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pediatrics, pediatric hematology

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https://www.readbyqxmd.com/read/28089099/clinical-characteristics-and-outcome-of-invasive-fungal-infections-in-pediatric-acute-myeloid-leukemia-patients-in-a-medical-center-in-taiwan
#1
Gu-Lung Lin, Hsiu-Hao Chang, Chun-Yi Lu, Chung-Ming Chen, Meng-Yao Lu, Ping-Ing Lee, Shiann-Tarng Jou, Yung-Li Yang, Li-Min Huang, Luan-Yin Chang
BACKGROUND: Invasive fungal infection (IFI) causes significant morbidity and mortality in patients with hematological malignancies, especially those with acute myeloid leukemia (AML), recurrent acute leukemia, high-risk acute lymphoblastic leukemia, and after allogeneic hematopoietic stem cell transplantation. The study aimed to investigate the clinical characteristics and outcome of IFIs in pediatric AML patients in a medical center in Taiwan. METHODS: We performed retrospective chart reviews...
December 19, 2016: Journal of Microbiology, Immunology, and Infection, Wei Mian Yu Gan Ran za Zhi
https://www.readbyqxmd.com/read/28079913/intravenous-immunoglobulin-in-the-treatment-of-hematologic-disorders-in-pediatrics
#2
Gabriela Villanueva, Jill L O de Jong, Jennifer L McNeer
Intravenous immunoglobulin (IVIG) is pooled immunoglobulin G derived from human blood donors. It was introduced in the early 1980s to treat immunodeficiency disorders. Since then, its use has expanded to other fields such as neurology, rheumatology, and hematology. IVIG has been used to provide passive immunity in qualitative and quantitative immunoglobulin disorders, to neutralize antibodies in immune-mediated diseases, and as an immune modulatory agent. The difficulty of producing IVIG in high quantities, in addition to a growing list of "off-label" indications, has resulted in a worldwide shortage and increase in cost...
January 1, 2017: Pediatric Annals
https://www.readbyqxmd.com/read/28069802/genomic-profiling-of-a-large-set-of-diverse-pediatric-cancers-identifies-known-and-novel-mutations-across-tumor-spectra
#3
Juliann Chmielecki, Mark Bailey, Jie He, Julia Elvin, Jo-Anne Vergilio, Shakti Ramkissoon, James Suh, Garrett M Frampton, James X Sun, Samantha Morley, Daniel Spritz, Siraj Ali, Laurie Gay, Rachel L Erlich, Jeffrey S Ross, Joana Buxhaku, Hilary Davies, Vinny Faso, Alexis Germain, Blair Glanville, Vincent A Miller, Philip J Stephens, Katherine A Janeway, John M Maris, Soheil Meshinchi, Trevor J Pugh, Jack F Shern, Doron Lipson
Pediatric cancers are generally characterized by low mutational burden and few recurrently mutated genes. Recent studies suggest that genomic alterations may help guide treatment decisions and clinical trial selection. Here, we describe genomic profiles from 1,215 pediatric tumors representing sarcomas, extracranial embryonal tumors, brain tumors, hematologic malignancies, carcinomas, and gonadal tumors. Comparable published datasets identified similar frequencies of clinically relevant alterations, validating this dataset as biologically relevant...
January 9, 2017: Cancer Research
https://www.readbyqxmd.com/read/28068559/the-ageing-genome-clonal-mosaicism-and-chronic-disease
#4
REVIEW
Mitchell J Machiela, Stephen J Chanock
Clonal mosaicism arises when a postzygotic mutational event is detectable in subpopulations of cells as an alternative genotype while not present in the germline genome. Although described in a subset of pediatric disorders, new genomic technologies have detected higher than anticipated frequencies of clonal mosaicism in adult population studies, stimulating investigation as to how clonal mosaicism could contribute to chronic human diseases, such as cancer, diabetes and neurodegenerative disorders. It has also been postulated to be an important mechanism for functional cellular diversity, including the brain...
