Abstract:
:Congenital amegakaryocytic thrombocytopenia (CAMT) is characterised by neonatal thrombocytopenia, with reduced or absent bone marrow megakaryocytes, leading eventually to pancytopenia. The mean age for progression to bone marrow failure is four years, with the earliest reported being six months. We describe a CAMT patient with compound heterozygous mutations of the causative MPL gene (one being a previously unreported splice site mutation in intron 11) who developed pancytopenia within the first month of life. This report emphasises the importance of considering CAMT in the differential diagnosis of congenital aplastic anaemia or idiopathic aplastic anaemia in babies.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Stoddart MT,Connor P,Germeshausen M,Ballmaier M,Steward CGdoi
10.1002/pbc.24566subject
Has Abstractpub_date
2013-09-01 00:00:00pages
E94-6issue
9eissn
1545-5009issn
1545-5017journal_volume
60pub_type
杂志文章abstract:BACKGROUND:This study investigates the effect of using patient reported outcomes (PROs) about health-related quality of life (HRQOL) in clinical practice on the type and amount of psychosocial topics discussed during a paediatric oncology consultation. PROCEDURE:Children (N = 193) with cancer participated in a sequent...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.24089
更新日期:2012-07-15 00:00:00
abstract:BACKGROUND:There is little information about factors modulating bone mineral density (BMD) in survivors of childhood acute lymphoblastic leukemia (ALL). PROCEDURE:We analyzed data from 57 survivors (26 male, 52 Caucasian) who underwent two serial quantitative computed tomography (QCT) studies of BMD. Using multiple li...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20553
更新日期:2006-01-01 00:00:00
abstract::Melanotic neuroectodermal tumor of infancy (MNTI) is a rare tumor arising mainly in the orofacial bones. Among more than 200 cases described in articles, only seven cases involved orbital regions. We present a case of newborn with recurrent MNTI in the lateral wall of right orbit. The patient underwent complete surgic...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21607
更新日期:2008-09-01 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:BACKGROUND:Clinical characteristics and optimal treatment strategies for spindle cell/sclerosing rhabdomyosarcoma (ssRMS) have not been well established because of its rarity. PROCEDURE:Retrospective re-evaluation of sarcoma specimens (1997-2014) identified 16 ssRMSs (median age 20 years, range 7-39 years). Clinicopat...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25367
更新日期:2015-06-01 00:00:00
abstract:BACKGROUND:Stroke is a severe clinical disorder in sickle cell disease (SCD), and few studies have evaluated transcranial Doppler (TCD) flow velocities in hemoglobin SC disease (HbSC). The guidelines for stroke risk are based on evaluations in sickle cell anemia (SCA) or HbS/β thalassemia. PROCEDURE:In this study, we ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26342
更新日期:2017-05-01 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:BACKGROUND:Cancer diagnoses pose challenges to adolescents' and young adults' (AYA) physical, mental, and emotional health, and developmental tasks. In order for AYAs, caregivers, clinicians, and other collaborators to optimize health outcomes (coproduction of health), understanding what living well means for AYAs who ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.28599
更新日期:2020-10-01 00:00:00
abstract::Immune-mediated hemolytic anemia is a well-recognized complication of hematopoietic stem cell transplantation. We report on a 6-year-old boy with X-linked adrenoleukodystrophy who developed severe delayed alloimmune hemolytic anemia associated with immune-mediated neutropenia and thrombocytopenia following major ABO i...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21187
更新日期:2008-04-01 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:Studies have shown that children with cancer globally lack access to palliative care. Little is known regarding physicians' perceptions of palliative care, treatment access, and self-reported competence in providing palliative care. PROCEDURE:Members of the Global Neuroblastoma Network (online tumor board) ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25900
更新日期:2016-05-01 00:00:00
abstract:BACKGROUND:Positron emission tomography (PET) and gallium scans facilitate diagnosis and staging, evaluation of response to therapy, and monitoring for relapse in Hodgkin lymphoma (HL), but have not been compared in pediatric HL. PROCEDURE:We performed concurrent PET and gallium scans on 44 pediatric HL patients at di...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21574
更新日期:2008-08-01 00:00:00
abstract:BACKGROUND:Total body irradiation (TBI) is an important component of hematopoietic stem cell transplant (SCT) for pediatric malignancies. With increasing survival rates, late effects of SCT become more important. Younger children may be at particular risk of late effects of radiation and SCT. METHODS:We retrospectivel...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24252
更新日期:2013-04-01 00:00:00
