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https://www.readbyqxmd.com/read/29790825/epidemiological-and-clinical-features-of-visceral-leishmaniasis-in-children-in-alicante-province-spain
#1
Jose M Ramos, Agustin Clavijo, Luis Moral, Cesar Gavilan, Tatiana Salvador, Javier González de Dios
Background Visceral leishmaniasis (VL) is endemic to the Mediterranean basin. In children, VL often presents with non-specific symptoms and can be life-threatening without proper treatment. Aim To describe the epidemiological and clinical features of pediatric VL in children in Alicante, Spain. Methods The study included all paediatric (<15 years) cases admitted to three hospitals in the province of Alicante from May 1992 to May 2015 with diagnosis of VL (detection was either by anti-Leishmania antibodies in serology or Leishmania in blood and/or bone marrow aspirates)...
May 23, 2018: Paediatrics and International Child Health
https://www.readbyqxmd.com/read/29788841/heparin-induced-thrombocytopenia-complicating-extracorporeal-membrane-oxygenation-support-in-pediatric-patients-review-of-the-literature-and-alternative-anticoagulants
#2
Uri Pollak
Heparin-induced thrombocytopenia (HIT) is a prothrombotic, immune-mediated complication of unfractionated heparin (UFH) and low molecular weight heparin therapy. HIT is characterized by moderate thrombocytopenia 5-10 days after initial heparin exposure, detection of platelet-activating anti-platelet factor 4/heparin antibodies and an increased risk of venous and arterial thrombosis. Extracorporeal membrane oxygenation (ECMO) is a form of mechanical circulatory support used in critically ill patients with respiratory or cardiac failure...
May 2018: Perfusion
https://www.readbyqxmd.com/read/29787942/thrombotic-and-hemorrhagic-complications-in-children-and-young-adult-recipients-of-hematopoietic-stem-cell-transplant-hsct
#3
Dominder Kaur, Aneel A Ashrani, Rajiv Pruthi, Shakila P Khan, Kent Bailey, Vilmarie Rodriguez
BACKGROUND: Overall incidence of hemostatic complications in pediatric recipients of Hematopoietic Stem Cell Transplant (HSCT) is scarcely studied. This retrospective review explored the incidence and underlying risk factors of bleeding and thrombotic complications in children. PROCEDURE: Clinical characteristics, hemorrhagic events (HE), thrombotic events (TE) and follow up data were abstracted from medical records on patients aged <21 years undergoing HSCT during January 2000-June 2015...
April 26, 2018: Thrombosis Research
https://www.readbyqxmd.com/read/29783818/-continuous-renal-replacement-therapy-combined-with-extracorporeal-membrane-oxygenation-for-pediatric-cardiopulmonary-failure
#4
Y P Zhou, J Y Shi, F Wang, Y Cui, T T Xu, C X Wang, Y C Zhang
Objective: To explore the effectiveness and safety of continuous renal replacement therapy (CRRT) combined with extracorporeal membrane oxygenation (ECMO) on rescuing pediatric patients with cardiopulmonary failure. Methods: The medical records of patients treated with ECMO admitted to pediatric intensive care unit (PICU) in Shanghai Children's Hospital from December 2015 to November 2017 were retrospectively extracted. There were 14 patients treated with ECMO combined with CRRT (ECMO+ CRRT group) due to acute kidney injury (AKI) or fluid overload, while 11 cases treated with ECMO only...
May 2, 2018: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
https://www.readbyqxmd.com/read/29774106/hematopoietic-restricted-ptpn11e76k-reveals-indolent-mpn-progression-in-mice
#5
Stefan P Tarnawsky, Wen-Mei Yu, Cheng-Kui Qu, Rebecca J Chan, Mervin C Yoder
Juvenile Myelomonocytic Leukemia (JMML) is a pediatric myeloproliferative neoplasm (MPN) that has a poor prognosis. Somatic mutations in Ptpn11 are the most frequent cause of JMML and they commonly occur in utero . Animal models of mutant Ptpn11 have probed the signaling pathways that contribute to JMML. However, existing models may inappropriately exacerbate MPN features by relying on non-hematopoietic-restricted Cre-loxP strains or transplantations into irradiated recipients. In this study we generate hematopoietic-restricted models of Ptpn11E76K-mediated disease using Csf1r-MCM and Flt3Cre...
