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pediatrics, thrombocytopenia

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https://www.readbyqxmd.com/read/28185830/children-with-type-1-gaucher-disease-changing-profiles-in-the-21st-century
#1
Deborah Elstein, Gheona Altarescu, Aya Abrahamov, Ari Zimran
Gaucher disease (GD) has phenotypic variability. Increased GD awareness especially among at-risk Ashkenazi Jews (AJ) and availability of non-invasive diagnosis induced trend to prenatal screening. We retrospectively assessed pediatric (<16years) Israeli AJ GD patients to ascertain demographics and phenotype at presentation and over-time because many were identified by large-scale screening. 55/67 patients born since 01/01/2000 are AJ with non-neuronopathic GD: 28 (50.9%) are N370S/N370S; 24 (43.6%) are N370S/other; 3 (3...
December 19, 2016: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/28176457/predictors-of-acupuncture-use-among-children-and-adolescents-with-cancer
#2
Sagar K Chokshi, Elena J Ladas, Katherine Taromina, Douglas McDaniel, Diane Rooney, Zhezhen Jin, Wei-Chun Hsu, Kara M Kelly
BACKGROUND: Evidence for the application of acupuncture in pediatric oncology is limited. We investigated the acceptance of acupuncture and factors associated with its use among children and adolescents with cancer. METHODS: Ninety acupuncture-naïve children receiving cancer treatment at Columbia University Medical Center (CUMC) provided consent/assent for participation. Participants could choose to receive or refuse integrative services offered at CUMC. Symptoms were collected for a 6-month period with the Memorial Symptom Assessment Scale (MSAS)...
February 8, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28150377/romiplostim-for-therapy-related-thrombocytopenia-in-pediatric-malignancies
#3
Amanda E Jacobson, Nilay Shah, Bhuvana A Setty
Therapy-related thrombocytopenia (TRT), due to chemotherapy and/or radiation therapy, is common with pediatric cancer treatments, and it can result in dose reductions and therapy delays. Romiplostim, a thrombopoietin mimetic, is efficacious as a second-line treatment for immune thrombocytopenia in children and for TRT in adult cancer patients. However, there are no data for its use for TRT in children. We report a case series of five children treated for solid tumors where romiplostim was used without adverse effects to successfully resolve and prevent therapy-limiting refractory TRT...
February 2, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28149700/retrospective-study-of-prognostic-factors-in-pediatric-invasive-pneumococcal-disease
#4
Nan-Chang Chiu, Hsin Chi, Chun-Chih Peng, Hung-Yang Chang, Daniel Tsung-Ning Huang, Lung Chang, Wei-Te Lei, Chien-Yu Lin
Streptococcus pneumoniae remains the leading causative pathogen in pediatric pneumonia and bacteremia throughout the world. The invasive pneumococcal disease (IPD) is known as isolation of S. pneumoniae from a normally sterile site (e.g., blood, cerebrospinal fluid, synovial fluid, pericardial fluid, pleural fluid, or peritoneal fluid). The aim of this study is to survey the clinical manifestations and laboratory results of IPD and identify the prognostic factors of mortality. From January 2001 to December 2006, a retrospective review of chart was performed in a teaching hospital in Taipei...
2017: PeerJ
https://www.readbyqxmd.com/read/28134038/outcomes-of-847-childhood-onset-systemic-lupus-erythematosus-patients-in-three-age-groups
#5
S R M Lopes, N W S Gormezano, R C Gomes, N E Aikawa, R M R Pereira, M T Terreri, C S Magalhães, J C Ferreira, E M Okuda, A P Sakamoto, A M E Sallum, S Appenzeller, V P L Ferriani, C M Barbosa, S Lotufo, A A Jesus, L E C Andrade, L M A Campos, E Bonfá, C A Silva
Objective The objective of this study was to assess outcomes of childhood systemic lupus erythematosus (cSLE) in three different age groups evaluated at last visit: group A early-onset disease (<6 years), group B school age (≥6 and <12 years) and group C adolescent (≥12 and <18 years). Methods An observational cohort study was performed in ten pediatric rheumatology centers, including 847 cSLE patients. Results Group A had 39 (4%), B 395 (47%) and C 413 (49%). Median disease duration was significantly higher in group A compared to groups B and C (8...