abstract:BACKGROUND:Although chemotherapy has improved outcome of osteosarcoma, 30-40% of patients succumb to this disease. Survivors experience substantial morbidity and mortality from anthracycline-induced cardiotoxicity. We hypothesized that the cardioprotectant dexrazoxane would (i) support escalation of the cumulative doxo...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25753
更新日期:2016-01-01 00:00:00
abstract:BACKGROUND:Central venous catheters (CVC) facilitate the management of patients with cancer. Optimal timing for placement of a CVC is controversial. We sought to determine whether early placement in children with acute lymphoblastic leukemia (ALL), a group at high risk for infection and thrombosis, was associated with ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24010
更新日期:2012-04-01 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:Children with acute lymphoblastic leukemia (ALL) who fail to achieve complete remission (CR) after induction therapy (induction failure: IF) have a poor prognosis; however, there have been few prospective studies in patients with IF. PATIENTS AND METHODS:Between April 1997 and March 2005, 27 of 1,237 leukem...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22217
更新日期:2010-01-01 00:00:00
abstract::Ovarian tumors have a low incidence in childhood, accounting for 1% of malignancies within the ages of 0-17 years. Small cell carcinoma of the ovary is a rare histology and historically has a poor prognosis. We report a case of an 11-year-old female diagnosed with small cell carcinoma of the ovary and hypercalcemia (S...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22184
更新日期:2009-12-15 00:00:00
abstract::Ependymoblastoma (EBL) is a rare malignant CNS tumor of early childhood, listed as a subgroup of primitive neuroectodermal tumors (PNET) in the 2007 WHO Classification of Tumours of the Central Nervous System. Histologically, EBL can be defined by multilayered, mitotically active "ependymoblastic" rosettes with centra...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24915
更新日期:2014-06-01 00:00:00
abstract:BACKGROUND:This study aimed to compare the health related quality of life (HRQoL) of children and adolescents after malignant bone tumor surgery of the leg with healthy controls. PROCEDURE:Patients between 8 and 25 years old were cross-sectional recruited. Patients under 16 years of age received the TNO (Netherlands O...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.22439
更新日期:2010-05-01 00:00:00
abstract:BACKGROUND:Retinoblastoma (Rb) is the most common intraocular primary malignancy in children. In industrialised countries, the cure rate is about 95%. We present the results of a prospective study on the management of Rb in the paediatric oncology unit of Gabriel Touré Teaching Hospital and African Institute of Tropica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27101
更新日期:2018-08-01 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::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: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:The hallmark of sickle cell disease (SCD) is recurrent, painful vaso-occlusive episodes (VOC) and is the most common reason for hospitalization in SCD patients. Narcotics, particularly morphine, along with fluid hydration are standard treatments for painful episodes but have been associated with the developm...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20403
更新日期:2005-10-15 00:00:00
abstract::A 6-year-old male with chronic granulomatous disease, who was transplanted with bone marrow and exhibited increasing mixed chimerism, subsequently received two donor lymphocyte infusions (DLI). Two weeks after the second DLI, the patient developed acute graft-versus-host disease (GVHD) and progressive pancytopenia tha...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22280
更新日期:2010-02-01 00:00:00
abstract:BACKGROUND:We examined patterns of failure in pediatric patients with thoracic sarcoma and pulmonary metastases treated with intensity-modulated radiation therapy with dose-painting (DP-IMRT). PROCEDURE:Eleven pediatric patients, five with Ewing sarcoma family tumors (ESFT) and six with rhabdomyosarcoma (RMS), with pr...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.24502
更新日期:2013-10-01 00:00:00
abstract::Steroid-induced neuropsychological side effects impact quality of life in children with acute lymphoblastic leukemia. Dexamethasone induces more metabolic side effects than prednisone. To evaluate whether dexamethasone also leads to more neuropsychological side effects, we reviewed all available literature. Randomized...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24988
更新日期:2014-07-01 00:00:00
abstract:BACKGROUND:Pediatric cancer-induced financial burden is source of stress for parents, particularly mothers, single parents, and parents with lower incomes. This financial burden has been linked to poorer family quality of life (QOL) in terms of new onset material hardships, and could also affect individual QOL in terms...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28093
更新日期:2020-03-01 00:00:00
abstract::The molecular detection of minimal residual disease (MRD) is standard of care in acute lymphoblastic leukemia to personalize the stratification of patients to appropriate intensity chemotherapy regimens. High-throughput sequencing (HTS) techniques are driving changes to MRD methodologies. Our study demonstrates HTS ca...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27787
更新日期:2019-08-01 00:00:00