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((Myelodysplastic Syndrome) OR (MD)) AND Pedia*

Patrick C Bonasso, Kevin W Sexton, Md Abul Hayat, Jingxian Wu, Hanna K Jensen, Morten O Jensen, Jeffrey M Burford, Melvin S Dassinger
BACKGROUND: No standard dehydration monitor exists for children. This study attempts to determine the utility of Fast Fourier Transform (FFT) of a peripheral venous pressure (PVP) waveform to predict dehydration. MATERIALS AND METHODS: PVP waveforms were collected from 18 patients. Groups were defined as resuscitated (serum chloride ≥ 100 mmol/L) and hypovolemic (serum chloride < 100 mmol/L). Data were collected on emergency department admission and after a 20 cc/kg fluid bolus...
February 15, 2019: Journal of Surgical Research
Josh M Colston, Ruthly Francois, Nora Pisanic, Pablo Peñataro Yori, Benjamin J J McCormick, Maribel Paredes Olortegui, Md Amran Gazi, Erling Svensen, Mondar Maruf Moin Ahmed, Esto Mduma, Jie Liu, Eric R Houpt, Robert Klapheke, Julia W Schwarz, Robert L Atmar, Robert E Black, Margaret N Kosek
BACKGROUND: Histo blood group antigens (HBGAs) such as fucosyltransferase FUT2 and 3 may act as innate host factors that differentially influence susceptibility of individuals and their offspring to pediatric enteric infections. METHODS: In three community-based birth cohorts, FUT2 and FUT3 statuses were ascertained for mother-child dyads. qPCR panels tested 3,663 diarrheal and 18,148 asymptomatic stool samples for 29 enteropathogens. Cumulative diarrhea and infection incidence were compared by child (n=520) and mothers' (n=519) HBGA status and hazard ratios (HRs) derived for all-cause diarrhea and specific enteropathogens...
February 15, 2019: Journal of Infectious Diseases
Leticia Manning Ryan, Sarah Lindstrom Johnson, Vanya Jones, Joel A Fein, Tina L Cheng
The association of household composition with violence-related injury risk has not been explored in the at-risk urban adolescent population. We hypothesize that, similar to the unintentional risk association, higher adult:child ratio, lower household size, and the presence of a grandparent are protective and thus associated with lower risk for repeat fight injury in this population. This is a cross-sectional study of 10- to 15-year-old adolescents who were evaluated in two urban, pediatric emergency departments (EDs; Baltimore, MD, Philadelphia, PA) for a peer fight-related injury between June 2014 and June 2016...
February 15, 2019: Journal of Interpersonal Violence
Yung Lee, Karanbir Brar, Aristithes G Doumouras, Dennis Hong
BACKGROUND: Achalasia is a rare primary esophageal dysmotility disorder in children. Peroral endoscopic myotomy (POEM) is a novel endoscopic technique which has shown promising results for treating achalasia in adults. However, limited data on efficacy and safety in pediatric patients are available. We performed a systematic review and meta-analysis to evaluate the efficacy and safety of POEM in pediatric achalasia. METHODS: We searched MEDLINE, Embase, PubMed, and Web of Science databases through July 2018...
February 14, 2019: Surgical Endoscopy
A Siu Uribe, J I Garrido Pérez, F Vázquez Rueda, M R Ibarra Rodrígue, F J Murcia Pascual, S D Ramnarine Sánchez, R M Paredes Esteban
AIM: To present our experience in pediatric patients with testicular torsion (TT) treated by manual detorsion (MD). PATIENTS AND METHODS: Retrospective analysis of patients treated by MD in a 10-year period in a single center. Description of symptoms, detorsion technique, follow-up and complications. RESULTS: 76 patients diagnosed with TT were studied in a 10-year period. 16 patients were treated by MD. Mean age was 12 years (Range: 10-13 years) and time from onset of pain was 5...
January 21, 2019: Cirugía Pediátrica: Organo Oficial de la Sociedad Española de Cirugía Pediátrica
Prabakaran Paulraj, Steven Diamond, Faisal Razzaqi, Dan Ozeran, Maria Longhurst, Erica F Andersen, Reha M Toydemir, Bo Hong
The t(7;21)(p22;q22) resulting in RUNX1-USP42 fusion, is a rare but recurrent cytogenetic abnormality associated with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The prognostic significance of this translocation has not been well established due to the limited number of patients. Herein, we report three pediatric AML patients with t(7;21)(p22;q22). All three patients presented with pancytopenia or leukopenia at diagnosis, accompanied by abnormal immunophenotypic expression of CD7 and CD56 on leukemic blasts...
