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Severe aplastic anemia

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https://www.readbyqxmd.com/read/28078653/meta-analysis-of-treatment-with-rabbit-and-horse-antithymocyte-globulin-for-aplastic-anemia
#1
Jin Hayakawa, Junya Kanda, Yu Akahoshi, Naonori Harada, Kazuaki Kameda, Tomotaka Ugai, Hidenori Wada, Yuko Ishihara, Koji Kawamura, Kana Sakamoto, Masahiro Ashizawa, Miki Sato, Kiriko Terasako-Saito, Shun-Ichi Kimura, Misato Kikuchi, Rie Yamazaki, Shinichi Kako, Yoshinobu Kanda
Aplastic anemia patients who received rabbit antithymocyte globulin exhibited response and survival rates inferior to those who received horse antithymocyte globulin in several studies. Therefore, we conducted a meta-analysis to compare rabbit and horse antithymocyte globulin as immunosuppressive therapy for aplastic anemia. We searched online databases for studies that compared antithymocyte globulin regimens as first-line treatment for aplastic anemia, including both randomized and non-randomized controlled trials...
January 11, 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/28067873/cotransplantation-of-bone-marrow-derived-mesenchymal-stem-cells-in-haploidentical-hematopoietic-stem-cell-transplantation-in-patients-with-severe-aplastic-anemia-an-interim-summary-for-a-multicenter-phase-ii-trial-results
#2
Z Liu, Y Zhang, H Xiao, Z Yao, H Zhang, Q Liu, B Wu, D Nie, Y Li, Y Pang, Z Fan, L Li, Z Jiang, F Duan, H Li, P Zhang, Y Gao, L Ouyang, C Yue, M Xie, C Shi, Y Xiao, S Wang
Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for severe aplastic anemia (SAA) is mainly limited by the high incidence of graft failure and GvHD. Mesenchymal stem cells (MSCs) have been shown to support hematopoiesis in vivo and to display potent immunosuppressive effects to prevent or treat GvHD after HSCT. In a multicenter phase II trial, we developed an approach with co-transplantation of MSCs in patients undergoing haplo-HSCT. Forty-four patients with SAA were included. The conditioning regimen included busulfan, cyclophosphamide and thymoglobulin (ATG)...
January 9, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28060106/interleukin-6-and-interleukin-8-levels-correlate-with-the-severity-of-aplastic-anemia-in-children
#3
Vineeta Gupta, Sushil Kumar, Rimjhim Sonowal, Surya K Singh
AIM: The aim of this study was to evaluate the levels of interleukin (IL)-6 and IL-8 in patients with aplastic anemia and its correlation with severity of the disease. MATERIALS AND METHODS: IL-6 and IL-8 levels were measured in 40 patients with aplastic anemia in the age group of 4 to 14 years. A total of 40 healthy children served as controls. Quantitative estimation of IL-6 and IL-8 was performed using a solid-phase sandwich ELISA kit. Results were presented as IL-6 and IL-8 concentrations in pg/mL...
January 5, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28030425/features-of-hepatitis-in-hepatitis-associated-aplastic-anemia-clinical-and-histopathologic-study
#4
Kalyani R Patel, Alison Bertuch, Ghadir S Sasa, Ryan W Himes, Hao Wu
OBJECTIVES: Hepatitis-associated aplastic anemia (HAA) is a rare variant of aplastic anemia in which patients present with severe pancytopenia after an episode of acute hepatitis. The marrow failure is often rapid, severe, and usually fatal if untreated. The preceding hepatitis is largely under-studied. METHODS: Retrospective study of the clinical and histopathologic features of hepatitis in pediatric patients who subsequently developed aplastic anemia and comparison with consecutive cases of acute liver failure and random cases of autoimmune hepatitis during the same time frame...
January 2017: Journal of Pediatric Gastroenterology and Nutrition
https://www.readbyqxmd.com/read/28024501/-effect-of-rabbit-anti-human-thymocyte-immunoglobulin-combined-with-cyclosporin-a-on-severe-aplastic-anemia
#5
Wei Luo, Lin-Hua Ji, Hui Geng, Xiao-Jing Ma, Hua Xiong, Qi-Chao Yin, Jie Ma
OBJECTIVE: To study the efficacy and safety of anti-human thymocyte immunoglobulin(ATG-F) combined with cyclosporin A(CsA) on patients with severe aplastic anemia (SSA), so as to provide support for clinical work. METHODS: From January 2010 to December 2015, 78 patients with SAA admitted in our hospital were divided into 2 groups: ATG-F+CsA group(40 cases) and ATG-F group(38 cases). After treatment for 6 months, the effective rate, side reaction rate and time of effect initiation were compared between 2 groups...
