Abstract:
:The FIP1L1-PDGFRA fusion gene is the most frequent genetic aberration in myeloid neoplasms associated with eosinophilia and abnormalities of PDGFRA, PDGFRB, or FGFR1. Affected patients in adult populations are very sensitive to imatinib therapy. Pediatric cases are rare and so far only one case of FIP1L1-PDGFRA positive disease has been reported. We report a 2-year-old female with a myeloid neoplasm associated with eosinophilia and rearrangement of PDGFRA. Treatment with imatinib resulted in complete and durable clinical, hematological, and molecular remission within 3 months after starting treatment.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Rathe M,Kristensen TK,Møller MB,Carlsen NLdoi
10.1002/pbc.22655subject
Has Abstractpub_date
2010-10-01 00:00:00pages
730-2issue
4eissn
1545-5009issn
1545-5017journal_volume
55pub_type
杂志文章abstract:BACKGROUND:Transcranial Doppler ultrasound (TCD) has been demonstrated to be a powerful predictor of stroke risk due to sickle cell disease (SCD) in pediatric populations. Little is known about how this healthcare innovation has disseminated into preventive care for SCD. The objective of this study was to determine TCD...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21677
更新日期:2008-11-01 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:Hemophagocytic Lymphohistiocytosis (HLH) is characterized by uncontrolled inflammation that is generally fatal without immune modulating chemotherapy. At Texas Children's Hospital, we have observed significant central nervous system (CNS) toxicity in several patients treated for HLH according to the Histiocy...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21838
更新日期:2009-05-01 00:00:00
abstract:INTRODUCTION:The rate of bacterial infections in children with sickle cell disease (SCD) has decreased in recent years, mainly due to penicillin prophylaxis and vaccination. OBJECTIVES:To determine the rate of severe bacterial infection (SBI) in a cohort of children with SCD and to describe low-risk factors for confir...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27667
更新日期:2019-06-01 00:00:00
abstract:BACKGROUND:Progressive/recurrent high-grade and diffuse intrinsic pontine gliomas (DIPGs) are fatal. Treatments targeting molecular pathways critical for these cancers are needed. METHODS:We conducted a phase 1 study (rolling-six design) to establish the safety and maximum tolerated dose (MTD) of dasatinib, an oral pl...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27035
更新日期:2018-07-01 00:00:00
abstract::Because of increasing survival rates in pediatric oncology, attention is focusing on cancer and its treatment-related side effects. Rehabilitation may reduce their impact. However, the literature does not provide strong evidence regarding rehabilitation pathways. Therefore, the Italian Association of Pediatric Hematol...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.28681
更新日期:2020-12-01 00:00:00
abstract:BACKGROUND:Compared to X-ray radiation therapy, proton therapy (PT) reduces the radiation dose to organs at risk, which is expected to translate into fewer second cancers and less cardiac morbidity decades after treatment. The Children's Oncology Group high-risk pediatric Hodgkin lymphoma (PHL) protocol, AHOD1331, allo...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26044
更新日期:2016-09-01 00:00:00
abstract::During ALL chemotherapy, a 4-year-old patient presented with febrile neutropenia and abdominal pain. Ultrasound examinations were repeatedly normal. Computerized tomography on day 7 demonstrated appendicitis and multiple hepatic foci identified as mucormycosis (Absidia corymbifera). Successful outcome was achieved by ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21918
更新日期:2009-07-01 00:00:00
abstract:BACKGROUND:Neurocognitive impairment occurs in 20-40% of childhood acute lymphoblastic leukemia (ALL) survivors, possibly mediated by folate depletion and homocysteine elevation following methotrexate treatment. We evaluated the relationship between folate pathway polymorphisms and neurocognitive impairment after child...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23162
更新日期:2011-09-01 00:00:00
abstract:BACKGROUND:Abandonment of treatment is one of the toughest challenges to deal with in pediatric oncology. It leads to unnecessary mortality and morbidity in patients from low- and middle-income countries. PROCEDURE:The objective of our retrospective study was to determine the prevalence and predictors for abandonment ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26818
更新日期:2018-02-01 00:00:00
abstract::We report two second malignant neoplasms (SMNs) of the parotid gland. Patient 1 was initially diagnosed with precursor B-cell lymphoblastic lymphoma of the scalp. Eight years after her initial diagnosis she presented with a small, painless mass in the region of her parotid gland. Patient 2 was diagnosed with pre-B-cel...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20306
更新日期:2005-12-01 00:00:00
abstract:BACKGROUND:The aim was to evaluate self-reported reproductive characteristics and markers of ovarian function in a nationwide cohort of female survivors of childhood acute lymphoblastic leukemia (ALL), because prior investigations have produced conflicting data. PROCEDURE:Self-reported reproductive characteristics wer...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28894
更新日期:2021-01-18 00:00:00
abstract:BACKGROUND:As pegylated asparaginase is becoming the preferred first-line asparaginase preparation in the chemotherapy regimens of childhood acute lymphoblastic leukemia (ALL), there is a need to evaluate this treatment. METHODS:The aim of this study was to evaluate the pharmacokinetics of prolonged upfront biweekly P...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.26686
