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https://www.readbyqxmd.com/read/30341538/donor-derived-marrow-mesenchymal-stromal-cell-co-transplantation-following-a-haploidentical-hematopoietic-stem-cell-transplantation-trail-to-treat-severe-aplastic-anemia-in-children
#1
Z -K Wang, H -J Yu, F -L Cao, Z -H Liu, Z -Y Liu, W -J Feng, X -L Liu, Y -Y Yu, Y Xiao, L -M Li, J Zhou
Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is associated with an increased risk of graft failure and severe graft-versus-host disease (GVHD). Recent studies have shown that mesenchymal stromal cells (MSCs) display potent immunosuppressive effects and can support normal hematopoiesis. In a multi-center trial, we co-transplanted culture-expanded donor-derived bone marrow MSCs (BM-MSCs) into 35 children with severe aplastic anemia (SAA) undergoing haplo-HSCT. All 35 patients (100%) achieved hematopoietic reconstitution and showed sustained full donor chimerism...
October 19, 2018: Annals of Hematology
https://www.readbyqxmd.com/read/30339655/evaluating-the-efficacy-and-anti-infective-effect-of-high-dose-intravenous-immunoglobulin-adjuvant-therapy-for-acquired-aplastic-anemia-children
#2
Zhuli Bian, Ning Zhou, Xiaohong Qiao, Shuanglong Lu, Xiaoxiao Song, Huimin Zhou
BACKGROUND: The efficacy and anti-infective effect of high-dose intravenous immunoglobulin (HDIVIG) in severe or very severe aplastic anemia children were evaluated. PATIENTS AND METHODS: In total, 61 patients who underwent immunosuppressive therapy were retrospectively reviewed. The non-IVIG group (30 cases) received rabbit-antithymocyte protein (R-ATG, 3 to 5 mg/kg/d, for 5 consecutive days)+cyclosporin A (CSA), and the HDIVIG group (31 cases) underwent R-ATG+CSA+immunoglobulin (1 g/kg/d, for 2 consecutive days, once a month, 6 times)...
October 18, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/30334930/clinical-and-genetic-study-of-children-with-peutz-jeghers-syndrome-identifies-a-high-frequency-of-stk11-de-novo-mutation
#3
Hong-Mei Zhao, Yong-Jia Yang, Jia-Qi Duan, Hong-Juan Ouyang, Li Liu, Li-Chun Yi, Thomas M Attard, Ding-You Li, Jie-Yu You
OBJECTIVES: The present study aims to identify the genotype-phenotype correlation in children with Peutz-Jeghers Syndrome (PJS) through the analysis of STK11 gene mutations in the context of clinical and pathological characteristics. METHOD: In this observational cohort study, the clinical characteristics of 18 families diagnosed with pediatric PJS were collected. Genomic DNA from the peripheral blood of affected children and their family members was collected. The coding region of STK11 was amplified by PCR and screened for mutation by Sanger sequencing...
October 16, 2018: Journal of Pediatric Gastroenterology and Nutrition
https://www.readbyqxmd.com/read/30334902/serum-and-urinary-hepcidin-for-diagnosing-iron-deficiency-anemia-in-under-5-children
#4
Pooja Dewan, Anupriya Dixit, Sunil Gomber, Mrinalini Kotru, Basu D Banerjee, Vipin Tyagi, Rajeev K Malhotra
Bone marrow iron estimation remains the gold standard for diagnosing iron-deficiency anemia (IDA); serum ferritin, total iron-binding capacity, and transferrin saturation are routinely used as surrogate markers of IDA. However, these tests are marred by problems like poor specificity and sensitivity. Recently, hepcidin, a protein hormone synthesized in the liver and excreted in urine, has been shown to be related to iron status. We estimated the serum and urinary hepcidin levels in healthy children aged 6 to 60 months with (n=30) and without IDA (n=30)...
