Abstract:
:Hemophilia is an excellent example in medicine where clinical translation of basic science discoveries has transformed the gloomy outlook of the disease. This review provides an overview of clinical advances in hemophilia management with a specific focus on the molecular heterogeneity of the disease and progress in management of patients with inhibitors. Novel therapeutics and the emerging ethical issues in the field of hemophilia are also discussed.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Sharathkumar AA,Carcao Mdoi
10.1002/pbc.23193subject
Has Abstractpub_date
2011-12-01 00:00:00pages
910-20issue
6eissn
1545-5009issn
1545-5017journal_volume
57pub_type
杂志文章,评审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::Increasingly young people survive cancer in childhood and as a result complications of its treatment are becoming more common and important. Premature ovarian failure is recognized as a complication of radiotherapy to a field that includes the pelvis and alkylating-agent-based chemotherapy. Young pre-pubertal girls ar...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.22012
更新日期:2009-08-01 00:00:00
abstract:BACKGROUND:Males have a higher incidence of medulloblastoma (MB) than females, but the effect of gender on survival is unclear. Studies have yielded conflicting results, possibly due to small sample sizes or differences in how researchers defined MB. We aimed to determine the effect of gender on survival in MB using a ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21832
更新日期:2009-01-01 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:To determine the maximum-tolerated duration and dose-limiting toxicity of a daily schedule of orally administered cyclophosphamide and topotecan in pediatric patients with recurrent or refractory malignant solid tumors. METHODS:Patients received oral cyclophosphamide (50 mg/m2/dose) in the morning followed ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.10456
更新日期:2004-01-01 00:00:00
abstract:BACKGROUND:In Germany, about 1,800 new cases of pediatric cancer under 15 years of age are diagnosed each year and survival rates approach 80%. Although treatment is covered by health insurance and is thus available for all patients at no cost, treatment refusal and treatment discontinuation have been observed. However...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26111
更新日期:2016-10-01 00:00:00
abstract:BACKGROUND:The Wnt/β-catenin pathway plays a central role in the pathogenesis of most hepatoblastomas (HBs), that is, up to 60-80% carry activating CTNNB1 mutations. HBs can however also be the first manifestation of familial adenomatous polyposis (FAP). As this is a severe disease, it is important for the patient and ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26991
更新日期:2018-06-01 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::Pneumocystis jiroveci pneumonia (PCP) is a serious complication in patients receiving chemotherapy or hematopoietic stem cell transplantation. Current recommendations for trimethoprim-sulfamethoxazole (TMP-SMZ) dosing as PCP prophylaxis in immunocompromised patients are based on either daily dosing or dosing three con...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21774
更新日期:2009-01-01 00:00:00
abstract:PURPOSE:To evaluate the association of central nervous system (CNS) treatment intensity and the social functioning of children who have completed treatment for leukemia, lymphoma, and solid tumors outside the CNS. Furthermore, we expected that these associations would be moderated by child age at diagnosis and gender. ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21062
更新日期:2007-10-15 00:00:00
abstract::Bridging the survival gap for children with cancer, between those (the great majority) in low and middle income countries (LMIC) and their economically advantaged counterparts, is a challenge that has been addressed by twinning institutions in high income countries with centers in LMIC. The long-established partnershi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24802
更新日期:2014-02-01 00:00:00
abstract::An 8-year old child had a pelvic MYCN-nonamplified neuroblastoma (NB) with retroperitoneal nodal extension. Multi-modality therapy achieved complete remission (CR). Small recurrences confined to left supraclavicular nodes were treated with surgery alone at 4.9, 6.5, 7.5, 9.5, and 12.9 years from diagnosis. Monitoring ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21658
更新日期:2008-10-01 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:Pain is the most common complication of sickle cell disease requiring emergency department (ED) visits and hospitalization. A Clinical Practice Guideline (CPG) to manage acute sickle cell pain offers clinicians a standardized approach for the provision of evidence-based, cost-effective care. After CPG implem...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21847
