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Aplastic anemia

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https://www.readbyqxmd.com/read/29467851/proteinase-3-expression-on-the-neutrophils-of-patients-with-paroxysmal-nocturnal-hemoglobinuria
#1
Hui Liu, Yi Liu, Yi Li, Zhaoyun Liu, Liyan Li, Shaoxue Ding, Yihao Wang, Tian Zhang, Lijuan Li, Zonghong Shao, Rong Fu
Proteinase 3 (PR3) is released from neutrophils and regulates platelet activity, which is associated with cluster of differentiation (CD)177 antigen (NB1), a glycosylphosphatidylinositol-linked protein. In the present study, the effect of PR3 on thrombosis in paroxysmal nocturnal hemoglobinuria (PNH) and PNH-aplastic anemia (AA) syndrome was explored. The expression of PR3 and NB1 on CD59- neutrophils was detected by flow cytometry, immunofluorescence (IF), reverse transcription-quantitative polymerase chain reaction analysis and western blotting...
March 2018: Experimental and Therapeutic Medicine
https://www.readbyqxmd.com/read/29459495/aplastic-anemia-and-risk-of-incident-atrial-fibrillation-a-nationwide-cohort-study
#2
Wei-Syun Hu, Fung-Chang Sung, Cheng-Li Lin
BACKGROUND: This retrospective cohort study sought to follow up patients with aplastic anemia (AA) to evaluate their risk of developing atrial fibrillation (AF).Methods and Results:From the National Health Insurance Research Database of Taiwan, this study identified an AA cohort (n=3,921), a general population cohort (n=17,617,843) and a propensity score-matched none AA cohort (PSM non-AA cohort in brief, n=15,684) in 2000-2010. By the end of 2011, the incident AF was higher in the AA cohort than in the general population and PSM non-AA cohorts (8...
February 16, 2018: Circulation Journal: Official Journal of the Japanese Circulation Society
https://www.readbyqxmd.com/read/29434791/efficacy-and-safety-of-combined-immunosuppressive-therapy-plus-umbilical-cord-blood-infusion-in-severe-aplastic-anemia-patients-a-cohort-study
#3
Xiu Luo, Huina Lu, Bing Xiu, Hao Wu, Bing Li, Ping Li, Yuhua Chen, Lili Zhou, Wenjun Zhang, Yan Dong, Aibin Liang, Yi Ding
The present study aimed to evaluate the efficacy and safety of combined immunosuppressive therapy (IST) plus umbilical cord blood infusion (UCBI) in severe aplastic anemia (SAA) patients. A total of 68 patients with SAA were enrolled in the current prospective cohort study and divided into the IST (n=35; positive control) and IST+UCBI (n=33; experimental) groups according to the treatment conditions. Patients in the IST group were treated with rabbit antithymocyte globulin (r-ATG) at a dose of 2.5 mg/kg through intravenous infusion once a day for five days...
February 2018: Experimental and Therapeutic Medicine
https://www.readbyqxmd.com/read/29434729/a-composite-mouse-model-of-aplastic-anemia-complicated-with-iron-overload
#4
Dijiong Wu, Xiaowen Wen, Wenbin Liu, Linlong Xu, Baodong Ye, Yuhong Zhou
Iron overload is commonly encountered during the course of aplastic anemia (AA), but no composite animal model has been developed yet, which hinders drug research. In the present study, the optimal dosage and duration of intraperitoneal iron dextran injection for the development of an iron overload model in mice were explored. A composite model of AA was successfully established on the principle of immune-mediated bone marrow failure. Liver volume, peripheral hemogram, bone marrow pathology, serum iron, serum ferritin, pathological iron deposition in multiple organs (liver, bone marrow, spleen), liver hepcidin, and bone morphogenetic protein 6 (BMP6), SMAD family member 4 (SMAD4) and transferrin receptor 2 (TfR2) mRNA expression levels were compared among the normal control, AA, iron overload and composite model groups to validate the composite model, and explore the pathogenesis and features of iron overload in this model...
February 2018: Experimental and Therapeutic Medicine
https://www.readbyqxmd.com/read/29432302/allogeneic-matched-related-donor-bone-marrow-transplantation-for-pediatric-patients-with-severe-aplastic-anemia-using-low-dose-cyclophosphamide-atg-plus-fludarabine
#5
Chayamon Takpradit, Susan E Prockop, Nancy A Kernan, Andromachi Scaradavou, Kevin Curran, Julianne Ruggiero, Nicole Zakak, Richard J O'Reilly, Farid Boulad
BACKGROUND: The combination of cyclophosphamide (CY) and antithymocyte globulin (ATG) has been used as a standard conditioning regimen for matched related donor transplantation in patients with severe aplastic anemia. PROCEDURE: To decrease the regimen-related toxicity while maintaining appropriate engraftment and survival rates, fludarabine (FLU) was added to the regimen. Four pediatric patients received matched related donor bone marrow transplantation with CY (50 mg/kg×2) (instead of the 50 mg/kg×4 standard dosing), equine ATG (30 mg/kg×3), with the addition of FLU (30 mg/m×4)...