October 16, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/30328134/impact-of-t-cell-depletion-strategies-on-outcomes-following-hematopoietic-stem-cell-transplantation-for-idiopathic-aplastic-anemia-a-study-on-behalf-of-the-european-blood-and-marrow-transplant-ebmt-saa-working-party
#5
Sujith Samarasinghe, Katherine Clesham, Simona Iacobelli, Giulia Sbianchi, Cora Knol, Rose-Marie Hamladji, Gerard Socié, Mahmoud Aljurf, Mickey Koh, Henrik Sengeloev, Jean-Hugues Dalle, Stephen Robinson, Maria Teresa Van Lint, Constantijn Jm Halkes, Dietrich Beelen, Ghulam J Mufti, John Snowden, Didier Blaise, Regis Peffault de Latour, Judith Marsh, Carlo Dufour, Antonio M Risitano
We retrospectively analyzed the outcomes of 1837 adults and children with severe aplastic anemia (SAA) who underwent matched sibling donor (MSD) and matched unrelated donor (MUD) haemopoietic stem cell transplantation (HSCT) between 2000 and 2013. Patients were grouped by transplant conditioning containing either ATG (n=1283), alemtuzumab (n=261) or no serotherapy (NS) (n=293). The risks of chronic GvHD were significantly reduced when ATG or alemtuzumab were compared to no serotherapy (p=0.021 and p=0.003, respectively)...
October 17, 2018: American Journal of Hematology
https://www.readbyqxmd.com/read/30327152/when-technology-is-scarce-assessing-maternal-child-anemia-in-rural-india
#6
Jane Greene-Ryan
The World Health Organization estimates that in 2011, the last year for which we have global data, 800 million women and children are anemic. Within the Southeast Asia World Health Region, which includes India, the focus of this article, this accounts for nearly half of the maternal-child population. There are a number of underlying causes of anemia including heomoglobinopathies either inherited or acquired, macronutrient deficiencies, infections such as hookworm infestations, HIV and malaria or diseases such as cancer and malaria...
October 13, 2018: Journal of Pediatric Nursing
https://www.readbyqxmd.com/read/30324698/differential-cerebral-hemometabolic-responses-to-blood-transfusions-in-adults-and-children-with-sickle-cell-anemia
#7
Meher R Juttukonda, Chelsea A Lee, Niral J Patel, Larry T Davis, Spencer L Waddle, Melissa C Gindville, Sumit Pruthi, Adetola A Kassim, Michael R DeBaun, Manus J Donahue, Lori C Jordan
BACKGROUND: Blood transfusions are administered to children and adults with sickle cell anemia (SCA) for secondary stroke prevention, or as treatment for recurrent pain crises or acute anemia, but transfusion effects on cerebral hemodynamics and metabolism are not well-characterized. PURPOSE: To compare blood transfusion-induced changes in hemometabolic parameters, including oxygen extraction fraction (OEF) and cerebral blood flow (CBF), within and between adults and children with SCA...
October 15, 2018: Journal of Magnetic Resonance Imaging: JMRI
https://www.readbyqxmd.com/read/30314363/micronutrient-deficiencies-over-and-undernutrition-and-their-contribution-to-anemia-in-azerbaijani-preschool-children-and-non-pregnant-women-of-reproductive-age
#8
James P Wirth, Tamerlan Rajabov, Nicolai Petry, Bradley A Woodruff, Nafisa Binte Shafique, Rashed Mustafa, Vilma Qahoush Tyler, Fabian Rohner
Data on the nutritional situation and prevalence of micronutrient deficiencies in Azerbaijan are scarce, and knowledge about anemia risk factors is needed for national and regional policymakers. A nationally representative cross-sectional survey was conducted to assess the prevalence of micronutrient deficiencies, over- and undernutrition, and to disentangle determinants of anemia in children and women in Azerbaijan. The survey generated estimates of micronutrient deficiency and growth indicators for children aged 0⁻59 months of age (6⁻59 months for blood biomarkers) and non-pregnant women 15⁻49 years of age...