January 6, 2017: Current Opinion in Genetics & Development
https://www.readbyqxmd.com/read/28067901/targeting-samhd1-with-the-vpx-protein-to-improve-cytarabine-therapy-for-hematological-malignancies
#5
Nikolas Herold, Sean G Rudd, Linda Ljungblad, Kumar Sanjiv, Ida Hed Myrberg, Cynthia B J Paulin, Yaser Heshmati, Anna Hagenkort, Juliane Kutzner, Brent D G Page, José M Calderón-Montaño, Olga Loseva, Ann-Sofie Jemth, Lorenzo Bulli, Hanna Axelsson, Bianca Tesi, Nicholas C K Valerie, Andreas Höglund, Julia Bladh, Elisée Wiita, Mikael Sundin, Michael Uhlin, Georgios Rassidakis, Mats Heyman, Katja Pokrovskaja Tamm, Ulrika Warpman-Berglund, Julian Walfridsson, Sören Lehmann, Dan Grandér, Thomas Lundbäck, Per Kogner, Jan-Inge Henter, Thomas Helleday, Torsten Schaller
The cytostatic deoxycytidine analog cytarabine (ara-C) is the most active agent available against acute myelogenous leukemia (AML). Together with anthracyclines, ara-C forms the backbone of AML treatment for children and adults. In AML, both the cytotoxicity of ara-C in vitro and the clinical response to ara-C therapy are correlated with the ability of AML blasts to accumulate the active metabolite ara-C triphosphate (ara-CTP), which causes DNA damage through perturbation of DNA synthesis. Differences in expression levels of known transporters or metabolic enzymes relevant to ara-C only partially account for patient-specific differential ara-CTP accumulation in AML blasts and response to ara-C treatment...
January 9, 2017: Nature Medicine
https://www.readbyqxmd.com/read/28067033/2016-updated-american-society-of-clinical-oncology-oncology-nursing-society-chemotherapy-administration-safety-standards-including-standards-for-pediatric-oncology
#6
Michael Neuss, Terry Gilmore, Kristin Belderson, Amy Billett, Tara Conti-Kalchik, Brittany Harvet, Carolyn Hendricks, Kristine LeFebvre, Pamela Mangu, Kristen McNiff, MiKaela Olsen, Lisa Schulmeister, Anne Von Gehr, Martha Polovich
Purpose: To update the American Society of Clinical Oncology (ASCO)/Oncology Nursing Society (ONS) Chemotherapy Administration Safety Standards and to highlight standards for pediatric oncology. Methods: The ASCO/ONS Chemotherapy Administration Safety Standards were first published in 2009 and updated in 2011 to include inpatient settings. A subsequent 2013 revision expanded the standards to include the safe administration and management of oral chemotherapy. A joint ASCO/ONS workshop with stakeholder participation, including that of the Association of Pediatric Hematology Oncology Nurses and American Society of Pediatric Hematology/Oncology, was held on May 12, 2015, to review the 2013 standards...
6, 2017: Oncology Nursing Forum
https://www.readbyqxmd.com/read/28054944/the-role-of-histone-protein-modifications-and-mutations-in-histone-modifiers-in-pediatric-b-cell-progenitor-acute-lymphoblastic-leukemia
#7
REVIEW
Szymon Janczar, Karolina Janczar, Agata Pastorczak, Hani Harb, Adam J W Paige, Beata Zalewska-Szewczyk, Marian Danilewicz, Wojciech Mlynarski
While cancer has been long recognized as a disease of the genome, the importance of epigenetic mechanisms in neoplasia was acknowledged more recently. The most active epigenetic marks are DNA methylation and histone protein modifications and they are involved in basic biological phenomena in every cell. Their role in tumorigenesis is stressed by recent unbiased large-scale studies providing evidence that several epigenetic modifiers are recurrently mutated or frequently dysregulated in multiple cancers. The interest in epigenetic marks is especially due to the fact that they are potentially reversible and thus druggable...
January 3, 2017: Cancers
https://www.readbyqxmd.com/read/28043682/efficacy-of-four-scoring-systems-in-predicting-intravenous-immunoglobulin-resistance-in-children-with-kawasaki-disease-in-a-children-s-hospital-in-beijing-north-china
#8
Ruixia Song, Wei Yao, Xiaohui Li
OBJECTIVE: To evaluate the predictive efficacies of 4 existing scoring systems for intravenous immunoglobulin (IVIG) resistance in Kawasaki disease (KD) in hospitalized children with KD in a children's hospital affiliated with the Capital Institute of Pediatrics, Beijing, China. STUDY DESIGN: We retrospectively analyzed 1569 children with KD treated at our children's hospital between January 2010 and December 2015. Age, sex, clinical manifestations, and pretreatment hematologic indicators were recorded...