January 1, 2017: Lupus
https://www.readbyqxmd.com/read/28111925/combination-of-bevacizumab-irinotecan-and-temozolomide-for-refractory-or-relapsed-neuroblastoma-results-of-a-phase-ii-study
#6
Shakeel Modak, Brian H Kushner, Ellen Basu, Stephen S Roberts, Nai-Kong V Cheung
BACKGROUND: The rationale for studying the combination of bevacizumab, irinotecan, and temozolomide (BIT) in neuroblastoma (NB) is based on the following: (i) vascular endothelial growth factor (VEGF) expression is associated with an aggressive phenotype, (ii) anti-VEGF antibody bevacizumab enhances irinotecan-mediated suppression of NB xenografts, (iii) bevacizumab safety has been established in pediatric phase I studies, and (iv) irinotecan + temozolomide (IT) is a standard salvage chemotherapy...
January 23, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28103732/evaluation-of-antivenom-therapy-for-vipera-palaestinae-bites-in-children-experience-of-two-large-tertiary-care-pediatric-hospitals
#7
Dikla Pivko-Levy, Itamar Munchnak, Ayelet Rimon, Uri Balla, Dennis Scolnik, Christopher Hoyte, Yair Voliovitch, Miguel Glatstein
BACKGROUND: Antivenom has been successfully used to treat systemic and progressive, local manifestations of envenomation inflicted by Vipera (V.) palaestinae, the most common venomous snake in Israel. The objective of this study was to evaluate the fixed dose V. palaestinae monovalent (equine) immunoglobulin G antivenom used in two pediatric emergency departments. In particular, we wanted to assess the need for repeated antivenom administration and the rate of adverse antivenom effects in children...
January 20, 2017: Clinical Toxicology
https://www.readbyqxmd.com/read/28098429/natural-history-of-nonimmune-mediated-thrombocytopenia-and-acute-kidney-injury-in-pediatric-open-heart-surgery
#8
Shannon Tew, Manuel L Fontes, Nathaniel H Greene, Miklos D Kertai, George Ofori-Amanfo, Robert D B Jaquiss, Andrew J Lodge, Warwick A Ames, Hercilia Mayumi Homi, Kelly A Machovec, Edmund H Jooste
OBJECTIVE: Thrombocytopenia and acute kidney injury (AKI) are common following pediatric cardiac surgery with cardiopulmonary bypass (CPB). However, the relationship between postoperative nadir platelet counts and AKI has not been investigated in the pediatric population. Our objective was to investigate this relationship and examine independent predictors of AKI. DESIGN: After IRB approval, we performed a retrospective review of the institution's medical records and database...
January 18, 2017: Paediatric Anaesthesia
https://www.readbyqxmd.com/read/28087540/international-evidence-based-consensus-diagnostic-criteria-for-hhv-8-negative-idiopathic-multicentric-castleman-disease
#9
David C Fajgenbaum, Thomas S Uldrick, Adam Bagg, Dale Frank, David Wu, Gordan Srkalovic, David Simpson, Amy Y Liu, David Menke, Shanmuganathan Chandrakasan, Mary Jo Lechowicz, Raymond S M Wong, Sheila Pierson, Michele Paessler, Jean-François Rossi, Makoto Ide, Jason Ruth, Michael Croglio, Alexander Suarez, Vera Krymskaya, Amy Chadburn, Gisele Colleoni, Sunita Nasta, Raj Jayanthan, Christopher S Nabel, Corey Casper, Angela Dispenzieri, Alexander Fosså, Dermot Kelleher, Razelle Kurzrock, Peter Voorhees, Ahmet Dogan, Kazuyuki Yoshizaki, Frits van Rhee, Eric Oksenhendler, Elaine S Jaffe, Kojo S J Elenitoba-Johnson, Megan S Lim
Human Herpesvirus-8(HHV-8)-negative, idiopathic multicentric Castleman disease (iMCD) is a rare and life-threatening disorder involving systemic inflammatory symptoms, polyclonal lymphoproliferation, cytopenias, and multiple organ system dysfunction due to a cytokine storm often including interleukin-6. iMCD accounts for one-third to one-half of all cases of MCD and can occur in individuals of any age. Accurate diagnosis is challenging, as no standard diagnostic criteria or diagnostic biomarkers currently exist, and there is significant overlap with malignant, autoimmune, and infectious disorders...