October 11, 2018: Nutrients
https://www.readbyqxmd.com/read/30311150/ultra-short-celiac-disease-is-a-distinct-and-milder-phenotype-of-the-disease-in-children
#9
Reut Doyev, Shlomi Cohen, Amir Ben-Tov, Yael Weintraub, Achiya Amir, Tut Galai, Hadar Moran-Lev, Anat Yerushalmy-Feler
BACKGROUND AND AIMS: Approximately 10% of children with celiac disease (CD) have ultra-short celiac disease (USCD), where histological abnormalities are limited to the duodenal bulb. The aim of our retrospective study was to identify clinical and serological characteristics at baseline and at follow-up of children with USCD. METHODS: All children that were diagnosed with CD in our unit during 7/2010-12/2017, in whom biopsies were taken from duodenal bulb and second part, were included...
October 11, 2018: Digestive Diseases and Sciences
https://www.readbyqxmd.com/read/30309846/cerebral-blood-flow-and-marrow-diffusion-alterations-in-children-with-sickle-cell-anemia-after-bone-marrow-transplantation-and-transfusion
#10
M T Whitehead, A Smitthimedhin, J Webb, E S Mahdi, Z P Khademian, J L Carpenter, A Abraham
BACKGROUND AND PURPOSE: Hematopoietic marrow hyperplasia and hyperperfusion are compensatory mechanisms in sickle cell anemia. We have observed marrow diffusion and arterial spin-labeling perfusion changes in sickle cell anemia following bone marrow transplantation. We aimed to compare arterial spin-labeling perfusion and marrow diffusion/ADC values in patients with sickle cell anemia before and after bone marrow transplantation or transfusion. MATERIALS AND METHODS: We reviewed brain MRIs from patients with sickle cell anemia obtained during 6 consecutive years at a children's hospital...
October 11, 2018: AJNR. American Journal of Neuroradiology
https://www.readbyqxmd.com/read/30309337/differentials-in-prevalence-and-correlates-of-metabolic-risk-factors-of-non-communicable-diseases-among-women-in-sub-saharan-africa-evidence-from-33-countries
#11
Sanni Yaya, Michael Ekholuenetale, Ghose Bishwajit
BACKGROUND: Even with the widespread recognition of non- communicable diseases (NCDs) in sub-Saharan Africa region, yet, sufficient evidence-based surveillance systems to confirm the prevalence and correlates of these diseases is lacking. In an attempt to understand the problem of NCDs in resource-constrained settings, this study was conducted to establish the pattern of the risk factors of NCDs in sub-Sahara Africa region. METHODS: The current Demographic and Health Survey (DHS) data sets from 33 countries in sub-Sahara Africa region were used in this study...
October 11, 2018: BMC Public Health
https://www.readbyqxmd.com/read/30307299/parasitic-infections-malnutrition-and-anemia-among-preschool-children-living-in-rural-areas-of-peshawar-pakistan
#12
Zia Ud Din, Lubna Pervez, Arshad Amir, Muhammad Abbas, Imran Khan, Zafar Iqbal, Mudassar Iqbal
INTRODUCTION: it has previously been shown that parasitic infections (PI) have deleterious effects on the nutritional status of the host, particularly among young children. OBJECTIVE: the objective of this study was to estimate the severity of the problem of malnutrition and anemia in association with PI in preschool children and to identify the possible risk factors that contribute to these health problems. METHODS: four hundred and thirty-seven mother-child pairs were randomly selected from rural areas of Peshawar, Pakistan...