February 9, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29419434/deep-sequencing-and-flow-cytometric-characterization-of-expanded-effector-memory-cd8-cd57-t-cells-frequently-reveals-t-cell-receptor-v%C3%AE-oligoclonality-and-cdr3-homology-in-acquired-aplastic-anemia
#6
Valentina Giudice, Xingmin Feng, Zenghua Lin, Wei Hu, Fanmao Zhang, Wangmin Qiao, Maria Del Pilar Fernandez Ibanez, Olga Rios, Neal S Young
Oligoclonal expansion of CD8+CD28- lymphocytes has been considered indirect evidence for a pathogenic immune response in acquired aplastic anemia. A subset of CD8+CD28- cells with CD57 expression termed effector memory cells is expanded in several immune mediated diseases and may have a role in immune surveillance. We hypothesized that effector memory CD8+CD28-CD57+ cells may drive aberrant oligoclonal expansion in aplastic anemia. We found CD8+CD57+ cells frequently expanded in the blood of aplastic anemia patients, with oligoclonal characteristics by flow cytometric Vβ usage analysis: skewing in 1 to 5 Vβ families and frequencies of immunodominant clones ranging from 1...
February 1, 2018: Haematologica
https://www.readbyqxmd.com/read/29416752/fanconi-anemia-germline-variants-as-susceptibility-factors-in-aplastic-anemia-mds-and-aml
#7
Bartlomiej Przychodzen, Hideki Makishima, Mikkael A Sekeres, Suresh Kumar Balasubramanian, Swapna Thota, Bhumika J Patel, Michael Clemente, Cassandra Hirsch, Brittney Dienes, Jaroslaw P Maciejewski
Using next generation sequencing we have systematically analyzed a large cohort of 489 patients with bone marrow failure (BMF), including myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), aplastic anemia (AA), and related conditions for the presence of germline (GL) alterations in Fanconi Anemia (FA) and telomerase genes. We have detected an increased frequency of heterozygous FA gene mutations in MDS and to lesser degree in AML suggesting that the presence of one normal allele may not be completely protective and indeed heterozygous FA lesions may have a long latency period before hematologic manifestation...
January 5, 2018: Oncotarget
https://www.readbyqxmd.com/read/29415940/-reduced-intensity-umbilical-cord-blood-transplantation-for-adult-patients-with-fulminant-aplastic-anemia
#8
Kaichi Nishiwaki, Koji Sano, Yutaro Kamiyama, Kazumi Hayashi, Susumu Tanoue, Mituji Katori, Hidekazu Masuoka, Keisuke Aiba
Allogeneic hematopoietic stem cell transplantation is recommended as a curative treatment option for fulminant aplastic anemia with no neutrophil despite the administration of granulocyte-colony stimulating factor. In the absence of an HLA-matched donor, unrelated cord blood transplantation (UCBT) is a treatment option that can be performed quickly. However, the optimal conditioning regimen of UCBT is yet to be established. We report two cases of fulminant aplastic anemia in adult patients who received UCBT...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/29415633/danazol-increases-t-regulatory-cells-in-patients-with-aplastic-anemia
#9
Harshit Khurana, Pankaj Malhotra, Man Updesh Sachdeva, Neelam Varma, Parveen Bose, Uday Yanamandra, Subhash Varma, Alka Khadwal, Deepesh Lad, Gaurav Prakash
OBJECTIVES: Danazol is an attenuated androgen and is used in the treatment of aplastic anemia (AA) in resource constraint settings. We chose to study the role of CD4+ CD25high CD127low FoxP3+ T regulatory cells (T-regs) in the pathophysiology of AA and their response to treatment with Danazol alone or in combination with immunosuppressive treatment (IST). METHODS: T-regs' percentages of 25 acquired idiopathic AA patients and 25 healthy controls who completed study protocol were analyzed by performing multicolor flowcytometry on peripheral blood samples...
February 8, 2018: Hematology (Amsterdam, Netherlands)
https://www.readbyqxmd.com/read/29410500/optimal-donor-for-severe-aplastic-anemia-patient-requiring-allogeneic-hematopoietic-stem-cell-transplantation-a-large-sample-study-from-china
#10
Yunjing Zeng, Sanbin Wang, Jishi Wang, Li Liu, Yi Su, Zhixiang Lu, Xuemei Zhang, Yanqi Zhang, Jiang Fan Zhong, Lihui Peng, Qiang Liu, Yinghao Lu, Lei Gao, Xi Zhang
HLA-haploidentical hematopoietic stem cell transplantation (HSCT) may be an option for severe aplastic anemia (SAA) patients. However, to date, no large-sample studies have been performed to determine which types of SAA patients are suitable for HLA-haploidentical HSCT. We retrospectively studied 189 consecutive patients with SAA who underwent HLA-identical or HLA-haploidentical HSCT at seven transplant centers in China. Propensity score matching (PSM) was applied in this study to reduce the influence of potential confounders...
