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 virus had independent effects, and hemophilia negatively impacts academic achievement, attention, and behavior. The introduction of prophylaxis treatment in hemophilia has created the need for re-evaluation of the effects of hemophilia on neurodevelopment and cognition. We outline the Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence, and Other Cognitive Functions (NCT03660774) study, which aims to meet this need.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Buranahirun C,Walsh KS,Mrakotsky C,Croteau SE,Rajpurkar M,Kearney S,Hannemann C,Wilkening GN,Shapiro KA,Cooper DLdoi
10.1002/pbc.28004subject
Has Abstractpub_date
2020-01-01 00:00:00pages
e28004issue
1eissn
1545-5009issn
1545-5017journal_volume
67pub_type
杂志文章,评审abstract:BACKGROUND:We have previously shown that aerosol interleukin-2 (IL-2) increased the number of intravenously injected human natural killer (NK) cells in the lungs. In this study we investigated whether this increase was secondary to NK cell proliferation and determined the site of the proliferation. MATERIALS AND METHO...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25019
更新日期:2014-08-01 00:00:00
abstract:BACKGROUND:Silent cerebral infarcts are both morbid and progressive in children with sickle cell disease (SCD). While blood transfusion therapy is effective primary and secondary stroke prevention, the efficacy and acceptance of blood transfusion therapy for children with silent cerebral infarcts is unknown. The overal...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.21338
更新日期:2008-03-01 00:00:00
abstract:BACKGROUND/OBJECTIVES:Sickle cell disease (SCD) is an important, hidden cause of childhood mortality worldwide. It is most prevalent in sub-Saharan Africa where national newborn screening programs remain unavailable and most children in rural areas are never diagnosed. We conducted a study at a rural district hospital ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28620
更新日期:2020-11-01 00:00:00
abstract::MIRAGE syndrome caused by mutations in SAMD9 is associated with potential loss of chromosome 7 (-7/7q-) and an increased risk to develop myelodysplastic syndrome (MDS). We report a case of MIRAGE syndrome, caused by a novel SAMD9 mutation p.Leu641Pro, leading to characteristic clinical features as well as to the coexi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27589
更新日期:2019-04-01 00:00:00
abstract:BACKGROUND:Over the past decade, PET-CT has been used to assess rhabdomyosarcoma (RMS) in children. However, the role of PET-CT in staging RMS is unknown. PROCEDURE:Thirty subjects with RMS, median age 7.3 years, underwent PET-CT before therapy. PET-CTs and conventional imaging (CI) were independently reviewed by two ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24430
更新日期:2013-07-01 00:00:00
abstract::To characterize radiation necrosis following hypofractionated brainstem re-irradiation in pediatric patients, we reviewed 23 cases with 28 tumors invading or abutting brainstem and treated with hypofractionated re-irradiation from 2004 to 2014. Re-irradiation delivered total doses of 16-30 Gy in two to five fractions....
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26341
更新日期:2017-05-01 00:00:00
abstract:PURPOSE:Research using banked tissue is key to advancing risk-stratification and treatment of children with cancer. Knowledge of parental attitudes to ethical issues arising in tissue banking is very limited but essential in obtaining respectful consent. METHODS:One hundred parents of consecutively diagnosed children ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22716
更新日期:2011-12-15 00:00:00
abstract:BACKGROUND:The use of carboplatin for the treatment of pediatric low grade gliomas (PLGG) is often limited by the development of carboplatin hypersensitivity. Reported rates of carboplatin hypersensitivity reactions vary between 6% and 32% in these patients. Here we report the frequency of carboplatin hypersensitivity ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25686
更新日期:2016-01-01 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::The rejection rate in cord blood transplants for chronic Epstein-Bar virus-associated T or natural killer cell lymphoproliferative diseases using our standard reduced-intensity conditioning "LPAM140 regimen," which includes fludarabine, melphalan (LPAM), etoposide, and antithymocyte globulin, has been high. To ensure ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28536
更新日期:2020-09-01 00:00:00
abstract:BACKGROUND:The presence of central nervous system (CNS) disease in pediatric acute myeloid leukemia (AML) is often thought to confer a worse prognosis. This study examined the outcome of children with AML who had CNS disease at diagnosis. METHODS:Patients enrolled on Children's Cancer Group protocols 2861, 2891, 2941,...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22511
更新日期:2010-09-01 00:00:00
abstract:BACKGROUND:Fever in neutropenic (FN) patients requires immediate broad-spectrum antibiotics, however, such patients do not represent a homogeneous population and the majority of them are at low risk of developing complication. Gatifloxacin (GA) is an alternative, though it has not been thoroughly studied in Pediatrics ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.21124
更新日期:2007-10-15 00:00:00
abstract::Scrotal ulcers are a rare manifestation in patients with acute promyelocytic leukemia. Fournier's gangrene (FG) is even rarer. We describe three adolescents and young adults who developed scrotal ulcerations during induction with all-trans-retinoic acid. One patient developed FG. These lesions are predominantly seen i...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21549
