Abstract:
BACKGROUND:Alveolar soft-part sarcoma (ASPS), a rare vascular sarcoma with a clinically indolent course, frequently presents with metastases. Vascular endothelial growth factor (VEGF) is a promising therapeutic target. In a phase-II trial of the VEGF receptor inhibitor cediranib for adults with ASPS, the partial response (PR) rate (response evaluation criteria in solid tumors [RECIST] v1.0) was 35% (15/43; 95% confidence interval: 21-51%). We evaluated cediranib in the pediatric population. PROCEDURE:Patients <16 years old with metastatic, unresectable ASPS received cediranib at the pediatric maximum tolerated dose of 12 mg/m2 (≈70% of the fixed adult phase-II dose orally daily). Tumor response was assessed every two cycles (RECIST v1.0). A Simon two-stage optimal design (target response rate 35%, rule out 5%) was used. RESULTS:Seven patients (four females), with a median age of 13 years, (range 9-15), were enrolled on stage 1. The most frequent grade 2 or 3 adverse events were neutropenia, diarrhea, hypertension, fatigue, and proteinuria. The best response was stable disease (SD) (median cycle number = 34). Three patients were removed from the study treatment for disease progression (cycles 4, 5, and 36). Five of seven patients had SD for ≥14 months. Two patients with SD remain on study (34-57+ cycles). CONCLUSIONS:Cediranib did not reach the target response rate in this small pediatric cohort, in contrast to the adult 35% PR rate. The pediatric dosing was 30% lower compared to the adult dosing, which may have contributed to response differences. Prolonged SD was observed in five patients, but given the indolent nature of ASPS, SD cannot be clearly attributed to cediranib. Cediranib has an acceptable safety profile.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Cohen JW,Widemann BC,Derdak J,Dombi E,Goodwin A,Dompierre J,Onukwubiri U,Steinberg SM,O'Sullivan Coyne G,Kummar S,Chen AP,Glod Jdoi
10.1002/pbc.27987subject
Has Abstractpub_date
2019-12-01 00:00:00pages
e27987issue
12eissn
1545-5009issn
1545-5017journal_volume
66pub_type
杂志文章,多中心研究abstract:BACKGROUND:The clinical impact of influenza in children undergoing therapy for cancer is not well-described in the literature. PROCEDURE:Laboratory-documented influenza infection in pediatric oncology patients cared for in a single regional pediatric medical center between July 2000 and June 2005 was identified by rev...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.21472
更新日期:2008-05-01 00:00:00
abstract:BACKGROUND:Childhood germ cell tumors (cGCTs), believed to arise from transformed primordial germ cells by an unknown mechanism, provide a unique model system for investigating cell signaling, pluripotency, and the microenvironment of neoplastic stem cells (NSCs) in vivo. This is the first report of proteomics of cGCTs...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23282
更新日期:2012-05-01 00:00:00
abstract:BACKGROUND:In low- and middle-income countries, therapeutic options for advanced, refractory, or relapsing malignancies are limited due to local constraints such as cost of drugs, distance from oncology centers, and lack of availability of new anticancer drugs. Metronomics, which combines metronomic chemotherapy (MC) a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28508
更新日期:2020-09-01 00:00:00
abstract:BACKGROUND:The median age of patients with Ewing sarcoma (EwS) at diagnosis is around 14-15 years. Older age is associated with a worse outcome. The correlation of age at diagnosis on sites of disease has not been fully described. OBJECTIVE:The goal of this study was to evaluate the differences in sites of primary tum...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27251
更新日期:2018-09-01 00:00:00
abstract::This study describes hydroxyurea use among children ages 1 to 17 with sickle cell anemia (SCA) enrolled in at least one year of Medicaid in six states from 2005 to 2012. Administrative claims were used to summarize the number of days' supply of hydroxyurea dispensed by state and year. A total of 7963 children with SCA...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27721
更新日期:2019-06-01 00:00:00
abstract::Familial hemophagocytic lymphohistiocytosis (FHLH) is an autosomal recessive disorder of cytotoxic cell function that results in abnormal proliferation of benign lymphocytes and histiocytes in response to infectious stimuli. FHLH generally occurs in very young children, and typically presents with fever, cytopenias, c...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21438
更新日期:2008-05-01 00:00:00
abstract:BACKGROUND:Patients with Langerhans cell histiocytosis (LCH) may develop neurodegeneration and significant CNS sequelae, affecting a significant proportion of the patients. We here aimed to investigate the neuropsychological consequences in more detail. METHODS:Using an extensive neuropsychological test battery, we ev...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21656
更新日期:2008-11-01 00:00:00
abstract:BACKGROUND:To investigate the incidence, clinical characteristics and survival of malignant tumours of the gastrointestinal tract in children in the West Midlands in Britain over a 44-year span time, to identify any change over this period and to compare the data with the world literature. PROCEDURE:Retrospective popu...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20086
更新日期:2004-09-01 00:00:00
abstract::The fundamental purposes underlying formal health care transition from the pediatric to adult setting for young adult survivors of childhood cancer are to facilitate the continuous, medically and developmentally appropriate implementation of risk-based guidelines for the monitoring and management of late effects of ch...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.21455
更新日期:2008-05-01 00:00:00
