Abstract:
:Evidence of the laboratory benefits of hydroxyurea and its clinical efficacy in reducing acute vaso-occlusive events in adults and children with sickle cell anemia has accumulated for more than 15 years. A definitive clinical trial showing that hydroxyurea can also prevent organ damage might support widespread use of the drug at an early age. BABY HUG is a randomized, double-blind placebo-controlled trial to test whether treating young children ages 9-17 months at entry with a liquid preparation of hydroxyurea (20 mg/kg/day for 2 years) can decrease organ damage in the kidneys and spleen by at least 50%. Creation of BABY HUG entailed unique challenges and opportunities. Although protection of brain function might be considered a more compelling endpoint, preservation of spleen and renal function has clinical relevance, and significant treatment effects might be discernable within the mandated sample size of 200. Concerns about unanticipated severe toxicity and burdensome testing and monitoring requirements were addressed in part by an internal Feasibility and Safety Pilot Study, the successful completion of which was required prior to enrolling a larger number of children on the protocol. Concerns over recruitment of potentially vulnerable subjects were allayed by inclusion of a research subject advocate, or ombudsman. Finally, maintenance of blinding of research personnel was aided by inclusion of an unblinded primary endpoint person, charged with transmitting endpoint data and monitoring blood work locally for toxicity (ClinicalTrials.gov number, NCT00006400).
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Thompson BW,Miller ST,Rogers ZR,Rees RC,Ware RE,Waclawiw MA,Iyer RV,Casella JF,Luchtman-Jones L,Rana S,Thornburg CD,Kalpatthi RV,Barredo JC,Brown RC,Sarnaik S,Howard TH,Luck L,Wang WCdoi
10.1002/pbc.22269subject
Has Abstractpub_date
2010-02-01 00:00:00pages
250-5issue
2eissn
1545-5009issn
1545-5017journal_volume
54pub_type
杂志文章,多中心研究abstract:BACKGROUND:The prevalence of nausea and vomiting after receipt of intrathecal methotrexate (IT-MTX) in pediatric oncology patients is unknown. METHODS:Patients (4-18 years) about to receive IT-MTX were eligible to participate in this prospective, observational study. Patients received antiemetics as prescribed by thei...
journal_title:Pediatric blood & cancer
pub_type: 评论,杂志文章
doi:10.1002/pbc.26603
更新日期:2017-10-01 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::Although investigators have been engaged in the study of human energy metabolism for over 100 years, there remain unanswered questions regarding the role that energy expenditure plays in human health and disease. A critical examination of available methods for measurement of the various components of daily energy expe...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23369
更新日期:2012-01-01 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:BACKGROUND:Hepatoblastoma is a rare malignancy of childhood. The scarcity of adequate cell models has limited our understanding of this tumor. Here we describe and characterize a new human liver tumor cell line, Hep293TT, derived from an aggressive childhood hepatoblastoma. PROCEDURES:Hep293TT cells were established u...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22187
更新日期:2009-12-01 00:00:00
abstract:BACKGROUND:Treatment with radiotherapy (RT) is associated with an increased risk of second malignant neoplasms (SMNs) in childhood cancer survivors; it is unclear how treatment with intensity-modulated radiation therapy (IMRT) impacts this risk. We provide the first report of SMN risk in a cohort of childhood cancer su...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25285
更新日期:2015-02-01 00:00:00
abstract:BACKGROUND:Most relapses from Wilms' tumor occur within 2 years from diagnosis. This study aims to describe the incidence and outcome of patients who experienced a late recurrence (LR) more than 5 years after diagnosis across several clinical trials, and to develop evidence-based recommendations for follow-up surveilla...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.24604
更新日期:2013-10-01 00:00:00
abstract:PURPOSE:To assess the safety of delayed high dose intravenous (i.v.) sodium thiosulfate (STS) in a case series of 12 children with malignant brain tumors who were treated with intraarterial (i.a.) carboplatin in conjunction with blood-brain-barrier disruption (BBBD). METHODS:Twelve children ages 17 months-12 years und...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20529
更新日期:2006-08-01 00:00:00
abstract:BACKGROUND:The identification of tissue-specific differentially methylated regions (tDMRs) is key to our understanding of mammalian development. Research has indicated that tDMRs are aberrantly methylated in cancer and may affect the oncogenic process. PROCEDURE:We used the MassARRAY EpiTYPER system to determine the q...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24282
更新日期:2013-03-01 00:00:00
abstract:BACKGROUND:The treatment of central nervous system (CNS) germ cell tumors (GCT) remains controversial. The purpose of this study was to demonstrate efficacy of a chemotherapy only strategy, with less morbidity, when compared to regimens with irradiation. METHODS:Between January 2001 and December 2004 newly diagnosed p...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.22381
更新日期:2010-03-01 00:00:00
abstract:BACKGROUND:Irinotecan is highly active against rhabdomyosarcoma (RMS), yet its tolerability and efficacy in combination with radiation is unknown. We examined local control and toxicities in RMS patients treated with radiotherapy (RT) in combination with radiosensitizing agents irinotecan + carboplatin (I + C). PROCED...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24205
更新日期:2013-02-01 00:00:00
abstract:BACKGROUND:Degenerative-like neuro-Langerhans cell histiocytosis (DN-LCH) is a rare complication of LCH marked by progressive cerebellar ataxia. No treatment has so far been shown to slow this progression. PROCEDURE:All-trans retinoic acid (ATRA) was administered orally at a dose of 45 mg/m(2) daily for 6 weeks and th...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.20040
