Abstract:
BACKGROUND:Based on the presumed clinical similarity between the two most severe sickle cell disease (SCD) genotypes, hemoglobin (Hb) Sβ0 thalassemia and HbSS, randomized controlled trials (RCTs) have included both genotypes. Our group has demonstrated that healthcare providers inadequately distinguish the two diagnoses through clinical and laboratory parameters. PROCEDURE:In a retrospective cohort study utilizing data from the Silent Cerebral Infarct Transfusion trial (NCT00072761), we tested the hypothesis that children with genotypic diagnoses of HbSβ0 thalassemia, when compared to HbSS, have significantly different rates of SCD comorbidities. Exclusion criteria included those with previous overt stroke or treatment with hydroxyurea or regular blood transfusion therapy. RESULTS:Among children with HbSβ0 thalassemia (n = 22) and HbSS (n = 786), the mean hemoglobin was higher in children with HbSβ0 thalassemia (9.2 g/dl) compared to HbSS (8.1 g/dl, P < 0.001). In children with HbSβ0 thalassemia, when compared to HbSS, the incidence rate of acute chest syndrome (ACS) was 3.0 and 14.4 events per 100 patient-years (P = 0.028), and mean transcranial Doppler (TCD) velocities were 112.6 and 135.6 cm/sec, respectively (P = 0.026). The number of children with HbSβ0 thalassemia and HbSS with conditional TCD velocities were zero (0%) and 26 (4.9%), respectively (P = 1.00), and the number with silent cerebral infarcts were five (27.8%) and 209 (30.8%), respectively (P = 0.78). CONCLUSIONS:We have provided preliminary evidence that clinically relevant differences occur in ACS rates and TCD velocities between children with HbSβ0 thalassemia and HbSS. Future SCD RCTs should consider balanced allocation of these SCD genotypes, particularly when ACS and abnormal TCD velocities are primary outcome measures.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Day ME,Rodeghier M,DeBaun MRdoi
10.1002/pbc.27352subject
Has Abstractpub_date
2018-11-01 00:00:00pages
e27352issue
11eissn
1545-5009issn
1545-5017journal_volume
65pub_type
杂志文章abstract::The global burden of sickle cell disease (SCD) is now being increasingly realized. SCD poses a significant public health problem in sub-Saharan Africa, the Middle East, some regions of India, the Caribbean, and Brazil. In many of these regions, progress in the management of SCD has been slow. Long-term North-South and...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24175
更新日期:2012-08-01 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:Children with cancer may be at an increased risk of infection with hepatitis B virus (HBV) when levels of hepatitis B antibodies are reduced owing to chemotherapy-induced immunosuppression. This study evaluated the changes in HBV antibody status and HBV infections after chemotherapy in children with acute ly...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27904
更新日期:2019-12-01 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:BACKGROUND:Cisplatin (CDDP) ototoxicity is a significant side effect of the current treatment of medulloblastoma (MB). Cumulative dose of CDDP and age are recognized risk factors for hearing loss, but inter-individual susceptibility limits our ability to identify patients at risk for hearing loss. We describe the kinet...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24307
更新日期:2013-02-01 00:00:00
abstract::In the present study, DNA sequencing of the genes SRGN, ARF6, AP3B1, and SH2D1A was performed in a well defined cohort of 18 families with familial hemophagocytic lymphohistiocytosis (FHL). A heterozygous nucleotide change (C > T) in the 3'untranslated region of the SRGN gene and a monoallelic 3-base pair deletion (c....
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21428
更新日期:2008-05-01 00:00:00
abstract:BACKGROUND:The optimal dose and schedule of mycophenolate mofetil (MMF) in pediatric allogeneic stem cell transplant recipients remains to be determined. We previously reported safety and pharmacokinetics of MMF at 900 mg/m2 q6h dosing. This study was conducted to investigate the efficacy of tacrolimus plus q8h MMF dos...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.27091
更新日期:2018-08-01 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::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: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::X-linked lymphoproliferative syndrome (XLP) is caused by mutations in SH2D1A, and is associated with overwhelming infectious mononucleosis, aplastic anemia, hypogammaglobulinemia, and B-cell lymphomas. However, the frequency of SH2D1A mutations in males who present with B NHL is unknown. Five cases of XLP were diagnos...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24525
更新日期:2013-09-01 00:00:00
abstract:BACKGROUND:Epoetin alfa (EPO, PROCRIT) pharmacokinetics and pharmacodynamics were evaluated in children with malignant solid tumors receiving chemotherapy. PROCEDURE:Children initially received IV EPO 600 IU/kg (max dose 40,000 IU) or placebo once weekly for 16 weeks. Dose was increased to 900 IU/kg (max dose 60,000 I...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.20685
更新日期:2006-10-15 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::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::Ovarian tumors have a low incidence in childhood, accounting for 1% of malignancies within the ages of 0-17 years. Small cell carcinoma of the ovary is a rare histology and historically has a poor prognosis. We report a case of an 11-year-old female diagnosed with small cell carcinoma of the ovary and hypercalcemia (S...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22184
更新日期:2009-12-15 00:00:00
abstract:BACKGROUND:The early effects of childhood acute lymphoblastic leukemia (ALL) include decreased physical function, bone mineral density (BMD/g/cm2 ), and health-related quality of life (HRQL). We assessed the capacity of a physical therapy and motivation-based intervention, beginning after diagnosis and continuing throu...
