Abstract:
:Chronic myeloid leukemia (CML) is a rare disease in children. Different from that in adults, childhood CML involves transformative events occurring over a short time period. CML transformation to lymphoid blast phase (BP) is associated with copy number abnormalities, characteristic of BCR-ABL1 positive acute lymphoblastic leukemia, but not of CML in the chronic phase. Here, we present an unusual case of CML progressing to BP in a 1.6-year-old child, harboring IKZF1, PAX5, CDKN2A, and ETV6 deletions at diagnosis. It remains to be addressed whether distinct mechanisms might account for CML pathogenesis in early childhood.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Klumb CE,Barbosa TDC,Nestal de Moraes G,Schramm MT,Emerenciano M,Maia RCdoi
10.1002/pbc.27570subject
Has Abstractpub_date
2019-04-01 00:00:00pages
e27570issue
4eissn
1545-5009issn
1545-5017journal_volume
66pub_type
杂志文章abstract:BACKGROUND:The treatment of central nervous system (CNS) germ cell tumors (GCT) remains controversial. The purpose of this study was to demonstrate efficacy of a chemotherapy only strategy, with less morbidity, when compared to regimens with irradiation. METHODS:Between January 2001 and December 2004 newly diagnosed p...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.22381
更新日期:2010-03-01 00:00:00
abstract::The BRAF V600E missense mutation is known to be present in a subset of central nervous system tumors. We report a patient with a BRAF V600E mutated pilomyxoid astrocytoma who failed multiple conventional chemotherapy regimens. Treatment with vemurafenib, a molecularly targeted therapy against the mutant BRAF V600E kin...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25084
更新日期:2014-11-01 00:00:00
abstract::We report three cases of hemophagocytic lymphohistiocytosis (HLH) in infants within the first 6 weeks of life. Diagnosis of HLH was made early after symptoms started. All three cases were successfully treated with dexamethasone and none relapsed, indicating that not all cases of HLH in very young infants are familial....
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21758
更新日期:2009-01-01 00:00:00
abstract::Although investigators have been engaged in the study of human energy metabolism for over 100 years, there remain unanswered questions regarding the role that energy expenditure plays in human health and disease. A critical examination of available methods for measurement of the various components of daily energy expe...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23369
更新日期:2012-01-01 00:00:00
abstract:BACKGROUND:This study evaluated the outcome of retinoblastoma patients, when employing a telemedicine-based twinning program in Jordan. PROCEDURE:This cohort study included patients at the King Hussein Cancer Centre (KHCC; Amman, Jordan) who received consultations for retinoblastoma from March 2003 to September 2006. ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21489
更新日期:2008-08-01 00:00:00
abstract::Germline or acquired mutations involving the GATA-binding protein gene (GATA2) have been linked to a variety of clinical conditions. In addition, patients harboring GATA2 mutations have a striking predisposition to develop myeloid malignancies, such as myelodysplastic syndrome or acute myeloid leukemia, but not acute ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27649
更新日期:2019-06-01 00:00:00
abstract:BACKGROUND:Primary hemophagocytic lymphohistiocytosis (HLH) represents a group of inherited hyperinflammatory immunodeficiencies, including familial HLH (FHL), Griscelli syndrome type 2 (GS2), and X-linked lymphoproliferative syndrome (XLP). We previously reported an annual incidence of suspected primary HLH in Sweden ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25308
更新日期:2015-02-01 00:00:00
abstract:BACKGROUND:Population-based evidence suggests that lower socioeconomic status (SES) negatively impacts the overall survival (OS) of children with leukemia; however, the relationships between SES and treatment-related mortality, relapse, and timing of relapse remain unclear. PROCEDURE:We examined OS, event-free surviva...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25928
更新日期:2016-06-01 00:00:00
abstract::Juvenile myelomonocytic leukemia (JMML) is a rare myelodysplastic/myeloproliferative disorder of early childhood characterized by mutations of the RAS-RAF-MAP kinase signaling pathway. We report the case of a child with a diagnosis of JMML carrying two mutations of NRAS gene (c.37G>C and c.38G>A) independently occurri...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23401
更新日期:2012-09-01 00:00:00
abstract:BACKGROUND:We report on the treatment of children and adolescents with acute lymphoblastic leukemia (ALL) in first relapse. The protocol focused on: (1) Intensive chemotherapy preceding allogeneic stem cell transplantation (SCT) in early bone marrow relapse; (2) Rotational chemotherapy in late relapse, without donor; (...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.22946
更新日期:2011-08-01 00:00:00
abstract:BACKGROUND:Childhood cancer is relatively rare and tends to present specific age distribution, as a prognostic factor for some of these diseases. Information on how young age affects prognosis, response to chemotherapy, and local control options in children versus AYA with osteosarcoma (OST) is minimal. METHODS:In ord...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25459
更新日期:2015-07-01 00:00:00
abstract:BACKGROUND:Langerhans cell histiocytosis (LCH) is a rare proliferative disorder of pathological Langerhans cells, for which the aetiology and pathogenesis remain largely unknown. PROCEDURE:Information on the 101 children with LCH registered with the population-based Manchester Children's Tumour Registry (MCTR) between...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20884
