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https://www.readbyqxmd.com/read/28774273/thrombotic-microangiopathy-associated-with-valproic-acid-toxicity
#1
Sean A Hebert, Timothy P Bohan, Christian L Erikson, Rita D Swinford
BACKGROUND: Thrombotic microangiopathy (TMA) is a serious, sometimes life-threatening disorder marked by the presence of endothelial injury and microvascular thrombi. Drug-induced thrombotic microangiopathy (DI-TMA) is one specific TMA syndrome that occurs following drug exposure via drug-dependent antibodies or direct tissue toxicity. Common examples include calcineurin inhibitors Tacrolimus and Cyclosporine and antineoplastics Gemcitabine and Mitomycin. Valproic acid has not been implicated in DI-TMA...
August 3, 2017: BMC Nephrology
https://www.readbyqxmd.com/read/28751444/pediatric-phase-1-trial-and-pharmacokinetic-study-of-trebananib-in-relapsed-solid-tumors-advl1115-a-children-s-oncology-group-phase-1-consortium-report
#2
Sarah E S Leary, Julie R Park, Joel M Reid, Andrew T Ralya, Sylvain Baruchel, Bing Wu, Timothy P L Roberts, Xiaowei Liu, Charles G Minard, Elizabeth Fox, Brenda J Weigel, Susan Blaney
Trebananib is a first-in-class anti-angiogenic peptibody (peptide-Fc fusion protein) that inhibits Angiopoietin 1 and 2. A pediatric phase 1 trial was performed to define trebananib dose limiting toxicities (DLT), recommended phase 2 dose (RP2D) and pharmacokinetics (PK).<br /><br />Experimental Design: Trebananib was administered by weekly infusion. Three dose levels (10, 15 or 30 mg/kg/dose) were evaluated using a rolling-six design. Part 2 evaluated a cohort of subjects with primary central nervous system (CNS) tumors...
July 27, 2017: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
https://www.readbyqxmd.com/read/28749962/severe-imported-malaria-in-children-in-france-a-national-retrospective-study-from-1996-to-2005
#3
Pierre Mornand, Catherine Verret, Philippe Minodier, Albert Faye, Marc Thellier, Patrick Imbert
BACKGROUNDS: Malaria is a leading cause of imported febrile illnesses in pediatric travelers, but few studies have addressed severe imported pediatric malaria. We aimed to determine the risk factors and the features of imported pediatric severe malaria. METHODS: We conducted a retrospective, descriptive study using the French National Reference Center for Imported Malaria database, in children aged 0-15 years who were hospitalized with a falciparum malaria from January 1st 1996 to December 31th 2005...
2017: PloS One
https://www.readbyqxmd.com/read/28737597/a-prospective-study-of-the-association-between-clinically-significant-bleeding-in-picu-patients-and-thrombocytopenia-or-prolonged-coagulation-times
#4
Paul C Moorehead, Nicholas J Barrowman, Janelle Cyr, Jamie Ray, Robert Klaassen, Kusum Menon
OBJECTIVE: There are no proven methods to predict the risk of clinically significant bleeding in the PICU. A retrospective study identified platelet count as a risk marker for clinically significant bleeding. We conducted a study to examine any association of platelet count, international normalized ratio, and activated partial thromboplastin time with bleeding risk in PICU patients. DESIGN: Prospective observational cohort study. SETTING: The PICU at the Children's Hospital of Eastern Ontario, a university-affiliated tertiary care pediatric center...
July 21, 2017: Pediatric Critical Care Medicine
https://www.readbyqxmd.com/read/28734656/patterns-of-emergency-department-care-for-newly-diagnosed-immune-thrombocytopenia-in-united-states-children-s-hospitals
#5
LaQuita Jones, Terah Koch, Joseph Stanek, Sarah H O'Brien
We used the Pediatric Health Information Systems database to ascertain treatment patterns of immune thrombocytopenia across the US. Despite the recently published guidelines by the American Society of Hematology, most patients are still being hospitalized for immune thrombocytopenia, even in the absence of documented bleeding symptoms.
