Abstract：
BACKGROUND:Sorafenib targets multiple pathways thought to be crucial in growth of plexiform neurofibroma (PN) in children with neurofibromatosis type 1 (NF1). Sorafenib has been tolerated with manageable toxicities in adults and children with refractory cancer. We conducted a separate study in this population. Monitoring long-term toxicities such as effects on growth and obtaining additional pharmacokinetic data were of importance due to the young age and long duration of therapy seen in previous phase I trials in children with NF1. PROCEDURE:Children ≥3 and ≤18-year-old with NF1 and inoperable PN were eligible. Sorafenib was administered orally twice daily for consecutive 28-day cycles. Maximum tolerated dose (MTD) was determined from toxicities observed during the first three cycles. RESULTS:Nine children enrolled, median age 8 (6-12) years. At the starting 115 mg/m(2) /dose (n = 5), two experienced dose-limiting grade 3 pain in their PN. At the de-escalated 80 mg/m(2) /dose (n = 4), approximately 40% of the pediatric solid tumor MTD, two had dose-limiting toxicity (grade 3 rash and grade 4 mood alteration), exceeding the MTD. At 80 mg/m(2) /dose, the median AUC(0-12 hours) at steady-state was 39.5 µg hours/ml. Toxicities appeared to correspond with decreases in quality of life (QOL). No tumor shrinkage was observed. CONCLUSIONS:Children with NF1 and PN did not tolerate sorafenib at doses substantially lower than the MTD in children and adults with malignant solid tumors. Future trials with targeted agents for children with NF1 may require a more conservative starting dose and separate definitions of dose limiting toxicities (DLT) than children with cancer.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Kim A,Dombi E,Tepas K,Fox E,Martin S,Wolters P,Balis FM,Jayaprakash N,Turkbey B,Muradyan N,Choyke PL,Reddy A,Korf B,Widemann BCdoi
10.1002/pbc.24281subject
Has Abstractpub_date
2013-03-01 00:00:00pages
396-401issue
3eissn
1545-5009issn
1545-5017journal_volume
60pub_type
杂志文章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:Sickle cell disease (SCD) is an inherited blood disorder characterized by a chronic hemolytic anemia that can contribute to fatigue and global cognitive impairment in patients. The study objective was to report on the feasibility, reliability, and validity of the PedsQL™ Multidimensional Fatigue Scale in SCD...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章,多中心研究
doi：10.1002/pbc.24776
更新日期：2014-01-01 00:00:00
abstract：BACKGROUND:Ewing sarcoma (ES), a highly aggressive tumor of children and young adults, is characterized most commonly by an 11;22 chromosomal translocation that fuses EWSR1 located at 22q12 with FLI1, coding for a member of the ETS family of transcription factors. Although genetic changes in ES have been extensively re...
journal_title：Pediatric blood & cancer
pub_type： 临床试验,杂志文章,多中心研究
doi：10.1002/pbc.24526
更新日期：2013-09-01 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 successful bone marrow transplantation in an 11-year-old male with chronic myeloid leukemia from his HLA-identical sibling selected by preimplantation HLA testing. Because collection of cord blood failed, the transplantation was performed when the donor reached the age of 19 months, and sufficient bone marro...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.23007
更新日期：2011-08-01 00:00:00
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 respon...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章,多中心研究
doi：10.1002/pbc.27987
更新日期：2019-12-01 00:00:00
abstract：OBJECTIVES:Palliative radiation therapy (pRT) is often used to improve quality of life for pediatric patients. Though palliative doses are generally lower than those for cure, pRT may still introduce undesirable effects. The decision to pursue additional therapy for a child may be challenging and depends on parents' kn...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.28003
更新日期：2020-01-01 00:00:00
abstract：BACKGROUND:Methylenetetrahydrofolate reductase (MTHFR) C677T and A1298C polymorphisms have been implicated in childhood acute lymphoblastic leukemia (ALL) risk, but previously published studies were inconsistent and recent meta-analyses were not adequate. PROCEDURES:In a meta-analysis of 21 publications with 4,706 cas...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章,meta分析,评审
doi：10.1002/pbc.23137
更新日期：2012-04-01 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
abstract：:High-risk (HR) acute lymphoblastic leukemia (ALL) remains one of the greatest challenges in pediatric oncology. Relapsed ALL is a leading cause of death in young people, and further improvements in outcome will required the development of therapeutic approaches directed against rational therapeutic targets, as escalat...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章,评审
doi：10.1002/pbc.22996
更新日期：2011-06-01 00:00:00
abstract：:Global variations in the incidence of pediatric cancers have been described; however, the causes of such differences are not known. We investigated the relationship between the incidence of embryonal tumors and human development index on a global scale. Increasing incidence of neuroblastoma correlates significantly wi...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.26244
更新日期：2017-02-01 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:Neuroblastoma (NB) is the most common solid extracranial tumor in childhood. Despite advances in therapy, the prognosis is poor and optimized therapies are urgently needed. Therefore, we investigated the antitumor potential of interleukin-15 (IL-15)-activated cytokine-induced killer (CIK) cells against diffe...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.26147
