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 case of a child with advanced PMH relapsing after surgery and chemotherapy. Sirolimus achieved significant clinical improvement and stabilization of the lesions without any remarkable toxicity. This case contributes to the growing evidence regarding the efficacy of mTOR inhibitors, such as sirolimus, in multifocal PMH.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Gabor KM,Sapi Z,Tiszlavicz LG,Fige A,Bereczki C,Bartyik Kdoi
10.1002/pbc.26781subject
Has Abstractpub_date
2018-02-01 00:00:00issue
2eissn
1545-5009issn
1545-5017journal_volume
65pub_type
杂志文章abstract:OBJECTIVE:To examine the level of children's understanding of informed consent in clinical trials and factors that may influence these processes. DESIGN:Twenty nine children who were included in clinical trials for treatment of cancer or HIV, were offered the possibility to complete a semidirective interview, with par...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.21359
更新日期:2008-05-01 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:Distinguishing alveolar rhabdomyosarcoma (ARMS) from embryonal rhabdomyosarcoma (ERMS) has historically been of prognostic and therapeutic importance. However, classification has been complicated by shifting histologic criteria required for an ARMS diagnosis. Children's Oncology Group (COG) studies after IRS...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26645
更新日期:2017-12-01 00:00:00
abstract:BACKGROUND:Extramedullary infiltration (EMI) is an occasional clinical symptom in childhood acute myelogenous leukemia (AML), but there is considerable controversy regarding the prognostic significance of EMI in AML. PROCEDURE:We evaluated the frequency and prognostic significance of EMI at diagnosis of AML in childre...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20824
更新日期:2007-04-01 00:00:00
abstract::Children with sickle cell anemia (SCA) have low bone mass though bone turnover has not been well described. In this study, growth and pubertal development were assessed twice, 1 year apart, in 80 young subjects with type-SS SCA, while whole body bone mineral content (BMC) and density where measured in a subset (n = 46...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21147
更新日期:2008-03-01 00:00:00
abstract:BACKGROUND:Previous studies of children with homozygous sickle cell anemia (SCA) show impaired growth and maturation. The correlation of this suboptimal growth with metabolic and hematological factors during puberty is poorly understood. PROCEDURE:We studied a group of pre-adolescent children with SCA (19 males, 14 fe...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22137
更新日期:2009-10-01 00:00:00
abstract::Pediatric cancer survivors have increased risk of obesity, hypertension, dyslipidemia, and type 2 diabetes, leading to premature cardiovascular disease (CVD). Multiple tissues that are involved in glucose homeostasis and lipid metabolism are adversely affected by chemotherapy. This review highlights the relevant tissu...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24703
更新日期:2013-12-01 00:00:00
abstract::We report the case of a drug interaction between methotrexate (MTX) and chloral hydrate (CH) observed in a child treated for acute leukemia. Significantly slower MTX clearance and increased MTX exposure occurred on the first three courses of a high-dose chemotherapy when co-administered with CH despite normal renal fu...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24393
更新日期:2013-03-01 00:00:00
abstract::In this retrospective study of patients with overt orbital retinoblastoma, we evaluated minimally disseminated disease (MDD) in bone marrow and cerebrospinal fluid (CSF) using CRX and/or GD2 synthase as markers. Ten patients were evaluated-five (50%) at diagnosis and five upon relapse. MDD was detected in four cases (...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27662
更新日期:2019-06-01 00:00:00
abstract::Langerhans cell histiocytosis is known to mimic many other conditions. We present two patients where anti-tubercular therapy was instituted when clinical and radiological features suggested tuberculosis. The correct diagnosis of histiocytosis was reached only on further work-up including immunohistochemistry following...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21930
更新日期:2009-07-01 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:Children with hepatoblastoma (HB) are at risk of sarcopenia due to immobility, chemotherapy, and malnutrition. We hypothesized that children with HB have a low preoperative total psoas muscle area (tPMA), reflecting sarcopenia, which negatively impacts outcome. PROCEDURE:Retrospective study of children (1-1...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28862