July 19, 2017: Journal of Pediatrics
https://www.readbyqxmd.com/read/28719512/a-pediatric-case-of-persistent-thrombocytopenia-in-a-patient-who-developed-all
#6
Haroon Shaukat, Jessica O'Mara, Michelle Neier
Inherited thrombocytopenia is a topic that was expanded greatly over the last decade and many new genes are being identified. However, inheritance patterns are not always easy to identify because sporadic cases from de novo mutations may in fact be more common. Few studies have assessed the relationship between thrombocytopenia and malignancies, specifically acute lymphoblastic leukemia (ALL). Here we present a pediatric case of persistent thrombocytopenia associated with T-cell ALL. Our patient was initially diagnosed with immune thrombocytopenic purpura with no evidence of malignancy on bone marrow biopsy but presented shortly after with ALL...
July 17, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28712817/clinical-features-and-dynamic-ordinary-laboratory-tests-differentiating-dengue-fever-from-other-febrile-illnesses-in-children
#7
Chih-Ho Chen, Yi-Chuan Huang, Kuang-Che Kuo, Chung-Chen Li
BACKGROUND: Dengue fever is not easily to be diagnosed before presentation of the classic symptoms. The study aimed to investigate the clinical features and dynamic laboratory tests in pediatric patients to facilitate dengue diagnosis. METHODS: This retrospective study examined the medical records of all pediatric patients who were clinically suspected to have dengue from June to December 2014. Laboratory-positive dengue cases were confirmed by detecting non-structural protein NS1, reverse transcription-polymerase chain reaction of dengue virus, and dengue-specific IgM seroconversion...
June 30, 2017: Journal of Microbiology, Immunology, and Infection, Wei Mian Yu Gan Ran za Zhi
https://www.readbyqxmd.com/read/28698848/treatment-and-response-of-autoimmune-cytopenia-occurring-after-allogeneic-hematopoietic-cell-transplantation-in-children
#8
Seok Hwang-Bo, Seong-Koo Kim, Jae Wook Lee, Pil-Sang Jang, Nack-Gyun Chung, Dae-Chul Jeong, Bin Cho, Hack-Ki Kim
BACKGROUND: Autoimmune cytopenia (AIC) is a rare complication of allogeneic hematopoietic cell transplantation (HCT). In this study, we reviewed the diagnosis, treatment and response to therapy for pediatric patients with post-HCT AIC at our institution. METHODS: Of the 292 allogeneic HCTs performed from January, 2011 to December, 2015 at the Department of Pediatrics, The Catholic University of Korea, seven were complicated by post-HCT AIC, resulting in an incidence of 2...
June 2017: Blood Research
https://www.readbyqxmd.com/read/28641574/intermittent-low-platelet-counts-hampering-diagnosis-of-x-linked-thrombocytopenia-in-children-report-of-two-unrelated-cases-and-a-novel-mutation-in-the-gene-coding-for-the-wiskott-aldrich-syndrome-protein
#9
Samuel Souza Medina, Lúcia Helena Siqueira, Marina Pereira Colella, Gabriela Goes Yamaguti-Hayakawa, Bruno Kosa Lino Duarte, Maria Marluce Dos Santos Vilela, Margareth Castro Ozelo
BACKGROUND: Thrombocytopenia can occur in different circumstances during childhood and although immune thrombocytopenia is its most frequent cause, it is important to consider other conditions, especially when there is a persistent or recurrent low platelet count. We report two cases of intermittent thrombocytopenia, previously misdiagnosed as immune thrombocytopenia. CASES PRESENTATION: Both cases described were boys who presented with an intermittent pattern of thrombocytopenia, with a persistently low mean platelet volume...
June 22, 2017: BMC Pediatrics
https://www.readbyqxmd.com/read/28627134/u-s-food-and-drug-administration-approval-summary-eltrombopag-for-the-treatment-of-pediatric-patients-with-chronic-immune-idiopathic-thrombocytopenia
#10
Lori A Ehrlich, Virginia E Kwitkowski, Gregory Reaman, Chia-Wen Ko, Lei Nie, Richard Pazdur, Ann T Farrell
The U.S. Food and Drug Administration (FDA) approved eltrombopag for pediatric patients with chronic immune (idiopathic) thrombocytopenia (ITP) ages ≥6 on June 11, 2015, and ages ≥1 on August 24, 2015. Approval was based on the FDA review of two randomized trials that included 159 pediatric patients with chronic ITP who had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. This manuscript describes the basis for approval of these applications. The FDA concluded that eltrombopag has shown efficacy and a favorable benefit to risk profile for pediatric patients with chronic ITP...