更新日期:2009-03-01 00:00:00
abstract:BACKGROUND:Acute and chronic clinical manifestations of sickle-cell disease (SCD) lead to significant healthcare utilization, especially of the emergency department (ED). Limited population-level data are available in SCD with the ability to connect patients to visits, leaving us with minimal description of utilization...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.22792
更新日期:2011-03-01 00:00:00
abstract:BACKGROUND:Ewing sarcoma of the thoracic spine and chest wall is frequently treated with concurrent chemotherapy and radiation therapy (RT). Treatment-related acute esophagitis can lead to hospitalization and treatment delays. The aim of this study was to analyze the incidence, risk factors, and management of esophagit...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.27006
更新日期:2018-06-01 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:BACKGROUND:Corticosteroids increase risk for decreased bone mineral density, which can be worsened by vitamin D insufficiency (VDI) or deficiency (VDD). PROCEDURE:In the Vanderbilt cancer survivorship clinic, we obtained screening total 25-hydroxy vitamin D levels (VDL) in 171 cancer survivors <23 years old who were t...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24844
更新日期:2014-04-01 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::Sickle cell disease (SCD) is an inherited red blood cell disorder that occurs worldwide and results in significant morbidity and mortality. In addition to affected children who are born in the US, an increasing number of children with SCD are migrating to the US with their families or through international adoption. C...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27000
更新日期:2018-06-01 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:Few studies have addressed the efficacy of palliative radiotherapy (RT) for pediatric osteosarcoma (OS), a disease generally considered to be radioresistant. We describe symptom relief, local control, and toxicity associated with palliative RT among children with OS. PROCEDURE:Patients diagnosed with OS at ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27967
更新日期:2020-01-01 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::Chronic granulomatous disease (CGD) is a primary immunodeficiency that affects the oxidative mechanism of microbial killing of phagocytic cells. The defect is characterized by a lack or severely reduced superoxide anion (O2-) production by phagocytes. Seventy percent of CGD cases are X-linked (X-CGD) and they are caus...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20204
更新日期:2005-04-01 00:00:00
abstract:BACKGROUND:Neuroblastoma (NB) is the most common solid extracranial tumor in childhood. Despite advances in therapy, the prognosis is poor and optimized therapies are urgently needed. Therefore, we investigated the antitumor potential of interleukin-15 (IL-15)-activated cytokine-induced killer (CIK) cells against diffe...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26147
更新日期:2016-12-01 00:00:00
abstract::There is mounting evidence that combination of antibiotic therapy with vancomycin and piperacillin/tazobactam (pip/tazo) is associated with acute kidney injury (AKI). To determine whether vancomycin plus pip/tazo is associated with higher rates of AKI compared to vancomycin plus cefepime among pediatric hematology/onc...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.27750
更新日期:2019-07-01 00:00:00
abstract:BACKGROUND:To examine the practical applicability, internal consistency, and validity of the Bt-DUX, a disease-specific Health Related Quality of Life (HRQoL) instrument. The Bt-Dux was developed to examine patients' individual values of their life after a malignant bone tumor of the lower extremity at four domains (co...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.22078
更新日期:2009-09-01 00:00:00
abstract:BACKGROUND:Medication errors occur universally. Inappropriate administration of chemotherapy drugs can have adverse effects in cancer patients. Our objective was to assess the rate and type of medication errors in children with acute lymphoblastic leukemia (ALL) receiving oral chemotherapy in outpatient setting. PROCE...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25203
更新日期:2014-12-01 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:Although screening for familial retinoblastoma has been shown to be beneficial we suspected that such screening programs may be less than optimal in developing countries (DC). METHODS:Retrospective cohort study comparing patients with familial retinoblastoma from five centers in DC (Argentina, Brazil, Turke...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21970
更新日期:2009-09-01 00:00:00