Abstract:
BACKGROUND:The high cost, coupled with the need for continuous infusion, renders Desferrioxamine (DFO), a non-feasible option for iron-chelation in a large majority of patients with β-thalassemia major in developing countries. Monotherapy with deferiprone (DFP) or deferasirox (DFX) may not always attain optimal control, particularly in heavily iron-loaded patients. Combination of DFP and DFX is a potential alternative. PROCEDURE:A prospective, single-center, open-label, uncontrolled study was conducted to evaluate the safety and efficacy of the combination in patients with β-thalassemia major. Patients who had received either DFP or DFX for >1 year and a serum ferritin >2,000 μg/L were enrolled. Blood counts, liver/renal functions, and serum ferritin were monitored during the 1-year study period. Facilities for cardiac T2*-MRI were unavailable. RESULTS:Thirty-six patients with a mean age of 13 ± 6.9 years (range: 4-29) and a ferritin of 6,768 ± 4,145 μg/L formed the study cohort. Eight (22%) patients had transient gastrointestinal adverse effects. DFX was discontinued in one patient for persistent abdominal pain/diarrhea. Eight (22%) had joint symptoms; DFP was discontinued in two. Four (11%) patients had elevation in AST/ALT levels, managed with temporary interruption of DFX. Nine (25%) had an inconsistent elevation of creatinine to >33% of baseline; no intervention was done. One had transient proteinuria. None had neutropenia. At the end of 1 year, the serum ferritin reduced by a mean value of 3,275.3 ± 618.2 μg/L (P < 0.001). CONCLUSIONS:The oral combination was found to be safe, efficacious, and a feasible option in patients with suboptimal response to monotherapy.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Totadri S,Bansal D,Bhatia P,Attri SV,Trehan A,Marwaha RKdoi
10.1002/pbc.25533subject
Has Abstractpub_date
2015-09-01 00:00:00pages
1592-6issue
9eissn
1545-5009issn
1545-5017journal_volume
62pub_type
杂志文章abstract:BACKGROUND:Influenza is a health risk to children receiving chemotherapy for cancer. An absolute lymphocyte count (ALC) >1,000 cells/mm(3) has been associated with the ability to produce an immune response to influenza vaccine during chemotherapy. However, clinical efficacy of influenza vaccination during chemotherapy ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26110
更新日期:2016-11-01 00:00:00
abstract::Surgery plays an important role as part of the treatment plan in most children with malignant solid tumors in regards to initial biopsy, upfront resection, and delayed resection. Surgeons also play a critical role in the treatment of surgical complications that may arise during medical treatment. The pediatric surgica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26618
更新日期:2017-10-01 00:00:00
abstract::Efficacy of therapeutic strategies relative to patient- and family-centered outcomes in pediatric oncology must be assessed. We sought to identify outcomes important to children with acute myeloid leukemia and their families related to inpatient versus at-home management of neutropenia. We conducted qualitative interv...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.26927
更新日期:2018-04-01 00:00:00
abstract::We describe an African American family with Hoyeraal-Hreidarrson syndrome (HHS) in which 2 TERT mutations (causing P530L and A880T amino acid changes) and two in the DKC1 variants (G486R and A487A) were segregating. Both genes are associated with dyskeratosis congenita and HHS. It was important to determine the import...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24389
更新日期:2013-06-01 00:00:00
abstract::Childhood obesity is a significant public health problem, affecting one in five children in the United States. At the crux of this issue is a dysregulation of energy intake and energy expenditure. This review will provide an overview on energy and nutrient balance. We discuss energy balance studies in children using i...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.23375
更新日期:2012-01-01 00:00:00
abstract:BACKGROUND:Survival rates for patients with bone tumors have increased dramatically over the past few decades. Unfortunately, many patients face functional limitations resulting from disease management, but there is little evidence regarding physical functioning in adolescents with bone tumors. This lack is largely due...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20383
更新日期:2005-10-15 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:To evaluate efficacy, pharmacokinetics (PK) and pharmacodynamics of single-agent everolimus in pediatric patients with radiographically progressive low-grade glioma (LGG). METHODS:Everolimus was administered at 5 mg/m2 once daily as a tablet or liquid for a planned 48-week duration or until unacceptable tox...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28787
更新日期:2021-02-01 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::A 4-year-old girl with Noonan syndrome (NS) and constitutive PTPN11 mutation presented with stage 4 neuroblastoma and was treated by intensive chemotherapy. During the treatment, cytogenetic analysis revealed the development of a hyperdiploid clone with duplication of the germline PTPN11 mutation in a morphologically ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20527
更新日期:2007-01-01 00:00:00
abstract::Osteosarcoma is the most common bone tumor in children and young adults, with few advances in survival and treatment, especially for metastatic disease, in the last 30 years. Recently, immunotherapy has begun to show promise in various adult cancers, but the utility of this approach for osteosarcoma remains relatively...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.27227
更新日期:2018-09-01 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:Long-term adverse effects of Imatinib in children with chronic myeloid leukemia (CML) are uncertain. The aim was to study the effect of imatinib on growth in children with CML. PROCEDURE:Children ≤13 years of age at diagnosis were enrolled retrospectively, from 2004 to 2011, from a single center in India. P...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23389
更新日期:2012-09-01 00:00:00
abstract:BACKGROUND:Pulmonary hypertension (PHTN) is a potentially life-threatening complication, detected by echocardiographic evidence of elevated tricuspid regurgitant velocity (TRV). This condition has been described in adults with sickle cell disease (SCD) and other hemolytic disorders; however, there is little information...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20791
更新日期:2006-12-01 00:00:00
abstract:BACKGROUND:Dexamethasone is more efficacious than prednisone in the treatment of acute lymphoblastic leukemia (ALL), but has also been associated with greater toxicity. We compared neuropsychological outcomes for patients treated on DFCI ALL Consortium Protocol 00-01, which included a randomized comparison of the two s...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.24666
更新日期:2013-11-01 00:00:00
abstract::Pediatric cancer programs in low-income countries (LIC) can improve outcomes. However, treatment must be tailored to the patient's living conditions and the availability of supportive care. In some cases, a more intense regimen will decrease survival since the increase in death from toxicity may exceed any decrease in...