December 30, 2016: Journal of Pediatrics
https://www.readbyqxmd.com/read/28040394/long-term-hematological-visceral-and-growth-outcomes-in-children-with-gaucher-disease-type-3-treated-with-imiglucerase-in-the-international-collaborative-gaucher-group-gaucher-registry
#9
Amal El-Beshlawy, Anna Tylki-Szymanska, Ashok Vellodi, Nadia Belmatoug, Gregory A Grabowski, Edwin H Kolodny, Julie L Batista, Gerald F Cox, Pramod K Mistry
In Gaucher disease (GD), deficiency of lysosomal acid β-glucosidase results in a broad phenotypic spectrum that is classified into three types based on the absence (type 1 [GD1]) or presence and severity of primary central nervous system involvement (type 2 [GD2], the fulminant neuronopathic form, and type 3 [GD3], the milder chronic neuronopathic form). Enzyme replacement therapy (ERT) with imiglucerase ameliorates and prevents hematological and visceral manifestations in GD1, but data in GD3 are limited to small, single-center series...
December 6, 2016: Molecular Genetics and Metabolism
https://www.readbyqxmd.com/read/28038498/risk-for-health-care-associated-bloodstream-infections-in-pediatric-oncology-patients-with-various-malignancies
#10
Cara B Thurman, Maura Abbott, Jinfang Liu, Elaine Larson
This was a retrospective cohort study to identify the rates, predictors, and outcomes of health care-associated bloodstream infections (HA-BSI) among children with solid tumors, lymphoma, lymphoid leukemia, and myeloid leukemia. The study population included 4500 children ≤18 years old at a pediatric hospital in New York City from 2006 to 2014. A total of 147 HA-BSI cases were identified; using multivariable logistic regression modeling, children with a hematologic diagnosis (lymphoma, lymphoid leukemia, myeloid leukemia) were at greater risk for HA-BSI than those with a solid tumor diagnosis (all P values <...
December 1, 2016: Journal of Pediatric Oncology Nursing: Official Journal of the Association of Pediatric Oncology Nurses
https://www.readbyqxmd.com/read/28030523/successful-introduction-of-fine-needle-aspiration-biopsy-for-diagnosis-of-pediatric-lymphadenopathy
#11
Candice Sher-Locketz, Pawel T Schubert, Samuel W Moore, Colleen A Wright
BACKGROUND: Accurate and rapid diagnosis of extrapulmonary nodal tuberculosis in children is of paramount importance in order to initiate immediate treatment and prevent complications. Fine needle aspiration biopsy (FNAB) provides a means of triage, and also identifies patients with suspected hematological or metastatic neoplasms.This study aims to demonstrate how a laboratory-run FNAB service, which largely replaced the previous surgical practice of lymph node excision biopsy can provide a clinically acceptable and effective diagnostic service...
December 27, 2016: Pediatric Infectious Disease Journal
https://www.readbyqxmd.com/read/28028966/glycogen-synthase-kinase-3%C3%AE-gsk-3%C3%AE-and-nuclear-factor-kappa-b-nfkb-in-childhood-acute-lymphoblastic-leukemia
#12
Cristian Fabian Layton Tovar, Hugo Mendieta Zerón, Maria Del Socorro Camarillo Romero, Yanko V Fabila Sánchez, Isidoro Tejocote Romero
BACKGROUND: Acute lymphocytic leukemia (ALL) is the most common hematologic malignancy in early childhood. In children with acute lymphoblastic leukemia (ALL), the activity of glycogen synthase kinase (GSK-3β) has been associated with changes in the transcriptional activity and expression of nuclear factor kappa beta (NFKB) in the mononuclear cells of bone marrow. OBJECTIVES: The aim of the study was to determine the possible role of glycogen synthase kinase 3beta (GSK-3β) and nuclear factor kappa beta (NFKB) as prognostic variables in pediatric patients with ALL...