January 31, 2019: Genes, Chromosomes & Cancer
X Y Peng, Y J Qu, F Song, X F Sun, X S Ge, H Jiao
Objective: To summarize the clinical manifestations and determine the molecular etiology for two collagen type Ⅵ-related myopathy pedigrees. Methods: Two spontaneous collagen type Ⅵ-related myopathy patients were admitted to Department of Neurology, Children's Hospital, Capital Institute of Pediatrics in October 2017. Clinical data of probands and their family members were collected and their genomic DNA was obtained for genetic testing. Next generation sequencing was performed and the variants were verified by the Sanger sequencing in the family members...
February 2, 2019: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
Ya-Lei Sun, Hai-Tao Zheng, Jia-Lei Tao, Ming-Chen Jiang, Chan-Chan Hu, Xin-Min Li, Bin Yuan
OBJECTIVE: Chinese herbal medicine has been gradually used to treat pediatric adenoid hypertrophy. This meta-analysis were conducted to evaluate the clinical efficacy and safety of Chinese herbal medicine in the treatment of pediatric adenoid hypertrophy. METHODS: Randomized controlled trials involving Chinese herbal medicine in the treatment of pediatric adenoid hypertrophy were identified from Cochrane Central Register of Controlled Trials, PubMed, EMBASE, Chinese National Knowledge Infrastructure, Chinese Biomedical Database, Wanfang Database and VIP Information Database...
January 19, 2019: International Journal of Pediatric Otorhinolaryngology
Mark A Schuster
The following is an address given by the author in receipt of the Joseph W. St. Geme, Jr. Leadership Award, presented by the Federation of Pediatric Organizations at the Pediatric Academic Societies Meeting in Toronto, Canada, on May 5, 2018. Gary R. Fleisher, MD, Chairman of the Department of (Pediatric) Medicine and Physician-in-Chief at Boston Children's Hospital, and the Egan Professor of Pediatrics at Harvard Medical School, introduced the author.
January 25, 2019: Pediatrics
Jonathan H Soslow, Matthew Hall, W Bryan Burnette, Kan Hor, Joanne Chisolm, Christopher Spurney, Justin Godown, Meng Xu, James C Slaughter, Larry W Markham
BACKGROUND: Outcome analyses in large administrative databases are ideal for rare diseases such as Becker and Duchenne muscular dystrophy. Unfortunately, Becker and Duchenne do not yet have specific International Classification of Disease-9/-10 codes. We hypothesised that an algorithm could accurately identify these patients within administrative data and improve assessment of cardiovascular morbidity. METHODS: Hospital discharges (n=13,189) for patients with muscular dystrophy classified by International Classification of Disease-9 code: 359...
January 26, 2019: Cardiology in the Young
Hermine I Brunner, Michael J Holland, Michael W Beresford, Stacy P Ardoin, Simone Appenzeller, Clovis A Silva, Francisco Flores, Beatrice Goilav, Pinar Ozge Avar Aydin, Scott E Wenderfer, Deborah M Levy, Angelo Ravelli, Raju Khubchandani, Tadej Avcin, Marisa S Klein-Gitelman, Nicolino Ruperto, Brian M Feldman, Jun Ying
OBJECTIVES: To develop a Childhood Lupus Improvement Index (CHILI) as a tool to measure response to therapy in childhood-onset systemic lupus erythematosus (cSLE), with focus on clinically relevant improvement (CRIc SLE ). METHODS: Pediatric nephrology and rheumatology subspecialists (n=213) experienced in cSLE management were invited to define CRIc SLE and rate a total of 433 unique patient-profiles for the presence/absence of CRIc SLE . Patient-profiles included the cSLE core response variables [cSLE-CRVs: global assessment of patient well-being (Patient-global), physician assessment of cSLE activity (MD-global), disease activity index score (here: Systemic Lupus Erythematosus Disease Activity Index), urine protein-to-creatinine ratio (UPCR), Child Health Questionnaire physical summary score (CHQ-PhS)]...