更新日期:2007-05-01 00:00:00
abstract::Enrollment of patients in sickle cell intervention trials has been challenging due to difficulty in obtaining consent from a legal guardian and lack of collaboration between emergency medicine and hematology. We utilized education and preconsent in a pediatric multisite sickle cell intervention trial to overcome these...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.26013
更新日期:2016-09-01 00:00:00
abstract:BACKGROUND:Retinoblastoma occurs in approximately 4 per million children per year in the United States, usually before 2 years of age. In developed countries, 95% of children with tumor in one eye can be cured with enucleation, while children with tumor in both eyes require individualized therapy to preserve vision. Al...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21155
更新日期:2008-02-01 00:00:00
abstract::Acute immune thrombocytopenic purpura (ITP) occurs most commonly in young children who present with severe isolated thrombocytopenia and purpura. A marrow examination is not required unless glucocorticoids are used, lest treatment mask incipient acute lymphoblastic leukemia, but controversy exists here. The recommenda...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.21934
更新日期:2009-09-01 00:00:00
abstract:BACKGROUND:Long-term adverse effects of Imatinib in children with chronic myeloid leukemia (CML) are uncertain. The aim was to study the effect of imatinib on growth in children with CML. PROCEDURE:Children ≤13 years of age at diagnosis were enrolled retrospectively, from 2004 to 2011, from a single center in India. P...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23389
更新日期:2012-09-01 00:00:00
abstract:BACKGROUND:A total of 201 pediatric cases of acute lymphoblastic leukemia were treated with the ALL-96 protocol by the Kyushu-Yamaguchi Children's Cancer Study Group. PROCEDURE:Risk stratification was based on white cell counts, immunophenotype, the presence of central nervous system disease at diagnosis, organomegaly...
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.22528
更新日期:2010-08-01 00:00:00
abstract:PURPOSE:About one-third of patients with rhabdomyosarcoma relapse despite appropriate treatment and experience a poor outcome. Little meaningful improvement in the outcome of this disease has been observed over the last 30 years. There is no clear international recommendation concerning the use of salvage chemotherapy ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.25622
更新日期:2015-11-01 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 report by the Radiation Oncology Discipline of Children's Oncology Group (COG) describes the practice patterns of pediatric image-guided radiotherapy (IGRT) based on a member survey and provides practice recommendations accordingly. The survey comprised of 11 vignettes asking clinicians about their recommended tr...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28629
更新日期:2020-10-01 00:00:00
abstract:BACKGROUND:Few data exist regarding the clinical outcome of patients with Ewing sarcoma (EWS) treated with pencil beam scanning proton therapy (PT). We report the outcome of children, adolescents and young adults (AYA) treated with PT at the Paul Scherrer Institute. MATERIALS:Thirty-eight patients (median age, 9.9 yea...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26688
更新日期:2017-12-01 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:BACKGROUND:Approximately one-third of patients with osteosarcoma who have a complete response to their initial treatment can be expected to relapse. It is important to define what host, tumor, or treatment characteristics determine outcome after relapse. We present findings in 59 patients treated at our institution fro...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20580
更新日期:2006-09-01 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
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:BACKGROUND:Self-concept was compared between adult survivors of childhood acute lymphoblastic leukemia (ALL) and sibling controls. Adult survivor subgroups at greatest risk for negative self-concept were identified. PROCEDURE:Survivors (n = 578) aged > or =18 years, treated before age 20 years on Children's Cancer Gro...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.10434
更新日期:2004-03-01 00:00:00
abstract:BACKGROUND:Radiotherapy (RT) is well established in the management of intracranial ependymoma (EP) and post-operative RT is employed for the majority of patients. There are no randomised trials of RT in EP and evidence for dose and volume relies on retrospective single institution series, usually comprising a heterogen...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.10470
更新日期:2004-05-01 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::Nutrition therapy is a therapeutic approach to treating medical conditions and symptoms via diet, which can be done by oral, enteral or parenteral routes. It is desirable to include nutritional interventions as a standard of care in pediatric cancer units (PCUs) at all levels of care. The interventions are dependent o...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.28378
更新日期:2020-06-01 00:00:00
abstract::Children suffering from Acute Lymphoblastic Leukaemia (ALL) treated with asparaginase and corticosteroids are at risk of developing severe lipid abnormalities. The authors report the case of a 10-year-old male with extremely high plasma triglyceride concentrations (4,000 mg/dl) during the induction phase of ALL associ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20986
更新日期:2008-02-01 00:00:00