December 2016: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/28024500/-hematopoietic-reconstitution-and-prognosis-of-hla-matched-and-haploidentical-hematopoietic-stem-cell-transplantation-using-modified-fc-atg-conditioning-for-treatment-of-severe-aplastic-anemia
#6
Li Wang, Ya-Mei Wu, Yong-Bin Cao, Xiao-Hong Li, Li-Xin Xu, Hai-Tao Wang, Ya-Hui Gao, Xiao-Xiong Wu
OBJECTIVE: To analyse the feasibility and compare differences between hematopoietic reconstitution and prognosis of patients with severe aplastic anemia(SAA) after matched sibling donor (MSD) or haploidentical family donor (HFD) hematopoietic stem cell transplantation (HSCT) using the modified FC/ATG conditioning. METHODS: The clinical data of 56 patients with SAA who received HSCT in First Affiliated Hospital of Chinese PLA General Hospital from January 2011 to June 2016 were analyzed retrospectively...
December 2016: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/28024499/-clinical-efficacy-of-haploidentical-allo-hsct-of-reduced-intensity-preconditioning-combined-with-induced-immune-tolerance-after-transplantation-for-severe-aplastic-anemia
#7
Zhi Guo, Hui-Ren Chen, Xiao-Dong Liu, Kai Yang, Jing-Xing Lou, Yuan Zhang, Peng Chen, Xue-Peng He
OBJECTIVE: To investigate the efficacy and safety of haploidentical allo-HSCT in combination of reduced intensity preconditioning combined with cyclophosphamid (CTX)-induced immune tolerance after transplanitation for treatment of severe aplastic anemia (SAA). METHODS: A total of 15 patients with SAA received the haploidentical allo-HSCT of reduced intensity preconditioning combined with CTX-induced immune tolerance after transplartation in the General hospital of Beijing military command of chinese PLA from June 2012 to December 2014...
December 2016: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/28013015/alternative-donor-transplantation-with-high-dose-post-transplantation-cyclophosphamide-for-refractory-severe-aplastic-anemia
#8
Amy E DeZern, Marianna Zahurak, Heather Symons, Kenneth Cooke, Richard J Jones, Robert A Brodsky
Severe aplastic anemia (SAA) is a life-threatening hematopoietic stem cell disorder that is treated with bone marrow transplantation (BMT) or immunosuppressive therapy (IST). The management of patients with refractory SAA after IST is a major challenge. Alternative donor BMT is best chance for cure in refractory SAA, but morbidity and mortality from graft failure and complications of graft-versus-host disease (GVHD) have limited enthusiasm for this approach. Here, we employed post-transplantation high-dose cyclophosphamide in an effort to safely expand the donor pool in 16 consecutive patients with refractory SAA who did not have a matched sibling donor...
December 21, 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28008152/dysregulated-mir34a-diacylglycerol-kinase-%C3%AE-interaction-enhances-t-cell-activation-in-acquired-aplastic-anemia
#9
Yuan-Xin Sun, Hui Li, Qi Feng, Xin Li, Ying-Yi Yu, Li-Wei Zhou, Yan Gao, Guo-Sheng Li, Juan Ren, Chun-Hong Ma, Cheng-Jiang Gao, Jun Peng
Acquired aplastic anemia is an idiopathic paradigm of human bone marrow failure syndrome, which involves active destruction of hematopoietic stem cells and progenitors by cytotoxic T cells in the bone marrow. Aberrant expression of microRNAs in T cells has been shown to lead to development of certain autoimmune diseases. In the present study, we performed a microarray analysis of miRNA expression in bone marrow CD3+ T cells from patients with aplastic anemia and healthy controls. Overexpression of miR34a and underexpression of its target gene diacylglycerol kinase (DGK) ζ in bone marrow mononuclear cells were validated in 41 patients and associated with the severity of aplastic anemia...