更新日期：2016-12-01 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：BACKGROUND:Ewing sarcoma (ES) is a malignant tumor of bone and soft tissue of children and young adults. Patients with ES are treated with intensive chemotherapy regimens. We describe predictors of acute chemotherapy-associated toxicity in this population. PROCEDURE:In this retrospective cohort study, records of ES pa...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.24031
更新日期：2012-10-01 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：:A 12-year-old boy in third remission of an acute lymphoblastic leukaemia developed infection of lung and paranasal sinuses with Aspergillus flavus in neutropenia. Because of the high risk of leukaemia-relapse bone marrow transplantation (BMT) from a matched unrelated donor was carried out despite invasive pulmonary as...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.20302
更新日期：2005-06-15 00:00:00
abstract：BACKGROUND:The male excess in childhood cancer incidence is well-established; however, the underlying biologic mechanisms remain unknown. Examining the association between male sex and childhood cancer by single year of age and tumor type may highlight important periods of risk such as variation in growth and hormonal ...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.27620
更新日期：2019-06-01 00:00:00
abstract：BACKGROUND:Pediatric aggressive fibromatosis (AF) is a rare, benign tumor with locally infiltrative growth. Therefore, how to prevent reoccurrence while maintaining the mandible contour and continuity as much as possible is very important when the mandible is involved. PROCEDURE:We selected 10 pediatric patients with ...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.24145
更新日期：2012-10-01 00:00:00
abstract：:This review of the experience of high dose chemotherapy in patients with recurrent or refractory intracranial germ cell tumor confirms that sustained tumor control can be achieved with this modality both in germinoma and non-germinomatous germ cell tumors. Data from cooperative groups in Europe and North America sugge...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章,评审
doi：10.1002/pbc.22376
更新日期：2010-04-01 00:00:00
abstract：BACKGROUND:The purpose of our study was to assess long-term cause-specific mortality of 5-year childhood cancer survivors. PROCEDURE:The study population consisted of 1,378 patients who had been treated for childhood cancer in The Netherlands between 1966 and 1996 and survived at least 5 years; follow-up was complete ...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.20028
更新日期：2004-06-01 00:00:00
abstract：:Data on prognostic factors in pelvic PNET are minimal. We analyzed patients with pelvic PNET treated between June 2003 and November 2011 for prognostic factors. Forty-eight (13%) of 374 patients with PNET were pelvic PNET with median age 14.5 years (range: 5-33); 31 (65%) had metastases. After median follow-up of 20.4...
journal_title：Pediatric blood & cancer
pub_type： 临床试验,杂志文章
doi：10.1002/pbc.24552
更新日期：2013-09-01 00:00:00
abstract：BACKGROUND:The introduction of the United Kingdom Medical Research Council's 10th AML trial (MRC AML 10) protocol incorporating high-dose anthracycline therapy has improved outcome of children with acute myeloid leukemia (AML). In this study, we review the results of childhood AML therapy in a Singapore university hosp...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.20834
更新日期：2007-03-01 00:00:00
abstract：BACKGROUND:Treatment abandonment (TxA) is a primary cause of therapy failure in children with cancer in low-/middle-income countries. We explored the absence of social support network (SSN), among other predictive factors, and TxA in children with cancer in Cali, Colombia. PROCEDURE:In this prospective cohort study, w...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.25919
更新日期：2016-05-01 00:00:00
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:Ultrasound-guided (USG) cannulation of the brachiocephalic vein (BCV) is gaining worldwide consensus for central venous access in children. This study reports a 20-month experience with this approach in children. METHODS:All patients who underwent percutaneous USG central venous catheter (CVC) positioning i...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.26202
更新日期：2017-02-01 00:00:00
abstract：BACKGROUND:Children with sickle cell disease (SCD) are at increased risk of death from invasive bacterial infections. Emergent evaluation of fever allows early treatment of potentially fatal infections. Limited data exist regarding caregiver adherence to physician recommendations of prompt medical evaluation of fever i...
journal_title：Pediatric blood & cancer
pub_type： 临床试验,杂志文章
doi：10.1002/pbc.25634
更新日期：2015-11-01 00:00:00
abstract：BACKGROUND:Follicular variant of papillary thyroid carcinoma (FVPTC) has been shown to be an intermediate entity between papillary (PTC) and follicular/Hurtle cell (FTC) thyroid carcinoma in adults. However, the tumor characteristics and prognosis of FVPTCs has not been studied in the pediatric population and is the fo...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.25623
更新日期：2015-11-01 00:00:00
abstract：BACKGROUND:The views and clinical practice of children's cancer units were surveyed regarding management of central venous catheter (CVC) occlusion (CVC-occlusion), CVC-related thrombosis (CVC-thrombosis) and thromboembolism (CVC-thromboembolism). PROCEDURE:A questionnaire was sent to all 22 United Kingdom Children's ...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章,多中心研究
doi：10.1002/pbc.21332
更新日期：2008-04-01 00:00:00
abstract：PURPOSE:To report the final analysis of survival outcomes for children with newly diagnosed high-grade glioma (HGG) treated on the "Head Start" (HS) II and III protocols with chemotherapy and intent to avoid irradiation in children <6 years old. PATIENTS AND METHODS:Between 1997 and 2009, 32 eligible children were enr...
journal_title：Pediatric blood & cancer
pub_type： 杂志文章
doi：10.1002/pbc.26118
更新日期：2016-10-01 00:00:00