更新日期:2008-08-01 00:00:00
abstract:BACKGROUND:Thrombosis in neonates is a rare but serious occurrence, usually associated with central catheterization. The objective of this study was to investigate the risk factors associated with catheter related thrombosis in very low birth weight (VLBW) infants. PROCEDURE:The present retrospective study was perform...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21714
更新日期:2009-01-01 00:00:00
abstract::Osteosarcoma is the most common bone tumor in children and young adults, with few advances in survival and treatment, especially for metastatic disease, in the last 30 years. Recently, immunotherapy has begun to show promise in various adult cancers, but the utility of this approach for osteosarcoma remains relatively...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.27227
更新日期:2018-09-01 00:00:00
abstract:AIM:Osteopontin (OPN) has been investigated as a biomarker for cancer and nonmalignant diseases during the last decades. Data about OPN as a potential biomarker in childhood diseases are still sparse, and reference values are not available in children. We aimed to establish reference values for children from birth to y...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28272
更新日期:2020-07-01 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::We report two cases of primary CNS lymphoma (PCNSL) treated with high-dose methotrexate. Though standard adult treatment of PCNSL incorporates whole-brain radiotherapy, the literature suggests it may be possible to delay or avoid radiotherapy and the associated increased risk of neurologic sequelae in pediatric patien...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22752
更新日期:2010-12-01 00:00:00
abstract:BACKGROUND:Asparaginase (Asp) and corticosteroid (CS) treatment in patients with acute lymphoblastic leukaemia (ALL) is associated with an increased risk of thrombotic events. OBJECTIVE:Characterization of global haemostatic phenotypes of patients with ALL during Asp therapy. PROCEDURE:Thrombin generation (TG) was mo...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26228
更新日期:2017-02-01 00:00:00
abstract:BACKGROUND:Outcomes for childhood brain tumors are now associated with a five-year survival rate of 75%. Endocrine effects of brain tumors are common, occurring in 43% of patients by 10 years from tumor diagnosis. Optimal timing of screening for endocrinopathies remains undefined. We aim to identify incidence and timin...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27631
更新日期:2019-05-01 00:00:00
abstract::Obesity is increasing in prevalence in the United States with over 65% of adults considered overweight and 16% of children with BMI > 95 percentile. The heritability of obesity is estimated between 40% and 70%, but the genetics of obesity for most individuals are complex and involve the interaction of multiple genes a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23372
更新日期:2012-01-01 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::Ameloblastic fibro-odontosarcoma (AFOS) is an extremely rare malignant odontogenic tumor. Complete surgical excision is the treatment of choice. Deaths due to disease recurrence and/or progression are documented. Here, we report the case of a 15-year-old female with multiple recurrent AFOS. She responded to chemothera...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25627
更新日期:2015-11-01 00:00:00
abstract:BACKGROUND:The early effects of childhood acute lymphoblastic leukemia (ALL) include decreased physical function, bone mineral density (BMD/g/cm2 ), and health-related quality of life (HRQL). We assessed the capacity of a physical therapy and motivation-based intervention, beginning after diagnosis and continuing throu...
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.26929
更新日期:2018-04-01 00:00:00
abstract::Unstable hemoglobin variants represent a rare etiology of congenital hemolytic anemia. Without a high index of suspicion, plus proper laboratory testing and interpretation, the correct diagnosis can be elusive. We report on five children who were initially thought to have other congenital disorders such as hereditary ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22702
更新日期:2010-12-15 00:00:00
abstract:BACKGROUND:The DNA methylating agent temozolomide was developed primarily for treatment of glioblastoma. However, preclinical data have suggested a broader application for treatment of childhood cancer. Temozolomide was tested against the PPTP solid tumor and ALL models. PROCEDURES:Temozolomide was tested against the ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24368
更新日期:2013-05-01 00:00:00
abstract::Bereaved families fear their child being forgotten by those who knew their loved child, including their child's oncology team. Thoughtfully timed, family-centric condolences shared by pediatric oncology team members have the potential to extend our compassion and kindness toward a family during the darkness of grief. ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27489
更新日期:2019-02-01 00:00:00
abstract::We report a novel glucose-6-phosphate dehydrogenase (G6PD) mutation, which we propose to name G6PD Cincinnati (c.1037A > T, p.N346I), found in combination with G6PD Gastonia (c.637G > T, p.V213L) in an infant who presented with neonatal cholestasis. The G6PD Cincinnati mutation results in a non-conservative amino acid...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22744
更新日期:2011-05-01 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::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