December 20, 2016: Oncotarget
https://www.readbyqxmd.com/read/28007665/cd25-blockade-delays-regulatory-t-cell-reconstitution-and-does-not-prevent-graft-versus-host-disease-after-allogeneic-hematopoietic-cell-transplantation
#10
Frederick L Locke, Joseph Pidala, Barry Storer, Paul J Martin, Michael A Pulsipher, Thomas R Chauncey, Niels Jacobsen, Nicolaus Kröger, Irwin Walker, Susan Light, Bronwen E Shaw, Francisca Beato, Ginna G Laport, Auayporn Nademanee, Armand Keating, Gerard Socie, Claudio Anasetti
Daclizumab, a humanized monoclonal antibody, binds CD25 and blocks formation of the IL-2 receptor on T cells. A study of daclizumab as acute graft-versus-host disease (GVHD) prophylaxis after unrelated bone marrow transplantation was conducted before the importance of CD25(+)FOXP3(+) regulatory T cells (Tregs) was recognized. Tregs can abrogate the onset of GVHD. The relation between Tregs and a graft-versus-malignancy effect is not fully understood. An international, multicenter, double-blind clinical trial randomized 210 adult or pediatric patients to receive 5 weekly doses of daclizumab at 0...
December 19, 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27995889/-ebv-related-post-transplantation-lymphoproliferative-diseases-in-patients-with-severe-aplastic-anemia-after-allogeneic-hematopoietic-stem-cell-transplantation-five-cases-report-and-literatures-review
#11
J Zhou, L J Han, Y L Zu, Yanli Zhang, F K Yu, Y W Fu, Q Wang, L J Liang, H Zhou, B J Fang, X D Wei, Y P Song
No abstract text is available yet for this article.
November 14, 2016: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/27941773/haploidentical-transplantation-for-pediatric-patients-with-acquired-severe-aplastic-anemia
#12
L P Xu, X H Zhang, F R Wang, X D Mo, T T Han, W Han, Y H Chen, Y Y Zhang, J Z Wang, C H Yan, Y Q Sun, S N Zuo, X J Huang
Techniques for haploidentical hematopoietic stem cell transplantation (haplo-HSCT) to treat severe aplastic anemia (SAA) have recently improved, but no protocol has been evaluated in a large number of pediatric patients. Fifty-two children with SAA received haplo-HSCT in our center. The treatment protocol used G-CSF-primed bone marrow with G-CSF-mobilized PBSCs without in vitro T-cell depletion. The conditioning regimen included busulfan/cyclophosphamide and antithymocyte globulin. Fifty-one patients achieved primary engraftment; one child died of regimen-related toxicity on the day +1...
December 12, 2016: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27933374/polymorphisms-of-the-tgf-%C3%AE-1-gene-and-the-risk-of-acquired-aplastic-anemia-in-a-chinese-population
#13
Xue-Hong Liang, Liucheng Rong, Guangsheng He, Hailong He, Shengyun Lin, Yan Yang, Yao Xue, Yongjun Fang
Acquired aplastic anemia (AA) is a hematological disease characterized by failure of bone marrow hematopoiesis resulting in pancytopenia. While immune-mediated destruction of hematopoietic stem/progenitor cells (HSPCs) plays a central role in the pathophysiology of acquired AA, the transforming growth factor-β1 (TGF-β1) is crucial in adjusting the immune system. The aim of our study was to investigate the role of TGF-β1 gene polymorphisms rs1800469 and rs2317130 in susceptibility to acquired AA. Via the approach of SNaPshot, we genotyped rs1800469 and rs2317130 in 101 patients with acquired AA and 165 controls...
December 9, 2016: Annals of Hematology
https://www.readbyqxmd.com/read/27923195/aplastic-anemia-and-mds-international-foundation-aamdsif-bone-marrow-failure-disease-scientific-symposium-2016
#14
Amer M Zeidan, Minoo Battiwalla, Deborah Berlyne, Thomas Winkler
Patients with acquired and inherited bone marrow failure syndromes (BMFS) have ineffective hematopoiesis due to impairments of the hematopoietic stem cell compartment. Common manifestations of BMFS include varying degrees of peripheral blood cytopenias and, sometimes, progression to acute myelogenous leukemia. Research efforts have been made all over the world to improve understanding of the pathogenesis of these diseases and their clinical implications. The Aplastic Anemia and MDS International Foundation (AAMDSIF) is an independent nonprofit organization whose mission is to help patients and family members cope with BMFS...