abstract:BACKGROUND:(131) I-metaiodobenzylguanidine ((131) I-MIBG) is a targeted radiopharmaceutical for patients with neuroblastoma. Despite its tumor-specific uptake, the treatment with (131) I-MIBG results in whole-body radiation exposure. Our aim was to correlate whole-body radiation dose (WBD) from (131) I-MIBG with tumor ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25816
更新日期:2016-03-01 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::We report a novel glucose-6-phosphate dehydrogenase (G6PD) mutation, which we propose to name G6PD Cincinnati (c.1037A > T, p.N346I), found in combination with G6PD Gastonia (c.637G > T, p.V213L) in an infant who presented with neonatal cholestasis. The G6PD Cincinnati mutation results in a non-conservative amino acid...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22744
更新日期:2011-05-01 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: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:Hepatic veno-occlusive disease (VOD) is one of the most serious complications in stem cell transplantation (SCT). Although plasma protein C activity decreases in VOD after SCT, the timeframe of plasma protein C activity decreases during SCT is not known. PROCEDURE:We examined levels of plasma protein C seri...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22314
更新日期:2010-03-01 00:00:00
abstract:BACKGROUND:Prophylactic use of cranial radiation therapy (CRT) in young children with acute lymphoblastic leukemia (ALL) is associated with significant long-term morbidity. Therefore, current treatment protocols for pediatric B-precursor ALL have abandoned prophylactic CRT in favor of intrathecal chemotherapy, combined...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.10392
更新日期:2004-01-01 00:00:00
abstract:BACKGROUND:The Wnt/β-catenin pathway plays a central role in the pathogenesis of most hepatoblastomas (HBs), that is, up to 60-80% carry activating CTNNB1 mutations. HBs can however also be the first manifestation of familial adenomatous polyposis (FAP). As this is a severe disease, it is important for the patient and ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26991
更新日期:2018-06-01 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::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::Scrotal ulcers are a rare manifestation in patients with acute promyelocytic leukemia. Fournier's gangrene (FG) is even rarer. We describe three adolescents and young adults who developed scrotal ulcerations during induction with all-trans-retinoic acid. One patient developed FG. These lesions are predominantly seen i...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21549
更新日期:2008-08-01 00:00:00
abstract:OBJECTIVE:To develop an objective tool for assessing disease activity in patients with Langerhans cell histiocytosis (LCH). METHOD:Scoring system was developed and applied to a database containing information on 612 patients. RESULTS:At diagnosis, the score distribution was highly asymmetrical: the score was between ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20160
更新日期:2004-12-01 00:00:00
abstract:BACKGROUND/OBJECTIVES:β-Thalassemia intermedia (β-TI) accounts for up to one-fourth of β-thalassemia patients. Evaluating and improving quality of life (QOL) should be a goal in β-TI follow-up and management strategies. Patients' perceptions of their illness and its treatment may impact their QOL. This study aimed to e...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.27735
更新日期:2019-07-01 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:An adapted LMB 96 derived protocol for B-cell non-Hodgkin lymphoma (NHL) was implemented at the pediatric oncology unit of the Children Welfare Teaching Hospital in Baghdad (Iraq) from 2000 to present. The purpose was to evaluate the feasibility and efficacy of this intensive therapeutic regimen in a limited...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22905
更新日期:2011-04-01 00:00:00
abstract:BACKGROUND:Iron overload is well documented in patients with β-thalassemia major, and patients who have undergone hematopoietic stem cell transplantation (HSCT) remain at risk as a result of pre- and immediate post-HSCT transfusions. PROCEDURE:This is a prospective, randomized, 1-year clinical trial that compares the ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.26213
更新日期:2017-01-01 00:00:00
abstract::Wiskott-Aldrich syndrome (WAS) is characterized by primary immunodeficiency, thrombocytopenia and eczema. Patients with WAS have an increased risk to develop tumors. Non-Hodgkin lymphoma (NHL) represents the most common malignancy occurring in WAS-affected patients, diffuse-large-B-cell lymphoma is the most frequently...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23393
更新日期:2012-08-01 00:00:00
abstract:BACKGROUND:Silent cerebral infarcts are both morbid and progressive in children with sickle cell disease (SCD). While blood transfusion therapy is effective primary and secondary stroke prevention, the efficacy and acceptance of blood transfusion therapy for children with silent cerebral infarcts is unknown. The overal...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.21338
更新日期:2008-03-01 00:00:00
abstract:BACKGROUND:The level of minimal residual disease (MRD) prior to allogeneic hematopoietic stem cell transplantation (HSCT) has been shown to be an independent prognostic factor for outcome of pediatric patients with high-risk acute lymphoblastic leukemia (ALL). Retrospective studies which used (semi-) quantitation of cl...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.20794
更新日期:2007-01-01 00:00:00
abstract::Thrombocytopenia and autoimmune hemolytic anemia (Evans syndrome) with the presence of both warm and cold autoantibodies (mixed type) are rare in the pediatric age group. This condition may be associated with other autoimmune disorders and is notoriously difficult to treat. This case describes an adolescent male who p...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20312
更新日期:2005-09-01 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