Abstract:
BACKGROUND:The aim of the study was to investigate factors affecting response to everolimus, a mammalian-target-of-rapamycin (mTOR) inhibitor, of subependymal giant cell astrocytomas (SEGA) in patients with tuberous sclerosis complex (TSC). METHODS:The study group consisted of 15 children with a diagnosis of TSC-related SEGA. Median therapy duration was 13 months. Age, sex, previous neurosurgical or mTOR inhibitor treatment, everolimus blood concentration and anticonvulsant therapy were analyzed as potential factors affecting reduction of SEGA tumor volume. RESULTS:Significant reductions in SEGA volumes were noted at 3 and 6 months (median tumor volume 0.97 cm(3) and 0.70 cm(3) , respectively, versus 2.70 cm(3) at baseline, P = 0.001). Responses were observed in 11/15 (73.3%) and 10/12 (83.3%) patients at 3 and 6 months, respectively. The most rapid reduction of SEGA volume (58.6%) was found during the initial 3 months of treatment. There was no statistical difference in the extent of SEGA volume reduction between patients with everolimus trough levels <5 ng/ml and ≥5 ng/ml. Patients treated with ≤1 anticonvulsant had greater tumor reduction after 6 months of treatment. CONCLUSIONS:Everolimus is an effective and safe treatment option for TSC-related SEGA. Drug dose titration according to blood concentration did not appear to be crucial to achieve clinical efficacy; however, concomitant anticonvulsant therapy may affect response to mTOR inhibitors.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Trelinska J,Dachowska I,Kotulska K,Baranska D,Fendler W,Jozwiak S,Mlynarski Wdoi
10.1002/pbc.25368subject
Has Abstractpub_date
2015-04-01 00:00:00pages
616-21issue
4eissn
1545-5009issn
1545-5017journal_volume
62pub_type
临床试验,杂志文章abstract:BACKGROUND:Juvenile xanthogranuloma (JXG) is the most common non-Langerhans cell histiocytosis in children. The mortality and morbidity of JXG with extracutaneous lesions remain unclear. METHODS:Data of patients aged < 18 years who were diagnosed with JXG between 2001 and 2010 were retrospectively collected through a ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28381
更新日期:2020-07-01 00:00:00
abstract:BACKGROUND:To evaluate outcomes of Group D eyes of bilateral retinoblastoma patients treated with primary chemoreduction and external beam radiation as salvage. PROCEDURE:Retrospective chart review of patients diagnosed with bilateral retinoblastoma and designated Group D in at least one eye from January 1, 2000 to De...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24303
更新日期:2013-04-01 00:00:00
abstract:BACKGROUND:The awareness that adolescents can have cancer is probably insufficient, not only among teenagers and their families, but also among physicians, and adolescent patients are reportedly often referred to qualified cancer institutes after a considerable delay. PROCEDURE:A prospective series of 425 patients (28...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24312
更新日期:2013-04-01 00:00:00
abstract::Beckwith-Wiedemann syndrome (BWS) is a genetic overgrowth and cancer predisposition syndrome, associated with both benign and malignant adrenal findings. Literature review and an institutional case series elucidate the wide spectrum of adrenal findings in BWS patients. The altered expression of the 11p15 region is lik...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.26432
更新日期:2017-08-01 00:00:00
abstract::With improvements in therapy for childhood cancer, the expectation that most childhood cancer patients will survive and enter adulthood is a reality. There is clear evidence that survivors are at risk for adverse health-related long-term sequelae associated with their cancer and its treatment, requiring appropriate he...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20611
更新日期:2006-02-01 00:00:00
abstract:BACKGROUND:Choroid plexus tumors (CPT) are rare, and predominate in early childhood. An association with the Li-Fraumeni syndrome (LFS) has been reported, but the biological and clinical implications of this association remain poorly defined. We have investigated the clinical features and overall survival of all CPT pa...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23349
更新日期:2012-06-01 00:00:00
abstract::We describe 12 pediatric patients (8-16 years) with primary refractory (N = 6) or first relapse (N = 6) Hodgkin lymphoma (HL) treated with ifosfamide, gemcitabine, and vinorelbine (IGEV). The overall response rate to IGEV was 100%, with seven (58%) complete responses (CR) and five (42%) partial responses. Successful C...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.28167
更新日期:2020-04-01 00:00:00
abstract:BACKGROUND:The purpose of this study was to determine the prevalence of pulmonary dysfunction in pediatric hematopoietic cell transplant (HCT) survivors and to identify associated risk factors. PROCEDURE:In a cross-sectional study, patients surviving at least 5 years after pediatric HCT were requested to undergo pulmo...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20531
更新日期:2006-10-15 00:00:00
abstract:BACKGROUND:Primary mediastinal germ cell tumors (GCTs) are rare in children and still represent a challenge for both adult and pediatric oncologists because of their worse outcome compared to their gonadal counterpart. PROCEDURE:Prospectively collected data concerning patients enrolled in the Italian Association of Pe...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.25895
更新日期:2016-05-01 00:00:00
abstract:BACKGROUND:Ewing sarcoma (EWS) is rarely diagnosed as a second malignancy. We sought to describe a cohort of patients with secondary EWS and investigate if patient characteristics and survival differ between patients with secondary and primary EWS. PROCEDURE:Patients with EWS or peripheral primitive neuroectodermal tu...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24251
更新日期:2013-04-01 00:00:00
abstract::Severe congenital neutropenia (SCN) is a bone marrow failure disease with an autosomal dominant inheritance from mutations in ELANE. Here, we report a 7-week-old Korean male with SCN. His elder sister died from pneumonia at 2 years. Direct sequencing of ELANE in the proband identified a heterozygous novel frameshift m...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25654