October 5, 2018: Nutrición Hospitalaria: Organo Oficial de la Sociedad Española de Nutrición Parenteral y Enteral
https://www.readbyqxmd.com/read/30305538/-diagnosis-surveillance-and-management-of-familial-leukemia
#13
Hiroshi Moritake
Recently, the modern technique of comprehensive genomic analysis has identified both somatic mutations originating from tumor cells and germline mutations as causative genes of inherited familial leukemias among which Fanconi anemia and Li-Fraumeni syndrome are well known. Pathogenic germline mutations occur in various pathways, affecting DNA repair, ribosome biogenesis, telomere biology, hematopoietic transcription factors, tumor suppressors, neutrophil development, and other critical cellular processes. The clinical manifestations of germline mutations present a wide phenotypic spectrum of patients displaying congenital anomalies, early-onset myelodysplastic syndrome, or no medical problems until the developing leukemia...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/30304524/homozygosity-for-a-mutation-affecting-the-catalytic-domain-of-tyrosyl-trna-synthetase-yars-causes-multisystem-disease
#14
Katie B Williams, Karlla W Brigatti, Erik G Puffenberger, Claudia Gonzaga-Jauregui, Laurie B Griffin, Erick D Martinez, Olivia K Wenger, Mark Yoder, Vinay V R Kandula, Michael D Fox, Matthew M Demczko, Laura Poskitt, Katryn N Furuya, Jeffrey G Reid, John D Overton, Aris Baras, Lili Miles, Kadakkal Radhakrishnan, Vincent J Carson, Anthony Antonellis, Robert N Jinks, Kevin A Strauss
Aminoacyl-tRNA synthetases (ARSs) are critical for protein translation. Pathogenic variants of ARSs have been previously associated with peripheral neuropathy and multisystem disease in heterozygotes and homozygotes, respectively. We report seven related children homozygous for a novel mutation in tyrosyl-tRNA synthetase (YARS, c.499C > A, p.Pro167Thr) identified by whole exome sequencing. This variant lies within a highly-conserved interface required for protein homodimerization, an essential step in YARS catalytic function...
October 9, 2018: Human Molecular Genetics
https://www.readbyqxmd.com/read/30300449/deferasirox-in-children-with-transfusion-dependent-thalassemia-or-sickle-cell-anemia-a-large-cohort-real-life-experience-from-turkey-reach-them
#15
Bulent Antmen, Zeynep Karakaş, Mehmet Akif Yeşilipek, Osman Alphan Küpesiz, İlgen Şaşmaz, Vedat Uygun, Erdal Kurtoğlu, Gonul Oktay, Gonul Aydogan, Mehmet Akın, Zafer Salcioglu, Canan Vergin, Elif Güler Kazancı, Selma Ünal, Ümran Çalışkan, Yusuf Ziya Aral, Emine Türkkan, Adalet Meral Güneş, Bahattin Tunç, Fatma Gümrük, Aylin Canbolat Ayhan, Murat Söker, Ahmet Koç, Yeşim Oymak, Mehmet Ertem, Çetin Timur, Yıldız Yıldırmak, Gülersu İrken, Hilmi Apak, Betül Biner, Tuğba Gürleyen Eren, Yasemin Işık Balcı, Ülker Koçak, Gülsün Karasu, Diyar Akkaynak, Türkan Patıroğlu
OBJECTIVES: To evaluate the long-term efficacy and safety of deferasirox therapy in a large observational cohort of children with transfusion-dependent thalassemia (TDT) and sickle cell anemia (SCA) in Turkey. METHODS: This was a multicenter, prospective cohort study including TDT and SCA patients aged 2-18 years with iron overload (≥100 mL/kg of pRBC or a serum ferritin [SF] level >1000 μg/L) receiving deferasirox. Patients were followed for up to 3 years according to standard practice...
October 9, 2018: European Journal of Haematology
https://www.readbyqxmd.com/read/30298806/-hymenolepis-nana-an-emerging-intestinal-parasite-associated-with-anemia-in-school-children-from-the-bolivian-chaco
#16
Michele Spinicci, Fabio Macchioni, Simona Gabrielli, David Rojo, Herlan Gamboa, Ana Liz Villagrán, Yolanda Vallejos, Marianne Strohmeyer, Mimmo Roselli, Gabriella Cancrini, Piero Olliaro, Antonio Montresor, Alessandro Bartoloni
Tropical anemia can have multiple causes, whether socioeconomic, dietary, or infectious. In the Bolivian Chaco, soil-transmitted helminthiases (STH), malaria, and Chagas disease are potential infectious causes of anemia among school-aged children (SAC). Following years of preventive chemotherapy with mebendazole, the prevalence of STH among SAC living in that area is now negligible, whereas protozoan infections are still highly prevalent (81%); Hymenolepis nana is the most frequent intestinal helminth (∼13%)...