April 24, 2018: Oncotarget
https://www.readbyqxmd.com/read/29746437/hematologic-findings-of-inherited-metabolic-disease-they-are-more-than-expected
#6
Ertan Sal, Idil Yenicesu, Ilyas Okur, Zuhre Kaya, Fatih S Ezgu, Ulker Kocak, Leyla Tumer, Turkiz Gursel, Alev Hasanoglu
Inherited metabolic diseases are pathologic conditions that generally develop as a result of impairment of the production or breakdown of protein, carbohydrate, and fatty acids. Early determination of hematological findings has a positive effect on the prognosis of metabolic diseases. Three hundred eighteen patients who were being followed-up within the previous 6 months at Department of Pediatric Nutrition and Metabolism, Gazi University, Turkey, were included in the study. The hematological findings were classified under 7 main groups: anemia of chronic disease, iron deficiency anemia, vitamin B12 deficiency anemia, hemophagocytosis, leukocytosis, and thrombocytosis...
May 9, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29713209/adoption-of-the-american-academy-of-pediatrics-neonatal-hyperbilirubinemia-guidelines-and-its-effect-on-blood-exchange-transfusion-rate-in-a-tertiary-care-center-in-amman-jordan
#7
Manar Al-Lawama, Eman Al-Rimawi, Rawan Al-Shibi, Eman Badran
Introduction: Severe neonatal hyperbilirubinemia can cause mortality and serious morbidities. When phototherapy fails, neonates with severe hyperbilirubinemia should undergo double volume blood exchange transfusion (BET). As this procedure carries a significant risk of mortality and morbidity, adopting guidelines for the treatment of neonatal hyperbilirubinemia is critical to avoid hyperbilirubinemia toxicity and also the complication of an unindicated procedure. Methods: This study investigated the causes, complications, and trend of BET rate in our unit over a 13-year period...
2018: Journal of Blood Medicine
https://www.readbyqxmd.com/read/29708883/graves-disease-following-allogenic-hematopoietic-stem-cell-transplantation-for-severe-aplastic-anemia-case-report-and-literature-review
#8
Ahu Paketçi, Korcan Demir, Özlem Tüfekçi, Sezer Acar, Ayhan Abacı, Şebnem Yılmaz, Ece Böber
BACKGROUND: Similar autoimmune processes (defective T-cell function) take place during the pathogenesis of aplastic anemia (AA) and Graves' disease (GD). Antithyroid drugs used for the management of GD may induce AA and GD may occur following treatment of severe aplastic anemia (SAA). CASE PRESENTATION: Clinical and laboratory investigations were performed for an 11-year-and-2-month-old girl who was referred for bilateral exophthalmus and abnormal thyroid function tests...
April 25, 2018: Journal of Pediatric Endocrinology & Metabolism: JPEM
https://www.readbyqxmd.com/read/29683873/eltrombopag-induced-acute-liver-failure-in-a-pediatric-patient-a-pharmacokinetic-and-pharmacogenetic-analysis
#9
M Marano, J Serafinelli, S Cairoli, D Martinelli, M Pisani, G Palumbo, M G Cefalo, C Cecchetti, M Di Nardo, F S Falvella, B M Goffredo
Eltrombopag is an oral thrombopoietin-receptor-agonist (TPO-RA) approved for the treatment of patients with chronic idiopathic thrombocytopenic purpura (ITP), who are more than one year old, and show poor response to first-line therapy. ITP is a hematological disorder characterized by isolated thrombocytopenia in the absence of secondary causes or disorders. Eltrombopag is generally well tolerated in the pediatric population, therefore therapeutic drug monitoring (TDM) is not usually performed in clinical practice...
April 20, 2018: Therapeutic Drug Monitoring
https://www.readbyqxmd.com/read/29668136/-a-fatal-case-series-of-rocky-mountain-spotted-fever-in-sonora-m%C3%A3-xico
#10
Jesús Delgado-De la Mora, Jesús David Licona-Enríquez, Marcia Leyva-Gastélum, David Delgado-De la Mora, Adela Rascón-Alcantar, Gerardo Álvarez-Hernández
INTRODUCTION: Rocky Mountain spotted fever is a highly lethal infectious disease, particularly if specific treatment with doxycycline is given belatedly. OBJECTIVE: To describe the clinical profile of fatal Rocky Mountain spotted fever cases in hospitalized patients in the state of Sonora, México. MATERIALS AND METHODS: We conducted a cross-sectional study on a series of 47 deaths caused by Rickettsia rickettsii from 2013 to 2016. The diagnosis of Rocky Mountain spotted fever was confirmed in a single blood sample by polymerase chain reaction (PCR) or by a four-fold increase in immunoglobulin G measured in paired samples analyzed by indirect immunofluorescence...