更新日期:2015-12-01 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:The aim of this study was to evaluate the value of follow-up investigations of T-cell acute lymphoblastic leukemia (T-ALL) and T-cell non-Hodgkin's lymphoma (T-NHL), including cerebrospinal fluid (CSF) examination, bone marrow (BM) aspiration, peripheral blood (PB) count, serum lactate dehydrogenase (LDH) an...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20813
更新日期:2007-04-01 00:00:00
abstract:BACKGROUND:Resource-rich countries tend to have a higher incidence of childhood acute lymphoblastic leukemia (ALL), whereas lower rates are seen in more deprived countries. This study describes the incidence of childhood acute leukemia in Brazil, an upper middle-income country, based on data from 16 population-based ca...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22736
更新日期:2011-01-01 00:00:00
abstract::As increasing numbers of childhood cancer patients are surviving, the long-term complications of the disease and its treatment have become ever more increasingly important. Reduced bone mineral density and increased fracture risk have been reported during and after treatment of children with cancer. The causes of oste...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.21407
更新日期:2008-02-01 00:00:00
abstract::2-Chlorodeoxyadenosine (2-CdA) has been successfully used in children to treat refractory Langerhans cell histiocytosis and juvenile xanthogranuloma (JXG) as salvage therapy. Although 2-CdA is generally well-tolerated, with temporary myelosuppression as the primary dose-limiting toxicity, prolonged myelosuppressive, a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23087
更新日期:2012-02-01 00:00:00
abstract:BACKGROUND:The Paediatric Oncology Centres (POCs) treating childhood cancer in South East England produce unified supportive care guidelines for use in the secondary pediatric (shared care) units. This study evaluated the adherence to current guidelines for febrile neutropenia (FN) and documented outcome in terms of ba...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21041
更新日期:2007-10-15 00:00:00
abstract:BACKGROUND:Children with cancer have high utilization of the emergency department (ED), but little is known about which outcomes are most important to them and their caregivers when they seek care in the ED. PROCEDURE:A qualitative evaluation of ED experience for children with cancer and their caregivers was performed...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27903
更新日期:2019-10-01 00:00:00
abstract::Risk-adapted, response-based therapies for pediatric Hodgkin lymphoma have resulted in 5-year survival exceeding 90%. Although high-dose chemotherapy and autologous hematopoietic stem cell transplantation (AHSCT) are considered standard for most patients with relapsed or refractory Hodgkin lymphoma, a subset of childr...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24851
更新日期:2014-04-01 00:00:00
abstract:BACKGROUND:Sleep disordered breathing (SDB) is emerging as a significant health condition for children. The purpose of this study is to evaluate SDB symptoms in childhood cancer survivors and identify associations with quality of life (QOL) and psychological symptoms. PROCEDURE:A sample of 62 survivors aged 8-18 years...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25394
更新日期:2015-04-01 00:00:00
abstract:INTRODUCTION:Major advances in the field of pediatric oncology have resulted from rigorous, prospective clinical oncology research trials. Optimizing access for all children and adolescents to clinical research trials is an important goal. Barriers to clinical trial enrollment are numerous, involving the health care sy...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28023
更新日期:2020-04-01 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::We evaluated the in vitro glucocorticoid (GC) responsiveness of 117 pediatric acute myeloid leukemia cells by considering GC resistance, GC-induced proliferation, and GC-induced differentiation. None of the samples was highly GC sensitive, and only 15% were intermediately sensitive. GC-induced differentiation was not ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26011
更新日期:2016-08-01 00:00:00
abstract:BACKGROUND:Children with terminal cancer and their families describe a preference for home-based end-of-life care. Inadequate support outside of the hospital is a limiting factor in home location feasibility, particularly in rural regions lacking pediatric-trained hospice providers. METHODS:The purpose of this longitu...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28921
更新日期:2021-02-01 00:00:00
abstract:BACKGROUND:Tumor cells frequently contaminate autologous stem cell products in a variety of malignancies, but their clinical significance remains controversial. We retrospectively monitored tumor contamination in stem cell harvests from patients with Ewing family of tumors (EFT) all harboring the specific translocation...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20022
更新日期:2004-05-01 00:00:00
abstract::Diffuse intrinsic pontine glioma (DIPG) is characterized by a short history of brainstem symptoms and well-known magnetic resonance imaging features with a fatal outcome. However, we report three unusual cases of brainstem tumors with an initial indolent and protracted course, which subsequently developed the classica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28102
更新日期:2020-03-01 00:00:00
abstract::Two thirds of children with cancer never reach a specialist centre for treatment in South Africa. The majority of those who present have advanced disease. A campaign was undertaken to educate the public and the primary health workers on the Saint Siluan early warning signs of cancer in children. There was a statistica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22853
更新日期:2011-02-01 00:00:00
abstract:BACKGROUND:Fanconi anaemia is a genetic disease characterized by congenital abnormalities, progressive bone marrow failure, and a higher predisposition of oral squamous cell carcinoma. The purpose of this study was to evaluate the prevalence of oral mucosa lesions in patients with Fanconi anaemia without hematopoietic ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25417
更新日期:2015-06-01 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:Veno-occlusive disease (VOD) is a major complication following hematopoietic stem cell transplantation (HSCT). Its diagnosis is based on clinical criteria, which have a limited sensitivity. Increased plasminogen activator inhibitor-1 (PAI-1) levels have been suggested as a marker of VOD. We aimed to prospect...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23213
更新日期:2012-05-01 00:00:00