle cell disease (SCD) and abnormal transcranial Doppler (TCD) ultrasonography have a high risk of stroke, but this risk is greatly reduced when chronic transfusion therapy is administered. The change in TCD velocities during chronic transfusion therapy and rate and frequency of normalizatio...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22951
更新日期:2011-05-01 00:00:00
abstract::Hyperglycemia during induction chemotherapy for childhood acute lymphoblastic leukemia (ALL) has been inconsistently associated with risk of infection. We investigated the incidence of hyperglycemia during induction for childhood ALL in a retrospective cohort study of 144 patients treated on a single national protocol...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24689
更新日期:2013-12-01 00:00:00
abstract::X-linked lymphoproliferative syndrome (XLP) is caused by mutations in SH2D1A, and is associated with overwhelming infectious mononucleosis, aplastic anemia, hypogammaglobulinemia, and B-cell lymphomas. However, the frequency of SH2D1A mutations in males who present with B NHL is unknown. Five cases of XLP were diagnos...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24525
更新日期:2013-09-01 00:00:00
abstract::Malignant degeneration frequently arises from preexisting plexiform neurofibroma in patients with neurofibromatosis type 1 (NF1). Image guided biopsy for diagnostic purposes, such as with CT guidance, can be technically challenging in these patients, as CT cannot distinguish malignant from benign areas within the same...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24668
更新日期:2013-12-01 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