March 15, 2018: Biomédica: Revista del Instituto Nacional de Salud
https://www.readbyqxmd.com/read/29659673/evaluation-of-the-utility-of-bone-marrow-morphology-and-ancillary-studies-in-pediatric-patients-under-surveillance-for-myelodysplastic-syndrome
#11
Karen M Chisholm, Min Xu, Billy Davis, Amy Ogi, M Cristina Pacheco, Amy E Geddis, Karen D Tsuchiya, Joe C Rutledge
Objectives: To evaluate the utility of flow cytometry, karyotype, and a fluorescence in situ hybridization (FISH) panel in screening children for myelodysplastic syndrome (MDS). Methods: Bone marrow morphology, flow cytometry, karyotype, and FISH reports from 595 bone marrow specimens (246 patients) were analyzed. Results: By morphology, 8.7% of cases demonstrated at least unilineage dysplasia and/or increased blasts. Flow cytometry identified definitive abnormalities in 2...
April 25, 2018: American Journal of Clinical Pathology
https://www.readbyqxmd.com/read/29659042/physician-decision-making-in-selection-of-second-line-treatments-in-immune-thrombocytopenia-in-children
#12
Rachael F Grace, Jenny M Despotovic, Carolyn M Bennett, James B Bussel, Michelle Neier, Cindy Neunert, Shelley E Crary, Yves D Pastore, Robert J Klaassen, Jennifer A Rothman, Kerry Hege, Vicky R Breakey, Melissa J Rose, Kristin A Shimano, George R Buchanan, Amy Geddis, Kristina M Haley, Adonis Lorenzana, Alexis Thompson, Michael Jeng, Ellis J Neufeld, Travis Brown, Peter W Forbes, Michele P Lambert
Immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder which presents with isolated thrombocytopenia and risk of hemorrhage. While most children with ITP promptly recover with or without drug therapy, ITP is persistent or chronic in others. When needed, how to select second-line therapies is not clear. ICON1, conducted within the Pediatric ITP Consortium of North America (ICON), is a prospective, observational, longitudinal cohort study of 120 children from 21 centers starting second-line treatments for ITP which examined treatment decisions...
April 16, 2018: American Journal of Hematology
https://www.readbyqxmd.com/read/29625627/a-pilot-screening-of-high-risk-gaucher-disease-children-using-dried-blood-spot-methods-in-shandong-province-of-china
#13
Ke Lei, Yanxia Zhao, Lirong Sun, Hui Liang, Ronghua Luo, Xiaojing Sun, Yanling Tao, Lijun Chen, Lingling Zhang, Aimin Li, Fu Li, Hongfang Ding
BACKGROUND: The study aim was to verify the feasibility of a diagnostic algorithm with the evaluation of beta glucocerebrosidase (GBA) activity on dried blood spots (DBS) in screening high-risk Gaucher disease (GD) children in China, and to investigate the GD prevalence in this selected population. METHODS: Children were recruited from 20 departments of pediatrics or children's hospitals in Shandong Province, China, due to splenomegaly and/or thrombocytopenia associated with one or more of the following creteria: anemia, history of bone pain, monoclonal gammopathy of unknown significance (MGUS), polyclonal gammopathy and splenectomy...
April 6, 2018: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/29589486/advances-in-chemical-pharmacotherapy-for-the-treatment-of-pediatric-immune-thrombocytopenia
#14
Thomas Kühne
Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder of heterogeneous pathophysiological mechanisms. Treatment endpoints include elevation of platelets and reduction of bleeding risk, elevation of quality of life, reduction of concomitant therapies and prevention from bleeding. Persistent and chronic ITP is more common in adults but occurs in children. Standard therapies include corticosteroids and immunoglobulins, both associated with side effects. There are new treatments, such as thrombopoietin-receptor agonists and promising investigational drugs...
May 2018: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/29558000/hemolytic-uremic-syndrome-with-dual-caution-in-an-infant-cobalamin-c-defect-and-complement-dysregulation-successfully-treated-with-eculizumab
#15
Ulkem Kocoglu Barlas, Hasan Serdar Kıhtır, Nilufer Goknar, Melike Ersoy, Nihal Akcay, Esra Sevketoglu
BACKGROUND: Hemolytic uremic syndrome (HUS) is a clinical syndrome characterized by hemolytic anemia, thrombocytopenia, and acute kidney injury. Atypical hemolytic uremic syndrome (aHUS) is a devastating disease with significant mortality and high risk of progression to end-stage kidney disease. It is mostly caused by dysregulation of the alternative complement pathway. Cobalamin C (Cbl C) defect is a genetic disorder of cobalamin metabolism and is a rare cause of HUS. CASE-DIAGNOSIS/TREATMENT: We present a 6-month-old male infant who was admitted to the pediatric intensive care unit (PICU) due to restlessness, severe hypertension, anemia, respiratory distress, and acute kidney injury...