更新日期:2004-07-01 00:00:00
abstract::Treatment recommendations for endemic Burkitt lymphoma (BL) in settings with only minimum requirements for curative treatment (PODC setting 1) are described. The reported cure rate for endemic BL is usually <50%. Facilities within setting 1 differ. Three treatment schedules are proposed based on: (1) when accurate sta...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,实务指引
doi:10.1002/pbc.24407
更新日期:2013-03-01 00:00:00
abstract:BACKGROUND:We report a 13-year-old male with Diamond Blackfan anemia and short stature. He had a normal biochemical response to growth hormone (GH) stimulation, but his bone age was delayed, his insulin-like growth factor 1 (IGF-1) was low, and he had a poor growth velocity. He was started on daily GH injections. METH...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20075
更新日期:2004-10-01 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::The molecular detection of minimal residual disease (MRD) is standard of care in acute lymphoblastic leukemia to personalize the stratification of patients to appropriate intensity chemotherapy regimens. High-throughput sequencing (HTS) techniques are driving changes to MRD methodologies. Our study demonstrates HTS ca...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27787
更新日期:2019-08-01 00:00:00
abstract:BACKGROUND:Approximately one-third of patients with osteosarcoma who have a complete response to their initial treatment can be expected to relapse. It is important to define what host, tumor, or treatment characteristics determine outcome after relapse. We present findings in 59 patients treated at our institution fro...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20580
更新日期:2006-09-01 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::We report two survivors of Wilms tumor (WT) who developed inflammatory myofibroblastic tumor (IMT). The first patient had IMT in the mainstem bronchus 1 year after WT therapy that included vincristine, dactinomycin, doxorubicin (VDA) and whole lung radiation therapy (XRT). The second patient had IMT in the cecum 7 yea...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25345
更新日期:2015-06-01 00:00:00
abstract:BACKGROUND:Acute and chronic clinical manifestations of sickle-cell disease (SCD) lead to significant healthcare utilization, especially of the emergency department (ED). Limited population-level data are available in SCD with the ability to connect patients to visits, leaving us with minimal description of utilization...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.22792
更新日期:2011-03-01 00:00:00
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:BACKGROUND:Asparaginase (Asp) and corticosteroid (CS) treatment in patients with acute lymphoblastic leukaemia (ALL) is associated with an increased risk of thrombotic events. OBJECTIVE:Characterization of global haemostatic phenotypes of patients with ALL during Asp therapy. PROCEDURE:Thrombin generation (TG) was mo...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26228
更新日期:2017-02-01 00:00:00
abstract:BACKGROUND:The objectives of this study were to determine the maximum tolerated dose (MTD), dose-limiting toxicities (DLTs), pharmacokinetics, and anti-tumor effect of irinotecan in pediatric patients with recurrent or refractory malignancies. PROCEDURE:Twenty-three patients between 1 and 21 years of age, with a solid...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20355
更新日期:2006-01-01 00:00:00
abstract:BACKGROUND:To examine the practical applicability, internal consistency, and validity of the Bt-DUX, a disease-specific Health Related Quality of Life (HRQoL) instrument. The Bt-Dux was developed to examine patients' individual values of their life after a malignant bone tumor of the lower extremity at four domains (co...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.22078
更新日期:2009-09-01 00:00:00
abstract:BACKGROUND:Fanconi anemia (FA) predisposes to hematologic disorders and myeloid neoplasia in childhood and to solid cancers, mainly oral carcinomas, in early adulthood. Few cases of solid cancers have been reported in childhood. PROCEDURES:We conducted a national retrospective study of solid tumors occurring in patien...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.25303
更新日期:2015-03-01 00:00:00
abstract:BACKGROUND:Patients with relapsed or refractory Ewing sarcoma have a poor outcome with conventional therapies. Cytarabine decreases EWS/FLI1 protein levels in Ewing sarcoma cells and has demonstrated preclinical activity against Ewing sarcoma in vitro and in vivo. The purpose of this phase II clinical trial was to esti...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21822
更新日期:2009-03-01 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::Tailoring pre-emptive CMV therapy to hematopoietic cell transplant recipients' risk for reactivation could make this approach more cost-effective. To determine the feasibility of creating a risk classification system for this purpose, we analyzed 169 pediatric HCTs involving seropositive recipients or donors. Using ri...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25237
更新日期:2015-02-01 00:00:00
abstract:INTRODUCTION:Regret about loss is one of the most intense types of regret experienced in life. Little is known about the bereavement regret of parents whose child has died of cancer. Although knowledge about parents' experiences after their child's death is vital for supporting these families, parents' regret is mostly...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28265
更新日期:2020-06-01 00:00:00
abstract::Rhabdomyosarcoma arising within a congenital cystic adenomatoid malformation (CCAM) is an unusual entity. The patient underwent a lobectomy of his right lower lobe of lung due to a CCAM at the age of two. One year later, he developed a solid embryonal rhabdomyosarcoma at the same location. He received 1-year period ch...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20481
更新日期:2005-11-01 00:00:00