更新日期:2021-01-12 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:BACKGROUND:Contemporary descriptions of classical Hodgkin lymphoma (cHL) are lacking from sub-Saharan Africa where human immunodeficiency virus (HIV) and Epstein-Barr virus (EBV) are prevalent. METHODS:We describe a prospective cHL cohort in Malawi enrolled from 2013 to 2015. Patients received standardized treatment a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26302
更新日期:2017-05-01 00:00:00
abstract:BACKGROUND:Sickle cell disease (SCD) is an autosomal recessive hemoglobinopathy associated with morbidity and mortality. We sought to understand family planning intentions of parents of young children with SCD including the awareness of three reproductive options (adoption, in vitro fertilization with egg/sperm donatio...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28227
更新日期:2020-05-01 00:00:00
abstract:BACKGROUND:Several sickle cell clinical trials have closed due to inability to enroll patients. To limit the early cessation of a proposed clinical trial due to low accrual rates, we sought to better understand barriers and facilitators to enrolling parents of children with sickle cell anemia (SCD) into clinical trials...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.24486
更新日期:2013-08-01 00:00:00
abstract::Malignant melanoma is rare in childhood and has never been reported to cause pancytopenia due to bone marrow metastases in a child. We report a 3-year-old boy with a large congenital melanocytic nevus who presented with bone pain and pancytopenia due to diffuse bone and bone marrow infiltration with metastatic melanom...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20200
更新日期:2005-07-01 00:00:00
abstract::A diverse panel of pediatric cancer advocates and experts, whose collective experience spans the continuum of international academic medicine, industry, government research, and cancer advocacy, recently discussed challenges for pediatric cancer research in the context of coronavirus disease 2019 (COVID-19). Specifica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.28435
更新日期:2020-09-01 00:00:00
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 pat...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24566
更新日期:2013-09-01 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:Traditionally, physicians have believed that limb-salvage surgery has functional and cosmetic advantages over amputation, yet the literature is equivocal. Therefore, we sought to compare the psychosocial and functional outcomes in osteosarcoma survivors after limb-salvage surgery and amputation. We hypothesi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22419
更新日期:2010-07-01 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::Juvenile xanthogranuloma (JXG) is a disorder of disputed origin thought to be related to the dermal/interstitial macrophage. A 5-year-old female presented with an aggressive systemic JXG that developed 5 months after the diagnosis of T-cell acute lymphoblastic leukemia (T-ALL). Examination of the T-cell receptor gamma...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22756
更新日期:2011-05-01 00:00:00
abstract:BACKGROUND:In malaria-endemic countries in West Africa, sickle cell disease (SCD) contributes to childhood mortality. Historically, Liberia had regions wherein hemoglobin S and beta-thalassemia trait were mutually exclusive. Data on hemoglobinopathies in the Monrovia, the capital, are outdated and do not reflect urban ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25875
更新日期:2016-04-01 00:00:00
abstract:BACKGROUND:Little is known about the visual outcome of children affected by an optic pathway glioma (OPG). PROCEDURES:We evaluated the long-term visual outcome of 32 consecutive children affected by OPG without neurofibromatosis type-1 referred to the Pediatric Department of Padua University and managed according to s...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.22748
更新日期:2010-12-01 00:00:00
abstract::Opportunities for cancer prevention in adolescents range from limitation of sun exposure to the use of human papillomavirus vaccines. Those who develop malignant disease experience longer waiting times for diagnosis and treatment than do children, especially when referred to adult treatment centers, and they are less ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20634
更新日期:2006-03-01 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::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::Phytosterolemia is a rare autosomal recessive sterol storage disease caused by mutations in ABCG5 and ABCG8 genes. A 9-year-old Turkish boy who was presented with exclusively hematologic abnormalities had elevated plant sterol levels. Sequencing of ABCG5 and ABCG8 genes revealed a novel homozygous IVS10-1 G>T mutation...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24934
更新日期:2014-08-01 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