June 19, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28617714/perioperative-chemotherapy-with-and-without-high-dose-methotrexate-in-adult-osteosarcoma
#11
Marcelo Vailati Negrão, Lucila S da Silva Rocha, Daniel da Motta Girardi, Olavo Feher
Treatment of adult osteosarcoma (AOS) includes perioperative chemotherapy and surgery. Standard chemotherapy consists of cisplatin (CP) and doxorubicin (DOX). Although considered the standard of care for pediatric patients, high-dose methotrexate (HDM) remains controversial in adults. We aimed to evaluate the role of HDM in AOS treated with curative intent. This study included patients with AOS who received perioperative chemotherapy with DOX and CP (group 1; N=16) and DOX, CP, and HDM (group 2; N=10). The primary endpoint was grade 3 or superior toxicities...
September 2017: Anti-cancer Drugs
https://www.readbyqxmd.com/read/28570300/impact-of-novel-polymorphisms-related-to-cytotoxicity-of-cytarabine-in-the-induction-treatment-of-acute-myeloid-leukemia
#12
Juan E Megías-Vericat, Pau Montesinos, María J Herrero, Federico Moscardó, Virginia Bosó, David Martínez-Cuadrón, José L Poveda, Miguel Á Sanz, Salvador F Aliño
Several novel single nucleotide polymorphisms (SNPs) involved in cytarabine cytotoxicity and related to clinical outcomes have been reported recently in a series of 232 pediatric patients with acute myeloid leukemia (AML). We report the first adult AML cohort in which the influence of these SNPs in cytarabine efficacy and toxicity was analyzed. Six of polymorphisms with clinical significance in the previous study [rs12036333, rs10758713, rs9883101, rs6550826, IRX2: rs2897047, mutated in colorectal cancers (MCC): rs7729269] were analyzed in a cohort of 225 adult patients at initial diagnosis of AML treated with an induction scheme of idarubicin plus cytarabine...
July 2017: Pharmacogenetics and Genomics
https://www.readbyqxmd.com/read/28562514/actn1-related-macrothrombocytopenia-a-novel-entity-in-the-progressing-field-of-pediatric-thrombocytopenia
#13
Helene Boutroux, Bianca David, Paul Guéguen, Pierre Frange, Anne Vincenot, Guy Leverger, Rémi Favier
The most common cause of thrombocytopenia in children is immune thrombocytopenia. Nevertheless, some atypical cases should evoke the hypothesis of genetic thrombocytopenia. Indeed, in the past years, 30 new genes had been described in the field of inherited thrombocytopenia. We report a series of 11 cases of a newly diagnosed entity: ACTN1-related macrothrombocytopenia. Mutations in the gene ACTN1 cause mild macrothrombocytopenia characterized by elevated mean platelet volume and elevated immature platelet fraction, and low bleeding tendency...
May 29, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28560174/pediatric-huaiyangshan-virus-infection-a-case-report-with-literature-review
#14
Chun-Hui Zhu, Dong Xu, Wei Liu, Di Guo, Qin Ning, Guang Chen
To define the clinical manifestations and laboratory characteristics of pediatric severe fever with thrombocytopenia syndrome (SFTS) case caused by a novel bunyavirus. we retrospectively analyzed a pediatric case of viral SFTS in a 13 year old successfully managed and confirmed to be due to the novel bunyavirus now referred to as Huaiyangshan virus. A literature review of related cases was performed.Our pediatric case was a 13.3-year-old middle school student no underlying disease. Major clinical features included a fever with chills, headache, and dizziness...
2017: IDCases
https://www.readbyqxmd.com/read/28544698/the-use-of-antifibrinolytics-in-pediatric-patients-with-hypoproliferative-thrombocytopenia
#15
Meghan Delaney, Dana C Matthews, Terry B Gernsheimer
Despite the use of evidence-based platelet transfusion therapy during periods of hypoproliferative thrombocytopenia, a large proportion of pediatric hematology/oncology patients continue to suffer from clinically significant bleeding. Antifibrinolytic (AF) drugs have been shown in certain surgical and trauma settings to decrease bleeding, blood transfusion, and improve survival. We conducted a retrospective assessment of the safety of using AF drugs in pediatric patients with hypoproliferative thrombocytopenia at our center as well as the impact on bleeding occurrence and severity...