January 25, 2019: Arthritis Care & Research
Pascal Amedro, Marie Vincenti, Gregoire De La Villeon, Kathleen Lavastre, Catherine Barrea, Sophie Guillaumont, Charlene Bredy, Lucie Gamon, Albano C Meli, Olivier Cazorla, Jeremy Fauconnier, Pierre Meyer, François Rivier, Jerome Adda, Thibault Mura, Alain Lacampagne
BACKGROUND: Prognosis of Duchenne muscular dystrophy (DMD) is related to cardiac dysfunction. Speckle-tracking echocardiographic (STE) imaging is emerging as a noninvasive functional biomarker to consider in the early detection of DMD-related cardiomyopathy. However, STE analysis has not been assessed in a prospectively controlled study, especially in presymptomatic children with DMD, and no study has used STE analysis in all three displacements (longitudinal, radial, and circumferential) and for both ventricles...
January 21, 2019: Journal of the American Society of Echocardiography
Linda Ewing-Cobbs, Dana M DeMaster, Christopher G Watson, Mary R Prasad, Charles S Cox, Larry A Kramer, Jesse T Fischer, Gerardo A Duque, Paul R Swank
Prefrontal-limbic circuitry is vulnerable to effects of stress and injury. We examined microstructure of prefrontal-limbic circuitry following traumatic brain (TBI) or extracranial injury (EI) and its relation to post-traumatic stress symptoms (PTSS). Participants aged 8 to 15 who sustained mild to severe TBI (n=53) or EI (n=26) in motor vehicle incidents were compared to healthy children (n=38) in a prospective longitudinal study. At the 7-week follow-up, diffusion tensor imaging was obtained in all groups; injured children completed PTSS ratings using a validated scale...
January 23, 2019: Journal of Neurotrauma
Yu Yu, Kang-Chen Chen, Jie Chen
BACKGROUND: Many studies have examined the effects of exclusive enteral nutrition (EEN) in children with Crohn's disease (CD), but corticosteroids are considered a superior therapy and are frequently used in China. This meta-analysis aims to compare the efficacy of EEN with corticosteroids in treating pediatric CD. METHODS: A comprehensive retrieval from medical databases, including PubMed, EMBASE, MEDLINE, Web of Science, Wanfang data, VIP and CNKI, was performed using the search terms "diet therapy", "exclusive enteral nutrition", "Crohn's disease", "inflammatory bowel diseases", "child" and "pediatrics" from January 1990 to April 2017...
January 21, 2019: World Journal of Pediatrics: WJP
Meng Lv, Shu-Zhen Zhai, Yu Wang, Lan-Ping Xu, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Yi-Fei Cheng, Chen-Hua Yan, Xiao-Dong Mo, Kai-Yan Liu, Ying-Jun Chang, Xiao-Jun Huang, Xiang-Yu Zhao
Donor-specific anti-human leukocyte antigen (HLA) antibodies (DSAs) were associated with graft failure (GF) following haploidentical stem cell transplantation (Haplo-HSCT). The prevalence and risk factors of DSAs in pediatric candidates remain to be determined. In a prospective trial (ChiCTR-OPC-15006672), 486 children with hematological diseases were enrolled to screen for the presence of anti-HLA class I and II antibodies of immunoglobulin G type. Fifty two patients (10.7%) demonstrated positive panel-reactive antibody (PRA) for class I; 24 (4...
January 17, 2019: Bone Marrow Transplantation
Francesco Muntoni, Bina Tejura, Stefan Spinty, Helen Roper, Imelda Hughes, Gary Layton, Kay E Davies, Shawn Harriman, Jonathon Tinsley
Ezutromid (SMT C1100) is a small-molecule utrophin modulator that was developed to treat Duchenne muscular dystrophy (DMD). Previous clinical trials of this agent revealed lower exposure in DMD patients compared with healthy volunteers, which may reflect differences in diet. This study evaluated the pharmacokinetics of ezutromid in patients with DMD who followed a balanced diet. This was a multicenter, double-blind, placebo-controlled, ascending single and multiple oral dose study. Twelve pediatric patients were randomly allocated to 1 of 3 treatment sequences within which were 3 treatment periods of 2 weeks each...