journal_title:Pediatric blood & cancer
pub_type:
doi:10.1002/pbc.20989
更新日期:2007-04-01 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::Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal stem cell disorder. Eculizumab and bone marrow transplantation are disease-modifying treatments for PNH but may not be readily available in resource-constrained settings. Of 52 pediatric patients with PNH, 20 had classical PNH and 32 had PNH/aplastic ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27712
更新日期:2020-04-01 00:00:00
abstract::The treatment of malignancy in cancer predisposition syndromes that also confer exquisite sensitivity to standard chemotherapy and radiation regimens remains a challenge. Bloom syndrome is one such disorder that is caused by a defect in DNA repair, predisposing to the development of early-onset age-related medical con...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28815
更新日期:2021-02-01 00:00:00
abstract::Primary choriocarcinoma of the liver is an extremely rare childhood malignancy frequently associated with clinical instability at initial presentation. It often mimics other benign and malignant childhood liver tumors. Prompt diagnosis and initiation of treatment are necessary to attain a successful outcome. We descri...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20623
更新日期:2007-07-01 00:00:00
abstract::Natural killer (NK) cell lymphoma of lung is very rare. We report a case of NK cell lymphoma presented in a 17-year-old male with bilateral pleural effusions and hemophagocytic lymphohistocytosis. Morphologic and immunohistochemical tests revealed an association of NK cell lymphoma with Epstein-Barr virus. A literatur...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21943
更新日期:2009-05-01 00:00:00
abstract:BACKGROUND:Corticosteroids increase risk for decreased bone mineral density, which can be worsened by vitamin D insufficiency (VDI) or deficiency (VDD). PROCEDURE:In the Vanderbilt cancer survivorship clinic, we obtained screening total 25-hydroxy vitamin D levels (VDL) in 171 cancer survivors <23 years old who were t...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24844
更新日期:2014-04-01 00:00:00
abstract:BACKGROUND:Retinoblastoma occurs in approximately 4 per million children per year in the United States, usually before 2 years of age. In developed countries, 95% of children with tumor in one eye can be cured with enucleation, while children with tumor in both eyes require individualized therapy to preserve vision. Al...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21155
更新日期:2008-02-01 00:00:00
abstract:BACKGROUND:In the pediatric population, pathologic bleeding is often challenging to identify. The pediatric bleeding questionnaire (PBQ) was developed as a screening tool for von Willebrand disease (VWD) but was designed to be self-completed by children above 12 years of age. The study objective was to determine whethe...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27709
更新日期:2019-06-01 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:BACKGROUND:Griscelli syndrome type 2 (GS2), the X-linked lymphoproliferative (XLP) and the Chédiak-Higashi (CHS) syndromes are diseases that all may develop hemophagocytic syndromes. We wanted to investigate whether the treatment protocols for hemophagocytic lymphohistiocytosis (HLH) can also be used for these syndrome...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21790
更新日期:2009-02-01 00:00:00
abstract::Bridging the survival gap for children with cancer, between those (the great majority) in low and middle income countries (LMIC) and their economically advantaged counterparts, is a challenge that has been addressed by twinning institutions in high income countries with centers in LMIC. The long-established partnershi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24802
更新日期:2014-02-01 00:00:00
abstract:BACKGROUND:Aberrant gangliosides are produced and shed by some tumors into the extracellular milieu. Their concentration is related to disease progression in children with neuroblastoma and in experimental models. The mechanism for this tumor promoting effect is not known. PURPOSE:Here, we investigated the effect of g...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20326
更新日期:2006-03-01 00:00:00
abstract:PURPOSE:This study compared the relative incidence of treatment-related toxicities and the event-free and overall survival between Hispanic and non-Hispanic children undergoing therapy for acute lymphoblastic leukemia (ALL) on Dana-Farber Cancer Institute ALL Consortium protocol 05-001. PATIENTS AND METHODS:Secondary ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.26871
更新日期:2018-03-01 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