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.26929
更新日期:2018-04-01 00:00:00
abstract:BACKGROUND:SCH 727965 is a novel drug in clinical development that potently and selectively inhibits CDK1, CDK2, CDK5, and CDK9. The activity of SCH 727965 was evaluated against the PPTP's in vitro and in vivo panels. PROCEDURES:SCH 727965 was tested against the PPTP in vitro panel using 96 hours exposure at concentra...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24073
更新日期:2012-12-15 00:00:00
abstract:PURPOSE:Pediatric head and neck malignancies are managed with intensive multimodality therapy. Proton beam therapy (PBT) may reduce toxicity by limiting exposure of normal tissue to radiation. In this study, we report acute toxicities and early outcomes following PBT for pediatric head and neck malignancies. MATERIALS...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26858
更新日期:2018-02-01 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:BACKGROUND:The prognostic significance of having extraskeletal (EES) versus skeletal Ewing sarcoma (ES) in the setting of modern chemotherapy protocols is unknown. The purpose of this study was to compare the clinical characteristics, biologic features, and outcomes for patients with EES and skeletal ES. METHODS:Patie...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26096
更新日期:2016-10-01 00:00:00
abstract:BACKGROUND:To determine prognostic factors for local control in the radiotherapeutic management of non-metastatic Ewing sarcoma. PROCEDURE:Forty patients with localized Ewing sarcoma (ES) were treated with primary site RT at one institution. Median RT dose was 55.8 Gy (range, 25.5-76 Gy). Chemotherapy was given to 34 ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20904
更新日期:2007-08-01 00:00:00
abstract:BACKGROUND:Opioids are a cornerstone of palliation of pain. We sought to assess variation in opioid prescription during the last week of life among a cohort of pediatric oncology patients who died while hospitalized. PROCEDURE:We used detailed hospital administrative data from the Pediatric Health Information System (...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.21824
更新日期:2009-07-01 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:Numerous recent reports have described the use of recombinant factor VIIa (rFVIIa) in non-hemophilia bleeding situations for achievement of hemostasis. However, its use in clinical situations other than hemophilia patients with inhibitors has been complicated by some reports of thrombotic events. rFVIIa has ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.22160
更新日期:2009-12-01 00:00:00
abstract:BACKGROUND:The improved survival rates for childhood acute lymphoblastic leukemia (ALL) may be jeopardized by the development of a second cancer, which has been associated with thiopurine therapy. PROCEDURE:We retrospectively analyzed three sequential Nordic Society of Paediatric Haematology and Oncology's protocols c...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26518
更新日期:2017-10-01 00:00:00
abstract:BACKGROUND:Information on the epidemiology of pediatric liver tumors in Latin America is limited. PURPOSE:To describe the incidence of liver tumors in a pediatric registry in Argentina according to geographic region, national trends over 16 years, and survival related to stage, age, sex, and care center. METHODS:Newl...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28583
更新日期:2020-11-01 00:00:00
abstract:BACKGROUND:Chronic myeloid leukemia (CML) is a rare disease in children and represents approximately 2% of all childhood leukemia. This results in difficulty creating large cohorts of patients for pediatric CML research. The Glivec International Patient Assistance Program (GIPAP) is a patient-access program sponsored b...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25120
更新日期:2014-10-01 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
abstract::Hepatoblastoma is a malignant pediatric liver tumor. The currently used diagnostic serum marker for hepatoblastoma, α-fetoprotein (AFP), is not always reliable in infants with hepatoblastoma, due to the physiologically elevated levels of AFP in this age group. In this report, we show that Delta-like 1 homolog (DLK1), ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24024
更新日期:2012-10-01 00:00:00
abstract:BACKGROUND:Knowledge about their past medical history is central for childhood cancer survivors to ensure informed decisions in their health management. Knowledge about information provision and information needs in this population is still scarce. We thus aimed to assess: (1) the information survivors reported to have...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24762
更新日期:2014-02-01 00:00:00