June 2018: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
https://www.readbyqxmd.com/read/29537138/-pre-emptive-strike-the-case-for-early-treatment-of-hepatic-sinusoidal-obstruction-syndrome-with-defibrotide
#16
Revathi Rajagopal, Marianne B Phillips, Nicholas G Gottardo
The initial signs of hepatic sinusoidal obstruction syndrome (HSOS) can be challenging to recognize in children, especially outside the hematopoietic stem cell transplantation setting. To assist clinicians to promptly identify HSOS, the European Society for Blood and Marrow Transplantation has proposed pediatric HSOS diagnostic criteria which emphasize unexplained consumptive and transfusion-refractory thrombocytopenia. To highlight the importance of these "bellwether" early signs of HSOS and the efficacy of pre-emptive treatment with defibrotide, we describe the case of a child with a right 11th rib primitive neuroectodermal tumor who developed HSOS following focal radiotherapy and actinomycin-D treatment...
March 14, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29536526/modeling-and-simulation-support-eltrombopag-dosing-in-pediatric-patients-with-immune-thrombocytopenia
#17
Mary B Wire, Xiaobin Li, Jianping Zhang, William Sallas, Vassilios Aslanis, Taoufik Ouatas
Our objective was to support initial eltrombopag doses and dose titration based on modeling and simulation of plasma exposure and platelet count response in pediatric patients aged 1-17 years with previously treated chronic immune thrombocytopenia enrolled in two clinical studies. Data from 168 pediatric patients were used to develop a life-span population pharmacokinetic and pharmacodynamic model including three pharmacokinetic and four pharmacodynamic compartments enabling simulation of platelet counts for various starting doses and dose titration schedules...
March 14, 2018: Clinical Pharmacology and Therapeutics
https://www.readbyqxmd.com/read/29533113/irinotecan-and-temozolomide-in-recurrent-ewing-sarcoma-an-analysis-in-51-adult-and-pediatric-patients
#18
E Palmerini, R L Jones, E Setola, P Picci, E Marchesi, R Luksch, G Grignani, M Cesari, A Longhi, M E Abate, A Paioli, Z Szucs, L D'ambrosio, K Scotlandi, F Fagioli, S Asaftei, S Ferrari
BACKGROUND: Data on temozolomide (TEM) and irinotecan (IRI) activity in recurrent Ewing sarcoma (EWS), especially in adult patients, are limited. METHODS: Patients receiving TEM 100 mg/m2 /day oral, and IRI 40 mg/m2 /day intravenous, days 1-5, every 21 days, were included in this multi-institutional retrospective study. Disease control rate (DCR) [overall response rate (ORR) [complete response (CR) + partial response (PR)] + stable disease (SD)], 6-months progression-free survival (6-mos PFS) and 1-year overall survival (OS) were assessed...
March 13, 2018: Acta Oncologica
https://www.readbyqxmd.com/read/29516627/a-comparative-prospective-observational-study-of-children-and-adults-with-immune-thrombocytopenia-2-year-follow-up
#19
Alexandra Schifferli, Andreas Holbro, Meera Chitlur, Michael Coslovsky, Paul Imbach, Hugo Donato, Mohsen Elalfy, Elena Graciela, John Grainger, Susanne Holzhauer, Cecilia Riccheri, Francesco Rodeghiero, Marco Ruggeri, Hannah Tamary, Tatjana Uglova, Runhui Wu, Thomas Kühne
Comparative clinical studies of children and adults with immune thrombocytopenia (ITP) are poorly covered in the literature. However, the accepted classification of ITP-childhood ITP and adult ITP-results in considerable differences in treatment protocols and practice guidelines. The analysis of the Pediatric and Adult Registry on Chronic ITP (PARC-ITP) of patients at first presentation demonstrated fewer differences in clinical and laboratory findings at initial diagnosis between children and adults than expected...
March 8, 2018: American Journal of Hematology
https://www.readbyqxmd.com/read/29487060/eltrombopag-for-use-in-children-with-immune-thrombocytopenia
#20
REVIEW
Taylor Olmsted Kim, Jenny Despotovic, Michele P Lambert
Eltrombopag is currently the only US Food and Drug Administration-approved thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia (ITP) in children. This oral, once-per-day therapy has shown favorable efficacy and adverse effect profiles in children. Two multicenter, double-blind, placebo controlled clinical trials (PETIT [Efficacy and Safety Study of Eltrombopag in Pediatric Patients With Thrombocytopenia From Chronic Idiopathic Thrombocytopenic Purpura (ITP)] and PETIT2 [Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body]) demonstrated efficacy in raising platelet counts, reducing bleeding, and reducing the need for concomitant ITP therapies with relatively few adverse effects...
February 27, 2018: Blood Advances
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