February 6, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29410341/evaluation-of-a-machine-learning-based-prognostic-model-for-unrelated-hematopoietic-cell-transplantation-donor-selection
#11
Ljubomir Buturovic, Jason Shelton, Stephen R Spellman, Tao Wang, Lyssa Friedman, David Loftus, Lyndal Hesterberg, Todd Woodring, Katharina Fleischhauer, Katharine C Hsu, Michael R Verneris, Mike Haagenson, Stephanie J Lee
Survival of patients undergoing hematopoietic cell transplantation (HCT) from unrelated donors for acute leukemia exhibits considerable variation, even after stringent genetic matching. In order to improve the donor selection process, we attempted to create an algorithm to quantify the likelihood of survival to five years after unrelated donor HCT for acute leukemia, based on the clinical characteristics of the donor selected. All standard clinical variables were included in the model, which also included average leukocyte telomere length (ATL) of the donor based on its association with recipient survival in severe aplastic anemia, and links to multiple malignancies...
February 1, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29409729/acquired-amegakaryocytic-thrombocytopenia-and-red-cell-aplasia-in-a-patient-with-thymoma-progressing-to-aplastic-anemia-successfully-treated-with-allogenic-stem-cell-transplantation
#12
Aron Simkins, Abhishek Maiti, Nicholas J Short, Nitin Jain, Uday Popat, Keyur P Patel, Thein H Oo
Association of pure red-cell aplasia with thymoma is well documented. However, acquired amegakaryocytic thrombocytopenia (AAMT) has been rarely associated with thymoma with only five reported cases in literature. We report a patient with thymoma complicated by pure red cell aplasia (PRCA) and AAMT who progressed to develop aplastic anemia (AA). The patient was refractory to 10-months of immunosuppressive therapy with cyclosporine, prednisone, and antithymocyte globulin. She was eventually treated with allogeneic stem cell transplantation (allo-SCT)...
January 31, 2018: Hematology/oncology and Stem Cell Therapy
https://www.readbyqxmd.com/read/29404507/sex-specific-analysis-post-liver-transplantation-in-hemochromatosis-with-aplastic-anemia-and-hepatocellular-carcinoma
#13
Paul Adams, Christopher Howlett, Anargyros Xenocostas, Subrata Chakrabarti
A 42-year-old man with hemochromatosis and cirrhosis developed aplastic anemia. He underwent liver transplantation from a female donor and splenectomy, and his aplastic anemia spontaneously resolved. A bone marrow examination 6 months after the liver transplant showed 17.5% female cells. He did well for 13 years without the need for any blood product support but then developed bone pain and was found to have metastatic hepatocellular carcinoma in the vertebral bodies. Molecular analysis demonstrated that the tumor cells were from his original liver...
January 2018: Hepatology Communications
https://www.readbyqxmd.com/read/29397846/-differential-expression-profiles-of-micrornas-between-de-novo-and-complete-response-severe-aplastic-anemia
#14
Ying-Qi Shao, Hong-Yu Dong, Mei-Li Ge, Feng Zhang, Jun Shi, Xing-Xin Li, Jin-Bo Huang, Zhen-Dong Huang, Jing Zhang, Neng Nie, Yi-Zhou Zheng
OBJECTIVE: To detect the expression of miRNA in de novo and complete response SAA patients and predict the targets of the miRNAs. METHODS: The expression profiles of miRNA from bone marrow mononuclear cells of the SAA patients with de novo and CR were detected by miRNA microarray. RESULTS: Totally 35 up-regulated and 37 down-regulated miRNA were identified in CR SAA patients in comparison with de novo SAA patients. Furthermore, by predicting the targets of the differentlly expressed miRNA, it was found that some targets associated with T cell receptor signaling pathway and cell adhesion molecules...
February 2018: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/29395255/lessons-learned-from-bone-marrow-failure-in-systemic-lupus-erythematosus-case-reports-and-review-of-the-literature
#15
Erik Anderson, Bhakti Shah, Anne Davidson, Richard Furie
OBJECTIVE: In the present review, four new cases of bone marrow failure are presented and the potential contribution of systemic lupus erythematosus (SLE) is discussed. Furthermore, a comprehensive literature review of cases of autoimmune myelofibrosis (AIMF), aplastic anemia (AA), and paroxysmal nocturnal hemoglobinuria (PNH) with concurrent SLE aims to allow their direct comparison. Based on a clearer characterization of reported cases and our own experience, diagnostic and therapeutic strategies of these disorders in SLE are proposed based on lessons learned from the present and previous cases...