May 24, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28543515/thrombocytopenia-associated-with-localized-scleroderma-report-of-four-pediatric-cases-and-review-of-the-literature
#16
Ankur Kumar Jindal, Anju Gupta, Sunil Dogra, Amit Rawat, Deepti Suri, Jasmina Ahluwalia, Surjit Singh
We report on four children with localized scleroderma (morphea) and thrombocytopenia. All four had the en coup de sabre subtype of morphea and had varying degrees of thrombocytopenia (8 × 10(9) /L to 120 × 10(9) /L). None of them had major bleeding manifestations, and thrombocytopenia resolved with treatment of morphea. (One patient was also given an injection of anti-D immunoglobulin.) We propose that thrombocytopenia associated with localized scleroderma is usually benign and requires no specific therapy...
May 23, 2017: Pediatric Dermatology
https://www.readbyqxmd.com/read/28537785/a-practical-guide-to-the-use-of-eltrombopag-in-children-with-chronic-immune-thrombocytopenia
#17
John D Grainger, Sharon Thind
INTRODUCTION: Pediatric immune thrombocytopenia (ITP) may be associated with significant burden on children and their parents/caregivers. Thrombopoietin (TPO) receptor agonists (RAs) have been used to treat adult patients with chronic ITP (cITP) for nearly a decade and following pediatric studies Eltrombopag has been recently approved for pediatric cITP in the United States and Europe. TPO-RA s may help reduce the risk of bleeding and the need for conventional ITP therapies. REVIEW: In this review, the clinical data demonstrating the efficacy and safety of TPO-RAs in pediatric ITP are evaluated, key recommendations regarding safe administration of eltrombopag are provided, and potential future directions in management of pediatric ITP are discussed...
March 2017: Pediatric Hematology and Oncology
https://www.readbyqxmd.com/read/28530458/-early-symptoms-of-childhood-malignant-diseases
#18
Zsuzsanna Erzsébet Papp, Izabella Kelemen, Adrienne Horváth
INTRODUCTION: Childhood malignant diseases are rare in pediatric pathology. Early symptoms are not specific, fatigue, pallor, compression signs and bone marrow failure are often mentioned. AIM: To summarize the most frequent early symptoms of childhood malignancies in order to help the physicians in the early recognition. METHOD: In our retrospective study, we processed a period of 5 years between 2012-2016, with an accent on the onset manifestations of malignancies...
May 2017: Orvosi Hetilap
https://www.readbyqxmd.com/read/28520981/clinical-presentation-and-outcomes-of-children-with-human-granulocytic-anaplasmosis
#19
Anna M Schotthoefer, Matthew C Hall, Satya Vittala, Raza Bajwa, Holly M Frost
Background.: Adults with the tick-borne disease human granulocytic anaplasmosis (HGA) have a spectrum of acute febrile illnesses that, if untreated, might be severe. Clinical presentation and outcomes of children with HGA have been poorly described. Methods.: A retrospective analysis was conducted to determine the frequency, presentation, and outcomes of pediatric patients with HGA between 1994 and 2015 in a region of Wisconsin in which HGA is highly endemic. Patients with related International Classification of Diseases Ninth and Tenth Revision (ICD-9 and ICD-10, respectively) codes or positive HGA laboratory test results were evaluated and classified as having had confirmed, probable, or suspected HGA on the basis of the Centers for Disease Control and Prevention (CDC) case definition...
May 16, 2017: Journal of the Pediatric Infectious Diseases Society
https://www.readbyqxmd.com/read/28499592/risk-factors-for-poor-outcomes-of-children-with-acute-acalculous-cholecystitis
#20
Yi-An Lu, Cheng-Hsun Chiu, Man-Shan Kong, Han-I Wang, Hsun-Chin Chao, Chien-Chang Chen
BACKGROUND: Acute acalculous cholecystitis (AAC) is generally considered to be a mild disease in children; however, if left untreated or treated without caution, AAC can lead to severe outcomes, such as death. The objectives of this study were to present the clinical features and identify the predictors of mortality in pediatric AAC. METHODS: Patients diagnosed with AAC between 2005 and 2012 were enrolled. AAC was defined by the presence of fever and an echo-proven thickened gallbladder wall exceeding 4 mm...
March 25, 2017: Pediatrics and Neonatology
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