November 24, 2016: Leukemia Research
https://www.readbyqxmd.com/read/27916046/-a-comparative-study-of-unrelated-donor-and-matched-sibling-donor-allogeneic-hematopoietic-stem-cell-transplantation-in-children-and-adolescents-with-acquired-severe-aplastic-anemia
#15
J Zhou, Y W Fu, L J Liang, Q Wang, L J Han, Y L Zu, Yanli Zhang, X H Zhu, F K Yu, B J Fang, X D Wei, Y P Song
Objective: To evaluate the efficacy of unrelated donor allogeneic hematopoietic stem cell transplantation(URD allo-HSCT) for children and adolescents with severe aplastic anemia (SAA). Methods: Clinical data of 34 SAA children and adolescents undergoing allo-HSCT were retrospectively analyzed from October 2001 to October 2015. According to the source of donor, the patients were divided into matched sibling donor allo-HSCT group (MSD group) and unrelated donor group (URD group). The clinical outcome of SAA children and adolescents receiving URD allo-HSCT was assessed, and patients in MSD allo-HSCT group were enrolled as control at the same period...
December 1, 2016: Zhonghua Nei Ke za Zhi [Chinese Journal of Internal Medicine]
https://www.readbyqxmd.com/read/27913467/transplantation-for-bone-marrow-failure-current-issues
#16
Régis Peffault de Latour
The preferred treatment of idiopathic aplastic anemia (AA) is allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA)-identical sibling donor. Transplantation from a well-matched unrelated donor (MUD) may be considered for patients without a sibling donor after failure of immunosuppressive therapy, as may alternative transplantation (mismatched, cord blood or haplo-identical HSCT) for patients without a MUD. HSCT may also be contemplated for congenital disorders in cases of pancytopenia or severe isolated cytopenia...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27885510/left-main-crossover-stenting-in-a-patient-with-severe-thrombocytopenia-due-to-aplastic-anemia
#17
Marie Nishikawa, Jun Shiraishi, Muneo Ohshiro, Masaki Yashige, Masayuki Hyogo, Takahisa Sawada
A 76-year-old man with aplastic anemia presented with recurrent acute myocardial infarction (AMI) with heart failure. After the initial appearance of AMI approximately 2 months earlier, he had received conservative treatment/transfusion alone because of severe thrombocytopenia and anemia (platelet 11 × 10(3)/μL, hemoglobin 6.4 g/dL). Refractory heart failure persisted despite repeated conservative treatment/transfusion for the second AMI, and therefore, we performed transradial coronary angiography and left main crossover stenting with a bare metal stent...
November 24, 2016: Cardiovascular Intervention and Therapeutics
https://www.readbyqxmd.com/read/27837250/development-of-a-disease-specific-quality-of-life-questionnaire-for-patients-with-aplastic-anemia-and-or-paroxysmal-nocturnal-hemoglobinuria-qlq-aa-pnh-report-on-phases-i-and-ii
#18
Martha Groth, Susanne Singer, Cathrin Niedeggen, Andrea Petermann-Meyer, Alexander Röth, Hubert Schrezenmeier, Britta Höchsmann, Tim H Brümmendorf, Jens Panse
Acquired aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated ultra-rare diseases. Quality of life (QoL) evaluation tools used in studies for AA and PNH are unspecific and designed for cancer patients (e.g., the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, EORTC QLQ-C30). Given the complexity of AA and PNH, variation in symptoms and treatments, younger age of many patients, and the fact that AA and PNH are not classified as malignant diseases, it is likely that cancer-specific questionnaires are inappropriate...
February 2017: Annals of Hematology
https://www.readbyqxmd.com/read/27816648/mixed-t-cell-chimerism-after-allogeneic-hematopoietic-stem-cell-transplantation-for-severe-aplastic-anemia-using-an-alemtuzumab-containing-regimen-is-shaped-by-persistence-of-recipient-cd8-t-cells
#19
Francesco Grimaldi, Victoria Potter, Pilar Perez-Abellan, John P Veluchamy, Muhammad Atif, Rosemary Grain, Monica Sen, Steven Best, Nicholas Lea, Carmel Rice, Antonio Pagliuca, Ghulam J Mufti, Judith C W Marsh, Linda D Barber
Prevention of graft-versus-host disease (GVHD) is paramount for allogeneic hematopoietic stem cell transplantation (HSCT) to treat nonmalignant diseases. We previously reported that allogeneic HSCT for severe aplastic anemia (SAA) using the fludarabine, cyclophosphamide, and alemtuzumab (Campath-1H) (FCC) regimen is associated with a very low risk of GVHD and excellent clinical outcomes. We now report a single-center study of 45 patients with longer follow-up and investigation of lymphocyte recovery. Overall survival (OS) was 93%, and event-free survival (EFS) was 90...
February 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27811186/camitta-bm-thomas-ed-nathan-dg-et-al-severe-aplastic-anemia-a-prospective-study-of-the-effect-of-early-marrow-transplantation-on-acute-mortality-blood-1976-48-1-63-70
#20
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