October 8, 2018: American Journal of Tropical Medicine and Hygiene
https://www.readbyqxmd.com/read/30298802/soil-transmitted-helminth-infections-and-anemia-in-schoolchildren-from-corn-island-archipelago-raas-nicaragua
#17
Carla Muñoz-Antoli, Paloma Pérez, Aleyda Pavón, Rafael Toledo, Jose-Guillermo Esteban
The prevalence and intensity of soil-transmitted helminth (STH) infections in schoolchildren from Corn Islands (Nicaragua) were examined to detect mono- or poly-STH infected children, measuring different intensity levels, and to elucidate measurably increased odds of being anemic. A total of 341 stool samples provided by 2- to 15-year-old children were examined using a concentration technique and a Kato-Katz slide. Intensity of infection was expressed as eggs per gram (epg) of feces to classify light, moderate, or heavy intensity infection...
October 8, 2018: American Journal of Tropical Medicine and Hygiene
https://www.readbyqxmd.com/read/30294530/the-rescue-effect-of-adalimumab-in-the-treatment-of-refractory-pediatric-panuveitis-complicated-with-steroid-induced-glaucoma
#18
Wei-Ning Ku, Chun-Ju Lin, Yi-Yu Tsai
We report the rescue effects of adalimumab (HUMIRA® ) therapy in a case of refractory pediatric panuveitis complicated with steroid-induced glaucoma. A 13-year-old girl had suffered from blurred vision since January 2013. Bilateral idiopathic panuveitis with macular edema (ME) was diagnosed. She had previously been treated with systemic steroids, methotrexate, cyclosporine, and mycophenolate. All treatments had failed to suppress the ocular inflammation and ME persisted. Intravitreal dexamethasone implant injections were given to both eyes and ME subsided; however, complications with steroid-induced glaucoma and cataract occurred...
July 2018: Taiwan Journal of Ophthalmology
https://www.readbyqxmd.com/read/30293279/-interleukin-10-receptor-gene-mutations-induced-very-early-onset-inflammatory-bowel-disease-in-6-infants
#19
F P Wang, X Ma, D Zhu, Y Z Gong, H J Ning, X M Zhong
Objective: To analyze the clinical features and interleukin-10 receptor gene mutations in six infants with very early onset inflammatory bowel disease (VEO-IBD). Methods: Four girls and two boys with VEO-IBD admitted to Children's Hospital Affiliated to Capital Institute of Pediatrics from June 2016 to September 2017 were reviewed. The clinical data including general condition, clinical symptoms, laboratory tests, and colonoscopy and pathological results were collected and analyzed. Interleukin-10 receptor α subunit (IL-10RA) gene was examined in all patients...
October 2, 2018: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
https://www.readbyqxmd.com/read/30290004/point-of-care-screening-for-sickle-cell-disease-in-low-resource-settings-a-multi-center-evaluation-of-hemotypesc-a-novel-rapid-test
#20
Cindy Steele, Annette Sinski, Jacqueline Asibey, Marie-Dominique Hardy-Dessources, Gisèle Elana, Colleen Brennan, Isaac Odame, Carolyn Hoppe, Mark Geisberg, Erik Serrao, Charles T Quinn
Sickle cell disease is a common, life-threatening genetic disorder that is best managed when diagnosed early by newborn screening. However, sickle cell disease is most prevalent in low-resource regions of the world where newborn screening is rare and diagnosis at the point-of-care is challenging. In many such regions, the majority of affected children die, undiagnosed, before the age of five years. A rapid and affordable point-of-care test for sickle cell disease is needed. The diagnostic accuracy of HemoTypeSC, a point-of-care immunoassay, for sickle cell disease was evaluated in individuals who had sickle cell disease, hemoglobin C disease, the related carrier (trait) states, or a normal hemoglobin phenotype...
October 5, 2018: American Journal of Hematology
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