December 8, 2017: Seminars in Arthritis and Rheumatism
https://www.readbyqxmd.com/read/29390381/mesalazine-as-a-cause-of-fetal-anemia-and-hydrops-fetalis-a-case-report
#16
Sverker Ek, Staffan Rosenborg
RATIONALE: Mesalazine and its prodrug sulfasalazine are both used for inflammatory bowel disease. Sulfasalazine has been associated with hematological side-effects such as aplastic and hemolytic anemia in patients, but also in fetuses after intrauterine exposure. To our knowledge, we describe the first case of a fetus with severe anemia, and subsequent hydrops, where this drug was found at concentrations in the fetus corresponding to those in the mother and most likely responsible for the fetal condition...
December 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29382003/hemoglobin-hornchurch-%C3%AE-43-cd2-glu-lys-hbb-c-130g-a-in-a-chinese-boy-complicated-with-thrombocytopenia-a-case-report-and-literature-review
#17
Huiping Shi, Zhaoyue Wang
RATIONALE: Hemoglobin Hornchurch is regarded as an asymptomatic hemoglobinopathy with no obvious hematological or clinical abnormalities. Recently, we identified hemoglobin Hornchurch in a 13-year-old Chinese boy complicated with thrombocytopenia, which displayed instability in isopropanol precipitation test. PATIENT CONCERNS: In this case report, we reported a Chinese boy with hemoglobin Hornchurch complicated by thrombocytopenia. The patients have been misdiagnosed as aplastic anemia and myelodysplastic syndrome before...
November 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29381836/skin-induration-in-a-patient-with-aplastic-anemia
#18
Liudmila Kastsianok, Prabha Ranganathan
One month prior to rheumatology evaluation he was admitted to the hospital for progressive worsening of shortness of breath on exertion, epistaxis, and gingival bleeding. He had fatigue, poor appetite, and an unintentional weight loss of about 30 pounds over the preceding two years. He denied symptoms of chest pain, orthopnea, paroxysmal nocturnal dyspnea, or leg swelling. This article is protected by copyright. All rights reserved.
January 30, 2018: Arthritis Care & Research
https://www.readbyqxmd.com/read/29377260/nationwide-survey-on-the-use-of-horse-antithymocyte-globulins-atgam-in-patients-with-acquired-aplastic-anemia-a-report-on-behalf-of-the-french-reference-center-for-aplastic-anemia
#19
Régis Peffault de Latour, Reza Tabrizi, Ambroise Marcais, Thierry Leblanc, Thierry Lamy, Mohamad Mohty, Suzanne Tavitian, Charlotte Jubert, Marlène Pasquet, Claire Galambrun, Stéphanie Nguyen, Jean Yves Cahn, Thorsten Braun, Eric Deconinck, Jacques Olivier Bay, Flore Sicre de Fontbrune, Fiorenza Barraco, Gérard Socié
Antithymocyte globulins (ATG) plus cyclosporine (CSA) is the gold standard immunosuppressive treatment (IST) for patients with aplastic anemia. A prospective randomized trial showed in 2011 that hATG was superior to rabbit ATG for first-line treatment of severe AA. The French Health Agency (ANSM) permitted a patient-named Authorization for Temporary Use (ATU) program of hATG (ATGAM, Pfizer) in patients with AA in 2011 since commercial access to hATG is not approved. We took advantage of this program to analyze the outcomes of 465 patients who received ATGAM plus CSA as first line treatment (n=379; 81...
January 27, 2018: American Journal of Hematology
https://www.readbyqxmd.com/read/29374141/ipsc-modeling-of-severe-aplastic-anemia-reveals-impaired-differentiation-and-telomere-shortening-in-blood-progenitors
#20
Dario Melguizo-Sanchis, Yaobo Xu, Dheraj Taheem, Min Yu, Katarzyna Tilgner, Tomas Barta, Katja Gassner, George Anyfantis, Tengfei Wan, Ramu Elango, Sameer Alharthi, Ashraf A El-Harouni, Stefan Przyborski, Soheir Adam, Gabriele Saretzki, Sujith Samarasinghe, Lyle Armstrong, Majlinda Lako
Aplastic Anemia (AA) is a bone marrow failure (BMF) disorder, resulting in bone marrow hypocellularity and peripheral pancytopenia. Severe aplastic anemia (SAA) is a subset of AA defined by a more severe phenotype. Although the immunological nature of SAA pathogenesis is widely accepted, there is an increasing recognition of the role of dysfunctional hematopoietic stem cells in the disease phenotype. While pediatric SAA can be attributable to genetic causes, evidence is evolving on previously unrecognized genetic etiologies in a proportion of adults with SAA...
January 26, 2018: Cell Death & Disease
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