January 13, 2017: Blood
https://www.readbyqxmd.com/read/28079914/intravenous-immunoglobulin-in-pediatric-rheumatology-when-to-use-it-and-what-is-the-evidence
#10
Martha M Rodriguez, Linda Wagner-Weiner
Intravenous immunoglobulin (IVIG) is given to children with a variety of rheumatologic illnesses. The mechanism of action by which it exerts therapeutic effects is not well understood and likely differs in the medical conditions for which it is given. IVIG is approved by the US Food and Drug Administration and is the standard of care for Kawasaki disease, but most IVIG use in pediatric rheumatology is "off-label. " The literature supports the use of IVIG for juvenile dermatomyositis, although it is unclear whether its use should be limited to those children with more severe or refractory disease...
January 1, 2017: Pediatric Annals
https://www.readbyqxmd.com/read/28062243/extramedullary-relapse-of-acute-lymphoblastic-leukemia-presenting-as-abnormal-uterine-bleeding-a-case-report
#11
Diana T Robillard, Matthew A Kutny, Joseph H Chewning, Janeen L Arbuckle
BACKGROUND: Acute lymphoblastic leukemia (ALL) is the most common childhood malignancy. Relapse of ALL occurs in 15-20% of patients, with 2-6% occurring exclusively in extramedullary sites. Relapse of ALL in gynecologic organs is extremely rare. CASE: We present a case of a 12 year old with a history of ALL who was referred to the pediatric gynecology clinic with abnormal uterine bleeding. She was determined to have an extramedullary uterine relapse of her ALL. SUMMARY AND CONCLUSION: Abnormal uterine bleeding in the setting of childhood malignancy is a frequent reason for consultation to Pediatric and Adolescent Gynecology services...
January 3, 2017: Journal of Pediatric and Adolescent Gynecology
https://www.readbyqxmd.com/read/28060550/evaluating-platelet-function-disorders-in-children-with-bleeding-tendency-a-single-center-study
#12
Osama Tanous, Orna Steinberg Shemer, Joanne Yacobovich, Meira Zoldan, Yoseph Horovitz, Isaac Yaniv, Esther Rabizadeh, Hannah Tamary, Sigal Nakav, Judith Lahav
Platelet function disorders (PFDs) are a common cause of mild bleeding tendency. However, they cannot be recognized by standard screening studies. The gold standard test for PFD is platelet aggregation, performed by light transmission aggregometry (LTA). A newer and less validated method is the closure time (CT), performed by the platelet function Analyzer 100 (PFA-100). Data regarding the validity of these tests in children are limited. The aim of this study was to evaluate the usefulness of LTA and PFA-100 for the diagnosis of pediatric patients with bleeding tendency...
January 6, 2017: Platelets
https://www.readbyqxmd.com/read/28059092/bone-marrow-morphology-and-disease-progression-in-congenital-thrombocytopenia-a-detailed-clinicopathologic-and-genetic-study-of-eight-cases
#13
Hamilton C Tsang, James B Bussel, Susan Mathew, Yen-Chun Liu, Allison A Imahiyerobo, Attilio Orazi, Julia T Geyer
Patients with congenital thrombocytopenia have an increased risk of developing myeloid neoplasms. In these cases, the morphologic distinction between disease at baseline and at progression is challenging. This report analyzes clinicopathologic features of congenital thrombocytopenia with long-term follow-up at one referral center. Records from the last 20 years were searched for cases of congenital thrombocytopenia with bone marrow biopsies and peripheral blood smears. The clinical, morphologic, immunophenotypic, and molecular features were analyzed...
January 6, 2017: Modern Pathology: An Official Journal of the United States and Canadian Academy of Pathology, Inc
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
#14
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/28035748/a-phase-i-study-of-perifosine-with-temsirolimus-for-recurrent-pediatric-solid-tumors
#15
Oren J Becher, Stephen W Gilheeney, Yasmin Khakoo, David C Lyden, Sofia Haque, Kevin C De Braganca, Jill M Kolesar, Jason T Huse, Shakeel Modak, Leonard H Wexler, Kim Kramer, Ivan Spasojevic, Ira J Dunkel
BACKGROUND: The PI3K/AKT/mTOR pathway is aberrantly activated in many pediatric solid tumors including gliomas and medulloblastomas. Preclinical data in a pediatric glioma model demonstrated that the combination of perifosine (AKT inhibitor) and temsirolimus (mTOR inhibitor) is more potent at inhibiting the axis than either agent alone. We conducted this study to assess pharmacokinetics and identify the maximum tolerated dose for the combination. PROCEDURE: We performed a standard 3+3 phase I, open-label, dose-escalation study in patients with recurrent/refractory pediatric solid tumors...