January 16, 2019: Clinical Pharmacology in Drug Development
Genovefa A Papanicolaou, Celalettin Ustun, Jo-Anne H Young, Min Chen, Soyoung Kim, Kwang Woo Ahn, Krishna Komanduri, Caroline Lindemans, Jeffery J Auletta, Marcie L Riches
Background: We examined the impact of Enterococcus (VRE) bloodstream infection (BSI) on outcomes of allogeneic hematopoietic cell transplantation (HCT) utilizing the Center for International Blood and Marrow Transplant Research (CIBMTR) database. Methods: Adult and pediatric patients (n=7,128), who underwent first HCT for acute leukemia or myelodysplastic syndrome from 2008 through 2012, were analyzed as 3 groups-VRE-BSI, non-VRE-BSI, without BSI-according to BSI status at 100 days (D100) after allogeneic HCT...
January 14, 2019: Clinical Infectious Diseases: An Official Publication of the Infectious Diseases Society of America
Cristina Jou, Juan D Ortigoza-Escobar, Maria M O'Callaghan, Andres Nascimento, Alejandra Darling, Leticia Pias-Peleteiro, Belén Perez-Dueñas, Mercedes Pineda, Anna Codina, César Arjona, Judith Armstrong, Francesc Palau, Antonia Ribes, Laura Gort, Frederic Tort, Placido Navas, Eduardo Ruiz-Pesini, Sonia Emperador, Ester Lopez-Gallardo, Pilar Bayona-Bafaluy, Raquel Montero, Cecilia Jimenez-Mallebrera, Angels Garcia-Cazorla, Julio Montoya, Delia Yubero, Rafael Artuch
Mitochondrial diseases (MD) are a group of genetic and acquired disorders which present significant diagnostic challenges. Here we report the disease characteristics of a large cohort of pediatric MD patients ( n = 95) with a definitive genetic diagnosis, giving special emphasis on clinical muscle involvement, biochemical and histopathological features. Of the whole cohort, 51 patients harbored mutations in nuclear DNA (nDNA) genes and 44 patients had mutations in mitochondrial DNA (mtDNA) genes. The nDNA patients were more likely to have a reduction in muscle fiber succinate dehydrogenase (SDH) stains and in SDH-positive blood vessels, while a higher frequency of mtDNA patients had ragged red (RRF) and blue fibers...
January 10, 2019: Journal of Clinical Medicine
Hemalatha G Rangarajan, Lauren C Smith, Joseph R Stanek, Matthew Hall, Rolla Abu-Arja, Jeffery J Auletta, Sarah H O'Brien
Allogeneic hematopoietic cell transplant (HCT) is a curative option for patients with acute leukemia and myelodysplastic syndromes (MDS), but is associated with significant cost. When compared to children (age <15 years), adolescents and young adults (AYA, ages 15-39 years) receiving HCT have an increased risk for transplant-related complications. Whether such complications translate into increased resource utilization and costs during HCT remains unknown. Therefore, we conducted a multicenter database study using the Pediatric Health Information System database, an administrative database containing resource utilization data from 49 tertiary children's hospitals in the United States, to compare inpatient costs and resource utilization in children and AYA undergoing HCT for acute leukemia and MDS...
January 6, 2019: Biology of Blood and Marrow Transplantation
Carla J Osigian, Sara F Grace, Kara M Cavuoto, William J Feuer, Mehdi Tavakoli, Piangporn Saksiriwutto, Mu Liu, Hilda Capo, Byron L Lam
PURPOSE: To assess the RETeval (LKC Technologies, Gaithersburg, MD) handheld electroretingram (ERG) device as a screening tool for cone dysfunction in pediatric patients by comparing it to conventional ERG. METHODS: Patients scheduled for ERG under general anesthesia (GA) underwent three tests: (1) RETeval standard 30 Hz cone flicker ERG using skin electrodes prior to GA, (2) E3 Diagnosys (Diagnosys LLC, Lowell, MA) conventional complete standard protocol full-field ERG using bipolar contact lens electrodes and handheld stimulus under GA, and (3) repeat RETeval testing under GA...
January 6, 2019: Journal of AAPOS: the Official Publication of the American Association for Pediatric Ophthalmology and Strabismus
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