November 2016: Advances in Clinical and Experimental Medicine: Official Organ Wroclaw Medical University
https://www.readbyqxmd.com/read/28027285/acute-rhinosinusitis-in-immunosuppressed-children-diagnostic-and-therapeutic-management
#13
Amit Ritter, Salvador Fischer, Eitan Yaniv, Tuvia Hadar, Ethan Soudry, Daniel Shai, Dror Gilony, Gideon Bachar
BACKGROUND: Immunosuppressive therapy places pediatric patients at risk of developing life-threatening sinonasal infections. Diagnosis and treatment is challenging owing to nonspecific signs and symptoms. The aim of the study was to present our department's experience with the surgical management of acute rhinosinusitis in immunosuppressed children. METHODS: The records of all children with a hematologic or oncologic disease who underwent endoscopic sinus surgery (ESS) for acute rhinosinusitis from January 2005 to May 2014 were reviewed...
December 23, 2016: Pediatric Infectious Disease Journal
https://www.readbyqxmd.com/read/28025240/safety-of-quadrivalent-meningococcal-conjugate-vaccine-in-11-to-21-year-olds
#14
Hung-Fu Tseng, Lina S Sy, Bradley K Ackerson, Rulin C Hechter, Sara Y Tartof, Mendel Haag, Jeffrey M Slezak, Yi Luo, Christine A Fischetti, Harp S Takhar, Yan Miao, Marianne Cunnington, Zendi Solano, Steven J Jacobsen
BACKGROUND: Meningococcal conjugate vaccination is recommended in the United States. This study evaluates the safety of quadrivalent meningococcal conjugate vaccine in a cohort aged 11 to 21 years. METHODS: This cohort study with self-controlled case-series analysis was conducted at Kaiser Permanente Southern California. Individuals receiving MenACWY-CRM, a quadrivalent meningococcal conjugate vaccine, during September 30, 2011 to June 30, 2013, were included. Twenty-six prespecified events of interest (EOIs), including neurologic, rheumatologic, hematologic, endocrine, renal, pediatric, and pediatric infectious disease EOIs, were identified through electronic health records 1 year after vaccination...
January 2017: Pediatrics
https://www.readbyqxmd.com/read/28024491/-children-s-nk-t-cell-lymphoma-associated-hemophagocytic-syndrome-clinical-analysis-of-6-cases
#15
Yong-Zhi Zheng, Hao Zheng, Jian Li, Shao-Hua LE, Xue-Ling Hua, Zai-Sheng Chen, Ling Zheng, Mei Li, Yi-Qiao Chen, Qin-Li Gao, Jing-Hui Yang, Jian-Da Hu
OBJECTIVE: To study the clinical features, treatment and prognosis of patients with NK/T cell lymphoma-associated hemophagocytic syndrome(NK/T-LAHPS). METHODS: Retrospective analysis was used to explore the clinical data of 6 children with NK/T-LAHPS who were admitted in Department of Pediatric Hematology of Fujian Medical University Union Hospital from July 2012 to June 2016. The 6 patients included 4 boys and 2 girls, with a median age of 4 years(range 1.75 to 11)...
December 2016: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/28024477/-a-retrospective-study-of-ph-negative-adolescent-and-young-adults-with-acute-b-lymphoblastic-leukemia-in-two-centers
#16
Hong-Hao Gao, Yu Jing, Ming-Juan Liu, Hong-Hua Li, Wen-Rong Huang, Chun-Ji Gao, Dai-Hong Liu, Yong-Hui Li, Sai Huang, Yan Li, Jing Liu, Yong-Qing Zhang, Li Yu
OBJECTIVE: To analyze the therapeutic efficacy of different consolidation therapies after induction remission on Ph negative adolescent and young adults with acute B lymphoblastic leukemia, and to explore the effect of different risk factors on prognosis. METHODS: The treatment and efficacy of 80 Ph negative B-ALL in patients of 16-39 years old in the Hematology Department of 301(65 cases) and 309(15 cases) hospital from 1999 to 2016 are retrospectively analyzed...