更新日期:2017-12-01 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:BACKGROUND:Recent studies suggest that SLCO1B1 c.521T > C variant decreases the clearance of methotrexate (MTX) and elevates its plasma concentration, hence leucovorin doses may need to be adjusted. However, high leucovorin doses may affect the cure rate in childhood acute lymphoblastic leukemia (ALL). Hitherto neither...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25191
更新日期:2014-12-01 00:00:00
abstract::Acute bilineal leukemias are rare and are commonly associated with t(9;22) and MLL abnormalities. Herein, we report a pediatric case of bilineal T/myeloid acute leukemia associated with del (9q)(q13q22) and TLX3/BCL11B fusion due to the cryptic t(5;14)(q35;32). FISH studies confirmed the TLX3/BCL11B fusion in both the...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22850
更新日期:2011-03-01 00:00:00
abstract:BACKGROUND:Advanced irradiation techniques, including intensity-modulated radiation therapy (IMRT), aim to limit irradiation to adjoining tissues by conforming beams to a well-defined volume. In intracranial germinomas, whole-ventricular IMRT decreases the volume of irradiation to surrounding parenchyma. This study exa...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28005
更新日期:2019-12-01 00:00:00
abstract::Pseudomyogenic hemangioendothelioma (PMH) is a rare, mostly indolent vascular tumor. Extensive cases are treated with amputation as chemotherapy seems to be ineffective. Recently, promising results were published using mammalian target of rapamycin (mTOR) inhibitors in tumors of vascular origin. Here, we present a cas...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26781
更新日期:2018-02-01 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::Chronic hypoxemia is a common manifestation among patients with sickle cell anemia (SCA) who develop chronic lung disease. We report the beneficial effect of hydroxyurea on chronic hypoxemia in three pediatric patients with SCA and recurrent episodes of acute chest syndrome (ACS). All three patients improved rapidly a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21480
更新日期:2008-06-01 00:00:00
abstract:BACKGROUND:Although hematopoietic stem cell transplantation (HSCT) is offered as a curative therapy for pediatric myelodysplastic syndrome (MDS), it may cause severe complications and mortality. Several reports have shown the efficacy of immunosuppressive therapy (IST) in adult patients with refractory anemia (RA), but...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.22121
更新日期:2009-12-01 00:00:00
abstract:BACKGROUND:Sorafenib is an inhibitor of multiple kinases (e.g., VEGF receptors, PDGFR, FLT3, RET, BRAF, KIT) and is approved by FDA for treatment of two adult cancers. The activity of sorafenib was evaluated against the PPTP's in vitro and in vivo panels. PROCEDURES:Sorafenib was evaluated against the PPTP in vitro pa...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22712
更新日期:2010-12-01 00:00:00
abstract::Hepatoblastoma (HB) and biliary atresia (BA) are both rare conditions that occurred in the patient described. This is the second such case in the literature. An explanation for this apparent coincidence could possibly be found in the existance of pluripotent liver stem cells. In humans, small epithelial cells (SEC) be...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20115
更新日期:2004-10-01 00:00:00
abstract:BACKGROUND:Diamond Blackfan Anemia (DBA) is a rare congenital, bone marrow failure syndrome characterized by normochromic macrocytic anemia, reticulocytopenia and absence or insufficiency of erythroid precursors in normocellular bone marrow, frequently associated with somatic malformations. Here, we present our finding...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25183
更新日期:2014-12-01 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::A 4-year-old girl with Noonan syndrome (NS) and constitutive PTPN11 mutation presented with stage 4 neuroblastoma and was treated by intensive chemotherapy. During the treatment, cytogenetic analysis revealed the development of a hyperdiploid clone with duplication of the germline PTPN11 mutation in a morphologically ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20527
更新日期:2007-01-01 00:00:00
abstract:BACKGROUND:Malignant peripheral nerve sheath tumor (MPNST) is an aggressive form of soft-tissue sarcoma (STS) in children. Despite intensive therapy, relatively few children with metastatic and unresectable disease survive beyond three years. RAS pathway activation is common in MPNST, suggesting MEK pathway inhibition ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28639
更新日期:2020-12-01 00:00:00
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:Infection in neutropenic children is a major cause of morbidity and mortality in children treated for cancer. In developing countries, children with cancer are often malnourished at diagnosis. In Blantyre, Malawi, children with Burkitt lymphoma are treated with a local protocol with limited toxicity. The aim...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22032
更新日期:2009-07-01 00:00:00
abstract::Hospital-based home care (HBHC) is widely applied in Pediatric Oncology. We reviewed the potential effect of HBHC on children's physical health and risk of adverse events, parental and child satisfaction, quality of life of children and their parents, and costs. A search of PubMed, CINAHL, and EMBASE led to identifica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.23047
更新日期:2011-09-01 00:00:00