December 30, 2016: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/27998672/quantitation-of-bleeding-symptoms-in-a-national-registry-of-patients-with-inherited-platelet-disorders
#16
Shoshana Revel-Vilk, Chana Richter, Tal Ben-Ami, Joanne Yacobovich, Shraga Aviner, Ayelet Ben-Barak, Amir Asher Kuperman, Shira Ben-Barak, Chaim Kaplinsky, Hagit Miskin, Hannah Tamary, Gili Kenet
BACKGROUND: Inherited platelet deficiency and/or dysfunction may be more common in the general population than has previously been appreciated. In 2013 the Israeli Inherited Platelet Disorder (IPD) Registry was established. METHODS: Clinical and laboratory data were collected to pre-specified registration forms. The study protocol was approved by the local hospital ethics committees. RESULTS: To date we have included in the registry 89 patients (male 52%) from 79 families...
December 17, 2016: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/27998318/management-of-severe-dengue-hemorrhagic-fever-and-bleeding-complications-in-a-primigravida%C3%A2-patient-a-case-report
#17
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
https://www.readbyqxmd.com/read/27998223/phase-i-phase-ii-study-of-blinatumomab-in-pediatric-patients-with-relapsed-refractory-acute-lymphoblastic-leukemia
#18
Arend von Stackelberg, Franco Locatelli, Gerhard Zugmaier, Rupert Handgretinger, Tanya M Trippett, Carmelo Rizzari, Peter Bader, Maureen M O'Brien, Benoît Brethon, Deepa Bhojwani, Paul Gerhardt Schlegel, Arndt Borkhardt, Susan R Rheingold, Todd Michael Cooper, Christian M Zwaan, Phillip Barnette, Chiara Messina, Gérard Michel, Steven G DuBois, Kuolung Hu, Min Zhu, James A Whitlock, Lia Gore
Purpose Blinatumomab is a bispecific T-cell engager antibody construct targeting CD19 on B-cell lymphoblasts. We evaluated the safety, pharmacokinetics, recommended dosage, and potential for efficacy of blinatumomab in children with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (BCP-ALL). Methods This open-label study enrolled children < 18 years old with relapsed/refractory BCP-ALL in a phase I dosage-escalation part and a phase II part, using 6-week treatment cycles. Primary end points were maximum-tolerated dosage (phase I) and complete remission rate within the first two cycles (phase II)...
December 20, 2016: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/27977557/safety-of-high-dose-acyclovir-in-infants-with-suspected-and-confirmed-neonatal-herpes-simplex-virus-infections
#19
Jessica E Ericson, Martyn Gostelow, Julie Autmizguine, Christoph P Hornik, Reese H Clark, Daniel K Benjamin, P Brian Smith
BACKGROUND: Acyclovir is used to treat herpes simplex virus (HSV) disease in infants. Treatment with high dose acyclovir, 60 mg/kg/day, is recommended; however, the safety of this dosage has not been assessed in the past 15 years, and this dosage is not currently approved for infants by the US Food and Drug Administration. METHODS: We included infants with neonatal HSV disease treated with ≥14 days of intravenous acyclovir starting in the first 120 days of life admitted to 1 of 42 neonatal intensive care units managed by the Pediatrix Medical Group from 2002-2012...
December 12, 2016: Pediatric Infectious Disease Journal
https://www.readbyqxmd.com/read/27976561/congenital-malaria-importance-of-diagnosis-and-treatment-in-pregnancy
#20
Selvi Gülaşı, Nureddin Özdener
Congenital malaria, in which infants are directly infected with malaria parasites from their mother prior to or during birth, is a potentially life-threatening condition that occurs at relatively low rates in malaria endemic regions. We report an unusual case of a 23-day-old girl with neonatal Plasmodium vivax malaria, suspected primarily on the basis of positive maternal history that her mother had malaria during her pregnancy and was cured with chloroquine therapy. Infant presented with fever, thrombocytopenia and a significant parasitemia...
2016: Turkish Journal of Pediatrics
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