December 2016: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/28007665/cd25-blockade-delays-regulatory-t-cell-reconstitution-and-does-not-prevent-graft-versus-host-disease-after-allogeneic-hematopoietic-cell-transplantation
#17
Frederick L Locke, Joseph Pidala, Barry Storer, Paul J Martin, Michael A Pulsipher, Thomas R Chauncey, Niels Jacobsen, Nicolaus Kröger, Irwin Walker, Susan Light, Bronwen E Shaw, Francisca Beato, Ginna G Laport, Auayporn Nademanee, Armand Keating, Gerard Socie, Claudio Anasetti
Daclizumab, a humanized monoclonal antibody, binds CD25 and blocks formation of the IL-2 receptor on T cells. A study of daclizumab as acute graft-versus-host disease (GVHD) prophylaxis after unrelated bone marrow transplantation was conducted before the importance of CD25(+)FOXP3(+) regulatory T cells (Tregs) was recognized. Tregs can abrogate the onset of GVHD. The relation between Tregs and a graft-versus-malignancy effect is not fully understood. An international, multicenter, double-blind clinical trial randomized 210 adult or pediatric patients to receive 5 weekly doses of daclizumab at 0...
December 19, 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28007021/implementation-of-next-generation-sequencing-into-pediatric-hematology-oncology-practice-moving-beyond-actionable-alterations
#18
Jennifer A Oberg, Julia L Glade Bender, Maria Luisa Sulis, Danielle Pendrick, Anthony N Sireci, Susan J Hsiao, Andrew T Turk, Filemon S Dela Cruz, Hanina Hibshoosh, Helen Remotti, Rebecca J Zylber, Jiuhong Pang, Daniel Diolaiti, Carrie Koval, Stuart J Andrews, James H Garvin, Darrell J Yamashiro, Wendy K Chung, Stephen G Emerson, Peter L Nagy, Mahesh M Mansukhani, Andrew L Kung
BACKGROUND: Molecular characterization has the potential to advance the management of pediatric cancer and high-risk hematologic disease. The clinical integration of genome sequencing into standard clinical practice has been limited and the potential utility of genome sequencing to identify clinically impactful information beyond targetable alterations has been underestimated. METHODS: The Precision in Pediatric Sequencing (PIPseq) Program at Columbia University Medical Center instituted prospective clinical next generation sequencing (NGS) for pediatric cancer and hematologic disorders at risk for treatment failure...
December 23, 2016: Genome Medicine
https://www.readbyqxmd.com/read/28004468/first-2-cases-with-thiamine-responsive-megaloblastic-anemia-in-the-czech-republic-a-rare-form-of-monogenic-diabetes-mellitus-a-novel-mutation-in-the-thiamine-transporter-slc19a2-gene-intron-1-mutation-c-204-2t-g
#19
Renata Pomahačová, Jana Zamboryová, Josef Sýkora, Petra Paterová, Karel Fiklík, Tomáš Votava, Zdeňka Černá, Petr Jehlička, Václav Lád, Ivan Šubrt, Jiří Dort, Eva Dortová
Thiamine-responsive megaloblastic anemia (TRMA) is a rare autosomal recessive disorder caused by mutations in the SLC19A2 gene. To date at least 43 mutations have been reported for the gene encoding a plasma membrane thiamine transporter protein (THTR-1). TRMA has been reported in less than 80 cases worldwide. Here, we illustrate 2 female patients with TRMA first diagnosed in the Czech Republic and in central Europe being confirmed by sequencing of the THTR-1 gene SLC19A2. Both subjects are compound heterozygotes with 3 different mutations in the SLC19A2 gene...
December 22, 2016: Pediatric Diabetes
https://www.readbyqxmd.com/read/27998318/management-of-severe-dengue-hemorrhagic-fever-and-bleeding-complications-in-a-primigravida%C3%A2-patient-a-case-report
#20
Hori Hariyanto, Corry Quando Yahya, Primartanto Wibowo, Oloan E Tampubolon
BACKGROUND: The incidence of dengue hemorrhagic fever is increasing among the adult population living in endemic areas. The disease carries a 0.73% fatality rate for the general population, but what happens when the disease strikes a special subpopulation group, the obstetrics? Perhaps the important question specific to this special subpopulation revolves around the right time and mode of delivery under severe coagulopathy and plasma leakage in conditions of imminent delivery. CASE PRESENTATION: A 24-year-old primigravid Sundanese woman presented to our intensive care unit due to acute pulmonary edema secondary to massive plasma leakage caused by severe dengue...
December 20, 2016: Journal of Medical Case Reports
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